Pediatric News

WASHINGTON – The LIBERTY AD open-label extension study of dupilumab is closing after 5 years with the small number of remaining patients showing stable and sustained improvements in skin lesions and pruritus and no new emergent side effects, Lisa Beck, MD, reported during a late-breaking session at the annual Revolutionizing Atopic Dermatitis conference.

Other recent research on the biologic has shown that it improves lesional skin barrier function and rapidly reduces the abundance of Staphylococcus aureus on lesional skin, Dr. Beck, professor of dermatology at the University of Rochester (N.Y.), said during another session at the meeting on long-term control of AD. Dr. Beck directs a laboratory at the University of Rochester Medical Center that focuses on understanding AD and is involved in the National Institute of Allergy and Infectious Diseases (NIAID)-funded Atopic Dermatitis Research Network (ADRN).

The LIBERTY AD open-label extension (OLE) study was a phase 3 trial of 2,677 adults with moderate to severe AD who had participated in previous dupilumab clinical trials and were treated with 300 mg dupilumab weekly or every other week. Concomitant treatments were permitted, including topical corticosteroids and topical calcineurin inhibitors. (The proportion of patients dosed on an every-other-week or weekly dosing schedule was not available.)

Of 334 patients (12.5%) who remained in the trial at week 260, or 5 years, 88.9% achieved at least a 75% improvement in lesion extent and severity (Eczema Area and Severity Index [EASI]-75), and 76.2% achieved an EASI-90. The proportion achieving at least a 4-point reduction in the Peak Pruritus Numerical Rating Scale (NRS) or a score of 0 was 66.5%. At 5 years, improvements “seem very stable,” with “no loss in efficacy,” Dr. Beck said.

The majority of patients who withdrew from the open-label extension trial did so because the study was terminated at their site or because of the drug’s approval and commercialization – not for a medical reason, Dr. Beck said. Over the course of the extension trial, 4% of those enrolled withdrew because of adverse events and about 2% withdrew because of lack of efficacy.

Safety of dupilumab

The extension trial lacked a control arm, so Dr. Beck and her colleagues compared safety results to those in the final data set for patients in the LIBERTY AD CHRONOS study who received dupilumab 300 mg weekly with concomitant corticosteroids. The CHRONOS study was a 1-year randomized, double-blinded placebo-controlled phase 3 trial.

The exposure-adjusted incidence rate of severe treatment-emergent adverse events (TEAE) was lower at the close of the extension trial (5 patients/100 patient years [PY]) than at the end of the CHRONOS study (5.9 patients/100 PY). The incidence of serious adverse events related to treatment was 0.6 patients/100 PY in the final open label extension study data set, compared with 0.7 patients/100 PY in the CHRONOS final data set.

Adverse event rates “are really, if anything, slightly less in the OLE study versus the CHRONOS study, which was 1 year of treatment,” Dr. Beck said. And “no new adverse events have emerged.”

During a question and answer period, Dr. Beck pointed out that existing and future “real world” registries of patients on dupilumab and other new therapies will better inform dermatologists of adverse events than clinical trials have done.
 

Ocular surface disease

In a separate presentation on the safety of biologics, Andrew Blauvelt, MD, MBA, of the Oregon Medical Research Center, Portland, said that in routine care, ocular surface disease is the most predominant side effect associated with dupilumab. “We don’t know the mechanism of action. But it’s not infectious, it’s not pink eye, and importantly, it’s not allergic conjunctivitis,” he said, noting that the spectrum of disease ranges from dry eye and eye itching to “frank conjunctivitis” and keratitis.

Most cases are mild to moderate and can often be managed with lubricating eye drops and periodic use of corticosteroid eye drops. Co-management with an ophthalmologist is often advisable, he said.

Dupilumab-associated erythema/eczema of the face was “not seen much” in clinical trials but is also being reported in the literature, largely by European researchers, Dr. Blauvelt said. “We hear a lot about red face, but I don’t think it’s much of an issue,” he said. “Most of the time, in my experience, it will [reflect] breakthrough residual AD, and I like to treat it with non-steroidal topicals.”

Occasionally, the withdrawal of steroids or allergic contact dermatitis are at play, Dr. Blauvelt said. “If you see red face in a person on dupilumab, use your clinical prowess, do a differential diagnosis, and treat accordingly.”
 

Effect on S. aureus

The vast majority of adults with moderate to severe AD have skin colonization with S. aureus, Dr. Beck said during the session on long-term control of AD. The presence of S. aureus in skin cultures correlates strongly with AD severity, type 2 immunity polarization, skin barrier disruption, and allergen sensitization, she said.

“So if we could do something to get rid of the staph and keep it away, one might imagine that would help” control the AD disease process, she said.

An ADRN study evaluated S. aureus in the skin of 71 patients who were randomized to receive dupilumab or placebo and found a “profound” effect of the biologic. “We were truly shocked by how quickly we saw a reduction in Staph aureus ... in lesional skin as early as 3 days” into treatment with dupilumab, she said of the unpublished findings. “And there is a pretty nice association with improvement in disease severity.”

Dr. Beck reported consultancy/advisory board work with Regeneron, Sanofi/Genzyme, among other disclosures. Dr. Blauvelt reported consultancy/advisory board work for Regeneron and Sanofi Genzyme and has received speakers bureau/honoraria for non-CME work for Regeneron and Sanofi, among other disclosures.

WASHINGTON – The LIBERTY AD open-label extension study of dupilumab is closing after 5 years with the small number of remaining patients showing stable and sustained improvements in skin lesions and pruritus and no new emergent side effects, Lisa Beck, MD, reported during a late-breaking session at the annual Revolutionizing Atopic Dermatitis conference.

Other recent research on the biologic has shown that it improves lesional skin barrier function and rapidly reduces the abundance of Staphylococcus aureus on lesional skin, Dr. Beck, professor of dermatology at the University of Rochester (N.Y.), said during another session at the meeting on long-term control of AD. Dr. Beck directs a laboratory at the University of Rochester Medical Center that focuses on understanding AD and is involved in the National Institute of Allergy and Infectious Diseases (NIAID)-funded Atopic Dermatitis Research Network (ADRN).

The LIBERTY AD open-label extension (OLE) study was a phase 3 trial of 2,677 adults with moderate to severe AD who had participated in previous dupilumab clinical trials and were treated with 300 mg dupilumab weekly or every other week. Concomitant treatments were permitted, including topical corticosteroids and topical calcineurin inhibitors. (The proportion of patients dosed on an every-other-week or weekly dosing schedule was not available.)

Of 334 patients (12.5%) who remained in the trial at week 260, or 5 years, 88.9% achieved at least a 75% improvement in lesion extent and severity (Eczema Area and Severity Index [EASI]-75), and 76.2% achieved an EASI-90. The proportion achieving at least a 4-point reduction in the Peak Pruritus Numerical Rating Scale (NRS) or a score of 0 was 66.5%. At 5 years, improvements “seem very stable,” with “no loss in efficacy,” Dr. Beck said.

The majority of patients who withdrew from the open-label extension trial did so because the study was terminated at their site or because of the drug’s approval and commercialization – not for a medical reason, Dr. Beck said. Over the course of the extension trial, 4% of those enrolled withdrew because of adverse events and about 2% withdrew because of lack of efficacy.

Safety of dupilumab

The extension trial lacked a control arm, so Dr. Beck and her colleagues compared safety results to those in the final data set for patients in the LIBERTY AD CHRONOS study who received dupilumab 300 mg weekly with concomitant corticosteroids. The CHRONOS study was a 1-year randomized, double-blinded placebo-controlled phase 3 trial.

The exposure-adjusted incidence rate of severe treatment-emergent adverse events (TEAE) was lower at the close of the extension trial (5 patients/100 patient years [PY]) than at the end of the CHRONOS study (5.9 patients/100 PY). The incidence of serious adverse events related to treatment was 0.6 patients/100 PY in the final open label extension study data set, compared with 0.7 patients/100 PY in the CHRONOS final data set.

Adverse event rates “are really, if anything, slightly less in the OLE study versus the CHRONOS study, which was 1 year of treatment,” Dr. Beck said. And “no new adverse events have emerged.”

During a question and answer period, Dr. Beck pointed out that existing and future “real world” registries of patients on dupilumab and other new therapies will better inform dermatologists of adverse events than clinical trials have done.
 

Ocular surface disease

In a separate presentation on the safety of biologics, Andrew Blauvelt, MD, MBA, of the Oregon Medical Research Center, Portland, said that in routine care, ocular surface disease is the most predominant side effect associated with dupilumab. “We don’t know the mechanism of action. But it’s not infectious, it’s not pink eye, and importantly, it’s not allergic conjunctivitis,” he said, noting that the spectrum of disease ranges from dry eye and eye itching to “frank conjunctivitis” and keratitis.

Most cases are mild to moderate and can often be managed with lubricating eye drops and periodic use of corticosteroid eye drops. Co-management with an ophthalmologist is often advisable, he said.

Dupilumab-associated erythema/eczema of the face was “not seen much” in clinical trials but is also being reported in the literature, largely by European researchers, Dr. Blauvelt said. “We hear a lot about red face, but I don’t think it’s much of an issue,” he said. “Most of the time, in my experience, it will [reflect] breakthrough residual AD, and I like to treat it with non-steroidal topicals.”

Occasionally, the withdrawal of steroids or allergic contact dermatitis are at play, Dr. Blauvelt said. “If you see red face in a person on dupilumab, use your clinical prowess, do a differential diagnosis, and treat accordingly.”
 

Effect on S. aureus

The vast majority of adults with moderate to severe AD have skin colonization with S. aureus, Dr. Beck said during the session on long-term control of AD. The presence of S. aureus in skin cultures correlates strongly with AD severity, type 2 immunity polarization, skin barrier disruption, and allergen sensitization, she said.

“So if we could do something to get rid of the staph and keep it away, one might imagine that would help” control the AD disease process, she said.

An ADRN study evaluated S. aureus in the skin of 71 patients who were randomized to receive dupilumab or placebo and found a “profound” effect of the biologic. “We were truly shocked by how quickly we saw a reduction in Staph aureus ... in lesional skin as early as 3 days” into treatment with dupilumab, she said of the unpublished findings. “And there is a pretty nice association with improvement in disease severity.”

Dr. Beck reported consultancy/advisory board work with Regeneron, Sanofi/Genzyme, among other disclosures. Dr. Blauvelt reported consultancy/advisory board work for Regeneron and Sanofi Genzyme and has received speakers bureau/honoraria for non-CME work for Regeneron and Sanofi, among other disclosures.

WASHINGTON – The LIBERTY AD open-label extension study of dupilumab is closing after 5 years with the small number of remaining patients showing stable and sustained improvements in skin lesions and pruritus and no new emergent side effects, Lisa Beck, MD, reported during a late-breaking session at the annual Revolutionizing Atopic Dermatitis conference.

Other recent research on the biologic has shown that it improves lesional skin barrier function and rapidly reduces the abundance of Staphylococcus aureus on lesional skin, Dr. Beck, professor of dermatology at the University of Rochester (N.Y.), said during another session at the meeting on long-term control of AD. Dr. Beck directs a laboratory at the University of Rochester Medical Center that focuses on understanding AD and is involved in the National Institute of Allergy and Infectious Diseases (NIAID)-funded Atopic Dermatitis Research Network (ADRN).

The LIBERTY AD open-label extension (OLE) study was a phase 3 trial of 2,677 adults with moderate to severe AD who had participated in previous dupilumab clinical trials and were treated with 300 mg dupilumab weekly or every other week. Concomitant treatments were permitted, including topical corticosteroids and topical calcineurin inhibitors. (The proportion of patients dosed on an every-other-week or weekly dosing schedule was not available.)

Of 334 patients (12.5%) who remained in the trial at week 260, or 5 years, 88.9% achieved at least a 75% improvement in lesion extent and severity (Eczema Area and Severity Index [EASI]-75), and 76.2% achieved an EASI-90. The proportion achieving at least a 4-point reduction in the Peak Pruritus Numerical Rating Scale (NRS) or a score of 0 was 66.5%. At 5 years, improvements “seem very stable,” with “no loss in efficacy,” Dr. Beck said.

The majority of patients who withdrew from the open-label extension trial did so because the study was terminated at their site or because of the drug’s approval and commercialization – not for a medical reason, Dr. Beck said. Over the course of the extension trial, 4% of those enrolled withdrew because of adverse events and about 2% withdrew because of lack of efficacy.

Safety of dupilumab

The extension trial lacked a control arm, so Dr. Beck and her colleagues compared safety results to those in the final data set for patients in the LIBERTY AD CHRONOS study who received dupilumab 300 mg weekly with concomitant corticosteroids. The CHRONOS study was a 1-year randomized, double-blinded placebo-controlled phase 3 trial.

The exposure-adjusted incidence rate of severe treatment-emergent adverse events (TEAE) was lower at the close of the extension trial (5 patients/100 patient years [PY]) than at the end of the CHRONOS study (5.9 patients/100 PY). The incidence of serious adverse events related to treatment was 0.6 patients/100 PY in the final open label extension study data set, compared with 0.7 patients/100 PY in the CHRONOS final data set.

Adverse event rates “are really, if anything, slightly less in the OLE study versus the CHRONOS study, which was 1 year of treatment,” Dr. Beck said. And “no new adverse events have emerged.”

During a question and answer period, Dr. Beck pointed out that existing and future “real world” registries of patients on dupilumab and other new therapies will better inform dermatologists of adverse events than clinical trials have done.
 

Ocular surface disease

In a separate presentation on the safety of biologics, Andrew Blauvelt, MD, MBA, of the Oregon Medical Research Center, Portland, said that in routine care, ocular surface disease is the most predominant side effect associated with dupilumab. “We don’t know the mechanism of action. But it’s not infectious, it’s not pink eye, and importantly, it’s not allergic conjunctivitis,” he said, noting that the spectrum of disease ranges from dry eye and eye itching to “frank conjunctivitis” and keratitis.

Most cases are mild to moderate and can often be managed with lubricating eye drops and periodic use of corticosteroid eye drops. Co-management with an ophthalmologist is often advisable, he said.

Dupilumab-associated erythema/eczema of the face was “not seen much” in clinical trials but is also being reported in the literature, largely by European researchers, Dr. Blauvelt said. “We hear a lot about red face, but I don’t think it’s much of an issue,” he said. “Most of the time, in my experience, it will [reflect] breakthrough residual AD, and I like to treat it with non-steroidal topicals.”

Occasionally, the withdrawal of steroids or allergic contact dermatitis are at play, Dr. Blauvelt said. “If you see red face in a person on dupilumab, use your clinical prowess, do a differential diagnosis, and treat accordingly.”
 

Effect on S. aureus

The vast majority of adults with moderate to severe AD have skin colonization with S. aureus, Dr. Beck said during the session on long-term control of AD. The presence of S. aureus in skin cultures correlates strongly with AD severity, type 2 immunity polarization, skin barrier disruption, and allergen sensitization, she said.

“So if we could do something to get rid of the staph and keep it away, one might imagine that would help” control the AD disease process, she said.

An ADRN study evaluated S. aureus in the skin of 71 patients who were randomized to receive dupilumab or placebo and found a “profound” effect of the biologic. “We were truly shocked by how quickly we saw a reduction in Staph aureus ... in lesional skin as early as 3 days” into treatment with dupilumab, she said of the unpublished findings. “And there is a pretty nice association with improvement in disease severity.”

Dr. Beck reported consultancy/advisory board work with Regeneron, Sanofi/Genzyme, among other disclosures. Dr. Blauvelt reported consultancy/advisory board work for Regeneron and Sanofi Genzyme and has received speakers bureau/honoraria for non-CME work for Regeneron and Sanofi, among other disclosures.

While physicians are getting less of a Medicare pay cut than they thought this year (Congress voted to cut Medicare payments by 2%, which was less than the expected 8.5%), Medicare still pays physicians only 80% of what many third-party insurers pay.

Moreover, those reimbursements are often slow to arrive, and the paperwork is burdensome. In fact, about 65% of doctors won’t accept new Medicare patients, down from 71% just 5 years ago, according to the Medscape Physician Compensation Report 2023.

Worse, inflation makes continuous cuts feel even steeper and trickles down to physicians and their patients as more and more doctors become disenchanted and consider dropping Medicare.
 

Medicare at a glance

Medicare pays physicians about 80% of the “reasonable charge” for covered services. At the same time, private insurers pay nearly double Medicare rates for hospital services.

The Medicare fee schedule is released each year. Physicians who accept Medicare can choose to be a “participating provider” by agreeing to the fee schedule and to not charging more than this amount. “Nonparticipating” providers can charge up to 15% more. Physicians can also opt out of Medicare entirely.

