Opinion

In my introductory comments to the practice management section last year, I wrote about cultivating competencies for value-based care. One of the key competencies was patient centeredness. Patient-reported outcomes (PROs) and patient experience measures specifically were highlighted as examples of meaningful tools for achieving patient centeredness. Starting with this month’s contribution by Drs Reed and Dellon on PROs in esophageal disease, we begin a series of articles focused on this important construct. We will follow this article with reports focused on PRO for patients with irritable bowel syndrome, inflammatory bowel disease, and chronic liver disease. These reports will not only review the importance of PROs, but also highlight the most practical approaches to measuring disease-specific PROs in clinical practice all with the goal of improving the care of our patients.
Ziad Gellad, MD, MPH, AGAF, Special Section Editor
 

Patients seek medical care for symptoms affecting their quality of life,¹ and this is particularly true of digestive diseases, in which many common conditions are symptom predominant. However, clinician and patient perception of symptoms often conflict,² and formalized measurement tools may have a role for optimizing symptom assessment. Patient-reported outcomes (PROs) directly capture patients’ health status from their own perspectives and can bridge the divide between patient and provider interpretation. The US Food and Drug Administration (FDA) defines PROs as “any report of the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else.”³

For the clinical assessment of esophageal diseases, existing physiologic and structural testing modalities cannot ascertain patient disease perception or measure the impact of symptoms on health care–associated quality of life. In contrast, by capturing patient-centric data, PROs can provide insight into the psychosocial aspects of patient disease perceptions; capture health-related quality of life (HRQL); improve provider understanding; highlight discordance between physiologic, symptom, and HRQL measures; and formalize follow-up evaluation of treatment response.^1,4 Following up symptoms such as dysphagia or heartburn over time in a structured way allows clinically obtained data to be used in pragmatic or comparative effectiveness studies. PROs are now an integral part of the FDA’s drug approval process.

In this article, we review the available PROs capturing esophageal symptoms with a focus on dysphagia and heartburn measures that were developed with rigorous methodology; it is beyond the scope of this article to perform a thorough review of all upper gastrointestinal (GI) PROs or quality-of-life PROs. We then discuss how esophageal PROs may be incorporated into clinical practice now, as well as opportunities for PRO use in the future.
 

Esophageal symptom-specific patient-reported outcomes

The literature pertinent to upper GI and esophageal-specific PROs is heterogeneous, and the development of PROs has been variable in rigor. Two recent systematic reviews identified PROs pertinent to dysphagia and heartburn (Table 1) and both emphasized rigorous measures developed in accordance with FDA guidance.³

Patel et al⁵ identified 34 dysphagia-specific PRO measures, of which 10 were rigorously developed (Table 1). These measures encompassed multiple conditions including esophageal cancer (Functional Assessment of Cancer Therapy Esophageal Cancer Subscale, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal Cancer 25 items, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal cancer 18 items, upper aerodigestive neoplasm-attributable oropharyngeal dysphagia [M.D. Anderson dysphagia inventory], mechanical and neuromyogenic oropharyngeal dysphagia [swallow quality-of-life questionnaire], Sydney Swallow Questionnaire, [swallowing quality of care], achalasia [Measure of Achalasia Disease Severity], eosinophilic esophagitis [Dysphagia Symptom Questionnaire], and general dysphagia symptoms and gastroesophageal reflux [Patient-Reported Outcomes Measurement Information System Gastrointestinal Symptom Scales (PROMIS-GI)]. PROMIS-GI, produced as part of the National Institutes of Health PROMIS program, includes rigorous measures for general dysphagia symptoms and gastroesophageal reflux in addition to lower gastrointestinal symptom measures.

The systematic review by Vakil et al⁶ found 15 PRO measures for gastroesophageal reflux disease (GERD) symptoms that underwent psychometric evaluation (Table 1). Of these, 5 measures were devised according to the developmental steps stipulated by the US FDA and the European Medicines Agency, and each measure has been used as an end point for a clinical trial. The 5 measures include the GERD Symptom Assessment Scale, the Nocturnal Gastro-oesophageal Reflux Disease Symptom Severity and Impact Questionnaire, the Reflux Questionnaire, the Reflux Disease Questionnaire, and the Proton Pump Inhibitor Acid Suppression Symptom Test (Table 1). Additional PROs capturing esophageal symptoms include the eosinophilic esophagitis symptom activity index, Eckardt score (used for achalasia), Mayo dysphagia questionnaire, and GERD-Q (Table 1).

Utilization of esophageal patient-reported outcomes in practice

Before incorporating a PRO into clinical practice, providers must appreciate the construct(s), intent, developmental measurement properties, validation strategies, and responsiveness characteristics associated with the measure.⁴ PROs can be symptom- and/or condition-specific. For example, this could include dysphagia associated with achalasia or eosinophilic esophagitis, postoperative dysphagia from spine surgery, or general dysphagia symptoms regardless of the etiology (Table 1). Intent refers to the context in which a PRO should be used and generally is stratified into 3 areas: population surveillance, individual patient-clinician interactions, and research studies.⁴ A thorough analysis of PRO developmental properties exceeds the scope of this article. However, several key considerations are worth discussing. Each measure should clearly delineate the construct, or outcome, in addition to the population used to create the measure (eg, patients with achalasia). PROs should be assessed for reliability, construct validity, and content validity. Reliability pertains to the degree in which scores are free from measurement error, the extent to which items (ie, questions) correlate, and test–retest reliability. Construct validity includes dimensionality (evidence of whether a single or multiple subscales exist in the measure), responsiveness to change (longitudinal validity), and convergent validity (correlation with additional construct-specific measures). Central to the PRO development process is the involvement of patients and content experts (content validity). PRO measures should be readily interpretable, and the handling of missing items should be stipulated. The burden, or time required for administering and scoring the instrument, and the reading level of the PRO need to be considered.⁸ In short, a PRO should measure something important to patients, in a way that patients can understand, and in a way that accurately reflects the underlying symptom and disease.

Although PROs traditionally represent a method for gathering data for research, they also should be viewed as a means of improving clinical care. The monitoring of change in a particular construct represents a common application of PROs in clinical practice. This helps quantify the efficacy of an intervention and can provide insight into the comparative effectiveness of alternative therapies. For example, in a patient with an esophageal stricture, a dysphagia-specific measure could be used at baseline before an endoscopy and dilation, in follow-up evaluation after dilation, and then as a monitoring tool to determine when repeat dilation may be needed. Similarly, the Eckardt score has been used commonly to monitor response to achalasia treatments. Clinicians also may use PROs in real time to optimize patient management. The data gathered from PROs may help triage patients into treatment pathways, trigger follow-up appointments, supply patient education prompts, and produce patient and provider alerts.⁸ For providers engaging in clinical research, PROs administered at the point of patient intake, whether electronically through a patient portal or in the clinic, provide a means of gathering baseline data.⁹ A key question, however, is whether it is practical to use a PRO routinely in the clinic, esophageal function laboratory, or endoscopy suite.

These practical issues include cultivating a conducive environment for PRO utilization, considering the burden of the measure on the patient, and utilization of the results in an expedient manner.⁹ To promote seamless use of a PRO in clinical work-flows, a multimodal means of collecting PRO data should be arranged. Electronic PROs available through a patient portal, designed with a user-friendly and intuitive interface, facilitate patient completion of PROs at their convenience, and ideally before a clinical or procedure visit. For patients without access to the internet, tablets and/or computer terminals within the office are convenient options. Nurses or clinic staff also could help patients complete a PRO during check-in for clinic, esophageal testing, or endoscopy. The burden a PRO imposes on patients also limits the utility of a measure. For instance, PROs with a small number of questions are more likely to be completed, while scales consisting of 30 of more items are infrequently finished. Clinicians also should consider how they plan to use the results of a PRO before implementing one; if the data will not be used, then the effort to implement and collect it will be wasted. Moreover, patients will anticipate that the time required to complete a PRO will translate to an impact on their management plan and will more readily complete additional PROs if previous measures expediently affected their care.⁹

Barriers to patient-reported outcome implementation and future directions

Given the potential benefits to PRO use, why are they not implemented routinely? In practice, there are multiple barriers that thwart the adoption of PROs into both health care systems and individual practices. The integration of PROs into large health care systems languishes partly because of technological and operational barriers.⁹ For instance, the manual distribution, collection, and transcription of handwritten information requires substantial investitures of time, which is magnified by the number of patients whose care is provided within a large health system. One approach to the technological barrier includes the creation of an electronic platform integrating with patient portals. Such a platform would obviate the need to manually collect and transcribe documents, and could import data directly into provider documentation and flowsheets. However, the programming time and costs are substantial upfront, and without clear data that this could lead to improved outcomes or decreased costs downstream there may be reluctance to devote resources to this. In clinical practice, the already significant demands on providers’ time mitigates enthusiasm to add additional tasks. Providers also could face annual licensing agreements, fees on a per-study basis, or royalties associated with particular PROs, and at the individual practice level, there may not be appropriate expertise to select and implement routine PRO monitoring. To address this, efforts are being made to simplify the process of incorporating PROs. For example, given the relatively large number of heterogeneous PROs, the PROMIS project1 endeavors to clarify which PROs constitute the best measure for each construct and condition.⁹ The PROMIS measures also are provided publicly and are available without license or fee.

Areas particularly well situated for growth in the use of PRO measures include comparative effectiveness studies and pragmatic clinical trials. PRO-derived data may promote a shift from explanatory randomized controlled trials to pragmatic randomized controlled trials because these data emphasize patient-centered care and are more broadly generalizable to clinical settings. Furthermore, the derivation of data directly from the health care delivery system through PROs, such as two-way text messages, increases the relevance and cost effectiveness of clinical trials. Given the current medical climate, pressures continue to mount to identify cost-efficient and efficacious medical therapies.¹⁰ In this capacity, PROs facilitate the understanding of changes in HRQL domains subject to treatment choices. PROs further consider the comparative symptom burden and side effects associated with competing treatment strategies.¹¹ Finally, PROs also have enabled the procurement of data from patient-powered research networks. Although this concept has not yet been applied to esophageal diseases, one example of this in the GI field is the Crohn’s and Colitis Foundation of America Partners project, which has built an internet cohort consisting of approximately 14,200 inflammatory bowel disease patients who are monitored with a series of PROs.¹² An endeavor such as this should be a model for esophageal conditions in the future.
 

Conclusions

PROs, as a structured means of directly assessing symptoms, help facilitate a provider’s understanding from a patient’s perspectives. Multiple PROs have been developed to characterize constructs pertinent to esophageal diseases and symptoms. These vary in methodologic rigor, but multiple well-constructed PROs exist for symptom domains such as dysphagia and heartburn, and can be used to monitor symptoms over time and assess treatment efficacy. Implementation of esophageal PROs, both in large health systems and in routine clinical practice, is not yet standard and faces a number of barriers. However, the potential benefits are substantial and include increased patient-centeredness, more accurate and timely disease monitoring, and applicability to comparative effectiveness studies, pragmatic clinical trials, and patient-powered research networks.
 

References

1. Spiegel B., Hays R., Bolus R., et al. Development of the NIH Patient-Reported Outcomes Measurement Information System (PROMIS) gastrointestinal symptom scales. Am J Gastroenterol. 2014;109:1804-14.

2. Chassany O., Shaheen N.J., Karlsson M., et al. Systematic review: symptom assessment using patient-reported outcomes in gastroesophageal reflux disease and dyspepsia. Scand J Gastroenterol. 2012;47:1412-21.

3. U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research, U.S. Department of Health and Human Services FDA Center for Biologics Evaluation and Research, U.S. Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79. Available from:http://www.ncbi.nlm.nih.gov/pubmed/17034633%0Ahttp://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=PMC1629006

Accessed May 23, 2017

4. Lipscomb J. Cancer outcomes research and the arenas of application. J Natl Cancer Inst Monogr. 2004;2004:1-7.

5. Patel D.A., Sharda R., Hovis K.L., et al. Patient-reported outcome measures in dysphagia: a systematic review of instrument development and validation. Dis Esophagus. 2017;30:1-23.

6. Vakil N.B., Halling K., Becher A., et al. Systematic review of patient-reported outcome instruments for gastroesophageal reflux disease symptoms. Eur J Gastroenterol Hepatol. 2013;25:2-14.

7. Bedell A., Taft T.H., Keefer L. Development of the Northwestern Esophageal Quality of Life Scale: a hybrid measure for use across esophageal conditions. Am J Gastroenterol. 2016;111:493-9.

8. Farnik M., Pierzchala W. Instrument development and evaluation for patient-related outcomes assessments. Patient Relat Outcome Meas. 2012;3:1-7.

9. Wagle N.W.. Implementing patient-reported outcome measures (PROMs). N Engl J Med Catal. 2016; :1-2. Available from:

http://catalyst.nejm.org/implementing-proms-patient-reported-outcome-measures/. Accessed July 14, 2017

10. Richesson R.L., Hammond W.E., Nahm M., et al. Electronic health records based phenotyping in next-generation clinical trials: a perspective from the NIH Health Care Systems Collaboratory. J Am Med Informatics Assoc. 2013;20: e226-e231.

11. Coon C.D., McLeod L.D. Patient-reported outcomes: current perspectives and future directions. Clin Ther. 2013;35:399-401.

12. Chung A.E., Sandler R.S., Long M.D., et al. Harnessing person-generated health data to accelerate patient-centered outcomes research: The Crohn’s and Colitis Foundation of America PCORnet Patient Powered Research Network (CCFA Partners)

J Am Med Informatics Assoc. 2016;23:485-90.

13. Darling G., Eton D.T., Sulman J., et al. Validation of the functional assessment of cancer therapy esophageal cancer subscale. Cancer. 2006;107:854-63.

14. Lagergren P., Fayers P., Conroy T., et al. Clinical and psychometric validation of a questionnaire module, the EORTC QLQ-OG25, to assess health-related quality of life in patients with cancer of the oesophagus, the oesophago-gastric junction and the stomach. Eur J Cancer. 2007;43:2066-73.

15. Blazeby J.M., Conroy T., Hammerlid E., et al. Clinical and psychometric validation of an EORTC questionnaire module, the EORTC QLQ-OES18, to assess quality of life in patients with oesophageal cancer. Eur J Cancer. 2003;39:1384-94.

16. Chen A.Y., Frankowski R., Bishop-Leone J., et al. The development and validation of a dysphagia-specific quality-of-life questionnaire for patients with head and neck cancer: the M. D. Anderson dysphagia inventory. Arch Otolaryngol Head Neck Surg. 2001;127:870-6.

17. McHorney C.A., Bricker D.E., Robbins J., et al. The SWAL-QOL outcomes tool for oropharyngeal dysphagia in adults: II. item reduction and preliminary scaling. Dysphagia. 2000;15:122-33.

18. Wallace K.L., Middleton S., Cook I.J. Development and validation of a self-report symptom inventory to assess the severity of oral-pharyngeal dysphagia. Gastroenterology. 2000;118:678-87.

19. McHorney C.A., Robbins J.A., Lomax K., et al. The SWAL-QOL and SWAL-CARE outcomes tool for oropharyngeal dysphagia in adults: III. Documentation of reliability and validity. Dysphagia. 2002;17:97-114.

20. Urbach D.R., Tomlinson G.A., Harnish J.L., et al. A measure of disease-specific health-related quality of life for achalasia. Am J Gastroenterol. 2005;100:1668-76.

21. Eckardt V., Aignherr C., Bernhard G. Predictors of outcome in patients with achalasia treated by pneumatic dilation. Gastroenterology. 1992;103:1732-8.

22. Dellon E.S., Irani A.M., Hill M.R., et al. Development and field testing of a novel patient-reported outcome measure of dysphagia in patients with eosinophilic esophagitis. Aliment Pharmacol Ther. 2013;38:634-42.

23. Franciosi J.P., Hommel K., DeBrosse C.W., et al. Development of a validated patient-reported symptom metric for pediatric eosinophilic esophagitis: qualitative methods. BMC Gastroenterol. 2011;11:126.

24. Schoepfer A.M., Straumann A., Panczak R., et al. Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. Gastroenterology. 2014;147:1-24.

25. Grudell A.B., Alexander J.A., Enders F.B., et al. Validation of the Mayo Dysphagia Questionnaire. Dis Esophagus. 2007;20:202-5.

26. Rothman M., Farup C., Steward W., et al. Symptoms associated with gastroesophageal reflux disease: Development of a questionnaire for use in clinical trials. Dig Dis Sci. 2001;46:1540-9.

27. Spiegel B.M., Roberts L., Mody R., et al. The development and validation of a nocturnal gastro-oesophageal reflux disease symptom severity and impact questionnaire for adults. Aliment Pharmacol Ther. 2010;32:591-602.

28. Bardhan K.D., Stanghellini V., Armstrong D., et al. International validation of ReQuest in patients with endoscopy-negative gastro-oesophageal reflux disease. Aliment Pharmacol Ther. 2004;20:891-8.

29. Van Zanten S.V., Armstrong D., Barkun A., et al. Symptom overlap in patients with upper gastrointestinal complaints in the Canadian confirmatory acid suppression test (CAST) study: Further psychometric validation of the reflux disease questionnaire. Aliment Pharmacol Ther. 2007;25:1087-97.

30. Armstrong D., Moayyedi P., Hunt R., et al. M1870 resolution of persistent GERD symptoms after a change in therapy: EncomPASS - a cluster-randomized study in primary care. Gastroenterology. 2009;136(Suppl 1):A-435.

31. Jones R., Junghard O., Dent J., et al. Developement of the GerdQ, a tool for the diagnosis and management of gastro-oesophageal reflux disease in primary care. Aliment Pharmacol Ther. 2009;30:1030-8.


Dr. Reed is a senior fellow and Dr. Dillon is an associate professor of medicine and epidemiology, Center for Esophageal Diseases and Swallowing, division of gastroenterology and hepatology, University of North Carolina School of Medicine, Chapel Hill. Dr. Dellon has received research funding from Adare, Allakos, GSK, Meritage, Miraca, Nutricia, Celgene/Receptos, Regeneron, and Shire; he has been a consultant for Adare, Alivio, Allakos, AstraZeneca, Banner, Calypso, Enumeral, EsoCap, Celgene/Receptos, GSK, Regeneron, Robarts, and Shire; and has received an educational grant from Banner and Holoclara.

In my introductory comments to the practice management section last year, I wrote about cultivating competencies for value-based care. One of the key competencies was patient centeredness. Patient-reported outcomes (PROs) and patient experience measures specifically were highlighted as examples of meaningful tools for achieving patient centeredness. Starting with this month’s contribution by Drs Reed and Dellon on PROs in esophageal disease, we begin a series of articles focused on this important construct. We will follow this article with reports focused on PRO for patients with irritable bowel syndrome, inflammatory bowel disease, and chronic liver disease. These reports will not only review the importance of PROs, but also highlight the most practical approaches to measuring disease-specific PROs in clinical practice all with the goal of improving the care of our patients.
Ziad Gellad, MD, MPH, AGAF, Special Section Editor
 

Patients seek medical care for symptoms affecting their quality of life,¹ and this is particularly true of digestive diseases, in which many common conditions are symptom predominant. However, clinician and patient perception of symptoms often conflict,² and formalized measurement tools may have a role for optimizing symptom assessment. Patient-reported outcomes (PROs) directly capture patients’ health status from their own perspectives and can bridge the divide between patient and provider interpretation. The US Food and Drug Administration (FDA) defines PROs as “any report of the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else.”³

For the clinical assessment of esophageal diseases, existing physiologic and structural testing modalities cannot ascertain patient disease perception or measure the impact of symptoms on health care–associated quality of life. In contrast, by capturing patient-centric data, PROs can provide insight into the psychosocial aspects of patient disease perceptions; capture health-related quality of life (HRQL); improve provider understanding; highlight discordance between physiologic, symptom, and HRQL measures; and formalize follow-up evaluation of treatment response.^1,4 Following up symptoms such as dysphagia or heartburn over time in a structured way allows clinically obtained data to be used in pragmatic or comparative effectiveness studies. PROs are now an integral part of the FDA’s drug approval process.

In this article, we review the available PROs capturing esophageal symptoms with a focus on dysphagia and heartburn measures that were developed with rigorous methodology; it is beyond the scope of this article to perform a thorough review of all upper gastrointestinal (GI) PROs or quality-of-life PROs. We then discuss how esophageal PROs may be incorporated into clinical practice now, as well as opportunities for PRO use in the future.
 

