A new vaccine to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults has been approved by the Food and Drug Administration. 

The injectable drug, Capvaxive (Pneumococcal 21-valent Conjugate Vaccine), protects against 22 serotypes that cause invasive pneumococcal disease in adults, the company said in a news release. These strains account for about 84% of invasive pneumococcal disease cases among adults aged 50 years or older and about 85% of these cases in adults aged 65 years or older. 

The drug company said about 150,000 adults in the United States are hospitalized annually because of pneumococcal pneumonia. 

“Many cases of adult disease are caused by serotypes not included in other approved pneumococcal conjugate vaccines,” Walter Orenstein, MD, a professor emeritus of medicine, epidemiology, global health, and pediatrics at Emory University, Atlanta, Georgia, and a member of Merck’s Scientific Advisory Committee, said in the release. “CAPVAXIVE is designed to include the serotypes that cause the majority of invasive pneumococcal disease in adults, helping to protect adults against invasive pneumococcal disease and pneumococcal pneumonia.”

A draft agenda shows a Centers for Disease Control and Prevention (CDC) advisory panel will meet on June 27 to discuss the vaccine. If the committee votes to approve Capvaxive, the CDC director will decide whether to make it available across the country.

Testing showed that Capvaxive was well tolerated by people it was tested on, with the main reports being pain where they got the shot, fatigue, headaches, and muscle aches, Merck said.

The eight unique serotypes included in CAPVAXIVE will protect against invasive pneumococcal disease and pneumococcal pneumonia, not just pneumonia. 

According to Reuters, Merck said Capvaxive has a wholesale acquisition price of $287 per dose, but most people will probably have access to it at no cost if the drug receives a routine CDC recommendation. Capvaxive’s main competition is expected to be Pfizer’s shot, Prevnar 20, which was approved in 2021 for use in adults aged 18 years or older, Reuters reported.

A version of this article appeared on Medscape.com.

A new vaccine to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults has been approved by the Food and Drug Administration. 

The injectable drug, Capvaxive (Pneumococcal 21-valent Conjugate Vaccine), protects against 22 serotypes that cause invasive pneumococcal disease in adults, the company said in a news release. These strains account for about 84% of invasive pneumococcal disease cases among adults aged 50 years or older and about 85% of these cases in adults aged 65 years or older. 

The drug company said about 150,000 adults in the United States are hospitalized annually because of pneumococcal pneumonia. 

“Many cases of adult disease are caused by serotypes not included in other approved pneumococcal conjugate vaccines,” Walter Orenstein, MD, a professor emeritus of medicine, epidemiology, global health, and pediatrics at Emory University, Atlanta, Georgia, and a member of Merck’s Scientific Advisory Committee, said in the release. “CAPVAXIVE is designed to include the serotypes that cause the majority of invasive pneumococcal disease in adults, helping to protect adults against invasive pneumococcal disease and pneumococcal pneumonia.”

A draft agenda shows a Centers for Disease Control and Prevention (CDC) advisory panel will meet on June 27 to discuss the vaccine. If the committee votes to approve Capvaxive, the CDC director will decide whether to make it available across the country.

Testing showed that Capvaxive was well tolerated by people it was tested on, with the main reports being pain where they got the shot, fatigue, headaches, and muscle aches, Merck said.

The eight unique serotypes included in CAPVAXIVE will protect against invasive pneumococcal disease and pneumococcal pneumonia, not just pneumonia. 

According to Reuters, Merck said Capvaxive has a wholesale acquisition price of $287 per dose, but most people will probably have access to it at no cost if the drug receives a routine CDC recommendation. Capvaxive’s main competition is expected to be Pfizer’s shot, Prevnar 20, which was approved in 2021 for use in adults aged 18 years or older, Reuters reported.

A version of this article appeared on Medscape.com.

A new vaccine to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults has been approved by the Food and Drug Administration. 

The injectable drug, Capvaxive (Pneumococcal 21-valent Conjugate Vaccine), protects against 22 serotypes that cause invasive pneumococcal disease in adults, the company said in a news release. These strains account for about 84% of invasive pneumococcal disease cases among adults aged 50 years or older and about 85% of these cases in adults aged 65 years or older. 

The drug company said about 150,000 adults in the United States are hospitalized annually because of pneumococcal pneumonia. 

“Many cases of adult disease are caused by serotypes not included in other approved pneumococcal conjugate vaccines,” Walter Orenstein, MD, a professor emeritus of medicine, epidemiology, global health, and pediatrics at Emory University, Atlanta, Georgia, and a member of Merck’s Scientific Advisory Committee, said in the release. “CAPVAXIVE is designed to include the serotypes that cause the majority of invasive pneumococcal disease in adults, helping to protect adults against invasive pneumococcal disease and pneumococcal pneumonia.”

A draft agenda shows a Centers for Disease Control and Prevention (CDC) advisory panel will meet on June 27 to discuss the vaccine. If the committee votes to approve Capvaxive, the CDC director will decide whether to make it available across the country.

Testing showed that Capvaxive was well tolerated by people it was tested on, with the main reports being pain where they got the shot, fatigue, headaches, and muscle aches, Merck said.

The eight unique serotypes included in CAPVAXIVE will protect against invasive pneumococcal disease and pneumococcal pneumonia, not just pneumonia. 

According to Reuters, Merck said Capvaxive has a wholesale acquisition price of $287 per dose, but most people will probably have access to it at no cost if the drug receives a routine CDC recommendation. Capvaxive’s main competition is expected to be Pfizer’s shot, Prevnar 20, which was approved in 2021 for use in adults aged 18 years or older, Reuters reported.

A version of this article appeared on Medscape.com.

SAN DIEGO — Nearly one in three patients with migraine are reluctant to seek medical help and many blame healthcare providers, results of a recent survey showed.

Participants cited concerns that their complaints would be dismissed, a belief that healthcare providers could offer no additional help, and a prior unsuccessful clinician visit as reasons for not seeking care. Survey respondents saw an average of four clinicians before finally receiving a diagnosis.

“I was shocked that a third of patients were reluctant to seek care,” said study investigator Elizabeth K. Seng, PhD, associate professor, Ferkauf Graduate School of Psychology, Yeshiva University, and research associate professor, department of neurology, Albert Einstein College of Medicine, both in New York City. “That just shows a much higher level of medical distress than I expected from this community of people who are obviously suffering from this significant neurologic disease.”

The findings were presented at the annual meeting of the American Headache Society.
 

‘Significant Disease’

The study included 500 adults with migraine (mean age, 40 years) who signed up for a patient support group sponsored by Eli Lilly and completed a comprehensive survey. Respondents were mostly female, White, non-Hispanic, and well-educated individuals.

Half of participants had episodic migraines, and half had chronic migraines; 46% reported experiencing anxiety and 33% reported depression.

Almost all respondents had initiated treatment with a first calcitonin gene-related peptide (CGRP) monoclonal antibody.

“These are people who have significant enough disease that eventually they needed our top-tier preventive medication,” Dr. Seng said.

Participants answered a variety of questions pertaining to disease factors and treatment seeking. Just over 70% said they suspected they had migraine prior to diagnosis, “which means for almost 30%, it was a surprise when they received the diagnosis,” said Dr. Seng. 

Nearly 40% reported that a relative first suggested they may have migraine, and 33% suspected it themselves. Only 17.4% said a healthcare provider suggested they may have the condition.

Almost a third of respondents (30.5%) reported they were reluctant to seek medical help.

“Some said they didn’t think their physician could do anything more than they were already doing for themselves, or that they’d be taken seriously, or they had had talked to doctors before and this wasn’t helpful,” said Dr. Seng. 

These responses speak to the need for better public health messaging, she said. “People have this idea that migraine attacks aren’t a big deal when, in fact, these attacks area big deal and certainly deserve treatment.” 

Family and friends were participants’ most common source of information on migraine, followed by the Internet. “This highlights the importance of getting migraine-related information out there so that when people talk to their friends and family, they’re receiving accurate information,” said Dr. Seng.

When asked about the path to a diagnosis, respondents reported consulting an average of four providers before receiving an accurate diagnosis. “That’s pretty remarkable,” Dr. Seng said.

An increase in frequency or severity of migraine attacks or attacks that interfered with work or school “pushed people over the threshold to seek care,” Dr. Seng said. 

A subset of patients was asked about the factors they believed could help with migraine attacks. Of these, 80% cited diet and 70% stress reduction. Supplements, exercise, and relaxation techniques were cited much less frequently, said Dr. Seng. 

The mean age of respondents’ migraine diagnosis was 26 years, so there was about 18 years from the time of diagnosis to participation in the survey, which could introduce recall bias. Other potential limitations included the fact that the survey had no open-ended questions, and men and ethnic minorities were underrepresented. 
 

Useful Data

Commenting on the study findings, Nina Riggins, MD, PhD, president, Brain Performance Center and Research Institute, and director of the Headache Center at The Neuron Clinic, San Diego, California, said the survey findings are “very useful” and highlight “significant opportunities for improvement in migraine education for clinicians and people living with migraine disease.”

The fact that participants reported consulting an average of four healthcare providers before receiving an accurate diagnosis underscores the importance of providing clinicians with tools to identify migraine, she said.

This is especially relevant as new migraine therapies that may improve efficacy and have fewer side effects become available, she added. 

“It would be interesting to see in future studies if migraine recognition by non-headache specialists improved after CGRP-blocking medications for migraine management became available,” said Dr. Riggins, who is cochair of the AHS First Contact program which is aimed at improving headache management in primary care.

She added that she and her colleagues will keep these survey results in mind when creating future educational materials for clinicians.

The study was supported by Eli Lily. Dr. Seng is a consultant for GlaxoSmithKline, Theranica, and Abbvie, and receives research support from the National Institutes of Health, National Center for Complementary and Integrative Health, National Institute of Neurological Disorders and Stroke, Veterans Health Administration, Cystic Fibrosis Foundation, and the American Heart Association. Dr. Riggins reported no relevant conflicts.

A version of this article appeared on Medscape.com.

SAN DIEGO — Nearly one in three patients with migraine are reluctant to seek medical help and many blame healthcare providers, results of a recent survey showed.

Participants cited concerns that their complaints would be dismissed, a belief that healthcare providers could offer no additional help, and a prior unsuccessful clinician visit as reasons for not seeking care. Survey respondents saw an average of four clinicians before finally receiving a diagnosis.

“I was shocked that a third of patients were reluctant to seek care,” said study investigator Elizabeth K. Seng, PhD, associate professor, Ferkauf Graduate School of Psychology, Yeshiva University, and research associate professor, department of neurology, Albert Einstein College of Medicine, both in New York City. “That just shows a much higher level of medical distress than I expected from this community of people who are obviously suffering from this significant neurologic disease.”

The findings were presented at the annual meeting of the American Headache Society.
 

‘Significant Disease’

The study included 500 adults with migraine (mean age, 40 years) who signed up for a patient support group sponsored by Eli Lilly and completed a comprehensive survey. Respondents were mostly female, White, non-Hispanic, and well-educated individuals.

Half of participants had episodic migraines, and half had chronic migraines; 46% reported experiencing anxiety and 33% reported depression.

Almost all respondents had initiated treatment with a first calcitonin gene-related peptide (CGRP) monoclonal antibody.

“These are people who have significant enough disease that eventually they needed our top-tier preventive medication,” Dr. Seng said.

Participants answered a variety of questions pertaining to disease factors and treatment seeking. Just over 70% said they suspected they had migraine prior to diagnosis, “which means for almost 30%, it was a surprise when they received the diagnosis,” said Dr. Seng. 

Nearly 40% reported that a relative first suggested they may have migraine, and 33% suspected it themselves. Only 17.4% said a healthcare provider suggested they may have the condition.

Almost a third of respondents (30.5%) reported they were reluctant to seek medical help.

“Some said they didn’t think their physician could do anything more than they were already doing for themselves, or that they’d be taken seriously, or they had had talked to doctors before and this wasn’t helpful,” said Dr. Seng. 

These responses speak to the need for better public health messaging, she said. “People have this idea that migraine attacks aren’t a big deal when, in fact, these attacks area big deal and certainly deserve treatment.” 

Family and friends were participants’ most common source of information on migraine, followed by the Internet. “This highlights the importance of getting migraine-related information out there so that when people talk to their friends and family, they’re receiving accurate information,” said Dr. Seng.

When asked about the path to a diagnosis, respondents reported consulting an average of four providers before receiving an accurate diagnosis. “That’s pretty remarkable,” Dr. Seng said.

An increase in frequency or severity of migraine attacks or attacks that interfered with work or school “pushed people over the threshold to seek care,” Dr. Seng said. 

A subset of patients was asked about the factors they believed could help with migraine attacks. Of these, 80% cited diet and 70% stress reduction. Supplements, exercise, and relaxation techniques were cited much less frequently, said Dr. Seng. 

The mean age of respondents’ migraine diagnosis was 26 years, so there was about 18 years from the time of diagnosis to participation in the survey, which could introduce recall bias. Other potential limitations included the fact that the survey had no open-ended questions, and men and ethnic minorities were underrepresented. 
 

Useful Data

Commenting on the study findings, Nina Riggins, MD, PhD, president, Brain Performance Center and Research Institute, and director of the Headache Center at The Neuron Clinic, San Diego, California, said the survey findings are “very useful” and highlight “significant opportunities for improvement in migraine education for clinicians and people living with migraine disease.”

The fact that participants reported consulting an average of four healthcare providers before receiving an accurate diagnosis underscores the importance of providing clinicians with tools to identify migraine, she said.

This is especially relevant as new migraine therapies that may improve efficacy and have fewer side effects become available, she added. 

“It would be interesting to see in future studies if migraine recognition by non-headache specialists improved after CGRP-blocking medications for migraine management became available,” said Dr. Riggins, who is cochair of the AHS First Contact program which is aimed at improving headache management in primary care.

She added that she and her colleagues will keep these survey results in mind when creating future educational materials for clinicians.

The study was supported by Eli Lily. Dr. Seng is a consultant for GlaxoSmithKline, Theranica, and Abbvie, and receives research support from the National Institutes of Health, National Center for Complementary and Integrative Health, National Institute of Neurological Disorders and Stroke, Veterans Health Administration, Cystic Fibrosis Foundation, and the American Heart Association. Dr. Riggins reported no relevant conflicts.

A version of this article appeared on Medscape.com.

SAN DIEGO — Nearly one in three patients with migraine are reluctant to seek medical help and many blame healthcare providers, results of a recent survey showed.

Participants cited concerns that their complaints would be dismissed, a belief that healthcare providers could offer no additional help, and a prior unsuccessful clinician visit as reasons for not seeking care. Survey respondents saw an average of four clinicians before finally receiving a diagnosis.

