Artificial intelligence (AI) is a loosely defined term that refers to machines (ie, algorithms) simulating facets of human intelligence. Some examples of AI are seen in natural language-processing algorithms, including autocorrect and search engine autocomplete functions; voice recognition in virtual assistants; autopilot systems in airplanes and self-driving cars; and computer vision in image and object recognition. Since the dawn of the century, various forms of AI have been tested and introduced in health care. However, a gap exists between clinician viewpoints on AI and the engineering world’s assumptions of what can be automated in medicine.

In this article, we review the history and evolution of AI in medicine, focusing on radiology and dermatology; current capabilities of AI; challenges to clinical integration; and future directions. Our aim is to provide realistic expectations of current technologies in solving complex problems and to empower dermatologists in planning for a future that likely includes various forms of AI.

Early Stages of AI in Medical Decision-making

Some of the earliest forms of clinical decision-support software in medicine were computer-aided detection and computer-aided diagnosis (CAD) used in screening for breast and lung cancer on mammography and computed tomography.^1-3 Early research on the use of CAD systems in radiology date to the 1960s (Figure), with the first US Food and Drug Administration–approved CAD system in mammography in 1998 and for Centers for Medicare & Medicaid Services reimbursement in 2002.^1,2

Early CAD systems relied on rule-based classifiers, which use predefined features to classify images into desired categories. For example, to classify an image as a high-risk or benign mass, features such as contour and texture had to be explicitly defined. Although these systems showed on par with, or higher, accuracy vs a radiologist in validation studies, early CAD systems never achieved wide adoption because of an increased rate of false positives as well as added work burden on a radiologist, who had to silence overcalling by the software.^1,2,4,5

Computer-aided diagnosis–based melanoma diagnosis was introduced in early 2000 in dermatology (Figure) using the same feature-based classifiers. These systems claimed expert-level accuracy in proof-of-concept studies and prospective uncontrolled trials on proprietary devices using these classifiers.^6,7 Similar to radiology, however, real-world adoption did not happen; in fact, the last of these devices was taken off the market in 2017. A recent meta-analysis of studies using CAD-based melanoma diagnosis point to study bias; data overfitting; and lack of large controlled, prospective trials as possible reasons why results could not be replicated in a clinical setting.⁸

Beyond 2010: Deep Learning

New techniques in machine learning (ML), called deep learning, began to emerge after 2010 (Figure). In deep learning, instead of directing the computer to look for certain discriminative features, the machine learns those features from the large amount of data without being explicitly programed to do so. In other words, compared to predecessor forms of computing, there is less human supervision in the learning process (Table). The concept of ML has existed since the 1980s. The field saw exponential growth in the last decade with the improvement of algorithms; an increase in computing power; and emergence of large training data sets, such as open-source platforms on the Web.^9,10

Most ML methods today incorporate artificial neural networks (ANN), computer programs that imitate the architecture of biological neural networks and form dynamically changing systems that improve with continuous data exposure. The performance of an ANN is dependent on the number and architecture of its neural layers and (similar to CAD systems) the size, quality, and generalizability of the training data set.^9-12

In medicine, images (eg, clinical or dermoscopic images and imaging scans) are the most commonly used form of data for AI development. Convolutional neural networks (CNN), a subtype of ANN, are frequently used for this purpose. These networks use a hierarchical neural network architecture, similar to the visual cortex, that allows for composition of complex features (eg, shapes) from simpler features (eg, image intensities), which leads to more efficient data processing.^10-12

In recent years, CNNs have been applied in a number of image-based medical fields, including radiology, dermatology, and pathology. Initially, studies were largely led by computer scientists trying to match clinician performance in detection of disease categories. However, there has been a shift toward more physicians getting involved, which has motivated development of large curated (ie, expert-labeled) and standardized clinical data sets in training the CNN. Although training on quality-controlled data is a work in progress across medical disciplines, it has led to improved machine performance.^11,12

Recent Advances in AI

In recent years, the number of studies covering CNN in diagnosis has increased exponentially in several medical specialties. The goal is to improve software to close the gap between experts and the machine in live clinical settings. The current literature focuses on a comparison of experts with the machine in simulated settings; prospective clinical trials are still lagging in the real world.^9,11,13

We look at radiology to explore recent advances in AI diagnosis for 3 reasons: (1) radiology has the largest repository of digital data (using a picture archiving and communication system) among medical specialties; (2) radiology has well-defined, image-acquisition protocols in its clinical workflow¹⁴; and (3) gray-scale images are easier to standardize because they are impervious to environmental variables that are difficult to control (eg, recent sun exposure, rosacea flare, lighting, sweating). These are some of the reasons we think radiology is, and will be, ahead in training AI algorithms and integrating them into clinical practice. However, even radiology AI studies have limitations, including a lack of prospective, real-world clinical setting, generalizable studies, and a lack of large standardized available databases for training algorithms.

Narrowing our discussion to studies of mammography—given the repetitive nature and binary output of this modality, which has made it one of the first targets of automation in diagnostic imaging^1,2,5,13—AI-based CAD in mammography, much like its predecessor feature-based CAD, has shown promising results in artificial settings. Five key mammography CNN studies have reported a wide range of diagnostic accuracy (area under the curve, 69.2 to 97.8 [mean, 88.2]) compared to radiologists.^15-19

In the most recent study (2019), Rodriguez-Ruiz et al¹⁵ compared machines and a cohort of 101 radiologists, in which AI showed performance comparability. However, results in this artificial setting were not followed up with prospective analysis of the technology in a clinical setting. First-generation, feature-based CADs in mammography also showed expert-level performance in artificial settings, but the technology became extinct because these results were not generalizable to real-world in prospective trials. To our knowledge, a limitation of radiology AI is that all current CNNs have not yet been tested in a live clinical setting.^13-19

The second limitation of radiology AI is lack of standardization, which also applies to mammography, despite this subset having the largest and oldest publicly available data set. In a recent review of 23 studies on AI-based algorithms in mammography (2010-2019), clinicians point to one of the biggest flaws: the use of small, nonstandardized, and skewed public databases (often enriched for malignancy) as training algorithms.¹³

Standardization refers to quality-control measures in acquisition, processing, and image labeling that need to be met for images to be included in the training data set. At present, large stores of radiologic data that are standardized within each institution are not publicly accessible through a unified reference platform. Lack of large standardized training data sets leads to selection bias and increases the risk for overfitting, which occurs when algorithm models incorporate background noise in the data into its prediction scheme. Overfitting has been noted in several AI-based studies in mammography,¹³ which limits the generalizability of algorithm performance in the real-world setting.

To overcome this limitation, the American College of Radiology Data Science Institute recently took the lead on creating a reference platform for quality control and standardized data generation for AI integration in radiology. The goal of the institute is for radiologists to work collaboratively with industry to ensure that algorithms are trained on quality data that produces clinically useable output for the clinician and patient.^11,20

Similar to initial radiology studies utilizing AI mainly as a screening tool, AI-driven studies in dermatology are focused on classification of melanocytic lesions; the goal is to aid in melanoma screening. Two of the most-recent, most-cited articles on this topic are by Esteva et al²¹ and Tschandl et al.²² Esteva et al²¹ matched the performance of 21 dermatologists in binary classification (malignant or nonmalignant) of clinical and dermoscopic images in pigmented and nonpigmented categories. A CNN developed by Google was trained on 130,000 clinical images encompassing more than 2000 dermatologist-labeled diagnoses from 18 sites. Despite promising results, the question remains whether these findings are transferrable to the clinical setting. In addition to the limitation on generalizability, the authors do not elaborate on standardization of training image data sets. For example, it is unclear what percentage of the training data set’s image labels were based on biopsy results vs clinical diagnosis.²¹

The second study was the largest Web-based study to compare the performance of more than 500 dermatologists worldwide.²² The top 3–performing algorithms (among a pool of 139) were at least as good as the performance of 27 expert dermatologists (defined as having more than 10 years’ experience) in the classification of pigmented lesions into 7 predefined categories.²² However, images came from nonstandardized sources gathered from a 20-year period at one European academic center and a private practice in Australia. Tschandl et al²² looked at external validation with an independent data set, outside the training data set. Although not generalizable to a real-world setting, looking at external data sets helps correct for overfitting and is a good first step in understanding transferability of results. However, the external data set was chosen by the authors and therefore might be tainted by selection bias. Although only a 10% drop in algorithmic accuracy was noted using the external data set chosen by the authors, this drop does not apply to other data sets or more importantly to a real-world setting.²²

Current limitations and future goals of radiology also will most likely apply to dermatology AI research. In medicine and radiology, the goal of AI is to first help users by prioritizing what they should focus on. The concept of comparing AI to a radiologist or dermatologist is potentially shortsighted. Shortcomings of the current supervised or semisupervised algorithms used in medicine underscore the points that, first, to make their outputs clinically usable, it should be clinicians who procure and standardize training data sets and, second, it appears logical that the performance of these category of algorithms requires constant monitoring for bias. Therefore, these algorithms cannot operate as stand-alone diagnostic machines but as an aid to the clinician—if the performance of the algorithms is proved in large trials.

Near-Future Directions and Projections

Almost all recent state-of-the-art AI systems tested in medical disciplines fall under the engineering terminology of narrow or weak AI, meaning any given algorithm is trained to do only one specific task.⁹ An example of a task is classification of images into multiple categories (ie, benign or malignant). However, task classification only works with preselected images that will need substantial improvements in standardization.

Although it has been demonstrated that AI systems can excel at one task at a time, such as classification, better than a human cohort in simulated settings, these literal machines lack the ability to incorporate context; integrate various forms of sensory input such as visual, voice, or text; or make associations the way humans do.⁹ Multiple tasks and clinical context integration are required for predictive diagnosis or clinical decision-making, even in a simulated environment. In this sense, CNN is still similar to its antiquated linear CAD predecessor: It cannot make a diagnosis or a clinical decision but might be appropriate for triaging cases that are referred for evaluation by a dermatologist.

Medical AI also may use electronic health records or patient-gathered data (eg, apps). However, clinical images are more structured and less noisy and are more easily incorporated in AI training. Therefore, as we are already witnessing, earlier validation and adoption of AI will occur in image-based disciplines, beginning with radiology; then pathology; and eventually dermatology, which will be the most challenging of the 3 medical specialties to standardize.

Final Thoughts

Artificial intelligence in health care is in its infancy; specific task-driven algorithms are only beginning to be introduced. We project that in the next 5 to 10 years, clinicians will become increasingly involved in training and testing large-scale validation as well as monitoring narrow AI in clinical trials. Radiology has served as the pioneering area in medicine and is just beginning to utilize narrow AI to help specialists with very specific tasks. For example, a task would be to triage which scans to look at first for a radiologist or which pigmented lesion might need prompt evaluation by a dermatologist. Artificial intelligence in medicine is not replacing specialists or placing decision-making in the hands of a nonexpert. At this point, CNNs have not proven that they make us better at diagnosing because real-world clinical data are lacking, which may change in the future with large standardized training data sets and validation with prospective clinical trials. The near future for dermatology and pathology will follow what is already happening in radiology, with AI substantially increasing workflow efficiency by prioritizing tasks.

Artificial intelligence (AI) is a loosely defined term that refers to machines (ie, algorithms) simulating facets of human intelligence. Some examples of AI are seen in natural language-processing algorithms, including autocorrect and search engine autocomplete functions; voice recognition in virtual assistants; autopilot systems in airplanes and self-driving cars; and computer vision in image and object recognition. Since the dawn of the century, various forms of AI have been tested and introduced in health care. However, a gap exists between clinician viewpoints on AI and the engineering world’s assumptions of what can be automated in medicine.

In this article, we review the history and evolution of AI in medicine, focusing on radiology and dermatology; current capabilities of AI; challenges to clinical integration; and future directions. Our aim is to provide realistic expectations of current technologies in solving complex problems and to empower dermatologists in planning for a future that likely includes various forms of AI.

Early Stages of AI in Medical Decision-making

Some of the earliest forms of clinical decision-support software in medicine were computer-aided detection and computer-aided diagnosis (CAD) used in screening for breast and lung cancer on mammography and computed tomography.^1-3 Early research on the use of CAD systems in radiology date to the 1960s (Figure), with the first US Food and Drug Administration–approved CAD system in mammography in 1998 and for Centers for Medicare & Medicaid Services reimbursement in 2002.^1,2

Early CAD systems relied on rule-based classifiers, which use predefined features to classify images into desired categories. For example, to classify an image as a high-risk or benign mass, features such as contour and texture had to be explicitly defined. Although these systems showed on par with, or higher, accuracy vs a radiologist in validation studies, early CAD systems never achieved wide adoption because of an increased rate of false positives as well as added work burden on a radiologist, who had to silence overcalling by the software.^1,2,4,5

Computer-aided diagnosis–based melanoma diagnosis was introduced in early 2000 in dermatology (Figure) using the same feature-based classifiers. These systems claimed expert-level accuracy in proof-of-concept studies and prospective uncontrolled trials on proprietary devices using these classifiers.^6,7 Similar to radiology, however, real-world adoption did not happen; in fact, the last of these devices was taken off the market in 2017. A recent meta-analysis of studies using CAD-based melanoma diagnosis point to study bias; data overfitting; and lack of large controlled, prospective trials as possible reasons why results could not be replicated in a clinical setting.⁸

Beyond 2010: Deep Learning

New techniques in machine learning (ML), called deep learning, began to emerge after 2010 (Figure). In deep learning, instead of directing the computer to look for certain discriminative features, the machine learns those features from the large amount of data without being explicitly programed to do so. In other words, compared to predecessor forms of computing, there is less human supervision in the learning process (Table). The concept of ML has existed since the 1980s. The field saw exponential growth in the last decade with the improvement of algorithms; an increase in computing power; and emergence of large training data sets, such as open-source platforms on the Web.^9,10

Most ML methods today incorporate artificial neural networks (ANN), computer programs that imitate the architecture of biological neural networks and form dynamically changing systems that improve with continuous data exposure. The performance of an ANN is dependent on the number and architecture of its neural layers and (similar to CAD systems) the size, quality, and generalizability of the training data set.^9-12

In medicine, images (eg, clinical or dermoscopic images and imaging scans) are the most commonly used form of data for AI development. Convolutional neural networks (CNN), a subtype of ANN, are frequently used for this purpose. These networks use a hierarchical neural network architecture, similar to the visual cortex, that allows for composition of complex features (eg, shapes) from simpler features (eg, image intensities), which leads to more efficient data processing.^10-12

In recent years, CNNs have been applied in a number of image-based medical fields, including radiology, dermatology, and pathology. Initially, studies were largely led by computer scientists trying to match clinician performance in detection of disease categories. However, there has been a shift toward more physicians getting involved, which has motivated development of large curated (ie, expert-labeled) and standardized clinical data sets in training the CNN. Although training on quality-controlled data is a work in progress across medical disciplines, it has led to improved machine performance.^11,12

Recent Advances in AI

In recent years, the number of studies covering CNN in diagnosis has increased exponentially in several medical specialties. The goal is to improve software to close the gap between experts and the machine in live clinical settings. The current literature focuses on a comparison of experts with the machine in simulated settings; prospective clinical trials are still lagging in the real world.^9,11,13

We look at radiology to explore recent advances in AI diagnosis for 3 reasons: (1) radiology has the largest repository of digital data (using a picture archiving and communication system) among medical specialties; (2) radiology has well-defined, image-acquisition protocols in its clinical workflow¹⁴; and (3) gray-scale images are easier to standardize because they are impervious to environmental variables that are difficult to control (eg, recent sun exposure, rosacea flare, lighting, sweating). These are some of the reasons we think radiology is, and will be, ahead in training AI algorithms and integrating them into clinical practice. However, even radiology AI studies have limitations, including a lack of prospective, real-world clinical setting, generalizable studies, and a lack of large standardized available databases for training algorithms.

Narrowing our discussion to studies of mammography—given the repetitive nature and binary output of this modality, which has made it one of the first targets of automation in diagnostic imaging^1,2,5,13—AI-based CAD in mammography, much like its predecessor feature-based CAD, has shown promising results in artificial settings. Five key mammography CNN studies have reported a wide range of diagnostic accuracy (area under the curve, 69.2 to 97.8 [mean, 88.2]) compared to radiologists.^15-19

In the most recent study (2019), Rodriguez-Ruiz et al¹⁵ compared machines and a cohort of 101 radiologists, in which AI showed performance comparability. However, results in this artificial setting were not followed up with prospective analysis of the technology in a clinical setting. First-generation, feature-based CADs in mammography also showed expert-level performance in artificial settings, but the technology became extinct because these results were not generalizable to real-world in prospective trials. To our knowledge, a limitation of radiology AI is that all current CNNs have not yet been tested in a live clinical setting.^13-19

The second limitation of radiology AI is lack of standardization, which also applies to mammography, despite this subset having the largest and oldest publicly available data set. In a recent review of 23 studies on AI-based algorithms in mammography (2010-2019), clinicians point to one of the biggest flaws: the use of small, nonstandardized, and skewed public databases (often enriched for malignancy) as training algorithms.¹³

Standardization refers to quality-control measures in acquisition, processing, and image labeling that need to be met for images to be included in the training data set. At present, large stores of radiologic data that are standardized within each institution are not publicly accessible through a unified reference platform. Lack of large standardized training data sets leads to selection bias and increases the risk for overfitting, which occurs when algorithm models incorporate background noise in the data into its prediction scheme. Overfitting has been noted in several AI-based studies in mammography,¹³ which limits the generalizability of algorithm performance in the real-world setting.

To overcome this limitation, the American College of Radiology Data Science Institute recently took the lead on creating a reference platform for quality control and standardized data generation for AI integration in radiology. The goal of the institute is for radiologists to work collaboratively with industry to ensure that algorithms are trained on quality data that produces clinically useable output for the clinician and patient.^11,20

Similar to initial radiology studies utilizing AI mainly as a screening tool, AI-driven studies in dermatology are focused on classification of melanocytic lesions; the goal is to aid in melanoma screening. Two of the most-recent, most-cited articles on this topic are by Esteva et al²¹ and Tschandl et al.²² Esteva et al²¹ matched the performance of 21 dermatologists in binary classification (malignant or nonmalignant) of clinical and dermoscopic images in pigmented and nonpigmented categories. A CNN developed by Google was trained on 130,000 clinical images encompassing more than 2000 dermatologist-labeled diagnoses from 18 sites. Despite promising results, the question remains whether these findings are transferrable to the clinical setting. In addition to the limitation on generalizability, the authors do not elaborate on standardization of training image data sets. For example, it is unclear what percentage of the training data set’s image labels were based on biopsy results vs clinical diagnosis.²¹

The second study was the largest Web-based study to compare the performance of more than 500 dermatologists worldwide.²² The top 3–performing algorithms (among a pool of 139) were at least as good as the performance of 27 expert dermatologists (defined as having more than 10 years’ experience) in the classification of pigmented lesions into 7 predefined categories.²² However, images came from nonstandardized sources gathered from a 20-year period at one European academic center and a private practice in Australia. Tschandl et al²² looked at external validation with an independent data set, outside the training data set. Although not generalizable to a real-world setting, looking at external data sets helps correct for overfitting and is a good first step in understanding transferability of results. However, the external data set was chosen by the authors and therefore might be tainted by selection bias. Although only a 10% drop in algorithmic accuracy was noted using the external data set chosen by the authors, this drop does not apply to other data sets or more importantly to a real-world setting.²²

Current limitations and future goals of radiology also will most likely apply to dermatology AI research. In medicine and radiology, the goal of AI is to first help users by prioritizing what they should focus on. The concept of comparing AI to a radiologist or dermatologist is potentially shortsighted. Shortcomings of the current supervised or semisupervised algorithms used in medicine underscore the points that, first, to make their outputs clinically usable, it should be clinicians who procure and standardize training data sets and, second, it appears logical that the performance of these category of algorithms requires constant monitoring for bias. Therefore, these algorithms cannot operate as stand-alone diagnostic machines but as an aid to the clinician—if the performance of the algorithms is proved in large trials.

Near-Future Directions and Projections

Almost all recent state-of-the-art AI systems tested in medical disciplines fall under the engineering terminology of narrow or weak AI, meaning any given algorithm is trained to do only one specific task.⁹ An example of a task is classification of images into multiple categories (ie, benign or malignant). However, task classification only works with preselected images that will need substantial improvements in standardization.

Although it has been demonstrated that AI systems can excel at one task at a time, such as classification, better than a human cohort in simulated settings, these literal machines lack the ability to incorporate context; integrate various forms of sensory input such as visual, voice, or text; or make associations the way humans do.⁹ Multiple tasks and clinical context integration are required for predictive diagnosis or clinical decision-making, even in a simulated environment. In this sense, CNN is still similar to its antiquated linear CAD predecessor: It cannot make a diagnosis or a clinical decision but might be appropriate for triaging cases that are referred for evaluation by a dermatologist.

Medical AI also may use electronic health records or patient-gathered data (eg, apps). However, clinical images are more structured and less noisy and are more easily incorporated in AI training. Therefore, as we are already witnessing, earlier validation and adoption of AI will occur in image-based disciplines, beginning with radiology; then pathology; and eventually dermatology, which will be the most challenging of the 3 medical specialties to standardize.

Final Thoughts

Artificial intelligence in health care is in its infancy; specific task-driven algorithms are only beginning to be introduced. We project that in the next 5 to 10 years, clinicians will become increasingly involved in training and testing large-scale validation as well as monitoring narrow AI in clinical trials. Radiology has served as the pioneering area in medicine and is just beginning to utilize narrow AI to help specialists with very specific tasks. For example, a task would be to triage which scans to look at first for a radiologist or which pigmented lesion might need prompt evaluation by a dermatologist. Artificial intelligence in medicine is not replacing specialists or placing decision-making in the hands of a nonexpert. At this point, CNNs have not proven that they make us better at diagnosing because real-world clinical data are lacking, which may change in the future with large standardized training data sets and validation with prospective clinical trials. The near future for dermatology and pathology will follow what is already happening in radiology, with AI substantially increasing workflow efficiency by prioritizing tasks.

Artificial intelligence (AI) is a loosely defined term that refers to machines (ie, algorithms) simulating facets of human intelligence. Some examples of AI are seen in natural language-processing algorithms, including autocorrect and search engine autocomplete functions; voice recognition in virtual assistants; autopilot systems in airplanes and self-driving cars; and computer vision in image and object recognition. Since the dawn of the century, various forms of AI have been tested and introduced in health care. However, a gap exists between clinician viewpoints on AI and the engineering world’s assumptions of what can be automated in medicine.

In this article, we review the history and evolution of AI in medicine, focusing on radiology and dermatology; current capabilities of AI; challenges to clinical integration; and future directions. Our aim is to provide realistic expectations of current technologies in solving complex problems and to empower dermatologists in planning for a future that likely includes various forms of AI.

Early Stages of AI in Medical Decision-making

Some of the earliest forms of clinical decision-support software in medicine were computer-aided detection and computer-aided diagnosis (CAD) used in screening for breast and lung cancer on mammography and computed tomography.^1-3 Early research on the use of CAD systems in radiology date to the 1960s (Figure), with the first US Food and Drug Administration–approved CAD system in mammography in 1998 and for Centers for Medicare & Medicaid Services reimbursement in 2002.^1,2

Early CAD systems relied on rule-based classifiers, which use predefined features to classify images into desired categories. For example, to classify an image as a high-risk or benign mass, features such as contour and texture had to be explicitly defined. Although these systems showed on par with, or higher, accuracy vs a radiologist in validation studies, early CAD systems never achieved wide adoption because of an increased rate of false positives as well as added work burden on a radiologist, who had to silence overcalling by the software.^1,2,4,5

Computer-aided diagnosis–based melanoma diagnosis was introduced in early 2000 in dermatology (Figure) using the same feature-based classifiers. These systems claimed expert-level accuracy in proof-of-concept studies and prospective uncontrolled trials on proprietary devices using these classifiers.^6,7 Similar to radiology, however, real-world adoption did not happen; in fact, the last of these devices was taken off the market in 2017. A recent meta-analysis of studies using CAD-based melanoma diagnosis point to study bias; data overfitting; and lack of large controlled, prospective trials as possible reasons why results could not be replicated in a clinical setting.⁸

Beyond 2010: Deep Learning

New techniques in machine learning (ML), called deep learning, began to emerge after 2010 (Figure). In deep learning, instead of directing the computer to look for certain discriminative features, the machine learns those features from the large amount of data without being explicitly programed to do so. In other words, compared to predecessor forms of computing, there is less human supervision in the learning process (Table). The concept of ML has existed since the 1980s. The field saw exponential growth in the last decade with the improvement of algorithms; an increase in computing power; and emergence of large training data sets, such as open-source platforms on the Web.^9,10

Most ML methods today incorporate artificial neural networks (ANN), computer programs that imitate the architecture of biological neural networks and form dynamically changing systems that improve with continuous data exposure. The performance of an ANN is dependent on the number and architecture of its neural layers and (similar to CAD systems) the size, quality, and generalizability of the training data set.^9-12

In medicine, images (eg, clinical or dermoscopic images and imaging scans) are the most commonly used form of data for AI development. Convolutional neural networks (CNN), a subtype of ANN, are frequently used for this purpose. These networks use a hierarchical neural network architecture, similar to the visual cortex, that allows for composition of complex features (eg, shapes) from simpler features (eg, image intensities), which leads to more efficient data processing.^10-12

In recent years, CNNs have been applied in a number of image-based medical fields, including radiology, dermatology, and pathology. Initially, studies were largely led by computer scientists trying to match clinician performance in detection of disease categories. However, there has been a shift toward more physicians getting involved, which has motivated development of large curated (ie, expert-labeled) and standardized clinical data sets in training the CNN. Although training on quality-controlled data is a work in progress across medical disciplines, it has led to improved machine performance.^11,12

Recent Advances in AI

In recent years, the number of studies covering CNN in diagnosis has increased exponentially in several medical specialties. The goal is to improve software to close the gap between experts and the machine in live clinical settings. The current literature focuses on a comparison of experts with the machine in simulated settings; prospective clinical trials are still lagging in the real world.^9,11,13

We look at radiology to explore recent advances in AI diagnosis for 3 reasons: (1) radiology has the largest repository of digital data (using a picture archiving and communication system) among medical specialties; (2) radiology has well-defined, image-acquisition protocols in its clinical workflow¹⁴; and (3) gray-scale images are easier to standardize because they are impervious to environmental variables that are difficult to control (eg, recent sun exposure, rosacea flare, lighting, sweating). These are some of the reasons we think radiology is, and will be, ahead in training AI algorithms and integrating them into clinical practice. However, even radiology AI studies have limitations, including a lack of prospective, real-world clinical setting, generalizable studies, and a lack of large standardized available databases for training algorithms.

Narrowing our discussion to studies of mammography—given the repetitive nature and binary output of this modality, which has made it one of the first targets of automation in diagnostic imaging^1,2,5,13—AI-based CAD in mammography, much like its predecessor feature-based CAD, has shown promising results in artificial settings. Five key mammography CNN studies have reported a wide range of diagnostic accuracy (area under the curve, 69.2 to 97.8 [mean, 88.2]) compared to radiologists.^15-19

In the most recent study (2019), Rodriguez-Ruiz et al¹⁵ compared machines and a cohort of 101 radiologists, in which AI showed performance comparability. However, results in this artificial setting were not followed up with prospective analysis of the technology in a clinical setting. First-generation, feature-based CADs in mammography also showed expert-level performance in artificial settings, but the technology became extinct because these results were not generalizable to real-world in prospective trials. To our knowledge, a limitation of radiology AI is that all current CNNs have not yet been tested in a live clinical setting.^13-19

The second limitation of radiology AI is lack of standardization, which also applies to mammography, despite this subset having the largest and oldest publicly available data set. In a recent review of 23 studies on AI-based algorithms in mammography (2010-2019), clinicians point to one of the biggest flaws: the use of small, nonstandardized, and skewed public databases (often enriched for malignancy) as training algorithms.¹³

Standardization refers to quality-control measures in acquisition, processing, and image labeling that need to be met for images to be included in the training data set. At present, large stores of radiologic data that are standardized within each institution are not publicly accessible through a unified reference platform. Lack of large standardized training data sets leads to selection bias and increases the risk for overfitting, which occurs when algorithm models incorporate background noise in the data into its prediction scheme. Overfitting has been noted in several AI-based studies in mammography,¹³ which limits the generalizability of algorithm performance in the real-world setting.

To overcome this limitation, the American College of Radiology Data Science Institute recently took the lead on creating a reference platform for quality control and standardized data generation for AI integration in radiology. The goal of the institute is for radiologists to work collaboratively with industry to ensure that algorithms are trained on quality data that produces clinically useable output for the clinician and patient.^11,20

Similar to initial radiology studies utilizing AI mainly as a screening tool, AI-driven studies in dermatology are focused on classification of melanocytic lesions; the goal is to aid in melanoma screening. Two of the most-recent, most-cited articles on this topic are by Esteva et al²¹ and Tschandl et al.²² Esteva et al²¹ matched the performance of 21 dermatologists in binary classification (malignant or nonmalignant) of clinical and dermoscopic images in pigmented and nonpigmented categories. A CNN developed by Google was trained on 130,000 clinical images encompassing more than 2000 dermatologist-labeled diagnoses from 18 sites. Despite promising results, the question remains whether these findings are transferrable to the clinical setting. In addition to the limitation on generalizability, the authors do not elaborate on standardization of training image data sets. For example, it is unclear what percentage of the training data set’s image labels were based on biopsy results vs clinical diagnosis.²¹

The second study was the largest Web-based study to compare the performance of more than 500 dermatologists worldwide.²² The top 3–performing algorithms (among a pool of 139) were at least as good as the performance of 27 expert dermatologists (defined as having more than 10 years’ experience) in the classification of pigmented lesions into 7 predefined categories.²² However, images came from nonstandardized sources gathered from a 20-year period at one European academic center and a private practice in Australia. Tschandl et al²² looked at external validation with an independent data set, outside the training data set. Although not generalizable to a real-world setting, looking at external data sets helps correct for overfitting and is a good first step in understanding transferability of results. However, the external data set was chosen by the authors and therefore might be tainted by selection bias. Although only a 10% drop in algorithmic accuracy was noted using the external data set chosen by the authors, this drop does not apply to other data sets or more importantly to a real-world setting.²²

Current limitations and future goals of radiology also will most likely apply to dermatology AI research. In medicine and radiology, the goal of AI is to first help users by prioritizing what they should focus on. The concept of comparing AI to a radiologist or dermatologist is potentially shortsighted. Shortcomings of the current supervised or semisupervised algorithms used in medicine underscore the points that, first, to make their outputs clinically usable, it should be clinicians who procure and standardize training data sets and, second, it appears logical that the performance of these category of algorithms requires constant monitoring for bias. Therefore, these algorithms cannot operate as stand-alone diagnostic machines but as an aid to the clinician—if the performance of the algorithms is proved in large trials.

Near-Future Directions and Projections

Almost all recent state-of-the-art AI systems tested in medical disciplines fall under the engineering terminology of narrow or weak AI, meaning any given algorithm is trained to do only one specific task.⁹ An example of a task is classification of images into multiple categories (ie, benign or malignant). However, task classification only works with preselected images that will need substantial improvements in standardization.

Although it has been demonstrated that AI systems can excel at one task at a time, such as classification, better than a human cohort in simulated settings, these literal machines lack the ability to incorporate context; integrate various forms of sensory input such as visual, voice, or text; or make associations the way humans do.⁹ Multiple tasks and clinical context integration are required for predictive diagnosis or clinical decision-making, even in a simulated environment. In this sense, CNN is still similar to its antiquated linear CAD predecessor: It cannot make a diagnosis or a clinical decision but might be appropriate for triaging cases that are referred for evaluation by a dermatologist.

Medical AI also may use electronic health records or patient-gathered data (eg, apps). However, clinical images are more structured and less noisy and are more easily incorporated in AI training. Therefore, as we are already witnessing, earlier validation and adoption of AI will occur in image-based disciplines, beginning with radiology; then pathology; and eventually dermatology, which will be the most challenging of the 3 medical specialties to standardize.

Final Thoughts

Artificial intelligence in health care is in its infancy; specific task-driven algorithms are only beginning to be introduced. We project that in the next 5 to 10 years, clinicians will become increasingly involved in training and testing large-scale validation as well as monitoring narrow AI in clinical trials. Radiology has served as the pioneering area in medicine and is just beginning to utilize narrow AI to help specialists with very specific tasks. For example, a task would be to triage which scans to look at first for a radiologist or which pigmented lesion might need prompt evaluation by a dermatologist. Artificial intelligence in medicine is not replacing specialists or placing decision-making in the hands of a nonexpert. At this point, CNNs have not proven that they make us better at diagnosing because real-world clinical data are lacking, which may change in the future with large standardized training data sets and validation with prospective clinical trials. The near future for dermatology and pathology will follow what is already happening in radiology, with AI substantially increasing workflow efficiency by prioritizing tasks.

Hyperbaric oxygen therapy (HOT) is a treatment modality dating to 1861 in the United States.¹ Today, there are 14 indications² for HOT (Table), issued by the Undersea & Hyperbaric Medical Society, which also administers an accreditation program for facilities providing HOT.³ The 14 indications also are relevant because it is unlikely that HOT will be covered by insurance for unapproved indications.⁴

Although HOT is not commonly seen as a first-line intervention in dermatology, there are scenarios in which it can be used to good effect: compromised grafts and flaps; poorly healing ulceration related to vasculitis and autoimmune disorders; and possibly for vascular compromise, including cutaneous ischemia caused by fillers. We review its indications, dermatologic applications, and potential complications.

