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Innovations in pediatric chronic pain management
At the new Walnut Creek Clinic in the East Bay of the San Francisco Bay area, kids get a “Comfort Promise.”
The clinic extends the work of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine beyond the locations in University of California San Francisco Benioff Children’s Hospitals in San Francisco and Oakland.
At Walnut Creek, clinical acupuncturists, massage therapists, and specialists in hypnosis complement advanced medical care with integrative techniques.
The “Comfort Promise” program, which is being rolled out at that clinic and other UCSF pediatric clinics through the end of 2024, is the clinicians’ pledge to do everything in their power to make tests, infusions, and vaccinations “practically pain free.”
Needle sticks, for example, can be a common source of pain and anxiety for kids. Techniques to minimize pain vary by age. Among the ways the clinicians minimize needle pain for a child 6- to 12-years-old are:
- Giving the child control options to pick which arm; and watch the injection, pause it, or stop it with a communication sign.
- Introducing memory shaping by asking the child about the experience afterward and presenting it in a positive way by praising the acts of sitting still, breathing deeply, or being brave.
- Using distractors such as asking the child to hold a favorite item from home, storytelling, coloring, singing, or using breathing exercises.
Stefan Friedrichsdorf, MD, chief of the UCSF division of pediatric pain, palliative & integrative medicine, said in a statement: “For kids with chronic pain, complex pain medications can cause more harm than benefit. Our goal is to combine exercise and physical therapy with integrative medicine and skills-based psychotherapy to help them become pain free in their everyday life.”
Bundling appointments for early impact
At Lurie Children’s Hospital of Chicago, the chronic pain treatment program bundles visits with experts in several disciplines, include social workers, psychologists, and physical therapists, in addition to the medical team, so that patients can complete a first round of visits with multiple specialists in a short period, as opposed to several months.
Natalie Weatherred, APRN-NP, CPNP-PC, a pediatric nurse practitioner in anesthesiology and the pain clinic coordinator, said in an interview that the up-front visits involve between four and eight follow-up sessions in a short period with everybody in the multidisciplinary team “to really help jump-start their pain treatment.”
She pointed out that many families come from distant parts of the state or beyond so the bundled appointments are also important for easing burden on families.
Sarah Duggan, APRN-NP, CPNP-PC, also a pediatric nurse practitioner in anesthesiology at Lurie’s, pointed out that patients at their clinic often have other chronic conditions as well, such as such as postural orthostatic tachycardia syndrome so the care integration is particularly important.
“We can get them the appropriate care that they need and the resources they need, much sooner than we would have been able to do 5 or 10 years ago,” Ms. Duggan said.
Virtual reality distraction instead of sedation
Henry Huang, MD, anesthesiologist and pain physician at Texas Children’s Hospital, Houston, said a special team there collaborates with the Chariot Program at Stanford (Calif.) University and incorporates virtual reality to distract children from pain and anxiety and harness their imaginations during induction for anesthesia, intravenous placement, and vaccinations.
“At our institution we’ve been recruiting patients to do a proof of concept to do virtual reality distraction for pain procedures, such as nerve blocks or steroid injections,” Dr. Huang said.
Traditionally, kids would have received oral or intravenous sedation to help them cope with the fear and pain.
“We’ve been successful in several cases without relying on any sedation,” he said. “The next target is to expand that to the chronic pain population.”
The distraction techniques are promising for a wide range of ages, he said, and the programming is tailored to the child’s ability to interact with the technology.
He said he is also part of a group promoting use of ultrasound instead of x-rays to guide injections to the spine and chest to reduce children’s exposure to radiation. His group is helping teach these methods to other clinicians nationally.
Dr. Huang said the most important development in chronic pediatric pain has been the growth of rehab centers that include the medical team, and practitioners from psychology as well as occupational and physical therapy.
“More and more hospitals are recognizing the importance of these pain rehab centers,” he said.
The problem, Dr. Huang said, is that these programs have always been resource intensive and involve highly specialized clinicians. The cost and the limited number of specialists make it difficult for widespread rollout.
“That’s always been the challenge from the pediatric pain world,” he said.
Recognizing the complexity of kids’ chronic pain
Angela Garcia, MD, a consulting physician for pediatric rehabilitation medicine at UPMC Children’s Hospital of Pittsburgh said
Techniques such as biofeedback and acupuncture are becoming more mainstream in pediatric chronic care, she said.