“I was shocked that a third of patients were reluctant to seek care,” said study investigator Elizabeth K. Seng, PhD, associate professor, Ferkauf Graduate School of Psychology, Yeshiva University, and research associate professor, department of neurology, Albert Einstein College of Medicine, both in New York City. “That just shows a much higher level of medical distress than I expected from this community of people who are obviously suffering from this significant neurologic disease.”

The findings were presented at the annual meeting of the American Headache Society.
 

‘Significant Disease’

The study included 500 adults with migraine (mean age, 40 years) who signed up for a patient support group sponsored by Eli Lilly and completed a comprehensive survey. Respondents were mostly female, White, non-Hispanic, and well-educated individuals.

Half of participants had episodic migraines, and half had chronic migraines; 46% reported experiencing anxiety and 33% reported depression.

Almost all respondents had initiated treatment with a first calcitonin gene-related peptide (CGRP) monoclonal antibody.

“These are people who have significant enough disease that eventually they needed our top-tier preventive medication,” Dr. Seng said.

Participants answered a variety of questions pertaining to disease factors and treatment seeking. Just over 70% said they suspected they had migraine prior to diagnosis, “which means for almost 30%, it was a surprise when they received the diagnosis,” said Dr. Seng. 

Nearly 40% reported that a relative first suggested they may have migraine, and 33% suspected it themselves. Only 17.4% said a healthcare provider suggested they may have the condition.

Almost a third of respondents (30.5%) reported they were reluctant to seek medical help.

“Some said they didn’t think their physician could do anything more than they were already doing for themselves, or that they’d be taken seriously, or they had had talked to doctors before and this wasn’t helpful,” said Dr. Seng. 

These responses speak to the need for better public health messaging, she said. “People have this idea that migraine attacks aren’t a big deal when, in fact, these attacks area big deal and certainly deserve treatment.” 

Family and friends were participants’ most common source of information on migraine, followed by the Internet. “This highlights the importance of getting migraine-related information out there so that when people talk to their friends and family, they’re receiving accurate information,” said Dr. Seng.

When asked about the path to a diagnosis, respondents reported consulting an average of four providers before receiving an accurate diagnosis. “That’s pretty remarkable,” Dr. Seng said.

An increase in frequency or severity of migraine attacks or attacks that interfered with work or school “pushed people over the threshold to seek care,” Dr. Seng said. 

A subset of patients was asked about the factors they believed could help with migraine attacks. Of these, 80% cited diet and 70% stress reduction. Supplements, exercise, and relaxation techniques were cited much less frequently, said Dr. Seng. 

The mean age of respondents’ migraine diagnosis was 26 years, so there was about 18 years from the time of diagnosis to participation in the survey, which could introduce recall bias. Other potential limitations included the fact that the survey had no open-ended questions, and men and ethnic minorities were underrepresented. 
 

Useful Data

Commenting on the study findings, Nina Riggins, MD, PhD, president, Brain Performance Center and Research Institute, and director of the Headache Center at The Neuron Clinic, San Diego, California, said the survey findings are “very useful” and highlight “significant opportunities for improvement in migraine education for clinicians and people living with migraine disease.”

The fact that participants reported consulting an average of four healthcare providers before receiving an accurate diagnosis underscores the importance of providing clinicians with tools to identify migraine, she said.

This is especially relevant as new migraine therapies that may improve efficacy and have fewer side effects become available, she added. 

“It would be interesting to see in future studies if migraine recognition by non-headache specialists improved after CGRP-blocking medications for migraine management became available,” said Dr. Riggins, who is cochair of the AHS First Contact program which is aimed at improving headache management in primary care.

She added that she and her colleagues will keep these survey results in mind when creating future educational materials for clinicians.

The study was supported by Eli Lily. Dr. Seng is a consultant for GlaxoSmithKline, Theranica, and Abbvie, and receives research support from the National Institutes of Health, National Center for Complementary and Integrative Health, National Institute of Neurological Disorders and Stroke, Veterans Health Administration, Cystic Fibrosis Foundation, and the American Heart Association. Dr. Riggins reported no relevant conflicts.

A version of this article appeared on Medscape.com.

WASHINGTON – Women with autoimmune liver diseases (AILD) may face increased risks for major adverse cardiovascular outcomes, according to a study presented at the annual Digestive Disease Week^® (DDW).

In particular, women with autoimmune hepatitis (AIH) and primary biliary cholangitis (PBC) appear to have higher risks than women without AIH or PBC. Those with primary sclerosing cholangitis (PSC) don’t seem to have increased risks.

“We know that cardiovascular disease remains the number one cause of death, but the mortality rate for women over the last decade has plateaued, whereas in men it’s actually declining due to interventions,” said lead author Rachel Redfield, MD, a transplant hepatology fellow at Thomas Jefferson University Hospital in Philadelphia.

“This is likely because we don’t have adequate risk stratification, especially for women,” she said. “We know that immune-mediated diseases — such as rheumatoid arthritis and psoriasis — carry a higher risk of cardiovascular disease, but there’s not a lot of data on our autoimmune liver disease patients.”

Dr. Redfield

Although being a female can offer protection against some CVD risks, the atherosclerotic cardiovascular disease (ASCVD) 10-year risk score calculator recommended by the American College of Cardiology doesn’t include chronic inflammatory diseases associated with increased CVD risk, including AILD.

Dr. Redfield and colleagues conducted a multicenter, retrospective cohort study of patients with AIH, PBC, and PSC from 1999-2019. Using TriNetX data, the researchers looked at women with AILD who also had diabetes mellitus, hypertension, and hyperlipidemia, as well as a control group of men and women with these same disorders, excluding those who used biologics, immune modulators, and steroids or had other autoimmune disorders.

The research team used 1:1 propensity-score matching for women in the study group and in the control group based on age, race, ethnicity, ASCVD risk factors, and tobacco use. Women in the study group and men in the control group were matched for age, race, ethnicity, and tobacco use.

The primary outcome was summative cardiovascular risk, including unstable angina, acute myocardial infarction, presence of coronary angioplasty implant, coronary artery bypass, percutaneous coronary intervention, and cerebral infarction.

Overall, women with AIH had a significantly higher cardiovascular risk compared to women without AIH, at 25.4% versus 20.6% (P = .0007).

Specifically, women with PBC had a significantly higher cardiovascular risk compared to women without PBC, at 27.05% versus 20.9% (P < .0001).

There wasn’t a significant difference in risk between women with and without PSC, at 27.5% versus 21.8% (P = .27).

When compared to men without disease, women with AIH didn’t have a statistically significant higher risk, at 25.3% versus 24.2% (P = .44). Similarly, there didn’t appear to be a significant difference between women with PBC and men without PBC, at 26.9% versus 25.9% (P = .52), or between women with PSC and men without PSC, at 27.7% versus 26.2% (P = .78).

Dr. Redfield and colleagues then compared the ASCVD-calculated risk versus database risk, finding that in each group of women with AILD — including AIH, PBC, and PSC — the ASCVD-calculated risk was around 11%, compared with database risk scores of 25% for AIH, 27% for PBC, and 28% for PSC. These database risks appeared similar to both the ASCVD and database risk percentages for men.

“So potentially there’s an oversight in women with any kind of inflammatory disease, but specifically here, autoimmune liver diseases,” she said. “We really need to enhance our risk assessment strategies to take into account their risk and optimize patient outcomes.”

Dr. Redfield noted the limitations with using TriNetX data, including coding consistency among providers and healthcare organizations, unknown patient follow-up dates, and the inability to capture various inflammatory disease phenotypes, such as autoimmune hepatitis with multiple flares, which may be associated with higher cardiovascular risks.

As an attendee of the DDW session, Kenneth Kelson, MD, a gastroenterologist with Fremont Medical Group and Washington Hospital Healthcare System in Fremont, California, noted the importance of investigating the effects of different types of statins in these patients. Although the research team looked at top-level differences among statin users, finding that women with AILD were more likely to be on a statin, they didn’t incorporate statin therapy in the propensity-score matching model.

“Lipid-soluble statins are known to cause more liver trouble, even though it’s pretty low,” Dr. Kelson said. “Whereas the water-soluble statins have a lower incidence of liver issues.”

Dr. Redfield and Dr. Kelson reported no relevant disclosures.

WASHINGTON – Women with autoimmune liver diseases (AILD) may face increased risks for major adverse cardiovascular outcomes, according to a study presented at the annual Digestive Disease Week^® (DDW).

In particular, women with autoimmune hepatitis (AIH) and primary biliary cholangitis (PBC) appear to have higher risks than women without AIH or PBC. Those with primary sclerosing cholangitis (PSC) don’t seem to have increased risks.

“We know that cardiovascular disease remains the number one cause of death, but the mortality rate for women over the last decade has plateaued, whereas in men it’s actually declining due to interventions,” said lead author Rachel Redfield, MD, a transplant hepatology fellow at Thomas Jefferson University Hospital in Philadelphia.

“This is likely because we don’t have adequate risk stratification, especially for women,” she said. “We know that immune-mediated diseases — such as rheumatoid arthritis and psoriasis — carry a higher risk of cardiovascular disease, but there’s not a lot of data on our autoimmune liver disease patients.”

Dr. Redfield

Although being a female can offer protection against some CVD risks, the atherosclerotic cardiovascular disease (ASCVD) 10-year risk score calculator recommended by the American College of Cardiology doesn’t include chronic inflammatory diseases associated with increased CVD risk, including AILD.

Dr. Redfield and colleagues conducted a multicenter, retrospective cohort study of patients with AIH, PBC, and PSC from 1999-2019. Using TriNetX data, the researchers looked at women with AILD who also had diabetes mellitus, hypertension, and hyperlipidemia, as well as a control group of men and women with these same disorders, excluding those who used biologics, immune modulators, and steroids or had other autoimmune disorders.

The research team used 1:1 propensity-score matching for women in the study group and in the control group based on age, race, ethnicity, ASCVD risk factors, and tobacco use. Women in the study group and men in the control group were matched for age, race, ethnicity, and tobacco use.

The primary outcome was summative cardiovascular risk, including unstable angina, acute myocardial infarction, presence of coronary angioplasty implant, coronary artery bypass, percutaneous coronary intervention, and cerebral infarction.

Overall, women with AIH had a significantly higher cardiovascular risk compared to women without AIH, at 25.4% versus 20.6% (P = .0007).

Specifically, women with PBC had a significantly higher cardiovascular risk compared to women without PBC, at 27.05% versus 20.9% (P < .0001).

There wasn’t a significant difference in risk between women with and without PSC, at 27.5% versus 21.8% (P = .27).

When compared to men without disease, women with AIH didn’t have a statistically significant higher risk, at 25.3% versus 24.2% (P = .44). Similarly, there didn’t appear to be a significant difference between women with PBC and men without PBC, at 26.9% versus 25.9% (P = .52), or between women with PSC and men without PSC, at 27.7% versus 26.2% (P = .78).

Dr. Redfield and colleagues then compared the ASCVD-calculated risk versus database risk, finding that in each group of women with AILD — including AIH, PBC, and PSC — the ASCVD-calculated risk was around 11%, compared with database risk scores of 25% for AIH, 27% for PBC, and 28% for PSC. These database risks appeared similar to both the ASCVD and database risk percentages for men.

“So potentially there’s an oversight in women with any kind of inflammatory disease, but specifically here, autoimmune liver diseases,” she said. “We really need to enhance our risk assessment strategies to take into account their risk and optimize patient outcomes.”

Dr. Redfield noted the limitations with using TriNetX data, including coding consistency among providers and healthcare organizations, unknown patient follow-up dates, and the inability to capture various inflammatory disease phenotypes, such as autoimmune hepatitis with multiple flares, which may be associated with higher cardiovascular risks.

As an attendee of the DDW session, Kenneth Kelson, MD, a gastroenterologist with Fremont Medical Group and Washington Hospital Healthcare System in Fremont, California, noted the importance of investigating the effects of different types of statins in these patients. Although the research team looked at top-level differences among statin users, finding that women with AILD were more likely to be on a statin, they didn’t incorporate statin therapy in the propensity-score matching model.

“Lipid-soluble statins are known to cause more liver trouble, even though it’s pretty low,” Dr. Kelson said. “Whereas the water-soluble statins have a lower incidence of liver issues.”

Dr. Redfield and Dr. Kelson reported no relevant disclosures.

WASHINGTON – Women with autoimmune liver diseases (AILD) may face increased risks for major adverse cardiovascular outcomes, according to a study presented at the annual Digestive Disease Week^® (DDW).

In particular, women with autoimmune hepatitis (AIH) and primary biliary cholangitis (PBC) appear to have higher risks than women without AIH or PBC. Those with primary sclerosing cholangitis (PSC) don’t seem to have increased risks.

“We know that cardiovascular disease remains the number one cause of death, but the mortality rate for women over the last decade has plateaued, whereas in men it’s actually declining due to interventions,” said lead author Rachel Redfield, MD, a transplant hepatology fellow at Thomas Jefferson University Hospital in Philadelphia.

“This is likely because we don’t have adequate risk stratification, especially for women,” she said. “We know that immune-mediated diseases — such as rheumatoid arthritis and psoriasis — carry a higher risk of cardiovascular disease, but there’s not a lot of data on our autoimmune liver disease patients.”

Dr. Redfield

Although being a female can offer protection against some CVD risks, the atherosclerotic cardiovascular disease (ASCVD) 10-year risk score calculator recommended by the American College of Cardiology doesn’t include chronic inflammatory diseases associated with increased CVD risk, including AILD.

Dr. Redfield and colleagues conducted a multicenter, retrospective cohort study of patients with AIH, PBC, and PSC from 1999-2019. Using TriNetX data, the researchers looked at women with AILD who also had diabetes mellitus, hypertension, and hyperlipidemia, as well as a control group of men and women with these same disorders, excluding those who used biologics, immune modulators, and steroids or had other autoimmune disorders.

The research team used 1:1 propensity-score matching for women in the study group and in the control group based on age, race, ethnicity, ASCVD risk factors, and tobacco use. Women in the study group and men in the control group were matched for age, race, ethnicity, and tobacco use.

The primary outcome was summative cardiovascular risk, including unstable angina, acute myocardial infarction, presence of coronary angioplasty implant, coronary artery bypass, percutaneous coronary intervention, and cerebral infarction.

Overall, women with AIH had a significantly higher cardiovascular risk compared to women without AIH, at 25.4% versus 20.6% (P = .0007).

Specifically, women with PBC had a significantly higher cardiovascular risk compared to women without PBC, at 27.05% versus 20.9% (P < .0001).