Overview of HOT

Hyperbaric oxygen therapy involves sitting or lying in a special chamber that allows for controlled levels of oxygen (O₂) at increased atmospheric pressure, which specifically involves breathing near 100% O₂ while inside a monoplace or multiplace chamber⁵ that is pressurized to greater than sea level pressure (≥1.4 atmosphere absolute).²

A monoplace chamber is designed to treat a single person (Figure 1); a multiplace chamber (Figure 2) accommodates as many as 5 to 25 patients.^5,6 The chambers also accommodate hospital beds and medical attendants, if needed. Hyperbaric O₂ is inhaled through a mask, a tight-fitting hood, or an endotracheal tube, depending on the patient’s status.⁷ Treatment ranges from only 1 or 2 iterations for acute conditions to 30 sessions or more for chronic conditions. Individual sessions last 45 minutes to 5 hours; 120 minutes is considered a safe maximum duration.⁷ A television often is provided to help the patient pass the time.⁸

Long-standing Use in Decompression Sickness

Hyperbaric oxygen therapy is best known for its effectiveness in treating decompression sickness (DCS) and carbon monoxide poisoning. Decompression sickness involves liberation of free gas from tissue, in the form of bubbles, when a person experiences a relative decrease in atmospheric pressure, which results in an imbalance in the sum of gas tensions in tissue compared to ambient pressure.

Decompression sickness has special military significance because it can affect divers and pilots, particularly those flying at high altitude. Over the course of 12 years, approximately 50 pilot trainees at an Air Force training site in Colorado required HOT when ground-level O₂ failed to resolve their DCS symptoms.¹⁰

Symptoms of DCS range from musculoskeletal pain to severe neurologic and pulmonary complications. First-line therapy for DCS is 100% O₂ at ground level. When symptoms are severe or persistent, HOT is the treatment of choice. It works by decreasing the volume of air bubbles (as predicted by Boyle’s Law), providing oxygenation to hypoxic tissue and mitigating inflammatory responses implicated in tissue injury⁹; HOT can be considered salvage treatment for rare, severe, or unresponsive complications of DCS during common activities such as diving and flying.

The emergent nature of DCS often necessitates an on-call, on-site HOT facility or contracted community services. Although DCS is a rare complication, it can be devastating, as was the case for a military pilot flying an ultrahigh altitude reconnaissance aircraft.¹¹ He developed a near fatal case of neurologic DCS during a military mission and required treatment with emergent HOT. Although his symptoms were reduced with therapy, he has persistent cognitive deficits.¹¹

Other Indications

Dermatologic Flaps and Grafts
Although less commonly discussed in dermatologic literature, the use of HOT in compromised grafts and flaps has been addressed in the plastic surgery literature. In a large multicenter study, researchers evaluated 20,821 Mohs micrographic surgery procedures and reported 149 adverse events, of which 20.1% were dehiscence and partial or full necrosis.¹² These complications, though rare, are potentially devastating, particularly in cosmetically sensitive locations such as the face. Traditional care for compromised grafts and flaps includes local wound care, surgical debridement, and additional reconstructive procedures. These interventions can be expensive and uncomfortable for patients and carry risk for further morbidity.¹³

Grafts become compromised when their metabolic demand outpaces the ability of the recipient bed due to characteristics of the graft or the recipient bed or both. Flaps carry their own blood supply, which can be compromised if the flap is too long or too large for the pedicle, there is notable tension on the wound, or blood flow is mechanically obstructed by kinking or twisting. Under these conditions, HOT can be beneficial, as O₂ dissolves in plasma, thus improving the O₂ tissue cellular diffusion gradient.⁷ An increased level of systemic O₂ promotes wound healing and graft or flap survival by improving fibroblast function, blood flow, and vascularity, and by mitigating ischemia-reperfusion injury.¹³

In a study, 105 patients with an ischemic flap or graft were treated with HOT; most (89% of threatened flaps and 91% of threatened grafts) were salvaged. In this series, the duration of latency from the creation of the flap to initiation of HOT was directly proportional to the failure rate of this treatment modality.¹⁴

Radiation-Induced Ulceration
Radionecrosis, a complication of radiotherapy, is caused by progressive obliterating endarteritis with resultant vascular stenosis and fibroatrophy, which eventually cause stromal fibrosis.¹⁵ In a study that looked at 1267 nonmelanoma skin cancers that had been treated with radiotherapy, the ulceration rate was 6.3%. Most of the ulcerated lesions were treatable conservatively, but some were more treatment resistant.¹⁶ Hampson et al¹⁷ reported on 58 patients with cutaneous wounds due to soft-tissue radionecrosis who were treated with HOT as part of a larger observational case series in which investigators looked at multiple types of radionecrosis. They found that 76% of these patients improved: 26% showed complete resolution and the remaining 50% had 50% to 90% improvement.¹⁷

Vasculitis or Autoimmune Ulceration
Vasculitis and vasculopathy can occur independent of, or in association with, connective tissue disease and can result in chronic ulceration. At our institution, a patient with antimelanoma differentiation-associated protein 5 dermatomyositis who had refractory digital ulcerations despite intensive systemic therapy had an excellent response to HOT; ulcerations resolved after 37 treatments.¹⁸

Efrati et al¹⁹ reported on 35 patients who had chronic nonhealing vasculitic ulcerations despite immunosuppression medication who were treated with HOT. Twenty-eight patients completely healed, 4 had partial healing, and 3 had no improvement.

Mirasoglu et al²⁰ reported on a case series of 6 systemic sclerosis patients who had ulcerations that persisted despite other treatments. After initiation of HOT, 4 patients experienced complete response and 2 experienced partial response, which is notable because such ulcerations are often extremely difficult to treat and have usually failed multiple therapies before being addressed with HOT.

Cutaneous Vascular Compromise
At our institution, a 36-year-old man was referred to the dermatology clinic 2 days after undergoing embolization of a symptomatic arteriovenous malformation in the right knee (Figure 3A). The procedure was complicated by cutaneous purpura concerning for necrosis, a known complication of this procedure. We referred the patient for evaluation to consider HOT. Although he was outside the ideal window for starting treatment, HOT was initiated. With a late start in treatment, areas of skin had already progressed to full necrosis, which did not respond to treatment; however, contiguous areas that initially looked very similar clinically did respond to treatment (Figure 3B). This case suggests a penumbralike effect in which vulnerable tissue that would most likely have been lost was salvaged by HOT.

Uittenbogaard et al²² reported the treatment of a patient who experienced dermal ischemia after receiving calcium hydroxylapatite at an aesthetic clinic. She did not improve with standard treatment but subsequently experienced resolution of symptoms after treatment with HOT. She had an excellent cosmetic outcome at 6-month follow-up.²²

Complications and a Contraindication

Hyperbaric oxygen therapy generally is safe, but there is potential for complications.

Fire
This rare risk has a catastrophic outcome.²³ Standards for fire prevention in hyperbaric facilities are issued by the National Fire Protection Association, covering construction and building materials, lighting, electrical wiring, exposure to flammable materials, and other possible ignition sources.²⁴

Middle Ear Barotrauma
The incidence of the most common adverse effect of HOT is reported at 2% to 30%.^7,25 Middle ear barotrauma occurs most commonly during the compression phase of treatment. It is more common in patients treated in a monoplace chamber because they are kept supine and are less able to regulate middle ear pressure.²⁶ Symptoms of middle ear barotrauma can be relieved by teaching patients autoinflation technique, such as the Valsalva maneuver, or by placing tympanoplasty tubes.²⁷

Reversible Myopia
Caused by direct O₂ toxicity to the lens, this complication can last for weeks, though it eventually resolves spontaneously. Reversible myopia has been reported to be at least as common as middle ear barotrauma.²⁷

Other Complications
Central nervous system complications, such as seizures, and pulmonary O₂ toxicity are rare, more serious complications.²⁷

Untreated Pneumothorax
The only absolute contraindication to HOT, pneumothorax can decompensate during HOT if left untreated. However, HOT can proceed once pneumothorax is addressed.⁷

Conclusion

Hyperbaric O₂ therapy can make a positive contribution to the dermatologic therapeutic armamentarium, in specific patients, for impending graft or flap failure, chronic wounds and ulcerations, and cutaneous vascular compromise. Although HOT is not a commonly needed treatment in dermatology, it is important to be aware of its potential because delay in treatment can decrease its effectiveness. It is recommended that dermatologists locate the nearest HOT facility and become familiar with its capabilities.

Hyperbaric oxygen therapy (HOT) is a treatment modality dating to 1861 in the United States.¹ Today, there are 14 indications² for HOT (Table), issued by the Undersea & Hyperbaric Medical Society, which also administers an accreditation program for facilities providing HOT.³ The 14 indications also are relevant because it is unlikely that HOT will be covered by insurance for unapproved indications.⁴

Although HOT is not commonly seen as a first-line intervention in dermatology, there are scenarios in which it can be used to good effect: compromised grafts and flaps; poorly healing ulceration related to vasculitis and autoimmune disorders; and possibly for vascular compromise, including cutaneous ischemia caused by fillers. We review its indications, dermatologic applications, and potential complications.

Overview of HOT

Hyperbaric oxygen therapy involves sitting or lying in a special chamber that allows for controlled levels of oxygen (O₂) at increased atmospheric pressure, which specifically involves breathing near 100% O₂ while inside a monoplace or multiplace chamber⁵ that is pressurized to greater than sea level pressure (≥1.4 atmosphere absolute).²

A monoplace chamber is designed to treat a single person (Figure 1); a multiplace chamber (Figure 2) accommodates as many as 5 to 25 patients.^5,6 The chambers also accommodate hospital beds and medical attendants, if needed. Hyperbaric O₂ is inhaled through a mask, a tight-fitting hood, or an endotracheal tube, depending on the patient’s status.⁷ Treatment ranges from only 1 or 2 iterations for acute conditions to 30 sessions or more for chronic conditions. Individual sessions last 45 minutes to 5 hours; 120 minutes is considered a safe maximum duration.⁷ A television often is provided to help the patient pass the time.⁸

Long-standing Use in Decompression Sickness

Hyperbaric oxygen therapy is best known for its effectiveness in treating decompression sickness (DCS) and carbon monoxide poisoning. Decompression sickness involves liberation of free gas from tissue, in the form of bubbles, when a person experiences a relative decrease in atmospheric pressure, which results in an imbalance in the sum of gas tensions in tissue compared to ambient pressure.

Decompression sickness has special military significance because it can affect divers and pilots, particularly those flying at high altitude. Over the course of 12 years, approximately 50 pilot trainees at an Air Force training site in Colorado required HOT when ground-level O₂ failed to resolve their DCS symptoms.¹⁰

Symptoms of DCS range from musculoskeletal pain to severe neurologic and pulmonary complications. First-line therapy for DCS is 100% O₂ at ground level. When symptoms are severe or persistent, HOT is the treatment of choice. It works by decreasing the volume of air bubbles (as predicted by Boyle’s Law), providing oxygenation to hypoxic tissue and mitigating inflammatory responses implicated in tissue injury⁹; HOT can be considered salvage treatment for rare, severe, or unresponsive complications of DCS during common activities such as diving and flying.

The emergent nature of DCS often necessitates an on-call, on-site HOT facility or contracted community services. Although DCS is a rare complication, it can be devastating, as was the case for a military pilot flying an ultrahigh altitude reconnaissance aircraft.¹¹ He developed a near fatal case of neurologic DCS during a military mission and required treatment with emergent HOT. Although his symptoms were reduced with therapy, he has persistent cognitive deficits.¹¹

Other Indications

Dermatologic Flaps and Grafts
Although less commonly discussed in dermatologic literature, the use of HOT in compromised grafts and flaps has been addressed in the plastic surgery literature. In a large multicenter study, researchers evaluated 20,821 Mohs micrographic surgery procedures and reported 149 adverse events, of which 20.1% were dehiscence and partial or full necrosis.¹² These complications, though rare, are potentially devastating, particularly in cosmetically sensitive locations such as the face. Traditional care for compromised grafts and flaps includes local wound care, surgical debridement, and additional reconstructive procedures. These interventions can be expensive and uncomfortable for patients and carry risk for further morbidity.¹³

Grafts become compromised when their metabolic demand outpaces the ability of the recipient bed due to characteristics of the graft or the recipient bed or both. Flaps carry their own blood supply, which can be compromised if the flap is too long or too large for the pedicle, there is notable tension on the wound, or blood flow is mechanically obstructed by kinking or twisting. Under these conditions, HOT can be beneficial, as O₂ dissolves in plasma, thus improving the O₂ tissue cellular diffusion gradient.⁷ An increased level of systemic O₂ promotes wound healing and graft or flap survival by improving fibroblast function, blood flow, and vascularity, and by mitigating ischemia-reperfusion injury.¹³

In a study, 105 patients with an ischemic flap or graft were treated with HOT; most (89% of threatened flaps and 91% of threatened grafts) were salvaged. In this series, the duration of latency from the creation of the flap to initiation of HOT was directly proportional to the failure rate of this treatment modality.¹⁴

Radiation-Induced Ulceration
Radionecrosis, a complication of radiotherapy, is caused by progressive obliterating endarteritis with resultant vascular stenosis and fibroatrophy, which eventually cause stromal fibrosis.¹⁵ In a study that looked at 1267 nonmelanoma skin cancers that had been treated with radiotherapy, the ulceration rate was 6.3%. Most of the ulcerated lesions were treatable conservatively, but some were more treatment resistant.¹⁶ Hampson et al¹⁷ reported on 58 patients with cutaneous wounds due to soft-tissue radionecrosis who were treated with HOT as part of a larger observational case series in which investigators looked at multiple types of radionecrosis. They found that 76% of these patients improved: 26% showed complete resolution and the remaining 50% had 50% to 90% improvement.¹⁷

Vasculitis or Autoimmune Ulceration
Vasculitis and vasculopathy can occur independent of, or in association with, connective tissue disease and can result in chronic ulceration. At our institution, a patient with antimelanoma differentiation-associated protein 5 dermatomyositis who had refractory digital ulcerations despite intensive systemic therapy had an excellent response to HOT; ulcerations resolved after 37 treatments.¹⁸

Efrati et al¹⁹ reported on 35 patients who had chronic nonhealing vasculitic ulcerations despite immunosuppression medication who were treated with HOT. Twenty-eight patients completely healed, 4 had partial healing, and 3 had no improvement.

Mirasoglu et al²⁰ reported on a case series of 6 systemic sclerosis patients who had ulcerations that persisted despite other treatments. After initiation of HOT, 4 patients experienced complete response and 2 experienced partial response, which is notable because such ulcerations are often extremely difficult to treat and have usually failed multiple therapies before being addressed with HOT.

Cutaneous Vascular Compromise
At our institution, a 36-year-old man was referred to the dermatology clinic 2 days after undergoing embolization of a symptomatic arteriovenous malformation in the right knee (Figure 3A). The procedure was complicated by cutaneous purpura concerning for necrosis, a known complication of this procedure. We referred the patient for evaluation to consider HOT. Although he was outside the ideal window for starting treatment, HOT was initiated. With a late start in treatment, areas of skin had already progressed to full necrosis, which did not respond to treatment; however, contiguous areas that initially looked very similar clinically did respond to treatment (Figure 3B). This case suggests a penumbralike effect in which vulnerable tissue that would most likely have been lost was salvaged by HOT.

Uittenbogaard et al²² reported the treatment of a patient who experienced dermal ischemia after receiving calcium hydroxylapatite at an aesthetic clinic. She did not improve with standard treatment but subsequently experienced resolution of symptoms after treatment with HOT. She had an excellent cosmetic outcome at 6-month follow-up.²²

Complications and a Contraindication

Hyperbaric oxygen therapy generally is safe, but there is potential for complications.

Fire
This rare risk has a catastrophic outcome.²³ Standards for fire prevention in hyperbaric facilities are issued by the National Fire Protection Association, covering construction and building materials, lighting, electrical wiring, exposure to flammable materials, and other possible ignition sources.²⁴

Middle Ear Barotrauma
The incidence of the most common adverse effect of HOT is reported at 2% to 30%.^7,25 Middle ear barotrauma occurs most commonly during the compression phase of treatment. It is more common in patients treated in a monoplace chamber because they are kept supine and are less able to regulate middle ear pressure.²⁶ Symptoms of middle ear barotrauma can be relieved by teaching patients autoinflation technique, such as the Valsalva maneuver, or by placing tympanoplasty tubes.²⁷

Reversible Myopia
Caused by direct O₂ toxicity to the lens, this complication can last for weeks, though it eventually resolves spontaneously. Reversible myopia has been reported to be at least as common as middle ear barotrauma.²⁷

Other Complications
Central nervous system complications, such as seizures, and pulmonary O₂ toxicity are rare, more serious complications.²⁷

Untreated Pneumothorax
The only absolute contraindication to HOT, pneumothorax can decompensate during HOT if left untreated. However, HOT can proceed once pneumothorax is addressed.⁷

Conclusion

Hyperbaric O₂ therapy can make a positive contribution to the dermatologic therapeutic armamentarium, in specific patients, for impending graft or flap failure, chronic wounds and ulcerations, and cutaneous vascular compromise. Although HOT is not a commonly needed treatment in dermatology, it is important to be aware of its potential because delay in treatment can decrease its effectiveness. It is recommended that dermatologists locate the nearest HOT facility and become familiar with its capabilities.

Hyperbaric oxygen therapy (HOT) is a treatment modality dating to 1861 in the United States.¹ Today, there are 14 indications² for HOT (Table), issued by the Undersea & Hyperbaric Medical Society, which also administers an accreditation program for facilities providing HOT.³ The 14 indications also are relevant because it is unlikely that HOT will be covered by insurance for unapproved indications.⁴

Although HOT is not commonly seen as a first-line intervention in dermatology, there are scenarios in which it can be used to good effect: compromised grafts and flaps; poorly healing ulceration related to vasculitis and autoimmune disorders; and possibly for vascular compromise, including cutaneous ischemia caused by fillers. We review its indications, dermatologic applications, and potential complications.

Overview of HOT

Hyperbaric oxygen therapy involves sitting or lying in a special chamber that allows for controlled levels of oxygen (O₂) at increased atmospheric pressure, which specifically involves breathing near 100% O₂ while inside a monoplace or multiplace chamber⁵ that is pressurized to greater than sea level pressure (≥1.4 atmosphere absolute).²

A monoplace chamber is designed to treat a single person (Figure 1); a multiplace chamber (Figure 2) accommodates as many as 5 to 25 patients.^5,6 The chambers also accommodate hospital beds and medical attendants, if needed. Hyperbaric O₂ is inhaled through a mask, a tight-fitting hood, or an endotracheal tube, depending on the patient’s status.⁷ Treatment ranges from only 1 or 2 iterations for acute conditions to 30 sessions or more for chronic conditions. Individual sessions last 45 minutes to 5 hours; 120 minutes is considered a safe maximum duration.⁷ A television often is provided to help the patient pass the time.⁸

Long-standing Use in Decompression Sickness

Hyperbaric oxygen therapy is best known for its effectiveness in treating decompression sickness (DCS) and carbon monoxide poisoning. Decompression sickness involves liberation of free gas from tissue, in the form of bubbles, when a person experiences a relative decrease in atmospheric pressure, which results in an imbalance in the sum of gas tensions in tissue compared to ambient pressure.

Decompression sickness has special military significance because it can affect divers and pilots, particularly those flying at high altitude. Over the course of 12 years, approximately 50 pilot trainees at an Air Force training site in Colorado required HOT when ground-level O₂ failed to resolve their DCS symptoms.¹⁰

Symptoms of DCS range from musculoskeletal pain to severe neurologic and pulmonary complications. First-line therapy for DCS is 100% O₂ at ground level. When symptoms are severe or persistent, HOT is the treatment of choice. It works by decreasing the volume of air bubbles (as predicted by Boyle’s Law), providing oxygenation to hypoxic tissue and mitigating inflammatory responses implicated in tissue injury⁹; HOT can be considered salvage treatment for rare, severe, or unresponsive complications of DCS during common activities such as diving and flying.

The emergent nature of DCS often necessitates an on-call, on-site HOT facility or contracted community services. Although DCS is a rare complication, it can be devastating, as was the case for a military pilot flying an ultrahigh altitude reconnaissance aircraft.¹¹ He developed a near fatal case of neurologic DCS during a military mission and required treatment with emergent HOT. Although his symptoms were reduced with therapy, he has persistent cognitive deficits.¹¹

Other Indications

Dermatologic Flaps and Grafts
Although less commonly discussed in dermatologic literature, the use of HOT in compromised grafts and flaps has been addressed in the plastic surgery literature. In a large multicenter study, researchers evaluated 20,821 Mohs micrographic surgery procedures and reported 149 adverse events, of which 20.1% were dehiscence and partial or full necrosis.¹² These complications, though rare, are potentially devastating, particularly in cosmetically sensitive locations such as the face. Traditional care for compromised grafts and flaps includes local wound care, surgical debridement, and additional reconstructive procedures. These interventions can be expensive and uncomfortable for patients and carry risk for further morbidity.¹³

Grafts become compromised when their metabolic demand outpaces the ability of the recipient bed due to characteristics of the graft or the recipient bed or both. Flaps carry their own blood supply, which can be compromised if the flap is too long or too large for the pedicle, there is notable tension on the wound, or blood flow is mechanically obstructed by kinking or twisting. Under these conditions, HOT can be beneficial, as O₂ dissolves in plasma, thus improving the O₂ tissue cellular diffusion gradient.⁷ An increased level of systemic O₂ promotes wound healing and graft or flap survival by improving fibroblast function, blood flow, and vascularity, and by mitigating ischemia-reperfusion injury.¹³

In a study, 105 patients with an ischemic flap or graft were treated with HOT; most (89% of threatened flaps and 91% of threatened grafts) were salvaged. In this series, the duration of latency from the creation of the flap to initiation of HOT was directly proportional to the failure rate of this treatment modality.¹⁴

Radiation-Induced Ulceration
Radionecrosis, a complication of radiotherapy, is caused by progressive obliterating endarteritis with resultant vascular stenosis and fibroatrophy, which eventually cause stromal fibrosis.¹⁵ In a study that looked at 1267 nonmelanoma skin cancers that had been treated with radiotherapy, the ulceration rate was 6.3%. Most of the ulcerated lesions were treatable conservatively, but some were more treatment resistant.¹⁶ Hampson et al¹⁷ reported on 58 patients with cutaneous wounds due to soft-tissue radionecrosis who were treated with HOT as part of a larger observational case series in which investigators looked at multiple types of radionecrosis. They found that 76% of these patients improved: 26% showed complete resolution and the remaining 50% had 50% to 90% improvement.¹⁷

Vasculitis or Autoimmune Ulceration
Vasculitis and vasculopathy can occur independent of, or in association with, connective tissue disease and can result in chronic ulceration. At our institution, a patient with antimelanoma differentiation-associated protein 5 dermatomyositis who had refractory digital ulcerations despite intensive systemic therapy had an excellent response to HOT; ulcerations resolved after 37 treatments.¹⁸

Efrati et al¹⁹ reported on 35 patients who had chronic nonhealing vasculitic ulcerations despite immunosuppression medication who were treated with HOT. Twenty-eight patients completely healed, 4 had partial healing, and 3 had no improvement.

Mirasoglu et al²⁰ reported on a case series of 6 systemic sclerosis patients who had ulcerations that persisted despite other treatments. After initiation of HOT, 4 patients experienced complete response and 2 experienced partial response, which is notable because such ulcerations are often extremely difficult to treat and have usually failed multiple therapies before being addressed with HOT.

Cutaneous Vascular Compromise
At our institution, a 36-year-old man was referred to the dermatology clinic 2 days after undergoing embolization of a symptomatic arteriovenous malformation in the right knee (Figure 3A). The procedure was complicated by cutaneous purpura concerning for necrosis, a known complication of this procedure. We referred the patient for evaluation to consider HOT. Although he was outside the ideal window for starting treatment, HOT was initiated. With a late start in treatment, areas of skin had already progressed to full necrosis, which did not respond to treatment; however, contiguous areas that initially looked very similar clinically did respond to treatment (Figure 3B). This case suggests a penumbralike effect in which vulnerable tissue that would most likely have been lost was salvaged by HOT.

Uittenbogaard et al²² reported the treatment of a patient who experienced dermal ischemia after receiving calcium hydroxylapatite at an aesthetic clinic. She did not improve with standard treatment but subsequently experienced resolution of symptoms after treatment with HOT. She had an excellent cosmetic outcome at 6-month follow-up.²²

Complications and a Contraindication

Hyperbaric oxygen therapy generally is safe, but there is potential for complications.

Fire
This rare risk has a catastrophic outcome.²³ Standards for fire prevention in hyperbaric facilities are issued by the National Fire Protection Association, covering construction and building materials, lighting, electrical wiring, exposure to flammable materials, and other possible ignition sources.²⁴

Middle Ear Barotrauma
The incidence of the most common adverse effect of HOT is reported at 2% to 30%.^7,25 Middle ear barotrauma occurs most commonly during the compression phase of treatment. It is more common in patients treated in a monoplace chamber because they are kept supine and are less able to regulate middle ear pressure.²⁶ Symptoms of middle ear barotrauma can be relieved by teaching patients autoinflation technique, such as the Valsalva maneuver, or by placing tympanoplasty tubes.²⁷

Reversible Myopia
Caused by direct O₂ toxicity to the lens, this complication can last for weeks, though it eventually resolves spontaneously. Reversible myopia has been reported to be at least as common as middle ear barotrauma.²⁷

Other Complications
Central nervous system complications, such as seizures, and pulmonary O₂ toxicity are rare, more serious complications.²⁷

Untreated Pneumothorax
The only absolute contraindication to HOT, pneumothorax can decompensate during HOT if left untreated. However, HOT can proceed once pneumothorax is addressed.⁷

Conclusion

Hyperbaric O₂ therapy can make a positive contribution to the dermatologic therapeutic armamentarium, in specific patients, for impending graft or flap failure, chronic wounds and ulcerations, and cutaneous vascular compromise. Although HOT is not a commonly needed treatment in dermatology, it is important to be aware of its potential because delay in treatment can decrease its effectiveness. It is recommended that dermatologists locate the nearest HOT facility and become familiar with its capabilities.

Identification

The Hymenoptera order of insects includes Apidae (bees), Vespidae (wasps, yellow jackets, hornets), and Formicidae (fire ants). All 3 of these families of insects inject venom into their prey or as a defense mechanism via ovipositors in their abdomen. Vespids are the most aggressive and are found in each of the United States.¹ They have membranous wings, broad antennae, and a nonbarbed stinger (Figure 1).² The nonbarbed stinger of Vespidae differentiates them from Apidae and allows these insects to sting their prey multiple times. Vespids can build nests in the ground (yellow jackets), trees (hornets), or areas of cover such as window shutters (mud wasps). Because only the queens survive winter, larger populations do not develop until late summer when the most stings take place. Stings most often take place near the nest of the vespid or while the victim is eating outdoors.³

Envenomation

When vespids sting their prey they inject venom via their ovipositors.¹ The venom is composed of a mixture of low-molecular-weight proteins, kinins, proteolytic enzymes, lipids, carbohydrates, and high-molecular-weight proteins that act as allergens.^1,4,5 The proteolytic enzymes degrade the surrounding tissue, basophils become activated, and histamine is released secondary to mast cell degranulation, which results in vasodilation and an inflammatory response characterized by edema, erythema, warmth, and pain.¹ The pain of the sting is immediate and can be intense; almost all victims are acutely aware of the discomforting sensation.⁴

Management of Reactions

Three types of reactions can be seen after a vespid sting: uncomplicated local reactions, large local reactions, and systemic reactions (SRs). The most common reaction is the self-limiting uncomplicated local reaction that includes a focal area of warmth, edema, erythema, induration, and tenderness.¹ Treatment of this kind of reaction is supportive, with ice, nonsteroidal anti-inflammatory drugs, and H₁ and H₂ blockers being commonly used methods. Large local reactions (Figure 2) are similar to uncomplicated local reactions but are greater than 10 cm in diameter and last longer. The same symptomatic treatment may be used along with possible short (3–5 days) oral glucocorticoid (40–60 mg prednisone) or potent topical steroid administration if symptoms persist. Systemic reactions involve IgE-mediated generalized urticaria, angioedema, face swelling, stridor, bronchospasm, nausea, vomiting, flushing, and respiratory distress.¹ Emergency management includes maintenance of airway, breathing, and circulation. Epinephrine injection commonly is employed and should be given via intramuscular injection into the anterolateral thigh; a dose of 0.3 to 0.5 mg can be repeatedly injected every 5 to 15 minutes, as needed.¹

If an individual has an SR, it is recommended to go to an emergency department after stabilization for monitoring. Referral to an allergist for desensitization is appropriate. A radioallergosorbent test to measure allergen-specific IgE can be helpful to confirm an allergy.⁴ This test also should be done weeks after the incident because during the first few days IgE may be too low to measure. Once the allergy is confirmed, the desensitization with venom immunotherapy (VIT) can begin. Venom immunotherapy is effective and reduces a patient’s risk for recurrent SRs to less than 5% to 20%.⁶ A 2015 study recommended longer duration of VIT therapy due to risk for repeat SRs after discontinuing therapy. This study concluded that VIT is to be administered for 5 years, unless the patient is at high risk for SRs after VIT therapy—risk factors include older age, cardiopulmonary disease, SR during VIT treatment, mast cell disorders, and elevated serum tryptase—in which case VIT may have to be continued indefinitely. It is recommended that all patients with history of SR carry an epinephrine autoinjector in case of emergency.⁶

Epidemiologic data show a prevalence of 0.3% to 7.5% for self-reported SRs due to stings, with lower prevalence in children (0.15%–0.3%).^4,7 An additional study looking at data from an allergy practice determined 24% of all cases of anaphylaxis were due to insect stings.⁵

Conclusion

Although many vespid stings can be managed symptomatically, it is imperative for patients and providers to be aware of the possible severe reactions that can take place. It is essential for providers to be aware of how to care for and treat large local reactions and SRs, as symptom recognition and timely treatment can improve patient safety and result in better outcomes.

Identification

The Hymenoptera order of insects includes Apidae (bees), Vespidae (wasps, yellow jackets, hornets), and Formicidae (fire ants). All 3 of these families of insects inject venom into their prey or as a defense mechanism via ovipositors in their abdomen. Vespids are the most aggressive and are found in each of the United States.¹ They have membranous wings, broad antennae, and a nonbarbed stinger (Figure 1).² The nonbarbed stinger of Vespidae differentiates them from Apidae and allows these insects to sting their prey multiple times. Vespids can build nests in the ground (yellow jackets), trees (hornets), or areas of cover such as window shutters (mud wasps). Because only the queens survive winter, larger populations do not develop until late summer when the most stings take place. Stings most often take place near the nest of the vespid or while the victim is eating outdoors.³

Envenomation

When vespids sting their prey they inject venom via their ovipositors.¹ The venom is composed of a mixture of low-molecular-weight proteins, kinins, proteolytic enzymes, lipids, carbohydrates, and high-molecular-weight proteins that act as allergens.^1,4,5 The proteolytic enzymes degrade the surrounding tissue, basophils become activated, and histamine is released secondary to mast cell degranulation, which results in vasodilation and an inflammatory response characterized by edema, erythema, warmth, and pain.¹ The pain of the sting is immediate and can be intense; almost all victims are acutely aware of the discomforting sensation.⁴

Management of Reactions

Three types of reactions can be seen after a vespid sting: uncomplicated local reactions, large local reactions, and systemic reactions (SRs). The most common reaction is the self-limiting uncomplicated local reaction that includes a focal area of warmth, edema, erythema, induration, and tenderness.¹ Treatment of this kind of reaction is supportive, with ice, nonsteroidal anti-inflammatory drugs, and H₁ and H₂ blockers being commonly used methods. Large local reactions (Figure 2) are similar to uncomplicated local reactions but are greater than 10 cm in diameter and last longer. The same symptomatic treatment may be used along with possible short (3–5 days) oral glucocorticoid (40–60 mg prednisone) or potent topical steroid administration if symptoms persist. Systemic reactions involve IgE-mediated generalized urticaria, angioedema, face swelling, stridor, bronchospasm, nausea, vomiting, flushing, and respiratory distress.¹ Emergency management includes maintenance of airway, breathing, and circulation. Epinephrine injection commonly is employed and should be given via intramuscular injection into the anterolateral thigh; a dose of 0.3 to 0.5 mg can be repeatedly injected every 5 to 15 minutes, as needed.¹

If an individual has an SR, it is recommended to go to an emergency department after stabilization for monitoring. Referral to an allergist for desensitization is appropriate. A radioallergosorbent test to measure allergen-specific IgE can be helpful to confirm an allergy.⁴ This test also should be done weeks after the incident because during the first few days IgE may be too low to measure. Once the allergy is confirmed, the desensitization with venom immunotherapy (VIT) can begin. Venom immunotherapy is effective and reduces a patient’s risk for recurrent SRs to less than 5% to 20%.⁶ A 2015 study recommended longer duration of VIT therapy due to risk for repeat SRs after discontinuing therapy. This study concluded that VIT is to be administered for 5 years, unless the patient is at high risk for SRs after VIT therapy—risk factors include older age, cardiopulmonary disease, SR during VIT treatment, mast cell disorders, and elevated serum tryptase—in which case VIT may have to be continued indefinitely. It is recommended that all patients with history of SR carry an epinephrine autoinjector in case of emergency.⁶

Epidemiologic data show a prevalence of 0.3% to 7.5% for self-reported SRs due to stings, with lower prevalence in children (0.15%–0.3%).^4,7 An additional study looking at data from an allergy practice determined 24% of all cases of anaphylaxis were due to insect stings.⁵

Conclusion

Although many vespid stings can be managed symptomatically, it is imperative for patients and providers to be aware of the possible severe reactions that can take place. It is essential for providers to be aware of how to care for and treat large local reactions and SRs, as symptom recognition and timely treatment can improve patient safety and result in better outcomes.