Esophageal symptom-specific patient-reported outcomes

The literature pertinent to upper GI and esophageal-specific PROs is heterogeneous, and the development of PROs has been variable in rigor. Two recent systematic reviews identified PROs pertinent to dysphagia and heartburn (Table 1) and both emphasized rigorous measures developed in accordance with FDA guidance.³

Patel et al⁵ identified 34 dysphagia-specific PRO measures, of which 10 were rigorously developed (Table 1). These measures encompassed multiple conditions including esophageal cancer (Functional Assessment of Cancer Therapy Esophageal Cancer Subscale, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal Cancer 25 items, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal cancer 18 items, upper aerodigestive neoplasm-attributable oropharyngeal dysphagia [M.D. Anderson dysphagia inventory], mechanical and neuromyogenic oropharyngeal dysphagia [swallow quality-of-life questionnaire], Sydney Swallow Questionnaire, [swallowing quality of care], achalasia [Measure of Achalasia Disease Severity], eosinophilic esophagitis [Dysphagia Symptom Questionnaire], and general dysphagia symptoms and gastroesophageal reflux [Patient-Reported Outcomes Measurement Information System Gastrointestinal Symptom Scales (PROMIS-GI)]. PROMIS-GI, produced as part of the National Institutes of Health PROMIS program, includes rigorous measures for general dysphagia symptoms and gastroesophageal reflux in addition to lower gastrointestinal symptom measures.

The systematic review by Vakil et al⁶ found 15 PRO measures for gastroesophageal reflux disease (GERD) symptoms that underwent psychometric evaluation (Table 1). Of these, 5 measures were devised according to the developmental steps stipulated by the US FDA and the European Medicines Agency, and each measure has been used as an end point for a clinical trial. The 5 measures include the GERD Symptom Assessment Scale, the Nocturnal Gastro-oesophageal Reflux Disease Symptom Severity and Impact Questionnaire, the Reflux Questionnaire, the Reflux Disease Questionnaire, and the Proton Pump Inhibitor Acid Suppression Symptom Test (Table 1). Additional PROs capturing esophageal symptoms include the eosinophilic esophagitis symptom activity index, Eckardt score (used for achalasia), Mayo dysphagia questionnaire, and GERD-Q (Table 1).

Utilization of esophageal patient-reported outcomes in practice

Before incorporating a PRO into clinical practice, providers must appreciate the construct(s), intent, developmental measurement properties, validation strategies, and responsiveness characteristics associated with the measure.⁴ PROs can be symptom- and/or condition-specific. For example, this could include dysphagia associated with achalasia or eosinophilic esophagitis, postoperative dysphagia from spine surgery, or general dysphagia symptoms regardless of the etiology (Table 1). Intent refers to the context in which a PRO should be used and generally is stratified into 3 areas: population surveillance, individual patient-clinician interactions, and research studies.⁴ A thorough analysis of PRO developmental properties exceeds the scope of this article. However, several key considerations are worth discussing. Each measure should clearly delineate the construct, or outcome, in addition to the population used to create the measure (eg, patients with achalasia). PROs should be assessed for reliability, construct validity, and content validity. Reliability pertains to the degree in which scores are free from measurement error, the extent to which items (ie, questions) correlate, and test–retest reliability. Construct validity includes dimensionality (evidence of whether a single or multiple subscales exist in the measure), responsiveness to change (longitudinal validity), and convergent validity (correlation with additional construct-specific measures). Central to the PRO development process is the involvement of patients and content experts (content validity). PRO measures should be readily interpretable, and the handling of missing items should be stipulated. The burden, or time required for administering and scoring the instrument, and the reading level of the PRO need to be considered.⁸ In short, a PRO should measure something important to patients, in a way that patients can understand, and in a way that accurately reflects the underlying symptom and disease.

Although PROs traditionally represent a method for gathering data for research, they also should be viewed as a means of improving clinical care. The monitoring of change in a particular construct represents a common application of PROs in clinical practice. This helps quantify the efficacy of an intervention and can provide insight into the comparative effectiveness of alternative therapies. For example, in a patient with an esophageal stricture, a dysphagia-specific measure could be used at baseline before an endoscopy and dilation, in follow-up evaluation after dilation, and then as a monitoring tool to determine when repeat dilation may be needed. Similarly, the Eckardt score has been used commonly to monitor response to achalasia treatments. Clinicians also may use PROs in real time to optimize patient management. The data gathered from PROs may help triage patients into treatment pathways, trigger follow-up appointments, supply patient education prompts, and produce patient and provider alerts.⁸ For providers engaging in clinical research, PROs administered at the point of patient intake, whether electronically through a patient portal or in the clinic, provide a means of gathering baseline data.⁹ A key question, however, is whether it is practical to use a PRO routinely in the clinic, esophageal function laboratory, or endoscopy suite.

These practical issues include cultivating a conducive environment for PRO utilization, considering the burden of the measure on the patient, and utilization of the results in an expedient manner.⁹ To promote seamless use of a PRO in clinical work-flows, a multimodal means of collecting PRO data should be arranged. Electronic PROs available through a patient portal, designed with a user-friendly and intuitive interface, facilitate patient completion of PROs at their convenience, and ideally before a clinical or procedure visit. For patients without access to the internet, tablets and/or computer terminals within the office are convenient options. Nurses or clinic staff also could help patients complete a PRO during check-in for clinic, esophageal testing, or endoscopy. The burden a PRO imposes on patients also limits the utility of a measure. For instance, PROs with a small number of questions are more likely to be completed, while scales consisting of 30 of more items are infrequently finished. Clinicians also should consider how they plan to use the results of a PRO before implementing one; if the data will not be used, then the effort to implement and collect it will be wasted. Moreover, patients will anticipate that the time required to complete a PRO will translate to an impact on their management plan and will more readily complete additional PROs if previous measures expediently affected their care.⁹

Barriers to patient-reported outcome implementation and future directions

Given the potential benefits to PRO use, why are they not implemented routinely? In practice, there are multiple barriers that thwart the adoption of PROs into both health care systems and individual practices. The integration of PROs into large health care systems languishes partly because of technological and operational barriers.⁹ For instance, the manual distribution, collection, and transcription of handwritten information requires substantial investitures of time, which is magnified by the number of patients whose care is provided within a large health system. One approach to the technological barrier includes the creation of an electronic platform integrating with patient portals. Such a platform would obviate the need to manually collect and transcribe documents, and could import data directly into provider documentation and flowsheets. However, the programming time and costs are substantial upfront, and without clear data that this could lead to improved outcomes or decreased costs downstream there may be reluctance to devote resources to this. In clinical practice, the already significant demands on providers’ time mitigates enthusiasm to add additional tasks. Providers also could face annual licensing agreements, fees on a per-study basis, or royalties associated with particular PROs, and at the individual practice level, there may not be appropriate expertise to select and implement routine PRO monitoring. To address this, efforts are being made to simplify the process of incorporating PROs. For example, given the relatively large number of heterogeneous PROs, the PROMIS project1 endeavors to clarify which PROs constitute the best measure for each construct and condition.⁹ The PROMIS measures also are provided publicly and are available without license or fee.

Areas particularly well situated for growth in the use of PRO measures include comparative effectiveness studies and pragmatic clinical trials. PRO-derived data may promote a shift from explanatory randomized controlled trials to pragmatic randomized controlled trials because these data emphasize patient-centered care and are more broadly generalizable to clinical settings. Furthermore, the derivation of data directly from the health care delivery system through PROs, such as two-way text messages, increases the relevance and cost effectiveness of clinical trials. Given the current medical climate, pressures continue to mount to identify cost-efficient and efficacious medical therapies.¹⁰ In this capacity, PROs facilitate the understanding of changes in HRQL domains subject to treatment choices. PROs further consider the comparative symptom burden and side effects associated with competing treatment strategies.¹¹ Finally, PROs also have enabled the procurement of data from patient-powered research networks. Although this concept has not yet been applied to esophageal diseases, one example of this in the GI field is the Crohn’s and Colitis Foundation of America Partners project, which has built an internet cohort consisting of approximately 14,200 inflammatory bowel disease patients who are monitored with a series of PROs.¹² An endeavor such as this should be a model for esophageal conditions in the future.
 

Conclusions

PROs, as a structured means of directly assessing symptoms, help facilitate a provider’s understanding from a patient’s perspectives. Multiple PROs have been developed to characterize constructs pertinent to esophageal diseases and symptoms. These vary in methodologic rigor, but multiple well-constructed PROs exist for symptom domains such as dysphagia and heartburn, and can be used to monitor symptoms over time and assess treatment efficacy. Implementation of esophageal PROs, both in large health systems and in routine clinical practice, is not yet standard and faces a number of barriers. However, the potential benefits are substantial and include increased patient-centeredness, more accurate and timely disease monitoring, and applicability to comparative effectiveness studies, pragmatic clinical trials, and patient-powered research networks.
 

References

1. Spiegel B., Hays R., Bolus R., et al. Development of the NIH Patient-Reported Outcomes Measurement Information System (PROMIS) gastrointestinal symptom scales. Am J Gastroenterol. 2014;109:1804-14.

2. Chassany O., Shaheen N.J., Karlsson M., et al. Systematic review: symptom assessment using patient-reported outcomes in gastroesophageal reflux disease and dyspepsia. Scand J Gastroenterol. 2012;47:1412-21.

3. U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research, U.S. Department of Health and Human Services FDA Center for Biologics Evaluation and Research, U.S. Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79. Available from:http://www.ncbi.nlm.nih.gov/pubmed/17034633%0Ahttp://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=PMC1629006

Accessed May 23, 2017

4. Lipscomb J. Cancer outcomes research and the arenas of application. J Natl Cancer Inst Monogr. 2004;2004:1-7.

5. Patel D.A., Sharda R., Hovis K.L., et al. Patient-reported outcome measures in dysphagia: a systematic review of instrument development and validation. Dis Esophagus. 2017;30:1-23.

6. Vakil N.B., Halling K., Becher A., et al. Systematic review of patient-reported outcome instruments for gastroesophageal reflux disease symptoms. Eur J Gastroenterol Hepatol. 2013;25:2-14.

7. Bedell A., Taft T.H., Keefer L. Development of the Northwestern Esophageal Quality of Life Scale: a hybrid measure for use across esophageal conditions. Am J Gastroenterol. 2016;111:493-9.

8. Farnik M., Pierzchala W. Instrument development and evaluation for patient-related outcomes assessments. Patient Relat Outcome Meas. 2012;3:1-7.

9. Wagle N.W.. Implementing patient-reported outcome measures (PROMs). N Engl J Med Catal. 2016; :1-2. Available from:

http://catalyst.nejm.org/implementing-proms-patient-reported-outcome-measures/. Accessed July 14, 2017

10. Richesson R.L., Hammond W.E., Nahm M., et al. Electronic health records based phenotyping in next-generation clinical trials: a perspective from the NIH Health Care Systems Collaboratory. J Am Med Informatics Assoc. 2013;20: e226-e231.

11. Coon C.D., McLeod L.D. Patient-reported outcomes: current perspectives and future directions. Clin Ther. 2013;35:399-401.

12. Chung A.E., Sandler R.S., Long M.D., et al. Harnessing person-generated health data to accelerate patient-centered outcomes research: The Crohn’s and Colitis Foundation of America PCORnet Patient Powered Research Network (CCFA Partners)

J Am Med Informatics Assoc. 2016;23:485-90.

13. Darling G., Eton D.T., Sulman J., et al. Validation of the functional assessment of cancer therapy esophageal cancer subscale. Cancer. 2006;107:854-63.

14. Lagergren P., Fayers P., Conroy T., et al. Clinical and psychometric validation of a questionnaire module, the EORTC QLQ-OG25, to assess health-related quality of life in patients with cancer of the oesophagus, the oesophago-gastric junction and the stomach. Eur J Cancer. 2007;43:2066-73.

15. Blazeby J.M., Conroy T., Hammerlid E., et al. Clinical and psychometric validation of an EORTC questionnaire module, the EORTC QLQ-OES18, to assess quality of life in patients with oesophageal cancer. Eur J Cancer. 2003;39:1384-94.

16. Chen A.Y., Frankowski R., Bishop-Leone J., et al. The development and validation of a dysphagia-specific quality-of-life questionnaire for patients with head and neck cancer: the M. D. Anderson dysphagia inventory. Arch Otolaryngol Head Neck Surg. 2001;127:870-6.

17. McHorney C.A., Bricker D.E., Robbins J., et al. The SWAL-QOL outcomes tool for oropharyngeal dysphagia in adults: II. item reduction and preliminary scaling. Dysphagia. 2000;15:122-33.

18. Wallace K.L., Middleton S., Cook I.J. Development and validation of a self-report symptom inventory to assess the severity of oral-pharyngeal dysphagia. Gastroenterology. 2000;118:678-87.

19. McHorney C.A., Robbins J.A., Lomax K., et al. The SWAL-QOL and SWAL-CARE outcomes tool for oropharyngeal dysphagia in adults: III. Documentation of reliability and validity. Dysphagia. 2002;17:97-114.

20. Urbach D.R., Tomlinson G.A., Harnish J.L., et al. A measure of disease-specific health-related quality of life for achalasia. Am J Gastroenterol. 2005;100:1668-76.

21. Eckardt V., Aignherr C., Bernhard G. Predictors of outcome in patients with achalasia treated by pneumatic dilation. Gastroenterology. 1992;103:1732-8.

22. Dellon E.S., Irani A.M., Hill M.R., et al. Development and field testing of a novel patient-reported outcome measure of dysphagia in patients with eosinophilic esophagitis. Aliment Pharmacol Ther. 2013;38:634-42.

23. Franciosi J.P., Hommel K., DeBrosse C.W., et al. Development of a validated patient-reported symptom metric for pediatric eosinophilic esophagitis: qualitative methods. BMC Gastroenterol. 2011;11:126.

24. Schoepfer A.M., Straumann A., Panczak R., et al. Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. Gastroenterology. 2014;147:1-24.

25. Grudell A.B., Alexander J.A., Enders F.B., et al. Validation of the Mayo Dysphagia Questionnaire. Dis Esophagus. 2007;20:202-5.

26. Rothman M., Farup C., Steward W., et al. Symptoms associated with gastroesophageal reflux disease: Development of a questionnaire for use in clinical trials. Dig Dis Sci. 2001;46:1540-9.

27. Spiegel B.M., Roberts L., Mody R., et al. The development and validation of a nocturnal gastro-oesophageal reflux disease symptom severity and impact questionnaire for adults. Aliment Pharmacol Ther. 2010;32:591-602.

28. Bardhan K.D., Stanghellini V., Armstrong D., et al. International validation of ReQuest in patients with endoscopy-negative gastro-oesophageal reflux disease. Aliment Pharmacol Ther. 2004;20:891-8.

29. Van Zanten S.V., Armstrong D., Barkun A., et al. Symptom overlap in patients with upper gastrointestinal complaints in the Canadian confirmatory acid suppression test (CAST) study: Further psychometric validation of the reflux disease questionnaire. Aliment Pharmacol Ther. 2007;25:1087-97.

30. Armstrong D., Moayyedi P., Hunt R., et al. M1870 resolution of persistent GERD symptoms after a change in therapy: EncomPASS - a cluster-randomized study in primary care. Gastroenterology. 2009;136(Suppl 1):A-435.

31. Jones R., Junghard O., Dent J., et al. Developement of the GerdQ, a tool for the diagnosis and management of gastro-oesophageal reflux disease in primary care. Aliment Pharmacol Ther. 2009;30:1030-8.


Dr. Reed is a senior fellow and Dr. Dillon is an associate professor of medicine and epidemiology, Center for Esophageal Diseases and Swallowing, division of gastroenterology and hepatology, University of North Carolina School of Medicine, Chapel Hill. Dr. Dellon has received research funding from Adare, Allakos, GSK, Meritage, Miraca, Nutricia, Celgene/Receptos, Regeneron, and Shire; he has been a consultant for Adare, Alivio, Allakos, AstraZeneca, Banner, Calypso, Enumeral, EsoCap, Celgene/Receptos, GSK, Regeneron, Robarts, and Shire; and has received an educational grant from Banner and Holoclara.

In my introductory comments to the practice management section last year, I wrote about cultivating competencies for value-based care. One of the key competencies was patient centeredness. Patient-reported outcomes (PROs) and patient experience measures specifically were highlighted as examples of meaningful tools for achieving patient centeredness. Starting with this month’s contribution by Drs Reed and Dellon on PROs in esophageal disease, we begin a series of articles focused on this important construct. We will follow this article with reports focused on PRO for patients with irritable bowel syndrome, inflammatory bowel disease, and chronic liver disease. These reports will not only review the importance of PROs, but also highlight the most practical approaches to measuring disease-specific PROs in clinical practice all with the goal of improving the care of our patients.
Ziad Gellad, MD, MPH, AGAF, Special Section Editor
 

Patients seek medical care for symptoms affecting their quality of life,¹ and this is particularly true of digestive diseases, in which many common conditions are symptom predominant. However, clinician and patient perception of symptoms often conflict,² and formalized measurement tools may have a role for optimizing symptom assessment. Patient-reported outcomes (PROs) directly capture patients’ health status from their own perspectives and can bridge the divide between patient and provider interpretation. The US Food and Drug Administration (FDA) defines PROs as “any report of the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else.”³

For the clinical assessment of esophageal diseases, existing physiologic and structural testing modalities cannot ascertain patient disease perception or measure the impact of symptoms on health care–associated quality of life. In contrast, by capturing patient-centric data, PROs can provide insight into the psychosocial aspects of patient disease perceptions; capture health-related quality of life (HRQL); improve provider understanding; highlight discordance between physiologic, symptom, and HRQL measures; and formalize follow-up evaluation of treatment response.^1,4 Following up symptoms such as dysphagia or heartburn over time in a structured way allows clinically obtained data to be used in pragmatic or comparative effectiveness studies. PROs are now an integral part of the FDA’s drug approval process.

In this article, we review the available PROs capturing esophageal symptoms with a focus on dysphagia and heartburn measures that were developed with rigorous methodology; it is beyond the scope of this article to perform a thorough review of all upper gastrointestinal (GI) PROs or quality-of-life PROs. We then discuss how esophageal PROs may be incorporated into clinical practice now, as well as opportunities for PRO use in the future.
 

Esophageal symptom-specific patient-reported outcomes

The literature pertinent to upper GI and esophageal-specific PROs is heterogeneous, and the development of PROs has been variable in rigor. Two recent systematic reviews identified PROs pertinent to dysphagia and heartburn (Table 1) and both emphasized rigorous measures developed in accordance with FDA guidance.³

Patel et al⁵ identified 34 dysphagia-specific PRO measures, of which 10 were rigorously developed (Table 1). These measures encompassed multiple conditions including esophageal cancer (Functional Assessment of Cancer Therapy Esophageal Cancer Subscale, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal Cancer 25 items, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal cancer 18 items, upper aerodigestive neoplasm-attributable oropharyngeal dysphagia [M.D. Anderson dysphagia inventory], mechanical and neuromyogenic oropharyngeal dysphagia [swallow quality-of-life questionnaire], Sydney Swallow Questionnaire, [swallowing quality of care], achalasia [Measure of Achalasia Disease Severity], eosinophilic esophagitis [Dysphagia Symptom Questionnaire], and general dysphagia symptoms and gastroesophageal reflux [Patient-Reported Outcomes Measurement Information System Gastrointestinal Symptom Scales (PROMIS-GI)]. PROMIS-GI, produced as part of the National Institutes of Health PROMIS program, includes rigorous measures for general dysphagia symptoms and gastroesophageal reflux in addition to lower gastrointestinal symptom measures.

The systematic review by Vakil et al⁶ found 15 PRO measures for gastroesophageal reflux disease (GERD) symptoms that underwent psychometric evaluation (Table 1). Of these, 5 measures were devised according to the developmental steps stipulated by the US FDA and the European Medicines Agency, and each measure has been used as an end point for a clinical trial. The 5 measures include the GERD Symptom Assessment Scale, the Nocturnal Gastro-oesophageal Reflux Disease Symptom Severity and Impact Questionnaire, the Reflux Questionnaire, the Reflux Disease Questionnaire, and the Proton Pump Inhibitor Acid Suppression Symptom Test (Table 1). Additional PROs capturing esophageal symptoms include the eosinophilic esophagitis symptom activity index, Eckardt score (used for achalasia), Mayo dysphagia questionnaire, and GERD-Q (Table 1).