There wasn’t a significant difference in risk between women with and without PSC, at 27.5% versus 21.8% (P = .27).

When compared to men without disease, women with AIH didn’t have a statistically significant higher risk, at 25.3% versus 24.2% (P = .44). Similarly, there didn’t appear to be a significant difference between women with PBC and men without PBC, at 26.9% versus 25.9% (P = .52), or between women with PSC and men without PSC, at 27.7% versus 26.2% (P = .78).

Dr. Redfield and colleagues then compared the ASCVD-calculated risk versus database risk, finding that in each group of women with AILD — including AIH, PBC, and PSC — the ASCVD-calculated risk was around 11%, compared with database risk scores of 25% for AIH, 27% for PBC, and 28% for PSC. These database risks appeared similar to both the ASCVD and database risk percentages for men.

“So potentially there’s an oversight in women with any kind of inflammatory disease, but specifically here, autoimmune liver diseases,” she said. “We really need to enhance our risk assessment strategies to take into account their risk and optimize patient outcomes.”

Dr. Redfield noted the limitations with using TriNetX data, including coding consistency among providers and healthcare organizations, unknown patient follow-up dates, and the inability to capture various inflammatory disease phenotypes, such as autoimmune hepatitis with multiple flares, which may be associated with higher cardiovascular risks.

As an attendee of the DDW session, Kenneth Kelson, MD, a gastroenterologist with Fremont Medical Group and Washington Hospital Healthcare System in Fremont, California, noted the importance of investigating the effects of different types of statins in these patients. Although the research team looked at top-level differences among statin users, finding that women with AILD were more likely to be on a statin, they didn’t incorporate statin therapy in the propensity-score matching model.

“Lipid-soluble statins are known to cause more liver trouble, even though it’s pretty low,” Dr. Kelson said. “Whereas the water-soluble statins have a lower incidence of liver issues.”

Dr. Redfield and Dr. Kelson reported no relevant disclosures.

At the same time the Medicare population is growing, the number of family practice (FP) physicians and the number of procedures they perform are decreasing, according to a new analysis in Annals of Family Medicine.

“The reasons might represent a changing scope of family practice, increasing referrals, shifting tasks to [physician assistants] and [nurse practitioners], or some combination,” wrote the authors, led by Robert McKenna, DMSc, MPH, PA-C, with Marshall B. Ketchum University, Fullerton, California. But understanding the reasons may be important to improve training programs.

The Council of Academic Family Medicine (CAFM) in 2014 addressed variability in how FPs perform procedures with a consensus statement on 44 procedures FPs should be competent to perform after a family medicine residency and 24 more for which residency programs should offer training.

Researchers wanted to see how often the procedures were being done so they performed a retrospective, observational study of the Medicare Part B cohort to understand how often FPs report CAFM-recommended procedures in an outpatient setting. They used data from 2014 to 2021, the most recent years for which data were available, and modified the list of procedures for Medicare beneficiaries to match with Current Procedural Terminology codes.
 

Procedures Dropped by a Third in 8 Years

For the 8 years ending in 2021, there was a 33% decrease in outpatient procedures filed and a 36% decrease in the number of FPs who filed them, according to the paper. However, the number of FPs who treat Medicare beneficiaries has remained relatively stable. Almost all procedures performed were for the skin; musculoskeletal; eyes, ears, nose, and throat; and pulmonary categories.

There was a 17% decrease in every category of modified CAFM procedures between 2014 and 2019 before the pandemic.

Concurrently, the U.S. Bureau of Labor Statistics reported a 7% decrease in clinically active FPs from 2013 to 2022; 100,940 FPs were employed clinically in 2022. The Association of American Medical Colleges estimates a shortage of 17,800-48,000 primary care physicians by 2034.

“In 2021, the number of Medicare Part B claims by FPs for all [modified] CAFM outpatient procedures was reported by 12% of these clinicians and represented less than 1% of all CPT claims submitted,” the researchers wrote.

“Meanwhile, the 17% of Americans aged [at least] 65 years is projected to represent 20% of the U.S. population by 2030,” the authors pointed out.

The reasons for the decrease in procedures need further study, the authors said. From 2010 to 2020, there was a 30% and 47% increase in the employment of PAs and NPs, respectively, across all specialties, many employed in primary care. And 42% and 56% of family physicians reported working with PAs and NPs, respectively, from 2014 to 2018, according to the paper.

One explanation is a shifting of roles. “NPs and PAs might increasingly serve as proceduralists, freeing FPs to focus on other aspects of their practice such as complex chronic disease management or annual wellness visits,” the authors wrote.

It’s also possible that more Medicare-eligible Americans are being referred to various specialists such as urologists, gynecologists, and cardiologists, they speculated, adding that, “because demographic trends show an increasingly older population, family medicine training programs might need to adjust to meet this change.”

Among the limitations of the data are that publicly available Medicare data are limited to the approximately 60% of beneficiaries who are traditional Part B enrollees. Those enrolled in Medicare Advantage plans were excluded.

The work was supported in part by a Small Research Grant from the American Academy of Physician Associates in 2022. The authors reported no relevant financial relationships.

At the same time the Medicare population is growing, the number of family practice (FP) physicians and the number of procedures they perform are decreasing, according to a new analysis in Annals of Family Medicine.

“The reasons might represent a changing scope of family practice, increasing referrals, shifting tasks to [physician assistants] and [nurse practitioners], or some combination,” wrote the authors, led by Robert McKenna, DMSc, MPH, PA-C, with Marshall B. Ketchum University, Fullerton, California. But understanding the reasons may be important to improve training programs.

The Council of Academic Family Medicine (CAFM) in 2014 addressed variability in how FPs perform procedures with a consensus statement on 44 procedures FPs should be competent to perform after a family medicine residency and 24 more for which residency programs should offer training.

Researchers wanted to see how often the procedures were being done so they performed a retrospective, observational study of the Medicare Part B cohort to understand how often FPs report CAFM-recommended procedures in an outpatient setting. They used data from 2014 to 2021, the most recent years for which data were available, and modified the list of procedures for Medicare beneficiaries to match with Current Procedural Terminology codes.
 

Procedures Dropped by a Third in 8 Years

For the 8 years ending in 2021, there was a 33% decrease in outpatient procedures filed and a 36% decrease in the number of FPs who filed them, according to the paper. However, the number of FPs who treat Medicare beneficiaries has remained relatively stable. Almost all procedures performed were for the skin; musculoskeletal; eyes, ears, nose, and throat; and pulmonary categories.

There was a 17% decrease in every category of modified CAFM procedures between 2014 and 2019 before the pandemic.

Concurrently, the U.S. Bureau of Labor Statistics reported a 7% decrease in clinically active FPs from 2013 to 2022; 100,940 FPs were employed clinically in 2022. The Association of American Medical Colleges estimates a shortage of 17,800-48,000 primary care physicians by 2034.

“In 2021, the number of Medicare Part B claims by FPs for all [modified] CAFM outpatient procedures was reported by 12% of these clinicians and represented less than 1% of all CPT claims submitted,” the researchers wrote.

“Meanwhile, the 17% of Americans aged [at least] 65 years is projected to represent 20% of the U.S. population by 2030,” the authors pointed out.

The reasons for the decrease in procedures need further study, the authors said. From 2010 to 2020, there was a 30% and 47% increase in the employment of PAs and NPs, respectively, across all specialties, many employed in primary care. And 42% and 56% of family physicians reported working with PAs and NPs, respectively, from 2014 to 2018, according to the paper.

One explanation is a shifting of roles. “NPs and PAs might increasingly serve as proceduralists, freeing FPs to focus on other aspects of their practice such as complex chronic disease management or annual wellness visits,” the authors wrote.

It’s also possible that more Medicare-eligible Americans are being referred to various specialists such as urologists, gynecologists, and cardiologists, they speculated, adding that, “because demographic trends show an increasingly older population, family medicine training programs might need to adjust to meet this change.”

Among the limitations of the data are that publicly available Medicare data are limited to the approximately 60% of beneficiaries who are traditional Part B enrollees. Those enrolled in Medicare Advantage plans were excluded.

The work was supported in part by a Small Research Grant from the American Academy of Physician Associates in 2022. The authors reported no relevant financial relationships.

At the same time the Medicare population is growing, the number of family practice (FP) physicians and the number of procedures they perform are decreasing, according to a new analysis in Annals of Family Medicine.

“The reasons might represent a changing scope of family practice, increasing referrals, shifting tasks to [physician assistants] and [nurse practitioners], or some combination,” wrote the authors, led by Robert McKenna, DMSc, MPH, PA-C, with Marshall B. Ketchum University, Fullerton, California. But understanding the reasons may be important to improve training programs.

The Council of Academic Family Medicine (CAFM) in 2014 addressed variability in how FPs perform procedures with a consensus statement on 44 procedures FPs should be competent to perform after a family medicine residency and 24 more for which residency programs should offer training.

Researchers wanted to see how often the procedures were being done so they performed a retrospective, observational study of the Medicare Part B cohort to understand how often FPs report CAFM-recommended procedures in an outpatient setting. They used data from 2014 to 2021, the most recent years for which data were available, and modified the list of procedures for Medicare beneficiaries to match with Current Procedural Terminology codes.
 

Procedures Dropped by a Third in 8 Years

For the 8 years ending in 2021, there was a 33% decrease in outpatient procedures filed and a 36% decrease in the number of FPs who filed them, according to the paper. However, the number of FPs who treat Medicare beneficiaries has remained relatively stable. Almost all procedures performed were for the skin; musculoskeletal; eyes, ears, nose, and throat; and pulmonary categories.

There was a 17% decrease in every category of modified CAFM procedures between 2014 and 2019 before the pandemic.

Concurrently, the U.S. Bureau of Labor Statistics reported a 7% decrease in clinically active FPs from 2013 to 2022; 100,940 FPs were employed clinically in 2022. The Association of American Medical Colleges estimates a shortage of 17,800-48,000 primary care physicians by 2034.

“In 2021, the number of Medicare Part B claims by FPs for all [modified] CAFM outpatient procedures was reported by 12% of these clinicians and represented less than 1% of all CPT claims submitted,” the researchers wrote.

“Meanwhile, the 17% of Americans aged [at least] 65 years is projected to represent 20% of the U.S. population by 2030,” the authors pointed out.

The reasons for the decrease in procedures need further study, the authors said. From 2010 to 2020, there was a 30% and 47% increase in the employment of PAs and NPs, respectively, across all specialties, many employed in primary care. And 42% and 56% of family physicians reported working with PAs and NPs, respectively, from 2014 to 2018, according to the paper.

One explanation is a shifting of roles. “NPs and PAs might increasingly serve as proceduralists, freeing FPs to focus on other aspects of their practice such as complex chronic disease management or annual wellness visits,” the authors wrote.

It’s also possible that more Medicare-eligible Americans are being referred to various specialists such as urologists, gynecologists, and cardiologists, they speculated, adding that, “because demographic trends show an increasingly older population, family medicine training programs might need to adjust to meet this change.”

Among the limitations of the data are that publicly available Medicare data are limited to the approximately 60% of beneficiaries who are traditional Part B enrollees. Those enrolled in Medicare Advantage plans were excluded.

The work was supported in part by a Small Research Grant from the American Academy of Physician Associates in 2022. The authors reported no relevant financial relationships.

The experimental drug inavolisib showed sustained benefit combined with standard treatment in advanced, PIK3CA-mutated, hormone receptor–positive, human epidermal growth factor receptor 2–negative (HR+/HER2-) locally advanced metastatic breast cancer (LA/mBC), the INAVO120 trial.

The U.S. Food and Drug Administration (FDA) recently granted Breakthrough Therapy Designation for inavolisib in combination with palbociclib and fulvestrant based on initial results of the study presented at a December 2023 meeting. The phase 3 results showed the inavolisib-based regimen more than doubled progression-free survival (PFS) compared with the two other drugs alone as first-line treatment, researchers reported.

The expanded analysis of the trial, which was presented at the annual meeting of the American Society of Clinical Oncology, looked at additional endpoints, including PFS2 (defined as time from randomization to end of next-line treatment), time to first chemotherapy, key adverse events (AEs) and patient-reported outcomes (PROs).

“Triple combination of inavolisib, a novel PI3K inhibitor, with palbociclib and fulvestrant, resulted in significant and clinically meaningful improvement in PFS (15.0 vs 7.3 months, hazard ratio [HR] 0.43, P less than .0001),” lead investigator Dejan Juric, MD, reported at the meeting, referring to the initial results.

In additional endpoints, the inavolisib-based triplet also “sustained benefit beyond disease progression, delay in chemotherapy initiation, a manageable safety profile, prolonged time to deterioration in pain severity, and maintained quality of life, supporting the overall conclusion that this triple combination is a promising new treatment option for patients with PIK3CA-mutated HR-positive, HER2-negative metastatic breast cancer,” said the oncologist, of Massachusetts General Hospital Cancer Center and assistant professor at Harvard Medical School in Boston.
 

Methods and Results

The trial enrolled 325 patients whose disease had progressed during or within 12 months of adjuvant endocrine therapy (ET) with an aromatase inhibitor or tamoxifen and who had not received prior systemic therapy for recurrent LA/mBC. Patients were enrolled from December 2019 to September 2023 and randomized to either the triplet combination of inavolisib with palbociclib and fulvestrant (n = 161) or the doublet therapy of placebo with palbociclib and fulvestrant (n = 164) until discontinuation due to progressive disease or toxicity.

At the analysis cutoff date at the end of September, 57.8% of patients in the experimental triple therapy arm and 70.1% in the doublet arm had discontinued treatment. In addition, “7.5% versus 11.6% of patients died without subsequent therapy,” said Dr. Juric, and 40.4% of those in the triplet arm, and 50% in the doublet arm received subsequent therapy.

In the expanded analysis, at a median follow-up of 21.3 months, the triplet combination was associated with a PFS2 benefit of 8.9 months over the doublet – meaning patients had 24 months versus 15.1 months from randomization to end of next-line treatment (HR = 0.54). There was a similar benefit in time to first chemotherapy.

Hyperglycemia, diarrhea, rash, and mucosal effects are a known toxicity of PI3K inhibition and were experienced more frequently in the inavolisib arm compared with the placebo arm: (59% vs 9%; 48% vs 16%; 25% vs 17%; and 51% vs 27% respectively). However, “in the vast majority of patients these AEs were experienced in a grade 1 or grade 2 level,” and had resolved by the cutoff date, said Dr. Juric.