Identification

The Hymenoptera order of insects includes Apidae (bees), Vespidae (wasps, yellow jackets, hornets), and Formicidae (fire ants). All 3 of these families of insects inject venom into their prey or as a defense mechanism via ovipositors in their abdomen. Vespids are the most aggressive and are found in each of the United States.¹ They have membranous wings, broad antennae, and a nonbarbed stinger (Figure 1).² The nonbarbed stinger of Vespidae differentiates them from Apidae and allows these insects to sting their prey multiple times. Vespids can build nests in the ground (yellow jackets), trees (hornets), or areas of cover such as window shutters (mud wasps). Because only the queens survive winter, larger populations do not develop until late summer when the most stings take place. Stings most often take place near the nest of the vespid or while the victim is eating outdoors.³

Envenomation

When vespids sting their prey they inject venom via their ovipositors.¹ The venom is composed of a mixture of low-molecular-weight proteins, kinins, proteolytic enzymes, lipids, carbohydrates, and high-molecular-weight proteins that act as allergens.^1,4,5 The proteolytic enzymes degrade the surrounding tissue, basophils become activated, and histamine is released secondary to mast cell degranulation, which results in vasodilation and an inflammatory response characterized by edema, erythema, warmth, and pain.¹ The pain of the sting is immediate and can be intense; almost all victims are acutely aware of the discomforting sensation.⁴

Management of Reactions

Three types of reactions can be seen after a vespid sting: uncomplicated local reactions, large local reactions, and systemic reactions (SRs). The most common reaction is the self-limiting uncomplicated local reaction that includes a focal area of warmth, edema, erythema, induration, and tenderness.¹ Treatment of this kind of reaction is supportive, with ice, nonsteroidal anti-inflammatory drugs, and H₁ and H₂ blockers being commonly used methods. Large local reactions (Figure 2) are similar to uncomplicated local reactions but are greater than 10 cm in diameter and last longer. The same symptomatic treatment may be used along with possible short (3–5 days) oral glucocorticoid (40–60 mg prednisone) or potent topical steroid administration if symptoms persist. Systemic reactions involve IgE-mediated generalized urticaria, angioedema, face swelling, stridor, bronchospasm, nausea, vomiting, flushing, and respiratory distress.¹ Emergency management includes maintenance of airway, breathing, and circulation. Epinephrine injection commonly is employed and should be given via intramuscular injection into the anterolateral thigh; a dose of 0.3 to 0.5 mg can be repeatedly injected every 5 to 15 minutes, as needed.¹

If an individual has an SR, it is recommended to go to an emergency department after stabilization for monitoring. Referral to an allergist for desensitization is appropriate. A radioallergosorbent test to measure allergen-specific IgE can be helpful to confirm an allergy.⁴ This test also should be done weeks after the incident because during the first few days IgE may be too low to measure. Once the allergy is confirmed, the desensitization with venom immunotherapy (VIT) can begin. Venom immunotherapy is effective and reduces a patient’s risk for recurrent SRs to less than 5% to 20%.⁶ A 2015 study recommended longer duration of VIT therapy due to risk for repeat SRs after discontinuing therapy. This study concluded that VIT is to be administered for 5 years, unless the patient is at high risk for SRs after VIT therapy—risk factors include older age, cardiopulmonary disease, SR during VIT treatment, mast cell disorders, and elevated serum tryptase—in which case VIT may have to be continued indefinitely. It is recommended that all patients with history of SR carry an epinephrine autoinjector in case of emergency.⁶

Epidemiologic data show a prevalence of 0.3% to 7.5% for self-reported SRs due to stings, with lower prevalence in children (0.15%–0.3%).^4,7 An additional study looking at data from an allergy practice determined 24% of all cases of anaphylaxis were due to insect stings.⁵

Conclusion

Although many vespid stings can be managed symptomatically, it is imperative for patients and providers to be aware of the possible severe reactions that can take place. It is essential for providers to be aware of how to care for and treat large local reactions and SRs, as symptom recognition and timely treatment can improve patient safety and result in better outcomes.

Major changes in continuing board certification are occurring across medical specialties. On January 6, 2020, the American Board of Dermatology (ABD) launches its new web-based longitudinal assessment program called CertLink (https://abderm.mycertlink.org/).¹ This new platform is designed to eventually replace the sit-down, high-stakes, once-every-10-year medical knowledge examination that dermatologists take to remain board certified. With this alternative, every participating dermatologist will receive a batch of 13 web-based questions every quarter that he/she may answer at a convenient time and place. Questions are answered one at a time or in batches, depending on the test taker’s preference, and can be completed on home or office computers (and eventually on smartphones). Participating in this type of testing does not require shutting down practice, traveling to a test center, or paying for expensive board review courses. CertLink is designed to be convenient, affordable, and relevant to an individual’s practice.

How did the ABD arrive at CertLink?

The ABD launched its original Maintenance of Certification (MOC) program in 2006. Since then, newly board-certified dermatologists, recertifying dermatologists with time-limited certificates, and time-unlimited dermatologists who volunteered to participate in MOC have experienced the dermatology MOC program. In its first 10 years, the program was met with very mixed reviews. The program was designed to assess and promote competence in a 4-part framework, including professionalism; commitment to lifelong learning and self-assessment; demonstration of knowledge, judgment, and skills; and improvement in medical practice. All 4 are areas of rational pursuit for medical professionals seeking to perform and maintain the highest quality patient care possible. But there were problems. First iterations are rarely perfect, and dermatology MOC was no exception.

At the onset, the ABD chose to oversee the MOC requirements and remained hands off in the delivery of education, relying instead on other organizations to fulfill the ABD’s requirements. Unfortunately, with limited educational offerings available, many diplomates paid notable registration fees for each qualifying MOC activity. Quality improvement activities were a relatively new experience for dermatologists and were time consuming. Required medical record reviews were onerous, often requiring more than 35 data points to be collected per medical record reviewed. The limited number and limited diversity of educational offerings also created circumstances in which the material covered was not maximally relevant to many participants. When paying to answer questions about patient populations or procedure types never encountered by the dermatologist who purchased the particular MOC activity, many asked the question “How does this make me a better doctor?” They were right to ask.

Cost, time commitment to participate in MOC, and relevance to practice were 3 key areas of concern for many dermatologists. In response to internal and external MOC feedback, in 2015 the ABD took a hard look at its 10-year experience with MOC. While contemplating its next strategies, the ABD temporarily put its component 4—practice improvement—requirements on hold. After much review, the ABD decided to take over a notable portion of the education delivery. Its goal was to provide education that would fulfill MOC requirements in a more affordable, relevant, quicker, and easier manner.

First, the ABD made the decision to assume a more notable role as educator, in part to offer qualifying activities at no additional cost to diplomates. By taking on the role as educator, 3 major changes resulted: the way ABD approached quality improvement activities, partnership to initiate a question-of-the-week self-assessment program, and initiation of a longitudinal assessment strategy that resulted in this month’s launch of CertLink.

The ABD revolutionized its quality improvement requirements with the launch of its practice improvement modules made available through its website.² These modules utilize recently published clinical practice gaps in 5 dermatology subspecialty domains to fulfill the practice improvement requirements. Participants read a brief synopsis of the supporting literature explaining practice improvement recommendations found in the module. Next, they find 5 patients in their practice with the condition, medication, or process in question and review whether they provided the care supported by the best available evidence. No module requires more than 5 medical records to review, and no more than 3 questions are answered per medical record review. If review confirms that the care was appropriate, no further action is needed. If a care gap is identified, then participants implement changes and later remeasure practice to detect any change. This certification activity was incredibly popular with the thousands of diplomates who have participated thus far; more than 97% stated the modules were relevant to practice, 98% stated they would recommend the modules to fellow dermatologists, and nearly 25% reported the module helped to change their practice for the better (unpublished data, July 2019). Relevance had been restored.

The ABD worked closely with the American Academy of Dermatology (AAD) to develop new education for weekly self-assessment. The ABD created the content and delivered to the AAD the first year of material for what would become the most successful and popular dermatology CME activity in history: the AAD Question of the Week (QOW). Thousands of dermatologists are registered to receive the QOW, with very active weekly participation. Participants receive 1 self-assessment point and 0.25 CME credits for each attempted question, right or wrong. This quizzing tool also was educational, with explanation of right answers and wrong choices included. The average amount of time spent answering each question was approximately 40 seconds. American Academy of Dermatology members can participate in its QOW as a member benefit. Self-assessment is no longer a time- consuming or costly process.

What will be part of CertLink?

In addition to standard multiple-choice questions, there are many interesting elements to the CertLink program, such as article-based questions. At the beginning of each year, dermatologists select 8 articles from a list of those hosted by CertLink. These are recently published articles, chosen for their meaningfulness to practicing dermatologists. Each subsequent quarter, 2 of these articles are issued to the diplomate to read at his/her leisure. Once ready, participants launch and answer 2 questions about the key points of each article. The article-based questions were designed to help the practicing dermatologist stay up-to-date and relevant in personally chosen areas.

Diplomates are offered a chance to learn from any question that was missed, with explanations or resources provided to help them understand why the correct answer is correct. In this new learn-to-competence model, diplomates are not penalized the first time they answer a particular question incorrectly. Each is provided an opportunity to learn through the explanations given, and then in a future quarter, the dermatologist is given a second chance to answer a similarly themed question, with only that second chance counting toward his/her overall score.

Another unique aspect of CertLink is the allowance of time off from assessment. The ABD recognizes that life happens, and that intermittent time off from career-long assessment will be necessary to accommodate life events, including but not limited to maternity leave, other medical leave, or mental health breaks. Diplomates may take off up to 1 quarter of testing each year to accommodate such life events. Those who need extra time (beyond 1 quarter per year) would need to communicate directly with ABD to request. Those who continue to answer questions throughout the year will have their lowest-performing quarter dropped, to maximize fairness to all. Only the top 3 quarters of CertLink test performance will be counted each year when making certification status decisions. Those who take 1 quarter off will have their other 3 quarters counted toward their scoring.

How will CertLink measure performance?

At the onset of CertLink, there is no predetermined passing score. It will take a few years for the ABD psychometricians to determine an acceptable performance. Questions are written not to be tricky but rather to assess patient issues the dermatologist is likely to encounter in practice. Article-based questions are designed to assess the key points of important recent articles to advance the dermatologist’s practice.

Final Thoughts

In the end, the ABD approach to the new area of continuing certification centers on strategies to be relevant, inexpensive, and minimally disruptive to practice, and to teach to competence and advance practice by bringing forward articles that address key recent literature. We think it is a much better approach to dermatology continuing certification.

Major changes in continuing board certification are occurring across medical specialties. On January 6, 2020, the American Board of Dermatology (ABD) launches its new web-based longitudinal assessment program called CertLink (https://abderm.mycertlink.org/).¹ This new platform is designed to eventually replace the sit-down, high-stakes, once-every-10-year medical knowledge examination that dermatologists take to remain board certified. With this alternative, every participating dermatologist will receive a batch of 13 web-based questions every quarter that he/she may answer at a convenient time and place. Questions are answered one at a time or in batches, depending on the test taker’s preference, and can be completed on home or office computers (and eventually on smartphones). Participating in this type of testing does not require shutting down practice, traveling to a test center, or paying for expensive board review courses. CertLink is designed to be convenient, affordable, and relevant to an individual’s practice.

How did the ABD arrive at CertLink?

The ABD launched its original Maintenance of Certification (MOC) program in 2006. Since then, newly board-certified dermatologists, recertifying dermatologists with time-limited certificates, and time-unlimited dermatologists who volunteered to participate in MOC have experienced the dermatology MOC program. In its first 10 years, the program was met with very mixed reviews. The program was designed to assess and promote competence in a 4-part framework, including professionalism; commitment to lifelong learning and self-assessment; demonstration of knowledge, judgment, and skills; and improvement in medical practice. All 4 are areas of rational pursuit for medical professionals seeking to perform and maintain the highest quality patient care possible. But there were problems. First iterations are rarely perfect, and dermatology MOC was no exception.

At the onset, the ABD chose to oversee the MOC requirements and remained hands off in the delivery of education, relying instead on other organizations to fulfill the ABD’s requirements. Unfortunately, with limited educational offerings available, many diplomates paid notable registration fees for each qualifying MOC activity. Quality improvement activities were a relatively new experience for dermatologists and were time consuming. Required medical record reviews were onerous, often requiring more than 35 data points to be collected per medical record reviewed. The limited number and limited diversity of educational offerings also created circumstances in which the material covered was not maximally relevant to many participants. When paying to answer questions about patient populations or procedure types never encountered by the dermatologist who purchased the particular MOC activity, many asked the question “How does this make me a better doctor?” They were right to ask.

Cost, time commitment to participate in MOC, and relevance to practice were 3 key areas of concern for many dermatologists. In response to internal and external MOC feedback, in 2015 the ABD took a hard look at its 10-year experience with MOC. While contemplating its next strategies, the ABD temporarily put its component 4—practice improvement—requirements on hold. After much review, the ABD decided to take over a notable portion of the education delivery. Its goal was to provide education that would fulfill MOC requirements in a more affordable, relevant, quicker, and easier manner.

First, the ABD made the decision to assume a more notable role as educator, in part to offer qualifying activities at no additional cost to diplomates. By taking on the role as educator, 3 major changes resulted: the way ABD approached quality improvement activities, partnership to initiate a question-of-the-week self-assessment program, and initiation of a longitudinal assessment strategy that resulted in this month’s launch of CertLink.

The ABD revolutionized its quality improvement requirements with the launch of its practice improvement modules made available through its website.² These modules utilize recently published clinical practice gaps in 5 dermatology subspecialty domains to fulfill the practice improvement requirements. Participants read a brief synopsis of the supporting literature explaining practice improvement recommendations found in the module. Next, they find 5 patients in their practice with the condition, medication, or process in question and review whether they provided the care supported by the best available evidence. No module requires more than 5 medical records to review, and no more than 3 questions are answered per medical record review. If review confirms that the care was appropriate, no further action is needed. If a care gap is identified, then participants implement changes and later remeasure practice to detect any change. This certification activity was incredibly popular with the thousands of diplomates who have participated thus far; more than 97% stated the modules were relevant to practice, 98% stated they would recommend the modules to fellow dermatologists, and nearly 25% reported the module helped to change their practice for the better (unpublished data, July 2019). Relevance had been restored.

The ABD worked closely with the American Academy of Dermatology (AAD) to develop new education for weekly self-assessment. The ABD created the content and delivered to the AAD the first year of material for what would become the most successful and popular dermatology CME activity in history: the AAD Question of the Week (QOW). Thousands of dermatologists are registered to receive the QOW, with very active weekly participation. Participants receive 1 self-assessment point and 0.25 CME credits for each attempted question, right or wrong. This quizzing tool also was educational, with explanation of right answers and wrong choices included. The average amount of time spent answering each question was approximately 40 seconds. American Academy of Dermatology members can participate in its QOW as a member benefit. Self-assessment is no longer a time- consuming or costly process.

What will be part of CertLink?

In addition to standard multiple-choice questions, there are many interesting elements to the CertLink program, such as article-based questions. At the beginning of each year, dermatologists select 8 articles from a list of those hosted by CertLink. These are recently published articles, chosen for their meaningfulness to practicing dermatologists. Each subsequent quarter, 2 of these articles are issued to the diplomate to read at his/her leisure. Once ready, participants launch and answer 2 questions about the key points of each article. The article-based questions were designed to help the practicing dermatologist stay up-to-date and relevant in personally chosen areas.

Diplomates are offered a chance to learn from any question that was missed, with explanations or resources provided to help them understand why the correct answer is correct. In this new learn-to-competence model, diplomates are not penalized the first time they answer a particular question incorrectly. Each is provided an opportunity to learn through the explanations given, and then in a future quarter, the dermatologist is given a second chance to answer a similarly themed question, with only that second chance counting toward his/her overall score.

Another unique aspect of CertLink is the allowance of time off from assessment. The ABD recognizes that life happens, and that intermittent time off from career-long assessment will be necessary to accommodate life events, including but not limited to maternity leave, other medical leave, or mental health breaks. Diplomates may take off up to 1 quarter of testing each year to accommodate such life events. Those who need extra time (beyond 1 quarter per year) would need to communicate directly with ABD to request. Those who continue to answer questions throughout the year will have their lowest-performing quarter dropped, to maximize fairness to all. Only the top 3 quarters of CertLink test performance will be counted each year when making certification status decisions. Those who take 1 quarter off will have their other 3 quarters counted toward their scoring.

How will CertLink measure performance?

At the onset of CertLink, there is no predetermined passing score. It will take a few years for the ABD psychometricians to determine an acceptable performance. Questions are written not to be tricky but rather to assess patient issues the dermatologist is likely to encounter in practice. Article-based questions are designed to assess the key points of important recent articles to advance the dermatologist’s practice.

Final Thoughts

In the end, the ABD approach to the new area of continuing certification centers on strategies to be relevant, inexpensive, and minimally disruptive to practice, and to teach to competence and advance practice by bringing forward articles that address key recent literature. We think it is a much better approach to dermatology continuing certification.

Major changes in continuing board certification are occurring across medical specialties. On January 6, 2020, the American Board of Dermatology (ABD) launches its new web-based longitudinal assessment program called CertLink (https://abderm.mycertlink.org/).¹ This new platform is designed to eventually replace the sit-down, high-stakes, once-every-10-year medical knowledge examination that dermatologists take to remain board certified. With this alternative, every participating dermatologist will receive a batch of 13 web-based questions every quarter that he/she may answer at a convenient time and place. Questions are answered one at a time or in batches, depending on the test taker’s preference, and can be completed on home or office computers (and eventually on smartphones). Participating in this type of testing does not require shutting down practice, traveling to a test center, or paying for expensive board review courses. CertLink is designed to be convenient, affordable, and relevant to an individual’s practice.

How did the ABD arrive at CertLink?

The ABD launched its original Maintenance of Certification (MOC) program in 2006. Since then, newly board-certified dermatologists, recertifying dermatologists with time-limited certificates, and time-unlimited dermatologists who volunteered to participate in MOC have experienced the dermatology MOC program. In its first 10 years, the program was met with very mixed reviews. The program was designed to assess and promote competence in a 4-part framework, including professionalism; commitment to lifelong learning and self-assessment; demonstration of knowledge, judgment, and skills; and improvement in medical practice. All 4 are areas of rational pursuit for medical professionals seeking to perform and maintain the highest quality patient care possible. But there were problems. First iterations are rarely perfect, and dermatology MOC was no exception.

At the onset, the ABD chose to oversee the MOC requirements and remained hands off in the delivery of education, relying instead on other organizations to fulfill the ABD’s requirements. Unfortunately, with limited educational offerings available, many diplomates paid notable registration fees for each qualifying MOC activity. Quality improvement activities were a relatively new experience for dermatologists and were time consuming. Required medical record reviews were onerous, often requiring more than 35 data points to be collected per medical record reviewed. The limited number and limited diversity of educational offerings also created circumstances in which the material covered was not maximally relevant to many participants. When paying to answer questions about patient populations or procedure types never encountered by the dermatologist who purchased the particular MOC activity, many asked the question “How does this make me a better doctor?” They were right to ask.

Cost, time commitment to participate in MOC, and relevance to practice were 3 key areas of concern for many dermatologists. In response to internal and external MOC feedback, in 2015 the ABD took a hard look at its 10-year experience with MOC. While contemplating its next strategies, the ABD temporarily put its component 4—practice improvement—requirements on hold. After much review, the ABD decided to take over a notable portion of the education delivery. Its goal was to provide education that would fulfill MOC requirements in a more affordable, relevant, quicker, and easier manner.

First, the ABD made the decision to assume a more notable role as educator, in part to offer qualifying activities at no additional cost to diplomates. By taking on the role as educator, 3 major changes resulted: the way ABD approached quality improvement activities, partnership to initiate a question-of-the-week self-assessment program, and initiation of a longitudinal assessment strategy that resulted in this month’s launch of CertLink.

The ABD revolutionized its quality improvement requirements with the launch of its practice improvement modules made available through its website.² These modules utilize recently published clinical practice gaps in 5 dermatology subspecialty domains to fulfill the practice improvement requirements. Participants read a brief synopsis of the supporting literature explaining practice improvement recommendations found in the module. Next, they find 5 patients in their practice with the condition, medication, or process in question and review whether they provided the care supported by the best available evidence. No module requires more than 5 medical records to review, and no more than 3 questions are answered per medical record review. If review confirms that the care was appropriate, no further action is needed. If a care gap is identified, then participants implement changes and later remeasure practice to detect any change. This certification activity was incredibly popular with the thousands of diplomates who have participated thus far; more than 97% stated the modules were relevant to practice, 98% stated they would recommend the modules to fellow dermatologists, and nearly 25% reported the module helped to change their practice for the better (unpublished data, July 2019). Relevance had been restored.

The ABD worked closely with the American Academy of Dermatology (AAD) to develop new education for weekly self-assessment. The ABD created the content and delivered to the AAD the first year of material for what would become the most successful and popular dermatology CME activity in history: the AAD Question of the Week (QOW). Thousands of dermatologists are registered to receive the QOW, with very active weekly participation. Participants receive 1 self-assessment point and 0.25 CME credits for each attempted question, right or wrong. This quizzing tool also was educational, with explanation of right answers and wrong choices included. The average amount of time spent answering each question was approximately 40 seconds. American Academy of Dermatology members can participate in its QOW as a member benefit. Self-assessment is no longer a time- consuming or costly process.

What will be part of CertLink?

In addition to standard multiple-choice questions, there are many interesting elements to the CertLink program, such as article-based questions. At the beginning of each year, dermatologists select 8 articles from a list of those hosted by CertLink. These are recently published articles, chosen for their meaningfulness to practicing dermatologists. Each subsequent quarter, 2 of these articles are issued to the diplomate to read at his/her leisure. Once ready, participants launch and answer 2 questions about the key points of each article. The article-based questions were designed to help the practicing dermatologist stay up-to-date and relevant in personally chosen areas.

Diplomates are offered a chance to learn from any question that was missed, with explanations or resources provided to help them understand why the correct answer is correct. In this new learn-to-competence model, diplomates are not penalized the first time they answer a particular question incorrectly. Each is provided an opportunity to learn through the explanations given, and then in a future quarter, the dermatologist is given a second chance to answer a similarly themed question, with only that second chance counting toward his/her overall score.

Another unique aspect of CertLink is the allowance of time off from assessment. The ABD recognizes that life happens, and that intermittent time off from career-long assessment will be necessary to accommodate life events, including but not limited to maternity leave, other medical leave, or mental health breaks. Diplomates may take off up to 1 quarter of testing each year to accommodate such life events. Those who need extra time (beyond 1 quarter per year) would need to communicate directly with ABD to request. Those who continue to answer questions throughout the year will have their lowest-performing quarter dropped, to maximize fairness to all. Only the top 3 quarters of CertLink test performance will be counted each year when making certification status decisions. Those who take 1 quarter off will have their other 3 quarters counted toward their scoring.

How will CertLink measure performance?

At the onset of CertLink, there is no predetermined passing score. It will take a few years for the ABD psychometricians to determine an acceptable performance. Questions are written not to be tricky but rather to assess patient issues the dermatologist is likely to encounter in practice. Article-based questions are designed to assess the key points of important recent articles to advance the dermatologist’s practice.

Final Thoughts

In the end, the ABD approach to the new area of continuing certification centers on strategies to be relevant, inexpensive, and minimally disruptive to practice, and to teach to competence and advance practice by bringing forward articles that address key recent literature. We think it is a much better approach to dermatology continuing certification.

WASHINGTON – Research on pravastatin for the prevention of preeclampsia is moving along after “reassuring” data from pilot studies, with a large National Institutes of Health–funded trial currently recruiting women with a prior history of the disorder with preterm delivery at less than 34 weeks, Maged Costantine, MD, said at the biennial Diabetes in Pregnancy Study Group of North America meeting.

Creatas Images

More should be learned about low-dose aspirin, in the meantime, once the outcomes of a global study involving first-trimester initiation are published, said another speaker, Cynthia Gyamfi-Bannerman, MD, MS. Low-dose aspirin currently is recommended for preeclampsia prevention starting between 12 and 28 weeks, optimally before 16 weeks.

The biological plausibility of using pravastatin for preeclampsia prevention stems from the overlapping pathophysiology of preeclampsia with atherosclerotic cardiovascular disease – endothelial dysfunction and inflammation are common key mechanisms – as well as common risk factors, including diabetes and obesity, said Dr. Costantine, director of the division of maternal-fetal medicine at Ohio State University, Columbus, who is chairing the study.

In animal models of preeclampsia, pravastatin has been shown to upregulate placental growth factor, reduce antiangiogenic factors such as soluble fms-like tyrosine kinase 1 (sFlt1), and upregulate endothelial nitric oxide synthase. Mice have shown improved vascular reactivity, decreased proteinuria, decreased oxidative stress, and other positive effects, without any detrimental outcomes.

A pilot randomized controlled trial conducted with the Obstetric-Fetal Pharmacology Research Units Network and published in the American Journal of Obstetrics and Gynecology in 2016 assigned 10 women to 10 mg daily pravastatin and 10 women to placebo. The drug reduced maternal cholesterol concentrations but there were no differences in birth weight or umbilical cord cholesterol concentrations between the two groups.

Women in the pravastatin group were less likely to develop preeclampsia (none, compared with four in the placebo group), less likely to have an indicated preterm delivery (one, compared with five in the placebo group), and less likely to have their neonates admitted to the neonatal ICU.

There were no differences in side effects, congenital anomalies, or other adverse events. Dr. Costantine, principal investigator of the pilot study, and his colleagues wrote in the paper that the “favorable risk-benefit analysis justifies continued research with a dose escalation” (Am J Obstet Gynecol. 2016 Jun;214[6]:720.e1-17).

The new multicenter randomized controlled trial is randomizing 1,550 women to either 20 mg pravastatin or placebo starting between 12 weeks 0 days and 16 weeks 6 days. The primary outcome is a composite of preeclampsia, maternal death, or fetal loss. Secondary outcomes include a composite of severe maternal morbidity and various measures representing preeclampsia severity and complications, as well as preterm delivery less than 37 weeks and less than 34 weeks and various fetal/neonatal outcomes.

“In addition, we’ll look at development,” Dr. Costantine said, with offspring assessed at 2 and 5 years of age. The trial is sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Heart, Lung, and Blood Institute.

In the meantime, he said, the use of pravastatin to ameliorate early-onset preeclampsia is being tested in a small European proof-of-concept trial that has randomized women with early-onset preeclampsia (between 24 and 31 6/7 weeks) to 40 mg pravastatin or placebo. The primary outcome is reduction of antiangiogenic markers. Results are expected in another year or 2, he said.

The aspirin trial referred to by Dr. Gyamfi-Bannerman has been looking at the 81-mg dose of aspirin initiated between 6 0/7 and 13 6/7 weeks in nulliparous women who had no more than two previous pregnancy losses. The key question of the Aspirin Supplementation for Pregnancy Indicated Risk Reduction in Nulliparas (ASPIRIN) trial – conducted in the NICHD Global Network for Women’s and Children’s Health – is whether low-dose aspirin can reduce the rate of preterm birth. Preeclampsia is a secondary outcome (https://clinicaltrials.gov/ct2/show/NCT02409680).

“It may eventually be that the use of baby aspirin is further expanded to reduce the risk of preterm birth,” she said.

Overall, “we need more data on first-trimester use [of low-dose aspirin] and long-term outcomes,” Dr. Gyamfi-Bannerman said. And with respect to preeclampsia prevention specifically, more research is needed looking at risk reduction levels within specific groups of patients.

Since 2014, the U.S. Preventive Services Task Force (USPSTF) has called for low-dose aspirin at 81 mg/day in women who have one or more high-risk factors for preeclampsia (including type 1 or type 2 diabetes mellitus), and consideration of such treatment in patients with several moderate-risk factors. The American College of Obstetricians and Gynecologists’ recommendation varies slightly in that it advises treatment in patients with more than one (versus several) moderate-level risk factors (Obstet Gynecol. 2018;132[1]:e44-52).

Moderate-level risk factors include nulliparity, obesity, family history of preeclampsia, a baseline demographic risk (African-American or low socioeconomic status), and prior poor history (intrauterine growth restriction/small-for-gestational-age, previous poor outcome). “This is just about everyone I see,” Dr. Gyamfi-Bannerman said.

Dr. Gyamfi-Bannerman said she’d “love to see more data on higher doses” of low-dose aspirin – data that compares 81 mg/day with 150 mg/day, for instance.

A study published in 2017 in the New England Journal of Medicine randomized 1,776 women at high risk for preeclampsia to 150 mg/day or placebo and found a significant reduction in preterm preeclampsia (4.3% vs. 1.6%) in the aspirin group. Women in this European trial were deemed to be at high risk, however, based on a first-trimester screening algorithm that incorporated serum markers (maternal serum pregnancy-associated plasma protein A and placental growth factor) and uterine artery Doppler measures (N Engl J Med. 2017 Aug 17;377[7]:613-22).

“So it was a very interesting study, very provocative, but it’s hard to know how it would translate to the U.S. population [given that such screening practices] are not the way most of us are practicing here,” said Dr. Gyamfi-Bannerman, codirector of the Preterm Birth Prevention Center at Columbia University, New York, and professor of obstetrics and gynecology at the university.

The USPSTF based its recommendations on a systematic review that pooled data from 15 high-quality randomized controlled trials, including 13 that reported preeclampsia incidence among women at highest risk of disease. They found a 24% reduction in preeclampsia, but the actual risk reduction depends on the baseline population risk and may be closer to 10%, she said.

In a presentation on gaps in knowledge, Leslie Myatt, PhD, of the department of obstetrics and gynecology at Oregon Health and Science University, Portland, emphasized that preeclampsia is a syndrome with a heterogeneity of presentation and pathophysiology. “We don’t completely understand the pathophysiology,” he said.

Research needs to be “directed at the existence of multiple pathways [and subtypes],” he said, such that future therapies can be targeted and personalized.

Dr. Costantine did not report any disclosures. Dr. Gyamfi-Bannerman reported a Society of Maternal Fetal Medicine/AMAG Pharmaceuticals unrestricted grant and Eunice Kennedy Shriver National Institute of Child Health and Human Development/National Heart, Lung and Blood Institute funding. Dr. Myatt reported that he has no financial or other ties that pose a conflict of interest.

WASHINGTON – Research on pravastatin for the prevention of preeclampsia is moving along after “reassuring” data from pilot studies, with a large National Institutes of Health–funded trial currently recruiting women with a prior history of the disorder with preterm delivery at less than 34 weeks, Maged Costantine, MD, said at the biennial Diabetes in Pregnancy Study Group of North America meeting.

Creatas Images

More should be learned about low-dose aspirin, in the meantime, once the outcomes of a global study involving first-trimester initiation are published, said another speaker, Cynthia Gyamfi-Bannerman, MD, MS. Low-dose aspirin currently is recommended for preeclampsia prevention starting between 12 and 28 weeks, optimally before 16 weeks.

The biological plausibility of using pravastatin for preeclampsia prevention stems from the overlapping pathophysiology of preeclampsia with atherosclerotic cardiovascular disease – endothelial dysfunction and inflammation are common key mechanisms – as well as common risk factors, including diabetes and obesity, said Dr. Costantine, director of the division of maternal-fetal medicine at Ohio State University, Columbus, who is chairing the study.

In animal models of preeclampsia, pravastatin has been shown to upregulate placental growth factor, reduce antiangiogenic factors such as soluble fms-like tyrosine kinase 1 (sFlt1), and upregulate endothelial nitric oxide synthase. Mice have shown improved vascular reactivity, decreased proteinuria, decreased oxidative stress, and other positive effects, without any detrimental outcomes.

A pilot randomized controlled trial conducted with the Obstetric-Fetal Pharmacology Research Units Network and published in the American Journal of Obstetrics and Gynecology in 2016 assigned 10 women to 10 mg daily pravastatin and 10 women to placebo. The drug reduced maternal cholesterol concentrations but there were no differences in birth weight or umbilical cord cholesterol concentrations between the two groups.

Women in the pravastatin group were less likely to develop preeclampsia (none, compared with four in the placebo group), less likely to have an indicated preterm delivery (one, compared with five in the placebo group), and less likely to have their neonates admitted to the neonatal ICU.

There were no differences in side effects, congenital anomalies, or other adverse events. Dr. Costantine, principal investigator of the pilot study, and his colleagues wrote in the paper that the “favorable risk-benefit analysis justifies continued research with a dose escalation” (Am J Obstet Gynecol. 2016 Jun;214[6]:720.e1-17).

The new multicenter randomized controlled trial is randomizing 1,550 women to either 20 mg pravastatin or placebo starting between 12 weeks 0 days and 16 weeks 6 days. The primary outcome is a composite of preeclampsia, maternal death, or fetal loss. Secondary outcomes include a composite of severe maternal morbidity and various measures representing preeclampsia severity and complications, as well as preterm delivery less than 37 weeks and less than 34 weeks and various fetal/neonatal outcomes.

“In addition, we’ll look at development,” Dr. Costantine said, with offspring assessed at 2 and 5 years of age. The trial is sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Heart, Lung, and Blood Institute.

In the meantime, he said, the use of pravastatin to ameliorate early-onset preeclampsia is being tested in a small European proof-of-concept trial that has randomized women with early-onset preeclampsia (between 24 and 31 6/7 weeks) to 40 mg pravastatin or placebo. The primary outcome is reduction of antiangiogenic markers. Results are expected in another year or 2, he said.

The aspirin trial referred to by Dr. Gyamfi-Bannerman has been looking at the 81-mg dose of aspirin initiated between 6 0/7 and 13 6/7 weeks in nulliparous women who had no more than two previous pregnancy losses. The key question of the Aspirin Supplementation for Pregnancy Indicated Risk Reduction in Nulliparas (ASPIRIN) trial – conducted in the NICHD Global Network for Women’s and Children’s Health – is whether low-dose aspirin can reduce the rate of preterm birth. Preeclampsia is a secondary outcome (https://clinicaltrials.gov/ct2/show/NCT02409680).