The earliest that physicians receive their payment is 14 days after electronic filing to 28 days after paper filing, but it often can take months.

Physicians lose an estimated 7.3% of Medicare claims to billing problems. With private insurers, an estimated 4.8% is lost.

In 2000, there were 50 million Medicare enrollees; it is projected that by 2050, there will be 87 million enrollees.
 

Why are doctors disenchanted?

“When Medicare started, the concept of the program was good,” said Rahul Gupta, MD, a geriatrician in Westport, Conn., and chief of internal medicine at St. Vincent’s Medical Center, Bridgeport, Conn. “However, over the years, with new developments in medicine and the explosion of the Medicare-eligible population, the program hasn’t kept up with coverages.” In addition, Medicare’s behemoth power as a government-run agency has ramifications that trickle down irrespective of a patient’s insurance carrier.

“Medicare sets the tone on price and reimbursement, and everyone follows suit,” Dr. Gupta said. “It’s a race to the bottom.”

“The program is great for patients when people need hospitalizations, skilled nursing, and physical therapy,” Dr. Gupta said. “But it’s not great about keeping people healthier and maintaining function via preventive treatments.” Many private insurers must become more adept at that too.

For instance, Dr. Gupta laments the lack of coverage for hearing aids, something his patients could greatly benefit from. Thanks to the Build Back Better bill, coverage of hearing aids will begin in 2024. But, again, most private insurers don’t cover hearing aids either. Some Medicare Advantage plans do.

Medicare doesn’t cover eye health (except for eye exams for diabetes patients), which is an issue for Daniel Laroche, MD, a glaucoma specialist and clinical associate professor of ophthalmology at Mount Sinai Medical Center, New York.

“I get paid less for Medicare patients by about 20% because of ‘lesser-of’ payments,” said Dr. Laroche. For example, as per Medicare, after patients meet their Part B deductible, they pay 20% of the Medicare-approved amount for glaucoma testing. “It would be nice to get the full amount for Medicare patients.”

“In addition, getting approvals for testing takes time and exhaustive amounts of paperwork, says Adeeti Gupta, MD, a gynecologist and founder of Walk In GYN Care in New York.

“Medicare only covers gynecologist visits every 2 years after the age of 65,” she said. “Any additional testing requires authorization, and Medicare doesn’t cover hormone replacement at all, which really makes me crazy. They will cover Viagra for men, but they won’t cover HRT, which prolongs life, reduces dementia, and prevents bone loss.”

While these three doctors find Medicare lacking in its coverage of their specialty, and their reimbursements are too low, many physicians also find fault regarding Medicare billing, which can put their patients at risk.
 

The problem with Medicare billing

Because claims are processed by Medicare administrative contractors, it can take about a month for the approval or denial process and for doctors to receive reimbursement.

Prior authorizations, especially with Medicare Advantage plans, are also problematic. For example, one 2022 study found that 18% of payment denials were for services that met coverage and billing rules.

Worse, all of this jockeying for coverage takes time. The average health care provider spends 16.4 hours a week on paperwork and on securing prior authorizations to cover services, according to the American Medical Association.

“A good 40% of my time is exclusively Medicare red tape paperwork,” Rahul Gupta says. “There’s a reason I spend 2-3 hours a night catching up on that stuff.”

Not only does this lead to burnout, but it also means that most physicians must hire an administrator to help with the paperwork.

In comparison, industry averages put the denial rate for all Medicare and private insurance claims at 20%.

“Excessive authorization controls required by health insurers are persistently responsible for serious harm to physician practices and patients when necessary medical care is delayed, denied, or disrupted in an attempt to increase profits,” Dr. Laroche said.

“Our office spends nearly 2 days per week on prior authorizations, creating costly administrative burdens.”

For Adeeti Gupta, the frustrations with Medicare have continued to mount. “We’re just at a dead end,” she said. “Authorizations keep getting denied, and the back-end paperwork is only increasing for us.”
 

Will more doctors opt out of Medicare?

When doctors don’t accept Medicare, it hurts the patients using it, especially patients who have selected either a Medicare Advantage plan or who become eligible for Medicare at age 65 only to find that fewer doctors take the government-sponsored insurance than in the past.

As of 2020, only 1% of nonpediatric physicians had formally opted out, per the Centers for Medicare & Medicaid Services. Psychiatrists account for the largest share of opt-outs (7.2%).

“Unfortunately, most doctors outside of hospital-based practices will reach a point when they can’t deal with Medicare paperwork, so they’ll stop taking it,” Rahul Gupta says.

A coalition of 120 physicians’ groups, including the American Medical Association, disputes that Medicare is paying a fair reimbursement rate to physicians and calls for an overhaul in how they adjust physician pay.

“Nothing much changes no matter how much the AMA shouts,” Rahul Gupta said in an interview.
 

What can doctors do

Prescription prices are another example of the challenges posed by Medicare. When prescriptions are denied because of Medicare’s medigap (or donut hole) program, which puts a cap on medication coverage, which was $4,660 in 2023, Dr. Gupta says she turns to alternative ways to fill them.

“I’ve been telling patients to pay out of pocket and use GoodRx, or we get medications compounded,” she said. “That’s cheaper. For example, for HRT, GoodRx can bring down the cost 40% to 50%.”

The American Medical Association as well as 150 other medical advocacy groups continue to urge Congress to work with the physician community to address the systematic problems within Medicare, especially reimbursement.

Despite the daily challenges, Rahul Gupta says he remains committed to caring for his patients.

“I want to care for the elderly, especially because they already have very few physicians to take care of them, and fortunately, I have a good practice with other coverages,” he said. “I can’t give up.”

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

While physicians are getting less of a Medicare pay cut than they thought this year (Congress voted to cut Medicare payments by 2%, which was less than the expected 8.5%), Medicare still pays physicians only 80% of what many third-party insurers pay.

Moreover, those reimbursements are often slow to arrive, and the paperwork is burdensome. In fact, about 65% of doctors won’t accept new Medicare patients, down from 71% just 5 years ago, according to the Medscape Physician Compensation Report 2023.

Worse, inflation makes continuous cuts feel even steeper and trickles down to physicians and their patients as more and more doctors become disenchanted and consider dropping Medicare.
 

Medicare at a glance

Medicare pays physicians about 80% of the “reasonable charge” for covered services. At the same time, private insurers pay nearly double Medicare rates for hospital services.

The Medicare fee schedule is released each year. Physicians who accept Medicare can choose to be a “participating provider” by agreeing to the fee schedule and to not charging more than this amount. “Nonparticipating” providers can charge up to 15% more. Physicians can also opt out of Medicare entirely.

The earliest that physicians receive their payment is 14 days after electronic filing to 28 days after paper filing, but it often can take months.

Physicians lose an estimated 7.3% of Medicare claims to billing problems. With private insurers, an estimated 4.8% is lost.

In 2000, there were 50 million Medicare enrollees; it is projected that by 2050, there will be 87 million enrollees.
 

Why are doctors disenchanted?

“When Medicare started, the concept of the program was good,” said Rahul Gupta, MD, a geriatrician in Westport, Conn., and chief of internal medicine at St. Vincent’s Medical Center, Bridgeport, Conn. “However, over the years, with new developments in medicine and the explosion of the Medicare-eligible population, the program hasn’t kept up with coverages.” In addition, Medicare’s behemoth power as a government-run agency has ramifications that trickle down irrespective of a patient’s insurance carrier.

“Medicare sets the tone on price and reimbursement, and everyone follows suit,” Dr. Gupta said. “It’s a race to the bottom.”

“The program is great for patients when people need hospitalizations, skilled nursing, and physical therapy,” Dr. Gupta said. “But it’s not great about keeping people healthier and maintaining function via preventive treatments.” Many private insurers must become more adept at that too.

For instance, Dr. Gupta laments the lack of coverage for hearing aids, something his patients could greatly benefit from. Thanks to the Build Back Better bill, coverage of hearing aids will begin in 2024. But, again, most private insurers don’t cover hearing aids either. Some Medicare Advantage plans do.

Medicare doesn’t cover eye health (except for eye exams for diabetes patients), which is an issue for Daniel Laroche, MD, a glaucoma specialist and clinical associate professor of ophthalmology at Mount Sinai Medical Center, New York.

“I get paid less for Medicare patients by about 20% because of ‘lesser-of’ payments,” said Dr. Laroche. For example, as per Medicare, after patients meet their Part B deductible, they pay 20% of the Medicare-approved amount for glaucoma testing. “It would be nice to get the full amount for Medicare patients.”

“In addition, getting approvals for testing takes time and exhaustive amounts of paperwork, says Adeeti Gupta, MD, a gynecologist and founder of Walk In GYN Care in New York.

“Medicare only covers gynecologist visits every 2 years after the age of 65,” she said. “Any additional testing requires authorization, and Medicare doesn’t cover hormone replacement at all, which really makes me crazy. They will cover Viagra for men, but they won’t cover HRT, which prolongs life, reduces dementia, and prevents bone loss.”

While these three doctors find Medicare lacking in its coverage of their specialty, and their reimbursements are too low, many physicians also find fault regarding Medicare billing, which can put their patients at risk.
 

The problem with Medicare billing

Because claims are processed by Medicare administrative contractors, it can take about a month for the approval or denial process and for doctors to receive reimbursement.

Prior authorizations, especially with Medicare Advantage plans, are also problematic. For example, one 2022 study found that 18% of payment denials were for services that met coverage and billing rules.

Worse, all of this jockeying for coverage takes time. The average health care provider spends 16.4 hours a week on paperwork and on securing prior authorizations to cover services, according to the American Medical Association.

“A good 40% of my time is exclusively Medicare red tape paperwork,” Rahul Gupta says. “There’s a reason I spend 2-3 hours a night catching up on that stuff.”

Not only does this lead to burnout, but it also means that most physicians must hire an administrator to help with the paperwork.

In comparison, industry averages put the denial rate for all Medicare and private insurance claims at 20%.

“Excessive authorization controls required by health insurers are persistently responsible for serious harm to physician practices and patients when necessary medical care is delayed, denied, or disrupted in an attempt to increase profits,” Dr. Laroche said.

“Our office spends nearly 2 days per week on prior authorizations, creating costly administrative burdens.”

For Adeeti Gupta, the frustrations with Medicare have continued to mount. “We’re just at a dead end,” she said. “Authorizations keep getting denied, and the back-end paperwork is only increasing for us.”
 

Will more doctors opt out of Medicare?

When doctors don’t accept Medicare, it hurts the patients using it, especially patients who have selected either a Medicare Advantage plan or who become eligible for Medicare at age 65 only to find that fewer doctors take the government-sponsored insurance than in the past.

As of 2020, only 1% of nonpediatric physicians had formally opted out, per the Centers for Medicare & Medicaid Services. Psychiatrists account for the largest share of opt-outs (7.2%).

“Unfortunately, most doctors outside of hospital-based practices will reach a point when they can’t deal with Medicare paperwork, so they’ll stop taking it,” Rahul Gupta says.

A coalition of 120 physicians’ groups, including the American Medical Association, disputes that Medicare is paying a fair reimbursement rate to physicians and calls for an overhaul in how they adjust physician pay.

“Nothing much changes no matter how much the AMA shouts,” Rahul Gupta said in an interview.
 

What can doctors do

Prescription prices are another example of the challenges posed by Medicare. When prescriptions are denied because of Medicare’s medigap (or donut hole) program, which puts a cap on medication coverage, which was $4,660 in 2023, Dr. Gupta says she turns to alternative ways to fill them.

“I’ve been telling patients to pay out of pocket and use GoodRx, or we get medications compounded,” she said. “That’s cheaper. For example, for HRT, GoodRx can bring down the cost 40% to 50%.”

The American Medical Association as well as 150 other medical advocacy groups continue to urge Congress to work with the physician community to address the systematic problems within Medicare, especially reimbursement.

Despite the daily challenges, Rahul Gupta says he remains committed to caring for his patients.

“I want to care for the elderly, especially because they already have very few physicians to take care of them, and fortunately, I have a good practice with other coverages,” he said. “I can’t give up.”

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

While physicians are getting less of a Medicare pay cut than they thought this year (Congress voted to cut Medicare payments by 2%, which was less than the expected 8.5%), Medicare still pays physicians only 80% of what many third-party insurers pay.

Moreover, those reimbursements are often slow to arrive, and the paperwork is burdensome. In fact, about 65% of doctors won’t accept new Medicare patients, down from 71% just 5 years ago, according to the Medscape Physician Compensation Report 2023.

Worse, inflation makes continuous cuts feel even steeper and trickles down to physicians and their patients as more and more doctors become disenchanted and consider dropping Medicare.
 

Medicare at a glance

Medicare pays physicians about 80% of the “reasonable charge” for covered services. At the same time, private insurers pay nearly double Medicare rates for hospital services.

The Medicare fee schedule is released each year. Physicians who accept Medicare can choose to be a “participating provider” by agreeing to the fee schedule and to not charging more than this amount. “Nonparticipating” providers can charge up to 15% more. Physicians can also opt out of Medicare entirely.

The earliest that physicians receive their payment is 14 days after electronic filing to 28 days after paper filing, but it often can take months.

Physicians lose an estimated 7.3% of Medicare claims to billing problems. With private insurers, an estimated 4.8% is lost.

In 2000, there were 50 million Medicare enrollees; it is projected that by 2050, there will be 87 million enrollees.
 

Why are doctors disenchanted?

“When Medicare started, the concept of the program was good,” said Rahul Gupta, MD, a geriatrician in Westport, Conn., and chief of internal medicine at St. Vincent’s Medical Center, Bridgeport, Conn. “However, over the years, with new developments in medicine and the explosion of the Medicare-eligible population, the program hasn’t kept up with coverages.” In addition, Medicare’s behemoth power as a government-run agency has ramifications that trickle down irrespective of a patient’s insurance carrier.

“Medicare sets the tone on price and reimbursement, and everyone follows suit,” Dr. Gupta said. “It’s a race to the bottom.”

“The program is great for patients when people need hospitalizations, skilled nursing, and physical therapy,” Dr. Gupta said. “But it’s not great about keeping people healthier and maintaining function via preventive treatments.” Many private insurers must become more adept at that too.

For instance, Dr. Gupta laments the lack of coverage for hearing aids, something his patients could greatly benefit from. Thanks to the Build Back Better bill, coverage of hearing aids will begin in 2024. But, again, most private insurers don’t cover hearing aids either. Some Medicare Advantage plans do.

Medicare doesn’t cover eye health (except for eye exams for diabetes patients), which is an issue for Daniel Laroche, MD, a glaucoma specialist and clinical associate professor of ophthalmology at Mount Sinai Medical Center, New York.

“I get paid less for Medicare patients by about 20% because of ‘lesser-of’ payments,” said Dr. Laroche. For example, as per Medicare, after patients meet their Part B deductible, they pay 20% of the Medicare-approved amount for glaucoma testing. “It would be nice to get the full amount for Medicare patients.”

“In addition, getting approvals for testing takes time and exhaustive amounts of paperwork, says Adeeti Gupta, MD, a gynecologist and founder of Walk In GYN Care in New York.

“Medicare only covers gynecologist visits every 2 years after the age of 65,” she said. “Any additional testing requires authorization, and Medicare doesn’t cover hormone replacement at all, which really makes me crazy. They will cover Viagra for men, but they won’t cover HRT, which prolongs life, reduces dementia, and prevents bone loss.”

While these three doctors find Medicare lacking in its coverage of their specialty, and their reimbursements are too low, many physicians also find fault regarding Medicare billing, which can put their patients at risk.
 

The problem with Medicare billing

Because claims are processed by Medicare administrative contractors, it can take about a month for the approval or denial process and for doctors to receive reimbursement.

Prior authorizations, especially with Medicare Advantage plans, are also problematic. For example, one 2022 study found that 18% of payment denials were for services that met coverage and billing rules.

Worse, all of this jockeying for coverage takes time. The average health care provider spends 16.4 hours a week on paperwork and on securing prior authorizations to cover services, according to the American Medical Association.

“A good 40% of my time is exclusively Medicare red tape paperwork,” Rahul Gupta says. “There’s a reason I spend 2-3 hours a night catching up on that stuff.”

Not only does this lead to burnout, but it also means that most physicians must hire an administrator to help with the paperwork.

In comparison, industry averages put the denial rate for all Medicare and private insurance claims at 20%.

“Excessive authorization controls required by health insurers are persistently responsible for serious harm to physician practices and patients when necessary medical care is delayed, denied, or disrupted in an attempt to increase profits,” Dr. Laroche said.