At the UPMC clinic, children and their families talk with a care team about their values and what they want to accomplish in managing the child’s pain. They ask what the pain is preventing the child from doing.
“Their goals really are our goals,” she said.
She said she also refers almost all patients to one of the center’s pain psychologists.
“Pain is biopsychosocial,” she said. “We want to make sure we’re addressing how to cope with pain.”
Dr. Garcia said she hopes nutritional therapy is one of the next approaches the clinic will incorporate, particularly surrounding how dietary changes can reduce inflammation “and heal the body from the inside out.”
She said the hospital is also looking at developing an inpatient pain program for kids whose functioning has changed so drastically that they need more intensive therapies.
Whatever the treatment approach, she said, addressing the pain early is critical.
“There is an increased risk of a child with chronic pain becoming an adult with chronic pain,” Dr. Garcia pointed out, “and that can lead to a decrease in the ability to participate in society.”
Ms. Weatherred, Ms. Duggan, Dr. Huang, and Dr. Garcia reported no relevant financial relationships.
At the new Walnut Creek Clinic in the East Bay of the San Francisco Bay area, kids get a “Comfort Promise.”
The clinic extends the work of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine beyond the locations in University of California San Francisco Benioff Children’s Hospitals in San Francisco and Oakland.
At Walnut Creek, clinical acupuncturists, massage therapists, and specialists in hypnosis complement advanced medical care with integrative techniques.
The “Comfort Promise” program, which is being rolled out at that clinic and other UCSF pediatric clinics through the end of 2024, is the clinicians’ pledge to do everything in their power to make tests, infusions, and vaccinations “practically pain free.”
Needle sticks, for example, can be a common source of pain and anxiety for kids. Techniques to minimize pain vary by age. Among the ways the clinicians minimize needle pain for a child 6- to 12-years-old are:
- Giving the child control options to pick which arm; and watch the injection, pause it, or stop it with a communication sign.
- Introducing memory shaping by asking the child about the experience afterward and presenting it in a positive way by praising the acts of sitting still, breathing deeply, or being brave.
- Using distractors such as asking the child to hold a favorite item from home, storytelling, coloring, singing, or using breathing exercises.
Stefan Friedrichsdorf, MD, chief of the UCSF division of pediatric pain, palliative & integrative medicine, said in a statement: “For kids with chronic pain, complex pain medications can cause more harm than benefit. Our goal is to combine exercise and physical therapy with integrative medicine and skills-based psychotherapy to help them become pain free in their everyday life.”
Bundling appointments for early impact
At Lurie Children’s Hospital of Chicago, the chronic pain treatment program bundles visits with experts in several disciplines, include social workers, psychologists, and physical therapists, in addition to the medical team, so that patients can complete a first round of visits with multiple specialists in a short period, as opposed to several months.
Natalie Weatherred, APRN-NP, CPNP-PC, a pediatric nurse practitioner in anesthesiology and the pain clinic coordinator, said in an interview that the up-front visits involve between four and eight follow-up sessions in a short period with everybody in the multidisciplinary team “to really help jump-start their pain treatment.”
She pointed out that many families come from distant parts of the state or beyond so the bundled appointments are also important for easing burden on families.
Sarah Duggan, APRN-NP, CPNP-PC, also a pediatric nurse practitioner in anesthesiology at Lurie’s, pointed out that patients at their clinic often have other chronic conditions as well, such as such as postural orthostatic tachycardia syndrome so the care integration is particularly important.
“We can get them the appropriate care that they need and the resources they need, much sooner than we would have been able to do 5 or 10 years ago,” Ms. Duggan said.
Virtual reality distraction instead of sedation
Henry Huang, MD, anesthesiologist and pain physician at Texas Children’s Hospital, Houston, said a special team there collaborates with the Chariot Program at Stanford (Calif.) University and incorporates virtual reality to distract children from pain and anxiety and harness their imaginations during induction for anesthesia, intravenous placement, and vaccinations.
“At our institution we’ve been recruiting patients to do a proof of concept to do virtual reality distraction for pain procedures, such as nerve blocks or steroid injections,” Dr. Huang said.
Traditionally, kids would have received oral or intravenous sedation to help them cope with the fear and pain.
“We’ve been successful in several cases without relying on any sedation,” he said. “The next target is to expand that to the chronic pain population.”
The distraction techniques are promising for a wide range of ages, he said, and the programming is tailored to the child’s ability to interact with the technology.
