The Cutis Editorial Board is now accepting applications for the 2025 Resident Corner column. The Editorial Board will select 2 to 3 residents to serve as the Resident Corner columnists for 1 year. Articles are posted online only at www.mdedge.com/dermatology but will be referenced in Index Medicus. All applicants must be current residents and will be in residency throughout 2025.

For consideration, send your curriculum vitae along with a brief (not to exceed 500 words) statement of why you enjoy Cutis and what you can offer your fellow residents in contributing a monthly column.

A signed letter of recommendation from the Director of the dermatology residency program also should be supplied.

All materials should be submitted via email to Alicia Sonners ([email protected]) by November 1. The residents who are selected to write the column for the upcoming year will be notified by November 15.

We look forward to continuing to educate dermatology residents on topics that are most important to them!

The Cutis Editorial Board is now accepting applications for the 2025 Resident Corner column. The Editorial Board will select 2 to 3 residents to serve as the Resident Corner columnists for 1 year. Articles are posted online only at www.mdedge.com/dermatology but will be referenced in Index Medicus. All applicants must be current residents and will be in residency throughout 2025.

For consideration, send your curriculum vitae along with a brief (not to exceed 500 words) statement of why you enjoy Cutis and what you can offer your fellow residents in contributing a monthly column.

A signed letter of recommendation from the Director of the dermatology residency program also should be supplied.

All materials should be submitted via email to Alicia Sonners ([email protected]) by November 1. The residents who are selected to write the column for the upcoming year will be notified by November 15.

We look forward to continuing to educate dermatology residents on topics that are most important to them!

The Cutis Editorial Board is now accepting applications for the 2025 Resident Corner column. The Editorial Board will select 2 to 3 residents to serve as the Resident Corner columnists for 1 year. Articles are posted online only at www.mdedge.com/dermatology but will be referenced in Index Medicus. All applicants must be current residents and will be in residency throughout 2025.

For consideration, send your curriculum vitae along with a brief (not to exceed 500 words) statement of why you enjoy Cutis and what you can offer your fellow residents in contributing a monthly column.

A signed letter of recommendation from the Director of the dermatology residency program also should be supplied.

All materials should be submitted via email to Alicia Sonners ([email protected]) by November 1. The residents who are selected to write the column for the upcoming year will be notified by November 15.

We look forward to continuing to educate dermatology residents on topics that are most important to them!

As more data show potentially dangerous effects of climate and weather on individuals with chronic medical conditions, CVS Health has introduced an initiative that uses technology to provide weather alerts and targeted outreach to those at increased risk, according to a press release from the company. Ultimately, the goals of the initiative are to improve health, reduce emergency department visits, hospital stays, and medical costs, according to the press release.

Extreme weather events such as heat waves are becoming more frequent and severe, but most heat-related deaths are preventable with outreach and intervention, Dan Knecht, MD, vice president and chief clinical innovation officer for CVS Caremark, a division of CVS Health, said in an interview. The approach will combine the company’s services, including care managers, health centers, and data, to aid patients vulnerable to severe weather.

The initiative is starting with a focus on extreme heat events and will expand this fall with alerts about high levels of air pollution for individuals with vulnerability to reduced lung function, asthma, and cardiac problems as a result of exposure to high air-pollution levels, according to Dr. Knecht.

For now, the initiative is available to members of Aetna Medicare, according to Dr. Knecht. “Our goal is to expand to other consumers, including those who visit MinuteClinic and CVS Pharmacy locations, where we can provide timely environment-related recommendations at time of care,” he said.

The alert system uses environmental data analytics to pair highly localized forecasts and real-time insights about air quality, wildfires, and high heat with medical and pharmacy data for high-risk patients in areas affected by extreme weather.

For example, for individuals who are at risk and living in areas facing extreme heat, “registered nurse care managers proactively reach out to vulnerable patients up to several days in advance of an extreme weather event and provide them personalized tips and resources,” said Dr. Knecht.

In addition, he added, “we talk to patients about how to manage their medications during periods of extreme heat and, when delivering medications, take weather data into account to determine appropriate packaging materials for shipments.”

These interventions direct patients to CVS Health–linked resources, such as Oak Street Health clinics available as cooling centers, health services provided at MinuteClinic locations, and medication management at CVS pharmacies. Other interventions include virtual or in-person mental health counseling through MinuteClinic.

Dr. Knecht offered additional guidance for clinicians and patients to help manage heat waves. “Heat and certain medications can impair heat tolerance and the ability to regulate body temperature,” he told this news organization. Extreme heat may affect the performance of some medications and their devices, such as inhalers and diabetes supplies, he added.
 

Health Alerts Have Potential, But Comprehensive Approach is Needed

“Patients with chronic lung conditions are highly susceptible to the impact of climate change,” MeiLan K. Han, MD, a pulmonologist and professor of internal medicine at the University of Michigan, Ann Arbor, said in an interview. “Increasing dust, hotter temperatures, and higher levels of air pollution make it more difficult for patients to breathe,” she said. Data also suggest that higher levels of air pollution may not only cause chronic lung disease but also cause worsening symptoms among those with existing disease, she added.

A weather-related health alert could be useful for patients so they can be prepared, Dr. Han told this news organization.

“For a patient with chronic lung disease, a hot weather alert may mean that it will be harder for patients to breathe, and [they] may [be] more susceptible to heat stroke and dehydration if they do not have access to air conditioning,” she said. “At a minimum, patients should ensure they are on their controller medications, which often means a daily inhaler for patients with conditions such as asthma and chronic obstructive pulmonary disease (COPD). However, patients also should have access to their short-term reliever medications so they can be prepared for increased shortness of breath that may accompany a hot weather day,” Dr. Han explained.

However, not all patients have access to technology such as smartphones or other devices that will alert them to impending weather events, such as heat waves, said Dr. Han. “For these patients, a standard phone call may be beneficial,” she said.

Looking ahead, “programs for weather-related health alerts will need to be comprehensive, focusing not only on access to needed medications but also climate-controlled settings for temporary relief of heat,” said Dr. Han. “For some of our most vulnerable patients, while they may have air conditioning, they may not be able to afford to run it, so this needs to be considered in developing a comprehensive program,” she emphasized.

Dr. Knecht had no financial conflicts to disclose. Dr. Han disclosed ties with Aerogen, Altesa BioSciences, American Lung Association, Amgen, Apreo Health, AstraZeneca, Biodesix, Boehringer Ingelheim, Chiesi, Cipla, COPD Foundation, DevPro, Gala Therapeutics, Genentech, GlaxoSmithKline, Integrity, MDBriefcase, Medscape, Medtronic, Medwiz, Meissa Vaccines, Merck, Mylan, NACE, National Institutes of Health, Novartis, Nuvaira, Polarian, Pulmonx, Regeneron, Roche, RS Biotherapeutics, Sanofi, Sunovion, Teva, UpToDate, and Verona..
 

A version of this article first appeared on Medscape.com.

As more data show potentially dangerous effects of climate and weather on individuals with chronic medical conditions, CVS Health has introduced an initiative that uses technology to provide weather alerts and targeted outreach to those at increased risk, according to a press release from the company. Ultimately, the goals of the initiative are to improve health, reduce emergency department visits, hospital stays, and medical costs, according to the press release.

Extreme weather events such as heat waves are becoming more frequent and severe, but most heat-related deaths are preventable with outreach and intervention, Dan Knecht, MD, vice president and chief clinical innovation officer for CVS Caremark, a division of CVS Health, said in an interview. The approach will combine the company’s services, including care managers, health centers, and data, to aid patients vulnerable to severe weather.

The initiative is starting with a focus on extreme heat events and will expand this fall with alerts about high levels of air pollution for individuals with vulnerability to reduced lung function, asthma, and cardiac problems as a result of exposure to high air-pollution levels, according to Dr. Knecht.

For now, the initiative is available to members of Aetna Medicare, according to Dr. Knecht. “Our goal is to expand to other consumers, including those who visit MinuteClinic and CVS Pharmacy locations, where we can provide timely environment-related recommendations at time of care,” he said.

The alert system uses environmental data analytics to pair highly localized forecasts and real-time insights about air quality, wildfires, and high heat with medical and pharmacy data for high-risk patients in areas affected by extreme weather.

For example, for individuals who are at risk and living in areas facing extreme heat, “registered nurse care managers proactively reach out to vulnerable patients up to several days in advance of an extreme weather event and provide them personalized tips and resources,” said Dr. Knecht.

In addition, he added, “we talk to patients about how to manage their medications during periods of extreme heat and, when delivering medications, take weather data into account to determine appropriate packaging materials for shipments.”

These interventions direct patients to CVS Health–linked resources, such as Oak Street Health clinics available as cooling centers, health services provided at MinuteClinic locations, and medication management at CVS pharmacies. Other interventions include virtual or in-person mental health counseling through MinuteClinic.

Dr. Knecht offered additional guidance for clinicians and patients to help manage heat waves. “Heat and certain medications can impair heat tolerance and the ability to regulate body temperature,” he told this news organization. Extreme heat may affect the performance of some medications and their devices, such as inhalers and diabetes supplies, he added.
 

Health Alerts Have Potential, But Comprehensive Approach is Needed

“Patients with chronic lung conditions are highly susceptible to the impact of climate change,” MeiLan K. Han, MD, a pulmonologist and professor of internal medicine at the University of Michigan, Ann Arbor, said in an interview. “Increasing dust, hotter temperatures, and higher levels of air pollution make it more difficult for patients to breathe,” she said. Data also suggest that higher levels of air pollution may not only cause chronic lung disease but also cause worsening symptoms among those with existing disease, she added.

A weather-related health alert could be useful for patients so they can be prepared, Dr. Han told this news organization.

“For a patient with chronic lung disease, a hot weather alert may mean that it will be harder for patients to breathe, and [they] may [be] more susceptible to heat stroke and dehydration if they do not have access to air conditioning,” she said. “At a minimum, patients should ensure they are on their controller medications, which often means a daily inhaler for patients with conditions such as asthma and chronic obstructive pulmonary disease (COPD). However, patients also should have access to their short-term reliever medications so they can be prepared for increased shortness of breath that may accompany a hot weather day,” Dr. Han explained.

However, not all patients have access to technology such as smartphones or other devices that will alert them to impending weather events, such as heat waves, said Dr. Han. “For these patients, a standard phone call may be beneficial,” she said.

Looking ahead, “programs for weather-related health alerts will need to be comprehensive, focusing not only on access to needed medications but also climate-controlled settings for temporary relief of heat,” said Dr. Han. “For some of our most vulnerable patients, while they may have air conditioning, they may not be able to afford to run it, so this needs to be considered in developing a comprehensive program,” she emphasized.

Dr. Knecht had no financial conflicts to disclose. Dr. Han disclosed ties with Aerogen, Altesa BioSciences, American Lung Association, Amgen, Apreo Health, AstraZeneca, Biodesix, Boehringer Ingelheim, Chiesi, Cipla, COPD Foundation, DevPro, Gala Therapeutics, Genentech, GlaxoSmithKline, Integrity, MDBriefcase, Medscape, Medtronic, Medwiz, Meissa Vaccines, Merck, Mylan, NACE, National Institutes of Health, Novartis, Nuvaira, Polarian, Pulmonx, Regeneron, Roche, RS Biotherapeutics, Sanofi, Sunovion, Teva, UpToDate, and Verona..
 

A version of this article first appeared on Medscape.com.

As more data show potentially dangerous effects of climate and weather on individuals with chronic medical conditions, CVS Health has introduced an initiative that uses technology to provide weather alerts and targeted outreach to those at increased risk, according to a press release from the company. Ultimately, the goals of the initiative are to improve health, reduce emergency department visits, hospital stays, and medical costs, according to the press release.

Extreme weather events such as heat waves are becoming more frequent and severe, but most heat-related deaths are preventable with outreach and intervention, Dan Knecht, MD, vice president and chief clinical innovation officer for CVS Caremark, a division of CVS Health, said in an interview. The approach will combine the company’s services, including care managers, health centers, and data, to aid patients vulnerable to severe weather.

The initiative is starting with a focus on extreme heat events and will expand this fall with alerts about high levels of air pollution for individuals with vulnerability to reduced lung function, asthma, and cardiac problems as a result of exposure to high air-pollution levels, according to Dr. Knecht.

For now, the initiative is available to members of Aetna Medicare, according to Dr. Knecht. “Our goal is to expand to other consumers, including those who visit MinuteClinic and CVS Pharmacy locations, where we can provide timely environment-related recommendations at time of care,” he said.

The alert system uses environmental data analytics to pair highly localized forecasts and real-time insights about air quality, wildfires, and high heat with medical and pharmacy data for high-risk patients in areas affected by extreme weather.

For example, for individuals who are at risk and living in areas facing extreme heat, “registered nurse care managers proactively reach out to vulnerable patients up to several days in advance of an extreme weather event and provide them personalized tips and resources,” said Dr. Knecht.

In addition, he added, “we talk to patients about how to manage their medications during periods of extreme heat and, when delivering medications, take weather data into account to determine appropriate packaging materials for shipments.”

These interventions direct patients to CVS Health–linked resources, such as Oak Street Health clinics available as cooling centers, health services provided at MinuteClinic locations, and medication management at CVS pharmacies. Other interventions include virtual or in-person mental health counseling through MinuteClinic.

Dr. Knecht offered additional guidance for clinicians and patients to help manage heat waves. “Heat and certain medications can impair heat tolerance and the ability to regulate body temperature,” he told this news organization. Extreme heat may affect the performance of some medications and their devices, such as inhalers and diabetes supplies, he added.
 

Health Alerts Have Potential, But Comprehensive Approach is Needed

“Patients with chronic lung conditions are highly susceptible to the impact of climate change,” MeiLan K. Han, MD, a pulmonologist and professor of internal medicine at the University of Michigan, Ann Arbor, said in an interview. “Increasing dust, hotter temperatures, and higher levels of air pollution make it more difficult for patients to breathe,” she said. Data also suggest that higher levels of air pollution may not only cause chronic lung disease but also cause worsening symptoms among those with existing disease, she added.

A weather-related health alert could be useful for patients so they can be prepared, Dr. Han told this news organization.

“For a patient with chronic lung disease, a hot weather alert may mean that it will be harder for patients to breathe, and [they] may [be] more susceptible to heat stroke and dehydration if they do not have access to air conditioning,” she said. “At a minimum, patients should ensure they are on their controller medications, which often means a daily inhaler for patients with conditions such as asthma and chronic obstructive pulmonary disease (COPD). However, patients also should have access to their short-term reliever medications so they can be prepared for increased shortness of breath that may accompany a hot weather day,” Dr. Han explained.

However, not all patients have access to technology such as smartphones or other devices that will alert them to impending weather events, such as heat waves, said Dr. Han. “For these patients, a standard phone call may be beneficial,” she said.

Looking ahead, “programs for weather-related health alerts will need to be comprehensive, focusing not only on access to needed medications but also climate-controlled settings for temporary relief of heat,” said Dr. Han. “For some of our most vulnerable patients, while they may have air conditioning, they may not be able to afford to run it, so this needs to be considered in developing a comprehensive program,” she emphasized.

Dr. Knecht had no financial conflicts to disclose. Dr. Han disclosed ties with Aerogen, Altesa BioSciences, American Lung Association, Amgen, Apreo Health, AstraZeneca, Biodesix, Boehringer Ingelheim, Chiesi, Cipla, COPD Foundation, DevPro, Gala Therapeutics, Genentech, GlaxoSmithKline, Integrity, MDBriefcase, Medscape, Medtronic, Medwiz, Meissa Vaccines, Merck, Mylan, NACE, National Institutes of Health, Novartis, Nuvaira, Polarian, Pulmonx, Regeneron, Roche, RS Biotherapeutics, Sanofi, Sunovion, Teva, UpToDate, and Verona..
 

A version of this article first appeared on Medscape.com.

Having an ostomy is a dreaded prospect for many patients with rectal cancer.

To defer, and potentially avoid, this life-altering surgery, the watch-and-wait approach has become increasingly common among patients with locally advanced disease who have a complete response to neoadjuvant chemoradiation.

About 80% of these patients who have a complete clinical response — a perfectly healed scar where the tumor used to be and other favorable features — can forgo total mesorectal excision and preserve their rectum.

The success of watch-and-wait among complete responders has led some centers to offer the approach in patients with near-complete responses to neoadjuvant chemoradiation.

But watch-and-wait for near-complete clinical responders “is very controversial,” Alan P. Venook, MD, a gastrointestinal oncologist at the University of California, San Francisco (UCSF), told this news organization.

“You sure as hell don’t want to miss a chance to cure a patient,” Dr. Venook said.

A near-complete clinical response essentially means there is no sign of the tumor 8 weeks after total neoadjuvant therapy, but the tumor bed hasn’t completely healed.

The goal of watch-and-wait in this scenario is to give near-complete response lesions time to become complete responses.

But there’s no clear way to predict which tumors will evolve into a clinical complete response.

Recent studies evaluating the conversion rate have reported that anywhere from 39% to about 90% of near-complete responders became complete responders. Some of the variation likely comes down to differences in the clinical stage of patients evaluated in each study as well as the limited number of patients who achieve a near-complete response overall.

Other concerns have emerged that waiting for near-complete responses to become complete leaves extra time for some tumors to metastasize and that tumor regrowth is much higher compared with complete responders.

A recent study found that 13% of near-complete responders who preserved their rectum on watch-and-wait developed distant metastases vs about 5% of long-term complete responders. The study also found that just over half of near-complete responders have tumor regrowth compared with about one in five complete responders.

But even with regrowth, “surgery is still curative,” explained Julio Garcia-Aguilar, MD, PhD, a pioneer of watch-and-wait for rectal cancer.

And overall, around 50%-60% of patients with a near-complete response can avoid surgery and preserve their rectum.
 

Selecting Patients for Watch-and-Wait

The key to deciding which patients are right for watch-and-wait is to understand how a near-complete clinical response was defined in the OPRA trial, a landmark randomized trial led by Dr. Garcia-Aguilar that helped establish watch-and-wait as an option in rectal cancer.

OPRA defined a near-complete response as no visible tumor but, in the tumor bed, mild erythema, superficial ulceration, minor mucosal abnormality or small nodules, and an irregular mucosa. The criteria also included no palpable tumor with smooth induration or a minor mucosal abnormality on the digital rectal exam.

The National Comprehensive Cancer Network mirrored the definition when, for the first time, it recommended watch-and-wait as an option for near-complete response in its 2023 rectal cancer guidelines. The group also added a few MRI requirements.

UCSF offers the watch-and-wait option to some patients with near-complete responses, but each decision is made on a case-by-case basis by a tumor board considering numerous measures of tumor aggressiveness.

Even then, “we have, in many cases, struggled to figure out what the right choices are,” Dr. Venook said.

For those chosen for watch-and-wait, Dr. Venook noted that UCSF has top-notch surgeons, radiation oncologists, medical oncologists, and pathologists who have the resources to follow patients closely.

For community practices without the resources of a major cancer center, watch-and-wait for near-complete response to rectal cancer “is really asking a lot,” Dr. Venook said.

Dr. Garcia-Aguilar, a colorectal surgeon at Memorial Sloan Kettering Cancer Center in New York City, explained that after years of studying the issue, he is comfortable with watch-and-wait in near-complete responders as long as it’s done carefully and in patients who will comply with ongoing surveillance.

Dr. Garcia-Aguilar explained that, after diagnosing a near-complete response 8 weeks following total neoadjuvant therapy, the patient needs to come back 6 weeks later. At that point, it’s time to assess whether that near-complete response is evolving into a complete response or not evolving into a complete response.

If it’s evolving into a complete response, surveillance continues about every 8 weeks, but if the tumor has stopped responding, “you take [the patient] to the operating room,” Dr. Garcia-Aguilar said.