There was a 6.2% rate of inavolisib discontinuation due to AEs, but most AEs could be managed with “common approaches” such as metformin for hyperglycemia, loperamide for diarrhea, topical hydrocortisone for rash, and steroid mouthwash for stomatitis/mucosal inflammation, he added.

Patients in the triple treatment arm experienced a longer interval before pain worsened, a median of 30.9 versus 18.1 months, and patient-reported outcomes and health-related quality of life measures showed no decrease with the addition of inavolisib, Dr. Juric reported.
 

Rationale for Using PFS2 as Endpoint

The PFS2 endpoint has emerged with studies of targeted cancer therapies, Kevin Kalinsky, MD, director of the Glenn Family Breast Center at Winship Cancer Institute of Emory University, in Atlanta, said in an interview.

“Presenting PFS2 is not a new thing — we’ve been doing this in other breast cancer studies (of CDK4/6 inhibitors),” said Dr. Kalinsky, a coauthor of the study. “The concern is that you give a drug, and then, after that, things grow so rapidly that then you’re actually not benefiting the patient.

“If you’re giving a targeted agent in the first-line, then the biology changes after that first-line, are you really even making a difference? Or is the drug so toxic that they’re not able to tolerate a next line of treatment?” Dr. Kalinsky continued. “So that’s really the intent of PFS2. The PFS2 included the next line of treatment, so it’s really a first, and second-line representation of treatment. The study presented at ASCO was really about toxicity.”

The study was funded by F. Hoffmann-La Roche Ltd. Dr. Juric disclosed having stock and other ownership interests in PIC Therapeutics, Relay Therapeutics, and Vibliome Therapeutics; consulting or advisory roles with AstraZeneca, Eisai, Genentech, Lilly, MapKure, Novartis, Pfizer, PIC Therapeutics, Relay Therapeutics, and Vibliome Therapeutics; and research funding from Amgen, Arvinas, AstraZeneca, Blueprint Medicines, Eisai, Genentech, Infinity Pharmaceuticals, InventisBio, Novartis, Pfizer, Ribon Therapeutics, Scorpion Therapeutics, Syros Pharmaceuticals, and Takeda.

The experimental drug inavolisib showed sustained benefit combined with standard treatment in advanced, PIK3CA-mutated, hormone receptor–positive, human epidermal growth factor receptor 2–negative (HR+/HER2-) locally advanced metastatic breast cancer (LA/mBC), the INAVO120 trial.

The U.S. Food and Drug Administration (FDA) recently granted Breakthrough Therapy Designation for inavolisib in combination with palbociclib and fulvestrant based on initial results of the study presented at a December 2023 meeting. The phase 3 results showed the inavolisib-based regimen more than doubled progression-free survival (PFS) compared with the two other drugs alone as first-line treatment, researchers reported.

The expanded analysis of the trial, which was presented at the annual meeting of the American Society of Clinical Oncology, looked at additional endpoints, including PFS2 (defined as time from randomization to end of next-line treatment), time to first chemotherapy, key adverse events (AEs) and patient-reported outcomes (PROs).

“Triple combination of inavolisib, a novel PI3K inhibitor, with palbociclib and fulvestrant, resulted in significant and clinically meaningful improvement in PFS (15.0 vs 7.3 months, hazard ratio [HR] 0.43, P less than .0001),” lead investigator Dejan Juric, MD, reported at the meeting, referring to the initial results.

In additional endpoints, the inavolisib-based triplet also “sustained benefit beyond disease progression, delay in chemotherapy initiation, a manageable safety profile, prolonged time to deterioration in pain severity, and maintained quality of life, supporting the overall conclusion that this triple combination is a promising new treatment option for patients with PIK3CA-mutated HR-positive, HER2-negative metastatic breast cancer,” said the oncologist, of Massachusetts General Hospital Cancer Center and assistant professor at Harvard Medical School in Boston.
 

Methods and Results

The trial enrolled 325 patients whose disease had progressed during or within 12 months of adjuvant endocrine therapy (ET) with an aromatase inhibitor or tamoxifen and who had not received prior systemic therapy for recurrent LA/mBC. Patients were enrolled from December 2019 to September 2023 and randomized to either the triplet combination of inavolisib with palbociclib and fulvestrant (n = 161) or the doublet therapy of placebo with palbociclib and fulvestrant (n = 164) until discontinuation due to progressive disease or toxicity.

At the analysis cutoff date at the end of September, 57.8% of patients in the experimental triple therapy arm and 70.1% in the doublet arm had discontinued treatment. In addition, “7.5% versus 11.6% of patients died without subsequent therapy,” said Dr. Juric, and 40.4% of those in the triplet arm, and 50% in the doublet arm received subsequent therapy.

In the expanded analysis, at a median follow-up of 21.3 months, the triplet combination was associated with a PFS2 benefit of 8.9 months over the doublet – meaning patients had 24 months versus 15.1 months from randomization to end of next-line treatment (HR = 0.54). There was a similar benefit in time to first chemotherapy.

Hyperglycemia, diarrhea, rash, and mucosal effects are a known toxicity of PI3K inhibition and were experienced more frequently in the inavolisib arm compared with the placebo arm: (59% vs 9%; 48% vs 16%; 25% vs 17%; and 51% vs 27% respectively). However, “in the vast majority of patients these AEs were experienced in a grade 1 or grade 2 level,” and had resolved by the cutoff date, said Dr. Juric.

There was a 6.2% rate of inavolisib discontinuation due to AEs, but most AEs could be managed with “common approaches” such as metformin for hyperglycemia, loperamide for diarrhea, topical hydrocortisone for rash, and steroid mouthwash for stomatitis/mucosal inflammation, he added.

Patients in the triple treatment arm experienced a longer interval before pain worsened, a median of 30.9 versus 18.1 months, and patient-reported outcomes and health-related quality of life measures showed no decrease with the addition of inavolisib, Dr. Juric reported.
 

Rationale for Using PFS2 as Endpoint

The PFS2 endpoint has emerged with studies of targeted cancer therapies, Kevin Kalinsky, MD, director of the Glenn Family Breast Center at Winship Cancer Institute of Emory University, in Atlanta, said in an interview.

“Presenting PFS2 is not a new thing — we’ve been doing this in other breast cancer studies (of CDK4/6 inhibitors),” said Dr. Kalinsky, a coauthor of the study. “The concern is that you give a drug, and then, after that, things grow so rapidly that then you’re actually not benefiting the patient.

“If you’re giving a targeted agent in the first-line, then the biology changes after that first-line, are you really even making a difference? Or is the drug so toxic that they’re not able to tolerate a next line of treatment?” Dr. Kalinsky continued. “So that’s really the intent of PFS2. The PFS2 included the next line of treatment, so it’s really a first, and second-line representation of treatment. The study presented at ASCO was really about toxicity.”

The study was funded by F. Hoffmann-La Roche Ltd. Dr. Juric disclosed having stock and other ownership interests in PIC Therapeutics, Relay Therapeutics, and Vibliome Therapeutics; consulting or advisory roles with AstraZeneca, Eisai, Genentech, Lilly, MapKure, Novartis, Pfizer, PIC Therapeutics, Relay Therapeutics, and Vibliome Therapeutics; and research funding from Amgen, Arvinas, AstraZeneca, Blueprint Medicines, Eisai, Genentech, Infinity Pharmaceuticals, InventisBio, Novartis, Pfizer, Ribon Therapeutics, Scorpion Therapeutics, Syros Pharmaceuticals, and Takeda.

The experimental drug inavolisib showed sustained benefit combined with standard treatment in advanced, PIK3CA-mutated, hormone receptor–positive, human epidermal growth factor receptor 2–negative (HR+/HER2-) locally advanced metastatic breast cancer (LA/mBC), the INAVO120 trial.

The U.S. Food and Drug Administration (FDA) recently granted Breakthrough Therapy Designation for inavolisib in combination with palbociclib and fulvestrant based on initial results of the study presented at a December 2023 meeting. The phase 3 results showed the inavolisib-based regimen more than doubled progression-free survival (PFS) compared with the two other drugs alone as first-line treatment, researchers reported.

The expanded analysis of the trial, which was presented at the annual meeting of the American Society of Clinical Oncology, looked at additional endpoints, including PFS2 (defined as time from randomization to end of next-line treatment), time to first chemotherapy, key adverse events (AEs) and patient-reported outcomes (PROs).

“Triple combination of inavolisib, a novel PI3K inhibitor, with palbociclib and fulvestrant, resulted in significant and clinically meaningful improvement in PFS (15.0 vs 7.3 months, hazard ratio [HR] 0.43, P less than .0001),” lead investigator Dejan Juric, MD, reported at the meeting, referring to the initial results.

In additional endpoints, the inavolisib-based triplet also “sustained benefit beyond disease progression, delay in chemotherapy initiation, a manageable safety profile, prolonged time to deterioration in pain severity, and maintained quality of life, supporting the overall conclusion that this triple combination is a promising new treatment option for patients with PIK3CA-mutated HR-positive, HER2-negative metastatic breast cancer,” said the oncologist, of Massachusetts General Hospital Cancer Center and assistant professor at Harvard Medical School in Boston.
 

Methods and Results

The trial enrolled 325 patients whose disease had progressed during or within 12 months of adjuvant endocrine therapy (ET) with an aromatase inhibitor or tamoxifen and who had not received prior systemic therapy for recurrent LA/mBC. Patients were enrolled from December 2019 to September 2023 and randomized to either the triplet combination of inavolisib with palbociclib and fulvestrant (n = 161) or the doublet therapy of placebo with palbociclib and fulvestrant (n = 164) until discontinuation due to progressive disease or toxicity.

At the analysis cutoff date at the end of September, 57.8% of patients in the experimental triple therapy arm and 70.1% in the doublet arm had discontinued treatment. In addition, “7.5% versus 11.6% of patients died without subsequent therapy,” said Dr. Juric, and 40.4% of those in the triplet arm, and 50% in the doublet arm received subsequent therapy.

In the expanded analysis, at a median follow-up of 21.3 months, the triplet combination was associated with a PFS2 benefit of 8.9 months over the doublet – meaning patients had 24 months versus 15.1 months from randomization to end of next-line treatment (HR = 0.54). There was a similar benefit in time to first chemotherapy.

Hyperglycemia, diarrhea, rash, and mucosal effects are a known toxicity of PI3K inhibition and were experienced more frequently in the inavolisib arm compared with the placebo arm: (59% vs 9%; 48% vs 16%; 25% vs 17%; and 51% vs 27% respectively). However, “in the vast majority of patients these AEs were experienced in a grade 1 or grade 2 level,” and had resolved by the cutoff date, said Dr. Juric.

There was a 6.2% rate of inavolisib discontinuation due to AEs, but most AEs could be managed with “common approaches” such as metformin for hyperglycemia, loperamide for diarrhea, topical hydrocortisone for rash, and steroid mouthwash for stomatitis/mucosal inflammation, he added.

Patients in the triple treatment arm experienced a longer interval before pain worsened, a median of 30.9 versus 18.1 months, and patient-reported outcomes and health-related quality of life measures showed no decrease with the addition of inavolisib, Dr. Juric reported.
 

Rationale for Using PFS2 as Endpoint

The PFS2 endpoint has emerged with studies of targeted cancer therapies, Kevin Kalinsky, MD, director of the Glenn Family Breast Center at Winship Cancer Institute of Emory University, in Atlanta, said in an interview.

“Presenting PFS2 is not a new thing — we’ve been doing this in other breast cancer studies (of CDK4/6 inhibitors),” said Dr. Kalinsky, a coauthor of the study. “The concern is that you give a drug, and then, after that, things grow so rapidly that then you’re actually not benefiting the patient.

“If you’re giving a targeted agent in the first-line, then the biology changes after that first-line, are you really even making a difference? Or is the drug so toxic that they’re not able to tolerate a next line of treatment?” Dr. Kalinsky continued. “So that’s really the intent of PFS2. The PFS2 included the next line of treatment, so it’s really a first, and second-line representation of treatment. The study presented at ASCO was really about toxicity.”

The study was funded by F. Hoffmann-La Roche Ltd. Dr. Juric disclosed having stock and other ownership interests in PIC Therapeutics, Relay Therapeutics, and Vibliome Therapeutics; consulting or advisory roles with AstraZeneca, Eisai, Genentech, Lilly, MapKure, Novartis, Pfizer, PIC Therapeutics, Relay Therapeutics, and Vibliome Therapeutics; and research funding from Amgen, Arvinas, AstraZeneca, Blueprint Medicines, Eisai, Genentech, Infinity Pharmaceuticals, InventisBio, Novartis, Pfizer, Ribon Therapeutics, Scorpion Therapeutics, Syros Pharmaceuticals, and Takeda.

The Food and Drug Administration has expanded the indication for pembrolizumab (Keytruda, Merck) to include the use of the targeted immunotherapy agent plus chemotherapy followed by single-agent pembrolizumab in adults with primary advanced or recurrent endometrial cancer.

Approval in this setting was granted following priority review and was based on efficacy demonstrated in the randomized, placebo-controlled, phase 3 KEYNOTE-868/NRG-GY018 trial. The multicenter trial showed improved progression-free survival (PFS) with chemotherapy plus pembrolizumab versus chemotherapy plus placebo in patients with stage 3 or 4 disease or stage IVB recurrent disease in two cohorts: 222 patients with mismatch repair (MMR) deficiency, and 588 patients with MMR proficiency.

Among the MMR-deficient patients, median PFS was not reached in the treatment arm and was 6.5 months in the control arm (hazard ratio, 0.30). Among the MMR-proficient patients, the median PFS was 11.1 versus 8.5 months in the study arms, respectively (HR, 0.60), according to an FDA press release.

Patients in both cohorts were randomized 1:1 to receive 200 mg of either pembrolizumab or placebo every 3 weeks, followed by paclitaxel at a dose of 175 mg/m² and carboplatin at a dose of AUC 5 mg/mL/min for six cycles and then 400 mg of pembrolizumab or placebo every 6 weeks for up to 14 cycles.

“Adverse reactions associated with pembrolizumab and chemotherapy were generally similar to those previously reported for pembrolizumab or chemotherapy with the exception of a higher incidence of rash,” the FDA noted.

According to the full prescribing information for pembrolizumab, the recommended dose is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.

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

The Food and Drug Administration has expanded the indication for pembrolizumab (Keytruda, Merck) to include the use of the targeted immunotherapy agent plus chemotherapy followed by single-agent pembrolizumab in adults with primary advanced or recurrent endometrial cancer.

Approval in this setting was granted following priority review and was based on efficacy demonstrated in the randomized, placebo-controlled, phase 3 KEYNOTE-868/NRG-GY018 trial. The multicenter trial showed improved progression-free survival (PFS) with chemotherapy plus pembrolizumab versus chemotherapy plus placebo in patients with stage 3 or 4 disease or stage IVB recurrent disease in two cohorts: 222 patients with mismatch repair (MMR) deficiency, and 588 patients with MMR proficiency.