Utilization of esophageal patient-reported outcomes in practice

Before incorporating a PRO into clinical practice, providers must appreciate the construct(s), intent, developmental measurement properties, validation strategies, and responsiveness characteristics associated with the measure.⁴ PROs can be symptom- and/or condition-specific. For example, this could include dysphagia associated with achalasia or eosinophilic esophagitis, postoperative dysphagia from spine surgery, or general dysphagia symptoms regardless of the etiology (Table 1). Intent refers to the context in which a PRO should be used and generally is stratified into 3 areas: population surveillance, individual patient-clinician interactions, and research studies.⁴ A thorough analysis of PRO developmental properties exceeds the scope of this article. However, several key considerations are worth discussing. Each measure should clearly delineate the construct, or outcome, in addition to the population used to create the measure (eg, patients with achalasia). PROs should be assessed for reliability, construct validity, and content validity. Reliability pertains to the degree in which scores are free from measurement error, the extent to which items (ie, questions) correlate, and test–retest reliability. Construct validity includes dimensionality (evidence of whether a single or multiple subscales exist in the measure), responsiveness to change (longitudinal validity), and convergent validity (correlation with additional construct-specific measures). Central to the PRO development process is the involvement of patients and content experts (content validity). PRO measures should be readily interpretable, and the handling of missing items should be stipulated. The burden, or time required for administering and scoring the instrument, and the reading level of the PRO need to be considered.⁸ In short, a PRO should measure something important to patients, in a way that patients can understand, and in a way that accurately reflects the underlying symptom and disease.

Although PROs traditionally represent a method for gathering data for research, they also should be viewed as a means of improving clinical care. The monitoring of change in a particular construct represents a common application of PROs in clinical practice. This helps quantify the efficacy of an intervention and can provide insight into the comparative effectiveness of alternative therapies. For example, in a patient with an esophageal stricture, a dysphagia-specific measure could be used at baseline before an endoscopy and dilation, in follow-up evaluation after dilation, and then as a monitoring tool to determine when repeat dilation may be needed. Similarly, the Eckardt score has been used commonly to monitor response to achalasia treatments. Clinicians also may use PROs in real time to optimize patient management. The data gathered from PROs may help triage patients into treatment pathways, trigger follow-up appointments, supply patient education prompts, and produce patient and provider alerts.⁸ For providers engaging in clinical research, PROs administered at the point of patient intake, whether electronically through a patient portal or in the clinic, provide a means of gathering baseline data.⁹ A key question, however, is whether it is practical to use a PRO routinely in the clinic, esophageal function laboratory, or endoscopy suite.

These practical issues include cultivating a conducive environment for PRO utilization, considering the burden of the measure on the patient, and utilization of the results in an expedient manner.⁹ To promote seamless use of a PRO in clinical work-flows, a multimodal means of collecting PRO data should be arranged. Electronic PROs available through a patient portal, designed with a user-friendly and intuitive interface, facilitate patient completion of PROs at their convenience, and ideally before a clinical or procedure visit. For patients without access to the internet, tablets and/or computer terminals within the office are convenient options. Nurses or clinic staff also could help patients complete a PRO during check-in for clinic, esophageal testing, or endoscopy. The burden a PRO imposes on patients also limits the utility of a measure. For instance, PROs with a small number of questions are more likely to be completed, while scales consisting of 30 of more items are infrequently finished. Clinicians also should consider how they plan to use the results of a PRO before implementing one; if the data will not be used, then the effort to implement and collect it will be wasted. Moreover, patients will anticipate that the time required to complete a PRO will translate to an impact on their management plan and will more readily complete additional PROs if previous measures expediently affected their care.⁹

Barriers to patient-reported outcome implementation and future directions

Given the potential benefits to PRO use, why are they not implemented routinely? In practice, there are multiple barriers that thwart the adoption of PROs into both health care systems and individual practices. The integration of PROs into large health care systems languishes partly because of technological and operational barriers.⁹ For instance, the manual distribution, collection, and transcription of handwritten information requires substantial investitures of time, which is magnified by the number of patients whose care is provided within a large health system. One approach to the technological barrier includes the creation of an electronic platform integrating with patient portals. Such a platform would obviate the need to manually collect and transcribe documents, and could import data directly into provider documentation and flowsheets. However, the programming time and costs are substantial upfront, and without clear data that this could lead to improved outcomes or decreased costs downstream there may be reluctance to devote resources to this. In clinical practice, the already significant demands on providers’ time mitigates enthusiasm to add additional tasks. Providers also could face annual licensing agreements, fees on a per-study basis, or royalties associated with particular PROs, and at the individual practice level, there may not be appropriate expertise to select and implement routine PRO monitoring. To address this, efforts are being made to simplify the process of incorporating PROs. For example, given the relatively large number of heterogeneous PROs, the PROMIS project1 endeavors to clarify which PROs constitute the best measure for each construct and condition.⁹ The PROMIS measures also are provided publicly and are available without license or fee.

Areas particularly well situated for growth in the use of PRO measures include comparative effectiveness studies and pragmatic clinical trials. PRO-derived data may promote a shift from explanatory randomized controlled trials to pragmatic randomized controlled trials because these data emphasize patient-centered care and are more broadly generalizable to clinical settings. Furthermore, the derivation of data directly from the health care delivery system through PROs, such as two-way text messages, increases the relevance and cost effectiveness of clinical trials. Given the current medical climate, pressures continue to mount to identify cost-efficient and efficacious medical therapies.¹⁰ In this capacity, PROs facilitate the understanding of changes in HRQL domains subject to treatment choices. PROs further consider the comparative symptom burden and side effects associated with competing treatment strategies.¹¹ Finally, PROs also have enabled the procurement of data from patient-powered research networks. Although this concept has not yet been applied to esophageal diseases, one example of this in the GI field is the Crohn’s and Colitis Foundation of America Partners project, which has built an internet cohort consisting of approximately 14,200 inflammatory bowel disease patients who are monitored with a series of PROs.¹² An endeavor such as this should be a model for esophageal conditions in the future.
 

Conclusions

PROs, as a structured means of directly assessing symptoms, help facilitate a provider’s understanding from a patient’s perspectives. Multiple PROs have been developed to characterize constructs pertinent to esophageal diseases and symptoms. These vary in methodologic rigor, but multiple well-constructed PROs exist for symptom domains such as dysphagia and heartburn, and can be used to monitor symptoms over time and assess treatment efficacy. Implementation of esophageal PROs, both in large health systems and in routine clinical practice, is not yet standard and faces a number of barriers. However, the potential benefits are substantial and include increased patient-centeredness, more accurate and timely disease monitoring, and applicability to comparative effectiveness studies, pragmatic clinical trials, and patient-powered research networks.
 

References

1. Spiegel B., Hays R., Bolus R., et al. Development of the NIH Patient-Reported Outcomes Measurement Information System (PROMIS) gastrointestinal symptom scales. Am J Gastroenterol. 2014;109:1804-14.

2. Chassany O., Shaheen N.J., Karlsson M., et al. Systematic review: symptom assessment using patient-reported outcomes in gastroesophageal reflux disease and dyspepsia. Scand J Gastroenterol. 2012;47:1412-21.

3. U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research, U.S. Department of Health and Human Services FDA Center for Biologics Evaluation and Research, U.S. Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79. Available from:http://www.ncbi.nlm.nih.gov/pubmed/17034633%0Ahttp://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=PMC1629006

Accessed May 23, 2017

4. Lipscomb J. Cancer outcomes research and the arenas of application. J Natl Cancer Inst Monogr. 2004;2004:1-7.

5. Patel D.A., Sharda R., Hovis K.L., et al. Patient-reported outcome measures in dysphagia: a systematic review of instrument development and validation. Dis Esophagus. 2017;30:1-23.

6. Vakil N.B., Halling K., Becher A., et al. Systematic review of patient-reported outcome instruments for gastroesophageal reflux disease symptoms. Eur J Gastroenterol Hepatol. 2013;25:2-14.

7. Bedell A., Taft T.H., Keefer L. Development of the Northwestern Esophageal Quality of Life Scale: a hybrid measure for use across esophageal conditions. Am J Gastroenterol. 2016;111:493-9.

8. Farnik M., Pierzchala W. Instrument development and evaluation for patient-related outcomes assessments. Patient Relat Outcome Meas. 2012;3:1-7.

9. Wagle N.W.. Implementing patient-reported outcome measures (PROMs). N Engl J Med Catal. 2016; :1-2. Available from:

http://catalyst.nejm.org/implementing-proms-patient-reported-outcome-measures/. Accessed July 14, 2017

10. Richesson R.L., Hammond W.E., Nahm M., et al. Electronic health records based phenotyping in next-generation clinical trials: a perspective from the NIH Health Care Systems Collaboratory. J Am Med Informatics Assoc. 2013;20: e226-e231.

11. Coon C.D., McLeod L.D. Patient-reported outcomes: current perspectives and future directions. Clin Ther. 2013;35:399-401.

12. Chung A.E., Sandler R.S., Long M.D., et al. Harnessing person-generated health data to accelerate patient-centered outcomes research: The Crohn’s and Colitis Foundation of America PCORnet Patient Powered Research Network (CCFA Partners)

J Am Med Informatics Assoc. 2016;23:485-90.

13. Darling G., Eton D.T., Sulman J., et al. Validation of the functional assessment of cancer therapy esophageal cancer subscale. Cancer. 2006;107:854-63.

14. Lagergren P., Fayers P., Conroy T., et al. Clinical and psychometric validation of a questionnaire module, the EORTC QLQ-OG25, to assess health-related quality of life in patients with cancer of the oesophagus, the oesophago-gastric junction and the stomach. Eur J Cancer. 2007;43:2066-73.

15. Blazeby J.M., Conroy T., Hammerlid E., et al. Clinical and psychometric validation of an EORTC questionnaire module, the EORTC QLQ-OES18, to assess quality of life in patients with oesophageal cancer. Eur J Cancer. 2003;39:1384-94.

16. Chen A.Y., Frankowski R., Bishop-Leone J., et al. The development and validation of a dysphagia-specific quality-of-life questionnaire for patients with head and neck cancer: the M. D. Anderson dysphagia inventory. Arch Otolaryngol Head Neck Surg. 2001;127:870-6.

17. McHorney C.A., Bricker D.E., Robbins J., et al. The SWAL-QOL outcomes tool for oropharyngeal dysphagia in adults: II. item reduction and preliminary scaling. Dysphagia. 2000;15:122-33.

18. Wallace K.L., Middleton S., Cook I.J. Development and validation of a self-report symptom inventory to assess the severity of oral-pharyngeal dysphagia. Gastroenterology. 2000;118:678-87.

19. McHorney C.A., Robbins J.A., Lomax K., et al. The SWAL-QOL and SWAL-CARE outcomes tool for oropharyngeal dysphagia in adults: III. Documentation of reliability and validity. Dysphagia. 2002;17:97-114.

20. Urbach D.R., Tomlinson G.A., Harnish J.L., et al. A measure of disease-specific health-related quality of life for achalasia. Am J Gastroenterol. 2005;100:1668-76.

21. Eckardt V., Aignherr C., Bernhard G. Predictors of outcome in patients with achalasia treated by pneumatic dilation. Gastroenterology. 1992;103:1732-8.

22. Dellon E.S., Irani A.M., Hill M.R., et al. Development and field testing of a novel patient-reported outcome measure of dysphagia in patients with eosinophilic esophagitis. Aliment Pharmacol Ther. 2013;38:634-42.

23. Franciosi J.P., Hommel K., DeBrosse C.W., et al. Development of a validated patient-reported symptom metric for pediatric eosinophilic esophagitis: qualitative methods. BMC Gastroenterol. 2011;11:126.

24. Schoepfer A.M., Straumann A., Panczak R., et al. Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. Gastroenterology. 2014;147:1-24.

25. Grudell A.B., Alexander J.A., Enders F.B., et al. Validation of the Mayo Dysphagia Questionnaire. Dis Esophagus. 2007;20:202-5.

26. Rothman M., Farup C., Steward W., et al. Symptoms associated with gastroesophageal reflux disease: Development of a questionnaire for use in clinical trials. Dig Dis Sci. 2001;46:1540-9.

27. Spiegel B.M., Roberts L., Mody R., et al. The development and validation of a nocturnal gastro-oesophageal reflux disease symptom severity and impact questionnaire for adults. Aliment Pharmacol Ther. 2010;32:591-602.

28. Bardhan K.D., Stanghellini V., Armstrong D., et al. International validation of ReQuest in patients with endoscopy-negative gastro-oesophageal reflux disease. Aliment Pharmacol Ther. 2004;20:891-8.

29. Van Zanten S.V., Armstrong D., Barkun A., et al. Symptom overlap in patients with upper gastrointestinal complaints in the Canadian confirmatory acid suppression test (CAST) study: Further psychometric validation of the reflux disease questionnaire. Aliment Pharmacol Ther. 2007;25:1087-97.

30. Armstrong D., Moayyedi P., Hunt R., et al. M1870 resolution of persistent GERD symptoms after a change in therapy: EncomPASS - a cluster-randomized study in primary care. Gastroenterology. 2009;136(Suppl 1):A-435.

31. Jones R., Junghard O., Dent J., et al. Developement of the GerdQ, a tool for the diagnosis and management of gastro-oesophageal reflux disease in primary care. Aliment Pharmacol Ther. 2009;30:1030-8.


Dr. Reed is a senior fellow and Dr. Dillon is an associate professor of medicine and epidemiology, Center for Esophageal Diseases and Swallowing, division of gastroenterology and hepatology, University of North Carolina School of Medicine, Chapel Hill. Dr. Dellon has received research funding from Adare, Allakos, GSK, Meritage, Miraca, Nutricia, Celgene/Receptos, Regeneron, and Shire; he has been a consultant for Adare, Alivio, Allakos, AstraZeneca, Banner, Calypso, Enumeral, EsoCap, Celgene/Receptos, GSK, Regeneron, Robarts, and Shire; and has received an educational grant from Banner and Holoclara.

CMS has released its proposed rule (see related articles and a commentary) and included changes as substantial as I have seen in the last two decades. Additionally, the Affordable Care Act has been under continued attack despite its majority support from our citizenry. Loss of the individual mandate, allowance of “skinny” health plans, a rewrite of association plan rules, elimination of cost-sharing reductions and premium support – all have contributed to a shifting away from socialized medical costs and toward a system of individual responsibility for health. Depending on one’s political philosophy (and income), that may be bad or good.

Our article list this month will be interesting to many. The AGA produced a Clinical Practice Update about tumor seeding with endoscopic procedures. This should give us pause and make us reconsider our endoscopic practices. My wife (an endoscopy nurse in Minneapolis) has been asking for years whether pulling a PEG tube past an esophageal cancer might cause tumor seeding, and physicians have reassured her that there is no cause for worry. Turns out she was right (as usual). Deaths from liver disease in the U.S. have seen a dramatic increase since 1999, driven substantially by increasing alcohol use. Fecal transplants in irritable bowel syndrome? Possibly helpful, as reported in an article from Digestive Disease Week.^®

As summer comes to an end, we head into a tumultuous fall that will be dominated by November elections.

John I. Allen, MD, MBA, AGAF
Editor in Chief

CMS has released its proposed rule (see related articles and a commentary) and included changes as substantial as I have seen in the last two decades. Additionally, the Affordable Care Act has been under continued attack despite its majority support from our citizenry. Loss of the individual mandate, allowance of “skinny” health plans, a rewrite of association plan rules, elimination of cost-sharing reductions and premium support – all have contributed to a shifting away from socialized medical costs and toward a system of individual responsibility for health. Depending on one’s political philosophy (and income), that may be bad or good.

Our article list this month will be interesting to many. The AGA produced a Clinical Practice Update about tumor seeding with endoscopic procedures. This should give us pause and make us reconsider our endoscopic practices. My wife (an endoscopy nurse in Minneapolis) has been asking for years whether pulling a PEG tube past an esophageal cancer might cause tumor seeding, and physicians have reassured her that there is no cause for worry. Turns out she was right (as usual). Deaths from liver disease in the U.S. have seen a dramatic increase since 1999, driven substantially by increasing alcohol use. Fecal transplants in irritable bowel syndrome? Possibly helpful, as reported in an article from Digestive Disease Week.^®

As summer comes to an end, we head into a tumultuous fall that will be dominated by November elections.

John I. Allen, MD, MBA, AGAF
Editor in Chief

CMS has released its proposed rule (see related articles and a commentary) and included changes as substantial as I have seen in the last two decades. Additionally, the Affordable Care Act has been under continued attack despite its majority support from our citizenry. Loss of the individual mandate, allowance of “skinny” health plans, a rewrite of association plan rules, elimination of cost-sharing reductions and premium support – all have contributed to a shifting away from socialized medical costs and toward a system of individual responsibility for health. Depending on one’s political philosophy (and income), that may be bad or good.

Our article list this month will be interesting to many. The AGA produced a Clinical Practice Update about tumor seeding with endoscopic procedures. This should give us pause and make us reconsider our endoscopic practices. My wife (an endoscopy nurse in Minneapolis) has been asking for years whether pulling a PEG tube past an esophageal cancer might cause tumor seeding, and physicians have reassured her that there is no cause for worry. Turns out she was right (as usual). Deaths from liver disease in the U.S. have seen a dramatic increase since 1999, driven substantially by increasing alcohol use. Fecal transplants in irritable bowel syndrome? Possibly helpful, as reported in an article from Digestive Disease Week.^®

As summer comes to an end, we head into a tumultuous fall that will be dominated by November elections.

John I. Allen, MD, MBA, AGAF
Editor in Chief

“Alex, I understand that you are upset that you left your little bulldozer at home. Let’s try to think of something else you can play with here at the restaurant that is kind of like a bulldozer.”

Ambernectar 13/CC BY-ND 2.0

Sounds like a reasonable strategy to calm an unruly preschooler, and it might have been had it not been the fifth attempt in a 45-minute dialogue between a mother and her overtired, misbehaving 3-year-old. There had been a lot of “I understand how you feel” and “use your words” woven into a gag-worthy and futile effort to forge a collaborative parent-child solution to the problem of an exhausted preschooler who is up past his bedtime in a public place.

My wife and I enjoy a night out with friends and prefer a quiet dining atmosphere. However, some evenings we eat earlier and choose a restaurant we know appeals to families with young children. At those meals, we anticipate being serenaded by a loud background buzz punctuated by the occasional shriek or short bout of crying. We expect a degree of childish behavior to come with the territory, and watching the dramas unfold brings back fond “been there, done that” memories. But, listening to those behaviors being horribly mismanaged can ruin even the most tolerant adult’s appetite in less time than it takes a parent to say, “I can see you’re unhappy, and we need to talk about why.”

In an op-ed piece, a psychotherapist asks the legitimate question, and provides the correct quick answer (“Which Is Better, Rewards or Punishments? Neither,” New York Times, Aug. 21, 2018). I couldn’t agree more. In my experience, rewards have a very short half-life and can become disastrously inflationary in the blink of an eye. On the other hand, punishments can be either too heavy-handed or so irrational that the child fails to make a logical connection between his misbehavior and his sentence.

Unfortunately, many child behavior advisers, including the op-ed author, offer alternatives to rewards and punishment that are unworkable in real-world circumstances, such as the restaurant scenario my wife and I endured.

While it sounds very democratic to ask a 3-year-old why he is misbehaving, more often than not it should be the parent who is asking what he or she could have done differently to avoid the situation. It is likely the child has been allowed to become overtired and/or the parent may be in denial about his/her child’s intolerance for stimulating environments.

Too often the parent takes too long to realize that the water is spilling over the dam and it is time to head for shore. Children who are overtired and having a tantrum can’t participate in a rational discussion about their feelings. If that dialogue needs to happen, and that is seldom, it should be the next day after the parent has time to consider his or her own mistakes.

When it comes to managing misbehaving children, I prefer well-tailored consequences and my favorite is a humanely crafted time-out. When presented and executed properly, a time-out can break the cycle of misbehavior and give both parent and child a chance to reconsider their positions.