As for the bigger safety concern — that near clinical complete response tumors will metastasize — Dr. Garcia-Aguilar’s opinion is that micrometastases are probably already there when the rectal cancer is first diagnosed and will manifest themselves “no matter what happens to the primary tumor.”

Because of that, he noted, “I don’t think the risk is very high” when surgery is delayed a few months to give near-complete response patients a chance to keep their rectum.

The way to answer the metastasis question is to do a randomized trial pitting surgery against watch-and-wait in patients with near-clinical complete response rectal cancer.

However, Dr. Garcia-Aguilar doesn’t think that trial will ever happen. Patients won’t allow themselves to be randomized to surgery once they find out they might be able to avoid a permanent ostomy, he said.

Dr. Venook had no disclosures. Dr. Garcia-Aguilar reported personal fees from Medtronic, Johnson & Johnson, and Intuitive Surgical.
 

A version of this article first appeared on Medscape.com.

Having an ostomy is a dreaded prospect for many patients with rectal cancer.

To defer, and potentially avoid, this life-altering surgery, the watch-and-wait approach has become increasingly common among patients with locally advanced disease who have a complete response to neoadjuvant chemoradiation.

About 80% of these patients who have a complete clinical response — a perfectly healed scar where the tumor used to be and other favorable features — can forgo total mesorectal excision and preserve their rectum.

The success of watch-and-wait among complete responders has led some centers to offer the approach in patients with near-complete responses to neoadjuvant chemoradiation.

But watch-and-wait for near-complete clinical responders “is very controversial,” Alan P. Venook, MD, a gastrointestinal oncologist at the University of California, San Francisco (UCSF), told this news organization.

“You sure as hell don’t want to miss a chance to cure a patient,” Dr. Venook said.

A near-complete clinical response essentially means there is no sign of the tumor 8 weeks after total neoadjuvant therapy, but the tumor bed hasn’t completely healed.

The goal of watch-and-wait in this scenario is to give near-complete response lesions time to become complete responses.

But there’s no clear way to predict which tumors will evolve into a clinical complete response.

Recent studies evaluating the conversion rate have reported that anywhere from 39% to about 90% of near-complete responders became complete responders. Some of the variation likely comes down to differences in the clinical stage of patients evaluated in each study as well as the limited number of patients who achieve a near-complete response overall.

Other concerns have emerged that waiting for near-complete responses to become complete leaves extra time for some tumors to metastasize and that tumor regrowth is much higher compared with complete responders.

A recent study found that 13% of near-complete responders who preserved their rectum on watch-and-wait developed distant metastases vs about 5% of long-term complete responders. The study also found that just over half of near-complete responders have tumor regrowth compared with about one in five complete responders.

But even with regrowth, “surgery is still curative,” explained Julio Garcia-Aguilar, MD, PhD, a pioneer of watch-and-wait for rectal cancer.

And overall, around 50%-60% of patients with a near-complete response can avoid surgery and preserve their rectum.
 

Selecting Patients for Watch-and-Wait

The key to deciding which patients are right for watch-and-wait is to understand how a near-complete clinical response was defined in the OPRA trial, a landmark randomized trial led by Dr. Garcia-Aguilar that helped establish watch-and-wait as an option in rectal cancer.

OPRA defined a near-complete response as no visible tumor but, in the tumor bed, mild erythema, superficial ulceration, minor mucosal abnormality or small nodules, and an irregular mucosa. The criteria also included no palpable tumor with smooth induration or a minor mucosal abnormality on the digital rectal exam.

The National Comprehensive Cancer Network mirrored the definition when, for the first time, it recommended watch-and-wait as an option for near-complete response in its 2023 rectal cancer guidelines. The group also added a few MRI requirements.

UCSF offers the watch-and-wait option to some patients with near-complete responses, but each decision is made on a case-by-case basis by a tumor board considering numerous measures of tumor aggressiveness.

Even then, “we have, in many cases, struggled to figure out what the right choices are,” Dr. Venook said.

For those chosen for watch-and-wait, Dr. Venook noted that UCSF has top-notch surgeons, radiation oncologists, medical oncologists, and pathologists who have the resources to follow patients closely.

For community practices without the resources of a major cancer center, watch-and-wait for near-complete response to rectal cancer “is really asking a lot,” Dr. Venook said.

Dr. Garcia-Aguilar, a colorectal surgeon at Memorial Sloan Kettering Cancer Center in New York City, explained that after years of studying the issue, he is comfortable with watch-and-wait in near-complete responders as long as it’s done carefully and in patients who will comply with ongoing surveillance.

Dr. Garcia-Aguilar explained that, after diagnosing a near-complete response 8 weeks following total neoadjuvant therapy, the patient needs to come back 6 weeks later. At that point, it’s time to assess whether that near-complete response is evolving into a complete response or not evolving into a complete response.

If it’s evolving into a complete response, surveillance continues about every 8 weeks, but if the tumor has stopped responding, “you take [the patient] to the operating room,” Dr. Garcia-Aguilar said.

As for the bigger safety concern — that near clinical complete response tumors will metastasize — Dr. Garcia-Aguilar’s opinion is that micrometastases are probably already there when the rectal cancer is first diagnosed and will manifest themselves “no matter what happens to the primary tumor.”

Because of that, he noted, “I don’t think the risk is very high” when surgery is delayed a few months to give near-complete response patients a chance to keep their rectum.

The way to answer the metastasis question is to do a randomized trial pitting surgery against watch-and-wait in patients with near-clinical complete response rectal cancer.

However, Dr. Garcia-Aguilar doesn’t think that trial will ever happen. Patients won’t allow themselves to be randomized to surgery once they find out they might be able to avoid a permanent ostomy, he said.

Dr. Venook had no disclosures. Dr. Garcia-Aguilar reported personal fees from Medtronic, Johnson & Johnson, and Intuitive Surgical.
 

A version of this article first appeared on Medscape.com.

Having an ostomy is a dreaded prospect for many patients with rectal cancer.

To defer, and potentially avoid, this life-altering surgery, the watch-and-wait approach has become increasingly common among patients with locally advanced disease who have a complete response to neoadjuvant chemoradiation.

About 80% of these patients who have a complete clinical response — a perfectly healed scar where the tumor used to be and other favorable features — can forgo total mesorectal excision and preserve their rectum.

The success of watch-and-wait among complete responders has led some centers to offer the approach in patients with near-complete responses to neoadjuvant chemoradiation.

But watch-and-wait for near-complete clinical responders “is very controversial,” Alan P. Venook, MD, a gastrointestinal oncologist at the University of California, San Francisco (UCSF), told this news organization.

“You sure as hell don’t want to miss a chance to cure a patient,” Dr. Venook said.

A near-complete clinical response essentially means there is no sign of the tumor 8 weeks after total neoadjuvant therapy, but the tumor bed hasn’t completely healed.

The goal of watch-and-wait in this scenario is to give near-complete response lesions time to become complete responses.

But there’s no clear way to predict which tumors will evolve into a clinical complete response.

Recent studies evaluating the conversion rate have reported that anywhere from 39% to about 90% of near-complete responders became complete responders. Some of the variation likely comes down to differences in the clinical stage of patients evaluated in each study as well as the limited number of patients who achieve a near-complete response overall.

Other concerns have emerged that waiting for near-complete responses to become complete leaves extra time for some tumors to metastasize and that tumor regrowth is much higher compared with complete responders.

A recent study found that 13% of near-complete responders who preserved their rectum on watch-and-wait developed distant metastases vs about 5% of long-term complete responders. The study also found that just over half of near-complete responders have tumor regrowth compared with about one in five complete responders.

But even with regrowth, “surgery is still curative,” explained Julio Garcia-Aguilar, MD, PhD, a pioneer of watch-and-wait for rectal cancer.

And overall, around 50%-60% of patients with a near-complete response can avoid surgery and preserve their rectum.
 

Selecting Patients for Watch-and-Wait

The key to deciding which patients are right for watch-and-wait is to understand how a near-complete clinical response was defined in the OPRA trial, a landmark randomized trial led by Dr. Garcia-Aguilar that helped establish watch-and-wait as an option in rectal cancer.

OPRA defined a near-complete response as no visible tumor but, in the tumor bed, mild erythema, superficial ulceration, minor mucosal abnormality or small nodules, and an irregular mucosa. The criteria also included no palpable tumor with smooth induration or a minor mucosal abnormality on the digital rectal exam.

The National Comprehensive Cancer Network mirrored the definition when, for the first time, it recommended watch-and-wait as an option for near-complete response in its 2023 rectal cancer guidelines. The group also added a few MRI requirements.

UCSF offers the watch-and-wait option to some patients with near-complete responses, but each decision is made on a case-by-case basis by a tumor board considering numerous measures of tumor aggressiveness.

Even then, “we have, in many cases, struggled to figure out what the right choices are,” Dr. Venook said.

For those chosen for watch-and-wait, Dr. Venook noted that UCSF has top-notch surgeons, radiation oncologists, medical oncologists, and pathologists who have the resources to follow patients closely.

For community practices without the resources of a major cancer center, watch-and-wait for near-complete response to rectal cancer “is really asking a lot,” Dr. Venook said.

Dr. Garcia-Aguilar, a colorectal surgeon at Memorial Sloan Kettering Cancer Center in New York City, explained that after years of studying the issue, he is comfortable with watch-and-wait in near-complete responders as long as it’s done carefully and in patients who will comply with ongoing surveillance.

Dr. Garcia-Aguilar explained that, after diagnosing a near-complete response 8 weeks following total neoadjuvant therapy, the patient needs to come back 6 weeks later. At that point, it’s time to assess whether that near-complete response is evolving into a complete response or not evolving into a complete response.

If it’s evolving into a complete response, surveillance continues about every 8 weeks, but if the tumor has stopped responding, “you take [the patient] to the operating room,” Dr. Garcia-Aguilar said.

As for the bigger safety concern — that near clinical complete response tumors will metastasize — Dr. Garcia-Aguilar’s opinion is that micrometastases are probably already there when the rectal cancer is first diagnosed and will manifest themselves “no matter what happens to the primary tumor.”

Because of that, he noted, “I don’t think the risk is very high” when surgery is delayed a few months to give near-complete response patients a chance to keep their rectum.

The way to answer the metastasis question is to do a randomized trial pitting surgery against watch-and-wait in patients with near-clinical complete response rectal cancer.

However, Dr. Garcia-Aguilar doesn’t think that trial will ever happen. Patients won’t allow themselves to be randomized to surgery once they find out they might be able to avoid a permanent ostomy, he said.

Dr. Venook had no disclosures. Dr. Garcia-Aguilar reported personal fees from Medtronic, Johnson & Johnson, and Intuitive Surgical.
 

A version of this article first appeared on Medscape.com.

STOCKHOLM — At the American Society of Retina Specialists (ASRS) 2024 Annual Meeting, researchers discussed how insights into potential risk factors and new treatments could improve outcomes for patients with diabetic retinopathy.

Jennifer Lim, MD, an ophthalmologist and director of the Retina Service at the University of Illinois Hospital & Health Sciences System in Chicago, told this news organization that emerging approaches to treating diabetic retinopathy offer hope because they address the root causes of the disease beyond just targeting vascular endothelial growth factor (VEGF). She said innovative methods and add-on treatments could lead to more durable and effective drugs.

Exploration of risk factors and treatment options for diabetic retinopathy could lead to more effective management strategies for the condition, agreed David Boyer, MD, an ophthalmologist at Retina Vitreous Associates Medical Group in Los Angeles, speaking with this news organization.
 

Risk Factors for Diabetic Retinopathy

Sodium-glucose cotransporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have gained popularity because of their benefits beyond glycemic control, including weight loss, and cardiovascular and kidney protection. However, the impact of these medications on vision-threatening retinal complications is not fully understood. “There has always been a question about whether these newer diabetes medications might exacerbate diabetic eye disease,” said Dr. Boyer.

In a retrospective observational study, researchers included adults with type 2 diabetes and moderate cardiovascular disease risk who had no history of advanced diabetic retinal complications. These patients initiated treatment with GLP-1 RA, SGLT2 inhibitors, DPP-4 inhibitors, or sulfonylureas. The study used inverse probability of treatment weighting to mimic randomization and compared the time to the first treatment for diabetic macular edema or proliferative diabetic retinopathy across the treatment groups.

Results, presented by Andrew J. Barkmeier, MD, an associate professor of ophthalmology at the Mayo Clinic, showed that among 371,698 patients, those who initiated therapy with SGLT2 inhibitors had a lower risk of requiring treatment for sight-threatening retinopathy compared with those using other medication classes. GLP-1 RA did not increase retinopathy risk relative to dipeptidyl peptidase 4 inhibitors and sulfonylurea medications.

“[This study] told us that we do have to keep an eye on patients’ retinopathy when they start on these new inhibitors. But the progression is minimal and, overall, I think most people today favor keeping blood sugar levels as good as possible,” said Dr. Boyer, who was not involved in the study.

Another factor that might increase diabetic retinopathy progression is obstructive sleep apnea. This underdiagnosed condition is linked to several health issues, including dementia, stroke, and myocardial infarctions. Although not easily treated, obstructive sleep apnea is manageable, Dr. Boyer explained.

Researchers utilized the TriNetX electronic health records research network to identify patients with nonproliferative diabetic retinopathy, both with and without obstructive sleep apnea. 

The results, presented by Ehsan Rahimy, MD, a retinal specialist at Palo Alto Medical Foundation and a professor at Stanford University, showed that patients with obstructive sleep apnea had a significantly higher risk of progressing to proliferative diabetic retinopathy and developing new-onset diabetic macular edema. These patients were more likely to require ocular interventions, such as intravitreal injections and laser photocoagulation. They also had greater risks for stroke, myocardial infarction, and death compared with those who did not have obstructive sleep apnea.

“It was good to bring this to everybody’s attention,” said Dr. Boyer, who was not involved in the study. “It’s an easy question to ask someone if they snore.”
 

New Treatments on the Horizon

In another presentation, Nathan C. Steinle, MD, of California Retina Consultants, presented a study that assessed the durability of response to sozinibercept in patients with retinal vascular diseases. This novel therapeutic agent is designed to inhibit VEGF-C and VEGF-D in conditions where VEGF-A suppression alone is insufficient. 

Sozinibercept was combined with standard anti–VEGF-A therapies such as ranibizumab or aflibercept. It involved a prospective, post hoc analysis of two phase 1b, open-label, dose-escalation studies, including 40 patients with neovascular age-related macular degeneration (nAMD; 31 patients) or diabetic macular edema (nine patients). These patients, either treatment-naive or previously treated, received three intravitreal injections of ranibizumab or aflibercept in combination with sozinibercept at various doses.

Results indicated that sozinibercept combination therapy was well tolerated, with no dose-limiting toxicities. In treatment-naive nAMD patients, the mean best-corrected visual acuity (BCVA) improved significantly from baseline at months 3 and 6. Previously treated nAMD patients also showed BCVA improvements, although to a lesser extent. For patients with persistent diabetic macular edema, switching to sozinibercept plus aflibercept resulted in notable BCVA gains. The mean time to requiring retreatment was longer in treatment-naive patients than in those previously treated, indicating a durable response. 

“Combination therapy with sozinibercept is going to be really important,” said Dr. Lim, who was not involved in the study, “because it attacks with a dual mechanism of action.”

Oral agents promise a potentially easier alternative for patients compared with frequent injections. CU06-1004 is a novel orally administered endothelial dysfunction blocker that has shown promise in stabilizing damaged capillaries, reducing abnormal angiogenesis, and inhibiting inflammatory activation in preclinical studies. “CU06 is very interesting to me because by preventing endothelial loss, it gets to the pathophysiology of why the blood vessels break down,” Dr. Lim said.

In a proof-of-concept phase 2a, multicenter, open-label, parallel-group trial, investigators randomly assigned 67 patients with diabetic macular edema to receive 100 mg, 200 mg, or 300 mg of CU06-1004 once daily for 12 weeks, followed by a 4-week follow-up. 

Results presented by Victor Gonzalez, MD, of Valley Retina Institute in Texas, indicated that the oral agent improved BCVA, stabilized central subfield thickness, and showed positive anatomical changes in optical coherence tomography images. CU06-1004 was well tolerated, with no drug-related serious adverse events. 

“The number [of patients] was very small, and we will need a much longer, larger trial to see if [CU06-1004] has benefits long term,” said Dr. Boyer, who was not involved in the study. “But I think we’re all very excited if we can find an oral agent for treating diabetic retinopathy. It would be easier for the patient to take a pill than having to come in for injections.” 

The sustained-release axitinib implant, OTX-TKI, is also generating significant interest, particularly for nonproliferative diabetic retinopathy. Axitinib, a tyrosine kinase inhibitor (TKI), targets signaling pathways crucial in cellular processes, providing a novel approach to managing diseases where traditional therapies might fall short. Unlike traditional anti-VEGF treatments that focus solely on cytokine levels, TKIs block the activation of signaling pathways, preventing downstream signaling regardless of cytokine levels. This mechanism is particularly important because it effectively inhibits disease progression even if levels of VEGF are high, Dr. Lim explained.

In the phase 1 HELIOS trial, OTX-TKI was assessed in patients with nonproliferative diabetic retinopathy. This multicenter, double-masked, parallel-group clinical study included 21 patients who had not received anti-VEGF treatment, dexamethasone intravitreal implants in the previous 12 months, or intraocular steroid injections in the prior 4 months. Patients were randomly assigned to receive either OTX-TKI or sham treatment.

Results presented by Dilsher S. Dhoot, MD, of California Retina Consultants, indicated that OTX-TKI was generally well tolerated, with no serious ocular adverse events. At 48 weeks, 46.2% of eyes treated with OTX-TKI showed a 1- or 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) compared with none in the sham arm. Additionally, no eyes treated with OTX-TKI experienced a worsening on the DRSS, whereas 25% of eyes in the sham arm did. Vision-threatening complications, such as proliferative diabetic retinopathy or diabetic macular edema, developed in 37.5% of the sham group but in none of the OTX-TKI treated eyes. A single injection of OTX-TKI provided durable DRSS improvement for up to 48 weeks, with no patients in either arm requiring rescue therapy.

“This is a really exciting add-on treatment,” Dr. Lim said, who was not involved in the study. She explained that it is initially necessary to control the disease with standard treatments, because TKIs may take longer to exhibit their effects. Once the disease is stabilized, TKIs can be used alongside other therapies, potentially reducing the reliance on frequent anti-VEGF injections. “These are preliminary results, but that’s the hope going forward.”

Dr. Lim and Dr. Boyer report no relevant financial relationships.

A version of this article first appeared on Medscape.com.

STOCKHOLM — At the American Society of Retina Specialists (ASRS) 2024 Annual Meeting, researchers discussed how insights into potential risk factors and new treatments could improve outcomes for patients with diabetic retinopathy.

Jennifer Lim, MD, an ophthalmologist and director of the Retina Service at the University of Illinois Hospital & Health Sciences System in Chicago, told this news organization that emerging approaches to treating diabetic retinopathy offer hope because they address the root causes of the disease beyond just targeting vascular endothelial growth factor (VEGF). She said innovative methods and add-on treatments could lead to more durable and effective drugs.

Exploration of risk factors and treatment options for diabetic retinopathy could lead to more effective management strategies for the condition, agreed David Boyer, MD, an ophthalmologist at Retina Vitreous Associates Medical Group in Los Angeles, speaking with this news organization.
 

Risk Factors for Diabetic Retinopathy

Sodium-glucose cotransporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have gained popularity because of their benefits beyond glycemic control, including weight loss, and cardiovascular and kidney protection. However, the impact of these medications on vision-threatening retinal complications is not fully understood. “There has always been a question about whether these newer diabetes medications might exacerbate diabetic eye disease,” said Dr. Boyer.