Among the MMR-deficient patients, median PFS was not reached in the treatment arm and was 6.5 months in the control arm (hazard ratio, 0.30). Among the MMR-proficient patients, the median PFS was 11.1 versus 8.5 months in the study arms, respectively (HR, 0.60), according to an FDA press release.

Patients in both cohorts were randomized 1:1 to receive 200 mg of either pembrolizumab or placebo every 3 weeks, followed by paclitaxel at a dose of 175 mg/m² and carboplatin at a dose of AUC 5 mg/mL/min for six cycles and then 400 mg of pembrolizumab or placebo every 6 weeks for up to 14 cycles.

“Adverse reactions associated with pembrolizumab and chemotherapy were generally similar to those previously reported for pembrolizumab or chemotherapy with the exception of a higher incidence of rash,” the FDA noted.

According to the full prescribing information for pembrolizumab, the recommended dose is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.

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

The Food and Drug Administration has expanded the indication for pembrolizumab (Keytruda, Merck) to include the use of the targeted immunotherapy agent plus chemotherapy followed by single-agent pembrolizumab in adults with primary advanced or recurrent endometrial cancer.

Approval in this setting was granted following priority review and was based on efficacy demonstrated in the randomized, placebo-controlled, phase 3 KEYNOTE-868/NRG-GY018 trial. The multicenter trial showed improved progression-free survival (PFS) with chemotherapy plus pembrolizumab versus chemotherapy plus placebo in patients with stage 3 or 4 disease or stage IVB recurrent disease in two cohorts: 222 patients with mismatch repair (MMR) deficiency, and 588 patients with MMR proficiency.

Among the MMR-deficient patients, median PFS was not reached in the treatment arm and was 6.5 months in the control arm (hazard ratio, 0.30). Among the MMR-proficient patients, the median PFS was 11.1 versus 8.5 months in the study arms, respectively (HR, 0.60), according to an FDA press release.

Patients in both cohorts were randomized 1:1 to receive 200 mg of either pembrolizumab or placebo every 3 weeks, followed by paclitaxel at a dose of 175 mg/m² and carboplatin at a dose of AUC 5 mg/mL/min for six cycles and then 400 mg of pembrolizumab or placebo every 6 weeks for up to 14 cycles.

“Adverse reactions associated with pembrolizumab and chemotherapy were generally similar to those previously reported for pembrolizumab or chemotherapy with the exception of a higher incidence of rash,” the FDA noted.

According to the full prescribing information for pembrolizumab, the recommended dose is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.

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

An ancient Chinese remedy for malaria could offer new hope to the 10% of reproductive-age women living with polycystic ovarian syndrome (PCOS), a poorly understood endocrine disorder that can cause hormonal imbalances, irregular periods, and cysts in the ovaries.

“PCOS is among the most common disorders of reproductive-age women,” said endocrinologist Andrea Dunaif, MD, a professor at the Icahn School of Medicine at Mount Sinai, New York City, who studies diabetes and women’s health. “It is a major risk factor for obesity, type 2 diabetes, and heart disease.” It’s also a leading cause of infertility.

Yet despite how common it is, PCOS has no Food and Drug Administration–approved treatments, though a few early-stage clinical trials are underway. Many women end up taking off-label medications such as oral contraceptives, insulin-sensitizing agents, and antiandrogens to help manage symptoms. Surgery can also be used to treat fertility problems associated with PCOS, though it may not work for everyone.

In a new study, a derivative of artemisinin — a molecule that comes from Artemisia plants, which have been used as far back as 1596 to treat malaria in China — helped relieve PCOS symptoms in rats and a small group of women.

Previously, the study’s lead researcher Qi-qun Tang, MD, PhD, had found that this derivative, called artemether, can increase thermogenesis, boosting metabolism. Dr. Tang and his team at Fudan University, Shanghai, China, wanted to see if it would help with PCOS, which is associated with metabolic problems such as insulin resistance.
 

What the Researchers Did

To simulate PCOS in rats, the team treated the rodents with insulin and human chorionic gonadotropin. Then, they tested artemether on the rats and found that it lowered androgen production in the ovaries.

“A common feature [of PCOS] is that the ovaries, and often the adrenal glands, make increased male hormones, nowhere near what a man makes but slightly above what a normal woman makes,” said Dr. Dunaif, who was not involved in the study.

Artemether “inhibits one of the steroidogenic enzymes, CYP11A1, which is important in the production of male hormones,” Dr. Tang said. It does this by increasing the enzyme’s interaction with a protein called LONP1, triggering the enzyme’s breakdown. Increased levels of LONP1 also appeared to suppress androgen production in the ovaries.

In a pilot clinical study of 19 women with PCOS, taking dihydroartemisinin — an approved drug used to treat malaria that contains active artemisinin derivatives — for 12 weeks substantially reduced serum testosterone and anti-Müllerian hormone levels (which are higher in women with PCOS). Using ultrasound, the researchers found that the antral follicle count (also higher than normal with PCOS) had been reduced. All participants had regular menstrual cycles during treatment. And no one reported significant side effects.

“Regular menstrual cycles suggest that there is ovulation, which can result in conception,” Dr. Dunaif said. Still, testing would be needed to confirm that cycles are ovulatory.

Lowering androgen levels “could improve a substantial portion of the symptoms of PCOS,” said Dr. Dunaif. But the research didn’t see an improvement in insulin sensitivity among the women, suggesting that targeting androgens may not help the metabolic symptoms.
 

What’s Next? 

A larger, placebo-controlled trial would still be needed to assess the drug’s efficacy, said Dr. Dunaif, pointing out that the human study did not have a placebo arm.

And unanswered questions remain. Are there any adrenal effects of the compound? “The enzymes that produce androgens are shared between the ovary and the adrenal [gland],” Dr. Dunaif said, but she pointed out that the study doesn’t address whether there is an adrenal benefit. It’s something to look at in future research.

Still, because artemisinin is an established drug, it may come to market faster than a new molecule would, she said. However, a pharmaceutical company would need to be willing to take on the drug. (Dr. Tang said several companies have already expressed interest.)

And while you can buy artemisinin on the Internet, Dr. Dunaif warned not to start taking it if you have PCOS. “I don’t think we’re at that point,” Dr. Dunaif said.

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

An ancient Chinese remedy for malaria could offer new hope to the 10% of reproductive-age women living with polycystic ovarian syndrome (PCOS), a poorly understood endocrine disorder that can cause hormonal imbalances, irregular periods, and cysts in the ovaries.

“PCOS is among the most common disorders of reproductive-age women,” said endocrinologist Andrea Dunaif, MD, a professor at the Icahn School of Medicine at Mount Sinai, New York City, who studies diabetes and women’s health. “It is a major risk factor for obesity, type 2 diabetes, and heart disease.” It’s also a leading cause of infertility.

Yet despite how common it is, PCOS has no Food and Drug Administration–approved treatments, though a few early-stage clinical trials are underway. Many women end up taking off-label medications such as oral contraceptives, insulin-sensitizing agents, and antiandrogens to help manage symptoms. Surgery can also be used to treat fertility problems associated with PCOS, though it may not work for everyone.

In a new study, a derivative of artemisinin — a molecule that comes from Artemisia plants, which have been used as far back as 1596 to treat malaria in China — helped relieve PCOS symptoms in rats and a small group of women.

Previously, the study’s lead researcher Qi-qun Tang, MD, PhD, had found that this derivative, called artemether, can increase thermogenesis, boosting metabolism. Dr. Tang and his team at Fudan University, Shanghai, China, wanted to see if it would help with PCOS, which is associated with metabolic problems such as insulin resistance.
 

What the Researchers Did

To simulate PCOS in rats, the team treated the rodents with insulin and human chorionic gonadotropin. Then, they tested artemether on the rats and found that it lowered androgen production in the ovaries.

“A common feature [of PCOS] is that the ovaries, and often the adrenal glands, make increased male hormones, nowhere near what a man makes but slightly above what a normal woman makes,” said Dr. Dunaif, who was not involved in the study.

Artemether “inhibits one of the steroidogenic enzymes, CYP11A1, which is important in the production of male hormones,” Dr. Tang said. It does this by increasing the enzyme’s interaction with a protein called LONP1, triggering the enzyme’s breakdown. Increased levels of LONP1 also appeared to suppress androgen production in the ovaries.

In a pilot clinical study of 19 women with PCOS, taking dihydroartemisinin — an approved drug used to treat malaria that contains active artemisinin derivatives — for 12 weeks substantially reduced serum testosterone and anti-Müllerian hormone levels (which are higher in women with PCOS). Using ultrasound, the researchers found that the antral follicle count (also higher than normal with PCOS) had been reduced. All participants had regular menstrual cycles during treatment. And no one reported significant side effects.

“Regular menstrual cycles suggest that there is ovulation, which can result in conception,” Dr. Dunaif said. Still, testing would be needed to confirm that cycles are ovulatory.

Lowering androgen levels “could improve a substantial portion of the symptoms of PCOS,” said Dr. Dunaif. But the research didn’t see an improvement in insulin sensitivity among the women, suggesting that targeting androgens may not help the metabolic symptoms.
 

What’s Next? 

A larger, placebo-controlled trial would still be needed to assess the drug’s efficacy, said Dr. Dunaif, pointing out that the human study did not have a placebo arm.

And unanswered questions remain. Are there any adrenal effects of the compound? “The enzymes that produce androgens are shared between the ovary and the adrenal [gland],” Dr. Dunaif said, but she pointed out that the study doesn’t address whether there is an adrenal benefit. It’s something to look at in future research.

Still, because artemisinin is an established drug, it may come to market faster than a new molecule would, she said. However, a pharmaceutical company would need to be willing to take on the drug. (Dr. Tang said several companies have already expressed interest.)

And while you can buy artemisinin on the Internet, Dr. Dunaif warned not to start taking it if you have PCOS. “I don’t think we’re at that point,” Dr. Dunaif said.

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

An ancient Chinese remedy for malaria could offer new hope to the 10% of reproductive-age women living with polycystic ovarian syndrome (PCOS), a poorly understood endocrine disorder that can cause hormonal imbalances, irregular periods, and cysts in the ovaries.

“PCOS is among the most common disorders of reproductive-age women,” said endocrinologist Andrea Dunaif, MD, a professor at the Icahn School of Medicine at Mount Sinai, New York City, who studies diabetes and women’s health. “It is a major risk factor for obesity, type 2 diabetes, and heart disease.” It’s also a leading cause of infertility.

Yet despite how common it is, PCOS has no Food and Drug Administration–approved treatments, though a few early-stage clinical trials are underway. Many women end up taking off-label medications such as oral contraceptives, insulin-sensitizing agents, and antiandrogens to help manage symptoms. Surgery can also be used to treat fertility problems associated with PCOS, though it may not work for everyone.

In a new study, a derivative of artemisinin — a molecule that comes from Artemisia plants, which have been used as far back as 1596 to treat malaria in China — helped relieve PCOS symptoms in rats and a small group of women.

Previously, the study’s lead researcher Qi-qun Tang, MD, PhD, had found that this derivative, called artemether, can increase thermogenesis, boosting metabolism. Dr. Tang and his team at Fudan University, Shanghai, China, wanted to see if it would help with PCOS, which is associated with metabolic problems such as insulin resistance.
 

What the Researchers Did

To simulate PCOS in rats, the team treated the rodents with insulin and human chorionic gonadotropin. Then, they tested artemether on the rats and found that it lowered androgen production in the ovaries.

“A common feature [of PCOS] is that the ovaries, and often the adrenal glands, make increased male hormones, nowhere near what a man makes but slightly above what a normal woman makes,” said Dr. Dunaif, who was not involved in the study.

Artemether “inhibits one of the steroidogenic enzymes, CYP11A1, which is important in the production of male hormones,” Dr. Tang said. It does this by increasing the enzyme’s interaction with a protein called LONP1, triggering the enzyme’s breakdown. Increased levels of LONP1 also appeared to suppress androgen production in the ovaries.

In a pilot clinical study of 19 women with PCOS, taking dihydroartemisinin — an approved drug used to treat malaria that contains active artemisinin derivatives — for 12 weeks substantially reduced serum testosterone and anti-Müllerian hormone levels (which are higher in women with PCOS). Using ultrasound, the researchers found that the antral follicle count (also higher than normal with PCOS) had been reduced. All participants had regular menstrual cycles during treatment. And no one reported significant side effects.

“Regular menstrual cycles suggest that there is ovulation, which can result in conception,” Dr. Dunaif said. Still, testing would be needed to confirm that cycles are ovulatory.

Lowering androgen levels “could improve a substantial portion of the symptoms of PCOS,” said Dr. Dunaif. But the research didn’t see an improvement in insulin sensitivity among the women, suggesting that targeting androgens may not help the metabolic symptoms.
 

What’s Next? 

A larger, placebo-controlled trial would still be needed to assess the drug’s efficacy, said Dr. Dunaif, pointing out that the human study did not have a placebo arm.

And unanswered questions remain. Are there any adrenal effects of the compound? “The enzymes that produce androgens are shared between the ovary and the adrenal [gland],” Dr. Dunaif said, but she pointed out that the study doesn’t address whether there is an adrenal benefit. It’s something to look at in future research.

Still, because artemisinin is an established drug, it may come to market faster than a new molecule would, she said. However, a pharmaceutical company would need to be willing to take on the drug. (Dr. Tang said several companies have already expressed interest.)

And while you can buy artemisinin on the Internet, Dr. Dunaif warned not to start taking it if you have PCOS. “I don’t think we’re at that point,” Dr. Dunaif said.

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

HOUSTON — A new analysis highlights the high burden of insomnia across the Americas, with about 17% of adults suffering from this chronic sleep disorder.

“Our findings underscore the urgent need for enhanced clinical care pathways and policy interventions to effectively diagnose and treat insomnia. It is crucial to foster greater awareness of the critical role that sleep plays in overall health,” lead investigator Adam Benjafield, PhD, vice president for medical affairs at ResMed, Sydney, Australia, said in an interview.

“Insomnia not only affects individuals’ health and quality of life but also has broader implications for public health systems. Developing comprehensive care strategies and promoting education about sleep health could significantly improve outcomes for individuals suffering from insomnia disorder,” Dr. Benjafield said.

The findings were presented at SLEEP 2024: 38th Annual Meeting of the Associated Professional Sleep Societies.
 