But at 7 p.m. on a Friday evening in a busy restaurant, neither a time-out nor philosophizing with a 3-year-old is going to work. It’s time to ask for the check and head home to bed.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

“Alex, I understand that you are upset that you left your little bulldozer at home. Let’s try to think of something else you can play with here at the restaurant that is kind of like a bulldozer.”

Ambernectar 13/CC BY-ND 2.0

Sounds like a reasonable strategy to calm an unruly preschooler, and it might have been had it not been the fifth attempt in a 45-minute dialogue between a mother and her overtired, misbehaving 3-year-old. There had been a lot of “I understand how you feel” and “use your words” woven into a gag-worthy and futile effort to forge a collaborative parent-child solution to the problem of an exhausted preschooler who is up past his bedtime in a public place.

My wife and I enjoy a night out with friends and prefer a quiet dining atmosphere. However, some evenings we eat earlier and choose a restaurant we know appeals to families with young children. At those meals, we anticipate being serenaded by a loud background buzz punctuated by the occasional shriek or short bout of crying. We expect a degree of childish behavior to come with the territory, and watching the dramas unfold brings back fond “been there, done that” memories. But, listening to those behaviors being horribly mismanaged can ruin even the most tolerant adult’s appetite in less time than it takes a parent to say, “I can see you’re unhappy, and we need to talk about why.”

In an op-ed piece, a psychotherapist asks the legitimate question, and provides the correct quick answer (“Which Is Better, Rewards or Punishments? Neither,” New York Times, Aug. 21, 2018). I couldn’t agree more. In my experience, rewards have a very short half-life and can become disastrously inflationary in the blink of an eye. On the other hand, punishments can be either too heavy-handed or so irrational that the child fails to make a logical connection between his misbehavior and his sentence.

Unfortunately, many child behavior advisers, including the op-ed author, offer alternatives to rewards and punishment that are unworkable in real-world circumstances, such as the restaurant scenario my wife and I endured.

While it sounds very democratic to ask a 3-year-old why he is misbehaving, more often than not it should be the parent who is asking what he or she could have done differently to avoid the situation. It is likely the child has been allowed to become overtired and/or the parent may be in denial about his/her child’s intolerance for stimulating environments.

Too often the parent takes too long to realize that the water is spilling over the dam and it is time to head for shore. Children who are overtired and having a tantrum can’t participate in a rational discussion about their feelings. If that dialogue needs to happen, and that is seldom, it should be the next day after the parent has time to consider his or her own mistakes.

When it comes to managing misbehaving children, I prefer well-tailored consequences and my favorite is a humanely crafted time-out. When presented and executed properly, a time-out can break the cycle of misbehavior and give both parent and child a chance to reconsider their positions.

But at 7 p.m. on a Friday evening in a busy restaurant, neither a time-out nor philosophizing with a 3-year-old is going to work. It’s time to ask for the check and head home to bed.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

“Alex, I understand that you are upset that you left your little bulldozer at home. Let’s try to think of something else you can play with here at the restaurant that is kind of like a bulldozer.”

Ambernectar 13/CC BY-ND 2.0

Sounds like a reasonable strategy to calm an unruly preschooler, and it might have been had it not been the fifth attempt in a 45-minute dialogue between a mother and her overtired, misbehaving 3-year-old. There had been a lot of “I understand how you feel” and “use your words” woven into a gag-worthy and futile effort to forge a collaborative parent-child solution to the problem of an exhausted preschooler who is up past his bedtime in a public place.

My wife and I enjoy a night out with friends and prefer a quiet dining atmosphere. However, some evenings we eat earlier and choose a restaurant we know appeals to families with young children. At those meals, we anticipate being serenaded by a loud background buzz punctuated by the occasional shriek or short bout of crying. We expect a degree of childish behavior to come with the territory, and watching the dramas unfold brings back fond “been there, done that” memories. But, listening to those behaviors being horribly mismanaged can ruin even the most tolerant adult’s appetite in less time than it takes a parent to say, “I can see you’re unhappy, and we need to talk about why.”

In an op-ed piece, a psychotherapist asks the legitimate question, and provides the correct quick answer (“Which Is Better, Rewards or Punishments? Neither,” New York Times, Aug. 21, 2018). I couldn’t agree more. In my experience, rewards have a very short half-life and can become disastrously inflationary in the blink of an eye. On the other hand, punishments can be either too heavy-handed or so irrational that the child fails to make a logical connection between his misbehavior and his sentence.

Unfortunately, many child behavior advisers, including the op-ed author, offer alternatives to rewards and punishment that are unworkable in real-world circumstances, such as the restaurant scenario my wife and I endured.

While it sounds very democratic to ask a 3-year-old why he is misbehaving, more often than not it should be the parent who is asking what he or she could have done differently to avoid the situation. It is likely the child has been allowed to become overtired and/or the parent may be in denial about his/her child’s intolerance for stimulating environments.

Too often the parent takes too long to realize that the water is spilling over the dam and it is time to head for shore. Children who are overtired and having a tantrum can’t participate in a rational discussion about their feelings. If that dialogue needs to happen, and that is seldom, it should be the next day after the parent has time to consider his or her own mistakes.

When it comes to managing misbehaving children, I prefer well-tailored consequences and my favorite is a humanely crafted time-out. When presented and executed properly, a time-out can break the cycle of misbehavior and give both parent and child a chance to reconsider their positions.

But at 7 p.m. on a Friday evening in a busy restaurant, neither a time-out nor philosophizing with a 3-year-old is going to work. It’s time to ask for the check and head home to bed.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

You probably think you know how to talk with a child about death. But somehow talking about a parent’s mental illness may seem more difficult. Even medical professionals, as most people, can find themselves feeling more judgmental or uneasy talking about mental illness than about physical problems. But with a prevalence of about one in four people having mental disorders, we need to be prepared for this discussion.

Steve Debenport/Getty Images

Sometimes family members, or even parents themselves, have asked me to tell a child about a parent’s mental illness or substance use. They know the child is confused and scared but don’t know what to say about this still-hushed issue. Other times, children’s behaviors show that they are struggling – by their aggression, depression, decline in school performance, anger, anxiety, or running away – and I find out only by asking that they are experiencing life with a mentally ill parent.

Both are times to get more information about the nature of the parent’s symptoms, what is being done about it; what the child has seen, heard, or experienced; the child’s safety; and what intrinsic or cultural attitudes the family has about the problem. It is best if the affected parents themselves are able to talk realistically about the illness with the child. Sometimes we, as pediatric professionals, can help get the conversation started or may need to step in.

What needs to be conveyed about parental mental illness depends on the developmental age and maturity of the child. Teens can understand the nature of mental illness as a brain disorder, but often have acquired misinformation from peers, the other parent who is hurt and angry, or the Internet that needs to be respectfully corrected. They may be less willing to have such a discussion than would younger children, as they may have experienced verbal or physical aggression from the parent, embarrassment in front of peers, or teasing by classmates. They may have developed coping strategies of writing off the parent, defiance, aggressive responses, or denial.

It is important to find a relaxed moment and begin by saying, “May I ask what you have noticed about your parent’s behavior?” and “What did you make of it?” If they do not answer, you could add, “You know, like acting different or strange.” It is very valuable to be able to name it, if you know the diagnosis, to make the illness more objective. Teens are typically egocentric and wonder if they are prone to develop a similar condition, as well as anxious about who will be there for them if the parent does not get better; both are good questions to address.

Both adolescents and school-aged children are very attuned to when things in life are “unfair,” and having an ill parent certainly qualifies. It is important for whoever has this discussion to empathize, and to acknowledge that it is unfair and that feeling angry, sad, or confused is natural – without giving them permission for misbehavior. It may be easier for some children to have a journal in which to write questions to have an adult answer later. Any child can be given hope by knowing that the parent is getting help (or that you will work on this), especially if the parents themselves say they are trying to get better. The children need to know that although mental illness tends to get better and worse at unexpected times, mental health can be improved. It is essential that children of school age and older have a clear plan for what to do if the parent loses control or is in danger. This might include getting out of the house and calling the parent’s partner, a trusted neighbor, or 911.

While teens may feel guilty about their anger or things they might have said or done in reaction to the ill parent, school-aged and preschool children are more likely to feel guilty that they somehow contributed to the parent’s condition through misbehavior or some imagined influence. Eliciting these thoughts may simply require asking, “What thoughts have you had about why this happened?” or having another family member prompting them by saying, “I have been wondering if there is something I should have done. Have you wondered about that, too?”

What is harder to explain is the impression children may get that their depressed or psychotic or drug-abusing parents no longer love them; and the parents may have said hurtful things to them. School-aged children can be told and can understand an illness of “the feelings and thinking parts of the brain” as being similar to other physical illnesses, such as the flu, that “make people act tired or grouchy or say things they do not mean.” Children of all ages need to be reassured that, inside, their parents still love them, and “it is the illness talking” if they act or speak otherwise. In the case of substance abuse disorders, which might seem more of a choice by the parent than would other mental illnesses, it can be helpful to point out that “drugs and alcohol can be stronger than people and can take over their brains.” The National Alliance on Mental Illness (www.nami.org) offers support programs that may be helpful for older children.

Sometimes families may resist telling a child about parental mental illness because of personal beliefs, cultural stigma, or privacy concerns. I try to emphasize that by being honest in giving children an understanding of their parents’ mental illness, one is helping them trust the adults in their lives. They need the truth and also to practice formulating things to say to counter those who have incorrect ideas, or who insult or make fun of them. They need the truth about mental illness to make sense of their experiences so they can feel lovable and hopeful about their own futures and can recognize illness symptoms in themselves. Giving them words, ensuring their safety and support, and strengthening their coping can help buffer the impact of this common adverse childhood experience to help prevent long-term negative effects.
 

Dr. Howard is assistant professor of pediatrics at Johns Hopkins University, Baltimore, and creator of CHADIS (www.CHADIS.com). She had no other relevant disclosures. Dr. Howard’s contribution to this publication was as a paid expert to MDedge News. E-mail her at [email protected].

You probably think you know how to talk with a child about death. But somehow talking about a parent’s mental illness may seem more difficult. Even medical professionals, as most people, can find themselves feeling more judgmental or uneasy talking about mental illness than about physical problems. But with a prevalence of about one in four people having mental disorders, we need to be prepared for this discussion.

Steve Debenport/Getty Images

Sometimes family members, or even parents themselves, have asked me to tell a child about a parent’s mental illness or substance use. They know the child is confused and scared but don’t know what to say about this still-hushed issue. Other times, children’s behaviors show that they are struggling – by their aggression, depression, decline in school performance, anger, anxiety, or running away – and I find out only by asking that they are experiencing life with a mentally ill parent.

Both are times to get more information about the nature of the parent’s symptoms, what is being done about it; what the child has seen, heard, or experienced; the child’s safety; and what intrinsic or cultural attitudes the family has about the problem. It is best if the affected parents themselves are able to talk realistically about the illness with the child. Sometimes we, as pediatric professionals, can help get the conversation started or may need to step in.

What needs to be conveyed about parental mental illness depends on the developmental age and maturity of the child. Teens can understand the nature of mental illness as a brain disorder, but often have acquired misinformation from peers, the other parent who is hurt and angry, or the Internet that needs to be respectfully corrected. They may be less willing to have such a discussion than would younger children, as they may have experienced verbal or physical aggression from the parent, embarrassment in front of peers, or teasing by classmates. They may have developed coping strategies of writing off the parent, defiance, aggressive responses, or denial.

It is important to find a relaxed moment and begin by saying, “May I ask what you have noticed about your parent’s behavior?” and “What did you make of it?” If they do not answer, you could add, “You know, like acting different or strange.” It is very valuable to be able to name it, if you know the diagnosis, to make the illness more objective. Teens are typically egocentric and wonder if they are prone to develop a similar condition, as well as anxious about who will be there for them if the parent does not get better; both are good questions to address.

Both adolescents and school-aged children are very attuned to when things in life are “unfair,” and having an ill parent certainly qualifies. It is important for whoever has this discussion to empathize, and to acknowledge that it is unfair and that feeling angry, sad, or confused is natural – without giving them permission for misbehavior. It may be easier for some children to have a journal in which to write questions to have an adult answer later. Any child can be given hope by knowing that the parent is getting help (or that you will work on this), especially if the parents themselves say they are trying to get better. The children need to know that although mental illness tends to get better and worse at unexpected times, mental health can be improved. It is essential that children of school age and older have a clear plan for what to do if the parent loses control or is in danger. This might include getting out of the house and calling the parent’s partner, a trusted neighbor, or 911.

While teens may feel guilty about their anger or things they might have said or done in reaction to the ill parent, school-aged and preschool children are more likely to feel guilty that they somehow contributed to the parent’s condition through misbehavior or some imagined influence. Eliciting these thoughts may simply require asking, “What thoughts have you had about why this happened?” or having another family member prompting them by saying, “I have been wondering if there is something I should have done. Have you wondered about that, too?”

What is harder to explain is the impression children may get that their depressed or psychotic or drug-abusing parents no longer love them; and the parents may have said hurtful things to them. School-aged children can be told and can understand an illness of “the feelings and thinking parts of the brain” as being similar to other physical illnesses, such as the flu, that “make people act tired or grouchy or say things they do not mean.” Children of all ages need to be reassured that, inside, their parents still love them, and “it is the illness talking” if they act or speak otherwise. In the case of substance abuse disorders, which might seem more of a choice by the parent than would other mental illnesses, it can be helpful to point out that “drugs and alcohol can be stronger than people and can take over their brains.” The National Alliance on Mental Illness (www.nami.org) offers support programs that may be helpful for older children.

Sometimes families may resist telling a child about parental mental illness because of personal beliefs, cultural stigma, or privacy concerns. I try to emphasize that by being honest in giving children an understanding of their parents’ mental illness, one is helping them trust the adults in their lives. They need the truth and also to practice formulating things to say to counter those who have incorrect ideas, or who insult or make fun of them. They need the truth about mental illness to make sense of their experiences so they can feel lovable and hopeful about their own futures and can recognize illness symptoms in themselves. Giving them words, ensuring their safety and support, and strengthening their coping can help buffer the impact of this common adverse childhood experience to help prevent long-term negative effects.
 

Dr. Howard is assistant professor of pediatrics at Johns Hopkins University, Baltimore, and creator of CHADIS (www.CHADIS.com). She had no other relevant disclosures. Dr. Howard’s contribution to this publication was as a paid expert to MDedge News. E-mail her at [email protected].

You probably think you know how to talk with a child about death. But somehow talking about a parent’s mental illness may seem more difficult. Even medical professionals, as most people, can find themselves feeling more judgmental or uneasy talking about mental illness than about physical problems. But with a prevalence of about one in four people having mental disorders, we need to be prepared for this discussion.

Steve Debenport/Getty Images

Sometimes family members, or even parents themselves, have asked me to tell a child about a parent’s mental illness or substance use. They know the child is confused and scared but don’t know what to say about this still-hushed issue. Other times, children’s behaviors show that they are struggling – by their aggression, depression, decline in school performance, anger, anxiety, or running away – and I find out only by asking that they are experiencing life with a mentally ill parent.

Both are times to get more information about the nature of the parent’s symptoms, what is being done about it; what the child has seen, heard, or experienced; the child’s safety; and what intrinsic or cultural attitudes the family has about the problem. It is best if the affected parents themselves are able to talk realistically about the illness with the child. Sometimes we, as pediatric professionals, can help get the conversation started or may need to step in.

What needs to be conveyed about parental mental illness depends on the developmental age and maturity of the child. Teens can understand the nature of mental illness as a brain disorder, but often have acquired misinformation from peers, the other parent who is hurt and angry, or the Internet that needs to be respectfully corrected. They may be less willing to have such a discussion than would younger children, as they may have experienced verbal or physical aggression from the parent, embarrassment in front of peers, or teasing by classmates. They may have developed coping strategies of writing off the parent, defiance, aggressive responses, or denial.

It is important to find a relaxed moment and begin by saying, “May I ask what you have noticed about your parent’s behavior?” and “What did you make of it?” If they do not answer, you could add, “You know, like acting different or strange.” It is very valuable to be able to name it, if you know the diagnosis, to make the illness more objective. Teens are typically egocentric and wonder if they are prone to develop a similar condition, as well as anxious about who will be there for them if the parent does not get better; both are good questions to address.

Both adolescents and school-aged children are very attuned to when things in life are “unfair,” and having an ill parent certainly qualifies. It is important for whoever has this discussion to empathize, and to acknowledge that it is unfair and that feeling angry, sad, or confused is natural – without giving them permission for misbehavior. It may be easier for some children to have a journal in which to write questions to have an adult answer later. Any child can be given hope by knowing that the parent is getting help (or that you will work on this), especially if the parents themselves say they are trying to get better. The children need to know that although mental illness tends to get better and worse at unexpected times, mental health can be improved. It is essential that children of school age and older have a clear plan for what to do if the parent loses control or is in danger. This might include getting out of the house and calling the parent’s partner, a trusted neighbor, or 911.

While teens may feel guilty about their anger or things they might have said or done in reaction to the ill parent, school-aged and preschool children are more likely to feel guilty that they somehow contributed to the parent’s condition through misbehavior or some imagined influence. Eliciting these thoughts may simply require asking, “What thoughts have you had about why this happened?” or having another family member prompting them by saying, “I have been wondering if there is something I should have done. Have you wondered about that, too?”

What is harder to explain is the impression children may get that their depressed or psychotic or drug-abusing parents no longer love them; and the parents may have said hurtful things to them. School-aged children can be told and can understand an illness of “the feelings and thinking parts of the brain” as being similar to other physical illnesses, such as the flu, that “make people act tired or grouchy or say things they do not mean.” Children of all ages need to be reassured that, inside, their parents still love them, and “it is the illness talking” if they act or speak otherwise. In the case of substance abuse disorders, which might seem more of a choice by the parent than would other mental illnesses, it can be helpful to point out that “drugs and alcohol can be stronger than people and can take over their brains.” The National Alliance on Mental Illness (www.nami.org) offers support programs that may be helpful for older children.

Sometimes families may resist telling a child about parental mental illness because of personal beliefs, cultural stigma, or privacy concerns. I try to emphasize that by being honest in giving children an understanding of their parents’ mental illness, one is helping them trust the adults in their lives. They need the truth and also to practice formulating things to say to counter those who have incorrect ideas, or who insult or make fun of them. They need the truth about mental illness to make sense of their experiences so they can feel lovable and hopeful about their own futures and can recognize illness symptoms in themselves. Giving them words, ensuring their safety and support, and strengthening their coping can help buffer the impact of this common adverse childhood experience to help prevent long-term negative effects.
 

Dr. Howard is assistant professor of pediatrics at Johns Hopkins University, Baltimore, and creator of CHADIS (www.CHADIS.com). She had no other relevant disclosures. Dr. Howard’s contribution to this publication was as a paid expert to MDedge News. E-mail her at [email protected].

Hand, foot, and mouth disease (HFMD) has become a global challenge since its first description in 1957 in New Zealand and Canada. Clinicians readily recognize the characteristic syndrome in young children: fever associated with a papulovesicular rash affecting the palms or soles, or both, usually in spring, summer, or fall. In most cases the disease is self-limited, and brief. However, aseptic meningitis, brainstem encephalitis, acute flaccid paralysis or autonomic nervous system deregulation or cardiorespiratory failure may complicate the clinical course.

Copyright MidgleyDJ/Wikimedia Commons

HFMD is caused by enterovirus A (formerly called human enterovirus A) which consists of 25 serotypes including multiple coxsackie A viruses, multiple enteroviruses, simian enteroviruses, and baboon enterovirus A13. The clinical spectrum spans from herpangina, characterized by fever and painful mouth ulcers most prominent in the posterior oral cavity (uvula, tonsils, soft plates, and anterior pharyngeal folds), to HFMD with papulovesicular rash on palms and soles with or without mouth ulcers/vesicles.¹ In atypical cases, the rash may be maculopapular and may include the buttocks, knees or elbows.

In the United States, the predominant cause is coxsackie A16. However, coxsackie A6 appears to be emerging; often more than one HFMD causing virus is circulating concurrently and clinically indistinguishable. Globally, especially, in Asia, enterovirus 1 is a major cause of HFMD and more often associated with prominent central nervous system involvement. Disease can be sporadic or epidemic. An outbreak is usually defined as two or more cases within a defined geographic area; global epidemics as large as 1.5 million cases (Taiwan, 1998) have been reported, and outbreaks in China involving tens of thousands with multiple deaths have been reported.