In a retrospective observational study, researchers included adults with type 2 diabetes and moderate cardiovascular disease risk who had no history of advanced diabetic retinal complications. These patients initiated treatment with GLP-1 RA, SGLT2 inhibitors, DPP-4 inhibitors, or sulfonylureas. The study used inverse probability of treatment weighting to mimic randomization and compared the time to the first treatment for diabetic macular edema or proliferative diabetic retinopathy across the treatment groups.

Results, presented by Andrew J. Barkmeier, MD, an associate professor of ophthalmology at the Mayo Clinic, showed that among 371,698 patients, those who initiated therapy with SGLT2 inhibitors had a lower risk of requiring treatment for sight-threatening retinopathy compared with those using other medication classes. GLP-1 RA did not increase retinopathy risk relative to dipeptidyl peptidase 4 inhibitors and sulfonylurea medications.

“[This study] told us that we do have to keep an eye on patients’ retinopathy when they start on these new inhibitors. But the progression is minimal and, overall, I think most people today favor keeping blood sugar levels as good as possible,” said Dr. Boyer, who was not involved in the study.

Another factor that might increase diabetic retinopathy progression is obstructive sleep apnea. This underdiagnosed condition is linked to several health issues, including dementia, stroke, and myocardial infarctions. Although not easily treated, obstructive sleep apnea is manageable, Dr. Boyer explained.

Researchers utilized the TriNetX electronic health records research network to identify patients with nonproliferative diabetic retinopathy, both with and without obstructive sleep apnea. 

The results, presented by Ehsan Rahimy, MD, a retinal specialist at Palo Alto Medical Foundation and a professor at Stanford University, showed that patients with obstructive sleep apnea had a significantly higher risk of progressing to proliferative diabetic retinopathy and developing new-onset diabetic macular edema. These patients were more likely to require ocular interventions, such as intravitreal injections and laser photocoagulation. They also had greater risks for stroke, myocardial infarction, and death compared with those who did not have obstructive sleep apnea.

“It was good to bring this to everybody’s attention,” said Dr. Boyer, who was not involved in the study. “It’s an easy question to ask someone if they snore.”
 

New Treatments on the Horizon

In another presentation, Nathan C. Steinle, MD, of California Retina Consultants, presented a study that assessed the durability of response to sozinibercept in patients with retinal vascular diseases. This novel therapeutic agent is designed to inhibit VEGF-C and VEGF-D in conditions where VEGF-A suppression alone is insufficient. 

Sozinibercept was combined with standard anti–VEGF-A therapies such as ranibizumab or aflibercept. It involved a prospective, post hoc analysis of two phase 1b, open-label, dose-escalation studies, including 40 patients with neovascular age-related macular degeneration (nAMD; 31 patients) or diabetic macular edema (nine patients). These patients, either treatment-naive or previously treated, received three intravitreal injections of ranibizumab or aflibercept in combination with sozinibercept at various doses.

Results indicated that sozinibercept combination therapy was well tolerated, with no dose-limiting toxicities. In treatment-naive nAMD patients, the mean best-corrected visual acuity (BCVA) improved significantly from baseline at months 3 and 6. Previously treated nAMD patients also showed BCVA improvements, although to a lesser extent. For patients with persistent diabetic macular edema, switching to sozinibercept plus aflibercept resulted in notable BCVA gains. The mean time to requiring retreatment was longer in treatment-naive patients than in those previously treated, indicating a durable response. 

“Combination therapy with sozinibercept is going to be really important,” said Dr. Lim, who was not involved in the study, “because it attacks with a dual mechanism of action.”

Oral agents promise a potentially easier alternative for patients compared with frequent injections. CU06-1004 is a novel orally administered endothelial dysfunction blocker that has shown promise in stabilizing damaged capillaries, reducing abnormal angiogenesis, and inhibiting inflammatory activation in preclinical studies. “CU06 is very interesting to me because by preventing endothelial loss, it gets to the pathophysiology of why the blood vessels break down,” Dr. Lim said.

In a proof-of-concept phase 2a, multicenter, open-label, parallel-group trial, investigators randomly assigned 67 patients with diabetic macular edema to receive 100 mg, 200 mg, or 300 mg of CU06-1004 once daily for 12 weeks, followed by a 4-week follow-up. 

Results presented by Victor Gonzalez, MD, of Valley Retina Institute in Texas, indicated that the oral agent improved BCVA, stabilized central subfield thickness, and showed positive anatomical changes in optical coherence tomography images. CU06-1004 was well tolerated, with no drug-related serious adverse events. 

“The number [of patients] was very small, and we will need a much longer, larger trial to see if [CU06-1004] has benefits long term,” said Dr. Boyer, who was not involved in the study. “But I think we’re all very excited if we can find an oral agent for treating diabetic retinopathy. It would be easier for the patient to take a pill than having to come in for injections.” 

The sustained-release axitinib implant, OTX-TKI, is also generating significant interest, particularly for nonproliferative diabetic retinopathy. Axitinib, a tyrosine kinase inhibitor (TKI), targets signaling pathways crucial in cellular processes, providing a novel approach to managing diseases where traditional therapies might fall short. Unlike traditional anti-VEGF treatments that focus solely on cytokine levels, TKIs block the activation of signaling pathways, preventing downstream signaling regardless of cytokine levels. This mechanism is particularly important because it effectively inhibits disease progression even if levels of VEGF are high, Dr. Lim explained.

In the phase 1 HELIOS trial, OTX-TKI was assessed in patients with nonproliferative diabetic retinopathy. This multicenter, double-masked, parallel-group clinical study included 21 patients who had not received anti-VEGF treatment, dexamethasone intravitreal implants in the previous 12 months, or intraocular steroid injections in the prior 4 months. Patients were randomly assigned to receive either OTX-TKI or sham treatment.

Results presented by Dilsher S. Dhoot, MD, of California Retina Consultants, indicated that OTX-TKI was generally well tolerated, with no serious ocular adverse events. At 48 weeks, 46.2% of eyes treated with OTX-TKI showed a 1- or 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) compared with none in the sham arm. Additionally, no eyes treated with OTX-TKI experienced a worsening on the DRSS, whereas 25% of eyes in the sham arm did. Vision-threatening complications, such as proliferative diabetic retinopathy or diabetic macular edema, developed in 37.5% of the sham group but in none of the OTX-TKI treated eyes. A single injection of OTX-TKI provided durable DRSS improvement for up to 48 weeks, with no patients in either arm requiring rescue therapy.

“This is a really exciting add-on treatment,” Dr. Lim said, who was not involved in the study. She explained that it is initially necessary to control the disease with standard treatments, because TKIs may take longer to exhibit their effects. Once the disease is stabilized, TKIs can be used alongside other therapies, potentially reducing the reliance on frequent anti-VEGF injections. “These are preliminary results, but that’s the hope going forward.”

Dr. Lim and Dr. Boyer report no relevant financial relationships.

A version of this article first appeared on Medscape.com.

STOCKHOLM — At the American Society of Retina Specialists (ASRS) 2024 Annual Meeting, researchers discussed how insights into potential risk factors and new treatments could improve outcomes for patients with diabetic retinopathy.

Jennifer Lim, MD, an ophthalmologist and director of the Retina Service at the University of Illinois Hospital & Health Sciences System in Chicago, told this news organization that emerging approaches to treating diabetic retinopathy offer hope because they address the root causes of the disease beyond just targeting vascular endothelial growth factor (VEGF). She said innovative methods and add-on treatments could lead to more durable and effective drugs.

Exploration of risk factors and treatment options for diabetic retinopathy could lead to more effective management strategies for the condition, agreed David Boyer, MD, an ophthalmologist at Retina Vitreous Associates Medical Group in Los Angeles, speaking with this news organization.
 

Risk Factors for Diabetic Retinopathy

Sodium-glucose cotransporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have gained popularity because of their benefits beyond glycemic control, including weight loss, and cardiovascular and kidney protection. However, the impact of these medications on vision-threatening retinal complications is not fully understood. “There has always been a question about whether these newer diabetes medications might exacerbate diabetic eye disease,” said Dr. Boyer.

In a retrospective observational study, researchers included adults with type 2 diabetes and moderate cardiovascular disease risk who had no history of advanced diabetic retinal complications. These patients initiated treatment with GLP-1 RA, SGLT2 inhibitors, DPP-4 inhibitors, or sulfonylureas. The study used inverse probability of treatment weighting to mimic randomization and compared the time to the first treatment for diabetic macular edema or proliferative diabetic retinopathy across the treatment groups.

Results, presented by Andrew J. Barkmeier, MD, an associate professor of ophthalmology at the Mayo Clinic, showed that among 371,698 patients, those who initiated therapy with SGLT2 inhibitors had a lower risk of requiring treatment for sight-threatening retinopathy compared with those using other medication classes. GLP-1 RA did not increase retinopathy risk relative to dipeptidyl peptidase 4 inhibitors and sulfonylurea medications.

“[This study] told us that we do have to keep an eye on patients’ retinopathy when they start on these new inhibitors. But the progression is minimal and, overall, I think most people today favor keeping blood sugar levels as good as possible,” said Dr. Boyer, who was not involved in the study.

Another factor that might increase diabetic retinopathy progression is obstructive sleep apnea. This underdiagnosed condition is linked to several health issues, including dementia, stroke, and myocardial infarctions. Although not easily treated, obstructive sleep apnea is manageable, Dr. Boyer explained.

Researchers utilized the TriNetX electronic health records research network to identify patients with nonproliferative diabetic retinopathy, both with and without obstructive sleep apnea. 

The results, presented by Ehsan Rahimy, MD, a retinal specialist at Palo Alto Medical Foundation and a professor at Stanford University, showed that patients with obstructive sleep apnea had a significantly higher risk of progressing to proliferative diabetic retinopathy and developing new-onset diabetic macular edema. These patients were more likely to require ocular interventions, such as intravitreal injections and laser photocoagulation. They also had greater risks for stroke, myocardial infarction, and death compared with those who did not have obstructive sleep apnea.

“It was good to bring this to everybody’s attention,” said Dr. Boyer, who was not involved in the study. “It’s an easy question to ask someone if they snore.”
 

New Treatments on the Horizon

In another presentation, Nathan C. Steinle, MD, of California Retina Consultants, presented a study that assessed the durability of response to sozinibercept in patients with retinal vascular diseases. This novel therapeutic agent is designed to inhibit VEGF-C and VEGF-D in conditions where VEGF-A suppression alone is insufficient. 

Sozinibercept was combined with standard anti–VEGF-A therapies such as ranibizumab or aflibercept. It involved a prospective, post hoc analysis of two phase 1b, open-label, dose-escalation studies, including 40 patients with neovascular age-related macular degeneration (nAMD; 31 patients) or diabetic macular edema (nine patients). These patients, either treatment-naive or previously treated, received three intravitreal injections of ranibizumab or aflibercept in combination with sozinibercept at various doses.

Results indicated that sozinibercept combination therapy was well tolerated, with no dose-limiting toxicities. In treatment-naive nAMD patients, the mean best-corrected visual acuity (BCVA) improved significantly from baseline at months 3 and 6. Previously treated nAMD patients also showed BCVA improvements, although to a lesser extent. For patients with persistent diabetic macular edema, switching to sozinibercept plus aflibercept resulted in notable BCVA gains. The mean time to requiring retreatment was longer in treatment-naive patients than in those previously treated, indicating a durable response. 

“Combination therapy with sozinibercept is going to be really important,” said Dr. Lim, who was not involved in the study, “because it attacks with a dual mechanism of action.”

Oral agents promise a potentially easier alternative for patients compared with frequent injections. CU06-1004 is a novel orally administered endothelial dysfunction blocker that has shown promise in stabilizing damaged capillaries, reducing abnormal angiogenesis, and inhibiting inflammatory activation in preclinical studies. “CU06 is very interesting to me because by preventing endothelial loss, it gets to the pathophysiology of why the blood vessels break down,” Dr. Lim said.

In a proof-of-concept phase 2a, multicenter, open-label, parallel-group trial, investigators randomly assigned 67 patients with diabetic macular edema to receive 100 mg, 200 mg, or 300 mg of CU06-1004 once daily for 12 weeks, followed by a 4-week follow-up. 

Results presented by Victor Gonzalez, MD, of Valley Retina Institute in Texas, indicated that the oral agent improved BCVA, stabilized central subfield thickness, and showed positive anatomical changes in optical coherence tomography images. CU06-1004 was well tolerated, with no drug-related serious adverse events. 

“The number [of patients] was very small, and we will need a much longer, larger trial to see if [CU06-1004] has benefits long term,” said Dr. Boyer, who was not involved in the study. “But I think we’re all very excited if we can find an oral agent for treating diabetic retinopathy. It would be easier for the patient to take a pill than having to come in for injections.” 

The sustained-release axitinib implant, OTX-TKI, is also generating significant interest, particularly for nonproliferative diabetic retinopathy. Axitinib, a tyrosine kinase inhibitor (TKI), targets signaling pathways crucial in cellular processes, providing a novel approach to managing diseases where traditional therapies might fall short. Unlike traditional anti-VEGF treatments that focus solely on cytokine levels, TKIs block the activation of signaling pathways, preventing downstream signaling regardless of cytokine levels. This mechanism is particularly important because it effectively inhibits disease progression even if levels of VEGF are high, Dr. Lim explained.

In the phase 1 HELIOS trial, OTX-TKI was assessed in patients with nonproliferative diabetic retinopathy. This multicenter, double-masked, parallel-group clinical study included 21 patients who had not received anti-VEGF treatment, dexamethasone intravitreal implants in the previous 12 months, or intraocular steroid injections in the prior 4 months. Patients were randomly assigned to receive either OTX-TKI or sham treatment.

Results presented by Dilsher S. Dhoot, MD, of California Retina Consultants, indicated that OTX-TKI was generally well tolerated, with no serious ocular adverse events. At 48 weeks, 46.2% of eyes treated with OTX-TKI showed a 1- or 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) compared with none in the sham arm. Additionally, no eyes treated with OTX-TKI experienced a worsening on the DRSS, whereas 25% of eyes in the sham arm did. Vision-threatening complications, such as proliferative diabetic retinopathy or diabetic macular edema, developed in 37.5% of the sham group but in none of the OTX-TKI treated eyes. A single injection of OTX-TKI provided durable DRSS improvement for up to 48 weeks, with no patients in either arm requiring rescue therapy.

“This is a really exciting add-on treatment,” Dr. Lim said, who was not involved in the study. She explained that it is initially necessary to control the disease with standard treatments, because TKIs may take longer to exhibit their effects. Once the disease is stabilized, TKIs can be used alongside other therapies, potentially reducing the reliance on frequent anti-VEGF injections. “These are preliminary results, but that’s the hope going forward.”

Dr. Lim and Dr. Boyer report no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Take a look at any of the evidence-based US obesity treatment guidelines. The key criteria for diagnosing overweight and obesity is based on the body mass index (BMI). 

The guidelines also use BMI to stratify care options to decrease cardiovascular risk. For example, persons with BMI ≥30 are classified as having obesity, and antiobesity medications are recommended. Those with BMI ≥ 40 are classified as having severe obesity, and metabolic bariatric surgery may be appropriate. 

But where did these cutoff points for more and less aggressive treatments come from? These BMI cutoffs are based primarily on mortality data collected from large non-Hispanic White populations, without data on potential differences by gender and ethnicity. In fact, by itself, BMI is an incomplete measure of cardiometabolic risk, especially in a multiethnic clinic with all genders represented.

For example, it is certainly true that those with BMI ≥ 30 have more cardiovascular risk factors than those with BMI < 30. But Asian American individuals have more risk factors at lower BMIs than do White or African American individuals likely because of more visceral fat accumulation at lower BMIs.

Besides the variation in gender and ethnicity, BMI does not take the type and location of body fat into consideration. Adipose tissue in visceral or ectopic areas have much higher risks for disease than subcutaneous adipose tissue because of the associated inflammation. Measures such as waist circumference, waist-to-hip ratio, and skinfold measurements aim to capture this aspect but often fall short because of variation in techniques.

BMI does not account for muscle mass either, so fit athletes and bodybuilders can be classified as having obesity by BMI alone. More accurate body fat percent measures, such as dual-energy X-ray absorptiometry or MRI specifically for ectopic fat, are labor intensive, expensive, and not feasible to perform in a busy primary care or endocrinology clinic.
 

Assessing Risks From Obesity Beyond BMI

Clearly, better risk measures than BMI are needed, but until they are available, supplemental clinical tools can aid diagnosis and treatment decisions at obesity medicine specialty centers, endocrinology and diabetes centers, and those centers that focus on the treatment of obesity.

For example, a seca scale can measure percent body fat by bioelectric impedance analysis. This technique also has its limitations, but for persons who are well hydrated, it can be used as a baseline to determine efficacy of behavioral interventions, such as resistance-exercise training and a high-protein diet to protect muscle mass as the patient loses weight.

A lot also can be gleaned from diet and exercise history, social history, family history, and physical exam as well as laboratory analyses. For example, an Asian American patient with a BMI of 26 who has been gaining weight mostly in the abdominal region after age 35 years is likely to have cardiometabolic risk, and a family history can solidify that. An exam can show signs of acanthosis nigricans or an enlarged liver and generous abdominal adipose tissue. This would be the patient in whom you would want to obtain a hemoglobin A1c measurement in the chance that it is elevated at > 5.7 mg/dL, suggesting high risk for type 2 diabetes. 

A Fibrosis-4 score can assess the risk for liver disease from aspartate transaminase and alanine aminotransferase and platelet count and age, providing clues to cardiometabolic disease risk.

In the next 10, years there may be a better measure for cardiometabolic risk that is more accurate than BMI is. It could be the sagittal abdominal diameter, which has been purported to more accurately measure visceral abdominal fat. But this has not made it to be one of the vital signs in a busy primary care clinic, however. 
 

Will New Body Fat Tools Change Practice?

In the next 10 years, there may be an affordable gadget to scan the body to determine visceral vs subcutaneous deposition of fat — like radiography for tissue. Now, three-dimensional (3D) total-body scanners can obtain body composition, but they are extremely expensive. The more important clinical question is: How will the use of these imaging modalities change your practice protocol for a particular patient? 

Think about the FibroScan, a type of ultrasound used to determine fatty liver disease and fibrosis. We order the test for those patients in whom we already have a strong suspicion for liver disease and, in obesity practices, for fatty liver and metabolic-associated fatty liver disease or metabolic associated steatohepatitis.

The test results do much to educate the patient and help the patient understand the need for aggressive treatment for their obesity. But it doesn’t necessarily change the clinician’s practice protocols and decisions. We would still recommend weight management and medications or surgery to patients regardless of the findings. 

A FibroScan is an expense, and not all primary care or endocrine practitioners may feel it necessary to purchase one for the added benefit of patient education. And I would argue that a 3D body scanner is a great tool but more for educational purposes than to really determine practice decision-making or outcomes. 

In the meantime, an old-fashioned physical examination, along with a thorough medical, social, and family history should give even the busiest primary care provider enough information to decide whether their patient is a candidate for preventive measures to reduce body fat with diet, exercise, and medication as well as whether the patient is a candidate for metabolic bariatric surgery. Higher suspicion of cardiovascular risk at lower BMI ranges for various ethnicities can help primary care providers pick up on the patients with low BMI but who are at higher risk for type 2 diabetes or prediabetes and cardiovascular disease. 

So the answer to whether we need a better measure than the BMI: Yes, we do. We need a physical examination on all patients.

Dr. Apovian, professor of medicine, Harvard Medical School, and codirector, Center for Weight Management and Wellness, Brigham and Women’s Hospital, both in Boston, Massachusetts, disclosed ties with Altimmune, CinFina Pharma, Cowen and Company, EPG Communication Holdings, Form Health, Gelesis, L-Nutra, NeuroBo Pharm, Novo, OptumRx, Pain Script, Palatin, Pursuit by You, Roman Health, Xeno, and Riverview School.

A version of this article appeared on Medscape.com.

Take a look at any of the evidence-based US obesity treatment guidelines. The key criteria for diagnosing overweight and obesity is based on the body mass index (BMI). 