“It may eventually be that the use of baby aspirin is further expanded to reduce the risk of preterm birth,” she said.

Overall, “we need more data on first-trimester use [of low-dose aspirin] and long-term outcomes,” Dr. Gyamfi-Bannerman said. And with respect to preeclampsia prevention specifically, more research is needed looking at risk reduction levels within specific groups of patients.

Since 2014, the U.S. Preventive Services Task Force (USPSTF) has called for low-dose aspirin at 81 mg/day in women who have one or more high-risk factors for preeclampsia (including type 1 or type 2 diabetes mellitus), and consideration of such treatment in patients with several moderate-risk factors. The American College of Obstetricians and Gynecologists’ recommendation varies slightly in that it advises treatment in patients with more than one (versus several) moderate-level risk factors (Obstet Gynecol. 2018;132[1]:e44-52).

Moderate-level risk factors include nulliparity, obesity, family history of preeclampsia, a baseline demographic risk (African-American or low socioeconomic status), and prior poor history (intrauterine growth restriction/small-for-gestational-age, previous poor outcome). “This is just about everyone I see,” Dr. Gyamfi-Bannerman said.

Dr. Gyamfi-Bannerman said she’d “love to see more data on higher doses” of low-dose aspirin – data that compares 81 mg/day with 150 mg/day, for instance.

A study published in 2017 in the New England Journal of Medicine randomized 1,776 women at high risk for preeclampsia to 150 mg/day or placebo and found a significant reduction in preterm preeclampsia (4.3% vs. 1.6%) in the aspirin group. Women in this European trial were deemed to be at high risk, however, based on a first-trimester screening algorithm that incorporated serum markers (maternal serum pregnancy-associated plasma protein A and placental growth factor) and uterine artery Doppler measures (N Engl J Med. 2017 Aug 17;377[7]:613-22).

“So it was a very interesting study, very provocative, but it’s hard to know how it would translate to the U.S. population [given that such screening practices] are not the way most of us are practicing here,” said Dr. Gyamfi-Bannerman, codirector of the Preterm Birth Prevention Center at Columbia University, New York, and professor of obstetrics and gynecology at the university.

The USPSTF based its recommendations on a systematic review that pooled data from 15 high-quality randomized controlled trials, including 13 that reported preeclampsia incidence among women at highest risk of disease. They found a 24% reduction in preeclampsia, but the actual risk reduction depends on the baseline population risk and may be closer to 10%, she said.

In a presentation on gaps in knowledge, Leslie Myatt, PhD, of the department of obstetrics and gynecology at Oregon Health and Science University, Portland, emphasized that preeclampsia is a syndrome with a heterogeneity of presentation and pathophysiology. “We don’t completely understand the pathophysiology,” he said.

Research needs to be “directed at the existence of multiple pathways [and subtypes],” he said, such that future therapies can be targeted and personalized.

Dr. Costantine did not report any disclosures. Dr. Gyamfi-Bannerman reported a Society of Maternal Fetal Medicine/AMAG Pharmaceuticals unrestricted grant and Eunice Kennedy Shriver National Institute of Child Health and Human Development/National Heart, Lung and Blood Institute funding. Dr. Myatt reported that he has no financial or other ties that pose a conflict of interest.

WASHINGTON – Research on pravastatin for the prevention of preeclampsia is moving along after “reassuring” data from pilot studies, with a large National Institutes of Health–funded trial currently recruiting women with a prior history of the disorder with preterm delivery at less than 34 weeks, Maged Costantine, MD, said at the biennial Diabetes in Pregnancy Study Group of North America meeting.

Creatas Images

More should be learned about low-dose aspirin, in the meantime, once the outcomes of a global study involving first-trimester initiation are published, said another speaker, Cynthia Gyamfi-Bannerman, MD, MS. Low-dose aspirin currently is recommended for preeclampsia prevention starting between 12 and 28 weeks, optimally before 16 weeks.

The biological plausibility of using pravastatin for preeclampsia prevention stems from the overlapping pathophysiology of preeclampsia with atherosclerotic cardiovascular disease – endothelial dysfunction and inflammation are common key mechanisms – as well as common risk factors, including diabetes and obesity, said Dr. Costantine, director of the division of maternal-fetal medicine at Ohio State University, Columbus, who is chairing the study.

In animal models of preeclampsia, pravastatin has been shown to upregulate placental growth factor, reduce antiangiogenic factors such as soluble fms-like tyrosine kinase 1 (sFlt1), and upregulate endothelial nitric oxide synthase. Mice have shown improved vascular reactivity, decreased proteinuria, decreased oxidative stress, and other positive effects, without any detrimental outcomes.

A pilot randomized controlled trial conducted with the Obstetric-Fetal Pharmacology Research Units Network and published in the American Journal of Obstetrics and Gynecology in 2016 assigned 10 women to 10 mg daily pravastatin and 10 women to placebo. The drug reduced maternal cholesterol concentrations but there were no differences in birth weight or umbilical cord cholesterol concentrations between the two groups.

Women in the pravastatin group were less likely to develop preeclampsia (none, compared with four in the placebo group), less likely to have an indicated preterm delivery (one, compared with five in the placebo group), and less likely to have their neonates admitted to the neonatal ICU.

There were no differences in side effects, congenital anomalies, or other adverse events. Dr. Costantine, principal investigator of the pilot study, and his colleagues wrote in the paper that the “favorable risk-benefit analysis justifies continued research with a dose escalation” (Am J Obstet Gynecol. 2016 Jun;214[6]:720.e1-17).

The new multicenter randomized controlled trial is randomizing 1,550 women to either 20 mg pravastatin or placebo starting between 12 weeks 0 days and 16 weeks 6 days. The primary outcome is a composite of preeclampsia, maternal death, or fetal loss. Secondary outcomes include a composite of severe maternal morbidity and various measures representing preeclampsia severity and complications, as well as preterm delivery less than 37 weeks and less than 34 weeks and various fetal/neonatal outcomes.

“In addition, we’ll look at development,” Dr. Costantine said, with offspring assessed at 2 and 5 years of age. The trial is sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Heart, Lung, and Blood Institute.

In the meantime, he said, the use of pravastatin to ameliorate early-onset preeclampsia is being tested in a small European proof-of-concept trial that has randomized women with early-onset preeclampsia (between 24 and 31 6/7 weeks) to 40 mg pravastatin or placebo. The primary outcome is reduction of antiangiogenic markers. Results are expected in another year or 2, he said.

The aspirin trial referred to by Dr. Gyamfi-Bannerman has been looking at the 81-mg dose of aspirin initiated between 6 0/7 and 13 6/7 weeks in nulliparous women who had no more than two previous pregnancy losses. The key question of the Aspirin Supplementation for Pregnancy Indicated Risk Reduction in Nulliparas (ASPIRIN) trial – conducted in the NICHD Global Network for Women’s and Children’s Health – is whether low-dose aspirin can reduce the rate of preterm birth. Preeclampsia is a secondary outcome (https://clinicaltrials.gov/ct2/show/NCT02409680).

“It may eventually be that the use of baby aspirin is further expanded to reduce the risk of preterm birth,” she said.

Overall, “we need more data on first-trimester use [of low-dose aspirin] and long-term outcomes,” Dr. Gyamfi-Bannerman said. And with respect to preeclampsia prevention specifically, more research is needed looking at risk reduction levels within specific groups of patients.

Since 2014, the U.S. Preventive Services Task Force (USPSTF) has called for low-dose aspirin at 81 mg/day in women who have one or more high-risk factors for preeclampsia (including type 1 or type 2 diabetes mellitus), and consideration of such treatment in patients with several moderate-risk factors. The American College of Obstetricians and Gynecologists’ recommendation varies slightly in that it advises treatment in patients with more than one (versus several) moderate-level risk factors (Obstet Gynecol. 2018;132[1]:e44-52).

Moderate-level risk factors include nulliparity, obesity, family history of preeclampsia, a baseline demographic risk (African-American or low socioeconomic status), and prior poor history (intrauterine growth restriction/small-for-gestational-age, previous poor outcome). “This is just about everyone I see,” Dr. Gyamfi-Bannerman said.

Dr. Gyamfi-Bannerman said she’d “love to see more data on higher doses” of low-dose aspirin – data that compares 81 mg/day with 150 mg/day, for instance.

A study published in 2017 in the New England Journal of Medicine randomized 1,776 women at high risk for preeclampsia to 150 mg/day or placebo and found a significant reduction in preterm preeclampsia (4.3% vs. 1.6%) in the aspirin group. Women in this European trial were deemed to be at high risk, however, based on a first-trimester screening algorithm that incorporated serum markers (maternal serum pregnancy-associated plasma protein A and placental growth factor) and uterine artery Doppler measures (N Engl J Med. 2017 Aug 17;377[7]:613-22).

“So it was a very interesting study, very provocative, but it’s hard to know how it would translate to the U.S. population [given that such screening practices] are not the way most of us are practicing here,” said Dr. Gyamfi-Bannerman, codirector of the Preterm Birth Prevention Center at Columbia University, New York, and professor of obstetrics and gynecology at the university.

The USPSTF based its recommendations on a systematic review that pooled data from 15 high-quality randomized controlled trials, including 13 that reported preeclampsia incidence among women at highest risk of disease. They found a 24% reduction in preeclampsia, but the actual risk reduction depends on the baseline population risk and may be closer to 10%, she said.

In a presentation on gaps in knowledge, Leslie Myatt, PhD, of the department of obstetrics and gynecology at Oregon Health and Science University, Portland, emphasized that preeclampsia is a syndrome with a heterogeneity of presentation and pathophysiology. “We don’t completely understand the pathophysiology,” he said.

Research needs to be “directed at the existence of multiple pathways [and subtypes],” he said, such that future therapies can be targeted and personalized.

Dr. Costantine did not report any disclosures. Dr. Gyamfi-Bannerman reported a Society of Maternal Fetal Medicine/AMAG Pharmaceuticals unrestricted grant and Eunice Kennedy Shriver National Institute of Child Health and Human Development/National Heart, Lung and Blood Institute funding. Dr. Myatt reported that he has no financial or other ties that pose a conflict of interest.

Diseases of the musculoskeletal (MSK) system are common, accounting for some of the most frequent visits to primary care clinics.^1-3 In addition, care for patients with chronic MSK diseases represents a substantial economic burden.^4-6Unfortunately, despite the high prevalence and associated costs of these conditions, numerous reports have concluded that primary care providers (PCPs) are not well prepared to effectively address these problems due to gaps in health professions education (HPE) programs.^7-12 Among other effects, overutilization of high-cost diagnostic tools or specialty care providers are increasingly recognized as important drivers of unnecessary spending.^13-16

In response to this clinical training need, the Veterans Health Administration (VHA) developed a portfolio of educational experiences for VHA health care providers and trainees, including both the Salt Lake City and National MSK “mini-residencies.”^17-19 These programs have educated more than 800 individuals. Early observations show a progressive increase in the number of joint injections performed at participant’s VHA clinics as well as a reduction in unnecessary magnetic resonance imaging orders of the knee.^20,21 These findings may be interpreted as markers for improved access to care for veterans as well as cost savings for the health care system.

The success of these early initiatives was recognized by the medical leadership of the VHA Simulation Learning, Education and Research Network (SimLEARN), who requested the Mini-Residency course directors to implement a similar educational program at the National Simulation Center in Orlando, Florida. SimLEARN was created to promote best practices in learning and education and provides a high-tech immersive environment for the development and delivery of simulation-based training curricula to facilitate workforce development.²² This article describes the initial experience of the VHA SimLEARN MSK continuing professional development (CPD) training programs, including curriculum design and educational impact on early learners, and how this informed additional CPD needs to continue advancing MSK education and care.

Methods

The initial vision was inspired by the national MSK Mini-Residency initiative for PCPs, which involved 13 US Department of Veterans Affairs (VA) medical centers; its development, dissemination, and validity evidence for assessment methods have been previously described.^17,18,23 SimLEARN leadership attended a Mini-Residency, observing the educational experience and identifying learning objectives most aligned with national goals. The director and codirector of the MSK Mini-Residency (MJB, AMB) then worked with SimLEARN using its educational platform and train-the-trainer model to create a condensed 2-day course, centered on primary care evaluation and management of shoulder and knee pain. The course also included elements supporting educational leaders in providing similar trainings at their local facility (Table 1).

Curriculum was introduced through didactics and reinforced in hands-on sessions enhanced by peer-teaching, arthrocentesis task trainers, and simulated patient experiences. At the end of day 1, participants engaged in critical reflection, reviewing knowledge and skills they had acquired.

On day 2, each participant was evaluated using an observed structured clinical examination (OSCE) for the shoulder, followed by an observed structured teaching experience (OSTE). Given the complexity of the physical examination and the greater potential for appropriate interpretation of clinical findings to influence best practice care, the shoulder was emphasized for these experiences. Time constraints of a 2-day program based on SimLEARN format requirements prevented including an additional OSCE for the knee. At the conclusion of the course, faculty and participants discussed strategies for bringing this educational experience to learners at their local facilities as well as for avoiding potential barriers to implementation. The course was accredited through the VHA Employee Education System (EES), and participants received 16 hours of CPD credit.

Participants

Opportunity to attend was communicated through national, regional, and local VHA organizational networks. Participants self-registered online through the VHA Talent Management System, the main learning resource for VHA employee education, and registration was open to both PCPs and clinician educators. Class size was limited to 10 to facilitate detailed faculty observation during skill acquisition experiences, simulations, and assessment exercises.

Program Evaluation

A standard process for evaluating and measuring learning objectives was performed through VHA EES. Self-assessment surveys and OSCEs were used to assess the activity.

Self-assessment surveys were administered at the beginning and end of the program. Content was adapted from that used in the national MSK Mini-Residency initiative and revised by experts in survey design.^18,24,25 Pre- and postcourse surveys asked participants to rate how important it was for them to be competent in evaluating shoulder and knee pain and in performing related joint injections, as well as to rate their level of confidence in their ability to evaluate and manage these conditions. The survey used 5 construct-specific response options distributed equally on a visual scale. Participants’ learning goals were collected on the precourse survey.

Participants’ competence in performing and interpreting a systematic and thorough physical examination of the shoulder and in suggesting a reasonable plan of management were assessed using a single-station OSCE. This tool, which presented learners with a simulated case depicting rotator cuff pathology, has been described in multiple educational settings, and validity evidence supporting its use has been published.^18,19,23 Course faculty conducted the OSCE, one as the simulated patient, the other as the rater. Immediately following the examination, both faculty conducted a debriefing session with each participant. The OSCE was scored using the validated checklist for specific elements of the shoulder exam, followed by a structured sequence of questions exploring participants’ interpretation of findings, diagnostic impressions, and recommendations for initial management. Scores for participants’ differential diagnosis were based on the completeness and specificity of diagnoses given; scores for management plans were based on appropriateness and accuracy of both the primary and secondary approach to treatment or further diagnostic efforts. A global rating (range 1 to 9) was assigned, independent of scores in other domains.

Following the OSCE, participants rotated through a 3-cycle OSTE where they practiced the roles of simulated patient, learner, and educator. Faculty observed each OSTE and led focused debriefing sessions immediately following each rotation to facilitate participants’ critical reflection of their involvement in these elements of the course. This exercise was formative without quantitative assessment of performance.

Statistical Analysis

Pre- and postsurvey data were analyzed using a paired Student t test. Comparisons between multiple variables (eg, OSCE scores by years of experience or level of credentials) were analyzed using analysis of variance. Relationships between variables were analyzed with a Pearson correlation. All statistical analyses were conducted using IBM SPSS, Version 24 (Armonk, NY).

This project was reviewed by the institutional review board of the University of Utah and the Salt Lake City VA and was determined to be exempt from review because the work did not meet the definition of research with human subjects and was considered a quality improvement study.

Results

Twenty-four participants completed the program over 3 course offerings between February and May 2016, and all completed pre- and postcourse self-assessment surveys (Table 2). Self-ratings of the importance of competence in shoulder and knee MSK skills remained high before and after the course, and confidence improved significantly across all learning objectives. Despite the emphasis on the evaluation and management of shoulder pain, participants’ self-confidence still improved significantly with the knee—though these improvements were generally smaller in scale compared with those of the shoulder.

Overall OSCE scores and scores by domain were not found to be statistically different based on either years of experience or by level of credential or specialty (advanced practice registered nurse/physician assistant, PCP, or specialty care physician)(Table 3). However, there was a trend toward higher performance among the specialty care physician group, and a trend toward lower performance among participants with less than 3 years’ experience.

Discussion

Building on the foundation of other successful innovations in MSK education, the first year of the SimLEARN National MSK Training Program demonstrated the feasibility of a 2-day centralized national course as a method to increase participants’ confidence and competence in evaluating and managing MSK problems, and to disseminate a portable curriculum to a range of clinician educators. Although this course focused on developing competence for shoulder skills, including an OSCE on day 2, self-perceived improvements in participants’ ability to evaluate and manage knee pain were observed. Future program refinement and follow-up of participants’ experience and needs may lead to increased time allocated to the knee exam as well as objective measures of competence for knee skills.

In comparing our findings to the work that others have previously described, we looked for reports of CPD programs in 2 contexts: those that focused on acquisition of MSK skills relevant to clinical practice, and those designed as clinician educator or faculty development initiatives. Although there are few reports of MSK-themed CPD experiences designed specifically for nurses and allied health professionals, a recent effort to survey members of these disciplines in the United Kingdom was an important contribution to a systematic needs assessment.^26-28 Increased support from leadership, mostly in terms of time allowance and budgetary support, was identified as an important driver to facilitate participation in MSK CPD experiences. Through SimLEARN, the VHA is investing in CPD, providing the MSK Training Programs and other courses at no cost to its employees.

Most published reports on physician education have not evaluated content knowledge or physical examination skills with measures for which validity evidence has been published.^19,29,30 One notable exception is the 2000 Canadian Viscosupplementation Injector Preceptor experience, in which Bellamy and colleagues examined patient outcomes in evaluating their program.³¹

Our experience is congruent with the work of Macedo and colleagues and Sturpe and colleagues, who described the effectiveness and acceptability of an OSTE for faculty development.^32,33 These studies emphasize debriefing, a critical element in faculty development identified by Steinert and colleagues in a 2006 best evidence medical education (BEME) review.³⁴ The shoulder OSTE was one of the most well-received elements of our course, and each debrief was critical to facilitating rich discussions between educators and practitioners playing the role of teacher or student during this simulated experience, gaining insight into each other’s perspectives.

This program has several significant strengths: First, this is the most recent step in the development of a portfolio of innovative MSK CPD programs that were envisioned through a systematic process involving projections of cost-effectiveness, local pilot testing, and national expansion.^17,18,35 Second, the SimLEARN program uses assessment tools for which validity evidence has been published, made available for reflective critique by educational scholars.^19,23 This supports a national consortium of MSK educators, advancing clinical teaching and educational scholarship, and creating opportunities for interprofessional collaboration in congruence with the vision expressed in the 2010 Institute of Medicine report, “Redesigning Continuing Education in the Health Professions,” as well as the 2016 update of the BEME recommendations for faculty development.^36,37

Our experience with the SimLEARN National MSK Training Program demonstrates need for 2 distinct courses: (1) the MSK Clinician—serving PCPs seeking to develop their skills in evaluating and managing patients with MSK problems; and (2), the MSK Master Educator—for those with preexisting content expertise who would value the introduction to a national curriculum and connections with other MSK master educators. Both of these are now offered regularly through SimLEARN for VHA and US Department of Defense employees. The MSK Clinician program establishes competence in systematically evaluating and managing shoulder and knee MSK problems in an educational setting and prepares participants for subsequent clinical experiences where they can perform related procedures if desired, under appropriate supervision. The Master Educator program introduces partici pants to the clinician curriculum and provides the opportunity to develop an individualized plan for implementation of an MSK educational program at their home institutions. Participants are selected through a competitive application process, and funding for travel to attend the Master Educator program is provided by SimLEARN for participants who are accepted. Additionally, the Master Educator program serves as a repository for potential future SimLEARN MSK Clinician course faculty.

Limitations

The small number of participants may limit the validity of our conclusions. Although we included an OSCE to measure competence in performing and interpreting the shoulder exam, the durability of these skills is not known. Periodic postcourse OSCEs could help determine this and refresh and preserve accuracy in the performance of specific maneuvers. Second, although this experience was rated highly by participants, we do not know the impact of the program on their daily work or career trajectory. Sustained follow-up of learners, perhaps developed on the model of the Long-Term Career Outcome Study, may increase the value of this experience for future participants.³⁸ This program appealed to a diverse pool of learners, with a broad range of precourse expertise and varied expectations of how course experiences would impact their future work and career development. Some clinical educator attendees came from tertiary care facilities affiliated with academic medical centers, held specialist or subspecialist credentials, and had formal responsibilities as leaders in HPE. Other clinical practitioner participants were solitary PCPs, often in rural or home-based settings; although they may have been eager to apply new knowledge and skills in patient care, they neither anticipated nor desired any role as an educator.

Conclusion

The initial SimLEARN MSK Training Program provides PCPs and clinician educators with rich learning experiences, increasing confidence in addressing MSK problems and competence in performing and interpreting a systematic physical examination of the shoulder. The success of this program has created new opportunities for practitioners seeking to strengthen clinical skills and for leaders in health professions education looking to disseminate similar trainings and connect with a national group of educators.

Acknowledgments

The authors gratefully acknowledge the faculty and staff at the Veterans Health Administration SimLEARN National Simulation Center, the faculty of the Salt Lake City Musculoskeletal Mini-Residency program, the supportive leadership of the George E. Wahlen Salt Lake City Veterans Affairs Medical Center, and the efforts of Danielle Blake for logistical support and data entry.

Diseases of the musculoskeletal (MSK) system are common, accounting for some of the most frequent visits to primary care clinics.^1-3 In addition, care for patients with chronic MSK diseases represents a substantial economic burden.^4-6Unfortunately, despite the high prevalence and associated costs of these conditions, numerous reports have concluded that primary care providers (PCPs) are not well prepared to effectively address these problems due to gaps in health professions education (HPE) programs.^7-12 Among other effects, overutilization of high-cost diagnostic tools or specialty care providers are increasingly recognized as important drivers of unnecessary spending.^13-16

In response to this clinical training need, the Veterans Health Administration (VHA) developed a portfolio of educational experiences for VHA health care providers and trainees, including both the Salt Lake City and National MSK “mini-residencies.”^17-19 These programs have educated more than 800 individuals. Early observations show a progressive increase in the number of joint injections performed at participant’s VHA clinics as well as a reduction in unnecessary magnetic resonance imaging orders of the knee.^20,21 These findings may be interpreted as markers for improved access to care for veterans as well as cost savings for the health care system.

The success of these early initiatives was recognized by the medical leadership of the VHA Simulation Learning, Education and Research Network (SimLEARN), who requested the Mini-Residency course directors to implement a similar educational program at the National Simulation Center in Orlando, Florida. SimLEARN was created to promote best practices in learning and education and provides a high-tech immersive environment for the development and delivery of simulation-based training curricula to facilitate workforce development.²² This article describes the initial experience of the VHA SimLEARN MSK continuing professional development (CPD) training programs, including curriculum design and educational impact on early learners, and how this informed additional CPD needs to continue advancing MSK education and care.

Methods

The initial vision was inspired by the national MSK Mini-Residency initiative for PCPs, which involved 13 US Department of Veterans Affairs (VA) medical centers; its development, dissemination, and validity evidence for assessment methods have been previously described.^17,18,23 SimLEARN leadership attended a Mini-Residency, observing the educational experience and identifying learning objectives most aligned with national goals. The director and codirector of the MSK Mini-Residency (MJB, AMB) then worked with SimLEARN using its educational platform and train-the-trainer model to create a condensed 2-day course, centered on primary care evaluation and management of shoulder and knee pain. The course also included elements supporting educational leaders in providing similar trainings at their local facility (Table 1).

Curriculum was introduced through didactics and reinforced in hands-on sessions enhanced by peer-teaching, arthrocentesis task trainers, and simulated patient experiences. At the end of day 1, participants engaged in critical reflection, reviewing knowledge and skills they had acquired.

On day 2, each participant was evaluated using an observed structured clinical examination (OSCE) for the shoulder, followed by an observed structured teaching experience (OSTE). Given the complexity of the physical examination and the greater potential for appropriate interpretation of clinical findings to influence best practice care, the shoulder was emphasized for these experiences. Time constraints of a 2-day program based on SimLEARN format requirements prevented including an additional OSCE for the knee. At the conclusion of the course, faculty and participants discussed strategies for bringing this educational experience to learners at their local facilities as well as for avoiding potential barriers to implementation. The course was accredited through the VHA Employee Education System (EES), and participants received 16 hours of CPD credit.

Participants

Opportunity to attend was communicated through national, regional, and local VHA organizational networks. Participants self-registered online through the VHA Talent Management System, the main learning resource for VHA employee education, and registration was open to both PCPs and clinician educators. Class size was limited to 10 to facilitate detailed faculty observation during skill acquisition experiences, simulations, and assessment exercises.

Program Evaluation

A standard process for evaluating and measuring learning objectives was performed through VHA EES. Self-assessment surveys and OSCEs were used to assess the activity.

Self-assessment surveys were administered at the beginning and end of the program. Content was adapted from that used in the national MSK Mini-Residency initiative and revised by experts in survey design.^18,24,25 Pre- and postcourse surveys asked participants to rate how important it was for them to be competent in evaluating shoulder and knee pain and in performing related joint injections, as well as to rate their level of confidence in their ability to evaluate and manage these conditions. The survey used 5 construct-specific response options distributed equally on a visual scale. Participants’ learning goals were collected on the precourse survey.

Participants’ competence in performing and interpreting a systematic and thorough physical examination of the shoulder and in suggesting a reasonable plan of management were assessed using a single-station OSCE. This tool, which presented learners with a simulated case depicting rotator cuff pathology, has been described in multiple educational settings, and validity evidence supporting its use has been published.^18,19,23 Course faculty conducted the OSCE, one as the simulated patient, the other as the rater. Immediately following the examination, both faculty conducted a debriefing session with each participant. The OSCE was scored using the validated checklist for specific elements of the shoulder exam, followed by a structured sequence of questions exploring participants’ interpretation of findings, diagnostic impressions, and recommendations for initial management. Scores for participants’ differential diagnosis were based on the completeness and specificity of diagnoses given; scores for management plans were based on appropriateness and accuracy of both the primary and secondary approach to treatment or further diagnostic efforts. A global rating (range 1 to 9) was assigned, independent of scores in other domains.

Following the OSCE, participants rotated through a 3-cycle OSTE where they practiced the roles of simulated patient, learner, and educator. Faculty observed each OSTE and led focused debriefing sessions immediately following each rotation to facilitate participants’ critical reflection of their involvement in these elements of the course. This exercise was formative without quantitative assessment of performance.

Statistical Analysis

Pre- and postsurvey data were analyzed using a paired Student t test. Comparisons between multiple variables (eg, OSCE scores by years of experience or level of credentials) were analyzed using analysis of variance. Relationships between variables were analyzed with a Pearson correlation. All statistical analyses were conducted using IBM SPSS, Version 24 (Armonk, NY).

This project was reviewed by the institutional review board of the University of Utah and the Salt Lake City VA and was determined to be exempt from review because the work did not meet the definition of research with human subjects and was considered a quality improvement study.

Results

Twenty-four participants completed the program over 3 course offerings between February and May 2016, and all completed pre- and postcourse self-assessment surveys (Table 2). Self-ratings of the importance of competence in shoulder and knee MSK skills remained high before and after the course, and confidence improved significantly across all learning objectives. Despite the emphasis on the evaluation and management of shoulder pain, participants’ self-confidence still improved significantly with the knee—though these improvements were generally smaller in scale compared with those of the shoulder.

Overall OSCE scores and scores by domain were not found to be statistically different based on either years of experience or by level of credential or specialty (advanced practice registered nurse/physician assistant, PCP, or specialty care physician)(Table 3). However, there was a trend toward higher performance among the specialty care physician group, and a trend toward lower performance among participants with less than 3 years’ experience.

Discussion

Building on the foundation of other successful innovations in MSK education, the first year of the SimLEARN National MSK Training Program demonstrated the feasibility of a 2-day centralized national course as a method to increase participants’ confidence and competence in evaluating and managing MSK problems, and to disseminate a portable curriculum to a range of clinician educators. Although this course focused on developing competence for shoulder skills, including an OSCE on day 2, self-perceived improvements in participants’ ability to evaluate and manage knee pain were observed. Future program refinement and follow-up of participants’ experience and needs may lead to increased time allocated to the knee exam as well as objective measures of competence for knee skills.

In comparing our findings to the work that others have previously described, we looked for reports of CPD programs in 2 contexts: those that focused on acquisition of MSK skills relevant to clinical practice, and those designed as clinician educator or faculty development initiatives. Although there are few reports of MSK-themed CPD experiences designed specifically for nurses and allied health professionals, a recent effort to survey members of these disciplines in the United Kingdom was an important contribution to a systematic needs assessment.^26-28 Increased support from leadership, mostly in terms of time allowance and budgetary support, was identified as an important driver to facilitate participation in MSK CPD experiences. Through SimLEARN, the VHA is investing in CPD, providing the MSK Training Programs and other courses at no cost to its employees.

Most published reports on physician education have not evaluated content knowledge or physical examination skills with measures for which validity evidence has been published.^19,29,30 One notable exception is the 2000 Canadian Viscosupplementation Injector Preceptor experience, in which Bellamy and colleagues examined patient outcomes in evaluating their program.³¹

Our experience is congruent with the work of Macedo and colleagues and Sturpe and colleagues, who described the effectiveness and acceptability of an OSTE for faculty development.^32,33 These studies emphasize debriefing, a critical element in faculty development identified by Steinert and colleagues in a 2006 best evidence medical education (BEME) review.³⁴ The shoulder OSTE was one of the most well-received elements of our course, and each debrief was critical to facilitating rich discussions between educators and practitioners playing the role of teacher or student during this simulated experience, gaining insight into each other’s perspectives.

This program has several significant strengths: First, this is the most recent step in the development of a portfolio of innovative MSK CPD programs that were envisioned through a systematic process involving projections of cost-effectiveness, local pilot testing, and national expansion.^17,18,35 Second, the SimLEARN program uses assessment tools for which validity evidence has been published, made available for reflective critique by educational scholars.^19,23 This supports a national consortium of MSK educators, advancing clinical teaching and educational scholarship, and creating opportunities for interprofessional collaboration in congruence with the vision expressed in the 2010 Institute of Medicine report, “Redesigning Continuing Education in the Health Professions,” as well as the 2016 update of the BEME recommendations for faculty development.^36,37

Our experience with the SimLEARN National MSK Training Program demonstrates need for 2 distinct courses: (1) the MSK Clinician—serving PCPs seeking to develop their skills in evaluating and managing patients with MSK problems; and (2), the MSK Master Educator—for those with preexisting content expertise who would value the introduction to a national curriculum and connections with other MSK master educators. Both of these are now offered regularly through SimLEARN for VHA and US Department of Defense employees. The MSK Clinician program establishes competence in systematically evaluating and managing shoulder and knee MSK problems in an educational setting and prepares participants for subsequent clinical experiences where they can perform related procedures if desired, under appropriate supervision. The Master Educator program introduces partici pants to the clinician curriculum and provides the opportunity to develop an individualized plan for implementation of an MSK educational program at their home institutions. Participants are selected through a competitive application process, and funding for travel to attend the Master Educator program is provided by SimLEARN for participants who are accepted. Additionally, the Master Educator program serves as a repository for potential future SimLEARN MSK Clinician course faculty.

Limitations

The small number of participants may limit the validity of our conclusions. Although we included an OSCE to measure competence in performing and interpreting the shoulder exam, the durability of these skills is not known. Periodic postcourse OSCEs could help determine this and refresh and preserve accuracy in the performance of specific maneuvers. Second, although this experience was rated highly by participants, we do not know the impact of the program on their daily work or career trajectory. Sustained follow-up of learners, perhaps developed on the model of the Long-Term Career Outcome Study, may increase the value of this experience for future participants.³⁸ This program appealed to a diverse pool of learners, with a broad range of precourse expertise and varied expectations of how course experiences would impact their future work and career development. Some clinical educator attendees came from tertiary care facilities affiliated with academic medical centers, held specialist or subspecialist credentials, and had formal responsibilities as leaders in HPE. Other clinical practitioner participants were solitary PCPs, often in rural or home-based settings; although they may have been eager to apply new knowledge and skills in patient care, they neither anticipated nor desired any role as an educator.

Conclusion

The initial SimLEARN MSK Training Program provides PCPs and clinician educators with rich learning experiences, increasing confidence in addressing MSK problems and competence in performing and interpreting a systematic physical examination of the shoulder. The success of this program has created new opportunities for practitioners seeking to strengthen clinical skills and for leaders in health professions education looking to disseminate similar trainings and connect with a national group of educators.

Acknowledgments

The authors gratefully acknowledge the faculty and staff at the Veterans Health Administration SimLEARN National Simulation Center, the faculty of the Salt Lake City Musculoskeletal Mini-Residency program, the supportive leadership of the George E. Wahlen Salt Lake City Veterans Affairs Medical Center, and the efforts of Danielle Blake for logistical support and data entry.