In 2015, an outbreak of HFMD occurred during basic military training at Lackland Air Force Base, Bexar County, Texas, due to coxsackie A6.² The illness was characterized by prodromal symptoms of fever and malaise followed by erosive stomatitis and a rash that began on the palms and soles. The rate of infection among trainees was 4.7% (50 of 1,054 persons).

The differential diagnosis includes aphthous ulcers and herpetic gingivostomatitis.³ Aphthous ulcers are seen more commonly in older children and adolescents, are often recurrent, are not seasonal, and are not associated with rash. Herpes simplex virus gingivostomatitis usually has a febrile prodrome, perioral lesions are frequent in addition to gum and tongue involvement, and gingival bleeding is common. HFMD usually has an incubation period of 3-5 days and fever, malaise, and myalgia prodrome followed by onset of oral and dermatologic manifestations in sequence. The skin rash has features that may overlap with varicella, erythema multiforme (EM) or drug eruption. Varicella usually involves the face before spreading to the extremities, and the lesions are characterized by umbilication and subsequent crusting. EM is characterized by target lesions and drug eruptions are morbilliform or maculopapular. The majority of cases of HFMD are diagnosed clinically; polymerase chain reaction testing is available and best performed on throat or vesicle specimens. Serologic testing for A16 and enterovirus 71 (IgM) is available. Infected patients shed virus for 2-4 weeks and virus is stable in the environment resulting in fecal-oral or oral-oral transmission.

Atypical features of HFMD include occurrence in the winter (outbreak in Alabama in 2011/2012) or an atypical distribution of rash involving the antecubital and popliteal fossae distribution of rash, or “eczema coxsackium” – the accentuation of rash in areas previously affected by atopic dermatitis. Additional features may include nail dystrophies that manifest as Beau lines (deep grooved lines that run from side to side on the fingernail or the toenail) and nail shedding.

A spectrum of neurologic complications has been observed, more frequently with EV71 and more frequently in Asia. The spectrum includes aseptic meningitis and brainstem encephalitis. Progressive cardiopulmonary failure also can be observed in severe cases. The hallmark of severe disease is often presentation with high fever, sweating, mottled skin, and tachycardia. Early signs of CNS involvement include myoclonic jerks, ataxia, and “wandering eyes.”³ Elevated white blood count and/or hyperglycemia may distinguish children with severe disease from benign disease. Anecdotal reports of response to treatment with high-dose methylprednisolone and intravenous immune globulin suggest that the neurologic disease may be an autoimmune phenomenon.

The clinician’s primary role is to accurately diagnose HFMD, provide supportive care for fever and dehydration, and identify those with early signs or laboratory features heralding a more severe course of disease.³ The Centers for Disease Control and Prevention recommends frequent hand washing after toileting and changing diapers, disinfecting surfaces such as toys, avoiding close contact with infected individuals or sharing of personal items for all affected patients. No antiviral treatment is available although improvement following early treatment with acyclovir has been reported anecdotally. Intravenous immunoglobulin has been used in severe cases in Asia with retrospective data analysis suggesting a potential for improvement when administered prior to cardiopulmonary arrest.¹

Dr. Pelton is professor of pediatrics and epidemiology at Boston University. Dr. Pelton said he had no relevant financial disclosures. Email him at [email protected].

References

1. Cleveland Clinic Journal of Medicine 2014;81(9):537-43.

2. Morbidity and Mortality Weekly Report MMWR. 2016 Jul 8;65(26);678-80.

3. A Guide to clinical management and public health response for hand, foot and mouth disease (HFMD).

Hand, foot, and mouth disease (HFMD) has become a global challenge since its first description in 1957 in New Zealand and Canada. Clinicians readily recognize the characteristic syndrome in young children: fever associated with a papulovesicular rash affecting the palms or soles, or both, usually in spring, summer, or fall. In most cases the disease is self-limited, and brief. However, aseptic meningitis, brainstem encephalitis, acute flaccid paralysis or autonomic nervous system deregulation or cardiorespiratory failure may complicate the clinical course.

Copyright MidgleyDJ/Wikimedia Commons

HFMD is caused by enterovirus A (formerly called human enterovirus A) which consists of 25 serotypes including multiple coxsackie A viruses, multiple enteroviruses, simian enteroviruses, and baboon enterovirus A13. The clinical spectrum spans from herpangina, characterized by fever and painful mouth ulcers most prominent in the posterior oral cavity (uvula, tonsils, soft plates, and anterior pharyngeal folds), to HFMD with papulovesicular rash on palms and soles with or without mouth ulcers/vesicles.¹ In atypical cases, the rash may be maculopapular and may include the buttocks, knees or elbows.

In the United States, the predominant cause is coxsackie A16. However, coxsackie A6 appears to be emerging; often more than one HFMD causing virus is circulating concurrently and clinically indistinguishable. Globally, especially, in Asia, enterovirus 1 is a major cause of HFMD and more often associated with prominent central nervous system involvement. Disease can be sporadic or epidemic. An outbreak is usually defined as two or more cases within a defined geographic area; global epidemics as large as 1.5 million cases (Taiwan, 1998) have been reported, and outbreaks in China involving tens of thousands with multiple deaths have been reported.

In 2015, an outbreak of HFMD occurred during basic military training at Lackland Air Force Base, Bexar County, Texas, due to coxsackie A6.² The illness was characterized by prodromal symptoms of fever and malaise followed by erosive stomatitis and a rash that began on the palms and soles. The rate of infection among trainees was 4.7% (50 of 1,054 persons).

The differential diagnosis includes aphthous ulcers and herpetic gingivostomatitis.³ Aphthous ulcers are seen more commonly in older children and adolescents, are often recurrent, are not seasonal, and are not associated with rash. Herpes simplex virus gingivostomatitis usually has a febrile prodrome, perioral lesions are frequent in addition to gum and tongue involvement, and gingival bleeding is common. HFMD usually has an incubation period of 3-5 days and fever, malaise, and myalgia prodrome followed by onset of oral and dermatologic manifestations in sequence. The skin rash has features that may overlap with varicella, erythema multiforme (EM) or drug eruption. Varicella usually involves the face before spreading to the extremities, and the lesions are characterized by umbilication and subsequent crusting. EM is characterized by target lesions and drug eruptions are morbilliform or maculopapular. The majority of cases of HFMD are diagnosed clinically; polymerase chain reaction testing is available and best performed on throat or vesicle specimens. Serologic testing for A16 and enterovirus 71 (IgM) is available. Infected patients shed virus for 2-4 weeks and virus is stable in the environment resulting in fecal-oral or oral-oral transmission.

Atypical features of HFMD include occurrence in the winter (outbreak in Alabama in 2011/2012) or an atypical distribution of rash involving the antecubital and popliteal fossae distribution of rash, or “eczema coxsackium” – the accentuation of rash in areas previously affected by atopic dermatitis. Additional features may include nail dystrophies that manifest as Beau lines (deep grooved lines that run from side to side on the fingernail or the toenail) and nail shedding.

A spectrum of neurologic complications has been observed, more frequently with EV71 and more frequently in Asia. The spectrum includes aseptic meningitis and brainstem encephalitis. Progressive cardiopulmonary failure also can be observed in severe cases. The hallmark of severe disease is often presentation with high fever, sweating, mottled skin, and tachycardia. Early signs of CNS involvement include myoclonic jerks, ataxia, and “wandering eyes.”³ Elevated white blood count and/or hyperglycemia may distinguish children with severe disease from benign disease. Anecdotal reports of response to treatment with high-dose methylprednisolone and intravenous immune globulin suggest that the neurologic disease may be an autoimmune phenomenon.

The clinician’s primary role is to accurately diagnose HFMD, provide supportive care for fever and dehydration, and identify those with early signs or laboratory features heralding a more severe course of disease.³ The Centers for Disease Control and Prevention recommends frequent hand washing after toileting and changing diapers, disinfecting surfaces such as toys, avoiding close contact with infected individuals or sharing of personal items for all affected patients. No antiviral treatment is available although improvement following early treatment with acyclovir has been reported anecdotally. Intravenous immunoglobulin has been used in severe cases in Asia with retrospective data analysis suggesting a potential for improvement when administered prior to cardiopulmonary arrest.¹

Dr. Pelton is professor of pediatrics and epidemiology at Boston University. Dr. Pelton said he had no relevant financial disclosures. Email him at [email protected].

References

1. Cleveland Clinic Journal of Medicine 2014;81(9):537-43.

2. Morbidity and Mortality Weekly Report MMWR. 2016 Jul 8;65(26);678-80.

3. A Guide to clinical management and public health response for hand, foot and mouth disease (HFMD).

Hand, foot, and mouth disease (HFMD) has become a global challenge since its first description in 1957 in New Zealand and Canada. Clinicians readily recognize the characteristic syndrome in young children: fever associated with a papulovesicular rash affecting the palms or soles, or both, usually in spring, summer, or fall. In most cases the disease is self-limited, and brief. However, aseptic meningitis, brainstem encephalitis, acute flaccid paralysis or autonomic nervous system deregulation or cardiorespiratory failure may complicate the clinical course.

Copyright MidgleyDJ/Wikimedia Commons

HFMD is caused by enterovirus A (formerly called human enterovirus A) which consists of 25 serotypes including multiple coxsackie A viruses, multiple enteroviruses, simian enteroviruses, and baboon enterovirus A13. The clinical spectrum spans from herpangina, characterized by fever and painful mouth ulcers most prominent in the posterior oral cavity (uvula, tonsils, soft plates, and anterior pharyngeal folds), to HFMD with papulovesicular rash on palms and soles with or without mouth ulcers/vesicles.¹ In atypical cases, the rash may be maculopapular and may include the buttocks, knees or elbows.

In the United States, the predominant cause is coxsackie A16. However, coxsackie A6 appears to be emerging; often more than one HFMD causing virus is circulating concurrently and clinically indistinguishable. Globally, especially, in Asia, enterovirus 1 is a major cause of HFMD and more often associated with prominent central nervous system involvement. Disease can be sporadic or epidemic. An outbreak is usually defined as two or more cases within a defined geographic area; global epidemics as large as 1.5 million cases (Taiwan, 1998) have been reported, and outbreaks in China involving tens of thousands with multiple deaths have been reported.

In 2015, an outbreak of HFMD occurred during basic military training at Lackland Air Force Base, Bexar County, Texas, due to coxsackie A6.² The illness was characterized by prodromal symptoms of fever and malaise followed by erosive stomatitis and a rash that began on the palms and soles. The rate of infection among trainees was 4.7% (50 of 1,054 persons).

The differential diagnosis includes aphthous ulcers and herpetic gingivostomatitis.³ Aphthous ulcers are seen more commonly in older children and adolescents, are often recurrent, are not seasonal, and are not associated with rash. Herpes simplex virus gingivostomatitis usually has a febrile prodrome, perioral lesions are frequent in addition to gum and tongue involvement, and gingival bleeding is common. HFMD usually has an incubation period of 3-5 days and fever, malaise, and myalgia prodrome followed by onset of oral and dermatologic manifestations in sequence. The skin rash has features that may overlap with varicella, erythema multiforme (EM) or drug eruption. Varicella usually involves the face before spreading to the extremities, and the lesions are characterized by umbilication and subsequent crusting. EM is characterized by target lesions and drug eruptions are morbilliform or maculopapular. The majority of cases of HFMD are diagnosed clinically; polymerase chain reaction testing is available and best performed on throat or vesicle specimens. Serologic testing for A16 and enterovirus 71 (IgM) is available. Infected patients shed virus for 2-4 weeks and virus is stable in the environment resulting in fecal-oral or oral-oral transmission.

Atypical features of HFMD include occurrence in the winter (outbreak in Alabama in 2011/2012) or an atypical distribution of rash involving the antecubital and popliteal fossae distribution of rash, or “eczema coxsackium” – the accentuation of rash in areas previously affected by atopic dermatitis. Additional features may include nail dystrophies that manifest as Beau lines (deep grooved lines that run from side to side on the fingernail or the toenail) and nail shedding.

A spectrum of neurologic complications has been observed, more frequently with EV71 and more frequently in Asia. The spectrum includes aseptic meningitis and brainstem encephalitis. Progressive cardiopulmonary failure also can be observed in severe cases. The hallmark of severe disease is often presentation with high fever, sweating, mottled skin, and tachycardia. Early signs of CNS involvement include myoclonic jerks, ataxia, and “wandering eyes.”³ Elevated white blood count and/or hyperglycemia may distinguish children with severe disease from benign disease. Anecdotal reports of response to treatment with high-dose methylprednisolone and intravenous immune globulin suggest that the neurologic disease may be an autoimmune phenomenon.

The clinician’s primary role is to accurately diagnose HFMD, provide supportive care for fever and dehydration, and identify those with early signs or laboratory features heralding a more severe course of disease.³ The Centers for Disease Control and Prevention recommends frequent hand washing after toileting and changing diapers, disinfecting surfaces such as toys, avoiding close contact with infected individuals or sharing of personal items for all affected patients. No antiviral treatment is available although improvement following early treatment with acyclovir has been reported anecdotally. Intravenous immunoglobulin has been used in severe cases in Asia with retrospective data analysis suggesting a potential for improvement when administered prior to cardiopulmonary arrest.¹

Dr. Pelton is professor of pediatrics and epidemiology at Boston University. Dr. Pelton said he had no relevant financial disclosures. Email him at [email protected].

References

1. Cleveland Clinic Journal of Medicine 2014;81(9):537-43.

2. Morbidity and Mortality Weekly Report MMWR. 2016 Jul 8;65(26);678-80.

3. A Guide to clinical management and public health response for hand, foot and mouth disease (HFMD).

On Aug.16, the New York University School of Medicine announced it was offering full-tuition scholarships to all current and future students in its MD Program, regardless of need or merit – and that this policy also would apply to all matriculating students for the remainder of their medical school education at NYU.

This bold initiative, they stated, was being instituted to simultaneously address the rising costs of medical education while still attracting the best and brightest to careers in medicine. In doing so, NYU School of Medicine (at which I hold a faculty appointment) became the first Top 10–ranked medical school in the nation to do so.

The symbolism of this announcement was noticeable: It was made at the medical school’s annual white coat ceremony, when each new student is presented with a white lab coat to mark the beginning of their medical education and training.

I count myself among many medical professionals and, indeed, others outside of medicine who have long advocated for free tuition for medical education, at the very least for those who have little or no means to pay. This particularly painful burden of debt often serves as a deterrent to many individuals who are considering a career in medicine or medical research.

According to the Association of American Medical Colleges, 75% of all U.S. doctors graduated with some degree of debt in 2017. For many, the total is staggering: The median current debt for a graduating medical student is $202,000, with debt for those pursuing private medical school education rising as high as $300,000.

What might develop as a result of NYU’s decision and that of several other schools who have adopted debt-reduction policies?

First, that these programs might have a ripple effect at other medical schools, and create a movement for more students to earn a medical degree without incurring a crushing financial burden. Some other schools, like Columbia University, already have taken steps, such as replacing all student loans with scholarships creating a “debt-free” medical school. It would, indeed, be a powerful message if other schools developed similar creative solutions to this problem.

Second, there is hope that debt relief will encourage more medical school graduates to pursue careers in such specialties as family medicine, psychiatry, pediatrics, and geriatrics – because they will not have the additional financial pressure to pursue careers in more lucrative specialties in order to pay off debt. While many medical school graduates point to other issues like complex reimbursement as a greater deterrent to a specific specialty choice, I certainly hope that debt relief will have a positive effect in shifting the subspecialty paradigm.

Third, these actions might incentivize the federal government to establish an AmeriCorps-type program, in which the cost of a medical education is covered in return for a commitment to practice medicine for a period of time in underserved areas of the country. Such an approach also might motivate more medical school graduates to pursue careers in primary care specialties and help address some of the ongoing concerns related to the uneven distribution of physicians in the United States.

Another issue that often comes up is the impact of debt burden on “burnout” among medical students. This is a complex subject – and one that actually affects physicians beyond medical school and into residency training and medical practice.

There is no doubt that debt weighs heavily on the minds of medical students – and many enter medical school having sustained significant debt already from previous education in colleges and universities. However, the causes and influences on burnout in training are multifactorial. Earlier in medical school, the impact of debt obligation may be less apparent because of the other challenges students face when beginning medical school.

However, once trainees begin residency and fellowship training – and especially during early career years when many are beginning to have families – concerns about increasing financial strain become even more prominent. For many young physicians, already stressed by other extenuating factors, it would be a tremendous relief not to have that debt pursuing them.

As someone who teaches and mentors medical students and residents, I firmly believe that most students pursue a career in medicine for altruistic reasons: to help cure illness and take care of patients, to make new scientific discoveries, and to train the next generation of physicians who will follow them into the medical profession. Unfortunately, outside economic influences – such as increased competition among health care systems, shrinking reimbursements, loss of joy and meaning in medicine, increasing isolation of the caregiver from the patient and the significant cost of a medical education – lead many physicians to burn out prematurely and, for some, to leave the profession altogether.

By eliminating medical school debt, we can remove one of these constraints and make the practice of medicine as rewarding and gratifying as it has been in the past, and more accessible to those who truly wish to care for others.
 

Dr. Bernstein is a professor in the departments of psychiatry and neurology at New York University and a past president of the American Psychiatric Association.

On Aug.16, the New York University School of Medicine announced it was offering full-tuition scholarships to all current and future students in its MD Program, regardless of need or merit – and that this policy also would apply to all matriculating students for the remainder of their medical school education at NYU.

This bold initiative, they stated, was being instituted to simultaneously address the rising costs of medical education while still attracting the best and brightest to careers in medicine. In doing so, NYU School of Medicine (at which I hold a faculty appointment) became the first Top 10–ranked medical school in the nation to do so.

The symbolism of this announcement was noticeable: It was made at the medical school’s annual white coat ceremony, when each new student is presented with a white lab coat to mark the beginning of their medical education and training.

I count myself among many medical professionals and, indeed, others outside of medicine who have long advocated for free tuition for medical education, at the very least for those who have little or no means to pay. This particularly painful burden of debt often serves as a deterrent to many individuals who are considering a career in medicine or medical research.

According to the Association of American Medical Colleges, 75% of all U.S. doctors graduated with some degree of debt in 2017. For many, the total is staggering: The median current debt for a graduating medical student is $202,000, with debt for those pursuing private medical school education rising as high as $300,000.

What might develop as a result of NYU’s decision and that of several other schools who have adopted debt-reduction policies?

First, that these programs might have a ripple effect at other medical schools, and create a movement for more students to earn a medical degree without incurring a crushing financial burden. Some other schools, like Columbia University, already have taken steps, such as replacing all student loans with scholarships creating a “debt-free” medical school. It would, indeed, be a powerful message if other schools developed similar creative solutions to this problem.

Second, there is hope that debt relief will encourage more medical school graduates to pursue careers in such specialties as family medicine, psychiatry, pediatrics, and geriatrics – because they will not have the additional financial pressure to pursue careers in more lucrative specialties in order to pay off debt. While many medical school graduates point to other issues like complex reimbursement as a greater deterrent to a specific specialty choice, I certainly hope that debt relief will have a positive effect in shifting the subspecialty paradigm.

Third, these actions might incentivize the federal government to establish an AmeriCorps-type program, in which the cost of a medical education is covered in return for a commitment to practice medicine for a period of time in underserved areas of the country. Such an approach also might motivate more medical school graduates to pursue careers in primary care specialties and help address some of the ongoing concerns related to the uneven distribution of physicians in the United States.

Another issue that often comes up is the impact of debt burden on “burnout” among medical students. This is a complex subject – and one that actually affects physicians beyond medical school and into residency training and medical practice.

There is no doubt that debt weighs heavily on the minds of medical students – and many enter medical school having sustained significant debt already from previous education in colleges and universities. However, the causes and influences on burnout in training are multifactorial. Earlier in medical school, the impact of debt obligation may be less apparent because of the other challenges students face when beginning medical school.

However, once trainees begin residency and fellowship training – and especially during early career years when many are beginning to have families – concerns about increasing financial strain become even more prominent. For many young physicians, already stressed by other extenuating factors, it would be a tremendous relief not to have that debt pursuing them.