Underdiagnosed, Undertreated

Sleep disruptions contribute to various medical problems, including cognitive impairment, reduced immune function, metabolic imbalance, and exacerbation of psychiatric conditions. While the prevalence of insomnia in developed countries like the United States and Canada is known, there is limited epidemiologic evidence, and no reliable estimate for the disorder across the Americas — especially in low- and middle-income countries.

The researchers used published nation-specific data to estimate the prevalence of adult insomnia disorder across the 55 countries defined by the United Nations as comprising the Americas.

Based on the available data, the researchers estimated that about 123 million adults across the Americas have insomnia disorder (16.8%) — with greater prevalence in women (73 million, 19.5%) than in men (50 million, 14%).

The nations with the greatest burden of insomnia disorder are the United States (37 million), Brazil (29 million), and Mexico (16 million).

“While our study did not specifically investigate trends over time due to its scope, evidence from other research suggests that insomnia is becoming more prevalent over the long term. This growing trend highlights the increasing need for awareness and intervention in managing sleep health,” Dr. Benjafield said.

Insomnia is underdiagnosed and undertreated partly because of general lack of awareness about the importance of addressing sleep disorders and the fact that cognitive-behavioral therapy for insomnia (CBT-I), which is recommended as first-line treatment, is not widely accessible because of a shortage of trained CBT-I practitioners.

“Many individuals with insomnia struggle to find and receive this effective nonpharmacological treatment. Consequently, there is an overreliance on pharmaceutical solutions, which are ideally used for short-term management but are often extended due to the lack of alternatives. These medications can lead to dependency and other side effects,” Dr. Benjafield said.
 

Ask About Sleep

Insomnia symptoms are a “common presenting complaint in doctors’ offices in the United States. The percentages in this poster show that insomnia disorder has a similar, high percent prevalence across countries in the Americas,” Boris Gilyadov, MD, assistant professor of pulmonary, critical care, and sleep medicine at the Icahn School of Medicine at Mount Sinai in New York City, said in an interview.

“During preventive care visits and general physical exams, patients should be asked about the quality of their sleep. Patients may benefit from a referral to the sleep medicine clinic when appropriate,” said Dr. Gilyadov, who wasn’t involved in the study.

“CBT-I is the first-line treatment for chronic insomnia disorder and can be an effective treatment for most patients. An alternative to CBT-I, when it is not available, is digital CBT-I,” Dr. Gilyadov said.

“There are also behavioral therapies called BBT-I [brief behavioral treatment for insomnia] and ACT [acceptance and commitment therapy]. These are therapies that may be offered by psychologists who specialize in the treatment of chronic insomnia disorder,” Dr. Gilyadov noted.

The study was conducted in collaboration with medXcloud and funded by ResMed. Dr. Benjafield is an employee of ResMed. Dr. Gilyadov had no relevant disclosures.

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

HOUSTON — A new analysis highlights the high burden of insomnia across the Americas, with about 17% of adults suffering from this chronic sleep disorder.

“Our findings underscore the urgent need for enhanced clinical care pathways and policy interventions to effectively diagnose and treat insomnia. It is crucial to foster greater awareness of the critical role that sleep plays in overall health,” lead investigator Adam Benjafield, PhD, vice president for medical affairs at ResMed, Sydney, Australia, said in an interview.

“Insomnia not only affects individuals’ health and quality of life but also has broader implications for public health systems. Developing comprehensive care strategies and promoting education about sleep health could significantly improve outcomes for individuals suffering from insomnia disorder,” Dr. Benjafield said.

The findings were presented at SLEEP 2024: 38th Annual Meeting of the Associated Professional Sleep Societies.
 

Underdiagnosed, Undertreated

Sleep disruptions contribute to various medical problems, including cognitive impairment, reduced immune function, metabolic imbalance, and exacerbation of psychiatric conditions. While the prevalence of insomnia in developed countries like the United States and Canada is known, there is limited epidemiologic evidence, and no reliable estimate for the disorder across the Americas — especially in low- and middle-income countries.

The researchers used published nation-specific data to estimate the prevalence of adult insomnia disorder across the 55 countries defined by the United Nations as comprising the Americas.

Based on the available data, the researchers estimated that about 123 million adults across the Americas have insomnia disorder (16.8%) — with greater prevalence in women (73 million, 19.5%) than in men (50 million, 14%).

The nations with the greatest burden of insomnia disorder are the United States (37 million), Brazil (29 million), and Mexico (16 million).

“While our study did not specifically investigate trends over time due to its scope, evidence from other research suggests that insomnia is becoming more prevalent over the long term. This growing trend highlights the increasing need for awareness and intervention in managing sleep health,” Dr. Benjafield said.

Insomnia is underdiagnosed and undertreated partly because of general lack of awareness about the importance of addressing sleep disorders and the fact that cognitive-behavioral therapy for insomnia (CBT-I), which is recommended as first-line treatment, is not widely accessible because of a shortage of trained CBT-I practitioners.

“Many individuals with insomnia struggle to find and receive this effective nonpharmacological treatment. Consequently, there is an overreliance on pharmaceutical solutions, which are ideally used for short-term management but are often extended due to the lack of alternatives. These medications can lead to dependency and other side effects,” Dr. Benjafield said.
 

Ask About Sleep

Insomnia symptoms are a “common presenting complaint in doctors’ offices in the United States. The percentages in this poster show that insomnia disorder has a similar, high percent prevalence across countries in the Americas,” Boris Gilyadov, MD, assistant professor of pulmonary, critical care, and sleep medicine at the Icahn School of Medicine at Mount Sinai in New York City, said in an interview.

“During preventive care visits and general physical exams, patients should be asked about the quality of their sleep. Patients may benefit from a referral to the sleep medicine clinic when appropriate,” said Dr. Gilyadov, who wasn’t involved in the study.

“CBT-I is the first-line treatment for chronic insomnia disorder and can be an effective treatment for most patients. An alternative to CBT-I, when it is not available, is digital CBT-I,” Dr. Gilyadov said.

“There are also behavioral therapies called BBT-I [brief behavioral treatment for insomnia] and ACT [acceptance and commitment therapy]. These are therapies that may be offered by psychologists who specialize in the treatment of chronic insomnia disorder,” Dr. Gilyadov noted.

The study was conducted in collaboration with medXcloud and funded by ResMed. Dr. Benjafield is an employee of ResMed. Dr. Gilyadov had no relevant disclosures.

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

HOUSTON — A new analysis highlights the high burden of insomnia across the Americas, with about 17% of adults suffering from this chronic sleep disorder.

“Our findings underscore the urgent need for enhanced clinical care pathways and policy interventions to effectively diagnose and treat insomnia. It is crucial to foster greater awareness of the critical role that sleep plays in overall health,” lead investigator Adam Benjafield, PhD, vice president for medical affairs at ResMed, Sydney, Australia, said in an interview.

“Insomnia not only affects individuals’ health and quality of life but also has broader implications for public health systems. Developing comprehensive care strategies and promoting education about sleep health could significantly improve outcomes for individuals suffering from insomnia disorder,” Dr. Benjafield said.

The findings were presented at SLEEP 2024: 38th Annual Meeting of the Associated Professional Sleep Societies.
 

Underdiagnosed, Undertreated

Sleep disruptions contribute to various medical problems, including cognitive impairment, reduced immune function, metabolic imbalance, and exacerbation of psychiatric conditions. While the prevalence of insomnia in developed countries like the United States and Canada is known, there is limited epidemiologic evidence, and no reliable estimate for the disorder across the Americas — especially in low- and middle-income countries.

The researchers used published nation-specific data to estimate the prevalence of adult insomnia disorder across the 55 countries defined by the United Nations as comprising the Americas.

Based on the available data, the researchers estimated that about 123 million adults across the Americas have insomnia disorder (16.8%) — with greater prevalence in women (73 million, 19.5%) than in men (50 million, 14%).

The nations with the greatest burden of insomnia disorder are the United States (37 million), Brazil (29 million), and Mexico (16 million).

“While our study did not specifically investigate trends over time due to its scope, evidence from other research suggests that insomnia is becoming more prevalent over the long term. This growing trend highlights the increasing need for awareness and intervention in managing sleep health,” Dr. Benjafield said.

Insomnia is underdiagnosed and undertreated partly because of general lack of awareness about the importance of addressing sleep disorders and the fact that cognitive-behavioral therapy for insomnia (CBT-I), which is recommended as first-line treatment, is not widely accessible because of a shortage of trained CBT-I practitioners.

“Many individuals with insomnia struggle to find and receive this effective nonpharmacological treatment. Consequently, there is an overreliance on pharmaceutical solutions, which are ideally used for short-term management but are often extended due to the lack of alternatives. These medications can lead to dependency and other side effects,” Dr. Benjafield said.
 

Ask About Sleep

Insomnia symptoms are a “common presenting complaint in doctors’ offices in the United States. The percentages in this poster show that insomnia disorder has a similar, high percent prevalence across countries in the Americas,” Boris Gilyadov, MD, assistant professor of pulmonary, critical care, and sleep medicine at the Icahn School of Medicine at Mount Sinai in New York City, said in an interview.

“During preventive care visits and general physical exams, patients should be asked about the quality of their sleep. Patients may benefit from a referral to the sleep medicine clinic when appropriate,” said Dr. Gilyadov, who wasn’t involved in the study.

“CBT-I is the first-line treatment for chronic insomnia disorder and can be an effective treatment for most patients. An alternative to CBT-I, when it is not available, is digital CBT-I,” Dr. Gilyadov said.

“There are also behavioral therapies called BBT-I [brief behavioral treatment for insomnia] and ACT [acceptance and commitment therapy]. These are therapies that may be offered by psychologists who specialize in the treatment of chronic insomnia disorder,” Dr. Gilyadov noted.

The study was conducted in collaboration with medXcloud and funded by ResMed. Dr. Benjafield is an employee of ResMed. Dr. Gilyadov had no relevant disclosures.

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

An intensive lifestyle intervention significantly improved cognition and function in many patients with mild cognitive impairment (MCI) or early dementia due to Alzheimer’s disease, in what authors said is the first randomized controlled trial of intensive lifestyle modification for patients diagnosed with Alzheimer’s disease. Results could help physicians address patients at risk of Alzheimer’s disease who reject relevant testing because they believe nothing can forestall development of the disease, the authors added. The study was published online in Alzheimer’s Research & Therapy.

Although technology allows probable Alzheimer’s disease diagnosis years before clinical symptoms appear, wrote investigators led by Dean Ornish, MD, of the Preventive Medicine Research Institute in Sausalito, California, “many people do not want to know if they are likely to get Alzheimer’s disease if they do not believe they can do anything about it. If intensive lifestyle changes may cause improvement in cognition and function in MCI or early dementia due to Alzheimer’s disease, then it is reasonable to think that these lifestyle changes may also help to prevent MCI or early dementia due to Alzheimer’s disease.” As with cardiovascular disease, the authors added, preventing Alzheimer’s disease might require less intensive lifestyle modifications than treating it.
 

Study Methodology

Investigators randomized 26 patients with Montréal Cognitive Assessment scores of 18 or higher to an intensive intervention involving nutrition, exercise, and stress management techniques. To improve adherence, the protocol included participants’ spouses or caregivers.

Two patients, both in the treatment group, withdrew over logistical concerns.

After 20 weeks, treated patients exhibited statistically significant differences in several key measures versus a 25-patient usual-care control group. Scores that improved in the intervention group and worsened among controls included the following:

• Clinical Global Impression of Change (CGIC, P = .001)
• Clinical Dementia Rating-Global (CDR-Global, -0.04, P = .037)
• Clinical Dementia Rating Sum of Boxes (CDR-SB, +0.08, P = .032)
• Alzheimer’s Disease Assessment Scale (ADAS-Cog, -1.01, P = .053)

The validity of these changes in cognition and function, and possible biological mechanisms of improvement, were supported by statistically significant improvements in several clinically relevant biomarkers versus controls, the investigators wrote. These biomarkers included Abeta42/40 ratio, HbA1c, insulin, and glycoprotein acetylation. “This information may also help in predicting which patients are more likely to show improvements in cognition and function by making these intensive lifestyle changes,” the authors added.

In primary analysis, the degree of lifestyle changes required to stop progression of MCI ranged from 71.4% (ADAS-Cog) to 120.6% (CDR-SB). “This helps to explain why other studies of less intensive lifestyle interventions may not have been sufficient to stop deterioration or improve cognition and function,” the authors wrote. Moreover, they added, variable adherence might explain why in the intervention group, 10 patients improved their CGIC scores, while the rest held static or worsened.
 

Caveats

Alzheimer’s Association Vice President of Medical and Scientific Relations Heather M. Snyder, PhD, said, “This is an interesting paper in an important area of research and adds to the growing body of literature on how behavior or lifestyle may be related to cognitive decline. However, because this is a small phase 2 study, it is important for this or similar work to be done in larger, more diverse populations and over a longer duration of the intervention.” She was not involved with the study but was asked to comment.

Investigators chose the 20-week duration, they explained, because control-group patients likely would not refrain from trying the lifestyle intervention beyond that timeframe. Perhaps more importantly, challenges created by the COVID-19 pandemic required researchers to cut planned enrollment in half, eliminate planned MRI and amyloid PET scans, and reduce the number of cognition and function tests.

Such shortcomings limit what neurologists can glean and generalize from the study, said Dr. Snyder. “That said,” she added, “it does demonstrate the potential of an intensive behavior/lifestyle intervention, and the importance of this sort of research in Alzheimer’s and dementia.” Although the complexity of the interventions makes these studies challenging, she added, “it is important that we continue to advance larger, longer studies in more representative study populations to develop specific recommendations.”
 

Further Study

The Alzheimer’s Association’s U.S. POINTER study is the first large-scale study in the United States to explore the impact of comprehensive lifestyle changes on cognitive health. About 2000 older adults at risk for cognitive decline are participating, from diverse locations across the country. More than 25% of participants come from groups typically underrepresented in dementia research, said Dr. Snyder. Initial results are expected in summer 2025.

Future research also should explore reasons (beyond adherence) why some patients respond to lifestyle interventions better than others, and the potential synergy of lifestyle changes with drug therapies, wrote Dr. Ornish and colleagues.

“For now,” said Dr. Snyder, “there is an opportunity for providers to incorporate or expand messaging with their patients and families about the habits that they can incorporate into their daily lives. The Alzheimer’s Association offers 10 Healthy Habits for Your Brain — everyday actions that can make a difference for your brain health.”

Investigators received study funding from more than two dozen charitable foundations and other organizations. Dr. Snyder is a full-time employee of the Alzheimer’s Association and in this role, serves on the leadership team of the U.S. POINTER study. Her partner works for Abbott in an unrelated field. 