The guidelines also use BMI to stratify care options to decrease cardiovascular risk. For example, persons with BMI ≥30 are classified as having obesity, and antiobesity medications are recommended. Those with BMI ≥ 40 are classified as having severe obesity, and metabolic bariatric surgery may be appropriate. 

But where did these cutoff points for more and less aggressive treatments come from? These BMI cutoffs are based primarily on mortality data collected from large non-Hispanic White populations, without data on potential differences by gender and ethnicity. In fact, by itself, BMI is an incomplete measure of cardiometabolic risk, especially in a multiethnic clinic with all genders represented.

For example, it is certainly true that those with BMI ≥ 30 have more cardiovascular risk factors than those with BMI < 30. But Asian American individuals have more risk factors at lower BMIs than do White or African American individuals likely because of more visceral fat accumulation at lower BMIs.

Besides the variation in gender and ethnicity, BMI does not take the type and location of body fat into consideration. Adipose tissue in visceral or ectopic areas have much higher risks for disease than subcutaneous adipose tissue because of the associated inflammation. Measures such as waist circumference, waist-to-hip ratio, and skinfold measurements aim to capture this aspect but often fall short because of variation in techniques.

BMI does not account for muscle mass either, so fit athletes and bodybuilders can be classified as having obesity by BMI alone. More accurate body fat percent measures, such as dual-energy X-ray absorptiometry or MRI specifically for ectopic fat, are labor intensive, expensive, and not feasible to perform in a busy primary care or endocrinology clinic.
 

Assessing Risks From Obesity Beyond BMI

Clearly, better risk measures than BMI are needed, but until they are available, supplemental clinical tools can aid diagnosis and treatment decisions at obesity medicine specialty centers, endocrinology and diabetes centers, and those centers that focus on the treatment of obesity.

For example, a seca scale can measure percent body fat by bioelectric impedance analysis. This technique also has its limitations, but for persons who are well hydrated, it can be used as a baseline to determine efficacy of behavioral interventions, such as resistance-exercise training and a high-protein diet to protect muscle mass as the patient loses weight.

A lot also can be gleaned from diet and exercise history, social history, family history, and physical exam as well as laboratory analyses. For example, an Asian American patient with a BMI of 26 who has been gaining weight mostly in the abdominal region after age 35 years is likely to have cardiometabolic risk, and a family history can solidify that. An exam can show signs of acanthosis nigricans or an enlarged liver and generous abdominal adipose tissue. This would be the patient in whom you would want to obtain a hemoglobin A1c measurement in the chance that it is elevated at > 5.7 mg/dL, suggesting high risk for type 2 diabetes. 

A Fibrosis-4 score can assess the risk for liver disease from aspartate transaminase and alanine aminotransferase and platelet count and age, providing clues to cardiometabolic disease risk.

In the next 10, years there may be a better measure for cardiometabolic risk that is more accurate than BMI is. It could be the sagittal abdominal diameter, which has been purported to more accurately measure visceral abdominal fat. But this has not made it to be one of the vital signs in a busy primary care clinic, however. 
 

Will New Body Fat Tools Change Practice?

In the next 10 years, there may be an affordable gadget to scan the body to determine visceral vs subcutaneous deposition of fat — like radiography for tissue. Now, three-dimensional (3D) total-body scanners can obtain body composition, but they are extremely expensive. The more important clinical question is: How will the use of these imaging modalities change your practice protocol for a particular patient? 

Think about the FibroScan, a type of ultrasound used to determine fatty liver disease and fibrosis. We order the test for those patients in whom we already have a strong suspicion for liver disease and, in obesity practices, for fatty liver and metabolic-associated fatty liver disease or metabolic associated steatohepatitis.

The test results do much to educate the patient and help the patient understand the need for aggressive treatment for their obesity. But it doesn’t necessarily change the clinician’s practice protocols and decisions. We would still recommend weight management and medications or surgery to patients regardless of the findings. 

A FibroScan is an expense, and not all primary care or endocrine practitioners may feel it necessary to purchase one for the added benefit of patient education. And I would argue that a 3D body scanner is a great tool but more for educational purposes than to really determine practice decision-making or outcomes. 

In the meantime, an old-fashioned physical examination, along with a thorough medical, social, and family history should give even the busiest primary care provider enough information to decide whether their patient is a candidate for preventive measures to reduce body fat with diet, exercise, and medication as well as whether the patient is a candidate for metabolic bariatric surgery. Higher suspicion of cardiovascular risk at lower BMI ranges for various ethnicities can help primary care providers pick up on the patients with low BMI but who are at higher risk for type 2 diabetes or prediabetes and cardiovascular disease. 

So the answer to whether we need a better measure than the BMI: Yes, we do. We need a physical examination on all patients.

Dr. Apovian, professor of medicine, Harvard Medical School, and codirector, Center for Weight Management and Wellness, Brigham and Women’s Hospital, both in Boston, Massachusetts, disclosed ties with Altimmune, CinFina Pharma, Cowen and Company, EPG Communication Holdings, Form Health, Gelesis, L-Nutra, NeuroBo Pharm, Novo, OptumRx, Pain Script, Palatin, Pursuit by You, Roman Health, Xeno, and Riverview School.

A version of this article appeared on Medscape.com.

Take a look at any of the evidence-based US obesity treatment guidelines. The key criteria for diagnosing overweight and obesity is based on the body mass index (BMI). 

The guidelines also use BMI to stratify care options to decrease cardiovascular risk. For example, persons with BMI ≥30 are classified as having obesity, and antiobesity medications are recommended. Those with BMI ≥ 40 are classified as having severe obesity, and metabolic bariatric surgery may be appropriate. 

But where did these cutoff points for more and less aggressive treatments come from? These BMI cutoffs are based primarily on mortality data collected from large non-Hispanic White populations, without data on potential differences by gender and ethnicity. In fact, by itself, BMI is an incomplete measure of cardiometabolic risk, especially in a multiethnic clinic with all genders represented.

For example, it is certainly true that those with BMI ≥ 30 have more cardiovascular risk factors than those with BMI < 30. But Asian American individuals have more risk factors at lower BMIs than do White or African American individuals likely because of more visceral fat accumulation at lower BMIs.

Besides the variation in gender and ethnicity, BMI does not take the type and location of body fat into consideration. Adipose tissue in visceral or ectopic areas have much higher risks for disease than subcutaneous adipose tissue because of the associated inflammation. Measures such as waist circumference, waist-to-hip ratio, and skinfold measurements aim to capture this aspect but often fall short because of variation in techniques.

BMI does not account for muscle mass either, so fit athletes and bodybuilders can be classified as having obesity by BMI alone. More accurate body fat percent measures, such as dual-energy X-ray absorptiometry or MRI specifically for ectopic fat, are labor intensive, expensive, and not feasible to perform in a busy primary care or endocrinology clinic.
 

Assessing Risks From Obesity Beyond BMI

Clearly, better risk measures than BMI are needed, but until they are available, supplemental clinical tools can aid diagnosis and treatment decisions at obesity medicine specialty centers, endocrinology and diabetes centers, and those centers that focus on the treatment of obesity.

For example, a seca scale can measure percent body fat by bioelectric impedance analysis. This technique also has its limitations, but for persons who are well hydrated, it can be used as a baseline to determine efficacy of behavioral interventions, such as resistance-exercise training and a high-protein diet to protect muscle mass as the patient loses weight.

A lot also can be gleaned from diet and exercise history, social history, family history, and physical exam as well as laboratory analyses. For example, an Asian American patient with a BMI of 26 who has been gaining weight mostly in the abdominal region after age 35 years is likely to have cardiometabolic risk, and a family history can solidify that. An exam can show signs of acanthosis nigricans or an enlarged liver and generous abdominal adipose tissue. This would be the patient in whom you would want to obtain a hemoglobin A1c measurement in the chance that it is elevated at > 5.7 mg/dL, suggesting high risk for type 2 diabetes. 

A Fibrosis-4 score can assess the risk for liver disease from aspartate transaminase and alanine aminotransferase and platelet count and age, providing clues to cardiometabolic disease risk.

In the next 10, years there may be a better measure for cardiometabolic risk that is more accurate than BMI is. It could be the sagittal abdominal diameter, which has been purported to more accurately measure visceral abdominal fat. But this has not made it to be one of the vital signs in a busy primary care clinic, however. 
 

Will New Body Fat Tools Change Practice?

In the next 10 years, there may be an affordable gadget to scan the body to determine visceral vs subcutaneous deposition of fat — like radiography for tissue. Now, three-dimensional (3D) total-body scanners can obtain body composition, but they are extremely expensive. The more important clinical question is: How will the use of these imaging modalities change your practice protocol for a particular patient? 

Think about the FibroScan, a type of ultrasound used to determine fatty liver disease and fibrosis. We order the test for those patients in whom we already have a strong suspicion for liver disease and, in obesity practices, for fatty liver and metabolic-associated fatty liver disease or metabolic associated steatohepatitis.

The test results do much to educate the patient and help the patient understand the need for aggressive treatment for their obesity. But it doesn’t necessarily change the clinician’s practice protocols and decisions. We would still recommend weight management and medications or surgery to patients regardless of the findings. 

A FibroScan is an expense, and not all primary care or endocrine practitioners may feel it necessary to purchase one for the added benefit of patient education. And I would argue that a 3D body scanner is a great tool but more for educational purposes than to really determine practice decision-making or outcomes. 

In the meantime, an old-fashioned physical examination, along with a thorough medical, social, and family history should give even the busiest primary care provider enough information to decide whether their patient is a candidate for preventive measures to reduce body fat with diet, exercise, and medication as well as whether the patient is a candidate for metabolic bariatric surgery. Higher suspicion of cardiovascular risk at lower BMI ranges for various ethnicities can help primary care providers pick up on the patients with low BMI but who are at higher risk for type 2 diabetes or prediabetes and cardiovascular disease. 

So the answer to whether we need a better measure than the BMI: Yes, we do. We need a physical examination on all patients.

Dr. Apovian, professor of medicine, Harvard Medical School, and codirector, Center for Weight Management and Wellness, Brigham and Women’s Hospital, both in Boston, Massachusetts, disclosed ties with Altimmune, CinFina Pharma, Cowen and Company, EPG Communication Holdings, Form Health, Gelesis, L-Nutra, NeuroBo Pharm, Novo, OptumRx, Pain Script, Palatin, Pursuit by You, Roman Health, Xeno, and Riverview School.

A version of this article appeared on Medscape.com.

The allegations against Algerian boxer Imane Khelif at the Paris Olympics raised the questions of intersexuality and its implications in competitive sports. This news organization has decided to delve into the topic to assist doctors who suspect a similar condition in their patients. No certain clinical data about Ms. Khelif have been made public, so this article does not concern the boxer but rather takes inspiration from the media controversy.

What Is Intersexuality?

Intersexuality encompasses a spectrum of variations in sexual development that lead to the simultaneous presence of typical male and female characteristics. As reiterated by the United Nations Office of the High Commissioner for Human Rights, the medical definition does not affect the patient’s self-identification of gender or sexual orientation.

“The percentage of people who fall within the intersexuality spectrum is less than 0.5 per thousand of the general population, but there are no precise statistics, given the difficulty of definition,” said Roberto Lala, MD, pediatric endocrinologist and president of the Federation of Rare Childhood Diseases.

Indeed, there is not only a strict definition of intersexuality that involves a significant presence of these mixed physical characteristics in a way that conditions the self-image of the subject but also a broad definition, said Dr. Lala. “For example, clitoral hypertrophy in a female otherwise conforming to the female gender, which does not raise doubts about identity,” he said.
 

Chromosomes, Genes, and Hormones

A patient’s sexual characteristics are determined by the complex interaction of chromosomal, genetic, and hormonal factors. “The human body is, so to speak, programmed to take on female appearances in development and shifts toward male ones only if exposed to testosterone and other factors. For this to happen, testosterone must be produced during embryonic development, and it must function properly,” said Paolo Moghetti, full professor of endocrinology at the University of Verona, Italy.

The protein encoded by SRY, which is located on the Y chromosome, determines the development of the testicles from undifferentiated tissue of the embryonic gonads. The testicles of the embryo then produce testosterone. The absence of the Y chromosome is a common characteristic of most female individuals. However, there are individuals with a female phenotype who have X and Y chromosomes but lack SRY or have a variant of it that is not entirely functional.

Numerous other chromosomal or genetic variations can lead to alterations in sexual differentiation. “In phenotypically male adult subjects (with a chromosomal makeup of 46XY) with complete androgen insensitivity (so-called Morris syndrome), testosterone levels in the blood are elevated, above normal even for a male, but the hormone is totally ineffective, and the phenotype is totally female at birth, with completely female development of secondary sexual characteristics at puberty,” said Dr. Moghetti.

This means that affected individuals have well-developed breasts and a complete lack or extremely reduced presence of hair, including underarm and pubic hair. Menstruation is also completely absent because there is no uterus, and there are testes, not visible because they are considered in the abdomen.

“There are syndromes that are currently considered congenital but not genetic, of which a genetic origin will probably be identified in the future,” said Dr. Lala.

Some variations in sexual development can be diagnosed prenatally, such as an alteration of the number of sex chromosomes or a discordance between the morphologic characteristics highlighted by ultrasound and the genotype detected by amniocentesis. Some variations are evident at birth because of atypical anatomical characteristics. Others are diagnosed during puberty or later in adulthood, in the presence of infertility. The Italian National Institute of Health details these variations on its website, describing the characteristics that determine diagnosis and treatment.
 

Pathologies or Variations?

Some anomalies in sexual development negatively affect the patient’s physical health. One example is congenital adrenal hyperplasia. “It results from an inherited defect of the adrenal glands, which reduces cortisol production while increasing testosterone production,” said Dr. Lala. “In addition to the appearance of male characteristics in females, in more severe forms, it carries the risk of collapse and shock and requires pharmacological treatment.” It is undoubtedly a pathology.

Other variations in sexual characteristics do not affect the patient’s physical health negatively. They may, however, have a psychologic effect, sometimes a significant one, because of the lack of social acceptance of a person who cannot be classified within the binary classification of sexes.

“Conditions in which mixed male and female aspects are clearly evident have been and are still pathologized by the family, the treating physician, and society,” said Dr. Lala. “In the late 1970s, when a child was born with intersexual anatomical characteristics, it was common practice to surgically intervene, making them female, because it was technically easier.”

Over the years, patients who, as they grew up, were dissatisfied with the solution adopted at birth began to make their voices heard, Dr. Lala added. Scientific societies and international organizations have spoken out against subjecting intersexual newborns to surgical interventions that are not medically necessary. “Nowadays, decisions are made on a case-by-case basis, taking into account the families’ wishes. Interventions are justified with medical reasons, which are often very nuanced,” Dr. Lala concluded.
 

Implications for Sports

Traditionally, athletes participating in competitions in certain sports have been divided into male and female categories to ensure a certain equity and uniformity in performance. Over the years, the emergence of new information about sexual development has made it necessary to update the criteria used in this division.

The main factor responsible for the performance diversity between males and females is the action of testosterone on the male and female organism. “Testosterone has important effects on muscle mass and enhances training results,” said Dr. Moghetti. “As a demonstration of this fact, before puberty, the best performances in athletics or swimming by males and females are similar, then males gain a significant advantage of around 10%-20%.”

A few years ago, the World Athletics Federation conducted widespread screening of athletes participating in its world championships. “It identified a small group of individuals with potentially abnormal testosterone levels for the female sex,” said Dr. Moghetti. “Some were found to be doping, others had genetic defects, and for some, an interpretation was not even possible.”

Some of the individuals had a male genotype but a defect in 5-alpha-reductase, an enzyme essential for the formation of male genitals and hair growth. An athlete with these characteristics, assigned female sex at birth, has a male level of testosterone that stimulates the accumulation of muscle mass, Dr. Moghetti explained. Therefore, the individual has a considerable advantage in performances influenced by this hormone.

“In the end, the Federation decided to set limits on the testosterone levels of athletes participating in certain types of races, especially those in middle distance, that appeared to be more sensitive to differences in hormone levels,” said Dr. Moghetti. “The limitation does not apply to athletes with Morris syndrome, ie, with a male genotype and complete resistance to testosterone, for whom the high level of this hormone does not provide any advantage.” Given the complexity of the problem, he hopes for a case-by-case policy that considers the needs of patients with genetic alterations and those of athletes who have to compete with them.
 

Not the First Time

A recent incident underscored the difficulty of regulating such complex issues. The World Athletics Federation excluded South African middle-distance runner Caster Semenya from competitions years ago because of excessively high testosterone levels.

“The Federation’s regulations recommend that athletes in these cases reduce hormone levels to values below the threshold of 5 nmol/L of blood for a period of at least 6 months before the race by using hormonal contraceptives. The use of such drugs does not pose a health risk, as they are substances normally taken by women for contraception purposes,” said Amelia Filippelli, a pharmacologist at the University of Salerno in Italy. The South African middle-distance runner refused the drug and appealed to the Court of Arbitration for Sport and later to the Swiss Federal Court. Both rejected her appeal. Finally, Ms. Semenya appealed to the European Court of Human Rights, which in 2023 recognized a violation of her rights but does not have the authority to order a change in the Federation’s regulations.

Beyond the ideologic positions of nonexperts, therefore, the issue is still the subject of debate in the scientific community, which is evaluating not only its medical aspects but also its ethical implications.
 

This story was translated from Univadis Italy, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

The allegations against Algerian boxer Imane Khelif at the Paris Olympics raised the questions of intersexuality and its implications in competitive sports. This news organization has decided to delve into the topic to assist doctors who suspect a similar condition in their patients. No certain clinical data about Ms. Khelif have been made public, so this article does not concern the boxer but rather takes inspiration from the media controversy.

What Is Intersexuality?

Intersexuality encompasses a spectrum of variations in sexual development that lead to the simultaneous presence of typical male and female characteristics. As reiterated by the United Nations Office of the High Commissioner for Human Rights, the medical definition does not affect the patient’s self-identification of gender or sexual orientation.

“The percentage of people who fall within the intersexuality spectrum is less than 0.5 per thousand of the general population, but there are no precise statistics, given the difficulty of definition,” said Roberto Lala, MD, pediatric endocrinologist and president of the Federation of Rare Childhood Diseases.

Indeed, there is not only a strict definition of intersexuality that involves a significant presence of these mixed physical characteristics in a way that conditions the self-image of the subject but also a broad definition, said Dr. Lala. “For example, clitoral hypertrophy in a female otherwise conforming to the female gender, which does not raise doubts about identity,” he said.
 

Chromosomes, Genes, and Hormones

A patient’s sexual characteristics are determined by the complex interaction of chromosomal, genetic, and hormonal factors. “The human body is, so to speak, programmed to take on female appearances in development and shifts toward male ones only if exposed to testosterone and other factors. For this to happen, testosterone must be produced during embryonic development, and it must function properly,” said Paolo Moghetti, full professor of endocrinology at the University of Verona, Italy.

The protein encoded by SRY, which is located on the Y chromosome, determines the development of the testicles from undifferentiated tissue of the embryonic gonads. The testicles of the embryo then produce testosterone. The absence of the Y chromosome is a common characteristic of most female individuals. However, there are individuals with a female phenotype who have X and Y chromosomes but lack SRY or have a variant of it that is not entirely functional.

Numerous other chromosomal or genetic variations can lead to alterations in sexual differentiation. “In phenotypically male adult subjects (with a chromosomal makeup of 46XY) with complete androgen insensitivity (so-called Morris syndrome), testosterone levels in the blood are elevated, above normal even for a male, but the hormone is totally ineffective, and the phenotype is totally female at birth, with completely female development of secondary sexual characteristics at puberty,” said Dr. Moghetti.

This means that affected individuals have well-developed breasts and a complete lack or extremely reduced presence of hair, including underarm and pubic hair. Menstruation is also completely absent because there is no uterus, and there are testes, not visible because they are considered in the abdomen.