As someone who teaches and mentors medical students and residents, I firmly believe that most students pursue a career in medicine for altruistic reasons: to help cure illness and take care of patients, to make new scientific discoveries, and to train the next generation of physicians who will follow them into the medical profession. Unfortunately, outside economic influences – such as increased competition among health care systems, shrinking reimbursements, loss of joy and meaning in medicine, increasing isolation of the caregiver from the patient and the significant cost of a medical education – lead many physicians to burn out prematurely and, for some, to leave the profession altogether.

By eliminating medical school debt, we can remove one of these constraints and make the practice of medicine as rewarding and gratifying as it has been in the past, and more accessible to those who truly wish to care for others.
 

Dr. Bernstein is a professor in the departments of psychiatry and neurology at New York University and a past president of the American Psychiatric Association.

On Aug.16, the New York University School of Medicine announced it was offering full-tuition scholarships to all current and future students in its MD Program, regardless of need or merit – and that this policy also would apply to all matriculating students for the remainder of their medical school education at NYU.

This bold initiative, they stated, was being instituted to simultaneously address the rising costs of medical education while still attracting the best and brightest to careers in medicine. In doing so, NYU School of Medicine (at which I hold a faculty appointment) became the first Top 10–ranked medical school in the nation to do so.

The symbolism of this announcement was noticeable: It was made at the medical school’s annual white coat ceremony, when each new student is presented with a white lab coat to mark the beginning of their medical education and training.

I count myself among many medical professionals and, indeed, others outside of medicine who have long advocated for free tuition for medical education, at the very least for those who have little or no means to pay. This particularly painful burden of debt often serves as a deterrent to many individuals who are considering a career in medicine or medical research.

According to the Association of American Medical Colleges, 75% of all U.S. doctors graduated with some degree of debt in 2017. For many, the total is staggering: The median current debt for a graduating medical student is $202,000, with debt for those pursuing private medical school education rising as high as $300,000.

What might develop as a result of NYU’s decision and that of several other schools who have adopted debt-reduction policies?

First, that these programs might have a ripple effect at other medical schools, and create a movement for more students to earn a medical degree without incurring a crushing financial burden. Some other schools, like Columbia University, already have taken steps, such as replacing all student loans with scholarships creating a “debt-free” medical school. It would, indeed, be a powerful message if other schools developed similar creative solutions to this problem.

Second, there is hope that debt relief will encourage more medical school graduates to pursue careers in such specialties as family medicine, psychiatry, pediatrics, and geriatrics – because they will not have the additional financial pressure to pursue careers in more lucrative specialties in order to pay off debt. While many medical school graduates point to other issues like complex reimbursement as a greater deterrent to a specific specialty choice, I certainly hope that debt relief will have a positive effect in shifting the subspecialty paradigm.

Third, these actions might incentivize the federal government to establish an AmeriCorps-type program, in which the cost of a medical education is covered in return for a commitment to practice medicine for a period of time in underserved areas of the country. Such an approach also might motivate more medical school graduates to pursue careers in primary care specialties and help address some of the ongoing concerns related to the uneven distribution of physicians in the United States.

Another issue that often comes up is the impact of debt burden on “burnout” among medical students. This is a complex subject – and one that actually affects physicians beyond medical school and into residency training and medical practice.

There is no doubt that debt weighs heavily on the minds of medical students – and many enter medical school having sustained significant debt already from previous education in colleges and universities. However, the causes and influences on burnout in training are multifactorial. Earlier in medical school, the impact of debt obligation may be less apparent because of the other challenges students face when beginning medical school.

However, once trainees begin residency and fellowship training – and especially during early career years when many are beginning to have families – concerns about increasing financial strain become even more prominent. For many young physicians, already stressed by other extenuating factors, it would be a tremendous relief not to have that debt pursuing them.

As someone who teaches and mentors medical students and residents, I firmly believe that most students pursue a career in medicine for altruistic reasons: to help cure illness and take care of patients, to make new scientific discoveries, and to train the next generation of physicians who will follow them into the medical profession. Unfortunately, outside economic influences – such as increased competition among health care systems, shrinking reimbursements, loss of joy and meaning in medicine, increasing isolation of the caregiver from the patient and the significant cost of a medical education – lead many physicians to burn out prematurely and, for some, to leave the profession altogether.

By eliminating medical school debt, we can remove one of these constraints and make the practice of medicine as rewarding and gratifying as it has been in the past, and more accessible to those who truly wish to care for others.
 

Dr. Bernstein is a professor in the departments of psychiatry and neurology at New York University and a past president of the American Psychiatric Association.

The management of bipolar disorder during pregnancy is a critical clinical situation demanding great attention to issues such as reproductive safety of psychiatric medications used by women with bipolar disorder to maintain emotional well-being, compared with the established risk of relapse if patients stopped those medications.

Antonio_Diaz/Thinkstock

Treatment of bipolar disorder frequently includes mainstay treatment with mood stabilizers such as sodium valproate, lithium, lamotrigine, and second-generation atypical antipsychotics. While we have robust information regarding the reproductive safety of sodium valproate, it is a teratogen with a very high risk for neural tube defects. In contrast, data over the 15 years have been very supportive of the reproductive safety of lamotrigine. The last decade has seen growing use of second-generation antipsychotics, so-called atypical antipsychotics. There has been growing interest in the reproductive safety of these medicines given their use both for acute mania and for prophylaxis of bipolar disorder; they also are used as an adjunct to treat patients with major depression. Atypical antipsychotics are widely used off-label to treat obsessive compulsive disorder, other anxiety disorders, and a spectrum of psychiatric illness.

Until relatively recently, data on the reproductive safety of second-generation atypical antipsychotics has been relatively sparse, with the small number of prospective studies yielding a small total number of patients. Over the same period of time, the National Pregnancy Registry for Atypical Antipsychotics (NPRAA) at Massachusetts General Hospital (MGH) was established, modeled after the North American Antiepileptic Drug Registry as a prospective registry of women with histories of first trimester exposure to atypical antipsychotics.

Over the last several years, the MGH NPRAA has accumulated very rigorous, prospectively ascertained data on outcomes following first trimester exposure to the atypical antipsychotics. Given the high prevalence of the use of this class of medications in reproductive-age women, data on the reproductive safety of atypical antipsychotics has been anxiously awaited and also has been relatively reassuring based on sources such as the NPRAA and also analyses of data from large administrative databases. For example, a recent paper published in JAMA Psychiatry by KF Huybrechts and her colleagues of 1,360,101 pregnant women who were enrolled in the Medicaid Analytic Extract database found an adjusted relative risk of 1.05 for congenital malformations in births for patients exposed to atypical antipsychotics (2016;73[9]:938-46).

Patients most often present with questions not about the reproductive safety of a class of medications, but about the safety of a particular medicine. A recent paper from our own group published in the American Journal of Psychiatry using data from the MGH NPRAA–described outcome of fetal exposure to quetiapine with a total of 152 women exposed to quetiapine and 205 unexposed patients. These patients were prospectively followed and compared with controls not exposed to the atypical antipsychotic but having a history of psychiatric morbidity. There was a 1.29% risk of major malformations in women exposed to quetiapine vs. 1.43% in the unexposed population (2018 Aug 16. doi: 10.1176/appi.ajp.2018.18010098).

The positive features of the MGH NPRAA include the careful rigorous assessments of patients over time as well as review of their obstetric, neonatal, and pediatric records up to 6 months, with blinded adjudication of outcome. The limitation of the small sample size remains with findings including relatively wide confidence intervals. With that being said, included in the paper in the discussion section is a pooled analysis of prospective data regarding quetiapine from the world’s literature that supports the findings of even this small prospective study in our registry, namely flat risk or absence of data suggesting that quetiapine is a major teratogen (pooled risk ratio, 1.03; 95% confidence interval, 0.89-1.19).

The delineation of risk for atypical antipsychotics is an extremely important area of research from a clinical point of view because it may help inform choices made by women with bipolar disorder who are well and maintained on these medicines as they wrestle with risk of relapse when agents are discontinued on one hand and reproductive safety concerns on the other.

Although not as widely used as perhaps a decade ago, data on the reproductive safety of lithium only continue to grow and become more refined. Use of lithium, a known teratogen with studies dating back to the 1970s, has an increased risk for cardiovascular malformations with the classic reference being to the small heightened risk of Ebstein’s anomaly (0.05%-0.1%). More recent studies from large administrative databases have been published with new data regarding risk of fetal exposure to lithium.

Two recent studies on lithium help to clarify some lingering questions about lithium use during pregnancy and risk for cardiovascular malformations. In one study published in the New England Journal of Medicine, researchers have demonstrated a small increased risk for cardiac malformations associated with using lithium during the first trimester (2017;376:2245-54). After researchers controlled for potential confounding factors, the adjusted risk ratio for cardiac malformations among infants exposed to lithium was 1.65 (95% CI, 1.02-2.68), compared with nonexposed infants. In a second study published in Lancet Psychiatry (2018 Aug;5[8]:644-52), a primary data meta-analysis of pregnant women and their children from six international cohorts in Denmark, Sweden, and Canada, there was no significant difference in major cardiac malformations between the lithium-exposed group, 2.1% (0.5%-3.7%), and the reference group, 1.6% (1.0%-2.1%).

Women with particularly brittle bipolar disorder or with histories of response to lithium may, in consultation with their doctors, consider use of lithium during pregnancy given the almost 50-year history of data accumulation on its reproductive safety, compared with some of the other mood stabilizers for which there is either confirmed teratogenicity (sodium valproate) or still incomplete data. Moreover, given the high risk for postpartum relapse of mood disorder in women who suffer from bipolar disorder, it is important to remember that the most robust data on prophylactic benefit of mood stabilizer during the peripartum period are with lithium.

Reproductive age women with bipolar disorder have for decades been caught between a teratologic rock and a clinical hard place. More recent data that have emerged from rigorously conducted registries and carefully analyzed administrative databases allow for more effective collaboration between patient and doctor as together they make personal decisions that match individual clinical situations with personal wishes.

Dr. Cohen is the director of the Ammon-Pinizzotto Center for Women’s Mental Health at Massachusetts General Hospital in Boston, which provides information resources and conducts clinical care and research in reproductive mental health. He has been a consultant to manufacturers of psychiatric medications. Email him at [email protected].

The management of bipolar disorder during pregnancy is a critical clinical situation demanding great attention to issues such as reproductive safety of psychiatric medications used by women with bipolar disorder to maintain emotional well-being, compared with the established risk of relapse if patients stopped those medications.

Antonio_Diaz/Thinkstock

Treatment of bipolar disorder frequently includes mainstay treatment with mood stabilizers such as sodium valproate, lithium, lamotrigine, and second-generation atypical antipsychotics. While we have robust information regarding the reproductive safety of sodium valproate, it is a teratogen with a very high risk for neural tube defects. In contrast, data over the 15 years have been very supportive of the reproductive safety of lamotrigine. The last decade has seen growing use of second-generation antipsychotics, so-called atypical antipsychotics. There has been growing interest in the reproductive safety of these medicines given their use both for acute mania and for prophylaxis of bipolar disorder; they also are used as an adjunct to treat patients with major depression. Atypical antipsychotics are widely used off-label to treat obsessive compulsive disorder, other anxiety disorders, and a spectrum of psychiatric illness.

Until relatively recently, data on the reproductive safety of second-generation atypical antipsychotics has been relatively sparse, with the small number of prospective studies yielding a small total number of patients. Over the same period of time, the National Pregnancy Registry for Atypical Antipsychotics (NPRAA) at Massachusetts General Hospital (MGH) was established, modeled after the North American Antiepileptic Drug Registry as a prospective registry of women with histories of first trimester exposure to atypical antipsychotics.

Over the last several years, the MGH NPRAA has accumulated very rigorous, prospectively ascertained data on outcomes following first trimester exposure to the atypical antipsychotics. Given the high prevalence of the use of this class of medications in reproductive-age women, data on the reproductive safety of atypical antipsychotics has been anxiously awaited and also has been relatively reassuring based on sources such as the NPRAA and also analyses of data from large administrative databases. For example, a recent paper published in JAMA Psychiatry by KF Huybrechts and her colleagues of 1,360,101 pregnant women who were enrolled in the Medicaid Analytic Extract database found an adjusted relative risk of 1.05 for congenital malformations in births for patients exposed to atypical antipsychotics (2016;73[9]:938-46).

Patients most often present with questions not about the reproductive safety of a class of medications, but about the safety of a particular medicine. A recent paper from our own group published in the American Journal of Psychiatry using data from the MGH NPRAA–described outcome of fetal exposure to quetiapine with a total of 152 women exposed to quetiapine and 205 unexposed patients. These patients were prospectively followed and compared with controls not exposed to the atypical antipsychotic but having a history of psychiatric morbidity. There was a 1.29% risk of major malformations in women exposed to quetiapine vs. 1.43% in the unexposed population (2018 Aug 16. doi: 10.1176/appi.ajp.2018.18010098).

The positive features of the MGH NPRAA include the careful rigorous assessments of patients over time as well as review of their obstetric, neonatal, and pediatric records up to 6 months, with blinded adjudication of outcome. The limitation of the small sample size remains with findings including relatively wide confidence intervals. With that being said, included in the paper in the discussion section is a pooled analysis of prospective data regarding quetiapine from the world’s literature that supports the findings of even this small prospective study in our registry, namely flat risk or absence of data suggesting that quetiapine is a major teratogen (pooled risk ratio, 1.03; 95% confidence interval, 0.89-1.19).

The delineation of risk for atypical antipsychotics is an extremely important area of research from a clinical point of view because it may help inform choices made by women with bipolar disorder who are well and maintained on these medicines as they wrestle with risk of relapse when agents are discontinued on one hand and reproductive safety concerns on the other.

Although not as widely used as perhaps a decade ago, data on the reproductive safety of lithium only continue to grow and become more refined. Use of lithium, a known teratogen with studies dating back to the 1970s, has an increased risk for cardiovascular malformations with the classic reference being to the small heightened risk of Ebstein’s anomaly (0.05%-0.1%). More recent studies from large administrative databases have been published with new data regarding risk of fetal exposure to lithium.

Two recent studies on lithium help to clarify some lingering questions about lithium use during pregnancy and risk for cardiovascular malformations. In one study published in the New England Journal of Medicine, researchers have demonstrated a small increased risk for cardiac malformations associated with using lithium during the first trimester (2017;376:2245-54). After researchers controlled for potential confounding factors, the adjusted risk ratio for cardiac malformations among infants exposed to lithium was 1.65 (95% CI, 1.02-2.68), compared with nonexposed infants. In a second study published in Lancet Psychiatry (2018 Aug;5[8]:644-52), a primary data meta-analysis of pregnant women and their children from six international cohorts in Denmark, Sweden, and Canada, there was no significant difference in major cardiac malformations between the lithium-exposed group, 2.1% (0.5%-3.7%), and the reference group, 1.6% (1.0%-2.1%).

Women with particularly brittle bipolar disorder or with histories of response to lithium may, in consultation with their doctors, consider use of lithium during pregnancy given the almost 50-year history of data accumulation on its reproductive safety, compared with some of the other mood stabilizers for which there is either confirmed teratogenicity (sodium valproate) or still incomplete data. Moreover, given the high risk for postpartum relapse of mood disorder in women who suffer from bipolar disorder, it is important to remember that the most robust data on prophylactic benefit of mood stabilizer during the peripartum period are with lithium.

Reproductive age women with bipolar disorder have for decades been caught between a teratologic rock and a clinical hard place. More recent data that have emerged from rigorously conducted registries and carefully analyzed administrative databases allow for more effective collaboration between patient and doctor as together they make personal decisions that match individual clinical situations with personal wishes.

Dr. Cohen is the director of the Ammon-Pinizzotto Center for Women’s Mental Health at Massachusetts General Hospital in Boston, which provides information resources and conducts clinical care and research in reproductive mental health. He has been a consultant to manufacturers of psychiatric medications. Email him at [email protected].

The management of bipolar disorder during pregnancy is a critical clinical situation demanding great attention to issues such as reproductive safety of psychiatric medications used by women with bipolar disorder to maintain emotional well-being, compared with the established risk of relapse if patients stopped those medications.

Antonio_Diaz/Thinkstock

Treatment of bipolar disorder frequently includes mainstay treatment with mood stabilizers such as sodium valproate, lithium, lamotrigine, and second-generation atypical antipsychotics. While we have robust information regarding the reproductive safety of sodium valproate, it is a teratogen with a very high risk for neural tube defects. In contrast, data over the 15 years have been very supportive of the reproductive safety of lamotrigine. The last decade has seen growing use of second-generation antipsychotics, so-called atypical antipsychotics. There has been growing interest in the reproductive safety of these medicines given their use both for acute mania and for prophylaxis of bipolar disorder; they also are used as an adjunct to treat patients with major depression. Atypical antipsychotics are widely used off-label to treat obsessive compulsive disorder, other anxiety disorders, and a spectrum of psychiatric illness.

Until relatively recently, data on the reproductive safety of second-generation atypical antipsychotics has been relatively sparse, with the small number of prospective studies yielding a small total number of patients. Over the same period of time, the National Pregnancy Registry for Atypical Antipsychotics (NPRAA) at Massachusetts General Hospital (MGH) was established, modeled after the North American Antiepileptic Drug Registry as a prospective registry of women with histories of first trimester exposure to atypical antipsychotics.

Over the last several years, the MGH NPRAA has accumulated very rigorous, prospectively ascertained data on outcomes following first trimester exposure to the atypical antipsychotics. Given the high prevalence of the use of this class of medications in reproductive-age women, data on the reproductive safety of atypical antipsychotics has been anxiously awaited and also has been relatively reassuring based on sources such as the NPRAA and also analyses of data from large administrative databases. For example, a recent paper published in JAMA Psychiatry by KF Huybrechts and her colleagues of 1,360,101 pregnant women who were enrolled in the Medicaid Analytic Extract database found an adjusted relative risk of 1.05 for congenital malformations in births for patients exposed to atypical antipsychotics (2016;73[9]:938-46).

Patients most often present with questions not about the reproductive safety of a class of medications, but about the safety of a particular medicine. A recent paper from our own group published in the American Journal of Psychiatry using data from the MGH NPRAA–described outcome of fetal exposure to quetiapine with a total of 152 women exposed to quetiapine and 205 unexposed patients. These patients were prospectively followed and compared with controls not exposed to the atypical antipsychotic but having a history of psychiatric morbidity. There was a 1.29% risk of major malformations in women exposed to quetiapine vs. 1.43% in the unexposed population (2018 Aug 16. doi: 10.1176/appi.ajp.2018.18010098).

The positive features of the MGH NPRAA include the careful rigorous assessments of patients over time as well as review of their obstetric, neonatal, and pediatric records up to 6 months, with blinded adjudication of outcome. The limitation of the small sample size remains with findings including relatively wide confidence intervals. With that being said, included in the paper in the discussion section is a pooled analysis of prospective data regarding quetiapine from the world’s literature that supports the findings of even this small prospective study in our registry, namely flat risk or absence of data suggesting that quetiapine is a major teratogen (pooled risk ratio, 1.03; 95% confidence interval, 0.89-1.19).

The delineation of risk for atypical antipsychotics is an extremely important area of research from a clinical point of view because it may help inform choices made by women with bipolar disorder who are well and maintained on these medicines as they wrestle with risk of relapse when agents are discontinued on one hand and reproductive safety concerns on the other.

Although not as widely used as perhaps a decade ago, data on the reproductive safety of lithium only continue to grow and become more refined. Use of lithium, a known teratogen with studies dating back to the 1970s, has an increased risk for cardiovascular malformations with the classic reference being to the small heightened risk of Ebstein’s anomaly (0.05%-0.1%). More recent studies from large administrative databases have been published with new data regarding risk of fetal exposure to lithium.

Two recent studies on lithium help to clarify some lingering questions about lithium use during pregnancy and risk for cardiovascular malformations. In one study published in the New England Journal of Medicine, researchers have demonstrated a small increased risk for cardiac malformations associated with using lithium during the first trimester (2017;376:2245-54). After researchers controlled for potential confounding factors, the adjusted risk ratio for cardiac malformations among infants exposed to lithium was 1.65 (95% CI, 1.02-2.68), compared with nonexposed infants. In a second study published in Lancet Psychiatry (2018 Aug;5[8]:644-52), a primary data meta-analysis of pregnant women and their children from six international cohorts in Denmark, Sweden, and Canada, there was no significant difference in major cardiac malformations between the lithium-exposed group, 2.1% (0.5%-3.7%), and the reference group, 1.6% (1.0%-2.1%).