An intensive lifestyle intervention significantly improved cognition and function in many patients with mild cognitive impairment (MCI) or early dementia due to Alzheimer’s disease, in what authors said is the first randomized controlled trial of intensive lifestyle modification for patients diagnosed with Alzheimer’s disease. Results could help physicians address patients at risk of Alzheimer’s disease who reject relevant testing because they believe nothing can forestall development of the disease, the authors added. The study was published online in Alzheimer’s Research & Therapy.

Although technology allows probable Alzheimer’s disease diagnosis years before clinical symptoms appear, wrote investigators led by Dean Ornish, MD, of the Preventive Medicine Research Institute in Sausalito, California, “many people do not want to know if they are likely to get Alzheimer’s disease if they do not believe they can do anything about it. If intensive lifestyle changes may cause improvement in cognition and function in MCI or early dementia due to Alzheimer’s disease, then it is reasonable to think that these lifestyle changes may also help to prevent MCI or early dementia due to Alzheimer’s disease.” As with cardiovascular disease, the authors added, preventing Alzheimer’s disease might require less intensive lifestyle modifications than treating it.
 

Study Methodology

Investigators randomized 26 patients with Montréal Cognitive Assessment scores of 18 or higher to an intensive intervention involving nutrition, exercise, and stress management techniques. To improve adherence, the protocol included participants’ spouses or caregivers.

Two patients, both in the treatment group, withdrew over logistical concerns.

After 20 weeks, treated patients exhibited statistically significant differences in several key measures versus a 25-patient usual-care control group. Scores that improved in the intervention group and worsened among controls included the following:

• Clinical Global Impression of Change (CGIC, P = .001)
• Clinical Dementia Rating-Global (CDR-Global, -0.04, P = .037)
• Clinical Dementia Rating Sum of Boxes (CDR-SB, +0.08, P = .032)
• Alzheimer’s Disease Assessment Scale (ADAS-Cog, -1.01, P = .053)

The validity of these changes in cognition and function, and possible biological mechanisms of improvement, were supported by statistically significant improvements in several clinically relevant biomarkers versus controls, the investigators wrote. These biomarkers included Abeta42/40 ratio, HbA1c, insulin, and glycoprotein acetylation. “This information may also help in predicting which patients are more likely to show improvements in cognition and function by making these intensive lifestyle changes,” the authors added.

In primary analysis, the degree of lifestyle changes required to stop progression of MCI ranged from 71.4% (ADAS-Cog) to 120.6% (CDR-SB). “This helps to explain why other studies of less intensive lifestyle interventions may not have been sufficient to stop deterioration or improve cognition and function,” the authors wrote. Moreover, they added, variable adherence might explain why in the intervention group, 10 patients improved their CGIC scores, while the rest held static or worsened.
 

Caveats

Alzheimer’s Association Vice President of Medical and Scientific Relations Heather M. Snyder, PhD, said, “This is an interesting paper in an important area of research and adds to the growing body of literature on how behavior or lifestyle may be related to cognitive decline. However, because this is a small phase 2 study, it is important for this or similar work to be done in larger, more diverse populations and over a longer duration of the intervention.” She was not involved with the study but was asked to comment.

Investigators chose the 20-week duration, they explained, because control-group patients likely would not refrain from trying the lifestyle intervention beyond that timeframe. Perhaps more importantly, challenges created by the COVID-19 pandemic required researchers to cut planned enrollment in half, eliminate planned MRI and amyloid PET scans, and reduce the number of cognition and function tests.

Such shortcomings limit what neurologists can glean and generalize from the study, said Dr. Snyder. “That said,” she added, “it does demonstrate the potential of an intensive behavior/lifestyle intervention, and the importance of this sort of research in Alzheimer’s and dementia.” Although the complexity of the interventions makes these studies challenging, she added, “it is important that we continue to advance larger, longer studies in more representative study populations to develop specific recommendations.”
 

Further Study

The Alzheimer’s Association’s U.S. POINTER study is the first large-scale study in the United States to explore the impact of comprehensive lifestyle changes on cognitive health. About 2000 older adults at risk for cognitive decline are participating, from diverse locations across the country. More than 25% of participants come from groups typically underrepresented in dementia research, said Dr. Snyder. Initial results are expected in summer 2025.

Future research also should explore reasons (beyond adherence) why some patients respond to lifestyle interventions better than others, and the potential synergy of lifestyle changes with drug therapies, wrote Dr. Ornish and colleagues.

“For now,” said Dr. Snyder, “there is an opportunity for providers to incorporate or expand messaging with their patients and families about the habits that they can incorporate into their daily lives. The Alzheimer’s Association offers 10 Healthy Habits for Your Brain — everyday actions that can make a difference for your brain health.”

Investigators received study funding from more than two dozen charitable foundations and other organizations. Dr. Snyder is a full-time employee of the Alzheimer’s Association and in this role, serves on the leadership team of the U.S. POINTER study. Her partner works for Abbott in an unrelated field. 

An intensive lifestyle intervention significantly improved cognition and function in many patients with mild cognitive impairment (MCI) or early dementia due to Alzheimer’s disease, in what authors said is the first randomized controlled trial of intensive lifestyle modification for patients diagnosed with Alzheimer’s disease. Results could help physicians address patients at risk of Alzheimer’s disease who reject relevant testing because they believe nothing can forestall development of the disease, the authors added. The study was published online in Alzheimer’s Research & Therapy.

Although technology allows probable Alzheimer’s disease diagnosis years before clinical symptoms appear, wrote investigators led by Dean Ornish, MD, of the Preventive Medicine Research Institute in Sausalito, California, “many people do not want to know if they are likely to get Alzheimer’s disease if they do not believe they can do anything about it. If intensive lifestyle changes may cause improvement in cognition and function in MCI or early dementia due to Alzheimer’s disease, then it is reasonable to think that these lifestyle changes may also help to prevent MCI or early dementia due to Alzheimer’s disease.” As with cardiovascular disease, the authors added, preventing Alzheimer’s disease might require less intensive lifestyle modifications than treating it.
 

Study Methodology

Investigators randomized 26 patients with Montréal Cognitive Assessment scores of 18 or higher to an intensive intervention involving nutrition, exercise, and stress management techniques. To improve adherence, the protocol included participants’ spouses or caregivers.

Two patients, both in the treatment group, withdrew over logistical concerns.

After 20 weeks, treated patients exhibited statistically significant differences in several key measures versus a 25-patient usual-care control group. Scores that improved in the intervention group and worsened among controls included the following:

• Clinical Global Impression of Change (CGIC, P = .001)
• Clinical Dementia Rating-Global (CDR-Global, -0.04, P = .037)
• Clinical Dementia Rating Sum of Boxes (CDR-SB, +0.08, P = .032)
• Alzheimer’s Disease Assessment Scale (ADAS-Cog, -1.01, P = .053)

The validity of these changes in cognition and function, and possible biological mechanisms of improvement, were supported by statistically significant improvements in several clinically relevant biomarkers versus controls, the investigators wrote. These biomarkers included Abeta42/40 ratio, HbA1c, insulin, and glycoprotein acetylation. “This information may also help in predicting which patients are more likely to show improvements in cognition and function by making these intensive lifestyle changes,” the authors added.

In primary analysis, the degree of lifestyle changes required to stop progression of MCI ranged from 71.4% (ADAS-Cog) to 120.6% (CDR-SB). “This helps to explain why other studies of less intensive lifestyle interventions may not have been sufficient to stop deterioration or improve cognition and function,” the authors wrote. Moreover, they added, variable adherence might explain why in the intervention group, 10 patients improved their CGIC scores, while the rest held static or worsened.
 

Caveats

Alzheimer’s Association Vice President of Medical and Scientific Relations Heather M. Snyder, PhD, said, “This is an interesting paper in an important area of research and adds to the growing body of literature on how behavior or lifestyle may be related to cognitive decline. However, because this is a small phase 2 study, it is important for this or similar work to be done in larger, more diverse populations and over a longer duration of the intervention.” She was not involved with the study but was asked to comment.

Investigators chose the 20-week duration, they explained, because control-group patients likely would not refrain from trying the lifestyle intervention beyond that timeframe. Perhaps more importantly, challenges created by the COVID-19 pandemic required researchers to cut planned enrollment in half, eliminate planned MRI and amyloid PET scans, and reduce the number of cognition and function tests.

Such shortcomings limit what neurologists can glean and generalize from the study, said Dr. Snyder. “That said,” she added, “it does demonstrate the potential of an intensive behavior/lifestyle intervention, and the importance of this sort of research in Alzheimer’s and dementia.” Although the complexity of the interventions makes these studies challenging, she added, “it is important that we continue to advance larger, longer studies in more representative study populations to develop specific recommendations.”
 

Further Study

The Alzheimer’s Association’s U.S. POINTER study is the first large-scale study in the United States to explore the impact of comprehensive lifestyle changes on cognitive health. About 2000 older adults at risk for cognitive decline are participating, from diverse locations across the country. More than 25% of participants come from groups typically underrepresented in dementia research, said Dr. Snyder. Initial results are expected in summer 2025.

Future research also should explore reasons (beyond adherence) why some patients respond to lifestyle interventions better than others, and the potential synergy of lifestyle changes with drug therapies, wrote Dr. Ornish and colleagues.

“For now,” said Dr. Snyder, “there is an opportunity for providers to incorporate or expand messaging with their patients and families about the habits that they can incorporate into their daily lives. The Alzheimer’s Association offers 10 Healthy Habits for Your Brain — everyday actions that can make a difference for your brain health.”

Investigators received study funding from more than two dozen charitable foundations and other organizations. Dr. Snyder is a full-time employee of the Alzheimer’s Association and in this role, serves on the leadership team of the U.S. POINTER study. Her partner works for Abbott in an unrelated field. 

The U.S. Preventive Services Task Force (USPSTF) is recommending that clinicians provide comprehensive, intensive behavioral interventions for children 6 years and older who have a high body mass index (BMI) at or above the 95th percentile (for age and sex) or refer those patients to an appropriate provider.

One in five children (19.7%) and adolescents ages 2-19 in the United States are at or above this range, based on Centers for Disease Control and Prevention growth charts from 2000, the task force wrote in its statement. The rate of BMI increase nearly doubled in this age group during the COVID pandemic, compared with prepandemic levels.

Publishing their recommendations in JAMA, the task force, with lead author Wanda K. Nicholson, MD, MPH, MBA, with the Milken Institute of Public Health, George Washington University, Washington, D.C., also noted that the prevalence of high BMI increases with age and rates are higher among children from lower-income families. Rates are also higher in Hispanic/Latino, Native American/Alaska Native and non-Hispanic Black children.
 

At Least 26 Hours of Interventions

It is important that children and adolescents 6 years or older with a high BMI receive intensive interventions for at least 26 contact hours for up to a year, as evidence showed that was the threshold for weight loss, the task force said.

Based on its evidence review, the USPSTF assigned this recommendation a B grade indicating “moderate certainty ... of moderate net benefit.” The task force analyzed 50 randomized clinical trials (RCTs) (n = 8,798) that examined behavioral interventions. They also analyzed eight trials that assessed pharmacotherapy interventions: liraglutide (three RCTs), semaglutide (one RCT), orlistat (two RCTs) and phentermine/topiramate (two RCTs). Five trials included behavioral counseling with the medication or placebo.

These new recommendations also reaffirm the task force’s 2010 and 2023 recommendations.

Effective interventions had multiple components. They included interventions targeting both the parent and child (separately, together or both); group sessions; information about healthy eating, information on reading food labels, and safe exercising; and interventions for encouraging behavioral changes, such as monitoring food intake and problem solving, changing physical activity behaviors, and goal setting.

These types of interventions are often delivered by multidisciplinary teams, including pediatricians, exercise physiologists or physical therapists, dietitians, psychologists, social workers, or other behavioral specialists.
 

Personalizing Treatment for Optimal Benefit

“The time to prevent and intervene on childhood obesity is now, and the need to start with ILT [intensive lifestyle therapy] is clear,” Roohi Y. Kharofa, MD, with the department of pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio, and colleagues wrote in a related editorial.

However, the editorialists noted it will be important to personalize the level of interventions as ILT won’t be enough for some to prevent serious outcomes. For such patients, bariatric surgery or pharmacotherapy may need to be considered as well.
 

Ways to Reach the 26 Hours

Dr. Kharofa and coauthors pointed out that, while the threshold of at least 26 contact hours is associated with significant improvement in BMI (mean BMI difference, –0.8; 95% CI, –1.2 to –0.4), and while it’s important to now have an evidence-based threshold, the number may be disheartening given limits on clinicians, staff, and resources. The key may be prescribing physical activity sessions outside the health system.

For patients not interested in group sports or burdened by participation fees, collaboration with local community organizations, such as the YMCA or the Boys & Girls Club, could be arranged, the authors suggested.

“The inability to attain 26 hours should not deter patients or practitioners from participating in, referring to, or implementing obesity interventions. Rather, clinical teams and families should work together to maximize intervention dose using clinical and community programs synergistically,” they wrote.

They noted that the USPSTF in this 2024 update found “inadequate evidence on the benefits of pharmacotherapy in youth with obesity, encouraging clinicians to use ILT as the primary intervention.”
 

What About Medications?

New since the previous USPSTF review, several new medications have been approved for weight loss in pediatric populations, Elizabeth A. O’Connor, PhD, with The Center for Health Research, Kaiser Permanente Northwest, Portland, Oregon, and colleagues noted in their updated evidence report.

They noted that the 2023 Clinical Practice Guideline developed by the American Academy of Pediatrics states that clinicians “may offer children ages 8 through 11 years of age with obesity weight loss pharmacotherapy, according to medication indications, risks, and benefits, as an adjunct to health behavior and lifestyle treatment.”

However, Dr. O’Connor and coauthors wrote, the evidence base for each agent is limited and there is no information in the literature supporting their findings on harms of medication use beyond 17 months.

“For pharmacotherapy, when evidence was available on weight maintenance after discontinuation, weight rebounded quickly after medication use ended,” the authors wrote. “This suggests that long-term use is required for weight maintenance and underscores the need for evidence about potential harms from long-term use.”
 

Changes in Investment, Food, Government Priorities Are Needed

In a separate accompanying editorial, Thomas N. Robinson, MD, MPH, with Stanford University’s Center for Healthy Weight and General Pediatrics Department in Palo Alto, California, and Sarah C. Armstrong, MD, with the Duke Center for Childhood Obesity Research, Chapel Hill, North Carolina, wrote that experience to date has shown that current approaches aren’t working and, in fact, pediatric obesity rates are worsening.