“There are syndromes that are currently considered congenital but not genetic, of which a genetic origin will probably be identified in the future,” said Dr. Lala.

Some variations in sexual development can be diagnosed prenatally, such as an alteration of the number of sex chromosomes or a discordance between the morphologic characteristics highlighted by ultrasound and the genotype detected by amniocentesis. Some variations are evident at birth because of atypical anatomical characteristics. Others are diagnosed during puberty or later in adulthood, in the presence of infertility. The Italian National Institute of Health details these variations on its website, describing the characteristics that determine diagnosis and treatment.
 

Pathologies or Variations?

Some anomalies in sexual development negatively affect the patient’s physical health. One example is congenital adrenal hyperplasia. “It results from an inherited defect of the adrenal glands, which reduces cortisol production while increasing testosterone production,” said Dr. Lala. “In addition to the appearance of male characteristics in females, in more severe forms, it carries the risk of collapse and shock and requires pharmacological treatment.” It is undoubtedly a pathology.

Other variations in sexual characteristics do not affect the patient’s physical health negatively. They may, however, have a psychologic effect, sometimes a significant one, because of the lack of social acceptance of a person who cannot be classified within the binary classification of sexes.

“Conditions in which mixed male and female aspects are clearly evident have been and are still pathologized by the family, the treating physician, and society,” said Dr. Lala. “In the late 1970s, when a child was born with intersexual anatomical characteristics, it was common practice to surgically intervene, making them female, because it was technically easier.”

Over the years, patients who, as they grew up, were dissatisfied with the solution adopted at birth began to make their voices heard, Dr. Lala added. Scientific societies and international organizations have spoken out against subjecting intersexual newborns to surgical interventions that are not medically necessary. “Nowadays, decisions are made on a case-by-case basis, taking into account the families’ wishes. Interventions are justified with medical reasons, which are often very nuanced,” Dr. Lala concluded.
 

Implications for Sports

Traditionally, athletes participating in competitions in certain sports have been divided into male and female categories to ensure a certain equity and uniformity in performance. Over the years, the emergence of new information about sexual development has made it necessary to update the criteria used in this division.

The main factor responsible for the performance diversity between males and females is the action of testosterone on the male and female organism. “Testosterone has important effects on muscle mass and enhances training results,” said Dr. Moghetti. “As a demonstration of this fact, before puberty, the best performances in athletics or swimming by males and females are similar, then males gain a significant advantage of around 10%-20%.”

A few years ago, the World Athletics Federation conducted widespread screening of athletes participating in its world championships. “It identified a small group of individuals with potentially abnormal testosterone levels for the female sex,” said Dr. Moghetti. “Some were found to be doping, others had genetic defects, and for some, an interpretation was not even possible.”

Some of the individuals had a male genotype but a defect in 5-alpha-reductase, an enzyme essential for the formation of male genitals and hair growth. An athlete with these characteristics, assigned female sex at birth, has a male level of testosterone that stimulates the accumulation of muscle mass, Dr. Moghetti explained. Therefore, the individual has a considerable advantage in performances influenced by this hormone.

“In the end, the Federation decided to set limits on the testosterone levels of athletes participating in certain types of races, especially those in middle distance, that appeared to be more sensitive to differences in hormone levels,” said Dr. Moghetti. “The limitation does not apply to athletes with Morris syndrome, ie, with a male genotype and complete resistance to testosterone, for whom the high level of this hormone does not provide any advantage.” Given the complexity of the problem, he hopes for a case-by-case policy that considers the needs of patients with genetic alterations and those of athletes who have to compete with them.
 

Not the First Time

A recent incident underscored the difficulty of regulating such complex issues. The World Athletics Federation excluded South African middle-distance runner Caster Semenya from competitions years ago because of excessively high testosterone levels.

“The Federation’s regulations recommend that athletes in these cases reduce hormone levels to values below the threshold of 5 nmol/L of blood for a period of at least 6 months before the race by using hormonal contraceptives. The use of such drugs does not pose a health risk, as they are substances normally taken by women for contraception purposes,” said Amelia Filippelli, a pharmacologist at the University of Salerno in Italy. The South African middle-distance runner refused the drug and appealed to the Court of Arbitration for Sport and later to the Swiss Federal Court. Both rejected her appeal. Finally, Ms. Semenya appealed to the European Court of Human Rights, which in 2023 recognized a violation of her rights but does not have the authority to order a change in the Federation’s regulations.

Beyond the ideologic positions of nonexperts, therefore, the issue is still the subject of debate in the scientific community, which is evaluating not only its medical aspects but also its ethical implications.
 

This story was translated from Univadis Italy, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

The allegations against Algerian boxer Imane Khelif at the Paris Olympics raised the questions of intersexuality and its implications in competitive sports. This news organization has decided to delve into the topic to assist doctors who suspect a similar condition in their patients. No certain clinical data about Ms. Khelif have been made public, so this article does not concern the boxer but rather takes inspiration from the media controversy.

What Is Intersexuality?

Intersexuality encompasses a spectrum of variations in sexual development that lead to the simultaneous presence of typical male and female characteristics. As reiterated by the United Nations Office of the High Commissioner for Human Rights, the medical definition does not affect the patient’s self-identification of gender or sexual orientation.

“The percentage of people who fall within the intersexuality spectrum is less than 0.5 per thousand of the general population, but there are no precise statistics, given the difficulty of definition,” said Roberto Lala, MD, pediatric endocrinologist and president of the Federation of Rare Childhood Diseases.

Indeed, there is not only a strict definition of intersexuality that involves a significant presence of these mixed physical characteristics in a way that conditions the self-image of the subject but also a broad definition, said Dr. Lala. “For example, clitoral hypertrophy in a female otherwise conforming to the female gender, which does not raise doubts about identity,” he said.
 

Chromosomes, Genes, and Hormones

A patient’s sexual characteristics are determined by the complex interaction of chromosomal, genetic, and hormonal factors. “The human body is, so to speak, programmed to take on female appearances in development and shifts toward male ones only if exposed to testosterone and other factors. For this to happen, testosterone must be produced during embryonic development, and it must function properly,” said Paolo Moghetti, full professor of endocrinology at the University of Verona, Italy.

The protein encoded by SRY, which is located on the Y chromosome, determines the development of the testicles from undifferentiated tissue of the embryonic gonads. The testicles of the embryo then produce testosterone. The absence of the Y chromosome is a common characteristic of most female individuals. However, there are individuals with a female phenotype who have X and Y chromosomes but lack SRY or have a variant of it that is not entirely functional.

Numerous other chromosomal or genetic variations can lead to alterations in sexual differentiation. “In phenotypically male adult subjects (with a chromosomal makeup of 46XY) with complete androgen insensitivity (so-called Morris syndrome), testosterone levels in the blood are elevated, above normal even for a male, but the hormone is totally ineffective, and the phenotype is totally female at birth, with completely female development of secondary sexual characteristics at puberty,” said Dr. Moghetti.

This means that affected individuals have well-developed breasts and a complete lack or extremely reduced presence of hair, including underarm and pubic hair. Menstruation is also completely absent because there is no uterus, and there are testes, not visible because they are considered in the abdomen.

“There are syndromes that are currently considered congenital but not genetic, of which a genetic origin will probably be identified in the future,” said Dr. Lala.

Some variations in sexual development can be diagnosed prenatally, such as an alteration of the number of sex chromosomes or a discordance between the morphologic characteristics highlighted by ultrasound and the genotype detected by amniocentesis. Some variations are evident at birth because of atypical anatomical characteristics. Others are diagnosed during puberty or later in adulthood, in the presence of infertility. The Italian National Institute of Health details these variations on its website, describing the characteristics that determine diagnosis and treatment.
 

Pathologies or Variations?

Some anomalies in sexual development negatively affect the patient’s physical health. One example is congenital adrenal hyperplasia. “It results from an inherited defect of the adrenal glands, which reduces cortisol production while increasing testosterone production,” said Dr. Lala. “In addition to the appearance of male characteristics in females, in more severe forms, it carries the risk of collapse and shock and requires pharmacological treatment.” It is undoubtedly a pathology.

Other variations in sexual characteristics do not affect the patient’s physical health negatively. They may, however, have a psychologic effect, sometimes a significant one, because of the lack of social acceptance of a person who cannot be classified within the binary classification of sexes.

“Conditions in which mixed male and female aspects are clearly evident have been and are still pathologized by the family, the treating physician, and society,” said Dr. Lala. “In the late 1970s, when a child was born with intersexual anatomical characteristics, it was common practice to surgically intervene, making them female, because it was technically easier.”

Over the years, patients who, as they grew up, were dissatisfied with the solution adopted at birth began to make their voices heard, Dr. Lala added. Scientific societies and international organizations have spoken out against subjecting intersexual newborns to surgical interventions that are not medically necessary. “Nowadays, decisions are made on a case-by-case basis, taking into account the families’ wishes. Interventions are justified with medical reasons, which are often very nuanced,” Dr. Lala concluded.
 

Implications for Sports

Traditionally, athletes participating in competitions in certain sports have been divided into male and female categories to ensure a certain equity and uniformity in performance. Over the years, the emergence of new information about sexual development has made it necessary to update the criteria used in this division.

The main factor responsible for the performance diversity between males and females is the action of testosterone on the male and female organism. “Testosterone has important effects on muscle mass and enhances training results,” said Dr. Moghetti. “As a demonstration of this fact, before puberty, the best performances in athletics or swimming by males and females are similar, then males gain a significant advantage of around 10%-20%.”

A few years ago, the World Athletics Federation conducted widespread screening of athletes participating in its world championships. “It identified a small group of individuals with potentially abnormal testosterone levels for the female sex,” said Dr. Moghetti. “Some were found to be doping, others had genetic defects, and for some, an interpretation was not even possible.”

Some of the individuals had a male genotype but a defect in 5-alpha-reductase, an enzyme essential for the formation of male genitals and hair growth. An athlete with these characteristics, assigned female sex at birth, has a male level of testosterone that stimulates the accumulation of muscle mass, Dr. Moghetti explained. Therefore, the individual has a considerable advantage in performances influenced by this hormone.

“In the end, the Federation decided to set limits on the testosterone levels of athletes participating in certain types of races, especially those in middle distance, that appeared to be more sensitive to differences in hormone levels,” said Dr. Moghetti. “The limitation does not apply to athletes with Morris syndrome, ie, with a male genotype and complete resistance to testosterone, for whom the high level of this hormone does not provide any advantage.” Given the complexity of the problem, he hopes for a case-by-case policy that considers the needs of patients with genetic alterations and those of athletes who have to compete with them.
 

Not the First Time

A recent incident underscored the difficulty of regulating such complex issues. The World Athletics Federation excluded South African middle-distance runner Caster Semenya from competitions years ago because of excessively high testosterone levels.

“The Federation’s regulations recommend that athletes in these cases reduce hormone levels to values below the threshold of 5 nmol/L of blood for a period of at least 6 months before the race by using hormonal contraceptives. The use of such drugs does not pose a health risk, as they are substances normally taken by women for contraception purposes,” said Amelia Filippelli, a pharmacologist at the University of Salerno in Italy. The South African middle-distance runner refused the drug and appealed to the Court of Arbitration for Sport and later to the Swiss Federal Court. Both rejected her appeal. Finally, Ms. Semenya appealed to the European Court of Human Rights, which in 2023 recognized a violation of her rights but does not have the authority to order a change in the Federation’s regulations.

Beyond the ideologic positions of nonexperts, therefore, the issue is still the subject of debate in the scientific community, which is evaluating not only its medical aspects but also its ethical implications.
 

This story was translated from Univadis Italy, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Since the COVID-19 pandemic, alcoholic hand sanitizers have become widely accessible nationwide. They can pose a problem, especially for emergency departments, when alcohol-dependent patients start drinking them. One example that demonstrates the challenge of diagnosing alcohol abuse is the medical history of a young man, as reported by Mahmoud El Hussein, MD, and colleagues from Hôpital Lariboisière in Paris, France.

Presentation and History

A 26-year-old man presented with severe abdominal pain at the emergency department. Upon arrival, he was hemodynamically stable but nervous and verbally aggressive at times. The patient reported no relevant preexisting conditions and was not taking any medications.

Findings

Upon initial physical examination, the patient had a soft, diffusely tender abdomen; tachycardia; and notably poor hygiene. The patient was afebrile. An ECG confirmed the tachycardia but showed no signs of ischemia. Blood work, except for slightly elevated liver values, did not reveal any abnormalities, particularly ruling out bleeding or kidney disease.

A urease rapid test to rule out kidney stones also showed no pathologic findings. In consultation with the surgical department, a CT scan of the abdomen was performed to rule out organ perforation, volvulus, or mesenteric ischemia. Only signs of fatty liver were found.
 

A Neighbor’s Tip

During all examinations, the patient’s abdomen was repeatedly palpated to promptly detect signs of an acute abdomen. However, there was never any defense tension at any point.

Intravenous analgesics and proton pump inhibitors (ie, paracetamol, phloroglucin, and pantoprazole) did not relieve the patient’s symptoms. Morphine was administered intravenously for sedation.

Only after a frustrating diagnostic process did a neighbor of the patient inform a nurse that he suspected the patient of stealing and consuming hand sanitizer. With the patient’s consent, a blood alcohol test was performed, revealing a blood alcohol concentration of 0.2% (2 g/L). A urine test, also conducted with the patient’s consent, tested positive for tetrahydrocannabinol. Additional tests showed the following results:

• Venous pH: 7.29 (normal, 7.32-7.38)
• Anion gap (mEq/L): 14 (normal, 3-9)
• Ketone bodies (mmol/L): 0.2 (normal, < 0.6)
• Calculated serum osmolality (mOsm/kg): 292 (normal, 285-295)
• Measured serum osmolality (mOsm/kg): 320 (normal, 285-295)
• Osmolality gap (mOsm/kg): 2 (normal, < 10)

The patient was informed of the test results and confessed to feigning abdominal pain. He was dependent on alcohol and experiencing withdrawal symptoms. The patient had stolen seven 475-mL bottles of hand sanitizer and consumed one and a half in the past 4-6 hours. According to the authors, the sanitizer consisted of 80% ethanol, 1.45% glycerol, and 0.13% hydrogen peroxide.
 

Discussion

In Germany, alcohol consumption results in approximately €57 billion in direct economic costs annually, according to data from the Federal Ministry of Health. In 2021, about 7.9 million people aged 18-64 years consumed alcohol in a risky manner (approximately 9.6% of the German population). About 9 million people (approximately 11%) were classified as alcoholics.

The authors of the case report pointed out that those in the advanced stages of alcohol addiction often consume any alcoholic liquid they can access. This includes alcoholic hand sanitizers commonly used in hospitals. Therefore, staff in emergency departments, where potential abusers encounter a wide range of potential abuse items, should exercise caution.

Although hand sanitizers are mainly composed of ethanol, they may also contain isopropanol, methanol, or acetone. Methanol poisoning can cause abdominal pain, visual disturbances, central nervous system damage, and death. Other alcohols such as ethylene glycol, commonly found in antifreeze, can distort blood values (lactate) and complicate a correct diagnosis.

Physicians working in emergency departments should proceed with caution when suspecting alcohol abuse. Questioning the patient’s environment and determining additional laboratory parameters (such as osmolality gap in the case report) can help unmask substance abuse if it is in doubt.

This story was translated from Univadis Germany, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Since the COVID-19 pandemic, alcoholic hand sanitizers have become widely accessible nationwide. They can pose a problem, especially for emergency departments, when alcohol-dependent patients start drinking them. One example that demonstrates the challenge of diagnosing alcohol abuse is the medical history of a young man, as reported by Mahmoud El Hussein, MD, and colleagues from Hôpital Lariboisière in Paris, France.

Presentation and History

A 26-year-old man presented with severe abdominal pain at the emergency department. Upon arrival, he was hemodynamically stable but nervous and verbally aggressive at times. The patient reported no relevant preexisting conditions and was not taking any medications.

Findings

Upon initial physical examination, the patient had a soft, diffusely tender abdomen; tachycardia; and notably poor hygiene. The patient was afebrile. An ECG confirmed the tachycardia but showed no signs of ischemia. Blood work, except for slightly elevated liver values, did not reveal any abnormalities, particularly ruling out bleeding or kidney disease.

A urease rapid test to rule out kidney stones also showed no pathologic findings. In consultation with the surgical department, a CT scan of the abdomen was performed to rule out organ perforation, volvulus, or mesenteric ischemia. Only signs of fatty liver were found.
 

A Neighbor’s Tip

During all examinations, the patient’s abdomen was repeatedly palpated to promptly detect signs of an acute abdomen. However, there was never any defense tension at any point.

Intravenous analgesics and proton pump inhibitors (ie, paracetamol, phloroglucin, and pantoprazole) did not relieve the patient’s symptoms. Morphine was administered intravenously for sedation.

Only after a frustrating diagnostic process did a neighbor of the patient inform a nurse that he suspected the patient of stealing and consuming hand sanitizer. With the patient’s consent, a blood alcohol test was performed, revealing a blood alcohol concentration of 0.2% (2 g/L). A urine test, also conducted with the patient’s consent, tested positive for tetrahydrocannabinol. Additional tests showed the following results:

• Venous pH: 7.29 (normal, 7.32-7.38)
• Anion gap (mEq/L): 14 (normal, 3-9)
• Ketone bodies (mmol/L): 0.2 (normal, < 0.6)
• Calculated serum osmolality (mOsm/kg): 292 (normal, 285-295)
• Measured serum osmolality (mOsm/kg): 320 (normal, 285-295)
• Osmolality gap (mOsm/kg): 2 (normal, < 10)

The patient was informed of the test results and confessed to feigning abdominal pain. He was dependent on alcohol and experiencing withdrawal symptoms. The patient had stolen seven 475-mL bottles of hand sanitizer and consumed one and a half in the past 4-6 hours. According to the authors, the sanitizer consisted of 80% ethanol, 1.45% glycerol, and 0.13% hydrogen peroxide.
 

Discussion

In Germany, alcohol consumption results in approximately €57 billion in direct economic costs annually, according to data from the Federal Ministry of Health. In 2021, about 7.9 million people aged 18-64 years consumed alcohol in a risky manner (approximately 9.6% of the German population). About 9 million people (approximately 11%) were classified as alcoholics.

The authors of the case report pointed out that those in the advanced stages of alcohol addiction often consume any alcoholic liquid they can access. This includes alcoholic hand sanitizers commonly used in hospitals. Therefore, staff in emergency departments, where potential abusers encounter a wide range of potential abuse items, should exercise caution.

Although hand sanitizers are mainly composed of ethanol, they may also contain isopropanol, methanol, or acetone. Methanol poisoning can cause abdominal pain, visual disturbances, central nervous system damage, and death. Other alcohols such as ethylene glycol, commonly found in antifreeze, can distort blood values (lactate) and complicate a correct diagnosis.

Physicians working in emergency departments should proceed with caution when suspecting alcohol abuse. Questioning the patient’s environment and determining additional laboratory parameters (such as osmolality gap in the case report) can help unmask substance abuse if it is in doubt.

This story was translated from Univadis Germany, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Since the COVID-19 pandemic, alcoholic hand sanitizers have become widely accessible nationwide. They can pose a problem, especially for emergency departments, when alcohol-dependent patients start drinking them. One example that demonstrates the challenge of diagnosing alcohol abuse is the medical history of a young man, as reported by Mahmoud El Hussein, MD, and colleagues from Hôpital Lariboisière in Paris, France.

Presentation and History

A 26-year-old man presented with severe abdominal pain at the emergency department. Upon arrival, he was hemodynamically stable but nervous and verbally aggressive at times. The patient reported no relevant preexisting conditions and was not taking any medications.