Women with particularly brittle bipolar disorder or with histories of response to lithium may, in consultation with their doctors, consider use of lithium during pregnancy given the almost 50-year history of data accumulation on its reproductive safety, compared with some of the other mood stabilizers for which there is either confirmed teratogenicity (sodium valproate) or still incomplete data. Moreover, given the high risk for postpartum relapse of mood disorder in women who suffer from bipolar disorder, it is important to remember that the most robust data on prophylactic benefit of mood stabilizer during the peripartum period are with lithium.

Reproductive age women with bipolar disorder have for decades been caught between a teratologic rock and a clinical hard place. More recent data that have emerged from rigorously conducted registries and carefully analyzed administrative databases allow for more effective collaboration between patient and doctor as together they make personal decisions that match individual clinical situations with personal wishes.

Dr. Cohen is the director of the Ammon-Pinizzotto Center for Women’s Mental Health at Massachusetts General Hospital in Boston, which provides information resources and conducts clinical care and research in reproductive mental health. He has been a consultant to manufacturers of psychiatric medications. Email him at [email protected].

Over the past several months, I’ve found myself increasingly reflective on the intersection of culture, relationships, and my professional work with those who have an autism spectrum disorder. Last winter, an adolescent boy treated by myself and other providers died by suicide. Adam (name changed) had been diagnosed with autism as a toddler and had struggled with anxiety and depression for several years; in the office, as he grew into an athletic teenager, Adam spoke more frequently about “not fitting in” with his peers and therapeutic focus was placed on building Adam’s sense of himself and fostering his self-confidence and perceived self-competence. His unexpected death was a tremendous shock, and his loved ones – including the clinical team – desperately searched for answers that could help add some understanding to the heartbreaking event.

Rawpixel/Thinkstock

As I attempted to search for answers and reconcile national news with my clinical and teaching experiences, I was struck by something that Andrew Solomon eloquently captured in his June 2018 New Yorker article that again touches upon the theme of connection.⁶ Mr. Solomon writes “modernity is alienating” and about how he receives correspondence from those who struggle with depression. “What is most striking to me is how alone many of them are ... these people are so alone that they are effectively invisible to the rest of us ... many of them describe suicidal feelings,” he noted.

The power of connection in our day-to-day work is undeniable. The influence of human interaction and appreciating one’s unique narrative is a bedrock of clinical care and can unquestionably allow us to better understand individual suffering, deliver optimal care, and combat shame as Hannah Gadsby boldly shared in her recent Netflix comedy special “Nanette.” This shame can drive one to experience earth-shattering depressive episodes and influence thoughts of suicide. “We simply cannot make it on our own,” Ms. Gadsby explains, “we’re humans. We’re to be connected.” Humans are indeed hardwired for connection; isolation and disconnection can lead to significant health problems and are linked with mental health concerns. The former U.S. Surgeon General Vivek H. Murthy, MD, has referred to loneliness as an epidemic, and those with autism may be at increased risk for feeling lonely and isolated.^7,8

Synthesizing thoughts about relationships, suicide, loneliness, love, well-being, and autism produces a complicated web of, well, connections. Suicide in the autism population hasn’t been well researched, but one 2016 study revealed sobering numbers about suicide being a leading cause of premature death in people with autism.⁹ How do these numbers associate with feelings of isolation, wanting to fit in, and troubles talking about emotions – all of which can characterize those with ASD? Data, not surprisingly, support the role of loneliness as a risk factor for the development of depression and suicidal ideation in those with ASD.¹⁰ In addition, social-communication challenges, even in the absence of an autism diagnosis, are related to depression and suicidality.¹¹ Another recent study showed a relationship between autistic traits and depression symptoms, an association seemingly linked to being bullied.¹² We cannot continue to hold onto the myth that individuals with autism don’t desire relationships and love because it’s these desires and not being able to fulfill them, limited opportunities to engage in meaningful experiences, and feeling different that can lead to negative outcomes.

Dr. Dickerson, a child and adolescent psychiatrist, is assistant professor of psychiatry at the University of Vermont, Burlington, where he is director of the autism diagnostic clinic. Email him at [email protected].

References

1. Harv Rev Psychiatry. 2018 Jul/Aug;26(4):201-15.

2. MMWR Morb Mortal Wkly Rep. 2018 Jun 8;67(22):617-24.

3. www.healthvermont.gov/YRBS

4. MMWR Surveill Summ. 2017 Oct;66(18):1-16.

5. JAMA Pediatr. 2015 May;169(5):466-73.

6. “Anthony Bourdain, Kate Spade, and the Preventable Tragedies of Suicide,” By Anthony Solomon, The New Yorker. Jun 8, 2018.

7. “Work and the Loneliness Epidemic,” By Vivek H. Murthy, Harvard Business Review. Sep 28, 2017.

8. Child Dev. 2000 Mar-Apr;71(2):447-56.

9. Br J Psychiatry. 2016 Mar;208(3):232-8.

10. Depress Anxiety. 2018 Jul;35(7):648-57.

11. J Am Acad Child Adolesc Psychiatry. 2018 May;57(5):313-20.

12. JAMA Psychiatry. 2018 Aug 1;75(8):835-43.

13. The Lancet Psychiatry. 2017 Jun;4(6):e11.

14. “Promoting Individual, Family, and Community Connectedness to Prevent Suicidal Behavior,” Centers for Disease Control and Prevention, National Center for Injury Prevention and Control, www.cdc.gov/injury.

15. JAMA Psychiatry. 2018 Jul 11. doi: 10.1001/jamapsychiatry.2018.1776.

Over the past several months, I’ve found myself increasingly reflective on the intersection of culture, relationships, and my professional work with those who have an autism spectrum disorder. Last winter, an adolescent boy treated by myself and other providers died by suicide. Adam (name changed) had been diagnosed with autism as a toddler and had struggled with anxiety and depression for several years; in the office, as he grew into an athletic teenager, Adam spoke more frequently about “not fitting in” with his peers and therapeutic focus was placed on building Adam’s sense of himself and fostering his self-confidence and perceived self-competence. His unexpected death was a tremendous shock, and his loved ones – including the clinical team – desperately searched for answers that could help add some understanding to the heartbreaking event.

Rawpixel/Thinkstock

As I attempted to search for answers and reconcile national news with my clinical and teaching experiences, I was struck by something that Andrew Solomon eloquently captured in his June 2018 New Yorker article that again touches upon the theme of connection.⁶ Mr. Solomon writes “modernity is alienating” and about how he receives correspondence from those who struggle with depression. “What is most striking to me is how alone many of them are ... these people are so alone that they are effectively invisible to the rest of us ... many of them describe suicidal feelings,” he noted.

The power of connection in our day-to-day work is undeniable. The influence of human interaction and appreciating one’s unique narrative is a bedrock of clinical care and can unquestionably allow us to better understand individual suffering, deliver optimal care, and combat shame as Hannah Gadsby boldly shared in her recent Netflix comedy special “Nanette.” This shame can drive one to experience earth-shattering depressive episodes and influence thoughts of suicide. “We simply cannot make it on our own,” Ms. Gadsby explains, “we’re humans. We’re to be connected.” Humans are indeed hardwired for connection; isolation and disconnection can lead to significant health problems and are linked with mental health concerns. The former U.S. Surgeon General Vivek H. Murthy, MD, has referred to loneliness as an epidemic, and those with autism may be at increased risk for feeling lonely and isolated.^7,8

Synthesizing thoughts about relationships, suicide, loneliness, love, well-being, and autism produces a complicated web of, well, connections. Suicide in the autism population hasn’t been well researched, but one 2016 study revealed sobering numbers about suicide being a leading cause of premature death in people with autism.⁹ How do these numbers associate with feelings of isolation, wanting to fit in, and troubles talking about emotions – all of which can characterize those with ASD? Data, not surprisingly, support the role of loneliness as a risk factor for the development of depression and suicidal ideation in those with ASD.¹⁰ In addition, social-communication challenges, even in the absence of an autism diagnosis, are related to depression and suicidality.¹¹ Another recent study showed a relationship between autistic traits and depression symptoms, an association seemingly linked to being bullied.¹² We cannot continue to hold onto the myth that individuals with autism don’t desire relationships and love because it’s these desires and not being able to fulfill them, limited opportunities to engage in meaningful experiences, and feeling different that can lead to negative outcomes.

Dr. Dickerson, a child and adolescent psychiatrist, is assistant professor of psychiatry at the University of Vermont, Burlington, where he is director of the autism diagnostic clinic. Email him at [email protected].

References

1. Harv Rev Psychiatry. 2018 Jul/Aug;26(4):201-15.

2. MMWR Morb Mortal Wkly Rep. 2018 Jun 8;67(22):617-24.

3. www.healthvermont.gov/YRBS

4. MMWR Surveill Summ. 2017 Oct;66(18):1-16.

5. JAMA Pediatr. 2015 May;169(5):466-73.

6. “Anthony Bourdain, Kate Spade, and the Preventable Tragedies of Suicide,” By Anthony Solomon, The New Yorker. Jun 8, 2018.

7. “Work and the Loneliness Epidemic,” By Vivek H. Murthy, Harvard Business Review. Sep 28, 2017.

8. Child Dev. 2000 Mar-Apr;71(2):447-56.

9. Br J Psychiatry. 2016 Mar;208(3):232-8.

10. Depress Anxiety. 2018 Jul;35(7):648-57.

11. J Am Acad Child Adolesc Psychiatry. 2018 May;57(5):313-20.

12. JAMA Psychiatry. 2018 Aug 1;75(8):835-43.

13. The Lancet Psychiatry. 2017 Jun;4(6):e11.

14. “Promoting Individual, Family, and Community Connectedness to Prevent Suicidal Behavior,” Centers for Disease Control and Prevention, National Center for Injury Prevention and Control, www.cdc.gov/injury.

15. JAMA Psychiatry. 2018 Jul 11. doi: 10.1001/jamapsychiatry.2018.1776.

Over the past several months, I’ve found myself increasingly reflective on the intersection of culture, relationships, and my professional work with those who have an autism spectrum disorder. Last winter, an adolescent boy treated by myself and other providers died by suicide. Adam (name changed) had been diagnosed with autism as a toddler and had struggled with anxiety and depression for several years; in the office, as he grew into an athletic teenager, Adam spoke more frequently about “not fitting in” with his peers and therapeutic focus was placed on building Adam’s sense of himself and fostering his self-confidence and perceived self-competence. His unexpected death was a tremendous shock, and his loved ones – including the clinical team – desperately searched for answers that could help add some understanding to the heartbreaking event.

Rawpixel/Thinkstock

As I attempted to search for answers and reconcile national news with my clinical and teaching experiences, I was struck by something that Andrew Solomon eloquently captured in his June 2018 New Yorker article that again touches upon the theme of connection.⁶ Mr. Solomon writes “modernity is alienating” and about how he receives correspondence from those who struggle with depression. “What is most striking to me is how alone many of them are ... these people are so alone that they are effectively invisible to the rest of us ... many of them describe suicidal feelings,” he noted.

The power of connection in our day-to-day work is undeniable. The influence of human interaction and appreciating one’s unique narrative is a bedrock of clinical care and can unquestionably allow us to better understand individual suffering, deliver optimal care, and combat shame as Hannah Gadsby boldly shared in her recent Netflix comedy special “Nanette.” This shame can drive one to experience earth-shattering depressive episodes and influence thoughts of suicide. “We simply cannot make it on our own,” Ms. Gadsby explains, “we’re humans. We’re to be connected.” Humans are indeed hardwired for connection; isolation and disconnection can lead to significant health problems and are linked with mental health concerns. The former U.S. Surgeon General Vivek H. Murthy, MD, has referred to loneliness as an epidemic, and those with autism may be at increased risk for feeling lonely and isolated.^7,8

Synthesizing thoughts about relationships, suicide, loneliness, love, well-being, and autism produces a complicated web of, well, connections. Suicide in the autism population hasn’t been well researched, but one 2016 study revealed sobering numbers about suicide being a leading cause of premature death in people with autism.⁹ How do these numbers associate with feelings of isolation, wanting to fit in, and troubles talking about emotions – all of which can characterize those with ASD? Data, not surprisingly, support the role of loneliness as a risk factor for the development of depression and suicidal ideation in those with ASD.¹⁰ In addition, social-communication challenges, even in the absence of an autism diagnosis, are related to depression and suicidality.¹¹ Another recent study showed a relationship between autistic traits and depression symptoms, an association seemingly linked to being bullied.¹² We cannot continue to hold onto the myth that individuals with autism don’t desire relationships and love because it’s these desires and not being able to fulfill them, limited opportunities to engage in meaningful experiences, and feeling different that can lead to negative outcomes.

Dr. Dickerson, a child and adolescent psychiatrist, is assistant professor of psychiatry at the University of Vermont, Burlington, where he is director of the autism diagnostic clinic. Email him at [email protected].

References

1. Harv Rev Psychiatry. 2018 Jul/Aug;26(4):201-15.

2. MMWR Morb Mortal Wkly Rep. 2018 Jun 8;67(22):617-24.

3. www.healthvermont.gov/YRBS

4. MMWR Surveill Summ. 2017 Oct;66(18):1-16.

5. JAMA Pediatr. 2015 May;169(5):466-73.

6. “Anthony Bourdain, Kate Spade, and the Preventable Tragedies of Suicide,” By Anthony Solomon, The New Yorker. Jun 8, 2018.

7. “Work and the Loneliness Epidemic,” By Vivek H. Murthy, Harvard Business Review. Sep 28, 2017.

8. Child Dev. 2000 Mar-Apr;71(2):447-56.

9. Br J Psychiatry. 2016 Mar;208(3):232-8.

10. Depress Anxiety. 2018 Jul;35(7):648-57.

11. J Am Acad Child Adolesc Psychiatry. 2018 May;57(5):313-20.

12. JAMA Psychiatry. 2018 Aug 1;75(8):835-43.

13. The Lancet Psychiatry. 2017 Jun;4(6):e11.

14. “Promoting Individual, Family, and Community Connectedness to Prevent Suicidal Behavior,” Centers for Disease Control and Prevention, National Center for Injury Prevention and Control, www.cdc.gov/injury.

15. JAMA Psychiatry. 2018 Jul 11. doi: 10.1001/jamapsychiatry.2018.1776.

Bruxism is grinding and clenching of the teeth with unconscious contractions of the temporal and masseter muscles while awake or during sleep. Bruxism occurs in 8%-16% of the population and is often an underdiagnosed condition that not only leads to dental problems but also to pain in the teeth, jaw, temporomandibular joint, and neck; headaches; and potentially, to tooth loss.

Although the pathogenesis of bruxism remains unclear, multiple factors, such as physical or psychological stress, malocclusion, sleep disorders and medication side effects, can cause bruxism. Treatment can be difficult given psychogenic and neurogenic components, and bruxism can be resistant to medical and behavioral therapy. There are various treatment options for bruxism, including oral splints; medications, such as muscle relaxants; antidepressants; and botulinum toxin. Multiple studies have shown that botulinum toxin injections into the masseter and temporalis muscles result in relaxation of the muscles and improvement of bruxism and the pain associated with chronic clenching and grinding.

McLean Dermatology and Skincare Center

In a recent study by Al-Wayli, 50 subjects who reported nocturnal bruxism were randomized to receive botulinum toxin versus conventional treatment (pharmacotherapy or oral splints). After 3 weeks, 2 months, 6 months, and 1 year, patients who received botulinum toxin had significantly less pain after only one treatment than did the traditional treatment group. Similarly, in a study by Lee et al., subjects randomized to receive botulinum toxin versus a placebo saline injections showed not only decreased pain but also decreased bruxism seen with nocturnal electromyography.

In our clinic, botulinum toxin is a safe, effective treatment for patients who grind, clench, develop temporal-mandibular joint pain, or have masseter hypertrophy. Botulinum toxin when injected into the temporalis and masseter muscles also helps with tension headaches and migraines related to clenching of the jaw. Albeit effective, the dose of botulinum toxin used in the aforementioned studies ranged between 25 U and 40 U of botulinum toxin and were lower than what we have found to be effective. Our patients receive 50 U botulinum toxin in each masseter muscle (100 U total). In a small minority of our patients, the temporalis muscle also needed 15-20 U per side as well. Clinical improvement starts within 3-5 days, and patients can expect to have relaxation of the muscle and decreased pain for 6 months. Side effects include mild swelling and bruising. Rarely, if the injection is not performed properly, the risorius muscle may be paralyzed, leading to an asymmetric smile. In addition, if the botulinum toxin is underdosed, the pain may not completely subside and the patient may report some symptoms returning within a couple of weeks of the initial treatment. Most patients also report thinning of the face and jaw, which is a much anticipated and appreciated result. Masseter hypertrophy with and without bruxism is treated similarly with botulinum toxin to sculpt the lower face.

Bruxism is a growing problem leading to facial pain, headaches, migraines, and significant dental pathology. Traditional treatments have been ineffective at treating the pain and masseter hypertrophy associated with chronic grinding and clenching. Botulinum toxin is a safe, effective, treatment with little downtime or side effects for treating both the neurogenic and muscular components of bruxism.


Dr. Lily Talakoub and Dr. Naissan Wesley and are co-contributors to this column. Dr. Talakoub is in private practice in McLean, Va. This month’s column is by Dr. Talakoub. Dr. Wesley practices dermatology in Beverly Hills, Calif. Write to them at [email protected]. They had no relevant disclosures.
 

References

Al-Wayli H. J Clin Exp Dent. 2017 Jan 1;9(1):e112-e117.

Asutay F et al. Pain Res Manag. 2017;2017:6264146. doi: 10.1155/2017/6264146.

Lee SJ et al. Am J Phys Med Rehabil. 2010 Jan;89(1):16-23.

Santamato A et al. J Chiropr Med. 2010 Sep;9(3):132-7.

Shetty S et al. J Indian Prosthodont Soc. 2010 Sep;10(3):141-8.

Tan EK et al. J Am Dent Assoc. 2000 Feb;131(2):211-6.

Bruxism is grinding and clenching of the teeth with unconscious contractions of the temporal and masseter muscles while awake or during sleep. Bruxism occurs in 8%-16% of the population and is often an underdiagnosed condition that not only leads to dental problems but also to pain in the teeth, jaw, temporomandibular joint, and neck; headaches; and potentially, to tooth loss.

Although the pathogenesis of bruxism remains unclear, multiple factors, such as physical or psychological stress, malocclusion, sleep disorders and medication side effects, can cause bruxism. Treatment can be difficult given psychogenic and neurogenic components, and bruxism can be resistant to medical and behavioral therapy. There are various treatment options for bruxism, including oral splints; medications, such as muscle relaxants; antidepressants; and botulinum toxin. Multiple studies have shown that botulinum toxin injections into the masseter and temporalis muscles result in relaxation of the muscles and improvement of bruxism and the pain associated with chronic clenching and grinding.

McLean Dermatology and Skincare Center

In a recent study by Al-Wayli, 50 subjects who reported nocturnal bruxism were randomized to receive botulinum toxin versus conventional treatment (pharmacotherapy or oral splints). After 3 weeks, 2 months, 6 months, and 1 year, patients who received botulinum toxin had significantly less pain after only one treatment than did the traditional treatment group. Similarly, in a study by Lee et al., subjects randomized to receive botulinum toxin versus a placebo saline injections showed not only decreased pain but also decreased bruxism seen with nocturnal electromyography.

In our clinic, botulinum toxin is a safe, effective treatment for patients who grind, clench, develop temporal-mandibular joint pain, or have masseter hypertrophy. Botulinum toxin when injected into the temporalis and masseter muscles also helps with tension headaches and migraines related to clenching of the jaw. Albeit effective, the dose of botulinum toxin used in the aforementioned studies ranged between 25 U and 40 U of botulinum toxin and were lower than what we have found to be effective. Our patients receive 50 U botulinum toxin in each masseter muscle (100 U total). In a small minority of our patients, the temporalis muscle also needed 15-20 U per side as well. Clinical improvement starts within 3-5 days, and patients can expect to have relaxation of the muscle and decreased pain for 6 months. Side effects include mild swelling and bruising. Rarely, if the injection is not performed properly, the risorius muscle may be paralyzed, leading to an asymmetric smile. In addition, if the botulinum toxin is underdosed, the pain may not completely subside and the patient may report some symptoms returning within a couple of weeks of the initial treatment. Most patients also report thinning of the face and jaw, which is a much anticipated and appreciated result. Masseter hypertrophy with and without bruxism is treated similarly with botulinum toxin to sculpt the lower face.