“Our office spends nearly 2 days per week on prior authorizations, creating costly administrative burdens.”

For Adeeti Gupta, the frustrations with Medicare have continued to mount. “We’re just at a dead end,” she said. “Authorizations keep getting denied, and the back-end paperwork is only increasing for us.”
 

Will more doctors opt out of Medicare?

When doctors don’t accept Medicare, it hurts the patients using it, especially patients who have selected either a Medicare Advantage plan or who become eligible for Medicare at age 65 only to find that fewer doctors take the government-sponsored insurance than in the past.

As of 2020, only 1% of nonpediatric physicians had formally opted out, per the Centers for Medicare & Medicaid Services. Psychiatrists account for the largest share of opt-outs (7.2%).

“Unfortunately, most doctors outside of hospital-based practices will reach a point when they can’t deal with Medicare paperwork, so they’ll stop taking it,” Rahul Gupta says.

A coalition of 120 physicians’ groups, including the American Medical Association, disputes that Medicare is paying a fair reimbursement rate to physicians and calls for an overhaul in how they adjust physician pay.

“Nothing much changes no matter how much the AMA shouts,” Rahul Gupta said in an interview.
 

What can doctors do

Prescription prices are another example of the challenges posed by Medicare. When prescriptions are denied because of Medicare’s medigap (or donut hole) program, which puts a cap on medication coverage, which was $4,660 in 2023, Dr. Gupta says she turns to alternative ways to fill them.

“I’ve been telling patients to pay out of pocket and use GoodRx, or we get medications compounded,” she said. “That’s cheaper. For example, for HRT, GoodRx can bring down the cost 40% to 50%.”

The American Medical Association as well as 150 other medical advocacy groups continue to urge Congress to work with the physician community to address the systematic problems within Medicare, especially reimbursement.

Despite the daily challenges, Rahul Gupta says he remains committed to caring for his patients.

“I want to care for the elderly, especially because they already have very few physicians to take care of them, and fortunately, I have a good practice with other coverages,” he said. “I can’t give up.”

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

The costs of providing evidence-based interventions in primary care to address social needs far exceed current federal funding streams, say the authors of a new analysis.

A microsimulation analysis by Sanjay Basu, MD, PhD, with Clinical Product Development, Waymark Care, San Francisco, and colleagues found that, as primary care practices are being asked to screen for social needs, the cost of providing evidence-based interventions for these needs averaged $60 per member/person per month (PMPM) (95% confidence interval, $55-$65).

However, less than half ($27) of the $60 cost had existing federal financing in place to pay for it. Of the $60, $5 was for screening and referral.

The study results were published in JAMA Internal Medicine.

The researchers looked at key social needs areas and found major gaps between what interventions cost and what’s covered by federal payers. They demonstrate the gaps in four key areas. Many people in the analysis have more than one need:

• Food insecurity: Cost was $23 PMPM and the proportion borne by existing federal payers was 61.6%.
• Housing insecurity: Cost was $3 PMPM; proportion borne by federal payers was 45.6%.
• Transportation insecurity: Cost was $0.1 PMPM; proportion borne by federal payers was 27.8%.
• Community-based care coordination: Cost was $0.6 PMPM; proportion borne by federal payers is 6.4%.

Gaps varied by type of center

Primary care practices were grouped into federally qualified health centers; non-FQHC urban practices in high-poverty areas; non-FQHC rural practices in high-poverty areas; and practices in lower-poverty areas. Gaps varied among the groups.

While disproportionate funding was available to populations seen at FQHCs, populations seen at non-FQHC practices in high-poverty areas had larger funding gaps.

The study population consisted of 19,225 patients seen in primary care practices; data on social needs were pulled from the National Center for Health Statistics from 2015 to 2018.

Dr. Basu said in an interview with the journal’s deputy editor, Mitchell Katz, MD, that new sustainable revenue streams need to be identified to close the gap. Primary care physicians should not be charged with tasks such as researching the best housing programs and food benefits.

“I can’t imagine fitting this into my primary care appointments,” he said.
 

Is primary care the best setting for addressing these needs?

In an accompanying comment, Jenifer Clapp, MPA, with the Office of Ambulatory Care and Population Health, NYC Health + Hospitals, New York, and colleagues wrote that the study raises the question of whether the health care setting is the right place for addressing social needs. Some aspects have to be addressed in health care, such as asking about the home environment for a patient with environmentally triggered asthma.

“But how involved should health care professionals be in identifying needs unrelated to illness and solving those needs?” Ms. Clapp and colleagues asked.

They wrote that the health care sector in the United States must address these needs because in the United States, unlike in many European countries, “there is an insufficient social service sector to address the basic human needs of children and working-age adults.”
 

Eligible but not enrolled

Importantly, both the study authors and editorialists pointed out, in many cases, intervening doesn’t mean paying for the social services, but helping patients enroll in the services for which they already qualify.

The study authors wrote that among people who had food and housing needs, most met the criteria for federally funded programs, but had low enrollment for reasons including inadequate program capacity.

For example, 78% of people with housing needs were eligible for federal programs but only 24% were enrolled, and 95.6% of people with food needs were eligible for programs but only 70.2% were enrolled in programs like the Supplemental Nutrition and Assistance Program and Women, Infants and Children.

Commentary coauthor Nichola Davis, MD, also with NYC Health + Hospitals, said one thing they’ve done at NYC Health + Hospitals is partner with community-based organizations that provide food navigators so when patients screen positive for food insecurity they can then be seen by a food navigator to pinpoint appropriate programs.

The referral for those who indicate food insecurity is automatically generated by the electronic health system and appears on the after-visit summary.

“At the bare minimum, the patient would leave with a list of resources,” Dr. Davis said.
 

One place primary care providers can make a difference

Dr. Katz said that the $60 cost per person is much lower than that for a service such as an MRI.

“We should be able to achieve that,” he said.

Will Bleser, PhD, MSPH, assistant research director of health care transformation for social needs and health equity at the Duke-Margolis Center for Health Policy, Washington, said it’s exciting to see the per-person cost for social needs quantified.

He pointed to existing revenue options that have been underutilized.

Through Medicare, he noted, if you are part of a Medicare Advantage plan, there is a program implemented in 2020 called Special Supplemental Benefits for the Chronically Ill. “That authorizes Medicare Advantage plans to offer non–primarily health-related services through Medicare Advantage to individuals who meet certain chronic illness conditions.”

Non–primarily health-related services may include meals, transportation, and pest control, for example, the Centers for Medicare & Medicaid Services notes.

Also, within the shared-savings program of traditional Medicare, if an accountable care organization is providing quality care under the cost target and is reaping the savings, “you could use those bonuses to do things that you couldn’t do under the normal Medicare fee schedule like address social needs,” Dr. Bleser said.

Medicaid, he said, offers the most opportunities to address social needs through the health system. One policy mechanism within Medicaid is the Section 1115 Waiver, where states can propose to provide new services as long as they comply with the core rules of Medicaid and meet certain qualifications.
 

Avoiding checking boxes with no benefit to patients

Ms. Clapp and colleagues noted that whether health care professionals agree that social needs can or should be addressed in primary care, CMS will mandate social needs screening and reporting for all hospitalized adults starting in 2024. Additionally, the Joint Commission will require health care systems to gauge social needs and report on resources.

“We need to ensure that these mandates do not become administrative checkboxes that frustrate clinical staff and ratchet up health care costs with no benefit to patients,” they wrote.

Dr. Basu reported receiving personal fees from the University of California, Healthright360, Waymark and Collective Health outside the submitted work; he has a patent issued for a multimodel member outreach system; and a patent pending for operationalizing predicted changes in risk based on interventions. A coauthor reported grants from the North Carolina Department of Health and Human Services, Blue Cross Blue Shield of North Carolina, and personal fees from several nonprofit organizations outside the submitted work. Another coauthor reported personal fees from ZealCare outside the submitted work.

The costs of providing evidence-based interventions in primary care to address social needs far exceed current federal funding streams, say the authors of a new analysis.

A microsimulation analysis by Sanjay Basu, MD, PhD, with Clinical Product Development, Waymark Care, San Francisco, and colleagues found that, as primary care practices are being asked to screen for social needs, the cost of providing evidence-based interventions for these needs averaged $60 per member/person per month (PMPM) (95% confidence interval, $55-$65).

However, less than half ($27) of the $60 cost had existing federal financing in place to pay for it. Of the $60, $5 was for screening and referral.

The study results were published in JAMA Internal Medicine.

The researchers looked at key social needs areas and found major gaps between what interventions cost and what’s covered by federal payers. They demonstrate the gaps in four key areas. Many people in the analysis have more than one need:

• Food insecurity: Cost was $23 PMPM and the proportion borne by existing federal payers was 61.6%.
• Housing insecurity: Cost was $3 PMPM; proportion borne by federal payers was 45.6%.
• Transportation insecurity: Cost was $0.1 PMPM; proportion borne by federal payers was 27.8%.
• Community-based care coordination: Cost was $0.6 PMPM; proportion borne by federal payers is 6.4%.

Gaps varied by type of center

Primary care practices were grouped into federally qualified health centers; non-FQHC urban practices in high-poverty areas; non-FQHC rural practices in high-poverty areas; and practices in lower-poverty areas. Gaps varied among the groups.

While disproportionate funding was available to populations seen at FQHCs, populations seen at non-FQHC practices in high-poverty areas had larger funding gaps.

The study population consisted of 19,225 patients seen in primary care practices; data on social needs were pulled from the National Center for Health Statistics from 2015 to 2018.

Dr. Basu said in an interview with the journal’s deputy editor, Mitchell Katz, MD, that new sustainable revenue streams need to be identified to close the gap. Primary care physicians should not be charged with tasks such as researching the best housing programs and food benefits.

“I can’t imagine fitting this into my primary care appointments,” he said.
 

Is primary care the best setting for addressing these needs?

In an accompanying comment, Jenifer Clapp, MPA, with the Office of Ambulatory Care and Population Health, NYC Health + Hospitals, New York, and colleagues wrote that the study raises the question of whether the health care setting is the right place for addressing social needs. Some aspects have to be addressed in health care, such as asking about the home environment for a patient with environmentally triggered asthma.

“But how involved should health care professionals be in identifying needs unrelated to illness and solving those needs?” Ms. Clapp and colleagues asked.

They wrote that the health care sector in the United States must address these needs because in the United States, unlike in many European countries, “there is an insufficient social service sector to address the basic human needs of children and working-age adults.”
 

Eligible but not enrolled

Importantly, both the study authors and editorialists pointed out, in many cases, intervening doesn’t mean paying for the social services, but helping patients enroll in the services for which they already qualify.

The study authors wrote that among people who had food and housing needs, most met the criteria for federally funded programs, but had low enrollment for reasons including inadequate program capacity.

For example, 78% of people with housing needs were eligible for federal programs but only 24% were enrolled, and 95.6% of people with food needs were eligible for programs but only 70.2% were enrolled in programs like the Supplemental Nutrition and Assistance Program and Women, Infants and Children.

Commentary coauthor Nichola Davis, MD, also with NYC Health + Hospitals, said one thing they’ve done at NYC Health + Hospitals is partner with community-based organizations that provide food navigators so when patients screen positive for food insecurity they can then be seen by a food navigator to pinpoint appropriate programs.

The referral for those who indicate food insecurity is automatically generated by the electronic health system and appears on the after-visit summary.

“At the bare minimum, the patient would leave with a list of resources,” Dr. Davis said.
 

One place primary care providers can make a difference

Dr. Katz said that the $60 cost per person is much lower than that for a service such as an MRI.

“We should be able to achieve that,” he said.

Will Bleser, PhD, MSPH, assistant research director of health care transformation for social needs and health equity at the Duke-Margolis Center for Health Policy, Washington, said it’s exciting to see the per-person cost for social needs quantified.

He pointed to existing revenue options that have been underutilized.

Through Medicare, he noted, if you are part of a Medicare Advantage plan, there is a program implemented in 2020 called Special Supplemental Benefits for the Chronically Ill. “That authorizes Medicare Advantage plans to offer non–primarily health-related services through Medicare Advantage to individuals who meet certain chronic illness conditions.”

Non–primarily health-related services may include meals, transportation, and pest control, for example, the Centers for Medicare & Medicaid Services notes.

Also, within the shared-savings program of traditional Medicare, if an accountable care organization is providing quality care under the cost target and is reaping the savings, “you could use those bonuses to do things that you couldn’t do under the normal Medicare fee schedule like address social needs,” Dr. Bleser said.

Medicaid, he said, offers the most opportunities to address social needs through the health system. One policy mechanism within Medicaid is the Section 1115 Waiver, where states can propose to provide new services as long as they comply with the core rules of Medicaid and meet certain qualifications.
 

Avoiding checking boxes with no benefit to patients

Ms. Clapp and colleagues noted that whether health care professionals agree that social needs can or should be addressed in primary care, CMS will mandate social needs screening and reporting for all hospitalized adults starting in 2024. Additionally, the Joint Commission will require health care systems to gauge social needs and report on resources.

“We need to ensure that these mandates do not become administrative checkboxes that frustrate clinical staff and ratchet up health care costs with no benefit to patients,” they wrote.

Dr. Basu reported receiving personal fees from the University of California, Healthright360, Waymark and Collective Health outside the submitted work; he has a patent issued for a multimodel member outreach system; and a patent pending for operationalizing predicted changes in risk based on interventions. A coauthor reported grants from the North Carolina Department of Health and Human Services, Blue Cross Blue Shield of North Carolina, and personal fees from several nonprofit organizations outside the submitted work. Another coauthor reported personal fees from ZealCare outside the submitted work.

The costs of providing evidence-based interventions in primary care to address social needs far exceed current federal funding streams, say the authors of a new analysis.

A microsimulation analysis by Sanjay Basu, MD, PhD, with Clinical Product Development, Waymark Care, San Francisco, and colleagues found that, as primary care practices are being asked to screen for social needs, the cost of providing evidence-based interventions for these needs averaged $60 per member/person per month (PMPM) (95% confidence interval, $55-$65).

However, less than half ($27) of the $60 cost had existing federal financing in place to pay for it. Of the $60, $5 was for screening and referral.

The study results were published in JAMA Internal Medicine.

The researchers looked at key social needs areas and found major gaps between what interventions cost and what’s covered by federal payers. They demonstrate the gaps in four key areas. Many people in the analysis have more than one need:

• Food insecurity: Cost was $23 PMPM and the proportion borne by existing federal payers was 61.6%.
• Housing insecurity: Cost was $3 PMPM; proportion borne by federal payers was 45.6%.
• Transportation insecurity: Cost was $0.1 PMPM; proportion borne by federal payers was 27.8%.
• Community-based care coordination: Cost was $0.6 PMPM; proportion borne by federal payers is 6.4%.

Gaps varied by type of center

Primary care practices were grouped into federally qualified health centers; non-FQHC urban practices in high-poverty areas; non-FQHC rural practices in high-poverty areas; and practices in lower-poverty areas. Gaps varied among the groups.

While disproportionate funding was available to populations seen at FQHCs, populations seen at non-FQHC practices in high-poverty areas had larger funding gaps.

The study population consisted of 19,225 patients seen in primary care practices; data on social needs were pulled from the National Center for Health Statistics from 2015 to 2018.

Dr. Basu said in an interview with the journal’s deputy editor, Mitchell Katz, MD, that new sustainable revenue streams need to be identified to close the gap. Primary care physicians should not be charged with tasks such as researching the best housing programs and food benefits.

“I can’t imagine fitting this into my primary care appointments,” he said.
 

Is primary care the best setting for addressing these needs?

In an accompanying comment, Jenifer Clapp, MPA, with the Office of Ambulatory Care and Population Health, NYC Health + Hospitals, New York, and colleagues wrote that the study raises the question of whether the health care setting is the right place for addressing social needs. Some aspects have to be addressed in health care, such as asking about the home environment for a patient with environmentally triggered asthma.

“But how involved should health care professionals be in identifying needs unrelated to illness and solving those needs?” Ms. Clapp and colleagues asked.

They wrote that the health care sector in the United States must address these needs because in the United States, unlike in many European countries, “there is an insufficient social service sector to address the basic human needs of children and working-age adults.”
 

Eligible but not enrolled

Importantly, both the study authors and editorialists pointed out, in many cases, intervening doesn’t mean paying for the social services, but helping patients enroll in the services for which they already qualify.

The study authors wrote that among people who had food and housing needs, most met the criteria for federally funded programs, but had low enrollment for reasons including inadequate program capacity.