Diseases of the musculoskeletal (MSK) system are common, accounting for some of the most frequent visits to primary care clinics.^1-3 In addition, care for patients with chronic MSK diseases represents a substantial economic burden.^4-6Unfortunately, despite the high prevalence and associated costs of these conditions, numerous reports have concluded that primary care providers (PCPs) are not well prepared to effectively address these problems due to gaps in health professions education (HPE) programs.^7-12 Among other effects, overutilization of high-cost diagnostic tools or specialty care providers are increasingly recognized as important drivers of unnecessary spending.^13-16

In response to this clinical training need, the Veterans Health Administration (VHA) developed a portfolio of educational experiences for VHA health care providers and trainees, including both the Salt Lake City and National MSK “mini-residencies.”^17-19 These programs have educated more than 800 individuals. Early observations show a progressive increase in the number of joint injections performed at participant’s VHA clinics as well as a reduction in unnecessary magnetic resonance imaging orders of the knee.^20,21 These findings may be interpreted as markers for improved access to care for veterans as well as cost savings for the health care system.

The success of these early initiatives was recognized by the medical leadership of the VHA Simulation Learning, Education and Research Network (SimLEARN), who requested the Mini-Residency course directors to implement a similar educational program at the National Simulation Center in Orlando, Florida. SimLEARN was created to promote best practices in learning and education and provides a high-tech immersive environment for the development and delivery of simulation-based training curricula to facilitate workforce development.²² This article describes the initial experience of the VHA SimLEARN MSK continuing professional development (CPD) training programs, including curriculum design and educational impact on early learners, and how this informed additional CPD needs to continue advancing MSK education and care.

Methods

The initial vision was inspired by the national MSK Mini-Residency initiative for PCPs, which involved 13 US Department of Veterans Affairs (VA) medical centers; its development, dissemination, and validity evidence for assessment methods have been previously described.^17,18,23 SimLEARN leadership attended a Mini-Residency, observing the educational experience and identifying learning objectives most aligned with national goals. The director and codirector of the MSK Mini-Residency (MJB, AMB) then worked with SimLEARN using its educational platform and train-the-trainer model to create a condensed 2-day course, centered on primary care evaluation and management of shoulder and knee pain. The course also included elements supporting educational leaders in providing similar trainings at their local facility (Table 1).

Curriculum was introduced through didactics and reinforced in hands-on sessions enhanced by peer-teaching, arthrocentesis task trainers, and simulated patient experiences. At the end of day 1, participants engaged in critical reflection, reviewing knowledge and skills they had acquired.

On day 2, each participant was evaluated using an observed structured clinical examination (OSCE) for the shoulder, followed by an observed structured teaching experience (OSTE). Given the complexity of the physical examination and the greater potential for appropriate interpretation of clinical findings to influence best practice care, the shoulder was emphasized for these experiences. Time constraints of a 2-day program based on SimLEARN format requirements prevented including an additional OSCE for the knee. At the conclusion of the course, faculty and participants discussed strategies for bringing this educational experience to learners at their local facilities as well as for avoiding potential barriers to implementation. The course was accredited through the VHA Employee Education System (EES), and participants received 16 hours of CPD credit.

Participants

Opportunity to attend was communicated through national, regional, and local VHA organizational networks. Participants self-registered online through the VHA Talent Management System, the main learning resource for VHA employee education, and registration was open to both PCPs and clinician educators. Class size was limited to 10 to facilitate detailed faculty observation during skill acquisition experiences, simulations, and assessment exercises.

Program Evaluation

A standard process for evaluating and measuring learning objectives was performed through VHA EES. Self-assessment surveys and OSCEs were used to assess the activity.

Self-assessment surveys were administered at the beginning and end of the program. Content was adapted from that used in the national MSK Mini-Residency initiative and revised by experts in survey design.^18,24,25 Pre- and postcourse surveys asked participants to rate how important it was for them to be competent in evaluating shoulder and knee pain and in performing related joint injections, as well as to rate their level of confidence in their ability to evaluate and manage these conditions. The survey used 5 construct-specific response options distributed equally on a visual scale. Participants’ learning goals were collected on the precourse survey.

Participants’ competence in performing and interpreting a systematic and thorough physical examination of the shoulder and in suggesting a reasonable plan of management were assessed using a single-station OSCE. This tool, which presented learners with a simulated case depicting rotator cuff pathology, has been described in multiple educational settings, and validity evidence supporting its use has been published.^18,19,23 Course faculty conducted the OSCE, one as the simulated patient, the other as the rater. Immediately following the examination, both faculty conducted a debriefing session with each participant. The OSCE was scored using the validated checklist for specific elements of the shoulder exam, followed by a structured sequence of questions exploring participants’ interpretation of findings, diagnostic impressions, and recommendations for initial management. Scores for participants’ differential diagnosis were based on the completeness and specificity of diagnoses given; scores for management plans were based on appropriateness and accuracy of both the primary and secondary approach to treatment or further diagnostic efforts. A global rating (range 1 to 9) was assigned, independent of scores in other domains.

Following the OSCE, participants rotated through a 3-cycle OSTE where they practiced the roles of simulated patient, learner, and educator. Faculty observed each OSTE and led focused debriefing sessions immediately following each rotation to facilitate participants’ critical reflection of their involvement in these elements of the course. This exercise was formative without quantitative assessment of performance.

Statistical Analysis

Pre- and postsurvey data were analyzed using a paired Student t test. Comparisons between multiple variables (eg, OSCE scores by years of experience or level of credentials) were analyzed using analysis of variance. Relationships between variables were analyzed with a Pearson correlation. All statistical analyses were conducted using IBM SPSS, Version 24 (Armonk, NY).

This project was reviewed by the institutional review board of the University of Utah and the Salt Lake City VA and was determined to be exempt from review because the work did not meet the definition of research with human subjects and was considered a quality improvement study.

Results

Twenty-four participants completed the program over 3 course offerings between February and May 2016, and all completed pre- and postcourse self-assessment surveys (Table 2). Self-ratings of the importance of competence in shoulder and knee MSK skills remained high before and after the course, and confidence improved significantly across all learning objectives. Despite the emphasis on the evaluation and management of shoulder pain, participants’ self-confidence still improved significantly with the knee—though these improvements were generally smaller in scale compared with those of the shoulder.

Overall OSCE scores and scores by domain were not found to be statistically different based on either years of experience or by level of credential or specialty (advanced practice registered nurse/physician assistant, PCP, or specialty care physician)(Table 3). However, there was a trend toward higher performance among the specialty care physician group, and a trend toward lower performance among participants with less than 3 years’ experience.

Discussion

Building on the foundation of other successful innovations in MSK education, the first year of the SimLEARN National MSK Training Program demonstrated the feasibility of a 2-day centralized national course as a method to increase participants’ confidence and competence in evaluating and managing MSK problems, and to disseminate a portable curriculum to a range of clinician educators. Although this course focused on developing competence for shoulder skills, including an OSCE on day 2, self-perceived improvements in participants’ ability to evaluate and manage knee pain were observed. Future program refinement and follow-up of participants’ experience and needs may lead to increased time allocated to the knee exam as well as objective measures of competence for knee skills.

In comparing our findings to the work that others have previously described, we looked for reports of CPD programs in 2 contexts: those that focused on acquisition of MSK skills relevant to clinical practice, and those designed as clinician educator or faculty development initiatives. Although there are few reports of MSK-themed CPD experiences designed specifically for nurses and allied health professionals, a recent effort to survey members of these disciplines in the United Kingdom was an important contribution to a systematic needs assessment.^26-28 Increased support from leadership, mostly in terms of time allowance and budgetary support, was identified as an important driver to facilitate participation in MSK CPD experiences. Through SimLEARN, the VHA is investing in CPD, providing the MSK Training Programs and other courses at no cost to its employees.

Most published reports on physician education have not evaluated content knowledge or physical examination skills with measures for which validity evidence has been published.^19,29,30 One notable exception is the 2000 Canadian Viscosupplementation Injector Preceptor experience, in which Bellamy and colleagues examined patient outcomes in evaluating their program.³¹

Our experience is congruent with the work of Macedo and colleagues and Sturpe and colleagues, who described the effectiveness and acceptability of an OSTE for faculty development.^32,33 These studies emphasize debriefing, a critical element in faculty development identified by Steinert and colleagues in a 2006 best evidence medical education (BEME) review.³⁴ The shoulder OSTE was one of the most well-received elements of our course, and each debrief was critical to facilitating rich discussions between educators and practitioners playing the role of teacher or student during this simulated experience, gaining insight into each other’s perspectives.

This program has several significant strengths: First, this is the most recent step in the development of a portfolio of innovative MSK CPD programs that were envisioned through a systematic process involving projections of cost-effectiveness, local pilot testing, and national expansion.^17,18,35 Second, the SimLEARN program uses assessment tools for which validity evidence has been published, made available for reflective critique by educational scholars.^19,23 This supports a national consortium of MSK educators, advancing clinical teaching and educational scholarship, and creating opportunities for interprofessional collaboration in congruence with the vision expressed in the 2010 Institute of Medicine report, “Redesigning Continuing Education in the Health Professions,” as well as the 2016 update of the BEME recommendations for faculty development.^36,37

Our experience with the SimLEARN National MSK Training Program demonstrates need for 2 distinct courses: (1) the MSK Clinician—serving PCPs seeking to develop their skills in evaluating and managing patients with MSK problems; and (2), the MSK Master Educator—for those with preexisting content expertise who would value the introduction to a national curriculum and connections with other MSK master educators. Both of these are now offered regularly through SimLEARN for VHA and US Department of Defense employees. The MSK Clinician program establishes competence in systematically evaluating and managing shoulder and knee MSK problems in an educational setting and prepares participants for subsequent clinical experiences where they can perform related procedures if desired, under appropriate supervision. The Master Educator program introduces partici pants to the clinician curriculum and provides the opportunity to develop an individualized plan for implementation of an MSK educational program at their home institutions. Participants are selected through a competitive application process, and funding for travel to attend the Master Educator program is provided by SimLEARN for participants who are accepted. Additionally, the Master Educator program serves as a repository for potential future SimLEARN MSK Clinician course faculty.

Limitations

The small number of participants may limit the validity of our conclusions. Although we included an OSCE to measure competence in performing and interpreting the shoulder exam, the durability of these skills is not known. Periodic postcourse OSCEs could help determine this and refresh and preserve accuracy in the performance of specific maneuvers. Second, although this experience was rated highly by participants, we do not know the impact of the program on their daily work or career trajectory. Sustained follow-up of learners, perhaps developed on the model of the Long-Term Career Outcome Study, may increase the value of this experience for future participants.³⁸ This program appealed to a diverse pool of learners, with a broad range of precourse expertise and varied expectations of how course experiences would impact their future work and career development. Some clinical educator attendees came from tertiary care facilities affiliated with academic medical centers, held specialist or subspecialist credentials, and had formal responsibilities as leaders in HPE. Other clinical practitioner participants were solitary PCPs, often in rural or home-based settings; although they may have been eager to apply new knowledge and skills in patient care, they neither anticipated nor desired any role as an educator.

Conclusion

The initial SimLEARN MSK Training Program provides PCPs and clinician educators with rich learning experiences, increasing confidence in addressing MSK problems and competence in performing and interpreting a systematic physical examination of the shoulder. The success of this program has created new opportunities for practitioners seeking to strengthen clinical skills and for leaders in health professions education looking to disseminate similar trainings and connect with a national group of educators.

Acknowledgments

The authors gratefully acknowledge the faculty and staff at the Veterans Health Administration SimLEARN National Simulation Center, the faculty of the Salt Lake City Musculoskeletal Mini-Residency program, the supportive leadership of the George E. Wahlen Salt Lake City Veterans Affairs Medical Center, and the efforts of Danielle Blake for logistical support and data entry.

Alkaptonuria is a rare autosomal recessive disorder uniquely known for causing black, or darkened, urine when left standing due to the renal excretion of excess homogentisic acid (HGA). When this disorder goes undiagnosed, as demonstrated in this case, patients experience its many complications without a unifying explanation. The disorder has 3 clinical stages that occur in a predictable order: clinical silence, clinical ochronosis, and ochronotic arthropathy. These stages lead to multiple musculoskeletal, cardiovascular (CV), and renal complications that can be mitigated with management focused on decreasing homogentisic acid buildup, alleviating symptoms, and close monitoring for these complications.

Case Presentation

A 61-year-old African American male with a medical history of multiple traumatic fractures, right Achilles tendon injury, early-onset multijoint osteoarthritis, chronic low back pain, and recurrent nephrolithiasis presented to the emergency department with sudden onset of sharp left ankle pain while moving furniture. His physical exam revealed a positive Thompson test—lack of foot plantar flexion with calf squeeze—and a subsequent magnetic resonance image (MRI) showed evidence of an acute Achilles tendon rupture.

Given these findings the patient was treated with nonsteroidal anti-inflammatory drugs (NSAIDs) and rest to allow for resolution of swelling and inflammation, followed by elective surgery a month later to repair the ruptured tendon. An operative report following his surgery described “black ends to the area where the Achilles was ruptured…and tendinopathy of the flexor hallucis longus with blackening of the flexor.”

A more in-depth patient history revealed that he underwent multiple invasive and noninvasive interventions for his chronic low back and joint pain with medical management of a prior right Achilles tendon injury. His medical history also included multiple nonspecific diagnoses, such as premature atherosclerosis (diagnosed in his third decade), severe lumbar degenerative disc disease, several tendonopathies and cartilage injuries (Figure 1), pseudogout (following calcium pyrophosphate dehydrate crystals found from a left knee aspirate), and chronic pain syndrome. Along this diagnostic journey, he had several health care providers (HCPs) in rheumatology, orthopedic surgery, pain management, and podiatry who offered a range of symptom management options, including physical therapy, NSAIDs, opioid agonists, tricyclic antidepressants, gabapentin, colchicine, and epidural steroid injections, all of which provided little or no relief of his pain. The patient reported that he told a HCP, “I’ll just live with [the pain].”

At the postsurgery follow-up, the patient reported that he had noticed dark urine and dark spots on his ears in the past. He also recounted that chronic joint pain was common in his family, with both his mother and brother receiving bilateral total knee replacements. Taking into consideration the surgical report and this new history, a urine assessment for HGA was ordered and yielded a diagnosis of alkaptonuria. 

He later suffered an acute myocardial infarction leading to an incidental discovery of severe aortic stenosis on echocardiography, requiring coronary stent placements and transcatheter aortic valve replacement, respectively. He reported that with CV interventions and joint replacement surgeries, including bilateral knees and hips, his symptoms and quality of life began to significantly improve. However, he continued to have diffuse chronic joint pain unimproved with any single agent or intervention.

Discussion

Alkaptonuria is a rare autosomal recessive disorder, with a prevalence of about 1 in 100,000 to 250,000, which results from an enzyme error in an essential amino acid metabolism pathway (Figure 2).¹ This inheritable gene mutation leads to ineffective homogentisate 1,2-dioxygenase (HGD), an enzyme required to break down HGA—which is a product of phenylalanine and tyrosine metabolism.² As these patients engage in normal dietary protein intake, which includes essential amino acid phenylalanine, they develop clinically evident manifestations of the buildup and deposition of HGA.

The rarity of alkaptonuria combined with the gradual buildup of HGA makes it difficult to diagnose. A common diagnostic technique is the visualization of discolored cartilage during surgical procedures, especially when discoloration in urine or skin is not immediately evident. A few case reports have noted surgical diagnosis of black or darkening tissue, known as ochronosis, following tendon rupture—a common complication of this disorder.^3-5 Additional intervention-related case reports linked to the discovery of ochronosis include aortic valve replacement, lumbar discectomy, and bronchoscopy.^6-9 Cases like these illustrate the complex, disabling, and unclear nature of this disorder when not diagnosed early in life.

The patient in this case communicated via e-mail about his tendon repair surgery. “Something very interesting was found during the surgery,” the patient explained. “I was diagnosed with the disease called ochronosis. I don’t know much about this disease but I am beginning to know why some of the things are happening to me and why I am always in constant pain.” This was the first recognized clue toward a diagnosis of alkaptonuria.

Pathophysiology

The pathophysiology of alkaptonuria is based on the extensive deposition of HGA throughout the body. Its progression is based on 3 clinical stages: clinical silence, clinical ochronosis, and ochronotic arthropathy.¹ In the first stage the disorder is asymptomatic but includes its most notable feature—the gradual darkening of urine when exposed to air through oxidation of the renally excreted HGA. A similar process occurs in the blood through formed HGA-melanin compounds, which cause discoloration in cartilage.¹ This internal metabolic disruption accounts for the disorder’s eventual second stage, clinical ochronosis, usually with an onset in the second or third decade. Prominent features noted on physical examination primarily include discoloration of ear pinnae and eye sclera but can involve the nose, gums, teeth, and hands. The third, final, and symptomatic stage, ochronotic arthropathy, occurs by the patient’s fourth to fifth decade and presents as joint pain, usually starting with the vertebrae and larger joints like hips, knees, and shoulders, that can appear as advanced early osteoarthritis on imaging.

Treatment

This clinical manifestation of alkaptonuria requires that HCPs manage patients with 3 strategies: decrease HGA buildup, alleviate symptoms, and monitor for disorder complications. Decreasing HGA buildup is a difficult aspect of management given the natural physiology of protein intake and metabolism. Three approaches to limit HGA buildup incorporate decreasing protein intake, inhibiting enzyme production of HGA, and increasing HGA excretion. Phenylalanine is an essential amino acid—meaning its levels are dependent on dietary protein intake. Patients should be advised to adhere to a low protein diet, especially phenylalanine and tyrosine, to lessen HGA concentrations.

Manipulating the metabolic pathway of phenylalanine with medication is a second option. An example of this is nitisinone, a US Food and Drug Administration-approved medication for treatment of tyrosinemia. It acts by inhibiting hydroxyphenylpyruvic acid dioxygenase, one of the enzymes that converts tyrosine into HGA, to prevent the buildup of damaging tyrosine byproducts. At low doses it has been effective in decreasing HGA concentrations in alkaptonuria and tyrosinemia.^10,11 Due to this mechanism of action, nitisinone directly causes increased tyrosine levels. Therefore, tyrosine toxicity, usually not predicted by tyrosine levels, has been associated with eye-related adverse effects (AEs), including keratopathy, diminished visual acuity, and corneal tissue damage.^1,2,10 Incidence of these AEs have not been clearly documented, but routine monitoring should include patient education on ocular symptoms and slit-lamp examinations.¹²

In addition, case reports have shown that high-dose ascorbic acid (vitamin C) promotes HGA, tyrosine, and phenylalanine excretion in urine, which may slow the progression of alkaptonuria, but clinical effect has not been proven.¹³ Additionally, high vitamin C intake is considered a risk factor for nephrolithiasis, which must be balanced with the increased risk of stone formation from HGA excretion.¹⁴ These dietary and medical options can be considered, especially in the setting of severe symptoms or complications, but the risks must be discussed with patients.

A second and commonly utilized strategy for caring for these patients is symptom management. As demonstrated through this case report, there is no clear medication that adequately addresses the pain caused by HGA deposition. Patients should be referred to a pain specialist to allow for single provider prescribing of pain medications. This patient found most relief and least AEs with tramadol but eventually self-discontinued due to diminishing pain relief. Given the eventual involvement of large joints, these patients will often require further symptom management with joint replacement surgery, usually much earlier than patients who undergo these surgeries for age-related osteoarthritis. The imperative aspect of symptom management is to engage patients in shared decision making with clear expectation setting.

Given the progressive nature of alkaptonuria, providers must monitor and address complications that are a result of this disorder. HGA becomes pathologic by binding to and weakening collagen fibers.⁵ This gradual buildup leads to degenerative changes in weight-bearing lower vertebrae and large joints that can become severe. Due to HGA’s interaction with collagen fibers, tendon involvement leading to inflammation, calcification, and rupture can result as patients enter the third stage, ochronotic arthropathy, of the disorder (Figure 3).¹⁵ Many of these arthropathies will require medical and surgical management and can be urgent in situations like tendon ruptures and meniscal tears. Understanding the pathophysiology of tendinopathies in patients with alkaptonuria also can aid orthopedic surgeons during the postoperative period where patients may be at risk for poor healing.⁵

A second area of complications includes CV involvement. This patient was diagnosed with premature atherosclerosis and underwent cardiac interventions, including coronary stent placement and valve replacements at age 63 years. This early cardiac involvement was likely due in part to the deposition of HGA and collagen injury in CV tissue leading to damage of the endocardium, aortic intima, heart valves, and coronary arteries.¹ HCPs should monitor for these manifestations with regular visits, chest computed tomography, and echocardiographic studies.²

The most classic aspect of this rare disorder is urine darkening due to the renal excretion of HGA and comprises the third area of complications. This process leads to chronically acidic urine—every urinalysis in this patient’s chart displayed the lowest pH measurable—and an increased risk for calcification and precipitation of solutes within the kidney and urinary tract (Figure 4). Both X-ray and ultrasound imaging should be used to identify kidney and prostate stones in the setting of abdominal or genitourinary pain or infection. Patients with diminished renal function may manifest a more severe and rapidly progressing form of alkaptonuria that exacerbates symptoms and complications, but direct damage to the kidneys by HGA is not evident.

Conclusion

Alkaptonuria is a rare autosomal recessive metabolic disorder that has a progressively debilitating pathophysiologic course spanning decades of a patient’s life. Its low prevalence and gradually progressive nature make it a difficult diagnosis to make without clinical suspicion. In patients with early-onset degenerative joint disease, tendinopathy, and cartilage or skin discoloration, congenital metabolic disorders like alkaptonuria should be considered.

As this case shows, suspicion and diagnosis can occur during surgical intervention in which tendon discoloration is directly visualized, especially in patients without prominent skin or cartilage discoloration. Once the diagnosis is made through elevated levels of urine HGA, there are 3 management strategies, including decreasing homogentisic acid buildup, providing symptom management, and monitoring for arthropathic, CV, and genitourinary complications.

Alkaptonuria is a rare autosomal recessive disorder uniquely known for causing black, or darkened, urine when left standing due to the renal excretion of excess homogentisic acid (HGA). When this disorder goes undiagnosed, as demonstrated in this case, patients experience its many complications without a unifying explanation. The disorder has 3 clinical stages that occur in a predictable order: clinical silence, clinical ochronosis, and ochronotic arthropathy. These stages lead to multiple musculoskeletal, cardiovascular (CV), and renal complications that can be mitigated with management focused on decreasing homogentisic acid buildup, alleviating symptoms, and close monitoring for these complications.

Case Presentation

A 61-year-old African American male with a medical history of multiple traumatic fractures, right Achilles tendon injury, early-onset multijoint osteoarthritis, chronic low back pain, and recurrent nephrolithiasis presented to the emergency department with sudden onset of sharp left ankle pain while moving furniture. His physical exam revealed a positive Thompson test—lack of foot plantar flexion with calf squeeze—and a subsequent magnetic resonance image (MRI) showed evidence of an acute Achilles tendon rupture.

Given these findings the patient was treated with nonsteroidal anti-inflammatory drugs (NSAIDs) and rest to allow for resolution of swelling and inflammation, followed by elective surgery a month later to repair the ruptured tendon. An operative report following his surgery described “black ends to the area where the Achilles was ruptured…and tendinopathy of the flexor hallucis longus with blackening of the flexor.”

A more in-depth patient history revealed that he underwent multiple invasive and noninvasive interventions for his chronic low back and joint pain with medical management of a prior right Achilles tendon injury. His medical history also included multiple nonspecific diagnoses, such as premature atherosclerosis (diagnosed in his third decade), severe lumbar degenerative disc disease, several tendonopathies and cartilage injuries (Figure 1), pseudogout (following calcium pyrophosphate dehydrate crystals found from a left knee aspirate), and chronic pain syndrome. Along this diagnostic journey, he had several health care providers (HCPs) in rheumatology, orthopedic surgery, pain management, and podiatry who offered a range of symptom management options, including physical therapy, NSAIDs, opioid agonists, tricyclic antidepressants, gabapentin, colchicine, and epidural steroid injections, all of which provided little or no relief of his pain. The patient reported that he told a HCP, “I’ll just live with [the pain].”

At the postsurgery follow-up, the patient reported that he had noticed dark urine and dark spots on his ears in the past. He also recounted that chronic joint pain was common in his family, with both his mother and brother receiving bilateral total knee replacements. Taking into consideration the surgical report and this new history, a urine assessment for HGA was ordered and yielded a diagnosis of alkaptonuria. 

He later suffered an acute myocardial infarction leading to an incidental discovery of severe aortic stenosis on echocardiography, requiring coronary stent placements and transcatheter aortic valve replacement, respectively. He reported that with CV interventions and joint replacement surgeries, including bilateral knees and hips, his symptoms and quality of life began to significantly improve. However, he continued to have diffuse chronic joint pain unimproved with any single agent or intervention.

Discussion

Alkaptonuria is a rare autosomal recessive disorder, with a prevalence of about 1 in 100,000 to 250,000, which results from an enzyme error in an essential amino acid metabolism pathway (Figure 2).¹ This inheritable gene mutation leads to ineffective homogentisate 1,2-dioxygenase (HGD), an enzyme required to break down HGA—which is a product of phenylalanine and tyrosine metabolism.² As these patients engage in normal dietary protein intake, which includes essential amino acid phenylalanine, they develop clinically evident manifestations of the buildup and deposition of HGA.

The rarity of alkaptonuria combined with the gradual buildup of HGA makes it difficult to diagnose. A common diagnostic technique is the visualization of discolored cartilage during surgical procedures, especially when discoloration in urine or skin is not immediately evident. A few case reports have noted surgical diagnosis of black or darkening tissue, known as ochronosis, following tendon rupture—a common complication of this disorder.^3-5 Additional intervention-related case reports linked to the discovery of ochronosis include aortic valve replacement, lumbar discectomy, and bronchoscopy.^6-9 Cases like these illustrate the complex, disabling, and unclear nature of this disorder when not diagnosed early in life.

The patient in this case communicated via e-mail about his tendon repair surgery. “Something very interesting was found during the surgery,” the patient explained. “I was diagnosed with the disease called ochronosis. I don’t know much about this disease but I am beginning to know why some of the things are happening to me and why I am always in constant pain.” This was the first recognized clue toward a diagnosis of alkaptonuria.

Pathophysiology

The pathophysiology of alkaptonuria is based on the extensive deposition of HGA throughout the body. Its progression is based on 3 clinical stages: clinical silence, clinical ochronosis, and ochronotic arthropathy.¹ In the first stage the disorder is asymptomatic but includes its most notable feature—the gradual darkening of urine when exposed to air through oxidation of the renally excreted HGA. A similar process occurs in the blood through formed HGA-melanin compounds, which cause discoloration in cartilage.¹ This internal metabolic disruption accounts for the disorder’s eventual second stage, clinical ochronosis, usually with an onset in the second or third decade. Prominent features noted on physical examination primarily include discoloration of ear pinnae and eye sclera but can involve the nose, gums, teeth, and hands. The third, final, and symptomatic stage, ochronotic arthropathy, occurs by the patient’s fourth to fifth decade and presents as joint pain, usually starting with the vertebrae and larger joints like hips, knees, and shoulders, that can appear as advanced early osteoarthritis on imaging.

Treatment

This clinical manifestation of alkaptonuria requires that HCPs manage patients with 3 strategies: decrease HGA buildup, alleviate symptoms, and monitor for disorder complications. Decreasing HGA buildup is a difficult aspect of management given the natural physiology of protein intake and metabolism. Three approaches to limit HGA buildup incorporate decreasing protein intake, inhibiting enzyme production of HGA, and increasing HGA excretion. Phenylalanine is an essential amino acid—meaning its levels are dependent on dietary protein intake. Patients should be advised to adhere to a low protein diet, especially phenylalanine and tyrosine, to lessen HGA concentrations.

Manipulating the metabolic pathway of phenylalanine with medication is a second option. An example of this is nitisinone, a US Food and Drug Administration-approved medication for treatment of tyrosinemia. It acts by inhibiting hydroxyphenylpyruvic acid dioxygenase, one of the enzymes that converts tyrosine into HGA, to prevent the buildup of damaging tyrosine byproducts. At low doses it has been effective in decreasing HGA concentrations in alkaptonuria and tyrosinemia.^10,11 Due to this mechanism of action, nitisinone directly causes increased tyrosine levels. Therefore, tyrosine toxicity, usually not predicted by tyrosine levels, has been associated with eye-related adverse effects (AEs), including keratopathy, diminished visual acuity, and corneal tissue damage.^1,2,10 Incidence of these AEs have not been clearly documented, but routine monitoring should include patient education on ocular symptoms and slit-lamp examinations.¹²

In addition, case reports have shown that high-dose ascorbic acid (vitamin C) promotes HGA, tyrosine, and phenylalanine excretion in urine, which may slow the progression of alkaptonuria, but clinical effect has not been proven.¹³ Additionally, high vitamin C intake is considered a risk factor for nephrolithiasis, which must be balanced with the increased risk of stone formation from HGA excretion.¹⁴ These dietary and medical options can be considered, especially in the setting of severe symptoms or complications, but the risks must be discussed with patients.

A second and commonly utilized strategy for caring for these patients is symptom management. As demonstrated through this case report, there is no clear medication that adequately addresses the pain caused by HGA deposition. Patients should be referred to a pain specialist to allow for single provider prescribing of pain medications. This patient found most relief and least AEs with tramadol but eventually self-discontinued due to diminishing pain relief. Given the eventual involvement of large joints, these patients will often require further symptom management with joint replacement surgery, usually much earlier than patients who undergo these surgeries for age-related osteoarthritis. The imperative aspect of symptom management is to engage patients in shared decision making with clear expectation setting.

Given the progressive nature of alkaptonuria, providers must monitor and address complications that are a result of this disorder. HGA becomes pathologic by binding to and weakening collagen fibers.⁵ This gradual buildup leads to degenerative changes in weight-bearing lower vertebrae and large joints that can become severe. Due to HGA’s interaction with collagen fibers, tendon involvement leading to inflammation, calcification, and rupture can result as patients enter the third stage, ochronotic arthropathy, of the disorder (Figure 3).¹⁵ Many of these arthropathies will require medical and surgical management and can be urgent in situations like tendon ruptures and meniscal tears. Understanding the pathophysiology of tendinopathies in patients with alkaptonuria also can aid orthopedic surgeons during the postoperative period where patients may be at risk for poor healing.⁵

A second area of complications includes CV involvement. This patient was diagnosed with premature atherosclerosis and underwent cardiac interventions, including coronary stent placement and valve replacements at age 63 years. This early cardiac involvement was likely due in part to the deposition of HGA and collagen injury in CV tissue leading to damage of the endocardium, aortic intima, heart valves, and coronary arteries.¹ HCPs should monitor for these manifestations with regular visits, chest computed tomography, and echocardiographic studies.²

The most classic aspect of this rare disorder is urine darkening due to the renal excretion of HGA and comprises the third area of complications. This process leads to chronically acidic urine—every urinalysis in this patient’s chart displayed the lowest pH measurable—and an increased risk for calcification and precipitation of solutes within the kidney and urinary tract (Figure 4). Both X-ray and ultrasound imaging should be used to identify kidney and prostate stones in the setting of abdominal or genitourinary pain or infection. Patients with diminished renal function may manifest a more severe and rapidly progressing form of alkaptonuria that exacerbates symptoms and complications, but direct damage to the kidneys by HGA is not evident.

Conclusion

Alkaptonuria is a rare autosomal recessive metabolic disorder that has a progressively debilitating pathophysiologic course spanning decades of a patient’s life. Its low prevalence and gradually progressive nature make it a difficult diagnosis to make without clinical suspicion. In patients with early-onset degenerative joint disease, tendinopathy, and cartilage or skin discoloration, congenital metabolic disorders like alkaptonuria should be considered.

As this case shows, suspicion and diagnosis can occur during surgical intervention in which tendon discoloration is directly visualized, especially in patients without prominent skin or cartilage discoloration. Once the diagnosis is made through elevated levels of urine HGA, there are 3 management strategies, including decreasing homogentisic acid buildup, providing symptom management, and monitoring for arthropathic, CV, and genitourinary complications.

Alkaptonuria is a rare autosomal recessive disorder uniquely known for causing black, or darkened, urine when left standing due to the renal excretion of excess homogentisic acid (HGA). When this disorder goes undiagnosed, as demonstrated in this case, patients experience its many complications without a unifying explanation. The disorder has 3 clinical stages that occur in a predictable order: clinical silence, clinical ochronosis, and ochronotic arthropathy. These stages lead to multiple musculoskeletal, cardiovascular (CV), and renal complications that can be mitigated with management focused on decreasing homogentisic acid buildup, alleviating symptoms, and close monitoring for these complications.

Case Presentation

A 61-year-old African American male with a medical history of multiple traumatic fractures, right Achilles tendon injury, early-onset multijoint osteoarthritis, chronic low back pain, and recurrent nephrolithiasis presented to the emergency department with sudden onset of sharp left ankle pain while moving furniture. His physical exam revealed a positive Thompson test—lack of foot plantar flexion with calf squeeze—and a subsequent magnetic resonance image (MRI) showed evidence of an acute Achilles tendon rupture.

Given these findings the patient was treated with nonsteroidal anti-inflammatory drugs (NSAIDs) and rest to allow for resolution of swelling and inflammation, followed by elective surgery a month later to repair the ruptured tendon. An operative report following his surgery described “black ends to the area where the Achilles was ruptured…and tendinopathy of the flexor hallucis longus with blackening of the flexor.”

A more in-depth patient history revealed that he underwent multiple invasive and noninvasive interventions for his chronic low back and joint pain with medical management of a prior right Achilles tendon injury. His medical history also included multiple nonspecific diagnoses, such as premature atherosclerosis (diagnosed in his third decade), severe lumbar degenerative disc disease, several tendonopathies and cartilage injuries (Figure 1), pseudogout (following calcium pyrophosphate dehydrate crystals found from a left knee aspirate), and chronic pain syndrome. Along this diagnostic journey, he had several health care providers (HCPs) in rheumatology, orthopedic surgery, pain management, and podiatry who offered a range of symptom management options, including physical therapy, NSAIDs, opioid agonists, tricyclic antidepressants, gabapentin, colchicine, and epidural steroid injections, all of which provided little or no relief of his pain. The patient reported that he told a HCP, “I’ll just live with [the pain].”