Findings

Upon initial physical examination, the patient had a soft, diffusely tender abdomen; tachycardia; and notably poor hygiene. The patient was afebrile. An ECG confirmed the tachycardia but showed no signs of ischemia. Blood work, except for slightly elevated liver values, did not reveal any abnormalities, particularly ruling out bleeding or kidney disease.

A urease rapid test to rule out kidney stones also showed no pathologic findings. In consultation with the surgical department, a CT scan of the abdomen was performed to rule out organ perforation, volvulus, or mesenteric ischemia. Only signs of fatty liver were found.
 

A Neighbor’s Tip

During all examinations, the patient’s abdomen was repeatedly palpated to promptly detect signs of an acute abdomen. However, there was never any defense tension at any point.

Intravenous analgesics and proton pump inhibitors (ie, paracetamol, phloroglucin, and pantoprazole) did not relieve the patient’s symptoms. Morphine was administered intravenously for sedation.

Only after a frustrating diagnostic process did a neighbor of the patient inform a nurse that he suspected the patient of stealing and consuming hand sanitizer. With the patient’s consent, a blood alcohol test was performed, revealing a blood alcohol concentration of 0.2% (2 g/L). A urine test, also conducted with the patient’s consent, tested positive for tetrahydrocannabinol. Additional tests showed the following results:

• Venous pH: 7.29 (normal, 7.32-7.38)
• Anion gap (mEq/L): 14 (normal, 3-9)
• Ketone bodies (mmol/L): 0.2 (normal, < 0.6)
• Calculated serum osmolality (mOsm/kg): 292 (normal, 285-295)
• Measured serum osmolality (mOsm/kg): 320 (normal, 285-295)
• Osmolality gap (mOsm/kg): 2 (normal, < 10)

The patient was informed of the test results and confessed to feigning abdominal pain. He was dependent on alcohol and experiencing withdrawal symptoms. The patient had stolen seven 475-mL bottles of hand sanitizer and consumed one and a half in the past 4-6 hours. According to the authors, the sanitizer consisted of 80% ethanol, 1.45% glycerol, and 0.13% hydrogen peroxide.
 

Discussion

In Germany, alcohol consumption results in approximately €57 billion in direct economic costs annually, according to data from the Federal Ministry of Health. In 2021, about 7.9 million people aged 18-64 years consumed alcohol in a risky manner (approximately 9.6% of the German population). About 9 million people (approximately 11%) were classified as alcoholics.

The authors of the case report pointed out that those in the advanced stages of alcohol addiction often consume any alcoholic liquid they can access. This includes alcoholic hand sanitizers commonly used in hospitals. Therefore, staff in emergency departments, where potential abusers encounter a wide range of potential abuse items, should exercise caution.

Although hand sanitizers are mainly composed of ethanol, they may also contain isopropanol, methanol, or acetone. Methanol poisoning can cause abdominal pain, visual disturbances, central nervous system damage, and death. Other alcohols such as ethylene glycol, commonly found in antifreeze, can distort blood values (lactate) and complicate a correct diagnosis.

Physicians working in emergency departments should proceed with caution when suspecting alcohol abuse. Questioning the patient’s environment and determining additional laboratory parameters (such as osmolality gap in the case report) can help unmask substance abuse if it is in doubt.

This story was translated from Univadis Germany, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

This transcript has been edited for clarity.

Robert A. Harrington, MD: I’m continuing my series of conversations with leaders in the field of cardiovascular medicine who are working on interesting projects and making contributions in the science and policy space. We have three guests joining us today who have recently written two papers in the Journal of the American College of Cardiology. One is an original research paper dealing with the issue of private equity’s acquisition of outpatient cardiology practices. And the second is an editorial that really tries to get at why this is happening. Is it a problem? Is it a solution to a problem?

Fortunately, I have all three as guests to think about this important issue that has implications for clinical care, reimbursement, physician wellness, and clinician wellness, and it has implications regarding public policy and how we should be thinking about the practice of medicine in this country.

Dr. Victoria L. Bartlett is an internal medicine resident at Brigham and Women’s Hospital in Boston, and a research fellow in the Smith Center at the Beth Israel Lahey medical center in Boston. Dr Rishi K. Wadhera is the senior author of the paper written by Dr. Bartlett. Dr. Rishi is associate professor of medicine at Harvard Medical School, and he is the associate director of the Smith Center at Beth Israel Lahey.

Rishi K. Wadhera, MD, MPP, MPhil: Thanks for having us, Bob.

Dr. Harrington: The editorialist, Dr. Ed Fry, is the national service line leader for cardiovascular medicine, for Ascension Health. Dr. Frey is a recent past president of the American College of Cardiology (ACC).

Edward T. A. Fry, MD: Great to be here. Thanks.
 

What is private equity? Why the interest in medicine?

Dr. Harrington: I was intrigued by the papers and it caused me to do a deeper dive into some of the earlier works that you have referenced about this growing topic of private equity making its way into medical practice. Rishi, I’ll start with you. For the casual reader like myself, what is the business of private equity?

Dr. Wadhera: Private equity firms basically used pooled investments from multiple sources. These can be individual and institutional investors, pension funds, endowments, and they use those funds to invest in private companies that have the potential to return a profit. Private equity firms typically try to add value to the company — or the case that we’re talking about today, the outpatient cardiology practices — within 3-7 years, and then subsequently tend to sell their stake in that entity or practice at a higher price than what they purchased it at. The goal really is to turn a profit for institutional investors over a shorter time horizon.

Dr. Harrington: How do they do that? I can understand, you buy a factory and you want to make the factory a little more efficient, and you think that perhaps, by combining some technologies, etc., that you might have in other factories, you can drive more value out of the one you just invested in in a short period of time. What’s the general business sense of how they’re going to do that in a cardiology practice? Is it all about making us more efficient?

Dr. Wadhera: Operational efficiency is the overarching theme here. One could argue that perhaps, private equity firms have the expertise to bring that kind of organizational know-how and operational efficiency to medicine. But there’s evidence that the way that private equity firms maximize their margin is maybe through mechanisms that aren’t necessarily good for patient care, such as reduced nursing staffing. When private equity acquires hospitals or practices in the same location, they have greater negotiating power at the payer table, to have higher prices for the services they deliver. There’s a lot of discussion about whether the sort of changes that private equity firms tend to implement are good or bad for patient care and also for clinicians.

Dr. Harrington: Great summary. Ed. Why is this happening in medicine? What did we do in medicine that made us ripe for investment by private equity? When you and I started out years ago, I don’t think we ever would have thought that this was in the future.

Dr. Fry: I think number one, as we know, is that medicine represents about 20% of our economy. There are huge amounts of money involved in these considerations. If players in this space can access even a small fraction of that money, it’s a lot of money and a lot of incentive for them.

In medicine in general, and then maybe more specifically, in cardiology, we’ve seen a shift away from private practice into employed practice. When people made those decisions over the past 10-15 years, there were certainly positives and risks that they took. I think for some, along the way, they realized that perhaps they gave up more than they thought in terms of control and running their own business and the opportunity to shape that themselves and be rewarded for that as they were in private practice. In cardiology, more specifically, we’ve seen this shift to the outpatient space: moving diagnostics and even therapeutics into ambulatory surgical centers and outpatient-based labs, and that is another potential source of revenue for these private equity companies.

As I wrote in the editorial, there are certainly a lot of pressures and frustrations that the day-to-day clinician feels, and maybe, this move to private equity is more of a symptom of those concerns and that this could be an opportunity to take the bull by the horns again in cardiology. We’ve evolved from a predominantly hospital-based acute care specialty into one of diagnosis, chronic disease management, and longitudinal care punctuated by diagnostics and therapeutics, which are, again, I think, attractive to private equity firms as potential sources for revenue.

Dr. Harrington: Ed, why cardiology? What’s happened over the years that has led to that appearance, if you will, of private equity and cardiovascular medicine?

Dr. Fry: Some of the earlier specialties were dermatology, ophthalmology, and gastroenterology, in particular. And interestingly, those tend to be specialties that have less chronic disease management and are more based on procedures and things like that. Within cardiology, obviously, the big driver is that our population is aging: 11,000 people turn 65 every day and become eligible for Medicare. With that, we see a rise in disease prevalence and then the rise in risk factors, obviously, with obesity and diabetes driving that, so there are more people who are going to have an illness that requires evaluation, diagnostics, and procedures. Because of that, it is a very target rich environment for private equity.

Dr. Harrington: That’s great background. Now, let’s dive into what you did, Victoria. What got you interested in the question? And give us some background on the literature that you were trying to build upon when you asked your series of questions.

Victoria L. Bartlett, MD: There’s been a lot of interest in private equity acquisitions and healthcare. A lot of the existing literature has been around hospital acquisitions and what happens there. There’s some literature, as you’ve mentioned, in outpatient practices, in certain specialties, where private equity has existed a little bit longer than in cardiology. They’ve been asking really similar questions to what we have been asking about cardiology, which is what happens when practices are acquired.

A kind of overview is that many of those studies have found increased costs to payers, to patients, and many have also found evidence of decreased quality. The evidence for the latter is honestly more difficult to figure out, but there has been evidence of decreased nursing ratios in nursing homes. There’s been evidence of changing the mix in clinics to more advanced practice providers than physicians. There’s been some evidence in hospitals that maybe quality doesn’t change too much. But the deeper layer under that is that these private equity–acquired hospitals may be selecting certain patients that are less sick, that are not going to negatively affect their metrics as much. That’s the environment that we had been reading about and starting to ask: Are we seeing that in cardiology too?

Dr. Harrington: Share with the audience what you did. You took what I would call a descriptive approach to try to understand the current landscape in cardiovascular medicine. As Ed already pointed out, a lot of the earlier data does not concern cardiology practices. My read of your paper is that you were trying to at least lay the groundwork for us to understand as a community what’s going on out there. Is that a fair interpretation?

Dr. Bartlett: Absolutely. Even that initial question of what’s happening is more challenging than it seems it might be to answer, partly because with private equity, these are private transactions. They don’t have to publicly report anything. So there’s a lot of manual work to gather these data. Our first questions were: What are these transactions? When are they happening? Where are they happening? What are the clinics that private equity is interested in? What are the community characteristics of those clinics? And what could that tell us about what’s going on?


 

Who Is Getting Acquired?

Dr. Harrington: Tell the audience broadly what you found. What are those clinics? And how often does this happen?

Dr. Bartlett: We looked at acquisitions between 2013 and 2023, and in that 10-year span, we found 41 acquisitions of outpatient cardiology practices, which corresponded to 342 acquisitions of clinics. The vast majority of these, pretty much 95%, occurred between 2021 and 2023. We calculated that about 3% of cardiology clinics in the US are owned by private equity. The states with the highest number of acquisitions were Florida, Texas, and Arizona, and particularly the urban areas in those states, ie, Jacksonville, Houston, Dallas. And interestingly, that mirrors what we’ve seen before in anesthesia and dermatology.

Our last question was around community characteristics, we looked at several that had a statistically significant association with private equity acquisition, and we found that private equity firms were less likely to acquire clinics in the highest poverty communities. Within the communities, we looked at the proportion of adults over 65, the proportion of racial and ethnic minorities, educational level, rurality, and didn’t find any significant associations between private equity acquisition and those characteristics.

Dr. Harrington: Thank you. Rishi, do you want to interpret why private equity was targeting certain areas?

Dr. Wadhera: Private equity goes where they can actually acquire practices. Those states, in particular, have more independent practices than, say, Massachusetts does. Then there’s the target population available in those states. Building on what Ed said earlier, why all of a sudden? Because Victoria just pointed out that the vast majority of these acquisitions happened between 2020 and 2023 and you see the surge, and I expect that surge to continue over the next several years. And the question is why?

We know with the rise in cardiometabolic risk factors at a population level, that the cardiovascular disease is only going to become more common. Cardiac procedures are very well reimbursed. There’s likely a lot of appeal in entering a specialty with a highly profitable service line. Over the past decade, federal policymakers very intentionally have created incentives to shift the delivery of cardiac procedures to nonhospital settings. We see that with the rise of ambulatory surgical centers and more cardiac procedures are being reimbursed in these types of settings. And I think that private equity firms may see this as an opportunity to maximize profits.

Victoria created this beautiful map in our study that showed how concentrated these acquisitions are. They really concentrated in specific markets. And I think that parallels what we’re seeing with health systems more broadly, this consolidation, and concentration is the ultimate goal. These different stakeholders, it’s not just private equity, have more market power, so that when they go to insurers, they can demand higher prices for procedures and services.

Dr. Harrington: It’s hard to look at the dates of 2021 or 2020 to 2023, and not wonder if there is a COVID effect. Victoria, do you think there’s a COVID effect, or is it just true, true, unrelated?

Dr. Bartlett: COVID definitely put a lot of financial pressure on providers, and particularly small independent practices. They would have felt that the most, and I certainly think is a piece of the picture but may not be all the picture.

Dr. Harrington: That’s what I would have guessed. We were all under financial pressures, but the small, independent practices didn’t have the big health system behind them to backstop things. Ed, as a former leader of the ACC, and the ACC very much works at the local level, are you hearing from the governors of these states that this is an issue, and not hearing from other states?

Dr. Fry: Certainly this activity is concentrated in the states that Victoria and Rishi described for the reasons that they outlined. This is still a very small number and probably will remain relatively small if we consider that 85% of cardiologists are employed, and the bar to exit an employment arrangement and enter into a private equity situation is pretty darn high. There’s a lot of costs associated with that. So it may have a finite cap to it, and that may be part of what buffers some of the response.

I would like to go back and address other reasons why this is happening. Particularly because of the aging population of cardiovascular patients, we’ve also seen the rise of Medicare Advantage, which is a type of value, if you consider it a type of value-based care. There are incentives built into Medicare Advantage to manage costs and to do various things so there is certainly a reward incentive. I am not wearing my hat as a representative of the ACC nor Ascension, and I will probably be a consumer of these services before I’m ever a participant, but I would say that private equity in some respects, is acting as a disruptor in this entire process. One of the positive outcomes from this is for a reevaluation of the role of clinicians in the overall delivery of care for health systems and academic medical centers. I think that can be a positive; I always try to look at the bright side of things too.
 

Patient and Clinician Satisfaction

Dr. Harrington: To your last comment. Ed, maybe I’ll ask you Rishi or Victoria, any insights into clinician wellness, how people feel when their practice has been bought by private equity? Are there any data out there?

Dr. Wadhera: Not that I know of. I will say that we have a study under review right now that doesn’t answer your question directly, Bob, but that looks at how private equity acquisitions of US hospitals affect the patient care experience. And what we found, using a rigorous, quasi experimental study design comparing private equity–acquired hospitals to neighboring control hospitals, is that private equity acquisition leads to a pretty marked decrease in patient care experience and satisfaction.

That’s capturing another dimension of quality that mortality and readmissions don’t necessarily reflect. It doesn’t answer your question directly, but I think an important area for future research is understanding the effects on the clinician experience as well as, most importantly, the patient experience.

Dr. Harrington: Nicely said, it seems like a good time to think about mixed qualitative methods such as focus groups, etc., coupled with the more quantitative research methods. Victoria, I suspect you talked to people in acquired practices. Any insight into whether it’s observational or rigorous data on the clinician experience?

Dr. Bartlett: Not that I have seen. I imagine it’s probably mixed because as we’ve been saying, there’s a lot of financial pressure on practices, small, independent practices, and it can become overwhelming to run them. Private equity firms offer a very attractive value proposition or can. But I think it’s a great point that should be highlighted.

Dr. Harrington: Ed, taking off your cardiovascular leadership hat, not representing any specific organization, what are the policy things that we should be thinking about?

Dr. Fry: There’s an opportunity to combine these conversations around research, collecting more data, and the advocacy issues related to that. One of the things that perhaps differentiates cardiology in this space from other specialties, or subspecialties, surgical subspecialties, is the plethora of data that we already have with well-established registry tools. We have good benchmarks. From a professional society standpoint, we have an obligation to make sure that the care that is provided in whatever environment meets the standards and is measurable, reportable, and provides a level of consumerism to patients and payers to be able to look at that. I think we have an obligation to advocate for the use of well-validated registry tools to track the data, to have objective data, to be able to demonstrate outcomes.

Interestingly, there’s an ACC/American Heart Association policy document from 2020 on professionalism and ethics in cardiology. And it calls for the obligation of the profession to make sure that in alternative sites of care, that we are achieving at least as good a result, if not better. We have to be true to that.

Dr. Harrington: I was actually a coauthor on that paper on professionalism and talking about some of the research and education issues within the academic medical centers. You’re spot on. And I love the comment about the importance of long-standing registries, whether maintained by the ACC, the Heart Association, or the Society of Thoracic Surgeons, where we can get insights into the quality issues.

We need more work done on the patient experience, the clinician experience, but I also take the positive, Ed, that this may be a disruptor that could lend itself to some positive change in other areas that need to change.

This has been a fantastic conversation on the appearance, if you will, of private equity in cardiovascular medicine and some of the observations made by colleagues at the Smith Center at the Beth Israel Lahey, with great commentary by Ed Fry on whether this is a symptom or a solution and what we should be thinking about from a broader societal perspective. I want to thank my three guests today, Victoria, Ed, and Rishi, for joining us here.


Dr. Harrington is the Stephen and Suzanne Weiss Dean of Weill Cornell Medicine and provost for medical affairs of Cornell University, as well as a former president of the American Heart Association. He disclosed ties with several companies. Dr. Bartlett is resident physician, Department of Internal Medicine, Brigham & women’s Hospital, Boston, and has disclosed no relevant financial relationships. Dr. Fry is chair, Ascension National Cardiovascular Service Line, Ascension St. Vincent Heart Center in Indianapolis, Indiana. Dr. Wadhera is associate professor, Harvard Medical School, and associate director, Richard A. and Susan F. Smith Center for Outcomes Research, Beth Israel Deaconess Medical Center, both in Boston. Dr. Wadhera disclosed ties with Abbott, ChamberCardio, CVS Health, the National Institutes of Health, American Heart Association, and the Donaghue Foundation.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Robert A. Harrington, MD: I’m continuing my series of conversations with leaders in the field of cardiovascular medicine who are working on interesting projects and making contributions in the science and policy space. We have three guests joining us today who have recently written two papers in the Journal of the American College of Cardiology. One is an original research paper dealing with the issue of private equity’s acquisition of outpatient cardiology practices. And the second is an editorial that really tries to get at why this is happening. Is it a problem? Is it a solution to a problem?

Fortunately, I have all three as guests to think about this important issue that has implications for clinical care, reimbursement, physician wellness, and clinician wellness, and it has implications regarding public policy and how we should be thinking about the practice of medicine in this country.

Dr. Victoria L. Bartlett is an internal medicine resident at Brigham and Women’s Hospital in Boston, and a research fellow in the Smith Center at the Beth Israel Lahey medical center in Boston. Dr Rishi K. Wadhera is the senior author of the paper written by Dr. Bartlett. Dr. Rishi is associate professor of medicine at Harvard Medical School, and he is the associate director of the Smith Center at Beth Israel Lahey.

Rishi K. Wadhera, MD, MPP, MPhil: Thanks for having us, Bob.

Dr. Harrington: The editorialist, Dr. Ed Fry, is the national service line leader for cardiovascular medicine, for Ascension Health. Dr. Frey is a recent past president of the American College of Cardiology (ACC).

Edward T. A. Fry, MD: Great to be here. Thanks.
 

What is private equity? Why the interest in medicine?

Dr. Harrington: I was intrigued by the papers and it caused me to do a deeper dive into some of the earlier works that you have referenced about this growing topic of private equity making its way into medical practice. Rishi, I’ll start with you. For the casual reader like myself, what is the business of private equity?

Dr. Wadhera: Private equity firms basically used pooled investments from multiple sources. These can be individual and institutional investors, pension funds, endowments, and they use those funds to invest in private companies that have the potential to return a profit. Private equity firms typically try to add value to the company — or the case that we’re talking about today, the outpatient cardiology practices — within 3-7 years, and then subsequently tend to sell their stake in that entity or practice at a higher price than what they purchased it at. The goal really is to turn a profit for institutional investors over a shorter time horizon.