For example, 78% of people with housing needs were eligible for federal programs but only 24% were enrolled, and 95.6% of people with food needs were eligible for programs but only 70.2% were enrolled in programs like the Supplemental Nutrition and Assistance Program and Women, Infants and Children.

Commentary coauthor Nichola Davis, MD, also with NYC Health + Hospitals, said one thing they’ve done at NYC Health + Hospitals is partner with community-based organizations that provide food navigators so when patients screen positive for food insecurity they can then be seen by a food navigator to pinpoint appropriate programs.

The referral for those who indicate food insecurity is automatically generated by the electronic health system and appears on the after-visit summary.

“At the bare minimum, the patient would leave with a list of resources,” Dr. Davis said.
 

One place primary care providers can make a difference

Dr. Katz said that the $60 cost per person is much lower than that for a service such as an MRI.

“We should be able to achieve that,” he said.

Will Bleser, PhD, MSPH, assistant research director of health care transformation for social needs and health equity at the Duke-Margolis Center for Health Policy, Washington, said it’s exciting to see the per-person cost for social needs quantified.

He pointed to existing revenue options that have been underutilized.

Through Medicare, he noted, if you are part of a Medicare Advantage plan, there is a program implemented in 2020 called Special Supplemental Benefits for the Chronically Ill. “That authorizes Medicare Advantage plans to offer non–primarily health-related services through Medicare Advantage to individuals who meet certain chronic illness conditions.”

Non–primarily health-related services may include meals, transportation, and pest control, for example, the Centers for Medicare & Medicaid Services notes.

Also, within the shared-savings program of traditional Medicare, if an accountable care organization is providing quality care under the cost target and is reaping the savings, “you could use those bonuses to do things that you couldn’t do under the normal Medicare fee schedule like address social needs,” Dr. Bleser said.

Medicaid, he said, offers the most opportunities to address social needs through the health system. One policy mechanism within Medicaid is the Section 1115 Waiver, where states can propose to provide new services as long as they comply with the core rules of Medicaid and meet certain qualifications.
 

Avoiding checking boxes with no benefit to patients

Ms. Clapp and colleagues noted that whether health care professionals agree that social needs can or should be addressed in primary care, CMS will mandate social needs screening and reporting for all hospitalized adults starting in 2024. Additionally, the Joint Commission will require health care systems to gauge social needs and report on resources.

“We need to ensure that these mandates do not become administrative checkboxes that frustrate clinical staff and ratchet up health care costs with no benefit to patients,” they wrote.

Dr. Basu reported receiving personal fees from the University of California, Healthright360, Waymark and Collective Health outside the submitted work; he has a patent issued for a multimodel member outreach system; and a patent pending for operationalizing predicted changes in risk based on interventions. A coauthor reported grants from the North Carolina Department of Health and Human Services, Blue Cross Blue Shield of North Carolina, and personal fees from several nonprofit organizations outside the submitted work. Another coauthor reported personal fees from ZealCare outside the submitted work.

More than 600,000 Americans have lost Medicaid coverage since pandemic protections ended on April 1. And a KFF Health News analysis of state data shows the vast majority were removed from state rolls for not completing paperwork.

Under normal circumstances, states review their Medicaid enrollment lists regularly to ensure every recipient qualifies for coverage. But because of a nationwide pause in those reviews during the pandemic, the health insurance program for low-income and disabled Americans kept people covered even if they no longer qualified.

Now, in what’s known as the Medicaid unwinding, states are combing through rolls and deciding who stays and who goes. People who are no longer eligible or don’t complete paperwork in time will be dropped.

The overwhelming majority of people who have lost coverage in most states were dropped because of technicalities, not because state officials determined they no longer meet Medicaid income limits. Four out of every five people dropped so far either never returned the paperwork or omitted required documents, according to a KFF Health News analysis of data from 11 states that provided details on recent cancellations. Now, lawmakers and advocates are expressing alarm over the volume of people losing coverage and, in some states, calling to pause the process.

KFF Health News sought data from the 19 states that started cancellations by May 1. Based on records from 14 states that provided detailed numbers, either in response to a public records request or by posting online, 36% of people whose eligibility was reviewed have been disenrolled.

In Indiana, 53,000 residents lost coverage in the first month of the unwinding, 89% for procedural reasons like not returning renewal forms. State Rep. Ed Clere, a Republican, expressed dismay at those “staggering numbers” in a May 24 Medicaid advisory group meeting, repeatedly questioning state officials about forms mailed to out-of-date addresses and urging them to give people more than 2 weeks’ notice before canceling their coverage.

Rep. Clere warned that the cancellations set in motion an avoidable revolving door. Some people dropped from Medicaid will have to forgo filling prescriptions and cancel doctor visits because they can’t afford care. Months down the line, after untreated chronic illnesses spiral out of control, they’ll end up in the emergency room where social workers will need to again help them join the program, he said.

Before the unwinding, more than one in four Americans – 93 million – were covered by Medicaid or CHIP, the Children’s Health Insurance Program, according to KFF Health News’ analysis of the latest enrollment data. Half of all kids are covered by the programs.

About 15 million people will be dropped over the next year as states review participants’ eligibility in monthly tranches.

Most people will find health coverage through new jobs or qualify for subsidized plans through the Affordable Care Act. But millions of others, including many children, will become uninsured and unable to afford basic prescriptions or preventive care. The uninsured rate among those under 65 is projected to rise from a historical low of 8.3% today to 9.3% next year, according to the Congressional Budget Office.

Because each state is handling the unwinding differently, the share of enrollees dropped in the first weeks varies widely.

Several states are first reviewing people officials believe are no longer eligible or who haven’t recently used their insurance. High cancellation rates in those states should level out as the agencies move on to people who likely still qualify.

In Utah, nearly 56% of people included in early reviews were dropped. In New Hampshire, 44% received cancellation letters within the first 2 months – almost all for procedural reasons, like not returning paperwork.

But New Hampshire officials found that thousands of people who didn’t fill out the forms indeed earn too much to qualify, according to Henry Lipman, the state’s Medicaid director. They would have been denied anyway. Even so, more people than he expected are not returning renewal forms. “That tells us that we need to change up our strategy,” said Mr. Lipman.

In other states, like Virginia and Nebraska, which aren’t prioritizing renewals by likely eligibility, about 90% have been renewed.

Because of the 3-year pause in renewals, many people on Medicaid have never been through the process or aren’t aware they may need to fill out long verification forms, as a recent KFF poll found. Some people moved and didn’t update their contact information.

And while agencies are required to assist enrollees who don’t speak English well, many are sending the forms in only a few common languages.

Tens of thousands of children are losing coverage, as researchers have warned, even though some may still qualify for Medicaid or CHIP. In its first month of reviews, South Dakota ended coverage for 10% of all Medicaid and CHIP enrollees in the state. More than half of them were children. In Arkansas, about 40% were kids.

Many parents don’t know that limits on household income are significantly higher for children than adults. Parents should fill out renewal forms even if they don’t qualify themselves, said Joan Alker, executive director of the Georgetown University Center for Children and Families, Washington.

New Hampshire has moved most families with children to the end of the review process. Mr. Lipman said his biggest worry is that a child will end up uninsured. Florida also planned to push kids with serious health conditions and other vulnerable groups to the end of the review line.

But according to Miriam Harmatz, advocacy director and founder of the Florida Health Justice Project, state officials sent cancellation letters to several clients with disabled children who probably still qualify. She’s helping those families appeal.

Nearly 250,000 Floridians reviewed in the first month of the unwinding lost coverage, 82% of them for reasons like incomplete paperwork, the state reported to federal authorities. House Democrats from the state petitioned Republican Gov. Ron DeSantis to pause the unwinding.

Advocacy coalitions in both Florida and Arkansas also have called for investigations into the review process and a pause on cancellations.

The state is contacting enrollees by phone, email, and text, and continues to process late applications, said Tori Cuddy, a spokesperson for the Florida Department of Children and Families. Ms. Cuddy did not respond to questions about issues raised in the petitions.

Federal officials are investigating those complaints and any other problems that emerge, said Dan Tsai, director of the Center for Medicaid & CHIP Services. “If we find that the rules are not being followed, we will take action.”

His agency has directed states to automatically reenroll residents using data from other government programs like unemployment and food assistance when possible. Anyone who can’t be approved through that process must act quickly.

“For the past 3 years, people have been told to ignore the mail around this, that the renewal was not going to lead to a termination.” Suddenly that mail matters, he said.

Federal law requires states to tell people why they’re losing Medicaid coverage and how to appeal the decision.

Ms. Harmatz said some cancellation notices in Florida are vague and could violate due process rules. Letters that she’s seen say “your Medicaid for this period is ending” rather than providing a specific reason for disenrollment, like having too high an income or incomplete paperwork.
If a person requests a hearing before their cancellation takes effect, they can stay covered during the appeals process. Even after being disenrolled, many still have a 90-day window to restore coverage.

In New Hampshire, 13% of people deemed ineligible in the first month have asked for extra time to provide the necessary records. “If you’re eligible for Medicaid, we don’t want you to lose it,” said Mr. Lipman.

Rep. Clere pushed Indiana’s Medicaid officials during the May meeting to immediately make changes to avoid people unnecessarily becoming uninsured. One official responded that they’ll learn and improve over time.

“I’m just concerned that we’re going to be ‘learning’ as a result of people losing coverage,” Rep. Clere replied. “So I don’t want to learn at their expense.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

More than 600,000 Americans have lost Medicaid coverage since pandemic protections ended on April 1. And a KFF Health News analysis of state data shows the vast majority were removed from state rolls for not completing paperwork.

Under normal circumstances, states review their Medicaid enrollment lists regularly to ensure every recipient qualifies for coverage. But because of a nationwide pause in those reviews during the pandemic, the health insurance program for low-income and disabled Americans kept people covered even if they no longer qualified.

Now, in what’s known as the Medicaid unwinding, states are combing through rolls and deciding who stays and who goes. People who are no longer eligible or don’t complete paperwork in time will be dropped.

The overwhelming majority of people who have lost coverage in most states were dropped because of technicalities, not because state officials determined they no longer meet Medicaid income limits. Four out of every five people dropped so far either never returned the paperwork or omitted required documents, according to a KFF Health News analysis of data from 11 states that provided details on recent cancellations. Now, lawmakers and advocates are expressing alarm over the volume of people losing coverage and, in some states, calling to pause the process.

KFF Health News sought data from the 19 states that started cancellations by May 1. Based on records from 14 states that provided detailed numbers, either in response to a public records request or by posting online, 36% of people whose eligibility was reviewed have been disenrolled.

In Indiana, 53,000 residents lost coverage in the first month of the unwinding, 89% for procedural reasons like not returning renewal forms. State Rep. Ed Clere, a Republican, expressed dismay at those “staggering numbers” in a May 24 Medicaid advisory group meeting, repeatedly questioning state officials about forms mailed to out-of-date addresses and urging them to give people more than 2 weeks’ notice before canceling their coverage.

Rep. Clere warned that the cancellations set in motion an avoidable revolving door. Some people dropped from Medicaid will have to forgo filling prescriptions and cancel doctor visits because they can’t afford care. Months down the line, after untreated chronic illnesses spiral out of control, they’ll end up in the emergency room where social workers will need to again help them join the program, he said.

Before the unwinding, more than one in four Americans – 93 million – were covered by Medicaid or CHIP, the Children’s Health Insurance Program, according to KFF Health News’ analysis of the latest enrollment data. Half of all kids are covered by the programs.

About 15 million people will be dropped over the next year as states review participants’ eligibility in monthly tranches.

Most people will find health coverage through new jobs or qualify for subsidized plans through the Affordable Care Act. But millions of others, including many children, will become uninsured and unable to afford basic prescriptions or preventive care. The uninsured rate among those under 65 is projected to rise from a historical low of 8.3% today to 9.3% next year, according to the Congressional Budget Office.

Because each state is handling the unwinding differently, the share of enrollees dropped in the first weeks varies widely.

Several states are first reviewing people officials believe are no longer eligible or who haven’t recently used their insurance. High cancellation rates in those states should level out as the agencies move on to people who likely still qualify.

In Utah, nearly 56% of people included in early reviews were dropped. In New Hampshire, 44% received cancellation letters within the first 2 months – almost all for procedural reasons, like not returning paperwork.

But New Hampshire officials found that thousands of people who didn’t fill out the forms indeed earn too much to qualify, according to Henry Lipman, the state’s Medicaid director. They would have been denied anyway. Even so, more people than he expected are not returning renewal forms. “That tells us that we need to change up our strategy,” said Mr. Lipman.

In other states, like Virginia and Nebraska, which aren’t prioritizing renewals by likely eligibility, about 90% have been renewed.

Because of the 3-year pause in renewals, many people on Medicaid have never been through the process or aren’t aware they may need to fill out long verification forms, as a recent KFF poll found. Some people moved and didn’t update their contact information.

And while agencies are required to assist enrollees who don’t speak English well, many are sending the forms in only a few common languages.

Tens of thousands of children are losing coverage, as researchers have warned, even though some may still qualify for Medicaid or CHIP. In its first month of reviews, South Dakota ended coverage for 10% of all Medicaid and CHIP enrollees in the state. More than half of them were children. In Arkansas, about 40% were kids.

Many parents don’t know that limits on household income are significantly higher for children than adults. Parents should fill out renewal forms even if they don’t qualify themselves, said Joan Alker, executive director of the Georgetown University Center for Children and Families, Washington.

New Hampshire has moved most families with children to the end of the review process. Mr. Lipman said his biggest worry is that a child will end up uninsured. Florida also planned to push kids with serious health conditions and other vulnerable groups to the end of the review line.

But according to Miriam Harmatz, advocacy director and founder of the Florida Health Justice Project, state officials sent cancellation letters to several clients with disabled children who probably still qualify. She’s helping those families appeal.

Nearly 250,000 Floridians reviewed in the first month of the unwinding lost coverage, 82% of them for reasons like incomplete paperwork, the state reported to federal authorities. House Democrats from the state petitioned Republican Gov. Ron DeSantis to pause the unwinding.

Advocacy coalitions in both Florida and Arkansas also have called for investigations into the review process and a pause on cancellations.

The state is contacting enrollees by phone, email, and text, and continues to process late applications, said Tori Cuddy, a spokesperson for the Florida Department of Children and Families. Ms. Cuddy did not respond to questions about issues raised in the petitions.

Federal officials are investigating those complaints and any other problems that emerge, said Dan Tsai, director of the Center for Medicaid & CHIP Services. “If we find that the rules are not being followed, we will take action.”

His agency has directed states to automatically reenroll residents using data from other government programs like unemployment and food assistance when possible. Anyone who can’t be approved through that process must act quickly.

“For the past 3 years, people have been told to ignore the mail around this, that the renewal was not going to lead to a termination.” Suddenly that mail matters, he said.

Federal law requires states to tell people why they’re losing Medicaid coverage and how to appeal the decision.

Ms. Harmatz said some cancellation notices in Florida are vague and could violate due process rules. Letters that she’s seen say “your Medicaid for this period is ending” rather than providing a specific reason for disenrollment, like having too high an income or incomplete paperwork.
If a person requests a hearing before their cancellation takes effect, they can stay covered during the appeals process. Even after being disenrolled, many still have a 90-day window to restore coverage.

In New Hampshire, 13% of people deemed ineligible in the first month have asked for extra time to provide the necessary records. “If you’re eligible for Medicaid, we don’t want you to lose it,” said Mr. Lipman.

Rep. Clere pushed Indiana’s Medicaid officials during the May meeting to immediately make changes to avoid people unnecessarily becoming uninsured. One official responded that they’ll learn and improve over time.

“I’m just concerned that we’re going to be ‘learning’ as a result of people losing coverage,” Rep. Clere replied. “So I don’t want to learn at their expense.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

More than 600,000 Americans have lost Medicaid coverage since pandemic protections ended on April 1. And a KFF Health News analysis of state data shows the vast majority were removed from state rolls for not completing paperwork.

Under normal circumstances, states review their Medicaid enrollment lists regularly to ensure every recipient qualifies for coverage. But because of a nationwide pause in those reviews during the pandemic, the health insurance program for low-income and disabled Americans kept people covered even if they no longer qualified.

Now, in what’s known as the Medicaid unwinding, states are combing through rolls and deciding who stays and who goes. People who are no longer eligible or don’t complete paperwork in time will be dropped.

The overwhelming majority of people who have lost coverage in most states were dropped because of technicalities, not because state officials determined they no longer meet Medicaid income limits. Four out of every five people dropped so far either never returned the paperwork or omitted required documents, according to a KFF Health News analysis of data from 11 states that provided details on recent cancellations. Now, lawmakers and advocates are expressing alarm over the volume of people losing coverage and, in some states, calling to pause the process.