At the postsurgery follow-up, the patient reported that he had noticed dark urine and dark spots on his ears in the past. He also recounted that chronic joint pain was common in his family, with both his mother and brother receiving bilateral total knee replacements. Taking into consideration the surgical report and this new history, a urine assessment for HGA was ordered and yielded a diagnosis of alkaptonuria. 

He later suffered an acute myocardial infarction leading to an incidental discovery of severe aortic stenosis on echocardiography, requiring coronary stent placements and transcatheter aortic valve replacement, respectively. He reported that with CV interventions and joint replacement surgeries, including bilateral knees and hips, his symptoms and quality of life began to significantly improve. However, he continued to have diffuse chronic joint pain unimproved with any single agent or intervention.

Discussion

Alkaptonuria is a rare autosomal recessive disorder, with a prevalence of about 1 in 100,000 to 250,000, which results from an enzyme error in an essential amino acid metabolism pathway (Figure 2).¹ This inheritable gene mutation leads to ineffective homogentisate 1,2-dioxygenase (HGD), an enzyme required to break down HGA—which is a product of phenylalanine and tyrosine metabolism.² As these patients engage in normal dietary protein intake, which includes essential amino acid phenylalanine, they develop clinically evident manifestations of the buildup and deposition of HGA.

The rarity of alkaptonuria combined with the gradual buildup of HGA makes it difficult to diagnose. A common diagnostic technique is the visualization of discolored cartilage during surgical procedures, especially when discoloration in urine or skin is not immediately evident. A few case reports have noted surgical diagnosis of black or darkening tissue, known as ochronosis, following tendon rupture—a common complication of this disorder.^3-5 Additional intervention-related case reports linked to the discovery of ochronosis include aortic valve replacement, lumbar discectomy, and bronchoscopy.^6-9 Cases like these illustrate the complex, disabling, and unclear nature of this disorder when not diagnosed early in life.

The patient in this case communicated via e-mail about his tendon repair surgery. “Something very interesting was found during the surgery,” the patient explained. “I was diagnosed with the disease called ochronosis. I don’t know much about this disease but I am beginning to know why some of the things are happening to me and why I am always in constant pain.” This was the first recognized clue toward a diagnosis of alkaptonuria.

Pathophysiology

The pathophysiology of alkaptonuria is based on the extensive deposition of HGA throughout the body. Its progression is based on 3 clinical stages: clinical silence, clinical ochronosis, and ochronotic arthropathy.¹ In the first stage the disorder is asymptomatic but includes its most notable feature—the gradual darkening of urine when exposed to air through oxidation of the renally excreted HGA. A similar process occurs in the blood through formed HGA-melanin compounds, which cause discoloration in cartilage.¹ This internal metabolic disruption accounts for the disorder’s eventual second stage, clinical ochronosis, usually with an onset in the second or third decade. Prominent features noted on physical examination primarily include discoloration of ear pinnae and eye sclera but can involve the nose, gums, teeth, and hands. The third, final, and symptomatic stage, ochronotic arthropathy, occurs by the patient’s fourth to fifth decade and presents as joint pain, usually starting with the vertebrae and larger joints like hips, knees, and shoulders, that can appear as advanced early osteoarthritis on imaging.

Treatment

This clinical manifestation of alkaptonuria requires that HCPs manage patients with 3 strategies: decrease HGA buildup, alleviate symptoms, and monitor for disorder complications. Decreasing HGA buildup is a difficult aspect of management given the natural physiology of protein intake and metabolism. Three approaches to limit HGA buildup incorporate decreasing protein intake, inhibiting enzyme production of HGA, and increasing HGA excretion. Phenylalanine is an essential amino acid—meaning its levels are dependent on dietary protein intake. Patients should be advised to adhere to a low protein diet, especially phenylalanine and tyrosine, to lessen HGA concentrations.

Manipulating the metabolic pathway of phenylalanine with medication is a second option. An example of this is nitisinone, a US Food and Drug Administration-approved medication for treatment of tyrosinemia. It acts by inhibiting hydroxyphenylpyruvic acid dioxygenase, one of the enzymes that converts tyrosine into HGA, to prevent the buildup of damaging tyrosine byproducts. At low doses it has been effective in decreasing HGA concentrations in alkaptonuria and tyrosinemia.^10,11 Due to this mechanism of action, nitisinone directly causes increased tyrosine levels. Therefore, tyrosine toxicity, usually not predicted by tyrosine levels, has been associated with eye-related adverse effects (AEs), including keratopathy, diminished visual acuity, and corneal tissue damage.^1,2,10 Incidence of these AEs have not been clearly documented, but routine monitoring should include patient education on ocular symptoms and slit-lamp examinations.¹²

In addition, case reports have shown that high-dose ascorbic acid (vitamin C) promotes HGA, tyrosine, and phenylalanine excretion in urine, which may slow the progression of alkaptonuria, but clinical effect has not been proven.¹³ Additionally, high vitamin C intake is considered a risk factor for nephrolithiasis, which must be balanced with the increased risk of stone formation from HGA excretion.¹⁴ These dietary and medical options can be considered, especially in the setting of severe symptoms or complications, but the risks must be discussed with patients.

A second and commonly utilized strategy for caring for these patients is symptom management. As demonstrated through this case report, there is no clear medication that adequately addresses the pain caused by HGA deposition. Patients should be referred to a pain specialist to allow for single provider prescribing of pain medications. This patient found most relief and least AEs with tramadol but eventually self-discontinued due to diminishing pain relief. Given the eventual involvement of large joints, these patients will often require further symptom management with joint replacement surgery, usually much earlier than patients who undergo these surgeries for age-related osteoarthritis. The imperative aspect of symptom management is to engage patients in shared decision making with clear expectation setting.

Given the progressive nature of alkaptonuria, providers must monitor and address complications that are a result of this disorder. HGA becomes pathologic by binding to and weakening collagen fibers.⁵ This gradual buildup leads to degenerative changes in weight-bearing lower vertebrae and large joints that can become severe. Due to HGA’s interaction with collagen fibers, tendon involvement leading to inflammation, calcification, and rupture can result as patients enter the third stage, ochronotic arthropathy, of the disorder (Figure 3).¹⁵ Many of these arthropathies will require medical and surgical management and can be urgent in situations like tendon ruptures and meniscal tears. Understanding the pathophysiology of tendinopathies in patients with alkaptonuria also can aid orthopedic surgeons during the postoperative period where patients may be at risk for poor healing.⁵

A second area of complications includes CV involvement. This patient was diagnosed with premature atherosclerosis and underwent cardiac interventions, including coronary stent placement and valve replacements at age 63 years. This early cardiac involvement was likely due in part to the deposition of HGA and collagen injury in CV tissue leading to damage of the endocardium, aortic intima, heart valves, and coronary arteries.¹ HCPs should monitor for these manifestations with regular visits, chest computed tomography, and echocardiographic studies.²

The most classic aspect of this rare disorder is urine darkening due to the renal excretion of HGA and comprises the third area of complications. This process leads to chronically acidic urine—every urinalysis in this patient’s chart displayed the lowest pH measurable—and an increased risk for calcification and precipitation of solutes within the kidney and urinary tract (Figure 4). Both X-ray and ultrasound imaging should be used to identify kidney and prostate stones in the setting of abdominal or genitourinary pain or infection. Patients with diminished renal function may manifest a more severe and rapidly progressing form of alkaptonuria that exacerbates symptoms and complications, but direct damage to the kidneys by HGA is not evident.

Conclusion

Alkaptonuria is a rare autosomal recessive metabolic disorder that has a progressively debilitating pathophysiologic course spanning decades of a patient’s life. Its low prevalence and gradually progressive nature make it a difficult diagnosis to make without clinical suspicion. In patients with early-onset degenerative joint disease, tendinopathy, and cartilage or skin discoloration, congenital metabolic disorders like alkaptonuria should be considered.

As this case shows, suspicion and diagnosis can occur during surgical intervention in which tendon discoloration is directly visualized, especially in patients without prominent skin or cartilage discoloration. Once the diagnosis is made through elevated levels of urine HGA, there are 3 management strategies, including decreasing homogentisic acid buildup, providing symptom management, and monitoring for arthropathic, CV, and genitourinary complications.

Case Presentation. A 69-year-old veteran presented with an intermittent, waxing and waning cough. He had never smoked and had no family history of lung cancer. His primary care physician ordered a chest radiograph, which revealed a nodular opacity within the lingula concerning for a parenchymal nodule. Further characterization with a chest computed tomography (CT) demonstrated a 1.4-cm left upper lobe subpleural nodule with small satellite nodules (Figure 1). Given these imaging findings, the patient was referred to the pulmonary clinic.

►Renda Wiener, MD, Pulmonary and Critical Care, VABHS, and Assistant Professor of Medicine, Boston University School of Medicine. Pulmonary nodules are well-defined lesions < 3 cm in diameter that are surrounded by lung parenchyma. Although cancer is a possibility (including primary lung cancers, metastatic cancers, or carcinoid tumors), most small nodules do not turn out to be malignant.¹ Benign etiologies include infections, benign tumors, vascular malformations, and inflammatory conditions. Infectious causes of nodules are often granulomatous in nature, including fungi, Mycobacterium tuberculosis, and nontuberculous mycobacteria. Benign tumors are most commonly hamartomas, and these may be clearly distinguished based on imaging characteristics. Pulmonary arteriovenous malformations, hematomas, and infarcts may present as nodules as well. Inflammatory causes of nodules are important and relatively common, including granulomatosis with polyangiitis, rheumatoid arthritis, sarcoidosis, amyloidosis, and rounded atelectasis. 

To distinguish benign from malignant etiologies, we look for several features of pulmonary nodules on imaging. Larger size, irregular borders, and upper lobe location all increase the likelihood of cancer, whereas solid attenuation and calcification make cancer less likely. One of the most reassuring findings that suggests a benign etiology is lack of growth over a period of surveillance; after 2 years without growth we typically consider a nodule benign.¹ And of course, we also consider the patient’s symptoms and risk factors: weight loss, hemoptysis, a history of cigarette smoking or asbestos exposure, or family history of cancer all increase the likelihood of malignancy.

►Dr. Kearney. Given that the differential diagnosis is so broad, how do you think about the next step in evaluating a pulmonary nodule? How do you approach shared decision making with the patient? 

►Dr. Wiener. The characteristics of the patient, the nodule, and the circumstances in which the nodule were discovered are all important to consider. Incidental pulmonary nodules are often found on chest imaging. The imaging characteristics of the nodule are important, as are the patient’s risk factors. A similarly appearing nodule can have very different implications if the patient is a never-smoker exposed to endemic fungi, or a long-time smoker enrolled in a lung cancer screening program. Consultation with a pulmonologist is often appropriate.

It’s important to note that we lack high-quality evidence on the optimal strategy to evaluate pulmonary nodules, and there is no single “right answer“ for all patients. For patients with a low risk of malignancy (< 5%-10%)—which comprises the majority of the incidental nodules discovered—we typically favor serial CT surveillance of the nodule over a period of a few years, whereas for patients at high risk of malignancy (> 65%), we favor early surgical resection if the patient is able to tolerate that. For patients with an intermediate risk of malignancy (~5%-65%), we might consider serial CT surveillance, positron emission tomography (PET) scan, or biopsy.¹ The American College of Chest Physicians guidelines for pulmonary nodule evaluation recommend discussing with patients the different options and the trade-offs of these options in a shared decision-making process.¹

►Dr. Kearney. The patient’s pulmonologist laid out options, including monitoring with serial CT scans, obtaining a PET scan, performing CT-guided needle biopsy, or referring for surgical excision. In this case, the patient elected to undergo CT-guided needle biopsy. Dr. Huang, can you discuss the pathology results?

►Qin Huang, MD, Pathology and Laboratory Medicine, VABHS, and Assistant Professor of Pathology, Harvard Medical School (HMS). The microscopic examination of the needle biopsy of the lung mass revealed rare clusters of atypical cells with crushed cells adjacent to an extensive area of necrosis with scarring. The atypical cells were suspicious for carcinoma. The Gomori methenamine silver (GMS) and periodic acid-Schiff (PAS) stains were negative for common bacterial and fungal microorganisms.

►Dr. Kearney. The tumor board, pulmonologist, and patient decide to move forward with video-assisted excisional biopsy with lymphadenectomy. Dr. Huang, can you interpret the pathology?

►Dr. Huang. Figure 2 showed an hemotoxylin and eosin (H&E)-stained lung resection tissue section with multiple caseating necrotic granulomas. No foreign bodies were identified. There was no evidence of malignancy. The GMS stain revealed a fungal microorganism oval with morphology typical of histoplasma capsulatum (Figure 3).

Pulmonary nodules are common incidental finding on chest imaging of patients who reside in histoplasmosis endemic regions, and they are often hard to differentiate from malignancies. There are 3 mediastinal manifestations: adenitis, granuloma, and fibrosis. Usually the syndromes are subclinical, but occasionally the nodes cause symptoms by impinging on other structures.²

This patient had a solitary pulmonary nodule with none of the associated features mentioned above. Pathology showed caseating granuloma and confirmed histoplasmosis.

►Dr. Kearney. Given the diagnosis of solitary histoplasmoma, how should this patient be managed?

►Dr. Strymish. The optimal therapy for histoplasmosis depends on the patient’s clinical syndrome. Most infections are self-limited and require no therapy. However, patients who are immunocompromised, exposed to large inoculum, and have progressive disease require antifungal treatment, usually with itraconazole for mild-to-moderate disease and a combination of azole therapy and amphotericin B with extensive disease. Patients with few solitary pulmonary nodules do not benefit from antifungal therapy as the nodule could represent quiescent disease that is unlikely to have clinical impact; in this case, the treatment would be higher risk than the nodule.³

^►Dr. Kearney. While the discussion of the diagnosis is interesting, it is also important to acknowledge what the patient went through to arrive at this, an essentially benign diagnosis: 8 months, multiple imaging studies, and 2 invasive diagnostic procedures. Further, the patient had to grapple with the possibility of a diagnosis of cancer. Dr. Wiener, can you talk about the challenges in communicating with patients about pulmonary nodules when cancer is on the differential? What are some of the harms patients face and how can clinicians work to mitigate these harms?

►Dr. Wiener. My colleague Dr. Christopher Slatore of the Portland VA Medical Center and I studied communication about pulmonary nodules in a series of surveys and qualitative studies of patients with pulmonary nodules and the clinicians who take care of them. We found that there seems to be a disconnect between patients’ perceptions of pulmonary nodules and their clinicians, often due to inadequate communication about the nodule. Many clinicians indicated that they do not tell patients about the chance that a nodule may be cancer, because the clinicians know that cancer is unlikely (< 5% of incidentally detected pulmonary nodules turn out to be malignant), and they do not want to alarm patients unnecessarily. However, we found that patients almost immediately wondered about cancer when they learned about their pulmonary nodule, and without hearing explicitly from their clinician that cancer was unlikely, patients tended to overestimate the likelihood of a malignant nodule. Moreover, patients often were not told much about the evaluation plan for the nodule or the rationale for CT surveillance of small nodules instead of biopsy. This uncertainty about the risk of cancer and the plan for evaluating the nodule was difficult for some patients to live with; we found that about one-quarter of patients with a small pulmonary nodule experienced mild-moderate distress during the period of radiographic surveillance. Reassuringly, high-quality patient-clinician communication was associated with lower distress and higher adherence to pulmonary nodule evaluation.⁴

►Dr. Kearney. The patient was educated about his diagnosis of solitary histoplasmoma. Given that the patient was otherwise well appearing with no complicating factors, he was not treated with antifungal therapy. After an 8-month-long workup, the patient was relieved to receive a diagnosis that excluded cancer and did not require any further treatment. His case provides a good example of how to proceed in the workup of a solitary pulmonary nodule and on the importance of communication and shared decision making with our patients.

Case Presentation. A 69-year-old veteran presented with an intermittent, waxing and waning cough. He had never smoked and had no family history of lung cancer. His primary care physician ordered a chest radiograph, which revealed a nodular opacity within the lingula concerning for a parenchymal nodule. Further characterization with a chest computed tomography (CT) demonstrated a 1.4-cm left upper lobe subpleural nodule with small satellite nodules (Figure 1). Given these imaging findings, the patient was referred to the pulmonary clinic.

►Renda Wiener, MD, Pulmonary and Critical Care, VABHS, and Assistant Professor of Medicine, Boston University School of Medicine. Pulmonary nodules are well-defined lesions < 3 cm in diameter that are surrounded by lung parenchyma. Although cancer is a possibility (including primary lung cancers, metastatic cancers, or carcinoid tumors), most small nodules do not turn out to be malignant.¹ Benign etiologies include infections, benign tumors, vascular malformations, and inflammatory conditions. Infectious causes of nodules are often granulomatous in nature, including fungi, Mycobacterium tuberculosis, and nontuberculous mycobacteria. Benign tumors are most commonly hamartomas, and these may be clearly distinguished based on imaging characteristics. Pulmonary arteriovenous malformations, hematomas, and infarcts may present as nodules as well. Inflammatory causes of nodules are important and relatively common, including granulomatosis with polyangiitis, rheumatoid arthritis, sarcoidosis, amyloidosis, and rounded atelectasis. 

To distinguish benign from malignant etiologies, we look for several features of pulmonary nodules on imaging. Larger size, irregular borders, and upper lobe location all increase the likelihood of cancer, whereas solid attenuation and calcification make cancer less likely. One of the most reassuring findings that suggests a benign etiology is lack of growth over a period of surveillance; after 2 years without growth we typically consider a nodule benign.¹ And of course, we also consider the patient’s symptoms and risk factors: weight loss, hemoptysis, a history of cigarette smoking or asbestos exposure, or family history of cancer all increase the likelihood of malignancy.

►Dr. Kearney. Given that the differential diagnosis is so broad, how do you think about the next step in evaluating a pulmonary nodule? How do you approach shared decision making with the patient? 

►Dr. Wiener. The characteristics of the patient, the nodule, and the circumstances in which the nodule were discovered are all important to consider. Incidental pulmonary nodules are often found on chest imaging. The imaging characteristics of the nodule are important, as are the patient’s risk factors. A similarly appearing nodule can have very different implications if the patient is a never-smoker exposed to endemic fungi, or a long-time smoker enrolled in a lung cancer screening program. Consultation with a pulmonologist is often appropriate.

It’s important to note that we lack high-quality evidence on the optimal strategy to evaluate pulmonary nodules, and there is no single “right answer“ for all patients. For patients with a low risk of malignancy (< 5%-10%)—which comprises the majority of the incidental nodules discovered—we typically favor serial CT surveillance of the nodule over a period of a few years, whereas for patients at high risk of malignancy (> 65%), we favor early surgical resection if the patient is able to tolerate that. For patients with an intermediate risk of malignancy (~5%-65%), we might consider serial CT surveillance, positron emission tomography (PET) scan, or biopsy.¹ The American College of Chest Physicians guidelines for pulmonary nodule evaluation recommend discussing with patients the different options and the trade-offs of these options in a shared decision-making process.¹

►Dr. Kearney. The patient’s pulmonologist laid out options, including monitoring with serial CT scans, obtaining a PET scan, performing CT-guided needle biopsy, or referring for surgical excision. In this case, the patient elected to undergo CT-guided needle biopsy. Dr. Huang, can you discuss the pathology results?

►Qin Huang, MD, Pathology and Laboratory Medicine, VABHS, and Assistant Professor of Pathology, Harvard Medical School (HMS). The microscopic examination of the needle biopsy of the lung mass revealed rare clusters of atypical cells with crushed cells adjacent to an extensive area of necrosis with scarring. The atypical cells were suspicious for carcinoma. The Gomori methenamine silver (GMS) and periodic acid-Schiff (PAS) stains were negative for common bacterial and fungal microorganisms.

►Dr. Kearney. The tumor board, pulmonologist, and patient decide to move forward with video-assisted excisional biopsy with lymphadenectomy. Dr. Huang, can you interpret the pathology?

►Dr. Huang. Figure 2 showed an hemotoxylin and eosin (H&E)-stained lung resection tissue section with multiple caseating necrotic granulomas. No foreign bodies were identified. There was no evidence of malignancy. The GMS stain revealed a fungal microorganism oval with morphology typical of histoplasma capsulatum (Figure 3).

Pulmonary nodules are common incidental finding on chest imaging of patients who reside in histoplasmosis endemic regions, and they are often hard to differentiate from malignancies. There are 3 mediastinal manifestations: adenitis, granuloma, and fibrosis. Usually the syndromes are subclinical, but occasionally the nodes cause symptoms by impinging on other structures.²

This patient had a solitary pulmonary nodule with none of the associated features mentioned above. Pathology showed caseating granuloma and confirmed histoplasmosis.

►Dr. Kearney. Given the diagnosis of solitary histoplasmoma, how should this patient be managed?

►Dr. Strymish. The optimal therapy for histoplasmosis depends on the patient’s clinical syndrome. Most infections are self-limited and require no therapy. However, patients who are immunocompromised, exposed to large inoculum, and have progressive disease require antifungal treatment, usually with itraconazole for mild-to-moderate disease and a combination of azole therapy and amphotericin B with extensive disease. Patients with few solitary pulmonary nodules do not benefit from antifungal therapy as the nodule could represent quiescent disease that is unlikely to have clinical impact; in this case, the treatment would be higher risk than the nodule.³

^►Dr. Kearney. While the discussion of the diagnosis is interesting, it is also important to acknowledge what the patient went through to arrive at this, an essentially benign diagnosis: 8 months, multiple imaging studies, and 2 invasive diagnostic procedures. Further, the patient had to grapple with the possibility of a diagnosis of cancer. Dr. Wiener, can you talk about the challenges in communicating with patients about pulmonary nodules when cancer is on the differential? What are some of the harms patients face and how can clinicians work to mitigate these harms?

►Dr. Wiener. My colleague Dr. Christopher Slatore of the Portland VA Medical Center and I studied communication about pulmonary nodules in a series of surveys and qualitative studies of patients with pulmonary nodules and the clinicians who take care of them. We found that there seems to be a disconnect between patients’ perceptions of pulmonary nodules and their clinicians, often due to inadequate communication about the nodule. Many clinicians indicated that they do not tell patients about the chance that a nodule may be cancer, because the clinicians know that cancer is unlikely (< 5% of incidentally detected pulmonary nodules turn out to be malignant), and they do not want to alarm patients unnecessarily. However, we found that patients almost immediately wondered about cancer when they learned about their pulmonary nodule, and without hearing explicitly from their clinician that cancer was unlikely, patients tended to overestimate the likelihood of a malignant nodule. Moreover, patients often were not told much about the evaluation plan for the nodule or the rationale for CT surveillance of small nodules instead of biopsy. This uncertainty about the risk of cancer and the plan for evaluating the nodule was difficult for some patients to live with; we found that about one-quarter of patients with a small pulmonary nodule experienced mild-moderate distress during the period of radiographic surveillance. Reassuringly, high-quality patient-clinician communication was associated with lower distress and higher adherence to pulmonary nodule evaluation.⁴

►Dr. Kearney. The patient was educated about his diagnosis of solitary histoplasmoma. Given that the patient was otherwise well appearing with no complicating factors, he was not treated with antifungal therapy. After an 8-month-long workup, the patient was relieved to receive a diagnosis that excluded cancer and did not require any further treatment. His case provides a good example of how to proceed in the workup of a solitary pulmonary nodule and on the importance of communication and shared decision making with our patients.

Case Presentation. A 69-year-old veteran presented with an intermittent, waxing and waning cough. He had never smoked and had no family history of lung cancer. His primary care physician ordered a chest radiograph, which revealed a nodular opacity within the lingula concerning for a parenchymal nodule. Further characterization with a chest computed tomography (CT) demonstrated a 1.4-cm left upper lobe subpleural nodule with small satellite nodules (Figure 1). Given these imaging findings, the patient was referred to the pulmonary clinic.

►Renda Wiener, MD, Pulmonary and Critical Care, VABHS, and Assistant Professor of Medicine, Boston University School of Medicine. Pulmonary nodules are well-defined lesions < 3 cm in diameter that are surrounded by lung parenchyma. Although cancer is a possibility (including primary lung cancers, metastatic cancers, or carcinoid tumors), most small nodules do not turn out to be malignant.¹ Benign etiologies include infections, benign tumors, vascular malformations, and inflammatory conditions. Infectious causes of nodules are often granulomatous in nature, including fungi, Mycobacterium tuberculosis, and nontuberculous mycobacteria. Benign tumors are most commonly hamartomas, and these may be clearly distinguished based on imaging characteristics. Pulmonary arteriovenous malformations, hematomas, and infarcts may present as nodules as well. Inflammatory causes of nodules are important and relatively common, including granulomatosis with polyangiitis, rheumatoid arthritis, sarcoidosis, amyloidosis, and rounded atelectasis. 

To distinguish benign from malignant etiologies, we look for several features of pulmonary nodules on imaging. Larger size, irregular borders, and upper lobe location all increase the likelihood of cancer, whereas solid attenuation and calcification make cancer less likely. One of the most reassuring findings that suggests a benign etiology is lack of growth over a period of surveillance; after 2 years without growth we typically consider a nodule benign.¹ And of course, we also consider the patient’s symptoms and risk factors: weight loss, hemoptysis, a history of cigarette smoking or asbestos exposure, or family history of cancer all increase the likelihood of malignancy.

►Dr. Kearney. Given that the differential diagnosis is so broad, how do you think about the next step in evaluating a pulmonary nodule? How do you approach shared decision making with the patient? 

►Dr. Wiener. The characteristics of the patient, the nodule, and the circumstances in which the nodule were discovered are all important to consider. Incidental pulmonary nodules are often found on chest imaging. The imaging characteristics of the nodule are important, as are the patient’s risk factors. A similarly appearing nodule can have very different implications if the patient is a never-smoker exposed to endemic fungi, or a long-time smoker enrolled in a lung cancer screening program. Consultation with a pulmonologist is often appropriate.

It’s important to note that we lack high-quality evidence on the optimal strategy to evaluate pulmonary nodules, and there is no single “right answer“ for all patients. For patients with a low risk of malignancy (< 5%-10%)—which comprises the majority of the incidental nodules discovered—we typically favor serial CT surveillance of the nodule over a period of a few years, whereas for patients at high risk of malignancy (> 65%), we favor early surgical resection if the patient is able to tolerate that. For patients with an intermediate risk of malignancy (~5%-65%), we might consider serial CT surveillance, positron emission tomography (PET) scan, or biopsy.¹ The American College of Chest Physicians guidelines for pulmonary nodule evaluation recommend discussing with patients the different options and the trade-offs of these options in a shared decision-making process.¹

►Dr. Kearney. The patient’s pulmonologist laid out options, including monitoring with serial CT scans, obtaining a PET scan, performing CT-guided needle biopsy, or referring for surgical excision. In this case, the patient elected to undergo CT-guided needle biopsy. Dr. Huang, can you discuss the pathology results?

►Qin Huang, MD, Pathology and Laboratory Medicine, VABHS, and Assistant Professor of Pathology, Harvard Medical School (HMS). The microscopic examination of the needle biopsy of the lung mass revealed rare clusters of atypical cells with crushed cells adjacent to an extensive area of necrosis with scarring. The atypical cells were suspicious for carcinoma. The Gomori methenamine silver (GMS) and periodic acid-Schiff (PAS) stains were negative for common bacterial and fungal microorganisms.

►Dr. Kearney. The tumor board, pulmonologist, and patient decide to move forward with video-assisted excisional biopsy with lymphadenectomy. Dr. Huang, can you interpret the pathology?

►Dr. Huang. Figure 2 showed an hemotoxylin and eosin (H&E)-stained lung resection tissue section with multiple caseating necrotic granulomas. No foreign bodies were identified. There was no evidence of malignancy. The GMS stain revealed a fungal microorganism oval with morphology typical of histoplasma capsulatum (Figure 3).

Pulmonary nodules are common incidental finding on chest imaging of patients who reside in histoplasmosis endemic regions, and they are often hard to differentiate from malignancies. There are 3 mediastinal manifestations: adenitis, granuloma, and fibrosis. Usually the syndromes are subclinical, but occasionally the nodes cause symptoms by impinging on other structures.²

This patient had a solitary pulmonary nodule with none of the associated features mentioned above. Pathology showed caseating granuloma and confirmed histoplasmosis.

►Dr. Kearney. Given the diagnosis of solitary histoplasmoma, how should this patient be managed?

►Dr. Strymish. The optimal therapy for histoplasmosis depends on the patient’s clinical syndrome. Most infections are self-limited and require no therapy. However, patients who are immunocompromised, exposed to large inoculum, and have progressive disease require antifungal treatment, usually with itraconazole for mild-to-moderate disease and a combination of azole therapy and amphotericin B with extensive disease. Patients with few solitary pulmonary nodules do not benefit from antifungal therapy as the nodule could represent quiescent disease that is unlikely to have clinical impact; in this case, the treatment would be higher risk than the nodule.³

^►Dr. Kearney. While the discussion of the diagnosis is interesting, it is also important to acknowledge what the patient went through to arrive at this, an essentially benign diagnosis: 8 months, multiple imaging studies, and 2 invasive diagnostic procedures. Further, the patient had to grapple with the possibility of a diagnosis of cancer. Dr. Wiener, can you talk about the challenges in communicating with patients about pulmonary nodules when cancer is on the differential? What are some of the harms patients face and how can clinicians work to mitigate these harms?

►Dr. Wiener. My colleague Dr. Christopher Slatore of the Portland VA Medical Center and I studied communication about pulmonary nodules in a series of surveys and qualitative studies of patients with pulmonary nodules and the clinicians who take care of them. We found that there seems to be a disconnect between patients’ perceptions of pulmonary nodules and their clinicians, often due to inadequate communication about the nodule. Many clinicians indicated that they do not tell patients about the chance that a nodule may be cancer, because the clinicians know that cancer is unlikely (< 5% of incidentally detected pulmonary nodules turn out to be malignant), and they do not want to alarm patients unnecessarily. However, we found that patients almost immediately wondered about cancer when they learned about their pulmonary nodule, and without hearing explicitly from their clinician that cancer was unlikely, patients tended to overestimate the likelihood of a malignant nodule. Moreover, patients often were not told much about the evaluation plan for the nodule or the rationale for CT surveillance of small nodules instead of biopsy. This uncertainty about the risk of cancer and the plan for evaluating the nodule was difficult for some patients to live with; we found that about one-quarter of patients with a small pulmonary nodule experienced mild-moderate distress during the period of radiographic surveillance. Reassuringly, high-quality patient-clinician communication was associated with lower distress and higher adherence to pulmonary nodule evaluation.⁴

►Dr. Kearney. The patient was educated about his diagnosis of solitary histoplasmoma. Given that the patient was otherwise well appearing with no complicating factors, he was not treated with antifungal therapy. After an 8-month-long workup, the patient was relieved to receive a diagnosis that excluded cancer and did not require any further treatment. His case provides a good example of how to proceed in the workup of a solitary pulmonary nodule and on the importance of communication and shared decision making with our patients.

Persons who trained for a marathon showed improvement in age-related aortic stiffness and reduction in blood pressure in a study of 138 first-time completers of the London Marathon.

Pavel1964/Getty Images

Compared with pretraining values, the descending aortas of marathon completers were 9% more distensible at the level of the bifurcation of the pulmonary artery and 16% more distensible at the level of the diaphragm (P = .0009 and .002, respectively). There was no change in distensibility of the ascending aorta.

Additionally, central systolic BP dropped by 4 mm Hg and diastolic BP by 3 mm Hg by the time marathon training was completed.

“Training and completion of a first-time marathon result in beneficial reductions in BP and intrinsic aortic stiffening in healthy participants,” concluded Anish Bhuva, MBBS, and coinvestigators. “These changes are equivalent to approximately a 4-year reduction in vascular age.”

The study points to a role for exercise in the reduction of arterial stiffness, a known aging-related contributor to cardiovascular disease for which there currently is no good pharmacologic option, said Julio Chirinos, MD, Phd, in an accompanying editorial (J Am Coll Cardiol. 2020 Jan 6. doi: 10.1016/j.jacc.2019.11.007). The challenge lies in implementing exercise interventions on a large scale in societies where “there remains an immense paradoxical gap” between the known benefits of physical activity and increasingly sedentary populations, he added, calling for increased implementation research.

Using cardiovascular magnetic resonance to assess aortic distensibility, Dr. Bhuva, of the Institute of Cardiovascular Science, University College London, and colleagues assessed aortic BP and aortic stiffness at two points via the noninvasive imaging method. The first assessment was conducted before the study participants began marathon training; the second was obtained between 1 and 3 weeks after marathon completion, after any acute effects of the marathon had abated.

Anthropometric data, peripheral BP, and aerobic capacity (peak VO₂) were also assessed at both study points.

Although the study wasn’t designed to track individual training regimens, first-time London Marathon participants were given a 17-week “Beginner’s Training Plan” by event organizers, and asked to follow the plan while participating in the study. The goal of the beginner’s plan was marathon completion, with a schedule of about three runs weekly increasing in duration and intensity over the training period.

Participants had to be first-time marathon participants and running less than 2 hours per week at enrollment. Only those who completed the marathon were included in the data analysis, though baseline characteristics didn’t differ between completers and those who dropped out.

For 2016, the first study year, only participants aged 18-39 years were included, while in 2017, all ages were included in the study. The final age range was 21-69 years, with a mean age of 37; 51% of participants were female. Those with a history of hypertension or taking antihypertensive medication and those who had other significant medical conditions were excluded.

The differential increase in distensibility along the length of the aorta reflects known differences in tissue composition, agreed the authors and Dr. Chirinos, a cardiologist at the University of Pennsylvania, Philadelphia. In addition to the magnetic resonance–obtained distensibility measurements, the investigators conducted further calculations to adjust for baseline mean central arterial pressure, since arterial stiffness is a function both of intrinsic tissue characteristics and loading conditions.