Dr. Harrington: How do they do that? I can understand, you buy a factory and you want to make the factory a little more efficient, and you think that perhaps, by combining some technologies, etc., that you might have in other factories, you can drive more value out of the one you just invested in in a short period of time. What’s the general business sense of how they’re going to do that in a cardiology practice? Is it all about making us more efficient?

Dr. Wadhera: Operational efficiency is the overarching theme here. One could argue that perhaps, private equity firms have the expertise to bring that kind of organizational know-how and operational efficiency to medicine. But there’s evidence that the way that private equity firms maximize their margin is maybe through mechanisms that aren’t necessarily good for patient care, such as reduced nursing staffing. When private equity acquires hospitals or practices in the same location, they have greater negotiating power at the payer table, to have higher prices for the services they deliver. There’s a lot of discussion about whether the sort of changes that private equity firms tend to implement are good or bad for patient care and also for clinicians.

Dr. Harrington: Great summary. Ed. Why is this happening in medicine? What did we do in medicine that made us ripe for investment by private equity? When you and I started out years ago, I don’t think we ever would have thought that this was in the future.

Dr. Fry: I think number one, as we know, is that medicine represents about 20% of our economy. There are huge amounts of money involved in these considerations. If players in this space can access even a small fraction of that money, it’s a lot of money and a lot of incentive for them.

In medicine in general, and then maybe more specifically, in cardiology, we’ve seen a shift away from private practice into employed practice. When people made those decisions over the past 10-15 years, there were certainly positives and risks that they took. I think for some, along the way, they realized that perhaps they gave up more than they thought in terms of control and running their own business and the opportunity to shape that themselves and be rewarded for that as they were in private practice. In cardiology, more specifically, we’ve seen this shift to the outpatient space: moving diagnostics and even therapeutics into ambulatory surgical centers and outpatient-based labs, and that is another potential source of revenue for these private equity companies.

As I wrote in the editorial, there are certainly a lot of pressures and frustrations that the day-to-day clinician feels, and maybe, this move to private equity is more of a symptom of those concerns and that this could be an opportunity to take the bull by the horns again in cardiology. We’ve evolved from a predominantly hospital-based acute care specialty into one of diagnosis, chronic disease management, and longitudinal care punctuated by diagnostics and therapeutics, which are, again, I think, attractive to private equity firms as potential sources for revenue.

Dr. Harrington: Ed, why cardiology? What’s happened over the years that has led to that appearance, if you will, of private equity and cardiovascular medicine?

Dr. Fry: Some of the earlier specialties were dermatology, ophthalmology, and gastroenterology, in particular. And interestingly, those tend to be specialties that have less chronic disease management and are more based on procedures and things like that. Within cardiology, obviously, the big driver is that our population is aging: 11,000 people turn 65 every day and become eligible for Medicare. With that, we see a rise in disease prevalence and then the rise in risk factors, obviously, with obesity and diabetes driving that, so there are more people who are going to have an illness that requires evaluation, diagnostics, and procedures. Because of that, it is a very target rich environment for private equity.

Dr. Harrington: That’s great background. Now, let’s dive into what you did, Victoria. What got you interested in the question? And give us some background on the literature that you were trying to build upon when you asked your series of questions.

Victoria L. Bartlett, MD: There’s been a lot of interest in private equity acquisitions and healthcare. A lot of the existing literature has been around hospital acquisitions and what happens there. There’s some literature, as you’ve mentioned, in outpatient practices, in certain specialties, where private equity has existed a little bit longer than in cardiology. They’ve been asking really similar questions to what we have been asking about cardiology, which is what happens when practices are acquired.

A kind of overview is that many of those studies have found increased costs to payers, to patients, and many have also found evidence of decreased quality. The evidence for the latter is honestly more difficult to figure out, but there has been evidence of decreased nursing ratios in nursing homes. There’s been evidence of changing the mix in clinics to more advanced practice providers than physicians. There’s been some evidence in hospitals that maybe quality doesn’t change too much. But the deeper layer under that is that these private equity–acquired hospitals may be selecting certain patients that are less sick, that are not going to negatively affect their metrics as much. That’s the environment that we had been reading about and starting to ask: Are we seeing that in cardiology too?

Dr. Harrington: Share with the audience what you did. You took what I would call a descriptive approach to try to understand the current landscape in cardiovascular medicine. As Ed already pointed out, a lot of the earlier data does not concern cardiology practices. My read of your paper is that you were trying to at least lay the groundwork for us to understand as a community what’s going on out there. Is that a fair interpretation?

Dr. Bartlett: Absolutely. Even that initial question of what’s happening is more challenging than it seems it might be to answer, partly because with private equity, these are private transactions. They don’t have to publicly report anything. So there’s a lot of manual work to gather these data. Our first questions were: What are these transactions? When are they happening? Where are they happening? What are the clinics that private equity is interested in? What are the community characteristics of those clinics? And what could that tell us about what’s going on?


 

Who Is Getting Acquired?

Dr. Harrington: Tell the audience broadly what you found. What are those clinics? And how often does this happen?

Dr. Bartlett: We looked at acquisitions between 2013 and 2023, and in that 10-year span, we found 41 acquisitions of outpatient cardiology practices, which corresponded to 342 acquisitions of clinics. The vast majority of these, pretty much 95%, occurred between 2021 and 2023. We calculated that about 3% of cardiology clinics in the US are owned by private equity. The states with the highest number of acquisitions were Florida, Texas, and Arizona, and particularly the urban areas in those states, ie, Jacksonville, Houston, Dallas. And interestingly, that mirrors what we’ve seen before in anesthesia and dermatology.

Our last question was around community characteristics, we looked at several that had a statistically significant association with private equity acquisition, and we found that private equity firms were less likely to acquire clinics in the highest poverty communities. Within the communities, we looked at the proportion of adults over 65, the proportion of racial and ethnic minorities, educational level, rurality, and didn’t find any significant associations between private equity acquisition and those characteristics.

Dr. Harrington: Thank you. Rishi, do you want to interpret why private equity was targeting certain areas?

Dr. Wadhera: Private equity goes where they can actually acquire practices. Those states, in particular, have more independent practices than, say, Massachusetts does. Then there’s the target population available in those states. Building on what Ed said earlier, why all of a sudden? Because Victoria just pointed out that the vast majority of these acquisitions happened between 2020 and 2023 and you see the surge, and I expect that surge to continue over the next several years. And the question is why?

We know with the rise in cardiometabolic risk factors at a population level, that the cardiovascular disease is only going to become more common. Cardiac procedures are very well reimbursed. There’s likely a lot of appeal in entering a specialty with a highly profitable service line. Over the past decade, federal policymakers very intentionally have created incentives to shift the delivery of cardiac procedures to nonhospital settings. We see that with the rise of ambulatory surgical centers and more cardiac procedures are being reimbursed in these types of settings. And I think that private equity firms may see this as an opportunity to maximize profits.

Victoria created this beautiful map in our study that showed how concentrated these acquisitions are. They really concentrated in specific markets. And I think that parallels what we’re seeing with health systems more broadly, this consolidation, and concentration is the ultimate goal. These different stakeholders, it’s not just private equity, have more market power, so that when they go to insurers, they can demand higher prices for procedures and services.

Dr. Harrington: It’s hard to look at the dates of 2021 or 2020 to 2023, and not wonder if there is a COVID effect. Victoria, do you think there’s a COVID effect, or is it just true, true, unrelated?

Dr. Bartlett: COVID definitely put a lot of financial pressure on providers, and particularly small independent practices. They would have felt that the most, and I certainly think is a piece of the picture but may not be all the picture.

Dr. Harrington: That’s what I would have guessed. We were all under financial pressures, but the small, independent practices didn’t have the big health system behind them to backstop things. Ed, as a former leader of the ACC, and the ACC very much works at the local level, are you hearing from the governors of these states that this is an issue, and not hearing from other states?

Dr. Fry: Certainly this activity is concentrated in the states that Victoria and Rishi described for the reasons that they outlined. This is still a very small number and probably will remain relatively small if we consider that 85% of cardiologists are employed, and the bar to exit an employment arrangement and enter into a private equity situation is pretty darn high. There’s a lot of costs associated with that. So it may have a finite cap to it, and that may be part of what buffers some of the response.

I would like to go back and address other reasons why this is happening. Particularly because of the aging population of cardiovascular patients, we’ve also seen the rise of Medicare Advantage, which is a type of value, if you consider it a type of value-based care. There are incentives built into Medicare Advantage to manage costs and to do various things so there is certainly a reward incentive. I am not wearing my hat as a representative of the ACC nor Ascension, and I will probably be a consumer of these services before I’m ever a participant, but I would say that private equity in some respects, is acting as a disruptor in this entire process. One of the positive outcomes from this is for a reevaluation of the role of clinicians in the overall delivery of care for health systems and academic medical centers. I think that can be a positive; I always try to look at the bright side of things too.
 

Patient and Clinician Satisfaction

Dr. Harrington: To your last comment. Ed, maybe I’ll ask you Rishi or Victoria, any insights into clinician wellness, how people feel when their practice has been bought by private equity? Are there any data out there?

Dr. Wadhera: Not that I know of. I will say that we have a study under review right now that doesn’t answer your question directly, Bob, but that looks at how private equity acquisitions of US hospitals affect the patient care experience. And what we found, using a rigorous, quasi experimental study design comparing private equity–acquired hospitals to neighboring control hospitals, is that private equity acquisition leads to a pretty marked decrease in patient care experience and satisfaction.

That’s capturing another dimension of quality that mortality and readmissions don’t necessarily reflect. It doesn’t answer your question directly, but I think an important area for future research is understanding the effects on the clinician experience as well as, most importantly, the patient experience.

Dr. Harrington: Nicely said, it seems like a good time to think about mixed qualitative methods such as focus groups, etc., coupled with the more quantitative research methods. Victoria, I suspect you talked to people in acquired practices. Any insight into whether it’s observational or rigorous data on the clinician experience?

Dr. Bartlett: Not that I have seen. I imagine it’s probably mixed because as we’ve been saying, there’s a lot of financial pressure on practices, small, independent practices, and it can become overwhelming to run them. Private equity firms offer a very attractive value proposition or can. But I think it’s a great point that should be highlighted.

Dr. Harrington: Ed, taking off your cardiovascular leadership hat, not representing any specific organization, what are the policy things that we should be thinking about?

Dr. Fry: There’s an opportunity to combine these conversations around research, collecting more data, and the advocacy issues related to that. One of the things that perhaps differentiates cardiology in this space from other specialties, or subspecialties, surgical subspecialties, is the plethora of data that we already have with well-established registry tools. We have good benchmarks. From a professional society standpoint, we have an obligation to make sure that the care that is provided in whatever environment meets the standards and is measurable, reportable, and provides a level of consumerism to patients and payers to be able to look at that. I think we have an obligation to advocate for the use of well-validated registry tools to track the data, to have objective data, to be able to demonstrate outcomes.

Interestingly, there’s an ACC/American Heart Association policy document from 2020 on professionalism and ethics in cardiology. And it calls for the obligation of the profession to make sure that in alternative sites of care, that we are achieving at least as good a result, if not better. We have to be true to that.

Dr. Harrington: I was actually a coauthor on that paper on professionalism and talking about some of the research and education issues within the academic medical centers. You’re spot on. And I love the comment about the importance of long-standing registries, whether maintained by the ACC, the Heart Association, or the Society of Thoracic Surgeons, where we can get insights into the quality issues.

We need more work done on the patient experience, the clinician experience, but I also take the positive, Ed, that this may be a disruptor that could lend itself to some positive change in other areas that need to change.

This has been a fantastic conversation on the appearance, if you will, of private equity in cardiovascular medicine and some of the observations made by colleagues at the Smith Center at the Beth Israel Lahey, with great commentary by Ed Fry on whether this is a symptom or a solution and what we should be thinking about from a broader societal perspective. I want to thank my three guests today, Victoria, Ed, and Rishi, for joining us here.


Dr. Harrington is the Stephen and Suzanne Weiss Dean of Weill Cornell Medicine and provost for medical affairs of Cornell University, as well as a former president of the American Heart Association. He disclosed ties with several companies. Dr. Bartlett is resident physician, Department of Internal Medicine, Brigham & women’s Hospital, Boston, and has disclosed no relevant financial relationships. Dr. Fry is chair, Ascension National Cardiovascular Service Line, Ascension St. Vincent Heart Center in Indianapolis, Indiana. Dr. Wadhera is associate professor, Harvard Medical School, and associate director, Richard A. and Susan F. Smith Center for Outcomes Research, Beth Israel Deaconess Medical Center, both in Boston. Dr. Wadhera disclosed ties with Abbott, ChamberCardio, CVS Health, the National Institutes of Health, American Heart Association, and the Donaghue Foundation.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Robert A. Harrington, MD: I’m continuing my series of conversations with leaders in the field of cardiovascular medicine who are working on interesting projects and making contributions in the science and policy space. We have three guests joining us today who have recently written two papers in the Journal of the American College of Cardiology. One is an original research paper dealing with the issue of private equity’s acquisition of outpatient cardiology practices. And the second is an editorial that really tries to get at why this is happening. Is it a problem? Is it a solution to a problem?

Fortunately, I have all three as guests to think about this important issue that has implications for clinical care, reimbursement, physician wellness, and clinician wellness, and it has implications regarding public policy and how we should be thinking about the practice of medicine in this country.

Dr. Victoria L. Bartlett is an internal medicine resident at Brigham and Women’s Hospital in Boston, and a research fellow in the Smith Center at the Beth Israel Lahey medical center in Boston. Dr Rishi K. Wadhera is the senior author of the paper written by Dr. Bartlett. Dr. Rishi is associate professor of medicine at Harvard Medical School, and he is the associate director of the Smith Center at Beth Israel Lahey.

Rishi K. Wadhera, MD, MPP, MPhil: Thanks for having us, Bob.

Dr. Harrington: The editorialist, Dr. Ed Fry, is the national service line leader for cardiovascular medicine, for Ascension Health. Dr. Frey is a recent past president of the American College of Cardiology (ACC).

Edward T. A. Fry, MD: Great to be here. Thanks.
 

What is private equity? Why the interest in medicine?

Dr. Harrington: I was intrigued by the papers and it caused me to do a deeper dive into some of the earlier works that you have referenced about this growing topic of private equity making its way into medical practice. Rishi, I’ll start with you. For the casual reader like myself, what is the business of private equity?

Dr. Wadhera: Private equity firms basically used pooled investments from multiple sources. These can be individual and institutional investors, pension funds, endowments, and they use those funds to invest in private companies that have the potential to return a profit. Private equity firms typically try to add value to the company — or the case that we’re talking about today, the outpatient cardiology practices — within 3-7 years, and then subsequently tend to sell their stake in that entity or practice at a higher price than what they purchased it at. The goal really is to turn a profit for institutional investors over a shorter time horizon.

Dr. Harrington: How do they do that? I can understand, you buy a factory and you want to make the factory a little more efficient, and you think that perhaps, by combining some technologies, etc., that you might have in other factories, you can drive more value out of the one you just invested in in a short period of time. What’s the general business sense of how they’re going to do that in a cardiology practice? Is it all about making us more efficient?

Dr. Wadhera: Operational efficiency is the overarching theme here. One could argue that perhaps, private equity firms have the expertise to bring that kind of organizational know-how and operational efficiency to medicine. But there’s evidence that the way that private equity firms maximize their margin is maybe through mechanisms that aren’t necessarily good for patient care, such as reduced nursing staffing. When private equity acquires hospitals or practices in the same location, they have greater negotiating power at the payer table, to have higher prices for the services they deliver. There’s a lot of discussion about whether the sort of changes that private equity firms tend to implement are good or bad for patient care and also for clinicians.

Dr. Harrington: Great summary. Ed. Why is this happening in medicine? What did we do in medicine that made us ripe for investment by private equity? When you and I started out years ago, I don’t think we ever would have thought that this was in the future.

Dr. Fry: I think number one, as we know, is that medicine represents about 20% of our economy. There are huge amounts of money involved in these considerations. If players in this space can access even a small fraction of that money, it’s a lot of money and a lot of incentive for them.

In medicine in general, and then maybe more specifically, in cardiology, we’ve seen a shift away from private practice into employed practice. When people made those decisions over the past 10-15 years, there were certainly positives and risks that they took. I think for some, along the way, they realized that perhaps they gave up more than they thought in terms of control and running their own business and the opportunity to shape that themselves and be rewarded for that as they were in private practice. In cardiology, more specifically, we’ve seen this shift to the outpatient space: moving diagnostics and even therapeutics into ambulatory surgical centers and outpatient-based labs, and that is another potential source of revenue for these private equity companies.

As I wrote in the editorial, there are certainly a lot of pressures and frustrations that the day-to-day clinician feels, and maybe, this move to private equity is more of a symptom of those concerns and that this could be an opportunity to take the bull by the horns again in cardiology. We’ve evolved from a predominantly hospital-based acute care specialty into one of diagnosis, chronic disease management, and longitudinal care punctuated by diagnostics and therapeutics, which are, again, I think, attractive to private equity firms as potential sources for revenue.

Dr. Harrington: Ed, why cardiology? What’s happened over the years that has led to that appearance, if you will, of private equity and cardiovascular medicine?

Dr. Fry: Some of the earlier specialties were dermatology, ophthalmology, and gastroenterology, in particular. And interestingly, those tend to be specialties that have less chronic disease management and are more based on procedures and things like that. Within cardiology, obviously, the big driver is that our population is aging: 11,000 people turn 65 every day and become eligible for Medicare. With that, we see a rise in disease prevalence and then the rise in risk factors, obviously, with obesity and diabetes driving that, so there are more people who are going to have an illness that requires evaluation, diagnostics, and procedures. Because of that, it is a very target rich environment for private equity.

Dr. Harrington: That’s great background. Now, let’s dive into what you did, Victoria. What got you interested in the question? And give us some background on the literature that you were trying to build upon when you asked your series of questions.

Victoria L. Bartlett, MD: There’s been a lot of interest in private equity acquisitions and healthcare. A lot of the existing literature has been around hospital acquisitions and what happens there. There’s some literature, as you’ve mentioned, in outpatient practices, in certain specialties, where private equity has existed a little bit longer than in cardiology. They’ve been asking really similar questions to what we have been asking about cardiology, which is what happens when practices are acquired.

A kind of overview is that many of those studies have found increased costs to payers, to patients, and many have also found evidence of decreased quality. The evidence for the latter is honestly more difficult to figure out, but there has been evidence of decreased nursing ratios in nursing homes. There’s been evidence of changing the mix in clinics to more advanced practice providers than physicians. There’s been some evidence in hospitals that maybe quality doesn’t change too much. But the deeper layer under that is that these private equity–acquired hospitals may be selecting certain patients that are less sick, that are not going to negatively affect their metrics as much. That’s the environment that we had been reading about and starting to ask: Are we seeing that in cardiology too?

Dr. Harrington: Share with the audience what you did. You took what I would call a descriptive approach to try to understand the current landscape in cardiovascular medicine. As Ed already pointed out, a lot of the earlier data does not concern cardiology practices. My read of your paper is that you were trying to at least lay the groundwork for us to understand as a community what’s going on out there. Is that a fair interpretation?

Dr. Bartlett: Absolutely. Even that initial question of what’s happening is more challenging than it seems it might be to answer, partly because with private equity, these are private transactions. They don’t have to publicly report anything. So there’s a lot of manual work to gather these data. Our first questions were: What are these transactions? When are they happening? Where are they happening? What are the clinics that private equity is interested in? What are the community characteristics of those clinics? And what could that tell us about what’s going on?


 

Who Is Getting Acquired?