Bruxism is a growing problem leading to facial pain, headaches, migraines, and significant dental pathology. Traditional treatments have been ineffective at treating the pain and masseter hypertrophy associated with chronic grinding and clenching. Botulinum toxin is a safe, effective, treatment with little downtime or side effects for treating both the neurogenic and muscular components of bruxism.


Dr. Lily Talakoub and Dr. Naissan Wesley and are co-contributors to this column. Dr. Talakoub is in private practice in McLean, Va. This month’s column is by Dr. Talakoub. Dr. Wesley practices dermatology in Beverly Hills, Calif. Write to them at [email protected]. They had no relevant disclosures.
 

References

Al-Wayli H. J Clin Exp Dent. 2017 Jan 1;9(1):e112-e117.

Asutay F et al. Pain Res Manag. 2017;2017:6264146. doi: 10.1155/2017/6264146.

Lee SJ et al. Am J Phys Med Rehabil. 2010 Jan;89(1):16-23.

Santamato A et al. J Chiropr Med. 2010 Sep;9(3):132-7.

Shetty S et al. J Indian Prosthodont Soc. 2010 Sep;10(3):141-8.

Tan EK et al. J Am Dent Assoc. 2000 Feb;131(2):211-6.

Bruxism is grinding and clenching of the teeth with unconscious contractions of the temporal and masseter muscles while awake or during sleep. Bruxism occurs in 8%-16% of the population and is often an underdiagnosed condition that not only leads to dental problems but also to pain in the teeth, jaw, temporomandibular joint, and neck; headaches; and potentially, to tooth loss.

Although the pathogenesis of bruxism remains unclear, multiple factors, such as physical or psychological stress, malocclusion, sleep disorders and medication side effects, can cause bruxism. Treatment can be difficult given psychogenic and neurogenic components, and bruxism can be resistant to medical and behavioral therapy. There are various treatment options for bruxism, including oral splints; medications, such as muscle relaxants; antidepressants; and botulinum toxin. Multiple studies have shown that botulinum toxin injections into the masseter and temporalis muscles result in relaxation of the muscles and improvement of bruxism and the pain associated with chronic clenching and grinding.

McLean Dermatology and Skincare Center

In a recent study by Al-Wayli, 50 subjects who reported nocturnal bruxism were randomized to receive botulinum toxin versus conventional treatment (pharmacotherapy or oral splints). After 3 weeks, 2 months, 6 months, and 1 year, patients who received botulinum toxin had significantly less pain after only one treatment than did the traditional treatment group. Similarly, in a study by Lee et al., subjects randomized to receive botulinum toxin versus a placebo saline injections showed not only decreased pain but also decreased bruxism seen with nocturnal electromyography.

In our clinic, botulinum toxin is a safe, effective treatment for patients who grind, clench, develop temporal-mandibular joint pain, or have masseter hypertrophy. Botulinum toxin when injected into the temporalis and masseter muscles also helps with tension headaches and migraines related to clenching of the jaw. Albeit effective, the dose of botulinum toxin used in the aforementioned studies ranged between 25 U and 40 U of botulinum toxin and were lower than what we have found to be effective. Our patients receive 50 U botulinum toxin in each masseter muscle (100 U total). In a small minority of our patients, the temporalis muscle also needed 15-20 U per side as well. Clinical improvement starts within 3-5 days, and patients can expect to have relaxation of the muscle and decreased pain for 6 months. Side effects include mild swelling and bruising. Rarely, if the injection is not performed properly, the risorius muscle may be paralyzed, leading to an asymmetric smile. In addition, if the botulinum toxin is underdosed, the pain may not completely subside and the patient may report some symptoms returning within a couple of weeks of the initial treatment. Most patients also report thinning of the face and jaw, which is a much anticipated and appreciated result. Masseter hypertrophy with and without bruxism is treated similarly with botulinum toxin to sculpt the lower face.

Bruxism is a growing problem leading to facial pain, headaches, migraines, and significant dental pathology. Traditional treatments have been ineffective at treating the pain and masseter hypertrophy associated with chronic grinding and clenching. Botulinum toxin is a safe, effective, treatment with little downtime or side effects for treating both the neurogenic and muscular components of bruxism.


Dr. Lily Talakoub and Dr. Naissan Wesley and are co-contributors to this column. Dr. Talakoub is in private practice in McLean, Va. This month’s column is by Dr. Talakoub. Dr. Wesley practices dermatology in Beverly Hills, Calif. Write to them at [email protected]. They had no relevant disclosures.
 

References

Al-Wayli H. J Clin Exp Dent. 2017 Jan 1;9(1):e112-e117.

Asutay F et al. Pain Res Manag. 2017;2017:6264146. doi: 10.1155/2017/6264146.

Lee SJ et al. Am J Phys Med Rehabil. 2010 Jan;89(1):16-23.

Santamato A et al. J Chiropr Med. 2010 Sep;9(3):132-7.

Shetty S et al. J Indian Prosthodont Soc. 2010 Sep;10(3):141-8.

Tan EK et al. J Am Dent Assoc. 2000 Feb;131(2):211-6.

The treatment choice is oral terbinafine for tinea capitis with kerion, a scalp dermatophyte infection with a concurrent inflammatory process. Tinea capitis is a very common infection with the peak occurrence at age 3-7 years. Tinea capitis is caused by a variety of dermatophyte species, most commonly by Trichophyton tonsurans or Microsporum canis. T. tonsurans is an endothrix infection, invading the hair shaft and superficial hair while M. canis is an ectothrix infection. T. tonsurans has a person to person transmission; in contrast, M. canis is a zoonotic infection most commonly acquired from infected pets.¹ The epidemiology of tinea capitis is affected by multiple factors, including immigration patterns. For example, in Montreal, a study showed a sixfold increase of African dermatophyte species, M. audouinii and T. soudanense.² Similarly, global variation has increased prevalence of T. violaceum, more commonly found in Europe and Africa, which has been seen in immigrant populations in the United States.¹ Kerion is thought to be a hypersensitivity reaction to dermatophytes. Often, misdiagnosis of kerion can result in unnecessary antibiotic prescription and delays in initiation of antifungal therapy.^3,4

Tinea capitis can present with focal, “patchy,” well-demarcated hair loss and overlying scale, broken-off hairs at the scalp, and often with pustules. It may be associated with occipital or posterior cervical lymphadenopathy. Tinea can be complicated by kerion development, which presents as a painful boggy scalp mass with or without purulent drainage. Kerions can have associated fever. It is important to differentiate tinea capitis with kerion from processes requiring a different treatment course.^3,5 Bacterial folliculitis may have erythema and pustules but only rarely causes hair loss. Similarly, alopecia areata can present with focal alopecia but lacks the scalp inflammation and pustules. Scalp psoriasis is quite scaly, but is more thickened, and usually does not have pustules, nor would purulent drainage be evident. There is a broader differential for other inflammatory focal alopecias including discoid lupus, lichen planopilaris, and dissecting cellulitis of the scalp, which can have similar pus-filled lumps, and cause permanent hair loss, but may be differentiated by associated conditions such as acne conglobata, hidradenitis suppurativa, and pilonidal sinus.

While some clinicians advocate clinical diagnosis of tinea capitis, we advocate confirmation of infection by fungal culture, which can identify the causative organism and influence therapy selection.¹ The presence of a fungal infection can be confirmed on potassium hydroxide wet mount prep if hyphae and small spores are seen.^6,7 Wood lamp examination will fluoresce if there are ectothrix species, however a negative fluorescence does not differentiate between an endothrix species or lack of infection.

It is important that tinea capitis with kerion is treated with systemic antifungal treatments to allow resolution of the infection, recovery of hair growth, and to prevent or minimize scarring. Systemic antifungal options include terbinafine, griseofulvin, and azoles. Terbinafine is becoming the treatment of choice given shorter duration of treatment with similar efficacy.¹T. tonsurans also is thought to respond better to terbinafine than to griseofulvin.⁸ Griseofulvin is the historical treatment of choice because of its history of clinical safety and no need for laboratory testing. It is important to note that higher doses of griseofulvin (20-25 mg/kg) are recommended, as older lower dose regimens have high rates of failure. Griseofulvin may be more effective for treatment of Microsporum spp than is terbinafine.⁸ Fluconazole is the only oral antifungal agent that is approved for patients younger than 2 years of age, however it has lower cure rates, compared with terbinafine and griseofulvin.¹ Tinea capitis with kerion does not generally require antibiotics unless there is superimposed bacterial infection. Kerion also do not require incision or drainage, which may increase scarring and complicate the clinical course.

Management of tinea capitis should include evaluation of any other household members for coinfection. Depending on the dermatophyte involved there can be risk of person-to-person transmission.⁵ Families should be educated about fomite transmission via shared combs or hats. Additionally, if a zoophilic dermatophyte is suspected, pets also should be appropriately examined and treated. Topical antifungals are insufficient to eradicate tinea capitis but can be used as adjunctive therapy. Kerion can be treated with oral prednisone in addition to oral antifungals if the lesions are very painful, however there is limited data on this treatment option.⁹

Ms. Kaushik is a research fellow and Dr. Eichenfield is professor of dermatology and pediatrics in the division of pediatric and adolescent dermatology at Rady Children’s Hospital and the University of California, both in San Diego. They have no conflicts of interest or relevant financial disclosures.

References

1. “Red Book: Report of the Committee on Infectious Diseases,” 31st Edition, (Elk Grove Village, Ill.: American Academy of Pediatrics, 2018, p. 1264).

2. Pediatr Dermatol. 2018 May;35(3):323-8

3. Arch Dis Child. 2016 May;101(5):503.

4. IDCases. 2018 Jun 28;14:e00418.

5. Int J Dermatol. 2018 Jan;57(1):3-9.

6. Pediatr Rev. 2007 May;28(5):164-74.

7. Clin Cosmet Investig Dermatol. 2010;3:89-98.

8. Cochrane Database Syst Rev. 2016 May 12;(5):CD004685.

9. Med Mycol. 1999 Apr;37(2):97-9.

The treatment choice is oral terbinafine for tinea capitis with kerion, a scalp dermatophyte infection with a concurrent inflammatory process. Tinea capitis is a very common infection with the peak occurrence at age 3-7 years. Tinea capitis is caused by a variety of dermatophyte species, most commonly by Trichophyton tonsurans or Microsporum canis. T. tonsurans is an endothrix infection, invading the hair shaft and superficial hair while M. canis is an ectothrix infection. T. tonsurans has a person to person transmission; in contrast, M. canis is a zoonotic infection most commonly acquired from infected pets.¹ The epidemiology of tinea capitis is affected by multiple factors, including immigration patterns. For example, in Montreal, a study showed a sixfold increase of African dermatophyte species, M. audouinii and T. soudanense.² Similarly, global variation has increased prevalence of T. violaceum, more commonly found in Europe and Africa, which has been seen in immigrant populations in the United States.¹ Kerion is thought to be a hypersensitivity reaction to dermatophytes. Often, misdiagnosis of kerion can result in unnecessary antibiotic prescription and delays in initiation of antifungal therapy.^3,4

Tinea capitis can present with focal, “patchy,” well-demarcated hair loss and overlying scale, broken-off hairs at the scalp, and often with pustules. It may be associated with occipital or posterior cervical lymphadenopathy. Tinea can be complicated by kerion development, which presents as a painful boggy scalp mass with or without purulent drainage. Kerions can have associated fever. It is important to differentiate tinea capitis with kerion from processes requiring a different treatment course.^3,5 Bacterial folliculitis may have erythema and pustules but only rarely causes hair loss. Similarly, alopecia areata can present with focal alopecia but lacks the scalp inflammation and pustules. Scalp psoriasis is quite scaly, but is more thickened, and usually does not have pustules, nor would purulent drainage be evident. There is a broader differential for other inflammatory focal alopecias including discoid lupus, lichen planopilaris, and dissecting cellulitis of the scalp, which can have similar pus-filled lumps, and cause permanent hair loss, but may be differentiated by associated conditions such as acne conglobata, hidradenitis suppurativa, and pilonidal sinus.

While some clinicians advocate clinical diagnosis of tinea capitis, we advocate confirmation of infection by fungal culture, which can identify the causative organism and influence therapy selection.¹ The presence of a fungal infection can be confirmed on potassium hydroxide wet mount prep if hyphae and small spores are seen.^6,7 Wood lamp examination will fluoresce if there are ectothrix species, however a negative fluorescence does not differentiate between an endothrix species or lack of infection.

It is important that tinea capitis with kerion is treated with systemic antifungal treatments to allow resolution of the infection, recovery of hair growth, and to prevent or minimize scarring. Systemic antifungal options include terbinafine, griseofulvin, and azoles. Terbinafine is becoming the treatment of choice given shorter duration of treatment with similar efficacy.¹T. tonsurans also is thought to respond better to terbinafine than to griseofulvin.⁸ Griseofulvin is the historical treatment of choice because of its history of clinical safety and no need for laboratory testing. It is important to note that higher doses of griseofulvin (20-25 mg/kg) are recommended, as older lower dose regimens have high rates of failure. Griseofulvin may be more effective for treatment of Microsporum spp than is terbinafine.⁸ Fluconazole is the only oral antifungal agent that is approved for patients younger than 2 years of age, however it has lower cure rates, compared with terbinafine and griseofulvin.¹ Tinea capitis with kerion does not generally require antibiotics unless there is superimposed bacterial infection. Kerion also do not require incision or drainage, which may increase scarring and complicate the clinical course.

Management of tinea capitis should include evaluation of any other household members for coinfection. Depending on the dermatophyte involved there can be risk of person-to-person transmission.⁵ Families should be educated about fomite transmission via shared combs or hats. Additionally, if a zoophilic dermatophyte is suspected, pets also should be appropriately examined and treated. Topical antifungals are insufficient to eradicate tinea capitis but can be used as adjunctive therapy. Kerion can be treated with oral prednisone in addition to oral antifungals if the lesions are very painful, however there is limited data on this treatment option.⁹

Ms. Kaushik is a research fellow and Dr. Eichenfield is professor of dermatology and pediatrics in the division of pediatric and adolescent dermatology at Rady Children’s Hospital and the University of California, both in San Diego. They have no conflicts of interest or relevant financial disclosures.

References

1. “Red Book: Report of the Committee on Infectious Diseases,” 31st Edition, (Elk Grove Village, Ill.: American Academy of Pediatrics, 2018, p. 1264).

2. Pediatr Dermatol. 2018 May;35(3):323-8

3. Arch Dis Child. 2016 May;101(5):503.

4. IDCases. 2018 Jun 28;14:e00418.

5. Int J Dermatol. 2018 Jan;57(1):3-9.

6. Pediatr Rev. 2007 May;28(5):164-74.

7. Clin Cosmet Investig Dermatol. 2010;3:89-98.

8. Cochrane Database Syst Rev. 2016 May 12;(5):CD004685.

9. Med Mycol. 1999 Apr;37(2):97-9.

The treatment choice is oral terbinafine for tinea capitis with kerion, a scalp dermatophyte infection with a concurrent inflammatory process. Tinea capitis is a very common infection with the peak occurrence at age 3-7 years. Tinea capitis is caused by a variety of dermatophyte species, most commonly by Trichophyton tonsurans or Microsporum canis. T. tonsurans is an endothrix infection, invading the hair shaft and superficial hair while M. canis is an ectothrix infection. T. tonsurans has a person to person transmission; in contrast, M. canis is a zoonotic infection most commonly acquired from infected pets.¹ The epidemiology of tinea capitis is affected by multiple factors, including immigration patterns. For example, in Montreal, a study showed a sixfold increase of African dermatophyte species, M. audouinii and T. soudanense.² Similarly, global variation has increased prevalence of T. violaceum, more commonly found in Europe and Africa, which has been seen in immigrant populations in the United States.¹ Kerion is thought to be a hypersensitivity reaction to dermatophytes. Often, misdiagnosis of kerion can result in unnecessary antibiotic prescription and delays in initiation of antifungal therapy.^3,4

Tinea capitis can present with focal, “patchy,” well-demarcated hair loss and overlying scale, broken-off hairs at the scalp, and often with pustules. It may be associated with occipital or posterior cervical lymphadenopathy. Tinea can be complicated by kerion development, which presents as a painful boggy scalp mass with or without purulent drainage. Kerions can have associated fever. It is important to differentiate tinea capitis with kerion from processes requiring a different treatment course.^3,5 Bacterial folliculitis may have erythema and pustules but only rarely causes hair loss. Similarly, alopecia areata can present with focal alopecia but lacks the scalp inflammation and pustules. Scalp psoriasis is quite scaly, but is more thickened, and usually does not have pustules, nor would purulent drainage be evident. There is a broader differential for other inflammatory focal alopecias including discoid lupus, lichen planopilaris, and dissecting cellulitis of the scalp, which can have similar pus-filled lumps, and cause permanent hair loss, but may be differentiated by associated conditions such as acne conglobata, hidradenitis suppurativa, and pilonidal sinus.

While some clinicians advocate clinical diagnosis of tinea capitis, we advocate confirmation of infection by fungal culture, which can identify the causative organism and influence therapy selection.¹ The presence of a fungal infection can be confirmed on potassium hydroxide wet mount prep if hyphae and small spores are seen.^6,7 Wood lamp examination will fluoresce if there are ectothrix species, however a negative fluorescence does not differentiate between an endothrix species or lack of infection.

It is important that tinea capitis with kerion is treated with systemic antifungal treatments to allow resolution of the infection, recovery of hair growth, and to prevent or minimize scarring. Systemic antifungal options include terbinafine, griseofulvin, and azoles. Terbinafine is becoming the treatment of choice given shorter duration of treatment with similar efficacy.¹T. tonsurans also is thought to respond better to terbinafine than to griseofulvin.⁸ Griseofulvin is the historical treatment of choice because of its history of clinical safety and no need for laboratory testing. It is important to note that higher doses of griseofulvin (20-25 mg/kg) are recommended, as older lower dose regimens have high rates of failure. Griseofulvin may be more effective for treatment of Microsporum spp than is terbinafine.⁸ Fluconazole is the only oral antifungal agent that is approved for patients younger than 2 years of age, however it has lower cure rates, compared with terbinafine and griseofulvin.¹ Tinea capitis with kerion does not generally require antibiotics unless there is superimposed bacterial infection. Kerion also do not require incision or drainage, which may increase scarring and complicate the clinical course.

Management of tinea capitis should include evaluation of any other household members for coinfection. Depending on the dermatophyte involved there can be risk of person-to-person transmission.⁵ Families should be educated about fomite transmission via shared combs or hats. Additionally, if a zoophilic dermatophyte is suspected, pets also should be appropriately examined and treated. Topical antifungals are insufficient to eradicate tinea capitis but can be used as adjunctive therapy. Kerion can be treated with oral prednisone in addition to oral antifungals if the lesions are very painful, however there is limited data on this treatment option.⁹

Ms. Kaushik is a research fellow and Dr. Eichenfield is professor of dermatology and pediatrics in the division of pediatric and adolescent dermatology at Rady Children’s Hospital and the University of California, both in San Diego. They have no conflicts of interest or relevant financial disclosures.

References

1. “Red Book: Report of the Committee on Infectious Diseases,” 31st Edition, (Elk Grove Village, Ill.: American Academy of Pediatrics, 2018, p. 1264).

2. Pediatr Dermatol. 2018 May;35(3):323-8

3. Arch Dis Child. 2016 May;101(5):503.

4. IDCases. 2018 Jun 28;14:e00418.

5. Int J Dermatol. 2018 Jan;57(1):3-9.

6. Pediatr Rev. 2007 May;28(5):164-74.

7. Clin Cosmet Investig Dermatol. 2010;3:89-98.

8. Cochrane Database Syst Rev. 2016 May 12;(5):CD004685.

9. Med Mycol. 1999 Apr;37(2):97-9.