In youth, aortic distensibility buffers the effect of pulse pressure on both the left ventricle and the peripheral vascular system. As the aorta and other large arteries stiffen predictably with age, isolated systolic hypertension can result. The stiffening “also favors adverse patterns of pulsatile left ventricular overload,” which can lead to left ventricular remodeling and, eventually, heart failure, noted Dr. Chirinos. Reduced aortic pliancy also allows pulse pressure variation to be transmitted downstream “into the microvasculature of target organs (such as the kidney and brain) that require high blood flow and thus operate at low arteriolar resistance,” he added.

The assessment that marathon training reversed aortic age by a median 3.9 years was derived from the baseline cross-sectional data regarding participants’ age and aortic stiffness. The effect size was largest in those older than 37 years and in those with higher baseline systolic BP, and men saw greater benefit by a median 1.4 years. Those with slower running times also saw greater benefit.

Study participants had small but significant reductions in heart rate, body fat percentage, and weight by the postmarathon assessment, but these differences were not associated with changes in aortic stiffness. Aerobic exercise capacity as measured by peak VO₂ didn’t change significantly from pre- to post training, but the fact that participants were semirecumbent during exercise testing (to allow concurrent echocardiography) may have affected results.

The real-world design of this study had the strengths of assessing free-living, healthy individuals who participated in a self-directed training plan. Dr. Bhuva and coauthors acknowledged that marathon training may include changes in diet, sleep, and other potentially confounding lifestyle factors, as well as improvement in lipid and glucose metabolism. Further, noted Dr. Chirinos, there was no control group. Also, results from individuals training for an endurance event may have limited generalizability to the general population.

Still, said Dr. Chirinos, the innovative study design took advantage of a large-scale athletic event to see how a realistic training regimen affected healthy individuals. “Perhaps the contemporary marathon can teach us some lessons about exploiting the confluence of interests of the general public, media, industry, scientific community, and government to accomplish worthy goals at the individual and societal levels.”

The study was funded by the British Heart Foundation, Cardiac Risk in the Young, and the Barts Cardiovascular Biomedical Research Centre. Exercise testing equipment and technical support were provided by COSMED. Dr. Bhuva reported receiving funding from the British Heart Foundation. Dr. Chirinos reported having been a consultant or receiving research funding from multiple pharmaceutical companies and Microsoft; he is also an inventor of University of Pennsylvania–held patents for cardiovascular pharmaceutical agents.

[email protected]

SOURCE: Bhuva A et al. J Am Coll Cardiol. 2020 Jan;75(1):60-71.

Persons who trained for a marathon showed improvement in age-related aortic stiffness and reduction in blood pressure in a study of 138 first-time completers of the London Marathon.

Pavel1964/Getty Images

Compared with pretraining values, the descending aortas of marathon completers were 9% more distensible at the level of the bifurcation of the pulmonary artery and 16% more distensible at the level of the diaphragm (P = .0009 and .002, respectively). There was no change in distensibility of the ascending aorta.

Additionally, central systolic BP dropped by 4 mm Hg and diastolic BP by 3 mm Hg by the time marathon training was completed.

“Training and completion of a first-time marathon result in beneficial reductions in BP and intrinsic aortic stiffening in healthy participants,” concluded Anish Bhuva, MBBS, and coinvestigators. “These changes are equivalent to approximately a 4-year reduction in vascular age.”

The study points to a role for exercise in the reduction of arterial stiffness, a known aging-related contributor to cardiovascular disease for which there currently is no good pharmacologic option, said Julio Chirinos, MD, Phd, in an accompanying editorial (J Am Coll Cardiol. 2020 Jan 6. doi: 10.1016/j.jacc.2019.11.007). The challenge lies in implementing exercise interventions on a large scale in societies where “there remains an immense paradoxical gap” between the known benefits of physical activity and increasingly sedentary populations, he added, calling for increased implementation research.

Using cardiovascular magnetic resonance to assess aortic distensibility, Dr. Bhuva, of the Institute of Cardiovascular Science, University College London, and colleagues assessed aortic BP and aortic stiffness at two points via the noninvasive imaging method. The first assessment was conducted before the study participants began marathon training; the second was obtained between 1 and 3 weeks after marathon completion, after any acute effects of the marathon had abated.

Anthropometric data, peripheral BP, and aerobic capacity (peak VO₂) were also assessed at both study points.

Although the study wasn’t designed to track individual training regimens, first-time London Marathon participants were given a 17-week “Beginner’s Training Plan” by event organizers, and asked to follow the plan while participating in the study. The goal of the beginner’s plan was marathon completion, with a schedule of about three runs weekly increasing in duration and intensity over the training period.

Participants had to be first-time marathon participants and running less than 2 hours per week at enrollment. Only those who completed the marathon were included in the data analysis, though baseline characteristics didn’t differ between completers and those who dropped out.

For 2016, the first study year, only participants aged 18-39 years were included, while in 2017, all ages were included in the study. The final age range was 21-69 years, with a mean age of 37; 51% of participants were female. Those with a history of hypertension or taking antihypertensive medication and those who had other significant medical conditions were excluded.

The differential increase in distensibility along the length of the aorta reflects known differences in tissue composition, agreed the authors and Dr. Chirinos, a cardiologist at the University of Pennsylvania, Philadelphia. In addition to the magnetic resonance–obtained distensibility measurements, the investigators conducted further calculations to adjust for baseline mean central arterial pressure, since arterial stiffness is a function both of intrinsic tissue characteristics and loading conditions.

In youth, aortic distensibility buffers the effect of pulse pressure on both the left ventricle and the peripheral vascular system. As the aorta and other large arteries stiffen predictably with age, isolated systolic hypertension can result. The stiffening “also favors adverse patterns of pulsatile left ventricular overload,” which can lead to left ventricular remodeling and, eventually, heart failure, noted Dr. Chirinos. Reduced aortic pliancy also allows pulse pressure variation to be transmitted downstream “into the microvasculature of target organs (such as the kidney and brain) that require high blood flow and thus operate at low arteriolar resistance,” he added.

The assessment that marathon training reversed aortic age by a median 3.9 years was derived from the baseline cross-sectional data regarding participants’ age and aortic stiffness. The effect size was largest in those older than 37 years and in those with higher baseline systolic BP, and men saw greater benefit by a median 1.4 years. Those with slower running times also saw greater benefit.

Study participants had small but significant reductions in heart rate, body fat percentage, and weight by the postmarathon assessment, but these differences were not associated with changes in aortic stiffness. Aerobic exercise capacity as measured by peak VO₂ didn’t change significantly from pre- to post training, but the fact that participants were semirecumbent during exercise testing (to allow concurrent echocardiography) may have affected results.

The real-world design of this study had the strengths of assessing free-living, healthy individuals who participated in a self-directed training plan. Dr. Bhuva and coauthors acknowledged that marathon training may include changes in diet, sleep, and other potentially confounding lifestyle factors, as well as improvement in lipid and glucose metabolism. Further, noted Dr. Chirinos, there was no control group. Also, results from individuals training for an endurance event may have limited generalizability to the general population.

Still, said Dr. Chirinos, the innovative study design took advantage of a large-scale athletic event to see how a realistic training regimen affected healthy individuals. “Perhaps the contemporary marathon can teach us some lessons about exploiting the confluence of interests of the general public, media, industry, scientific community, and government to accomplish worthy goals at the individual and societal levels.”

The study was funded by the British Heart Foundation, Cardiac Risk in the Young, and the Barts Cardiovascular Biomedical Research Centre. Exercise testing equipment and technical support were provided by COSMED. Dr. Bhuva reported receiving funding from the British Heart Foundation. Dr. Chirinos reported having been a consultant or receiving research funding from multiple pharmaceutical companies and Microsoft; he is also an inventor of University of Pennsylvania–held patents for cardiovascular pharmaceutical agents.

[email protected]

SOURCE: Bhuva A et al. J Am Coll Cardiol. 2020 Jan;75(1):60-71.

Persons who trained for a marathon showed improvement in age-related aortic stiffness and reduction in blood pressure in a study of 138 first-time completers of the London Marathon.

Pavel1964/Getty Images

Compared with pretraining values, the descending aortas of marathon completers were 9% more distensible at the level of the bifurcation of the pulmonary artery and 16% more distensible at the level of the diaphragm (P = .0009 and .002, respectively). There was no change in distensibility of the ascending aorta.

Additionally, central systolic BP dropped by 4 mm Hg and diastolic BP by 3 mm Hg by the time marathon training was completed.

“Training and completion of a first-time marathon result in beneficial reductions in BP and intrinsic aortic stiffening in healthy participants,” concluded Anish Bhuva, MBBS, and coinvestigators. “These changes are equivalent to approximately a 4-year reduction in vascular age.”

The study points to a role for exercise in the reduction of arterial stiffness, a known aging-related contributor to cardiovascular disease for which there currently is no good pharmacologic option, said Julio Chirinos, MD, Phd, in an accompanying editorial (J Am Coll Cardiol. 2020 Jan 6. doi: 10.1016/j.jacc.2019.11.007). The challenge lies in implementing exercise interventions on a large scale in societies where “there remains an immense paradoxical gap” between the known benefits of physical activity and increasingly sedentary populations, he added, calling for increased implementation research.

Using cardiovascular magnetic resonance to assess aortic distensibility, Dr. Bhuva, of the Institute of Cardiovascular Science, University College London, and colleagues assessed aortic BP and aortic stiffness at two points via the noninvasive imaging method. The first assessment was conducted before the study participants began marathon training; the second was obtained between 1 and 3 weeks after marathon completion, after any acute effects of the marathon had abated.

Anthropometric data, peripheral BP, and aerobic capacity (peak VO₂) were also assessed at both study points.

Although the study wasn’t designed to track individual training regimens, first-time London Marathon participants were given a 17-week “Beginner’s Training Plan” by event organizers, and asked to follow the plan while participating in the study. The goal of the beginner’s plan was marathon completion, with a schedule of about three runs weekly increasing in duration and intensity over the training period.

Participants had to be first-time marathon participants and running less than 2 hours per week at enrollment. Only those who completed the marathon were included in the data analysis, though baseline characteristics didn’t differ between completers and those who dropped out.

For 2016, the first study year, only participants aged 18-39 years were included, while in 2017, all ages were included in the study. The final age range was 21-69 years, with a mean age of 37; 51% of participants were female. Those with a history of hypertension or taking antihypertensive medication and those who had other significant medical conditions were excluded.

The differential increase in distensibility along the length of the aorta reflects known differences in tissue composition, agreed the authors and Dr. Chirinos, a cardiologist at the University of Pennsylvania, Philadelphia. In addition to the magnetic resonance–obtained distensibility measurements, the investigators conducted further calculations to adjust for baseline mean central arterial pressure, since arterial stiffness is a function both of intrinsic tissue characteristics and loading conditions.

In youth, aortic distensibility buffers the effect of pulse pressure on both the left ventricle and the peripheral vascular system. As the aorta and other large arteries stiffen predictably with age, isolated systolic hypertension can result. The stiffening “also favors adverse patterns of pulsatile left ventricular overload,” which can lead to left ventricular remodeling and, eventually, heart failure, noted Dr. Chirinos. Reduced aortic pliancy also allows pulse pressure variation to be transmitted downstream “into the microvasculature of target organs (such as the kidney and brain) that require high blood flow and thus operate at low arteriolar resistance,” he added.

The assessment that marathon training reversed aortic age by a median 3.9 years was derived from the baseline cross-sectional data regarding participants’ age and aortic stiffness. The effect size was largest in those older than 37 years and in those with higher baseline systolic BP, and men saw greater benefit by a median 1.4 years. Those with slower running times also saw greater benefit.

Study participants had small but significant reductions in heart rate, body fat percentage, and weight by the postmarathon assessment, but these differences were not associated with changes in aortic stiffness. Aerobic exercise capacity as measured by peak VO₂ didn’t change significantly from pre- to post training, but the fact that participants were semirecumbent during exercise testing (to allow concurrent echocardiography) may have affected results.

The real-world design of this study had the strengths of assessing free-living, healthy individuals who participated in a self-directed training plan. Dr. Bhuva and coauthors acknowledged that marathon training may include changes in diet, sleep, and other potentially confounding lifestyle factors, as well as improvement in lipid and glucose metabolism. Further, noted Dr. Chirinos, there was no control group. Also, results from individuals training for an endurance event may have limited generalizability to the general population.

Still, said Dr. Chirinos, the innovative study design took advantage of a large-scale athletic event to see how a realistic training regimen affected healthy individuals. “Perhaps the contemporary marathon can teach us some lessons about exploiting the confluence of interests of the general public, media, industry, scientific community, and government to accomplish worthy goals at the individual and societal levels.”

The study was funded by the British Heart Foundation, Cardiac Risk in the Young, and the Barts Cardiovascular Biomedical Research Centre. Exercise testing equipment and technical support were provided by COSMED. Dr. Bhuva reported receiving funding from the British Heart Foundation. Dr. Chirinos reported having been a consultant or receiving research funding from multiple pharmaceutical companies and Microsoft; he is also an inventor of University of Pennsylvania–held patents for cardiovascular pharmaceutical agents.

[email protected]

SOURCE: Bhuva A et al. J Am Coll Cardiol. 2020 Jan;75(1):60-71.

Providing high-quality, patient-centered health care is a top priority for the US Department of Veterans Affairs (VA) Veteran Health Administration (VHA), whose core mission is to improve the health and well-being of US veterans. Thus, news of long wait times for medical appointments in the VHA sparked intense national attention and debate and led to changes in senior management and legislative action. ¹ On August 8, 2014, President Bara c k Obama signed the Veterans Access, Choice, and Accountability Act of 2014, also known as the Choice Act, which provided an additional $16 billion in emergency spending over 3 years to improve veterans’ access to timely health care. ² The Choice Act sought to develop an integrated health care network that allowed qualified VHA patients to receive specific health care services in their communities delivered by non-VHA health care providers (HCPs) but paid for by the VHA. The Choice Act also laid out explicit criteria for how to prioritize who would be eligible for VHA-purchased civilian care: (1) veterans who could not get timely appointments at a VHA medical facility within 30 days of referral; or (2) veterans who lived > 40 miles from the closest VHA medical facility.

VHA decision makers seeking to improve care delivery also need to weigh trade-offs between alternative approaches to providing rapid access. For instance, increasing access to non-VHA HCPs may not always decrease wait times and could result in loss of continuity, limited care coordination, limited ability to ensure and enforce high-quality standards at the VHA, and other challenges.^3-6 Although the concerns and views of elected representatives, advocacy groups, and health system leaders are important, it is unknown whether these views and preferences align with those of veterans. Arguably, the range of views and concerns of informed veterans whose health is at stake should be particularly prominent in such policy decision making.

To identify the considerations that were most important to veterans regarding VHA policy around decreasing wait times, a study was designed to engage a group of veterans who were eligible for civilian care under the Choice Act. The study took place 1 year after the Choice Act was passed. Veterans were asked to focus on 2 related questions: First, how should funding be used for building VHA capacity (build) vs purchasing civilian care (buy)? Second, under what circumstances should civilian care be prioritized?

The aim of this paper is to describe democratic deliberation (DD), a specific method that engaged veteran patients in complex policy decisions around access to care. DD methods have been used increasingly in health care for developing policy guidance, setting priorities, providing advice on ethical dilemmas, weighing risk-benefit trade-offs, and determining decision-making authority.^7-12 For example, DD helped guide national policy for mammography screening for breast cancer in New Zealand.¹³ The Agency for Healthcare Research and Quality has completed a systematic review and a large, randomized experiment on best practices for carrying out public deliberation.^8,13,14 However, despite the potential value of this approach, there has been little use of deliberative methods within the VHA for the explicit purpose of informing veteran health care delivery.

This paper describes the experience engaging veterans by using DD methodology and informing VHA leadership about the results of those deliberations. The specific aims were to understand whether DD is an acceptable approach to use to engage patients in the medical services policy-making process within VHA and whether veterans are able to come to an informed consensus.

Methods

Engaging patients and incorporating their needs and concerns within the policy-making process may improve health system policies and make those policies more patient centered. Such engagement also could be a way to generate creative solutions. However, because health-system decisions often involve making difficult trade-offs, effectively obtaining patient population input on complex care delivery issues can be challenging.

Although surveys can provide intuitive, top-of-mind input from respondents, these opinions are generally not sufficient for resolving complex problems.¹⁵ Focus groups and interviews may produce results that are more in-depth than surveys, but these methods tend to elicit settled private preferences rather than opinions about what the community should do.¹⁶ DD, on the other hand, is designed to elicit deeply informed public opinions on complex, value-laden topics to develop recommendations and policies for a larger community.¹⁷ The goal is to find collective solutions to challenging social problems. DD achieves this by giving participants an opportunity to explore a topic in-depth, question experts, and engage peers in reason-based discussions.^18,19 This method has its roots in political science and has been used over several decades to successfully inform policy making on a broad array of topics nationally and internationally—from health research ethics in the US to nuclear and energy policy in Japan.^7,16,20,21 DD has been found to promote ownership of public programs and lend legitimacy to policy decisions, political institutions, and democracy itself.¹⁸

A single day (8 hours) DD session was convened, following a Citizens Jury model of deliberation, which brings veteran patients together to learn about a topic, ask questions of experts, deliberate with peers, and generate a “citizen’s report” that contains a set of recommendations (Table 1). An overview of the different models of DD and rationale for each can be found elsewhere.^8,15

Recruitment Considerations

A purposively selected sample of civilian care-eligible veterans from a midwestern VHA health care system (1 medical center and 3 community-based outpatient clinics [CBOCs]) were invited to the DD session. The targeted number of participants was 30. Female veterans, who comprise only 7% of the local veteran population, were oversampled to account for their potentially different health care needs and to create balance between males and females in the session. Oversampling for other characteristics was not possible due to the relatively small sample size. Based on prior experience,⁷ it was assumed that 70% of willing participants would attend the session; therefore 34 veterans were invited and 24 attended. Each participant received a $200 incentive in appreciation for their substantial time commitment and to offset transportation costs.

Background Materials

A packet with educational materials (Flesch-Kincaid Grade Level of 10.5) was mailed to participants about 2 weeks before the DD session. Participants were asked to review prior to attending the session. These materials described the session (eg, purpose, organizers, importance) and provided factual information about the Choice Act (eg, eligibility, out-of-pocket costs, travel pay, prescription drug policies).

Session Overview

The session was structured to accomplish the following goals: (1) Elicit participants’ opinions about access to health care and reasons for those opinions; (2) Provide in-depth education about the Choice Act through presentations and discussions with topical experts; and (3) Elicit reasoning and recommendations on both the criteria by which participants prioritize candidates for civilian care and how participants would allocate additional funding to improve access (ie, by building VHA capacity to deliver more timely health care vs purchasing health care from civilian HCPs).

Participants were asked to fill out a survey on arrival in the morning and were assigned to 1 of 3 tables or small groups. Each table had a facilitator who had extensive experience in qualitative data collection methods and guided the dialogue using a scripted protocol that they helped develop and refine. The facilitation materials drew from and used previously published studies.^22,23 Each facilitator audio recorded the sessions and took notes. Three experts presented during plenary education sessions. Presentations were designed to provide balanced factual information and included a veteran’s perspective. One presenter was a clinician on the project team, another was a local clinical leader responsible for making decisions about what services to provide via civilian care (buy) vs enhancing the local VHA health system’s ability to provide those services (build), and the third presenter was a veteran who was on the project team.

Education Session 1

The first plenary education session with expert presentations was conducted after each table completed an icebreaker exercise. The project team physician provided a brief history and description of the Choice Act to reinforce educational materials sent to participants prior to the session. The health system clinical leader described his decision process and principles and highlighted constraints placed on him by the Choice Act that were in place at the time of the DD session. He also described existing local and national programs to provide civilian care (eg, local fee-basis non-VHA care programs) and how these programs sought to achieve goals similar to the Choice Act. The veteran presenter focused on the importance of session participants providing candid insight and observations and emphasized that this session was a significant opportunity to “have their voices heard.”

Deliberation 1: What criteria should be used to prioritize patients for receiving civilian care paid for by the VHA? To elicit preferences on the central question of this deliberation, participants were presented with 8 real-world cases that were based on interviews conducted with Choice Act-eligible veterans (Table 2 and eAppendices A

Education Session 2

In the second plenary session, the project team physician provided information about health care access issues, both inside and outside of the VHA, particularly between urban and rural areas. He also discussed factors related to the insufficient capacity to meet growing demand that contributed to the VHA wait-time crisis. The veteran presenter shared reflections on health care access from a veteran’s perspective.

Deliberation 2: How should additional funding be divided between increasing the ability of the VHA to (1) provide care by VHA HCPs; and (2) pay for care from non-VHA civilian HCPs? Participants were presented the patient examples and Choice Act funding scenarios (the buy policy option) and contrasted that with a build policy option. Participants were explicitly encouraged to shift their perspectives from thinking about individual cases to considering policy-level decisions and the broader social good (Table 2).

Ensuring Robust Deliberations

If participants do not adequately grasp the complexities of the topic, a deliberation can fail. To facilitate nuanced reasoning, real-world concrete examples were developed as the starting point of each deliberation based on interviews with actual patients (deliberation 1) and actual policy proposals relevant to the funding allocation decisions within the Choice Act (deliberation 2).

A deliberation also can fail with self-silencing, where participants withhold opinions that differ from those articulated first or by more vocal members of the group.²⁴ To combat self-silencing, highly experienced facilitators were used to ensure sharing from all participants and to support an open-minded, courteous, and reason-based environment for discourse. It was specified that the best solutions are achieved through reason-based and cordial disagreement and that success can be undermined when participants simply agree because it is easier or more comfortable.

A third way a deliberation can fail is if individuals do not adopt a group or system-level perspective. To counter this, facilitators reinforced at multiple points the importance of taking a broader social perspective rather than sharing only one’s personal preferences.

Finally, it is important to assess the quality of the deliberative process itself, to ensure that results are trustworthy.²⁵ To assess the quality of the deliberative process, participants knowledge about key issues pre- and postdeliberation were assessed. Participants also were asked to rate the quality of the facilitators and how well they felt their voice was heard and respected, and facilitators made qualitative assessments about the extent to which participants were engaged in reason-based and collaborative discussion.

Data

Quantitative data were collected via pre- and postsession surveys. The surveys contained items related to knowledge about the Choice Act, expectations for the DD session, beliefs and opinions about the provision of health care for veterans, recommended funding allocations between build vs buy policy options, and general demographics. Qualitative data were collected through detailed notes taken by the 3 facilitators. Each table’s deliberations were audio recorded so that gaps in the notes could be filled.

The 3 facilitators, who were all experienced qualitative researchers, typed their written notes into a template immediately after the session. Two of the 3 facilitators led the analysis of the session notes. Findings within and across the 3 deliberation tables were developed using content and matrix analysis methods.²⁶ Descriptive statistics were generated from survey responses and compared survey items pre- and postsession using paired t tests or χ² tests for categorical responses.

Results

Thirty-three percent of individuals invited (n = 127) agreed to participate. Those who declined cited conflicts related to distance, transportation, work/school, medical appointments, family commitments, or were not interested. In all, 24 (69%) of the 35 veterans who accepted the invitation attended the deliberation session. Of the 11 who accepted but did not attend, 5 cancelled ahead of time because of conflicts (Figure). Most participants were male (70%), 48% were aged 61 to 75 years, 65% were white, 43% had some college education, 43% reported an annual income of between $25,000 and $40,000, and only 35% reported very good health (eAppendix D).

Deliberation 1

During the deliberation on the prioritization criteria, the concept of “condition severity” emerged as an important criterion for veterans. This criterion captured simultaneous consideration of both clinical necessity and burden on the veteran to obtain care. For example, participants felt that patients with a life-threatening illness should be prioritized for civilian care over patients who need preventative or primary care (clinical necessity) and that elderly patients with substantial difficulty traveling to VHA appointments should be prioritized over patients who can travel more easily (burden). The Choice Act regulations at the time of the DD session did not reflect this nuanced perspective, stipulating only that veterans must live > 40 miles from the nearest VHA medical facility.

One of the 3 groups did not prioritize the patient cases because some members felt that no veteran should be constrained from receiving civilian care if they desired it. Nonetheless, this group did agree with prioritizing the first 2 cases in Table 3. The other groups prioritized all 8 cases in generally similar ways.

Deliberation 2

No clear consensus emerged on the buy vs build question. A representative from each table presented their group’s positions, rationale, and recommendations after deliberations were completed. After hearing the range of positions, the groups then had another opportunity to deliberate based on what they heard from the other tables; no new recommendations or consensus emerged.

Participants who were in favor of allocating more funds toward the build policy offered a range of rationales, saying that it would (1) increase access for rural veterans by building CBOCs and deploying more mobile units that could bring outlets for health care closer to their home communities; (2) provide critical and unique medical expertise to address veteran-specific issues such as prosthetics, traumatic brain injury, posttraumatic stress disorder, spinal cord injury, and shrapnel wounds that are typically not available through civilian providers; (3) give VHA more oversight over the quality and cost of care, which is more challenging to do with civilian providers; and (4) Improve VHA infrastructure by, for example, upgrading technology and attracting the best clinicians and staff to support “our VHA.”

Participants who were in favor of allocating more funds toward the buy policy also offered a range of rationales, saying that it would (1) decrease patient burden by increasing access through community providers, decreasing wait time, and lessening personal cost and travel time; (2) allow more patients to receive civilian care, which was generally seen as beneficial by a few participants because of perceptions that the VHA provides lower quality care due to a shortage of VHA providers, run-down/older facilities, lack of technology, and poorer-quality VHA providers; and (3) provide an opportunity to divest of costly facilities and invest in other innovative approaches. Regarding this last reason, a few participants felt that the VHA is “gouged” when building medical centers that overrun budgets. They also were concerned that investing in facilities tied VHA to specific locations when current locations of veterans may change “25 years from now.”

Survey Results

Twenty-three of the 24 participants completed both pre- and postsession surveys. The majority of participants in the session felt people in the group respected their opinion (96%); felt that the facilitator did not try to influence the group with her own opinions (96%); indicated they understood the information enough to participate as much as they wanted (100%); and were hopeful that their reasoning and recommendations would help inform VHA policy makers (82%).

The surveys also provided an opportunity to examine the extent to which knowledge, attitudes, and opinions changed from before to after the deliberation. Even with the small sample, responses revealed a trend toward improved knowledge about key elements of the Choice Act and its goals. Further, there was a shift in some participants’ opinions about how patients should be prioritized to receive civilian care. For example, before the deliberation participants generally felt that all veterans should be able to receive civilian care, whereas postdeliberation this was not the case. Postdeliberation, most participants felt that primary care should not be a high priority for civilian care but continued to endorse prioritizing civilian care for specialty services like orthopedic or cardiology-related care. Finally, participants moved from more diverse recommendations regarding additional funds allocations, toward consensus after the deliberation around allocating funds to the build policy. Eight participants supported a build policy beforehand, whereas 16 supported this policy afterward.

Discussion

This study explored DD as a method for deeply engaging veterans in complex policy making to guide funding allocation and prioritization decisions related to the Choice Act, decisions that are still very relevant today within the context of the Mission Act and have substantial implications for how health care is delivered in the VHA. The Mission Act passed on June 6, 2018, with the goal of improving access to and the reliability of civilian or community care for eligible veterans.²⁷ Decisions related to appropriating scarce funding to improve access to care is an emotional and value-laden topic that elicited strong and divergent opinions among the participants. Veterans were eager to have their voices heard and had strong expectations that VHA leadership would be briefed about their recommendations. The majority of participants were satisfied with the deliberation process, felt they understood the issues, and felt their opinions were respected. They expressed feelings of comradery and community throughout the process.

In this single deliberation session, the groups did not achieve a single, final consensus regarding how VHA funding should ultimately be allocated between buy and build policy options. Nonetheless, participants provided a rich array of recommendations and rationale for them. Session moderators observed rich, sophisticated, fair, and reason-based discussions on this complex topic. Participants left with a deeper knowledge and appreciation for the complex trade-offs and expressed strong rationales for both sides of the policy debate on build vs buy. In addition, the project yielded results of high interest to VHA policy makers.

This work was presented in multiple venues between 2015 to 2016, and to both local and national VHA leadership, including the local Executive Quality Leadership Boards, the VHA Central Office Committee on the Future State of VA Community Care, the VA Office of Patient Centered Care, and the National Veteran Experience Committee. Through these discussions and others, we saw great interest within the VHA system and high-level leaders to explore ways to include veterans’ voices in the policy-making process. This work was invaluable to our research team (eAppendix E 

Many health system decisions regarding what care should be delivered (and how) involve making difficult, value-laden choices in the context of limited resources. DD methods can be used to target and obtain specific viewpoints from diverse populations, such as the informed perspectives of minority and underrepresented populations within the VHA.¹⁹ For example, female veterans were oversampled to ensure that the informed preferences of this population was obtained. Thus, DD methods could provide a valuable tool for health systems to elicit in-depth diverse patient input on high-profile policies that will have a substantial impact on the system’s patient population.

Limitations

One potential downside of DD is that, because of the resource-intensive nature of deliberation sessions, they are often conducted with relatively small groups.⁹ Viewpoints of those within these small samples who are willing to spend an entire day discussing a complex topic may not be representative of the larger patient community. However, the core goal of DD is diversity of opinions rather than representativeness.

A stratified random sampling strategy that oversampled for underrepresented and minority populations was used to help select a diverse group that represents the population on key characteristics and partially addresses concern about representativeness. Efforts to optimize participation rates, including providing monetary incentives, also are helpful and have led to high participation rates in past deliberations.⁷

Health system communication strategies that promote the importance of becoming involved in DD sessions also may be helpful in improving rates of recruitment. On particularly important topics where health system leaders feel a larger resource investment is justified, conducting larger scale deliberations with many small groups may obtain more generalizable evidence about what individual patients and groups of patients recommend.⁷ However, due to the inherent limitations of surveys and focus group approaches for obtaining informed views on complex topics, there are no clear systematic alternatives to the DD approach.

Conclusion

DD is an effective method to meaningfully engage patients in deep deliberations to guide complex policy making. Although design of deliberative sessions is resource-intensive, patient engagement efforts, such as those described in this paper, could be an important aspect of a well-functioning learning health system. Further research into alternative, streamlined methods that can also engage veterans more deeply is needed. DD also can be combined with other approaches to broaden and confirm findings, including focus groups, town hall meetings, or surveys.

Although this study did not provide consensus on how the VHA should allocate funds with respect to the Choice Act, it did provide insight into the importance and feasibility of engaging veterans in the policy-making process. As more policies aimed at improving veterans’ access to civilian care are created, such as the most recent Mission Act, policy makers should strongly consider using the DD method of obtaining informed veteran input into future policy decisions.

Acknowledgments
Funding was provided by the US Department of Veterans Affairs Office of Analytics and Business Intelligence (OABI) and the VA Quality Enhancement Research Initiative (QUERI). Dr. Caverly was supported in part by a VA Career Development Award (CDA 16-151). Dr. Krein is supported by a VA Health Services Research and Development Research Career Scientist Award (RCS 11-222). The authors thank the veterans who participated in this work. They also thank Caitlin Reardon and Natalya Wawrin for their assistance in organizing the deliberation session.

Providing high-quality, patient-centered health care is a top priority for the US Department of Veterans Affairs (VA) Veteran Health Administration (VHA), whose core mission is to improve the health and well-being of US veterans. Thus, news of long wait times for medical appointments in the VHA sparked intense national attention and debate and led to changes in senior management and legislative action. ¹ On August 8, 2014, President Bara c k Obama signed the Veterans Access, Choice, and Accountability Act of 2014, also known as the Choice Act, which provided an additional $16 billion in emergency spending over 3 years to improve veterans’ access to timely health care. ² The Choice Act sought to develop an integrated health care network that allowed qualified VHA patients to receive specific health care services in their communities delivered by non-VHA health care providers (HCPs) but paid for by the VHA. The Choice Act also laid out explicit criteria for how to prioritize who would be eligible for VHA-purchased civilian care: (1) veterans who could not get timely appointments at a VHA medical facility within 30 days of referral; or (2) veterans who lived > 40 miles from the closest VHA medical facility.

VHA decision makers seeking to improve care delivery also need to weigh trade-offs between alternative approaches to providing rapid access. For instance, increasing access to non-VHA HCPs may not always decrease wait times and could result in loss of continuity, limited care coordination, limited ability to ensure and enforce high-quality standards at the VHA, and other challenges.^3-6 Although the concerns and views of elected representatives, advocacy groups, and health system leaders are important, it is unknown whether these views and preferences align with those of veterans. Arguably, the range of views and concerns of informed veterans whose health is at stake should be particularly prominent in such policy decision making.

To identify the considerations that were most important to veterans regarding VHA policy around decreasing wait times, a study was designed to engage a group of veterans who were eligible for civilian care under the Choice Act. The study took place 1 year after the Choice Act was passed. Veterans were asked to focus on 2 related questions: First, how should funding be used for building VHA capacity (build) vs purchasing civilian care (buy)? Second, under what circumstances should civilian care be prioritized?

The aim of this paper is to describe democratic deliberation (DD), a specific method that engaged veteran patients in complex policy decisions around access to care. DD methods have been used increasingly in health care for developing policy guidance, setting priorities, providing advice on ethical dilemmas, weighing risk-benefit trade-offs, and determining decision-making authority.^7-12 For example, DD helped guide national policy for mammography screening for breast cancer in New Zealand.¹³ The Agency for Healthcare Research and Quality has completed a systematic review and a large, randomized experiment on best practices for carrying out public deliberation.^8,13,14 However, despite the potential value of this approach, there has been little use of deliberative methods within the VHA for the explicit purpose of informing veteran health care delivery.