Dr. Harrington: Tell the audience broadly what you found. What are those clinics? And how often does this happen?

Dr. Bartlett: We looked at acquisitions between 2013 and 2023, and in that 10-year span, we found 41 acquisitions of outpatient cardiology practices, which corresponded to 342 acquisitions of clinics. The vast majority of these, pretty much 95%, occurred between 2021 and 2023. We calculated that about 3% of cardiology clinics in the US are owned by private equity. The states with the highest number of acquisitions were Florida, Texas, and Arizona, and particularly the urban areas in those states, ie, Jacksonville, Houston, Dallas. And interestingly, that mirrors what we’ve seen before in anesthesia and dermatology.

Our last question was around community characteristics, we looked at several that had a statistically significant association with private equity acquisition, and we found that private equity firms were less likely to acquire clinics in the highest poverty communities. Within the communities, we looked at the proportion of adults over 65, the proportion of racial and ethnic minorities, educational level, rurality, and didn’t find any significant associations between private equity acquisition and those characteristics.

Dr. Harrington: Thank you. Rishi, do you want to interpret why private equity was targeting certain areas?

Dr. Wadhera: Private equity goes where they can actually acquire practices. Those states, in particular, have more independent practices than, say, Massachusetts does. Then there’s the target population available in those states. Building on what Ed said earlier, why all of a sudden? Because Victoria just pointed out that the vast majority of these acquisitions happened between 2020 and 2023 and you see the surge, and I expect that surge to continue over the next several years. And the question is why?

We know with the rise in cardiometabolic risk factors at a population level, that the cardiovascular disease is only going to become more common. Cardiac procedures are very well reimbursed. There’s likely a lot of appeal in entering a specialty with a highly profitable service line. Over the past decade, federal policymakers very intentionally have created incentives to shift the delivery of cardiac procedures to nonhospital settings. We see that with the rise of ambulatory surgical centers and more cardiac procedures are being reimbursed in these types of settings. And I think that private equity firms may see this as an opportunity to maximize profits.

Victoria created this beautiful map in our study that showed how concentrated these acquisitions are. They really concentrated in specific markets. And I think that parallels what we’re seeing with health systems more broadly, this consolidation, and concentration is the ultimate goal. These different stakeholders, it’s not just private equity, have more market power, so that when they go to insurers, they can demand higher prices for procedures and services.

Dr. Harrington: It’s hard to look at the dates of 2021 or 2020 to 2023, and not wonder if there is a COVID effect. Victoria, do you think there’s a COVID effect, or is it just true, true, unrelated?

Dr. Bartlett: COVID definitely put a lot of financial pressure on providers, and particularly small independent practices. They would have felt that the most, and I certainly think is a piece of the picture but may not be all the picture.

Dr. Harrington: That’s what I would have guessed. We were all under financial pressures, but the small, independent practices didn’t have the big health system behind them to backstop things. Ed, as a former leader of the ACC, and the ACC very much works at the local level, are you hearing from the governors of these states that this is an issue, and not hearing from other states?

Dr. Fry: Certainly this activity is concentrated in the states that Victoria and Rishi described for the reasons that they outlined. This is still a very small number and probably will remain relatively small if we consider that 85% of cardiologists are employed, and the bar to exit an employment arrangement and enter into a private equity situation is pretty darn high. There’s a lot of costs associated with that. So it may have a finite cap to it, and that may be part of what buffers some of the response.

I would like to go back and address other reasons why this is happening. Particularly because of the aging population of cardiovascular patients, we’ve also seen the rise of Medicare Advantage, which is a type of value, if you consider it a type of value-based care. There are incentives built into Medicare Advantage to manage costs and to do various things so there is certainly a reward incentive. I am not wearing my hat as a representative of the ACC nor Ascension, and I will probably be a consumer of these services before I’m ever a participant, but I would say that private equity in some respects, is acting as a disruptor in this entire process. One of the positive outcomes from this is for a reevaluation of the role of clinicians in the overall delivery of care for health systems and academic medical centers. I think that can be a positive; I always try to look at the bright side of things too.
 

Patient and Clinician Satisfaction

Dr. Harrington: To your last comment. Ed, maybe I’ll ask you Rishi or Victoria, any insights into clinician wellness, how people feel when their practice has been bought by private equity? Are there any data out there?

Dr. Wadhera: Not that I know of. I will say that we have a study under review right now that doesn’t answer your question directly, Bob, but that looks at how private equity acquisitions of US hospitals affect the patient care experience. And what we found, using a rigorous, quasi experimental study design comparing private equity–acquired hospitals to neighboring control hospitals, is that private equity acquisition leads to a pretty marked decrease in patient care experience and satisfaction.

That’s capturing another dimension of quality that mortality and readmissions don’t necessarily reflect. It doesn’t answer your question directly, but I think an important area for future research is understanding the effects on the clinician experience as well as, most importantly, the patient experience.

Dr. Harrington: Nicely said, it seems like a good time to think about mixed qualitative methods such as focus groups, etc., coupled with the more quantitative research methods. Victoria, I suspect you talked to people in acquired practices. Any insight into whether it’s observational or rigorous data on the clinician experience?

Dr. Bartlett: Not that I have seen. I imagine it’s probably mixed because as we’ve been saying, there’s a lot of financial pressure on practices, small, independent practices, and it can become overwhelming to run them. Private equity firms offer a very attractive value proposition or can. But I think it’s a great point that should be highlighted.

Dr. Harrington: Ed, taking off your cardiovascular leadership hat, not representing any specific organization, what are the policy things that we should be thinking about?

Dr. Fry: There’s an opportunity to combine these conversations around research, collecting more data, and the advocacy issues related to that. One of the things that perhaps differentiates cardiology in this space from other specialties, or subspecialties, surgical subspecialties, is the plethora of data that we already have with well-established registry tools. We have good benchmarks. From a professional society standpoint, we have an obligation to make sure that the care that is provided in whatever environment meets the standards and is measurable, reportable, and provides a level of consumerism to patients and payers to be able to look at that. I think we have an obligation to advocate for the use of well-validated registry tools to track the data, to have objective data, to be able to demonstrate outcomes.

Interestingly, there’s an ACC/American Heart Association policy document from 2020 on professionalism and ethics in cardiology. And it calls for the obligation of the profession to make sure that in alternative sites of care, that we are achieving at least as good a result, if not better. We have to be true to that.

Dr. Harrington: I was actually a coauthor on that paper on professionalism and talking about some of the research and education issues within the academic medical centers. You’re spot on. And I love the comment about the importance of long-standing registries, whether maintained by the ACC, the Heart Association, or the Society of Thoracic Surgeons, where we can get insights into the quality issues.

We need more work done on the patient experience, the clinician experience, but I also take the positive, Ed, that this may be a disruptor that could lend itself to some positive change in other areas that need to change.

This has been a fantastic conversation on the appearance, if you will, of private equity in cardiovascular medicine and some of the observations made by colleagues at the Smith Center at the Beth Israel Lahey, with great commentary by Ed Fry on whether this is a symptom or a solution and what we should be thinking about from a broader societal perspective. I want to thank my three guests today, Victoria, Ed, and Rishi, for joining us here.


Dr. Harrington is the Stephen and Suzanne Weiss Dean of Weill Cornell Medicine and provost for medical affairs of Cornell University, as well as a former president of the American Heart Association. He disclosed ties with several companies. Dr. Bartlett is resident physician, Department of Internal Medicine, Brigham & women’s Hospital, Boston, and has disclosed no relevant financial relationships. Dr. Fry is chair, Ascension National Cardiovascular Service Line, Ascension St. Vincent Heart Center in Indianapolis, Indiana. Dr. Wadhera is associate professor, Harvard Medical School, and associate director, Richard A. and Susan F. Smith Center for Outcomes Research, Beth Israel Deaconess Medical Center, both in Boston. Dr. Wadhera disclosed ties with Abbott, ChamberCardio, CVS Health, the National Institutes of Health, American Heart Association, and the Donaghue Foundation.

A version of this article first appeared on Medscape.com.

TORONTO — Reading a storybook about embracing differences can reduce anxiety and boost self-esteem in children with a visible skin condition, the early results of an ongoing study suggested.

So far, “the study demonstrates that these books have value to patients and families,” one of the study authors, Sonia Havele, MD, a pediatrician and dermatology resident at Children’s Mercy Hospital Kansas City, Kansas City, Missouri, said in an interview.

“There are tools to help kids cope with their skin conditions, but we’re underutilizing them,” she added. “And part of the reason we’re underutilizing storybooks is that we just don’t know what’s out there.” For the study, the researchers received funding to purchase 18 “creative and thoughtful” storybooks related to pediatric skin conditions, reviewed by at least two pediatric dermatologists before being selected, which are just a sample of related books that are available.

The study results were presented as a poster at the annual meeting of the Society for Pediatric Dermatology.

Children with visible skin conditions, which can include port-wine stains, capillary malformations, and congenital moles, may be subjected to teasing or bullying at school, and the conditions can also affect their quality of life.
 

Beauty and the Birthmark

The books include one titled “Beauty with a Birthmark” and another, “My Hair Went on Vacation.” An illustrated book, “Just Ask: Be Different, Be Brave, Be You,” by US Supreme Court Justice Sonia Sotomayor, offers tips on how to answer common questions about someone’s appearance.

Dr. Havele said that Justice Sotomayor’s book “empowers kids, their siblings, their classmates ... to ask questions, and it teaches patients not to be afraid of those questions, and to really lean into educating their peers, and their family members.”

“Kids are really just curious,” she added. “They’ll make comments like: ‘Hey, what’s that spot on your face?’ Or, they’ll ask about vitiligo because they’ve never seen somebody with it before.”

To evaluate the psychosocial impact of these types of books for children with visible skin conditions, Dr. Havele and colleagues designed a study that includes patients aged 2-12 years dealing with issues related to self-esteem, acceptance, coping, or bullying. Parents are provided with a relevant storybook to read at home with their child in a “safe and comfortable space” and “at their own pace and their own time,” said Dr. Havele.

Inside the book is a QR code to access the validated Children’s Dermatology Life Quality Index (CDLQI). Families complete the survey at baseline and provide feedback after reading the book. Researchers collect information about demographics, age, gender, and skin conditions, which included atopic dermatitis, alopecia areata, vitiligo, hemangioma, and port-wine stain.

The response rate so far is 34%, and close to 80 parents have completed the survey with their child, Dr. Havele said.

At baseline, many of the children were either moderately or severely affected in terms of their quality of life (45% scored ≥ 6 on the CDLQI).

After reading the book, about 80% of parents reported it had a positive impact, and about 20% said it had a somewhat positive impact on their child’s self-image or confidence. Almost 80% agreed, and the remainder somewhat agreed it encouraged their child to embrace differences.

Most respondents also said the book helped the parent and child cope with the child’s condition. “So really, it was overall a positive response,” said Dr. Havele. “We are able to demonstrate that these books have value in a more scientific or objective way.”

This may not be surprising. Dr. Havele referred to more formal bibliotherapy (book therapy), which has been studied in other pediatric populations, including patients with cancer and those who have experienced trauma.
 

Awesome Space

Pediatric dermatologists are perfectly positioned to play a role in improving the lives of their patients with skin issues. “We see the impact of visible skin disease on children all the time,” said Dr. Havele. “The dermatology visit is an awesome space and opportunity to introduce these books to families and potentially help them talk about the skin condition with their child.”

In addition to prescribing therapies, “we’re also with these kids through an emotional journey, and I think giving them tools for that emotional journey is very helpful,” she added.

Such books would have been a great help to Dr. Havele herself. Growing up, she had severe atopic dermatitis covering much of her body. “Having such a resource would have helped me better cope with my reality of being different than everyone else.”

She hopes a database will be established to house these resources so other providers can refer patients to the list of books. Other books include “The Itchy-saurus: The Dino with an itch that can’t be scratched,” “Hair in My Brush,” and “I am Unique!”

Dr. Havele had no relevant disclosures.

A version of this article first appeared on Medscape.com.

TORONTO — Reading a storybook about embracing differences can reduce anxiety and boost self-esteem in children with a visible skin condition, the early results of an ongoing study suggested.

So far, “the study demonstrates that these books have value to patients and families,” one of the study authors, Sonia Havele, MD, a pediatrician and dermatology resident at Children’s Mercy Hospital Kansas City, Kansas City, Missouri, said in an interview.

“There are tools to help kids cope with their skin conditions, but we’re underutilizing them,” she added. “And part of the reason we’re underutilizing storybooks is that we just don’t know what’s out there.” For the study, the researchers received funding to purchase 18 “creative and thoughtful” storybooks related to pediatric skin conditions, reviewed by at least two pediatric dermatologists before being selected, which are just a sample of related books that are available.

The study results were presented as a poster at the annual meeting of the Society for Pediatric Dermatology.

Children with visible skin conditions, which can include port-wine stains, capillary malformations, and congenital moles, may be subjected to teasing or bullying at school, and the conditions can also affect their quality of life.
 

Beauty and the Birthmark

The books include one titled “Beauty with a Birthmark” and another, “My Hair Went on Vacation.” An illustrated book, “Just Ask: Be Different, Be Brave, Be You,” by US Supreme Court Justice Sonia Sotomayor, offers tips on how to answer common questions about someone’s appearance.

Dr. Havele said that Justice Sotomayor’s book “empowers kids, their siblings, their classmates ... to ask questions, and it teaches patients not to be afraid of those questions, and to really lean into educating their peers, and their family members.”

“Kids are really just curious,” she added. “They’ll make comments like: ‘Hey, what’s that spot on your face?’ Or, they’ll ask about vitiligo because they’ve never seen somebody with it before.”

To evaluate the psychosocial impact of these types of books for children with visible skin conditions, Dr. Havele and colleagues designed a study that includes patients aged 2-12 years dealing with issues related to self-esteem, acceptance, coping, or bullying. Parents are provided with a relevant storybook to read at home with their child in a “safe and comfortable space” and “at their own pace and their own time,” said Dr. Havele.

Inside the book is a QR code to access the validated Children’s Dermatology Life Quality Index (CDLQI). Families complete the survey at baseline and provide feedback after reading the book. Researchers collect information about demographics, age, gender, and skin conditions, which included atopic dermatitis, alopecia areata, vitiligo, hemangioma, and port-wine stain.

The response rate so far is 34%, and close to 80 parents have completed the survey with their child, Dr. Havele said.

At baseline, many of the children were either moderately or severely affected in terms of their quality of life (45% scored ≥ 6 on the CDLQI).

After reading the book, about 80% of parents reported it had a positive impact, and about 20% said it had a somewhat positive impact on their child’s self-image or confidence. Almost 80% agreed, and the remainder somewhat agreed it encouraged their child to embrace differences.

Most respondents also said the book helped the parent and child cope with the child’s condition. “So really, it was overall a positive response,” said Dr. Havele. “We are able to demonstrate that these books have value in a more scientific or objective way.”

This may not be surprising. Dr. Havele referred to more formal bibliotherapy (book therapy), which has been studied in other pediatric populations, including patients with cancer and those who have experienced trauma.
 

Awesome Space

Pediatric dermatologists are perfectly positioned to play a role in improving the lives of their patients with skin issues. “We see the impact of visible skin disease on children all the time,” said Dr. Havele. “The dermatology visit is an awesome space and opportunity to introduce these books to families and potentially help them talk about the skin condition with their child.”

In addition to prescribing therapies, “we’re also with these kids through an emotional journey, and I think giving them tools for that emotional journey is very helpful,” she added.

Such books would have been a great help to Dr. Havele herself. Growing up, she had severe atopic dermatitis covering much of her body. “Having such a resource would have helped me better cope with my reality of being different than everyone else.”

She hopes a database will be established to house these resources so other providers can refer patients to the list of books. Other books include “The Itchy-saurus: The Dino with an itch that can’t be scratched,” “Hair in My Brush,” and “I am Unique!”

Dr. Havele had no relevant disclosures.

A version of this article first appeared on Medscape.com.

TORONTO — Reading a storybook about embracing differences can reduce anxiety and boost self-esteem in children with a visible skin condition, the early results of an ongoing study suggested.

So far, “the study demonstrates that these books have value to patients and families,” one of the study authors, Sonia Havele, MD, a pediatrician and dermatology resident at Children’s Mercy Hospital Kansas City, Kansas City, Missouri, said in an interview.

“There are tools to help kids cope with their skin conditions, but we’re underutilizing them,” she added. “And part of the reason we’re underutilizing storybooks is that we just don’t know what’s out there.” For the study, the researchers received funding to purchase 18 “creative and thoughtful” storybooks related to pediatric skin conditions, reviewed by at least two pediatric dermatologists before being selected, which are just a sample of related books that are available.

The study results were presented as a poster at the annual meeting of the Society for Pediatric Dermatology.

Children with visible skin conditions, which can include port-wine stains, capillary malformations, and congenital moles, may be subjected to teasing or bullying at school, and the conditions can also affect their quality of life.
 

Beauty and the Birthmark

The books include one titled “Beauty with a Birthmark” and another, “My Hair Went on Vacation.” An illustrated book, “Just Ask: Be Different, Be Brave, Be You,” by US Supreme Court Justice Sonia Sotomayor, offers tips on how to answer common questions about someone’s appearance.

Dr. Havele said that Justice Sotomayor’s book “empowers kids, their siblings, their classmates ... to ask questions, and it teaches patients not to be afraid of those questions, and to really lean into educating their peers, and their family members.”

“Kids are really just curious,” she added. “They’ll make comments like: ‘Hey, what’s that spot on your face?’ Or, they’ll ask about vitiligo because they’ve never seen somebody with it before.”

To evaluate the psychosocial impact of these types of books for children with visible skin conditions, Dr. Havele and colleagues designed a study that includes patients aged 2-12 years dealing with issues related to self-esteem, acceptance, coping, or bullying. Parents are provided with a relevant storybook to read at home with their child in a “safe and comfortable space” and “at their own pace and their own time,” said Dr. Havele.

Inside the book is a QR code to access the validated Children’s Dermatology Life Quality Index (CDLQI). Families complete the survey at baseline and provide feedback after reading the book. Researchers collect information about demographics, age, gender, and skin conditions, which included atopic dermatitis, alopecia areata, vitiligo, hemangioma, and port-wine stain.

The response rate so far is 34%, and close to 80 parents have completed the survey with their child, Dr. Havele said.

At baseline, many of the children were either moderately or severely affected in terms of their quality of life (45% scored ≥ 6 on the CDLQI).

After reading the book, about 80% of parents reported it had a positive impact, and about 20% said it had a somewhat positive impact on their child’s self-image or confidence. Almost 80% agreed, and the remainder somewhat agreed it encouraged their child to embrace differences.

Most respondents also said the book helped the parent and child cope with the child’s condition. “So really, it was overall a positive response,” said Dr. Havele. “We are able to demonstrate that these books have value in a more scientific or objective way.”

This may not be surprising. Dr. Havele referred to more formal bibliotherapy (book therapy), which has been studied in other pediatric populations, including patients with cancer and those who have experienced trauma.
 

Awesome Space

Pediatric dermatologists are perfectly positioned to play a role in improving the lives of their patients with skin issues. “We see the impact of visible skin disease on children all the time,” said Dr. Havele. “The dermatology visit is an awesome space and opportunity to introduce these books to families and potentially help them talk about the skin condition with their child.”

In addition to prescribing therapies, “we’re also with these kids through an emotional journey, and I think giving them tools for that emotional journey is very helpful,” she added.

Such books would have been a great help to Dr. Havele herself. Growing up, she had severe atopic dermatitis covering much of her body. “Having such a resource would have helped me better cope with my reality of being different than everyone else.”

She hopes a database will be established to house these resources so other providers can refer patients to the list of books. Other books include “The Itchy-saurus: The Dino with an itch that can’t be scratched,” “Hair in My Brush,” and “I am Unique!”

Dr. Havele had no relevant disclosures.

A version of this article first appeared on Medscape.com.