KFF Health News sought data from the 19 states that started cancellations by May 1. Based on records from 14 states that provided detailed numbers, either in response to a public records request or by posting online, 36% of people whose eligibility was reviewed have been disenrolled.

In Indiana, 53,000 residents lost coverage in the first month of the unwinding, 89% for procedural reasons like not returning renewal forms. State Rep. Ed Clere, a Republican, expressed dismay at those “staggering numbers” in a May 24 Medicaid advisory group meeting, repeatedly questioning state officials about forms mailed to out-of-date addresses and urging them to give people more than 2 weeks’ notice before canceling their coverage.

Rep. Clere warned that the cancellations set in motion an avoidable revolving door. Some people dropped from Medicaid will have to forgo filling prescriptions and cancel doctor visits because they can’t afford care. Months down the line, after untreated chronic illnesses spiral out of control, they’ll end up in the emergency room where social workers will need to again help them join the program, he said.

Before the unwinding, more than one in four Americans – 93 million – were covered by Medicaid or CHIP, the Children’s Health Insurance Program, according to KFF Health News’ analysis of the latest enrollment data. Half of all kids are covered by the programs.

About 15 million people will be dropped over the next year as states review participants’ eligibility in monthly tranches.

Most people will find health coverage through new jobs or qualify for subsidized plans through the Affordable Care Act. But millions of others, including many children, will become uninsured and unable to afford basic prescriptions or preventive care. The uninsured rate among those under 65 is projected to rise from a historical low of 8.3% today to 9.3% next year, according to the Congressional Budget Office.

Because each state is handling the unwinding differently, the share of enrollees dropped in the first weeks varies widely.

Several states are first reviewing people officials believe are no longer eligible or who haven’t recently used their insurance. High cancellation rates in those states should level out as the agencies move on to people who likely still qualify.

In Utah, nearly 56% of people included in early reviews were dropped. In New Hampshire, 44% received cancellation letters within the first 2 months – almost all for procedural reasons, like not returning paperwork.

But New Hampshire officials found that thousands of people who didn’t fill out the forms indeed earn too much to qualify, according to Henry Lipman, the state’s Medicaid director. They would have been denied anyway. Even so, more people than he expected are not returning renewal forms. “That tells us that we need to change up our strategy,” said Mr. Lipman.

In other states, like Virginia and Nebraska, which aren’t prioritizing renewals by likely eligibility, about 90% have been renewed.

Because of the 3-year pause in renewals, many people on Medicaid have never been through the process or aren’t aware they may need to fill out long verification forms, as a recent KFF poll found. Some people moved and didn’t update their contact information.

And while agencies are required to assist enrollees who don’t speak English well, many are sending the forms in only a few common languages.

Tens of thousands of children are losing coverage, as researchers have warned, even though some may still qualify for Medicaid or CHIP. In its first month of reviews, South Dakota ended coverage for 10% of all Medicaid and CHIP enrollees in the state. More than half of them were children. In Arkansas, about 40% were kids.

Many parents don’t know that limits on household income are significantly higher for children than adults. Parents should fill out renewal forms even if they don’t qualify themselves, said Joan Alker, executive director of the Georgetown University Center for Children and Families, Washington.

New Hampshire has moved most families with children to the end of the review process. Mr. Lipman said his biggest worry is that a child will end up uninsured. Florida also planned to push kids with serious health conditions and other vulnerable groups to the end of the review line.

But according to Miriam Harmatz, advocacy director and founder of the Florida Health Justice Project, state officials sent cancellation letters to several clients with disabled children who probably still qualify. She’s helping those families appeal.

Nearly 250,000 Floridians reviewed in the first month of the unwinding lost coverage, 82% of them for reasons like incomplete paperwork, the state reported to federal authorities. House Democrats from the state petitioned Republican Gov. Ron DeSantis to pause the unwinding.

Advocacy coalitions in both Florida and Arkansas also have called for investigations into the review process and a pause on cancellations.

The state is contacting enrollees by phone, email, and text, and continues to process late applications, said Tori Cuddy, a spokesperson for the Florida Department of Children and Families. Ms. Cuddy did not respond to questions about issues raised in the petitions.

Federal officials are investigating those complaints and any other problems that emerge, said Dan Tsai, director of the Center for Medicaid & CHIP Services. “If we find that the rules are not being followed, we will take action.”

His agency has directed states to automatically reenroll residents using data from other government programs like unemployment and food assistance when possible. Anyone who can’t be approved through that process must act quickly.

“For the past 3 years, people have been told to ignore the mail around this, that the renewal was not going to lead to a termination.” Suddenly that mail matters, he said.

Federal law requires states to tell people why they’re losing Medicaid coverage and how to appeal the decision.

Ms. Harmatz said some cancellation notices in Florida are vague and could violate due process rules. Letters that she’s seen say “your Medicaid for this period is ending” rather than providing a specific reason for disenrollment, like having too high an income or incomplete paperwork.
If a person requests a hearing before their cancellation takes effect, they can stay covered during the appeals process. Even after being disenrolled, many still have a 90-day window to restore coverage.

In New Hampshire, 13% of people deemed ineligible in the first month have asked for extra time to provide the necessary records. “If you’re eligible for Medicaid, we don’t want you to lose it,” said Mr. Lipman.

Rep. Clere pushed Indiana’s Medicaid officials during the May meeting to immediately make changes to avoid people unnecessarily becoming uninsured. One official responded that they’ll learn and improve over time.

“I’m just concerned that we’re going to be ‘learning’ as a result of people losing coverage,” Rep. Clere replied. “So I don’t want to learn at their expense.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Myopia affects roughly one-third of the population worldwide - a figure that is projected to reach 50% by 2050. Low-dose atropine, which helps curb the condition, currently is available in the United States only through compounding pharmacies. The products contain preservatives – raising questions about potential toxicities to the eye – and may not be of pharmaceutical grade. In a new study published in JAMA Ophthalmology, a preservative-free eyedrop containing 0.01% atropine led to significant improvements in several markers of myopia in children who received the experimental therapy.

Methodology

• The CHAMP study was a double-masked, placebo-controlled, randomized, phase 3 trial conducted between Nov. 20, 2017, and Aug. 22, 2022, that involved children at 26 sites in North America and 5 centers in Europe.
• Children received either 0.01% or 0.02% atropine drops once per day.
• Patients were aged 3-16 years. They demonstrated a spherical equivalent refractive error (SER) of −0.50 diopter (D) to −6.00 D astigmatism no worse than −1.50 D.
• Of these patients, 573 were included in a safety analysis, and 489 were included in a modified intention-to-treat analysis.

Takeaways

• After 36 months, the 0.01% dose of atropine was associated with a significantly lower responder proportion (odds ratio, 4.54) and slower progression of SER and axial elongation.
• The effect of the 0.02% dose on responder proportion and SER progression was not statistically significant, but the treatment was associated with slower axial elongation.
• The researchers observed no serious ocular adverse events and few serious nonocular events, none of which was determined to be associated with the treatment.

In practice: According to the researchers, “from a risk/benefit perspective, the efficacy and safety observed suggests that low-dose atropine may provide a treatment option for children aged 3-17 years with myopia progression, which may lead to less frequent or delayed change in glasses, progression to less severe correction, and potentially reduce long-term sequelae, which could lead to vision loss later in life, such as myopic maculopathy.”

Study details: The CHAMP study was led by Karla Zadnik, OD, PhD, of Ohio State University, Columbus, and was funded by Vylulma.

Limitations: The researchers said the trial was potentially limited by the fact that patients switched from the study drug to confounding treatments. In addition, patients at the low and high age ranges were not well represented.

Disclosures: Dr. Zadnik received consultant fees from Vyluma during the study.

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

Myopia affects roughly one-third of the population worldwide - a figure that is projected to reach 50% by 2050. Low-dose atropine, which helps curb the condition, currently is available in the United States only through compounding pharmacies. The products contain preservatives – raising questions about potential toxicities to the eye – and may not be of pharmaceutical grade. In a new study published in JAMA Ophthalmology, a preservative-free eyedrop containing 0.01% atropine led to significant improvements in several markers of myopia in children who received the experimental therapy.

Methodology

• The CHAMP study was a double-masked, placebo-controlled, randomized, phase 3 trial conducted between Nov. 20, 2017, and Aug. 22, 2022, that involved children at 26 sites in North America and 5 centers in Europe.
• Children received either 0.01% or 0.02% atropine drops once per day.
• Patients were aged 3-16 years. They demonstrated a spherical equivalent refractive error (SER) of −0.50 diopter (D) to −6.00 D astigmatism no worse than −1.50 D.
• Of these patients, 573 were included in a safety analysis, and 489 were included in a modified intention-to-treat analysis.

Takeaways

• After 36 months, the 0.01% dose of atropine was associated with a significantly lower responder proportion (odds ratio, 4.54) and slower progression of SER and axial elongation.
• The effect of the 0.02% dose on responder proportion and SER progression was not statistically significant, but the treatment was associated with slower axial elongation.
• The researchers observed no serious ocular adverse events and few serious nonocular events, none of which was determined to be associated with the treatment.

In practice: According to the researchers, “from a risk/benefit perspective, the efficacy and safety observed suggests that low-dose atropine may provide a treatment option for children aged 3-17 years with myopia progression, which may lead to less frequent or delayed change in glasses, progression to less severe correction, and potentially reduce long-term sequelae, which could lead to vision loss later in life, such as myopic maculopathy.”

Study details: The CHAMP study was led by Karla Zadnik, OD, PhD, of Ohio State University, Columbus, and was funded by Vylulma.

Limitations: The researchers said the trial was potentially limited by the fact that patients switched from the study drug to confounding treatments. In addition, patients at the low and high age ranges were not well represented.

Disclosures: Dr. Zadnik received consultant fees from Vyluma during the study.

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

Myopia affects roughly one-third of the population worldwide - a figure that is projected to reach 50% by 2050. Low-dose atropine, which helps curb the condition, currently is available in the United States only through compounding pharmacies. The products contain preservatives – raising questions about potential toxicities to the eye – and may not be of pharmaceutical grade. In a new study published in JAMA Ophthalmology, a preservative-free eyedrop containing 0.01% atropine led to significant improvements in several markers of myopia in children who received the experimental therapy.

Methodology

• The CHAMP study was a double-masked, placebo-controlled, randomized, phase 3 trial conducted between Nov. 20, 2017, and Aug. 22, 2022, that involved children at 26 sites in North America and 5 centers in Europe.
• Children received either 0.01% or 0.02% atropine drops once per day.
• Patients were aged 3-16 years. They demonstrated a spherical equivalent refractive error (SER) of −0.50 diopter (D) to −6.00 D astigmatism no worse than −1.50 D.
• Of these patients, 573 were included in a safety analysis, and 489 were included in a modified intention-to-treat analysis.

Takeaways

• After 36 months, the 0.01% dose of atropine was associated with a significantly lower responder proportion (odds ratio, 4.54) and slower progression of SER and axial elongation.
• The effect of the 0.02% dose on responder proportion and SER progression was not statistically significant, but the treatment was associated with slower axial elongation.
• The researchers observed no serious ocular adverse events and few serious nonocular events, none of which was determined to be associated with the treatment.

In practice: According to the researchers, “from a risk/benefit perspective, the efficacy and safety observed suggests that low-dose atropine may provide a treatment option for children aged 3-17 years with myopia progression, which may lead to less frequent or delayed change in glasses, progression to less severe correction, and potentially reduce long-term sequelae, which could lead to vision loss later in life, such as myopic maculopathy.”

Study details: The CHAMP study was led by Karla Zadnik, OD, PhD, of Ohio State University, Columbus, and was funded by Vylulma.

Limitations: The researchers said the trial was potentially limited by the fact that patients switched from the study drug to confounding treatments. In addition, patients at the low and high age ranges were not well represented.

Disclosures: Dr. Zadnik received consultant fees from Vyluma during the study.

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

The American Psychiatric Association has released an online screening and assessment tool for eating disorders.

“People with eating disorders have a high rate of mortality and [the disorder is] growing in prevalence among young adults and adolescents,” APA CEO and medical director Saul Levin, MD, MPA, said in a news release.

“It is vital that we equip our clinicians, especially primary care clinicians, with the latest evidence from the APA to empower their decision-making and improve care for their patients,” Dr. Levin added.

The clinical decision support tool was developed by the APA’s guideline writing group in collaboration with AvoMD, a software company that translates clinical evidence into the workflow.

The tool guides clinicians through the screening, assessing, diagnosing, and treatment planning of patients with anorexia nervosa, bulimia nervosa, binge-eating disorder, and other eating disorders.

It’s available free in the electronic health record, on the APA website, and on the AvoMD mobile app.

The tool is based on the APA’s updated practice guidelines for the management of eating disorders, which were released in March.

The tool incorporates guidance on screening tools, levels of care, nutrition, exercise, psychotherapy, and more. Additionally, the tool provides a summary of responses that can be leveraged for easy documentation.

It is intended for use by pediatricians, family physicians, and other primary care and mental health clinicians, including psychiatrists and therapists.

Data published earlier this year show that more than 1 in 5 children worldwide are at risk of developing an eating disorder. Girls are significantly more likely than boys to have disordered eating.

Early diagnosis and treatment are associated with a higher rate of recovery, and extended illness is associated with potentially devastating consequences, the APA notes.

“As an internal medicine physician myself, I see a wide variety of patients and clinical issues,” Joongheum Park, MD, head of product and engineering at AvoMD, said in the release.

“Easy access to the expertise in me and my peers’ workflow is essential to ensuring high-quality care, and partnering with the leading authority on eating disorders to provide this tool will improve clinician efficiency and most importantly, patient outcomes,” Dr. Park added.

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

The American Psychiatric Association has released an online screening and assessment tool for eating disorders.

“People with eating disorders have a high rate of mortality and [the disorder is] growing in prevalence among young adults and adolescents,” APA CEO and medical director Saul Levin, MD, MPA, said in a news release.

“It is vital that we equip our clinicians, especially primary care clinicians, with the latest evidence from the APA to empower their decision-making and improve care for their patients,” Dr. Levin added.

The clinical decision support tool was developed by the APA’s guideline writing group in collaboration with AvoMD, a software company that translates clinical evidence into the workflow.

The tool guides clinicians through the screening, assessing, diagnosing, and treatment planning of patients with anorexia nervosa, bulimia nervosa, binge-eating disorder, and other eating disorders.

It’s available free in the electronic health record, on the APA website, and on the AvoMD mobile app.

The tool is based on the APA’s updated practice guidelines for the management of eating disorders, which were released in March.

The tool incorporates guidance on screening tools, levels of care, nutrition, exercise, psychotherapy, and more. Additionally, the tool provides a summary of responses that can be leveraged for easy documentation.

It is intended for use by pediatricians, family physicians, and other primary care and mental health clinicians, including psychiatrists and therapists.

Data published earlier this year show that more than 1 in 5 children worldwide are at risk of developing an eating disorder. Girls are significantly more likely than boys to have disordered eating.

Early diagnosis and treatment are associated with a higher rate of recovery, and extended illness is associated with potentially devastating consequences, the APA notes.

“As an internal medicine physician myself, I see a wide variety of patients and clinical issues,” Joongheum Park, MD, head of product and engineering at AvoMD, said in the release.

“Easy access to the expertise in me and my peers’ workflow is essential to ensuring high-quality care, and partnering with the leading authority on eating disorders to provide this tool will improve clinician efficiency and most importantly, patient outcomes,” Dr. Park added.

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

The American Psychiatric Association has released an online screening and assessment tool for eating disorders.

“People with eating disorders have a high rate of mortality and [the disorder is] growing in prevalence among young adults and adolescents,” APA CEO and medical director Saul Levin, MD, MPA, said in a news release.

“It is vital that we equip our clinicians, especially primary care clinicians, with the latest evidence from the APA to empower their decision-making and improve care for their patients,” Dr. Levin added.

The clinical decision support tool was developed by the APA’s guideline writing group in collaboration with AvoMD, a software company that translates clinical evidence into the workflow.

The tool guides clinicians through the screening, assessing, diagnosing, and treatment planning of patients with anorexia nervosa, bulimia nervosa, binge-eating disorder, and other eating disorders.

It’s available free in the electronic health record, on the APA website, and on the AvoMD mobile app.

The tool is based on the APA’s updated practice guidelines for the management of eating disorders, which were released in March.

The tool incorporates guidance on screening tools, levels of care, nutrition, exercise, psychotherapy, and more. Additionally, the tool provides a summary of responses that can be leveraged for easy documentation.