This paper describes the experience engaging veterans by using DD methodology and informing VHA leadership about the results of those deliberations. The specific aims were to understand whether DD is an acceptable approach to use to engage patients in the medical services policy-making process within VHA and whether veterans are able to come to an informed consensus.

Methods

Engaging patients and incorporating their needs and concerns within the policy-making process may improve health system policies and make those policies more patient centered. Such engagement also could be a way to generate creative solutions. However, because health-system decisions often involve making difficult trade-offs, effectively obtaining patient population input on complex care delivery issues can be challenging.

Although surveys can provide intuitive, top-of-mind input from respondents, these opinions are generally not sufficient for resolving complex problems.¹⁵ Focus groups and interviews may produce results that are more in-depth than surveys, but these methods tend to elicit settled private preferences rather than opinions about what the community should do.¹⁶ DD, on the other hand, is designed to elicit deeply informed public opinions on complex, value-laden topics to develop recommendations and policies for a larger community.¹⁷ The goal is to find collective solutions to challenging social problems. DD achieves this by giving participants an opportunity to explore a topic in-depth, question experts, and engage peers in reason-based discussions.^18,19 This method has its roots in political science and has been used over several decades to successfully inform policy making on a broad array of topics nationally and internationally—from health research ethics in the US to nuclear and energy policy in Japan.^7,16,20,21 DD has been found to promote ownership of public programs and lend legitimacy to policy decisions, political institutions, and democracy itself.¹⁸

A single day (8 hours) DD session was convened, following a Citizens Jury model of deliberation, which brings veteran patients together to learn about a topic, ask questions of experts, deliberate with peers, and generate a “citizen’s report” that contains a set of recommendations (Table 1). An overview of the different models of DD and rationale for each can be found elsewhere.^8,15

Recruitment Considerations

A purposively selected sample of civilian care-eligible veterans from a midwestern VHA health care system (1 medical center and 3 community-based outpatient clinics [CBOCs]) were invited to the DD session. The targeted number of participants was 30. Female veterans, who comprise only 7% of the local veteran population, were oversampled to account for their potentially different health care needs and to create balance between males and females in the session. Oversampling for other characteristics was not possible due to the relatively small sample size. Based on prior experience,⁷ it was assumed that 70% of willing participants would attend the session; therefore 34 veterans were invited and 24 attended. Each participant received a $200 incentive in appreciation for their substantial time commitment and to offset transportation costs.

Background Materials

A packet with educational materials (Flesch-Kincaid Grade Level of 10.5) was mailed to participants about 2 weeks before the DD session. Participants were asked to review prior to attending the session. These materials described the session (eg, purpose, organizers, importance) and provided factual information about the Choice Act (eg, eligibility, out-of-pocket costs, travel pay, prescription drug policies).

Session Overview

The session was structured to accomplish the following goals: (1) Elicit participants’ opinions about access to health care and reasons for those opinions; (2) Provide in-depth education about the Choice Act through presentations and discussions with topical experts; and (3) Elicit reasoning and recommendations on both the criteria by which participants prioritize candidates for civilian care and how participants would allocate additional funding to improve access (ie, by building VHA capacity to deliver more timely health care vs purchasing health care from civilian HCPs).

Participants were asked to fill out a survey on arrival in the morning and were assigned to 1 of 3 tables or small groups. Each table had a facilitator who had extensive experience in qualitative data collection methods and guided the dialogue using a scripted protocol that they helped develop and refine. The facilitation materials drew from and used previously published studies.^22,23 Each facilitator audio recorded the sessions and took notes. Three experts presented during plenary education sessions. Presentations were designed to provide balanced factual information and included a veteran’s perspective. One presenter was a clinician on the project team, another was a local clinical leader responsible for making decisions about what services to provide via civilian care (buy) vs enhancing the local VHA health system’s ability to provide those services (build), and the third presenter was a veteran who was on the project team.

Education Session 1

The first plenary education session with expert presentations was conducted after each table completed an icebreaker exercise. The project team physician provided a brief history and description of the Choice Act to reinforce educational materials sent to participants prior to the session. The health system clinical leader described his decision process and principles and highlighted constraints placed on him by the Choice Act that were in place at the time of the DD session. He also described existing local and national programs to provide civilian care (eg, local fee-basis non-VHA care programs) and how these programs sought to achieve goals similar to the Choice Act. The veteran presenter focused on the importance of session participants providing candid insight and observations and emphasized that this session was a significant opportunity to “have their voices heard.”

Deliberation 1: What criteria should be used to prioritize patients for receiving civilian care paid for by the VHA? To elicit preferences on the central question of this deliberation, participants were presented with 8 real-world cases that were based on interviews conducted with Choice Act-eligible veterans (Table 2 and eAppendices A

Education Session 2

In the second plenary session, the project team physician provided information about health care access issues, both inside and outside of the VHA, particularly between urban and rural areas. He also discussed factors related to the insufficient capacity to meet growing demand that contributed to the VHA wait-time crisis. The veteran presenter shared reflections on health care access from a veteran’s perspective.

Deliberation 2: How should additional funding be divided between increasing the ability of the VHA to (1) provide care by VHA HCPs; and (2) pay for care from non-VHA civilian HCPs? Participants were presented the patient examples and Choice Act funding scenarios (the buy policy option) and contrasted that with a build policy option. Participants were explicitly encouraged to shift their perspectives from thinking about individual cases to considering policy-level decisions and the broader social good (Table 2).

Ensuring Robust Deliberations

If participants do not adequately grasp the complexities of the topic, a deliberation can fail. To facilitate nuanced reasoning, real-world concrete examples were developed as the starting point of each deliberation based on interviews with actual patients (deliberation 1) and actual policy proposals relevant to the funding allocation decisions within the Choice Act (deliberation 2).

A deliberation also can fail with self-silencing, where participants withhold opinions that differ from those articulated first or by more vocal members of the group.²⁴ To combat self-silencing, highly experienced facilitators were used to ensure sharing from all participants and to support an open-minded, courteous, and reason-based environment for discourse. It was specified that the best solutions are achieved through reason-based and cordial disagreement and that success can be undermined when participants simply agree because it is easier or more comfortable.

A third way a deliberation can fail is if individuals do not adopt a group or system-level perspective. To counter this, facilitators reinforced at multiple points the importance of taking a broader social perspective rather than sharing only one’s personal preferences.

Finally, it is important to assess the quality of the deliberative process itself, to ensure that results are trustworthy.²⁵ To assess the quality of the deliberative process, participants knowledge about key issues pre- and postdeliberation were assessed. Participants also were asked to rate the quality of the facilitators and how well they felt their voice was heard and respected, and facilitators made qualitative assessments about the extent to which participants were engaged in reason-based and collaborative discussion.

Data

Quantitative data were collected via pre- and postsession surveys. The surveys contained items related to knowledge about the Choice Act, expectations for the DD session, beliefs and opinions about the provision of health care for veterans, recommended funding allocations between build vs buy policy options, and general demographics. Qualitative data were collected through detailed notes taken by the 3 facilitators. Each table’s deliberations were audio recorded so that gaps in the notes could be filled.

The 3 facilitators, who were all experienced qualitative researchers, typed their written notes into a template immediately after the session. Two of the 3 facilitators led the analysis of the session notes. Findings within and across the 3 deliberation tables were developed using content and matrix analysis methods.²⁶ Descriptive statistics were generated from survey responses and compared survey items pre- and postsession using paired t tests or χ² tests for categorical responses.

Results

Thirty-three percent of individuals invited (n = 127) agreed to participate. Those who declined cited conflicts related to distance, transportation, work/school, medical appointments, family commitments, or were not interested. In all, 24 (69%) of the 35 veterans who accepted the invitation attended the deliberation session. Of the 11 who accepted but did not attend, 5 cancelled ahead of time because of conflicts (Figure). Most participants were male (70%), 48% were aged 61 to 75 years, 65% were white, 43% had some college education, 43% reported an annual income of between $25,000 and $40,000, and only 35% reported very good health (eAppendix D).

Deliberation 1

During the deliberation on the prioritization criteria, the concept of “condition severity” emerged as an important criterion for veterans. This criterion captured simultaneous consideration of both clinical necessity and burden on the veteran to obtain care. For example, participants felt that patients with a life-threatening illness should be prioritized for civilian care over patients who need preventative or primary care (clinical necessity) and that elderly patients with substantial difficulty traveling to VHA appointments should be prioritized over patients who can travel more easily (burden). The Choice Act regulations at the time of the DD session did not reflect this nuanced perspective, stipulating only that veterans must live > 40 miles from the nearest VHA medical facility.

One of the 3 groups did not prioritize the patient cases because some members felt that no veteran should be constrained from receiving civilian care if they desired it. Nonetheless, this group did agree with prioritizing the first 2 cases in Table 3. The other groups prioritized all 8 cases in generally similar ways.

Deliberation 2

No clear consensus emerged on the buy vs build question. A representative from each table presented their group’s positions, rationale, and recommendations after deliberations were completed. After hearing the range of positions, the groups then had another opportunity to deliberate based on what they heard from the other tables; no new recommendations or consensus emerged.

Participants who were in favor of allocating more funds toward the build policy offered a range of rationales, saying that it would (1) increase access for rural veterans by building CBOCs and deploying more mobile units that could bring outlets for health care closer to their home communities; (2) provide critical and unique medical expertise to address veteran-specific issues such as prosthetics, traumatic brain injury, posttraumatic stress disorder, spinal cord injury, and shrapnel wounds that are typically not available through civilian providers; (3) give VHA more oversight over the quality and cost of care, which is more challenging to do with civilian providers; and (4) Improve VHA infrastructure by, for example, upgrading technology and attracting the best clinicians and staff to support “our VHA.”

Participants who were in favor of allocating more funds toward the buy policy also offered a range of rationales, saying that it would (1) decrease patient burden by increasing access through community providers, decreasing wait time, and lessening personal cost and travel time; (2) allow more patients to receive civilian care, which was generally seen as beneficial by a few participants because of perceptions that the VHA provides lower quality care due to a shortage of VHA providers, run-down/older facilities, lack of technology, and poorer-quality VHA providers; and (3) provide an opportunity to divest of costly facilities and invest in other innovative approaches. Regarding this last reason, a few participants felt that the VHA is “gouged” when building medical centers that overrun budgets. They also were concerned that investing in facilities tied VHA to specific locations when current locations of veterans may change “25 years from now.”

Survey Results

Twenty-three of the 24 participants completed both pre- and postsession surveys. The majority of participants in the session felt people in the group respected their opinion (96%); felt that the facilitator did not try to influence the group with her own opinions (96%); indicated they understood the information enough to participate as much as they wanted (100%); and were hopeful that their reasoning and recommendations would help inform VHA policy makers (82%).

The surveys also provided an opportunity to examine the extent to which knowledge, attitudes, and opinions changed from before to after the deliberation. Even with the small sample, responses revealed a trend toward improved knowledge about key elements of the Choice Act and its goals. Further, there was a shift in some participants’ opinions about how patients should be prioritized to receive civilian care. For example, before the deliberation participants generally felt that all veterans should be able to receive civilian care, whereas postdeliberation this was not the case. Postdeliberation, most participants felt that primary care should not be a high priority for civilian care but continued to endorse prioritizing civilian care for specialty services like orthopedic or cardiology-related care. Finally, participants moved from more diverse recommendations regarding additional funds allocations, toward consensus after the deliberation around allocating funds to the build policy. Eight participants supported a build policy beforehand, whereas 16 supported this policy afterward.

Discussion

This study explored DD as a method for deeply engaging veterans in complex policy making to guide funding allocation and prioritization decisions related to the Choice Act, decisions that are still very relevant today within the context of the Mission Act and have substantial implications for how health care is delivered in the VHA. The Mission Act passed on June 6, 2018, with the goal of improving access to and the reliability of civilian or community care for eligible veterans.²⁷ Decisions related to appropriating scarce funding to improve access to care is an emotional and value-laden topic that elicited strong and divergent opinions among the participants. Veterans were eager to have their voices heard and had strong expectations that VHA leadership would be briefed about their recommendations. The majority of participants were satisfied with the deliberation process, felt they understood the issues, and felt their opinions were respected. They expressed feelings of comradery and community throughout the process.

In this single deliberation session, the groups did not achieve a single, final consensus regarding how VHA funding should ultimately be allocated between buy and build policy options. Nonetheless, participants provided a rich array of recommendations and rationale for them. Session moderators observed rich, sophisticated, fair, and reason-based discussions on this complex topic. Participants left with a deeper knowledge and appreciation for the complex trade-offs and expressed strong rationales for both sides of the policy debate on build vs buy. In addition, the project yielded results of high interest to VHA policy makers.

This work was presented in multiple venues between 2015 to 2016, and to both local and national VHA leadership, including the local Executive Quality Leadership Boards, the VHA Central Office Committee on the Future State of VA Community Care, the VA Office of Patient Centered Care, and the National Veteran Experience Committee. Through these discussions and others, we saw great interest within the VHA system and high-level leaders to explore ways to include veterans’ voices in the policy-making process. This work was invaluable to our research team (eAppendix E 

Many health system decisions regarding what care should be delivered (and how) involve making difficult, value-laden choices in the context of limited resources. DD methods can be used to target and obtain specific viewpoints from diverse populations, such as the informed perspectives of minority and underrepresented populations within the VHA.¹⁹ For example, female veterans were oversampled to ensure that the informed preferences of this population was obtained. Thus, DD methods could provide a valuable tool for health systems to elicit in-depth diverse patient input on high-profile policies that will have a substantial impact on the system’s patient population.

Limitations

One potential downside of DD is that, because of the resource-intensive nature of deliberation sessions, they are often conducted with relatively small groups.⁹ Viewpoints of those within these small samples who are willing to spend an entire day discussing a complex topic may not be representative of the larger patient community. However, the core goal of DD is diversity of opinions rather than representativeness.

A stratified random sampling strategy that oversampled for underrepresented and minority populations was used to help select a diverse group that represents the population on key characteristics and partially addresses concern about representativeness. Efforts to optimize participation rates, including providing monetary incentives, also are helpful and have led to high participation rates in past deliberations.⁷

Health system communication strategies that promote the importance of becoming involved in DD sessions also may be helpful in improving rates of recruitment. On particularly important topics where health system leaders feel a larger resource investment is justified, conducting larger scale deliberations with many small groups may obtain more generalizable evidence about what individual patients and groups of patients recommend.⁷ However, due to the inherent limitations of surveys and focus group approaches for obtaining informed views on complex topics, there are no clear systematic alternatives to the DD approach.

Conclusion

DD is an effective method to meaningfully engage patients in deep deliberations to guide complex policy making. Although design of deliberative sessions is resource-intensive, patient engagement efforts, such as those described in this paper, could be an important aspect of a well-functioning learning health system. Further research into alternative, streamlined methods that can also engage veterans more deeply is needed. DD also can be combined with other approaches to broaden and confirm findings, including focus groups, town hall meetings, or surveys.

Although this study did not provide consensus on how the VHA should allocate funds with respect to the Choice Act, it did provide insight into the importance and feasibility of engaging veterans in the policy-making process. As more policies aimed at improving veterans’ access to civilian care are created, such as the most recent Mission Act, policy makers should strongly consider using the DD method of obtaining informed veteran input into future policy decisions.

Acknowledgments
Funding was provided by the US Department of Veterans Affairs Office of Analytics and Business Intelligence (OABI) and the VA Quality Enhancement Research Initiative (QUERI). Dr. Caverly was supported in part by a VA Career Development Award (CDA 16-151). Dr. Krein is supported by a VA Health Services Research and Development Research Career Scientist Award (RCS 11-222). The authors thank the veterans who participated in this work. They also thank Caitlin Reardon and Natalya Wawrin for their assistance in organizing the deliberation session.

Providing high-quality, patient-centered health care is a top priority for the US Department of Veterans Affairs (VA) Veteran Health Administration (VHA), whose core mission is to improve the health and well-being of US veterans. Thus, news of long wait times for medical appointments in the VHA sparked intense national attention and debate and led to changes in senior management and legislative action. ¹ On August 8, 2014, President Bara c k Obama signed the Veterans Access, Choice, and Accountability Act of 2014, also known as the Choice Act, which provided an additional $16 billion in emergency spending over 3 years to improve veterans’ access to timely health care. ² The Choice Act sought to develop an integrated health care network that allowed qualified VHA patients to receive specific health care services in their communities delivered by non-VHA health care providers (HCPs) but paid for by the VHA. The Choice Act also laid out explicit criteria for how to prioritize who would be eligible for VHA-purchased civilian care: (1) veterans who could not get timely appointments at a VHA medical facility within 30 days of referral; or (2) veterans who lived > 40 miles from the closest VHA medical facility.

VHA decision makers seeking to improve care delivery also need to weigh trade-offs between alternative approaches to providing rapid access. For instance, increasing access to non-VHA HCPs may not always decrease wait times and could result in loss of continuity, limited care coordination, limited ability to ensure and enforce high-quality standards at the VHA, and other challenges.^3-6 Although the concerns and views of elected representatives, advocacy groups, and health system leaders are important, it is unknown whether these views and preferences align with those of veterans. Arguably, the range of views and concerns of informed veterans whose health is at stake should be particularly prominent in such policy decision making.

To identify the considerations that were most important to veterans regarding VHA policy around decreasing wait times, a study was designed to engage a group of veterans who were eligible for civilian care under the Choice Act. The study took place 1 year after the Choice Act was passed. Veterans were asked to focus on 2 related questions: First, how should funding be used for building VHA capacity (build) vs purchasing civilian care (buy)? Second, under what circumstances should civilian care be prioritized?

The aim of this paper is to describe democratic deliberation (DD), a specific method that engaged veteran patients in complex policy decisions around access to care. DD methods have been used increasingly in health care for developing policy guidance, setting priorities, providing advice on ethical dilemmas, weighing risk-benefit trade-offs, and determining decision-making authority.^7-12 For example, DD helped guide national policy for mammography screening for breast cancer in New Zealand.¹³ The Agency for Healthcare Research and Quality has completed a systematic review and a large, randomized experiment on best practices for carrying out public deliberation.^8,13,14 However, despite the potential value of this approach, there has been little use of deliberative methods within the VHA for the explicit purpose of informing veteran health care delivery.

This paper describes the experience engaging veterans by using DD methodology and informing VHA leadership about the results of those deliberations. The specific aims were to understand whether DD is an acceptable approach to use to engage patients in the medical services policy-making process within VHA and whether veterans are able to come to an informed consensus.

Methods

Engaging patients and incorporating their needs and concerns within the policy-making process may improve health system policies and make those policies more patient centered. Such engagement also could be a way to generate creative solutions. However, because health-system decisions often involve making difficult trade-offs, effectively obtaining patient population input on complex care delivery issues can be challenging.

Although surveys can provide intuitive, top-of-mind input from respondents, these opinions are generally not sufficient for resolving complex problems.¹⁵ Focus groups and interviews may produce results that are more in-depth than surveys, but these methods tend to elicit settled private preferences rather than opinions about what the community should do.¹⁶ DD, on the other hand, is designed to elicit deeply informed public opinions on complex, value-laden topics to develop recommendations and policies for a larger community.¹⁷ The goal is to find collective solutions to challenging social problems. DD achieves this by giving participants an opportunity to explore a topic in-depth, question experts, and engage peers in reason-based discussions.^18,19 This method has its roots in political science and has been used over several decades to successfully inform policy making on a broad array of topics nationally and internationally—from health research ethics in the US to nuclear and energy policy in Japan.^7,16,20,21 DD has been found to promote ownership of public programs and lend legitimacy to policy decisions, political institutions, and democracy itself.¹⁸

A single day (8 hours) DD session was convened, following a Citizens Jury model of deliberation, which brings veteran patients together to learn about a topic, ask questions of experts, deliberate with peers, and generate a “citizen’s report” that contains a set of recommendations (Table 1). An overview of the different models of DD and rationale for each can be found elsewhere.^8,15

Recruitment Considerations

A purposively selected sample of civilian care-eligible veterans from a midwestern VHA health care system (1 medical center and 3 community-based outpatient clinics [CBOCs]) were invited to the DD session. The targeted number of participants was 30. Female veterans, who comprise only 7% of the local veteran population, were oversampled to account for their potentially different health care needs and to create balance between males and females in the session. Oversampling for other characteristics was not possible due to the relatively small sample size. Based on prior experience,⁷ it was assumed that 70% of willing participants would attend the session; therefore 34 veterans were invited and 24 attended. Each participant received a $200 incentive in appreciation for their substantial time commitment and to offset transportation costs.

Background Materials

A packet with educational materials (Flesch-Kincaid Grade Level of 10.5) was mailed to participants about 2 weeks before the DD session. Participants were asked to review prior to attending the session. These materials described the session (eg, purpose, organizers, importance) and provided factual information about the Choice Act (eg, eligibility, out-of-pocket costs, travel pay, prescription drug policies).

Session Overview

The session was structured to accomplish the following goals: (1) Elicit participants’ opinions about access to health care and reasons for those opinions; (2) Provide in-depth education about the Choice Act through presentations and discussions with topical experts; and (3) Elicit reasoning and recommendations on both the criteria by which participants prioritize candidates for civilian care and how participants would allocate additional funding to improve access (ie, by building VHA capacity to deliver more timely health care vs purchasing health care from civilian HCPs).

Participants were asked to fill out a survey on arrival in the morning and were assigned to 1 of 3 tables or small groups. Each table had a facilitator who had extensive experience in qualitative data collection methods and guided the dialogue using a scripted protocol that they helped develop and refine. The facilitation materials drew from and used previously published studies.^22,23 Each facilitator audio recorded the sessions and took notes. Three experts presented during plenary education sessions. Presentations were designed to provide balanced factual information and included a veteran’s perspective. One presenter was a clinician on the project team, another was a local clinical leader responsible for making decisions about what services to provide via civilian care (buy) vs enhancing the local VHA health system’s ability to provide those services (build), and the third presenter was a veteran who was on the project team.

Education Session 1

The first plenary education session with expert presentations was conducted after each table completed an icebreaker exercise. The project team physician provided a brief history and description of the Choice Act to reinforce educational materials sent to participants prior to the session. The health system clinical leader described his decision process and principles and highlighted constraints placed on him by the Choice Act that were in place at the time of the DD session. He also described existing local and national programs to provide civilian care (eg, local fee-basis non-VHA care programs) and how these programs sought to achieve goals similar to the Choice Act. The veteran presenter focused on the importance of session participants providing candid insight and observations and emphasized that this session was a significant opportunity to “have their voices heard.”

Deliberation 1: What criteria should be used to prioritize patients for receiving civilian care paid for by the VHA? To elicit preferences on the central question of this deliberation, participants were presented with 8 real-world cases that were based on interviews conducted with Choice Act-eligible veterans (Table 2 and eAppendices A

Education Session 2

In the second plenary session, the project team physician provided information about health care access issues, both inside and outside of the VHA, particularly between urban and rural areas. He also discussed factors related to the insufficient capacity to meet growing demand that contributed to the VHA wait-time crisis. The veteran presenter shared reflections on health care access from a veteran’s perspective.

Deliberation 2: How should additional funding be divided between increasing the ability of the VHA to (1) provide care by VHA HCPs; and (2) pay for care from non-VHA civilian HCPs? Participants were presented the patient examples and Choice Act funding scenarios (the buy policy option) and contrasted that with a build policy option. Participants were explicitly encouraged to shift their perspectives from thinking about individual cases to considering policy-level decisions and the broader social good (Table 2).

Ensuring Robust Deliberations

If participants do not adequately grasp the complexities of the topic, a deliberation can fail. To facilitate nuanced reasoning, real-world concrete examples were developed as the starting point of each deliberation based on interviews with actual patients (deliberation 1) and actual policy proposals relevant to the funding allocation decisions within the Choice Act (deliberation 2).

A deliberation also can fail with self-silencing, where participants withhold opinions that differ from those articulated first or by more vocal members of the group.²⁴ To combat self-silencing, highly experienced facilitators were used to ensure sharing from all participants and to support an open-minded, courteous, and reason-based environment for discourse. It was specified that the best solutions are achieved through reason-based and cordial disagreement and that success can be undermined when participants simply agree because it is easier or more comfortable.

A third way a deliberation can fail is if individuals do not adopt a group or system-level perspective. To counter this, facilitators reinforced at multiple points the importance of taking a broader social perspective rather than sharing only one’s personal preferences.

Finally, it is important to assess the quality of the deliberative process itself, to ensure that results are trustworthy.²⁵ To assess the quality of the deliberative process, participants knowledge about key issues pre- and postdeliberation were assessed. Participants also were asked to rate the quality of the facilitators and how well they felt their voice was heard and respected, and facilitators made qualitative assessments about the extent to which participants were engaged in reason-based and collaborative discussion.

Data

Quantitative data were collected via pre- and postsession surveys. The surveys contained items related to knowledge about the Choice Act, expectations for the DD session, beliefs and opinions about the provision of health care for veterans, recommended funding allocations between build vs buy policy options, and general demographics. Qualitative data were collected through detailed notes taken by the 3 facilitators. Each table’s deliberations were audio recorded so that gaps in the notes could be filled.

The 3 facilitators, who were all experienced qualitative researchers, typed their written notes into a template immediately after the session. Two of the 3 facilitators led the analysis of the session notes. Findings within and across the 3 deliberation tables were developed using content and matrix analysis methods.²⁶ Descriptive statistics were generated from survey responses and compared survey items pre- and postsession using paired t tests or χ² tests for categorical responses.

Results

Thirty-three percent of individuals invited (n = 127) agreed to participate. Those who declined cited conflicts related to distance, transportation, work/school, medical appointments, family commitments, or were not interested. In all, 24 (69%) of the 35 veterans who accepted the invitation attended the deliberation session. Of the 11 who accepted but did not attend, 5 cancelled ahead of time because of conflicts (Figure). Most participants were male (70%), 48% were aged 61 to 75 years, 65% were white, 43% had some college education, 43% reported an annual income of between $25,000 and $40,000, and only 35% reported very good health (eAppendix D).

Deliberation 1

During the deliberation on the prioritization criteria, the concept of “condition severity” emerged as an important criterion for veterans. This criterion captured simultaneous consideration of both clinical necessity and burden on the veteran to obtain care. For example, participants felt that patients with a life-threatening illness should be prioritized for civilian care over patients who need preventative or primary care (clinical necessity) and that elderly patients with substantial difficulty traveling to VHA appointments should be prioritized over patients who can travel more easily (burden). The Choice Act regulations at the time of the DD session did not reflect this nuanced perspective, stipulating only that veterans must live > 40 miles from the nearest VHA medical facility.

One of the 3 groups did not prioritize the patient cases because some members felt that no veteran should be constrained from receiving civilian care if they desired it. Nonetheless, this group did agree with prioritizing the first 2 cases in Table 3. The other groups prioritized all 8 cases in generally similar ways.

Deliberation 2

No clear consensus emerged on the buy vs build question. A representative from each table presented their group’s positions, rationale, and recommendations after deliberations were completed. After hearing the range of positions, the groups then had another opportunity to deliberate based on what they heard from the other tables; no new recommendations or consensus emerged.

Participants who were in favor of allocating more funds toward the build policy offered a range of rationales, saying that it would (1) increase access for rural veterans by building CBOCs and deploying more mobile units that could bring outlets for health care closer to their home communities; (2) provide critical and unique medical expertise to address veteran-specific issues such as prosthetics, traumatic brain injury, posttraumatic stress disorder, spinal cord injury, and shrapnel wounds that are typically not available through civilian providers; (3) give VHA more oversight over the quality and cost of care, which is more challenging to do with civilian providers; and (4) Improve VHA infrastructure by, for example, upgrading technology and attracting the best clinicians and staff to support “our VHA.”

Participants who were in favor of allocating more funds toward the buy policy also offered a range of rationales, saying that it would (1) decrease patient burden by increasing access through community providers, decreasing wait time, and lessening personal cost and travel time; (2) allow more patients to receive civilian care, which was generally seen as beneficial by a few participants because of perceptions that the VHA provides lower quality care due to a shortage of VHA providers, run-down/older facilities, lack of technology, and poorer-quality VHA providers; and (3) provide an opportunity to divest of costly facilities and invest in other innovative approaches. Regarding this last reason, a few participants felt that the VHA is “gouged” when building medical centers that overrun budgets. They also were concerned that investing in facilities tied VHA to specific locations when current locations of veterans may change “25 years from now.”

Survey Results

Twenty-three of the 24 participants completed both pre- and postsession surveys. The majority of participants in the session felt people in the group respected their opinion (96%); felt that the facilitator did not try to influence the group with her own opinions (96%); indicated they understood the information enough to participate as much as they wanted (100%); and were hopeful that their reasoning and recommendations would help inform VHA policy makers (82%).

The surveys also provided an opportunity to examine the extent to which knowledge, attitudes, and opinions changed from before to after the deliberation. Even with the small sample, responses revealed a trend toward improved knowledge about key elements of the Choice Act and its goals. Further, there was a shift in some participants’ opinions about how patients should be prioritized to receive civilian care. For example, before the deliberation participants generally felt that all veterans should be able to receive civilian care, whereas postdeliberation this was not the case. Postdeliberation, most participants felt that primary care should not be a high priority for civilian care but continued to endorse prioritizing civilian care for specialty services like orthopedic or cardiology-related care. Finally, participants moved from more diverse recommendations regarding additional funds allocations, toward consensus after the deliberation around allocating funds to the build policy. Eight participants supported a build policy beforehand, whereas 16 supported this policy afterward.

Discussion

This study explored DD as a method for deeply engaging veterans in complex policy making to guide funding allocation and prioritization decisions related to the Choice Act, decisions that are still very relevant today within the context of the Mission Act and have substantial implications for how health care is delivered in the VHA. The Mission Act passed on June 6, 2018, with the goal of improving access to and the reliability of civilian or community care for eligible veterans.²⁷ Decisions related to appropriating scarce funding to improve access to care is an emotional and value-laden topic that elicited strong and divergent opinions among the participants. Veterans were eager to have their voices heard and had strong expectations that VHA leadership would be briefed about their recommendations. The majority of participants were satisfied with the deliberation process, felt they understood the issues, and felt their opinions were respected. They expressed feelings of comradery and community throughout the process.

In this single deliberation session, the groups did not achieve a single, final consensus regarding how VHA funding should ultimately be allocated between buy and build policy options. Nonetheless, participants provided a rich array of recommendations and rationale for them. Session moderators observed rich, sophisticated, fair, and reason-based discussions on this complex topic. Participants left with a deeper knowledge and appreciation for the complex trade-offs and expressed strong rationales for both sides of the policy debate on build vs buy. In addition, the project yielded results of high interest to VHA policy makers.

This work was presented in multiple venues between 2015 to 2016, and to both local and national VHA leadership, including the local Executive Quality Leadership Boards, the VHA Central Office Committee on the Future State of VA Community Care, the VA Office of Patient Centered Care, and the National Veteran Experience Committee. Through these discussions and others, we saw great interest within the VHA system and high-level leaders to explore ways to include veterans’ voices in the policy-making process. This work was invaluable to our research team (eAppendix E 

Many health system decisions regarding what care should be delivered (and how) involve making difficult, value-laden choices in the context of limited resources. DD methods can be used to target and obtain specific viewpoints from diverse populations, such as the informed perspectives of minority and underrepresented populations within the VHA.¹⁹ For example, female veterans were oversampled to ensure that the informed preferences of this population was obtained. Thus, DD methods could provide a valuable tool for health systems to elicit in-depth diverse patient input on high-profile policies that will have a substantial impact on the system’s patient population.

Limitations

One potential downside of DD is that, because of the resource-intensive nature of deliberation sessions, they are often conducted with relatively small groups.⁹ Viewpoints of those within these small samples who are willing to spend an entire day discussing a complex topic may not be representative of the larger patient community. However, the core goal of DD is diversity of opinions rather than representativeness.

A stratified random sampling strategy that oversampled for underrepresented and minority populations was used to help select a diverse group that represents the population on key characteristics and partially addresses concern about representativeness. Efforts to optimize participation rates, including providing monetary incentives, also are helpful and have led to high participation rates in past deliberations.⁷

Health system communication strategies that promote the importance of becoming involved in DD sessions also may be helpful in improving rates of recruitment. On particularly important topics where health system leaders feel a larger resource investment is justified, conducting larger scale deliberations with many small groups may obtain more generalizable evidence about what individual patients and groups of patients recommend.⁷ However, due to the inherent limitations of surveys and focus group approaches for obtaining informed views on complex topics, there are no clear systematic alternatives to the DD approach.

Conclusion

DD is an effective method to meaningfully engage patients in deep deliberations to guide complex policy making. Although design of deliberative sessions is resource-intensive, patient engagement efforts, such as those described in this paper, could be an important aspect of a well-functioning learning health system. Further research into alternative, streamlined methods that can also engage veterans more deeply is needed. DD also can be combined with other approaches to broaden and confirm findings, including focus groups, town hall meetings, or surveys.

Although this study did not provide consensus on how the VHA should allocate funds with respect to the Choice Act, it did provide insight into the importance and feasibility of engaging veterans in the policy-making process. As more policies aimed at improving veterans’ access to civilian care are created, such as the most recent Mission Act, policy makers should strongly consider using the DD method of obtaining informed veteran input into future policy decisions.

Acknowledgments
Funding was provided by the US Department of Veterans Affairs Office of Analytics and Business Intelligence (OABI) and the VA Quality Enhancement Research Initiative (QUERI). Dr. Caverly was supported in part by a VA Career Development Award (CDA 16-151). Dr. Krein is supported by a VA Health Services Research and Development Research Career Scientist Award (RCS 11-222). The authors thank the veterans who participated in this work. They also thank Caitlin Reardon and Natalya Wawrin for their assistance in organizing the deliberation session.