“After nearly 15 years of authoritative, evidence-backed USPSTF recommendations for effective interventions for children with high BMI, it is long past time to implement them,” they wrote.

But changes will need to go far beyond clinicians’ offices and priorities must change at local, state, and federal levels, Dr. Robinson and Dr. Armstrong wrote. A shift in priorities is needed to make screening and behavioral interventions available to all children and teens with obesity.

Public policies, they wrote, must address larger issues, such as food content and availability of healthy foods, transportation innovations, and ways to make active lifestyles available equitably.

The authors said that strategies may include taxing sugary drinks, regulating marketing of unhealthful foods, crafting legislation to regulate the nutritional content of school meals, and creating policies to reduce poverty and address social drivers of health.

“A synergistic combination of effective clinical care, as recommended by the USPSTF, and public policy interventions is critically needed to turn the tide on childhood obesity,” Dr. Robinson and Dr. Armstrong wrote.

The full recommendation statement is available at the USPSTF website or the JAMA website.

One coauthor of the recommendation statement reported receiving publications and federal grand funding to his institution for the relationship between obesity and the potential effect of nutrition policy interventions on cardiovascular disease and cancer and for a meta-analysis of the effect of dietary counseling for weight loss. The authors of the evidence report had no relevant conflicts of interest. Dr. Kharofa reported receiving grants from Rhythm Pharmaceuticals outside the submitted work. Dr. Robinson has served on the scientific advisory board of WW International (through December 2022). Dr. Armstrong has served as chair of the Section on Obesity, American Academy of Pediatrics; and is a coauthor of the Clinical Practice Guidelines for the Evaluation and Treatment of Children and Adolescents with Obesity.
 

The U.S. Preventive Services Task Force (USPSTF) is recommending that clinicians provide comprehensive, intensive behavioral interventions for children 6 years and older who have a high body mass index (BMI) at or above the 95th percentile (for age and sex) or refer those patients to an appropriate provider.

One in five children (19.7%) and adolescents ages 2-19 in the United States are at or above this range, based on Centers for Disease Control and Prevention growth charts from 2000, the task force wrote in its statement. The rate of BMI increase nearly doubled in this age group during the COVID pandemic, compared with prepandemic levels.

Publishing their recommendations in JAMA, the task force, with lead author Wanda K. Nicholson, MD, MPH, MBA, with the Milken Institute of Public Health, George Washington University, Washington, D.C., also noted that the prevalence of high BMI increases with age and rates are higher among children from lower-income families. Rates are also higher in Hispanic/Latino, Native American/Alaska Native and non-Hispanic Black children.
 

At Least 26 Hours of Interventions

It is important that children and adolescents 6 years or older with a high BMI receive intensive interventions for at least 26 contact hours for up to a year, as evidence showed that was the threshold for weight loss, the task force said.

Based on its evidence review, the USPSTF assigned this recommendation a B grade indicating “moderate certainty ... of moderate net benefit.” The task force analyzed 50 randomized clinical trials (RCTs) (n = 8,798) that examined behavioral interventions. They also analyzed eight trials that assessed pharmacotherapy interventions: liraglutide (three RCTs), semaglutide (one RCT), orlistat (two RCTs) and phentermine/topiramate (two RCTs). Five trials included behavioral counseling with the medication or placebo.

These new recommendations also reaffirm the task force’s 2010 and 2023 recommendations.

Effective interventions had multiple components. They included interventions targeting both the parent and child (separately, together or both); group sessions; information about healthy eating, information on reading food labels, and safe exercising; and interventions for encouraging behavioral changes, such as monitoring food intake and problem solving, changing physical activity behaviors, and goal setting.

These types of interventions are often delivered by multidisciplinary teams, including pediatricians, exercise physiologists or physical therapists, dietitians, psychologists, social workers, or other behavioral specialists.
 

Personalizing Treatment for Optimal Benefit

“The time to prevent and intervene on childhood obesity is now, and the need to start with ILT [intensive lifestyle therapy] is clear,” Roohi Y. Kharofa, MD, with the department of pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio, and colleagues wrote in a related editorial.

However, the editorialists noted it will be important to personalize the level of interventions as ILT won’t be enough for some to prevent serious outcomes. For such patients, bariatric surgery or pharmacotherapy may need to be considered as well.
 

Ways to Reach the 26 Hours

Dr. Kharofa and coauthors pointed out that, while the threshold of at least 26 contact hours is associated with significant improvement in BMI (mean BMI difference, –0.8; 95% CI, –1.2 to –0.4), and while it’s important to now have an evidence-based threshold, the number may be disheartening given limits on clinicians, staff, and resources. The key may be prescribing physical activity sessions outside the health system.

For patients not interested in group sports or burdened by participation fees, collaboration with local community organizations, such as the YMCA or the Boys & Girls Club, could be arranged, the authors suggested.

“The inability to attain 26 hours should not deter patients or practitioners from participating in, referring to, or implementing obesity interventions. Rather, clinical teams and families should work together to maximize intervention dose using clinical and community programs synergistically,” they wrote.

They noted that the USPSTF in this 2024 update found “inadequate evidence on the benefits of pharmacotherapy in youth with obesity, encouraging clinicians to use ILT as the primary intervention.”
 

What About Medications?

New since the previous USPSTF review, several new medications have been approved for weight loss in pediatric populations, Elizabeth A. O’Connor, PhD, with The Center for Health Research, Kaiser Permanente Northwest, Portland, Oregon, and colleagues noted in their updated evidence report.

They noted that the 2023 Clinical Practice Guideline developed by the American Academy of Pediatrics states that clinicians “may offer children ages 8 through 11 years of age with obesity weight loss pharmacotherapy, according to medication indications, risks, and benefits, as an adjunct to health behavior and lifestyle treatment.”

However, Dr. O’Connor and coauthors wrote, the evidence base for each agent is limited and there is no information in the literature supporting their findings on harms of medication use beyond 17 months.

“For pharmacotherapy, when evidence was available on weight maintenance after discontinuation, weight rebounded quickly after medication use ended,” the authors wrote. “This suggests that long-term use is required for weight maintenance and underscores the need for evidence about potential harms from long-term use.”
 

Changes in Investment, Food, Government Priorities Are Needed

In a separate accompanying editorial, Thomas N. Robinson, MD, MPH, with Stanford University’s Center for Healthy Weight and General Pediatrics Department in Palo Alto, California, and Sarah C. Armstrong, MD, with the Duke Center for Childhood Obesity Research, Chapel Hill, North Carolina, wrote that experience to date has shown that current approaches aren’t working and, in fact, pediatric obesity rates are worsening.

“After nearly 15 years of authoritative, evidence-backed USPSTF recommendations for effective interventions for children with high BMI, it is long past time to implement them,” they wrote.

But changes will need to go far beyond clinicians’ offices and priorities must change at local, state, and federal levels, Dr. Robinson and Dr. Armstrong wrote. A shift in priorities is needed to make screening and behavioral interventions available to all children and teens with obesity.

Public policies, they wrote, must address larger issues, such as food content and availability of healthy foods, transportation innovations, and ways to make active lifestyles available equitably.

The authors said that strategies may include taxing sugary drinks, regulating marketing of unhealthful foods, crafting legislation to regulate the nutritional content of school meals, and creating policies to reduce poverty and address social drivers of health.

“A synergistic combination of effective clinical care, as recommended by the USPSTF, and public policy interventions is critically needed to turn the tide on childhood obesity,” Dr. Robinson and Dr. Armstrong wrote.

The full recommendation statement is available at the USPSTF website or the JAMA website.

One coauthor of the recommendation statement reported receiving publications and federal grand funding to his institution for the relationship between obesity and the potential effect of nutrition policy interventions on cardiovascular disease and cancer and for a meta-analysis of the effect of dietary counseling for weight loss. The authors of the evidence report had no relevant conflicts of interest. Dr. Kharofa reported receiving grants from Rhythm Pharmaceuticals outside the submitted work. Dr. Robinson has served on the scientific advisory board of WW International (through December 2022). Dr. Armstrong has served as chair of the Section on Obesity, American Academy of Pediatrics; and is a coauthor of the Clinical Practice Guidelines for the Evaluation and Treatment of Children and Adolescents with Obesity.
 

The U.S. Preventive Services Task Force (USPSTF) is recommending that clinicians provide comprehensive, intensive behavioral interventions for children 6 years and older who have a high body mass index (BMI) at or above the 95th percentile (for age and sex) or refer those patients to an appropriate provider.

One in five children (19.7%) and adolescents ages 2-19 in the United States are at or above this range, based on Centers for Disease Control and Prevention growth charts from 2000, the task force wrote in its statement. The rate of BMI increase nearly doubled in this age group during the COVID pandemic, compared with prepandemic levels.

Publishing their recommendations in JAMA, the task force, with lead author Wanda K. Nicholson, MD, MPH, MBA, with the Milken Institute of Public Health, George Washington University, Washington, D.C., also noted that the prevalence of high BMI increases with age and rates are higher among children from lower-income families. Rates are also higher in Hispanic/Latino, Native American/Alaska Native and non-Hispanic Black children.
 

At Least 26 Hours of Interventions

It is important that children and adolescents 6 years or older with a high BMI receive intensive interventions for at least 26 contact hours for up to a year, as evidence showed that was the threshold for weight loss, the task force said.

Based on its evidence review, the USPSTF assigned this recommendation a B grade indicating “moderate certainty ... of moderate net benefit.” The task force analyzed 50 randomized clinical trials (RCTs) (n = 8,798) that examined behavioral interventions. They also analyzed eight trials that assessed pharmacotherapy interventions: liraglutide (three RCTs), semaglutide (one RCT), orlistat (two RCTs) and phentermine/topiramate (two RCTs). Five trials included behavioral counseling with the medication or placebo.

These new recommendations also reaffirm the task force’s 2010 and 2023 recommendations.

Effective interventions had multiple components. They included interventions targeting both the parent and child (separately, together or both); group sessions; information about healthy eating, information on reading food labels, and safe exercising; and interventions for encouraging behavioral changes, such as monitoring food intake and problem solving, changing physical activity behaviors, and goal setting.

These types of interventions are often delivered by multidisciplinary teams, including pediatricians, exercise physiologists or physical therapists, dietitians, psychologists, social workers, or other behavioral specialists.
 

Personalizing Treatment for Optimal Benefit

“The time to prevent and intervene on childhood obesity is now, and the need to start with ILT [intensive lifestyle therapy] is clear,” Roohi Y. Kharofa, MD, with the department of pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio, and colleagues wrote in a related editorial.

However, the editorialists noted it will be important to personalize the level of interventions as ILT won’t be enough for some to prevent serious outcomes. For such patients, bariatric surgery or pharmacotherapy may need to be considered as well.
 

Ways to Reach the 26 Hours

Dr. Kharofa and coauthors pointed out that, while the threshold of at least 26 contact hours is associated with significant improvement in BMI (mean BMI difference, –0.8; 95% CI, –1.2 to –0.4), and while it’s important to now have an evidence-based threshold, the number may be disheartening given limits on clinicians, staff, and resources. The key may be prescribing physical activity sessions outside the health system.

For patients not interested in group sports or burdened by participation fees, collaboration with local community organizations, such as the YMCA or the Boys & Girls Club, could be arranged, the authors suggested.

“The inability to attain 26 hours should not deter patients or practitioners from participating in, referring to, or implementing obesity interventions. Rather, clinical teams and families should work together to maximize intervention dose using clinical and community programs synergistically,” they wrote.

They noted that the USPSTF in this 2024 update found “inadequate evidence on the benefits of pharmacotherapy in youth with obesity, encouraging clinicians to use ILT as the primary intervention.”
 

What About Medications?

New since the previous USPSTF review, several new medications have been approved for weight loss in pediatric populations, Elizabeth A. O’Connor, PhD, with The Center for Health Research, Kaiser Permanente Northwest, Portland, Oregon, and colleagues noted in their updated evidence report.

They noted that the 2023 Clinical Practice Guideline developed by the American Academy of Pediatrics states that clinicians “may offer children ages 8 through 11 years of age with obesity weight loss pharmacotherapy, according to medication indications, risks, and benefits, as an adjunct to health behavior and lifestyle treatment.”

However, Dr. O’Connor and coauthors wrote, the evidence base for each agent is limited and there is no information in the literature supporting their findings on harms of medication use beyond 17 months.

“For pharmacotherapy, when evidence was available on weight maintenance after discontinuation, weight rebounded quickly after medication use ended,” the authors wrote. “This suggests that long-term use is required for weight maintenance and underscores the need for evidence about potential harms from long-term use.”
 

Changes in Investment, Food, Government Priorities Are Needed

In a separate accompanying editorial, Thomas N. Robinson, MD, MPH, with Stanford University’s Center for Healthy Weight and General Pediatrics Department in Palo Alto, California, and Sarah C. Armstrong, MD, with the Duke Center for Childhood Obesity Research, Chapel Hill, North Carolina, wrote that experience to date has shown that current approaches aren’t working and, in fact, pediatric obesity rates are worsening.

“After nearly 15 years of authoritative, evidence-backed USPSTF recommendations for effective interventions for children with high BMI, it is long past time to implement them,” they wrote.

But changes will need to go far beyond clinicians’ offices and priorities must change at local, state, and federal levels, Dr. Robinson and Dr. Armstrong wrote. A shift in priorities is needed to make screening and behavioral interventions available to all children and teens with obesity.

Public policies, they wrote, must address larger issues, such as food content and availability of healthy foods, transportation innovations, and ways to make active lifestyles available equitably.

The authors said that strategies may include taxing sugary drinks, regulating marketing of unhealthful foods, crafting legislation to regulate the nutritional content of school meals, and creating policies to reduce poverty and address social drivers of health.

“A synergistic combination of effective clinical care, as recommended by the USPSTF, and public policy interventions is critically needed to turn the tide on childhood obesity,” Dr. Robinson and Dr. Armstrong wrote.

The full recommendation statement is available at the USPSTF website or the JAMA website.

One coauthor of the recommendation statement reported receiving publications and federal grand funding to his institution for the relationship between obesity and the potential effect of nutrition policy interventions on cardiovascular disease and cancer and for a meta-analysis of the effect of dietary counseling for weight loss. The authors of the evidence report had no relevant conflicts of interest. Dr. Kharofa reported receiving grants from Rhythm Pharmaceuticals outside the submitted work. Dr. Robinson has served on the scientific advisory board of WW International (through December 2022). Dr. Armstrong has served as chair of the Section on Obesity, American Academy of Pediatrics; and is a coauthor of the Clinical Practice Guidelines for the Evaluation and Treatment of Children and Adolescents with Obesity.