It is intended for use by pediatricians, family physicians, and other primary care and mental health clinicians, including psychiatrists and therapists.

Data published earlier this year show that more than 1 in 5 children worldwide are at risk of developing an eating disorder. Girls are significantly more likely than boys to have disordered eating.

Early diagnosis and treatment are associated with a higher rate of recovery, and extended illness is associated with potentially devastating consequences, the APA notes.

“As an internal medicine physician myself, I see a wide variety of patients and clinical issues,” Joongheum Park, MD, head of product and engineering at AvoMD, said in the release.

“Easy access to the expertise in me and my peers’ workflow is essential to ensuring high-quality care, and partnering with the leading authority on eating disorders to provide this tool will improve clinician efficiency and most importantly, patient outcomes,” Dr. Park added.

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

MANNHEIM, Germany – Lomitapide, which reduces lipoprotein production in the liver, could help manage pediatric homozygous familial hypercholesterolemia (HoFH), suggest results of a trial that showed large reductions in circulating lipids.

The research was presented May 23 at the 91st European Atherosclerosis Society Congress.

Lomitapide inhibits microsomal triglyceride transfer protein, which plays a key role in apolipoprotein B-containing lipoprotein assembly and secretion in the liver and intestines. Crucially, the drug acts independently of the LDL cholesterol receptor.

It was approved in December 2012 by the U.S. Food and Drug Administration for use in adults with HoFH, sold under the name Juxtapid, and by the European Medicines Agency, where the brand name is Lojuxta.

The current trial involved more than 40 children and teenagers with HoFH aged 5-17 years; they were treated with the drug for 24 weeks, resulting in reductions of low density lipoprotein cholesterol of almost 54%, with nearly 42% reaching target levels.

The drug was also associated with marked reductions in other key lipids of at least 50%. However, 67% of patients also experienced gastrointestinal adverse events, and around 25% saw their levels of liver enzymes increase.
 

Early diagnosis ‘imperative’

The findings show that the “early diagnosis and treatment of HoFH is imperative,” said study presenter Luis Masana, MD, PhD, director of the Vascular Medicine and Metabolism Unit at Sant Joan de Reus University Hospital, Tarragona, Spain.

“I think that, with these results, we are bringing a new hope for this group of patients,” he continued. “I also think we will increase the quality of life, not just of the patients but also all the families involved in [managing] this problem.”

Session co-chair Andreas Zirlik, MD, PhD, head of the department of cardiology and chairman of the University Heart Center Graz, LKH-University Hospital, and Medical University of Graz (Austria), was more circumspect in his appraisal of the results.

He told this news organization that it is “always very difficult to establish therapy in pediatrics,” and believes that the drug “will give us an additional option” in managing HoFH.

However, Dr. Zirlik warned that he is a “little bit concerned” about lomitapide’s adverse event profile, and “would need to see a little bit deeper into the safety data.”

Highlighting the elevations in liver enzymes of around 25%, he asked: “What does it mean?” And how will it “play out in the long run?”

Beyond lomitapide, Dr. Zirlik pointed out that there are other drugs that have shown potential in managing HoFH and could potentially be used in the pediatric population, such as angiopoietin-like 3 protein (ANGPTL3) inhibitors and small interfering RNA (siRNA) compounds that target upstream production. “So, let’s see how they pan out,” he said.
 

Life-limiting condition

HoFH is an “ultra-rare, life-limiting condition,” with an estimated prevalence of approximately 3 per 1 million people, and a life expectancy in untreated patients of just 18 years, Dr. Masana said during his presentation.

Case series of lomitapide use in pediatric HoFH patients have shown encouraging results that are consistent with those seen in adults, he noted, with many able to achieve their LDL cholesterol target and stop or reduce apheresis.

To investigate further, a phase 3, single arm, open-label study was conducted. Following screening, 46 children and teenagers with HoFH underwent a 6- to 12-week run-in period, during which they were put on a low-fat diet with nutritional supplements.

“As you can imagine,” Dr. Masana said, “we are reducing the capacity for fat absorption with lomitapide, so the supplements and low-fat diet are necessary.”

Of these, 43 participants then entered a 24-week treatment period in which they were started on one of three doses, before undergoing dose escalation to the maximally tolerated dose. This was followed by an 80-week open-label safety phase, in which they continued on the maximally tolerated dose, then a follow-up period.

For the current presentation, Dr. Masana focused on the efficacy phase, showing that the mean age of participants was 10.7 years and that 55.8% were female. The HoFH diagnosis was confirmed genetically in 88.4% of cases.

Results showed that lomitapide was associated with a significant reduction in LDL cholesterol levels, from 435.8 mg/dL at baseline to 176.5 mg/dL at Week 24, which corresponded to a 53.5% overall reduction (P < .0001).

This meant that 41.9% of patients achieved their EAS LDL cholesterol target of less than 135 mg/dL at some point during the 24-week treatment period.

Stratifying by age, the reduction between baseline and week 24 was 538.5 mg/dL to 207.2 mg/dL, or 56.5%, in the 20 children aged 5-10 years, and 346.5 mg/dL to 149.9 mg/L, or 50.9%, in the 23 patients aged 11-17 years.

Dr. Masana explained that the results were “a little bit better in the younger group because they were receiving less treatment at this stage of the disease” than the older group.

He showed that lomitapide was associated with significant reductions in other lipid markers, including a 53.9% reduction in non–HDL cholesterol (P < .0001), a 50.1% drop in total cholesterol (P < .0001), and a 50.2% fall in very-low-density lipoprotein cholesterol (P < .0001).

Results showed 93% of patients experienced treatment-related adverse events, with 11.6% having serious events and 4.7% having events that led to study discontinuation. There was one (2.3%) major adverse cardiac event but no deaths.

He said that, despite these figures, the adverse events were “mostly mild or moderate.”

The majority (67%) of patients nevertheless had gastrointestinal adverse events, which were, “in general, associated with a lack of adherence to the low-fat diet.”

Aspartate aminotransferase levels were elevated in 23% of patients, while 28% had elevations in alanine aminotransferase, which were described by Dr. Masana as “moderate.”

The study was sponsored by Amryt Pharma. Dr. Masana declares relationships with Amarin, Amryt, Daiichi-Sankyo, Novartis, Sanofi, Servier, Servier, and Viatrix.

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

MANNHEIM, Germany – Lomitapide, which reduces lipoprotein production in the liver, could help manage pediatric homozygous familial hypercholesterolemia (HoFH), suggest results of a trial that showed large reductions in circulating lipids.

The research was presented May 23 at the 91st European Atherosclerosis Society Congress.

Lomitapide inhibits microsomal triglyceride transfer protein, which plays a key role in apolipoprotein B-containing lipoprotein assembly and secretion in the liver and intestines. Crucially, the drug acts independently of the LDL cholesterol receptor.

It was approved in December 2012 by the U.S. Food and Drug Administration for use in adults with HoFH, sold under the name Juxtapid, and by the European Medicines Agency, where the brand name is Lojuxta.

The current trial involved more than 40 children and teenagers with HoFH aged 5-17 years; they were treated with the drug for 24 weeks, resulting in reductions of low density lipoprotein cholesterol of almost 54%, with nearly 42% reaching target levels.

The drug was also associated with marked reductions in other key lipids of at least 50%. However, 67% of patients also experienced gastrointestinal adverse events, and around 25% saw their levels of liver enzymes increase.
 

Early diagnosis ‘imperative’

The findings show that the “early diagnosis and treatment of HoFH is imperative,” said study presenter Luis Masana, MD, PhD, director of the Vascular Medicine and Metabolism Unit at Sant Joan de Reus University Hospital, Tarragona, Spain.

“I think that, with these results, we are bringing a new hope for this group of patients,” he continued. “I also think we will increase the quality of life, not just of the patients but also all the families involved in [managing] this problem.”

Session co-chair Andreas Zirlik, MD, PhD, head of the department of cardiology and chairman of the University Heart Center Graz, LKH-University Hospital, and Medical University of Graz (Austria), was more circumspect in his appraisal of the results.

He told this news organization that it is “always very difficult to establish therapy in pediatrics,” and believes that the drug “will give us an additional option” in managing HoFH.

However, Dr. Zirlik warned that he is a “little bit concerned” about lomitapide’s adverse event profile, and “would need to see a little bit deeper into the safety data.”

Highlighting the elevations in liver enzymes of around 25%, he asked: “What does it mean?” And how will it “play out in the long run?”

Beyond lomitapide, Dr. Zirlik pointed out that there are other drugs that have shown potential in managing HoFH and could potentially be used in the pediatric population, such as angiopoietin-like 3 protein (ANGPTL3) inhibitors and small interfering RNA (siRNA) compounds that target upstream production. “So, let’s see how they pan out,” he said.
 

Life-limiting condition

HoFH is an “ultra-rare, life-limiting condition,” with an estimated prevalence of approximately 3 per 1 million people, and a life expectancy in untreated patients of just 18 years, Dr. Masana said during his presentation.

Case series of lomitapide use in pediatric HoFH patients have shown encouraging results that are consistent with those seen in adults, he noted, with many able to achieve their LDL cholesterol target and stop or reduce apheresis.

To investigate further, a phase 3, single arm, open-label study was conducted. Following screening, 46 children and teenagers with HoFH underwent a 6- to 12-week run-in period, during which they were put on a low-fat diet with nutritional supplements.

“As you can imagine,” Dr. Masana said, “we are reducing the capacity for fat absorption with lomitapide, so the supplements and low-fat diet are necessary.”

Of these, 43 participants then entered a 24-week treatment period in which they were started on one of three doses, before undergoing dose escalation to the maximally tolerated dose. This was followed by an 80-week open-label safety phase, in which they continued on the maximally tolerated dose, then a follow-up period.

For the current presentation, Dr. Masana focused on the efficacy phase, showing that the mean age of participants was 10.7 years and that 55.8% were female. The HoFH diagnosis was confirmed genetically in 88.4% of cases.

Results showed that lomitapide was associated with a significant reduction in LDL cholesterol levels, from 435.8 mg/dL at baseline to 176.5 mg/dL at Week 24, which corresponded to a 53.5% overall reduction (P < .0001).

This meant that 41.9% of patients achieved their EAS LDL cholesterol target of less than 135 mg/dL at some point during the 24-week treatment period.

Stratifying by age, the reduction between baseline and week 24 was 538.5 mg/dL to 207.2 mg/dL, or 56.5%, in the 20 children aged 5-10 years, and 346.5 mg/dL to 149.9 mg/L, or 50.9%, in the 23 patients aged 11-17 years.

Dr. Masana explained that the results were “a little bit better in the younger group because they were receiving less treatment at this stage of the disease” than the older group.

He showed that lomitapide was associated with significant reductions in other lipid markers, including a 53.9% reduction in non–HDL cholesterol (P < .0001), a 50.1% drop in total cholesterol (P < .0001), and a 50.2% fall in very-low-density lipoprotein cholesterol (P < .0001).

Results showed 93% of patients experienced treatment-related adverse events, with 11.6% having serious events and 4.7% having events that led to study discontinuation. There was one (2.3%) major adverse cardiac event but no deaths.

He said that, despite these figures, the adverse events were “mostly mild or moderate.”

The majority (67%) of patients nevertheless had gastrointestinal adverse events, which were, “in general, associated with a lack of adherence to the low-fat diet.”

Aspartate aminotransferase levels were elevated in 23% of patients, while 28% had elevations in alanine aminotransferase, which were described by Dr. Masana as “moderate.”

The study was sponsored by Amryt Pharma. Dr. Masana declares relationships with Amarin, Amryt, Daiichi-Sankyo, Novartis, Sanofi, Servier, Servier, and Viatrix.

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

MANNHEIM, Germany – Lomitapide, which reduces lipoprotein production in the liver, could help manage pediatric homozygous familial hypercholesterolemia (HoFH), suggest results of a trial that showed large reductions in circulating lipids.

The research was presented May 23 at the 91st European Atherosclerosis Society Congress.

Lomitapide inhibits microsomal triglyceride transfer protein, which plays a key role in apolipoprotein B-containing lipoprotein assembly and secretion in the liver and intestines. Crucially, the drug acts independently of the LDL cholesterol receptor.

It was approved in December 2012 by the U.S. Food and Drug Administration for use in adults with HoFH, sold under the name Juxtapid, and by the European Medicines Agency, where the brand name is Lojuxta.

The current trial involved more than 40 children and teenagers with HoFH aged 5-17 years; they were treated with the drug for 24 weeks, resulting in reductions of low density lipoprotein cholesterol of almost 54%, with nearly 42% reaching target levels.

The drug was also associated with marked reductions in other key lipids of at least 50%. However, 67% of patients also experienced gastrointestinal adverse events, and around 25% saw their levels of liver enzymes increase.
 

Early diagnosis ‘imperative’

The findings show that the “early diagnosis and treatment of HoFH is imperative,” said study presenter Luis Masana, MD, PhD, director of the Vascular Medicine and Metabolism Unit at Sant Joan de Reus University Hospital, Tarragona, Spain.

“I think that, with these results, we are bringing a new hope for this group of patients,” he continued. “I also think we will increase the quality of life, not just of the patients but also all the families involved in [managing] this problem.”

Session co-chair Andreas Zirlik, MD, PhD, head of the department of cardiology and chairman of the University Heart Center Graz, LKH-University Hospital, and Medical University of Graz (Austria), was more circumspect in his appraisal of the results.

He told this news organization that it is “always very difficult to establish therapy in pediatrics,” and believes that the drug “will give us an additional option” in managing HoFH.

However, Dr. Zirlik warned that he is a “little bit concerned” about lomitapide’s adverse event profile, and “would need to see a little bit deeper into the safety data.”

Highlighting the elevations in liver enzymes of around 25%, he asked: “What does it mean?” And how will it “play out in the long run?”

Beyond lomitapide, Dr. Zirlik pointed out that there are other drugs that have shown potential in managing HoFH and could potentially be used in the pediatric population, such as angiopoietin-like 3 protein (ANGPTL3) inhibitors and small interfering RNA (siRNA) compounds that target upstream production. “So, let’s see how they pan out,” he said.
 

Life-limiting condition

HoFH is an “ultra-rare, life-limiting condition,” with an estimated prevalence of approximately 3 per 1 million people, and a life expectancy in untreated patients of just 18 years, Dr. Masana said during his presentation.

Case series of lomitapide use in pediatric HoFH patients have shown encouraging results that are consistent with those seen in adults, he noted, with many able to achieve their LDL cholesterol target and stop or reduce apheresis.

To investigate further, a phase 3, single arm, open-label study was conducted. Following screening, 46 children and teenagers with HoFH underwent a 6- to 12-week run-in period, during which they were put on a low-fat diet with nutritional supplements.

“As you can imagine,” Dr. Masana said, “we are reducing the capacity for fat absorption with lomitapide, so the supplements and low-fat diet are necessary.”

Of these, 43 participants then entered a 24-week treatment period in which they were started on one of three doses, before undergoing dose escalation to the maximally tolerated dose. This was followed by an 80-week open-label safety phase, in which they continued on the maximally tolerated dose, then a follow-up period.

For the current presentation, Dr. Masana focused on the efficacy phase, showing that the mean age of participants was 10.7 years and that 55.8% were female. The HoFH diagnosis was confirmed genetically in 88.4% of cases.

Results showed that lomitapide was associated with a significant reduction in LDL cholesterol levels, from 435.8 mg/dL at baseline to 176.5 mg/dL at Week 24, which corresponded to a 53.5% overall reduction (P < .0001).

This meant that 41.9% of patients achieved their EAS LDL cholesterol target of less than 135 mg/dL at some point during the 24-week treatment period.

Stratifying by age, the reduction between baseline and week 24 was 538.5 mg/dL to 207.2 mg/dL, or 56.5%, in the 20 children aged 5-10 years, and 346.5 mg/dL to 149.9 mg/L, or 50.9%, in the 23 patients aged 11-17 years.

Dr. Masana explained that the results were “a little bit better in the younger group because they were receiving less treatment at this stage of the disease” than the older group.

He showed that lomitapide was associated with significant reductions in other lipid markers, including a 53.9% reduction in non–HDL cholesterol (P < .0001), a 50.1% drop in total cholesterol (P < .0001), and a 50.2% fall in very-low-density lipoprotein cholesterol (P < .0001).

Results showed 93% of patients experienced treatment-related adverse events, with 11.6% having serious events and 4.7% having events that led to study discontinuation. There was one (2.3%) major adverse cardiac event but no deaths.

He said that, despite these figures, the adverse events were “mostly mild or moderate.”

The majority (67%) of patients nevertheless had gastrointestinal adverse events, which were, “in general, associated with a lack of adherence to the low-fat diet.”

Aspartate aminotransferase levels were elevated in 23% of patients, while 28% had elevations in alanine aminotransferase, which were described by Dr. Masana as “moderate.”

The study was sponsored by Amryt Pharma. Dr. Masana declares relationships with Amarin, Amryt, Daiichi-Sankyo, Novartis, Sanofi, Servier, Servier, and Viatrix.

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

A team of researchers has identified genomic variants that cause disabling pansclerotic morphea (DPM), a rare, severe inflammatory skin disorder, and report that the Janus kinase (JAK) inhibitor ruxolitinib may be a useful therapy, especially in patients who have not responded to other interventions.

DPM was first reported in 1923, and while a genetic cause has been suspected, it had not been identified until now. The disease is the most severe form of deep morphea, which affects individuals with juvenile localized scleroderma. Patients, generally children under age 14, experience rapid sclerosis of all layers of the skin, fascia, muscle, and bone. DPM is also deadly: Most patients do not live more than 10 years after diagnosis, as they contract squamous cell carcinoma, restrictive pulmonary disease, sepsis, and gangrene.

In the study, published in the New England Journal of Medicine, the researchers discovered that people with DPM have an overactive version of the protein STAT4, which regulates inflammation and wound healing. The scientists studied four patients from three unrelated families with an autosomal dominant pattern of inheritance of DPM.

“Researchers previously thought that this disorder was caused by the immune system attacking the skin,” Sarah Blackstone, a predoctoral fellow in the inflammatory disease section at the National Human Genome Research Institute and co–first author of the study, said in a statement from the National Institutes of Health describing the results. “However, we found that this is an oversimplification, and that both skin and the immune system play an active role in disabling pansclerotic morphea,” added Ms. Blackstone, also a medical student at the University of South Dakota, Sioux Falls.

The overactive STAT4 protein creates a positive feedback loop of inflammation and impaired wound-healing. By targeting JAK, the researchers were able to stop the feedback and patients’ wounds dramatically improved. After 18 months of treatment with oral ruxolitinib, one patient had discontinued all other medications, and had complete resolution of a chest rash, substantial clearing on the arms and legs, and global clinical improvement.

The authors said that oral systemic JAK inhibitor therapy is preferred over topical therapy. Their research also suggested that anti–interleukin-6 monoclonal antibodies – such as tocilizumab, approved for indications that include rheumatoid arthritis and systemic sclerosis–associated interstitial lung disease, “may be an alternative therapy or may be useful in combination with JAK inhibitors in patients with DPM,” the authors wrote.

Most current DPM therapies – including methotrexate, mycophenolate mofetil, and ultraviolet A light therapy – have been ineffective, and some have severe side effects.

“The findings of this study open doors for JAK inhibitors to be a potential treatment for other inflammatory skin disorders or disorders related to tissue scarring, whether it is scarring of the lungs, liver or bone marrow,” Dan Kastner, MD, PhD, an NIH distinguished investigator, head of the NHGRI’s inflammatory disease section, and a senior author of the paper, said in the NIH statement.

“We hope to continue studying other molecules in this pathway and how they are altered in patients with disabling pansclerotic morphea and related conditions to find clues to understanding a broader array of more common diseases,” Lori Broderick, MD, PhD, a senior author of the paper and an associate professor at University of California, San Diego, said in the statement.

The study was led by researchers at NHGRI in collaboration with researchers from UCSD and the University of Pittsburgh. Researchers from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the National Institute of Allergy and Infectious Diseases also participated.

The study was supported by grants from the American Academy of Allergy, Asthma, and Immunology Foundation; the Ludwig Institute for Cancer Research; the University of California, San Diego, department of pediatrics; and the Novo Nordisk Foundation. Additional support and grants were given by the Deutsche Forschungsgemeinschaft, various institutes at the NIH, the California Institute for Regenerative Medicine, the Hydrocephalus Association, the Scleroderma Research Foundation, the Biowulf High-Performance Computing Cluster of the Center for Information Technology, the Undiagnosed Diseases Program of the Common Fund of the Office of the Director of the NIH, and the NIH Clinical Center.

A team of researchers has identified genomic variants that cause disabling pansclerotic morphea (DPM), a rare, severe inflammatory skin disorder, and report that the Janus kinase (JAK) inhibitor ruxolitinib may be a useful therapy, especially in patients who have not responded to other interventions.

DPM was first reported in 1923, and while a genetic cause has been suspected, it had not been identified until now. The disease is the most severe form of deep morphea, which affects individuals with juvenile localized scleroderma. Patients, generally children under age 14, experience rapid sclerosis of all layers of the skin, fascia, muscle, and bone. DPM is also deadly: Most patients do not live more than 10 years after diagnosis, as they contract squamous cell carcinoma, restrictive pulmonary disease, sepsis, and gangrene.

In the study, published in the New England Journal of Medicine, the researchers discovered that people with DPM have an overactive version of the protein STAT4, which regulates inflammation and wound healing. The scientists studied four patients from three unrelated families with an autosomal dominant pattern of inheritance of DPM.

“Researchers previously thought that this disorder was caused by the immune system attacking the skin,” Sarah Blackstone, a predoctoral fellow in the inflammatory disease section at the National Human Genome Research Institute and co–first author of the study, said in a statement from the National Institutes of Health describing the results. “However, we found that this is an oversimplification, and that both skin and the immune system play an active role in disabling pansclerotic morphea,” added Ms. Blackstone, also a medical student at the University of South Dakota, Sioux Falls.

The overactive STAT4 protein creates a positive feedback loop of inflammation and impaired wound-healing. By targeting JAK, the researchers were able to stop the feedback and patients’ wounds dramatically improved. After 18 months of treatment with oral ruxolitinib, one patient had discontinued all other medications, and had complete resolution of a chest rash, substantial clearing on the arms and legs, and global clinical improvement.

The authors said that oral systemic JAK inhibitor therapy is preferred over topical therapy. Their research also suggested that anti–interleukin-6 monoclonal antibodies – such as tocilizumab, approved for indications that include rheumatoid arthritis and systemic sclerosis–associated interstitial lung disease, “may be an alternative therapy or may be useful in combination with JAK inhibitors in patients with DPM,” the authors wrote.

Most current DPM therapies – including methotrexate, mycophenolate mofetil, and ultraviolet A light therapy – have been ineffective, and some have severe side effects.

“The findings of this study open doors for JAK inhibitors to be a potential treatment for other inflammatory skin disorders or disorders related to tissue scarring, whether it is scarring of the lungs, liver or bone marrow,” Dan Kastner, MD, PhD, an NIH distinguished investigator, head of the NHGRI’s inflammatory disease section, and a senior author of the paper, said in the NIH statement.

“We hope to continue studying other molecules in this pathway and how they are altered in patients with disabling pansclerotic morphea and related conditions to find clues to understanding a broader array of more common diseases,” Lori Broderick, MD, PhD, a senior author of the paper and an associate professor at University of California, San Diego, said in the statement.

The study was led by researchers at NHGRI in collaboration with researchers from UCSD and the University of Pittsburgh. Researchers from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the National Institute of Allergy and Infectious Diseases also participated.

The study was supported by grants from the American Academy of Allergy, Asthma, and Immunology Foundation; the Ludwig Institute for Cancer Research; the University of California, San Diego, department of pediatrics; and the Novo Nordisk Foundation. Additional support and grants were given by the Deutsche Forschungsgemeinschaft, various institutes at the NIH, the California Institute for Regenerative Medicine, the Hydrocephalus Association, the Scleroderma Research Foundation, the Biowulf High-Performance Computing Cluster of the Center for Information Technology, the Undiagnosed Diseases Program of the Common Fund of the Office of the Director of the NIH, and the NIH Clinical Center.

A team of researchers has identified genomic variants that cause disabling pansclerotic morphea (DPM), a rare, severe inflammatory skin disorder, and report that the Janus kinase (JAK) inhibitor ruxolitinib may be a useful therapy, especially in patients who have not responded to other interventions.

DPM was first reported in 1923, and while a genetic cause has been suspected, it had not been identified until now. The disease is the most severe form of deep morphea, which affects individuals with juvenile localized scleroderma. Patients, generally children under age 14, experience rapid sclerosis of all layers of the skin, fascia, muscle, and bone. DPM is also deadly: Most patients do not live more than 10 years after diagnosis, as they contract squamous cell carcinoma, restrictive pulmonary disease, sepsis, and gangrene.

In the study, published in the New England Journal of Medicine, the researchers discovered that people with DPM have an overactive version of the protein STAT4, which regulates inflammation and wound healing. The scientists studied four patients from three unrelated families with an autosomal dominant pattern of inheritance of DPM.

“Researchers previously thought that this disorder was caused by the immune system attacking the skin,” Sarah Blackstone, a predoctoral fellow in the inflammatory disease section at the National Human Genome Research Institute and co–first author of the study, said in a statement from the National Institutes of Health describing the results. “However, we found that this is an oversimplification, and that both skin and the immune system play an active role in disabling pansclerotic morphea,” added Ms. Blackstone, also a medical student at the University of South Dakota, Sioux Falls.

The overactive STAT4 protein creates a positive feedback loop of inflammation and impaired wound-healing. By targeting JAK, the researchers were able to stop the feedback and patients’ wounds dramatically improved. After 18 months of treatment with oral ruxolitinib, one patient had discontinued all other medications, and had complete resolution of a chest rash, substantial clearing on the arms and legs, and global clinical improvement.

The authors said that oral systemic JAK inhibitor therapy is preferred over topical therapy. Their research also suggested that anti–interleukin-6 monoclonal antibodies – such as tocilizumab, approved for indications that include rheumatoid arthritis and systemic sclerosis–associated interstitial lung disease, “may be an alternative therapy or may be useful in combination with JAK inhibitors in patients with DPM,” the authors wrote.

Most current DPM therapies – including methotrexate, mycophenolate mofetil, and ultraviolet A light therapy – have been ineffective, and some have severe side effects.

“The findings of this study open doors for JAK inhibitors to be a potential treatment for other inflammatory skin disorders or disorders related to tissue scarring, whether it is scarring of the lungs, liver or bone marrow,” Dan Kastner, MD, PhD, an NIH distinguished investigator, head of the NHGRI’s inflammatory disease section, and a senior author of the paper, said in the NIH statement.

“We hope to continue studying other molecules in this pathway and how they are altered in patients with disabling pansclerotic morphea and related conditions to find clues to understanding a broader array of more common diseases,” Lori Broderick, MD, PhD, a senior author of the paper and an associate professor at University of California, San Diego, said in the statement.

The study was led by researchers at NHGRI in collaboration with researchers from UCSD and the University of Pittsburgh. Researchers from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the National Institute of Allergy and Infectious Diseases also participated.

The study was supported by grants from the American Academy of Allergy, Asthma, and Immunology Foundation; the Ludwig Institute for Cancer Research; the University of California, San Diego, department of pediatrics; and the Novo Nordisk Foundation. Additional support and grants were given by the Deutsche Forschungsgemeinschaft, various institutes at the NIH, the California Institute for Regenerative Medicine, the Hydrocephalus Association, the Scleroderma Research Foundation, the Biowulf High-Performance Computing Cluster of the Center for Information Technology, the Undiagnosed Diseases Program of the Common Fund of the Office of the Director of the NIH, and the NIH Clinical Center.

More children are getting surgical procedures to lose weight, according to a report published in JAMA Pediatrics.

Metabolic and bariatric surgeries have been on the rise among youths aged 10 to 19 since 2016, the report says. From 2020 to 2021, the number shot up by 19%.

The procedures change parts of the digestive system, helping the person feel more full and less hungry – thereby contributing to weight loss.

Among American children, obesity affects 20%, or 15 million people between 2 and 19. In addition, more are becoming afflicted with severe obesity, with a body mass index 20% higher than the marker for obesity.

“Behavioral lifestyle interventions alone do not result in long-term, clinically important weight loss among youth with severe obesity,” the study’s authors wrote. “Metabolic and bariatric surgery (MBS) is a safe and effective treatment.”

The American Academy of Pediatrics updated its guidelines for the treatment of obesity this year for the first time in 15 years, CNN reported. “The new guidelines urge prompt use of behavior therapy and lifestyle changes and, for the first time, recommend surgery and medications for some young people,” CNN wrote.

Black and Hispanic children have higher rates of childhood obesity, the CDC says. Use of surgeries rose 42% among Black youths and 53% among Hispanic youths between 2020 and 2021.

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

More children are getting surgical procedures to lose weight, according to a report published in JAMA Pediatrics.

Metabolic and bariatric surgeries have been on the rise among youths aged 10 to 19 since 2016, the report says. From 2020 to 2021, the number shot up by 19%.

The procedures change parts of the digestive system, helping the person feel more full and less hungry – thereby contributing to weight loss.

Among American children, obesity affects 20%, or 15 million people between 2 and 19. In addition, more are becoming afflicted with severe obesity, with a body mass index 20% higher than the marker for obesity.

“Behavioral lifestyle interventions alone do not result in long-term, clinically important weight loss among youth with severe obesity,” the study’s authors wrote. “Metabolic and bariatric surgery (MBS) is a safe and effective treatment.”

The American Academy of Pediatrics updated its guidelines for the treatment of obesity this year for the first time in 15 years, CNN reported. “The new guidelines urge prompt use of behavior therapy and lifestyle changes and, for the first time, recommend surgery and medications for some young people,” CNN wrote.

Black and Hispanic children have higher rates of childhood obesity, the CDC says. Use of surgeries rose 42% among Black youths and 53% among Hispanic youths between 2020 and 2021.

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

More children are getting surgical procedures to lose weight, according to a report published in JAMA Pediatrics.

Metabolic and bariatric surgeries have been on the rise among youths aged 10 to 19 since 2016, the report says. From 2020 to 2021, the number shot up by 19%.

The procedures change parts of the digestive system, helping the person feel more full and less hungry – thereby contributing to weight loss.

Among American children, obesity affects 20%, or 15 million people between 2 and 19. In addition, more are becoming afflicted with severe obesity, with a body mass index 20% higher than the marker for obesity.

“Behavioral lifestyle interventions alone do not result in long-term, clinically important weight loss among youth with severe obesity,” the study’s authors wrote. “Metabolic and bariatric surgery (MBS) is a safe and effective treatment.”

The American Academy of Pediatrics updated its guidelines for the treatment of obesity this year for the first time in 15 years, CNN reported. “The new guidelines urge prompt use of behavior therapy and lifestyle changes and, for the first time, recommend surgery and medications for some young people,” CNN wrote.

Black and Hispanic children have higher rates of childhood obesity, the CDC says. Use of surgeries rose 42% among Black youths and 53% among Hispanic youths between 2020 and 2021.

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

SAN FRANCISCO – Children randomly assigned to foster care after being institutionalized in infancy have significantly better cognitive and physical outcomes and less severe symptoms of psychopathology than their peers who remain in institutional care, results of a new study suggest.

The study shows that sustained recovery is possible after severe, early-life adversity, study author Kathryn L. Humphreys, PhD, assistant professor, department of psychology and human development, Vanderbilt University, Nashville, Tenn., said in an interview.

Vanderbilt University

“Given the strong evidence from the present study, I hope physicians will play a role in promoting family placements as an alternative to institutional care for children who have been orphaned,” she said.

The findings were presented at the annual meeting of the American Psychiatric Association and were published online in the American Journal of Psychiatry.

Millions of children around the world experience psychosocial deprivation while living in institutions, and many more are neglected in their families of origin. In addition, about 6.7 million children lost a parent or caregiver during the COVID-19 pandemic.

In particular, Romania has a history of institutionalizing children. Through decades of repressive policies from the Romanian dictator Nicolae Ceausescu, child abandonment became a national disaster. Families couldn’t afford to keep their children and were encouraged to turn them over to the state.

The current study was part of the Bucharest Early Intervention Project, initiated in 2001 to examine the impact of high-quality, family-based care on development. It included 136 Romanian children (mean age, about 22 months) who were abandoned at or shortly after birth and were placed in an institution.

Researchers randomly assigned each toddler to 1 of 56 foster families or to continue living in an institution (care as usual). The researchers had to create a foster care network, because such care was extremely limited at the start of the study.
 

Providing stimulating care

Foster parents in the study received regular support from social workers and U.S.-based psychologists. They were encouraged to “make a commitment to treat the child as if it was their own, providing sensitive, stimulating, and nurturing care, not just in the short term but for their whole life,” said Dr. Humphreys.

Foster care programs in the United States have been criticized for focusing on short-term care, she said. “It’s really just a bed to sleep on, clothes to wear, and food to eat rather than the psychological component we think is really important for child development.”

For the study, the researchers assessed the children across multiple developmental domains at baseline and at ages 30, 42, and 54 months. They conducted additional assessments when the kids were aged 8, 12, and 16-18 years.

The primary outcomes were cognitive functioning (IQ), physical growth (height, weight, head circumference), brain electrical activity (relative electroencephalography power in the alpha frequency band), and symptoms of five types of psychopathology (disinhibited social engagement disorder, reactive attachment disorder, ADHD symptoms, externalizing symptoms, and internalizing symptoms).

From over 7,000 observations analyzed across follow-ups, the investigators found that the intervention had an overall significant effect on cognitive, physical, and neural outcomes when considered collectively across waves (beta, 0.26; 95% confidence interval, 0.07-0.46; P = .012). Compared to children who received care as usual, those in foster homes had significantly higher average IQ scores (P < .001) and physical size (P = .008).

The intervention had an overall beneficial effect in regard to psychopathology. The greatest impact involved a reduction in symptoms of reactive attachment disorder (P < .001).

“There are a few forms of psychopathology that seem to almost entirely occur after severe neglect, including reactive attachment disorder; we think of these as disorders of social relatedness that derive from aberrant or insufficient early caregiving experiences,” said Dr. Humphreys. “Being placed in a family reduced the symptoms of reactive attachment disorder to pretty much nonexistent.”

To a lesser extent, the intervention reduced symptoms of disinhibited social engagement disorder. The foster care group also had significantly fewer internalizing symptoms than did children in the care-as-usual group.

But there was no significant overall effect of the intervention on symptoms of ADHD or externalizing problems.
 

Positive effects persisted

For the most part, the positive effects of the intervention on children’s functioning persisted during nearly 2 decades of follow-up. The impact of the intervention “can be described as rapidly apparent by age 30 months and sustained through late adolescence,” wrote the authors.

Regarding the impact of age at the time of placement, the study found that, compared with children placed into foster care later, those who entered foster care earlier (younger than 33 months) had significantly higher IQ scores and relative alpha power, but there was no difference in physical growth.

For some outcomes, the benefits of earlier placement were apparent in early childhood but faded by adolescence. But Dr. Humphreys noted all placements were early by most definitions.

The researchers also assessed stability of foster care placements. Children were considered “stable” if they remained with their original foster family; they were considered “disrupted” if they no longer resided with the family.

Here, the study found some “striking results,” said Dr. Humphreys. The effect of placement stability was largest in adolescence, when, overall, those who had remained with their original foster family had better cognitive and physical outcomes and less severe symptoms of psychopathology compared to those who experienced placement disruptions.

As for sex differences, “it’s a mixed bag,” said Dr. Humphreys, although overall, “we didn’t see strong evidence of sex differences” in terms of outcomes.

The investigators were unable to examine trajectories of children’s functioning, which would have provided important information on aspects such as rate of growth and the shape of growth curves. Specific features of the institutional or foster care environment in Bucharest during the study may limit the generalizability of the findings to other settings.
 

Absolutely unique project

The study examined an “absolutely unique project” and had “very exciting” results that should have “important clinical implications,” commented the American Journal of Psychiatry editor-in-chief Ned Kalin, MD, Hedberg Professor and chair, department of psychiatry, University of Wisconsin–Madison.

The findings are “pretty dramatic,” added Dr. Kalin. “This is probably the study to be thinking about when considering the future of treatment and interventions in children who have suffered from this type of neglect, which is unfortunately extremely common worldwide, including in the U.S.”

In particular, the findings regarding improved psychopathology “bode well for the future,” said Dr. Kalin. “We know these types of problems are risk factors for the later development of depression and anxiety disorders. It will be really interesting to find out, but my guess is these kids will be protected as they mature further.”

The study was supported by the NIH, the John D. and Catherine T. MacArthur Foundation, the Palix Foundation, and the Jacobs Foundation. Dr. Humphreys has received research funding from the Brain and Behavior Research Foundation, the Caplan Foundation, the Jacobs Foundation, the National Science Foundation, the NIH, the Vanderbilt Institute for Clinical and Translational Research, the Vanderbilt Kennedy Center, and Vanderbilt University; she has received honoraria from the Journal of Clinical Child and Adolescent Psychology Future Directions Forum, Learning Grove, the University of Iowa, the University of Texas at Austin, and ZERO TO THREE.

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

SAN FRANCISCO – Children randomly assigned to foster care after being institutionalized in infancy have significantly better cognitive and physical outcomes and less severe symptoms of psychopathology than their peers who remain in institutional care, results of a new study suggest.

The study shows that sustained recovery is possible after severe, early-life adversity, study author Kathryn L. Humphreys, PhD, assistant professor, department of psychology and human development, Vanderbilt University, Nashville, Tenn., said in an interview.

Vanderbilt University

“Given the strong evidence from the present study, I hope physicians will play a role in promoting family placements as an alternative to institutional care for children who have been orphaned,” she said.

The findings were presented at the annual meeting of the American Psychiatric Association and were published online in the American Journal of Psychiatry.

Millions of children around the world experience psychosocial deprivation while living in institutions, and many more are neglected in their families of origin. In addition, about 6.7 million children lost a parent or caregiver during the COVID-19 pandemic.

In particular, Romania has a history of institutionalizing children. Through decades of repressive policies from the Romanian dictator Nicolae Ceausescu, child abandonment became a national disaster. Families couldn’t afford to keep their children and were encouraged to turn them over to the state.

The current study was part of the Bucharest Early Intervention Project, initiated in 2001 to examine the impact of high-quality, family-based care on development. It included 136 Romanian children (mean age, about 22 months) who were abandoned at or shortly after birth and were placed in an institution.

Researchers randomly assigned each toddler to 1 of 56 foster families or to continue living in an institution (care as usual). The researchers had to create a foster care network, because such care was extremely limited at the start of the study.
 

Providing stimulating care

Foster parents in the study received regular support from social workers and U.S.-based psychologists. They were encouraged to “make a commitment to treat the child as if it was their own, providing sensitive, stimulating, and nurturing care, not just in the short term but for their whole life,” said Dr. Humphreys.

Foster care programs in the United States have been criticized for focusing on short-term care, she said. “It’s really just a bed to sleep on, clothes to wear, and food to eat rather than the psychological component we think is really important for child development.”

For the study, the researchers assessed the children across multiple developmental domains at baseline and at ages 30, 42, and 54 months. They conducted additional assessments when the kids were aged 8, 12, and 16-18 years.

The primary outcomes were cognitive functioning (IQ), physical growth (height, weight, head circumference), brain electrical activity (relative electroencephalography power in the alpha frequency band), and symptoms of five types of psychopathology (disinhibited social engagement disorder, reactive attachment disorder, ADHD symptoms, externalizing symptoms, and internalizing symptoms).

From over 7,000 observations analyzed across follow-ups, the investigators found that the intervention had an overall significant effect on cognitive, physical, and neural outcomes when considered collectively across waves (beta, 0.26; 95% confidence interval, 0.07-0.46; P = .012). Compared to children who received care as usual, those in foster homes had significantly higher average IQ scores (P < .001) and physical size (P = .008).

The intervention had an overall beneficial effect in regard to psychopathology. The greatest impact involved a reduction in symptoms of reactive attachment disorder (P < .001).

“There are a few forms of psychopathology that seem to almost entirely occur after severe neglect, including reactive attachment disorder; we think of these as disorders of social relatedness that derive from aberrant or insufficient early caregiving experiences,” said Dr. Humphreys. “Being placed in a family reduced the symptoms of reactive attachment disorder to pretty much nonexistent.”

To a lesser extent, the intervention reduced symptoms of disinhibited social engagement disorder. The foster care group also had significantly fewer internalizing symptoms than did children in the care-as-usual group.

But there was no significant overall effect of the intervention on symptoms of ADHD or externalizing problems.
 

Positive effects persisted

For the most part, the positive effects of the intervention on children’s functioning persisted during nearly 2 decades of follow-up. The impact of the intervention “can be described as rapidly apparent by age 30 months and sustained through late adolescence,” wrote the authors.

Regarding the impact of age at the time of placement, the study found that, compared with children placed into foster care later, those who entered foster care earlier (younger than 33 months) had significantly higher IQ scores and relative alpha power, but there was no difference in physical growth.

For some outcomes, the benefits of earlier placement were apparent in early childhood but faded by adolescence. But Dr. Humphreys noted all placements were early by most definitions.

The researchers also assessed stability of foster care placements. Children were considered “stable” if they remained with their original foster family; they were considered “disrupted” if they no longer resided with the family.

Here, the study found some “striking results,” said Dr. Humphreys. The effect of placement stability was largest in adolescence, when, overall, those who had remained with their original foster family had better cognitive and physical outcomes and less severe symptoms of psychopathology compared to those who experienced placement disruptions.

As for sex differences, “it’s a mixed bag,” said Dr. Humphreys, although overall, “we didn’t see strong evidence of sex differences” in terms of outcomes.

The investigators were unable to examine trajectories of children’s functioning, which would have provided important information on aspects such as rate of growth and the shape of growth curves. Specific features of the institutional or foster care environment in Bucharest during the study may limit the generalizability of the findings to other settings.
 

Absolutely unique project

The study examined an “absolutely unique project” and had “very exciting” results that should have “important clinical implications,” commented the American Journal of Psychiatry editor-in-chief Ned Kalin, MD, Hedberg Professor and chair, department of psychiatry, University of Wisconsin–Madison.

The findings are “pretty dramatic,” added Dr. Kalin. “This is probably the study to be thinking about when considering the future of treatment and interventions in children who have suffered from this type of neglect, which is unfortunately extremely common worldwide, including in the U.S.”

In particular, the findings regarding improved psychopathology “bode well for the future,” said Dr. Kalin. “We know these types of problems are risk factors for the later development of depression and anxiety disorders. It will be really interesting to find out, but my guess is these kids will be protected as they mature further.”

The study was supported by the NIH, the John D. and Catherine T. MacArthur Foundation, the Palix Foundation, and the Jacobs Foundation. Dr. Humphreys has received research funding from the Brain and Behavior Research Foundation, the Caplan Foundation, the Jacobs Foundation, the National Science Foundation, the NIH, the Vanderbilt Institute for Clinical and Translational Research, the Vanderbilt Kennedy Center, and Vanderbilt University; she has received honoraria from the Journal of Clinical Child and Adolescent Psychology Future Directions Forum, Learning Grove, the University of Iowa, the University of Texas at Austin, and ZERO TO THREE.

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

SAN FRANCISCO – Children randomly assigned to foster care after being institutionalized in infancy have significantly better cognitive and physical outcomes and less severe symptoms of psychopathology than their peers who remain in institutional care, results of a new study suggest.

The study shows that sustained recovery is possible after severe, early-life adversity, study author Kathryn L. Humphreys, PhD, assistant professor, department of psychology and human development, Vanderbilt University, Nashville, Tenn., said in an interview.

Vanderbilt University

“Given the strong evidence from the present study, I hope physicians will play a role in promoting family placements as an alternative to institutional care for children who have been orphaned,” she said.

The findings were presented at the annual meeting of the American Psychiatric Association and were published online in the American Journal of Psychiatry.

Millions of children around the world experience psychosocial deprivation while living in institutions, and many more are neglected in their families of origin. In addition, about 6.7 million children lost a parent or caregiver during the COVID-19 pandemic.

In particular, Romania has a history of institutionalizing children. Through decades of repressive policies from the Romanian dictator Nicolae Ceausescu, child abandonment became a national disaster. Families couldn’t afford to keep their children and were encouraged to turn them over to the state.

The current study was part of the Bucharest Early Intervention Project, initiated in 2001 to examine the impact of high-quality, family-based care on development. It included 136 Romanian children (mean age, about 22 months) who were abandoned at or shortly after birth and were placed in an institution.

Researchers randomly assigned each toddler to 1 of 56 foster families or to continue living in an institution (care as usual). The researchers had to create a foster care network, because such care was extremely limited at the start of the study.
 

Providing stimulating care

Foster parents in the study received regular support from social workers and U.S.-based psychologists. They were encouraged to “make a commitment to treat the child as if it was their own, providing sensitive, stimulating, and nurturing care, not just in the short term but for their whole life,” said Dr. Humphreys.

Foster care programs in the United States have been criticized for focusing on short-term care, she said. “It’s really just a bed to sleep on, clothes to wear, and food to eat rather than the psychological component we think is really important for child development.”

For the study, the researchers assessed the children across multiple developmental domains at baseline and at ages 30, 42, and 54 months. They conducted additional assessments when the kids were aged 8, 12, and 16-18 years.

The primary outcomes were cognitive functioning (IQ), physical growth (height, weight, head circumference), brain electrical activity (relative electroencephalography power in the alpha frequency band), and symptoms of five types of psychopathology (disinhibited social engagement disorder, reactive attachment disorder, ADHD symptoms, externalizing symptoms, and internalizing symptoms).

From over 7,000 observations analyzed across follow-ups, the investigators found that the intervention had an overall significant effect on cognitive, physical, and neural outcomes when considered collectively across waves (beta, 0.26; 95% confidence interval, 0.07-0.46; P = .012). Compared to children who received care as usual, those in foster homes had significantly higher average IQ scores (P < .001) and physical size (P = .008).

The intervention had an overall beneficial effect in regard to psychopathology. The greatest impact involved a reduction in symptoms of reactive attachment disorder (P < .001).

“There are a few forms of psychopathology that seem to almost entirely occur after severe neglect, including reactive attachment disorder; we think of these as disorders of social relatedness that derive from aberrant or insufficient early caregiving experiences,” said Dr. Humphreys. “Being placed in a family reduced the symptoms of reactive attachment disorder to pretty much nonexistent.”

To a lesser extent, the intervention reduced symptoms of disinhibited social engagement disorder. The foster care group also had significantly fewer internalizing symptoms than did children in the care-as-usual group.

But there was no significant overall effect of the intervention on symptoms of ADHD or externalizing problems.
 

Positive effects persisted

For the most part, the positive effects of the intervention on children’s functioning persisted during nearly 2 decades of follow-up. The impact of the intervention “can be described as rapidly apparent by age 30 months and sustained through late adolescence,” wrote the authors.

Regarding the impact of age at the time of placement, the study found that, compared with children placed into foster care later, those who entered foster care earlier (younger than 33 months) had significantly higher IQ scores and relative alpha power, but there was no difference in physical growth.

For some outcomes, the benefits of earlier placement were apparent in early childhood but faded by adolescence. But Dr. Humphreys noted all placements were early by most definitions.

The researchers also assessed stability of foster care placements. Children were considered “stable” if they remained with their original foster family; they were considered “disrupted” if they no longer resided with the family.

Here, the study found some “striking results,” said Dr. Humphreys. The effect of placement stability was largest in adolescence, when, overall, those who had remained with their original foster family had better cognitive and physical outcomes and less severe symptoms of psychopathology compared to those who experienced placement disruptions.

As for sex differences, “it’s a mixed bag,” said Dr. Humphreys, although overall, “we didn’t see strong evidence of sex differences” in terms of outcomes.

The investigators were unable to examine trajectories of children’s functioning, which would have provided important information on aspects such as rate of growth and the shape of growth curves. Specific features of the institutional or foster care environment in Bucharest during the study may limit the generalizability of the findings to other settings.
 

Absolutely unique project

The study examined an “absolutely unique project” and had “very exciting” results that should have “important clinical implications,” commented the American Journal of Psychiatry editor-in-chief Ned Kalin, MD, Hedberg Professor and chair, department of psychiatry, University of Wisconsin–Madison.

The findings are “pretty dramatic,” added Dr. Kalin. “This is probably the study to be thinking about when considering the future of treatment and interventions in children who have suffered from this type of neglect, which is unfortunately extremely common worldwide, including in the U.S.”

In particular, the findings regarding improved psychopathology “bode well for the future,” said Dr. Kalin. “We know these types of problems are risk factors for the later development of depression and anxiety disorders. It will be really interesting to find out, but my guess is these kids will be protected as they mature further.”

The study was supported by the NIH, the John D. and Catherine T. MacArthur Foundation, the Palix Foundation, and the Jacobs Foundation. Dr. Humphreys has received research funding from the Brain and Behavior Research Foundation, the Caplan Foundation, the Jacobs Foundation, the National Science Foundation, the NIH, the Vanderbilt Institute for Clinical and Translational Research, the Vanderbilt Kennedy Center, and Vanderbilt University; she has received honoraria from the Journal of Clinical Child and Adolescent Psychology Future Directions Forum, Learning Grove, the University of Iowa, the University of Texas at Austin, and ZERO TO THREE.

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