He said he is also part of a group promoting use of ultrasound instead of x-rays to guide injections to the spine and chest to reduce children’s exposure to radiation. His group is helping teach these methods to other clinicians nationally.
Dr. Huang said the most important development in chronic pediatric pain has been the growth of rehab centers that include the medical team, and practitioners from psychology as well as occupational and physical therapy.
“More and more hospitals are recognizing the importance of these pain rehab centers,” he said.
The problem, Dr. Huang said, is that these programs have always been resource intensive and involve highly specialized clinicians. The cost and the limited number of specialists make it difficult for widespread rollout.
“That’s always been the challenge from the pediatric pain world,” he said.
Recognizing the complexity of kids’ chronic pain
Angela Garcia, MD, a consulting physician for pediatric rehabilitation medicine at UPMC Children’s Hospital of Pittsburgh said
Techniques such as biofeedback and acupuncture are becoming more mainstream in pediatric chronic care, she said.
At the UPMC clinic, children and their families talk with a care team about their values and what they want to accomplish in managing the child’s pain. They ask what the pain is preventing the child from doing.
“Their goals really are our goals,” she said.
She said she also refers almost all patients to one of the center’s pain psychologists.
“Pain is biopsychosocial,” she said. “We want to make sure we’re addressing how to cope with pain.”
Dr. Garcia said she hopes nutritional therapy is one of the next approaches the clinic will incorporate, particularly surrounding how dietary changes can reduce inflammation “and heal the body from the inside out.”
She said the hospital is also looking at developing an inpatient pain program for kids whose functioning has changed so drastically that they need more intensive therapies.
Whatever the treatment approach, she said, addressing the pain early is critical.
“There is an increased risk of a child with chronic pain becoming an adult with chronic pain,” Dr. Garcia pointed out, “and that can lead to a decrease in the ability to participate in society.”
Ms. Weatherred, Ms. Duggan, Dr. Huang, and Dr. Garcia reported no relevant financial relationships.
At the new Walnut Creek Clinic in the East Bay of the San Francisco Bay area, kids get a “Comfort Promise.”
The clinic extends the work of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine beyond the locations in University of California San Francisco Benioff Children’s Hospitals in San Francisco and Oakland.
At Walnut Creek, clinical acupuncturists, massage therapists, and specialists in hypnosis complement advanced medical care with integrative techniques.
The “Comfort Promise” program, which is being rolled out at that clinic and other UCSF pediatric clinics through the end of 2024, is the clinicians’ pledge to do everything in their power to make tests, infusions, and vaccinations “practically pain free.”
Needle sticks, for example, can be a common source of pain and anxiety for kids. Techniques to minimize pain vary by age. Among the ways the clinicians minimize needle pain for a child 6- to 12-years-old are:
- Giving the child control options to pick which arm; and watch the injection, pause it, or stop it with a communication sign.
- Introducing memory shaping by asking the child about the experience afterward and presenting it in a positive way by praising the acts of sitting still, breathing deeply, or being brave.
- Using distractors such as asking the child to hold a favorite item from home, storytelling, coloring, singing, or using breathing exercises.
Stefan Friedrichsdorf, MD, chief of the UCSF division of pediatric pain, palliative & integrative medicine, said in a statement: “For kids with chronic pain, complex pain medications can cause more harm than benefit. Our goal is to combine exercise and physical therapy with integrative medicine and skills-based psychotherapy to help them become pain free in their everyday life.”
Bundling appointments for early impact
At Lurie Children’s Hospital of Chicago, the chronic pain treatment program bundles visits with experts in several disciplines, include social workers, psychologists, and physical therapists, in addition to the medical team, so that patients can complete a first round of visits with multiple specialists in a short period, as opposed to several months.
Natalie Weatherred, APRN-NP, CPNP-PC, a pediatric nurse practitioner in anesthesiology and the pain clinic coordinator, said in an interview that the up-front visits involve between four and eight follow-up sessions in a short period with everybody in the multidisciplinary team “to really help jump-start their pain treatment.”
She pointed out that many families come from distant parts of the state or beyond so the bundled appointments are also important for easing burden on families.
Sarah Duggan, APRN-NP, CPNP-PC, also a pediatric nurse practitioner in anesthesiology at Lurie’s, pointed out that patients at their clinic often have other chronic conditions as well, such as such as postural orthostatic tachycardia syndrome so the care integration is particularly important.
“We can get them the appropriate care that they need and the resources they need, much sooner than we would have been able to do 5 or 10 years ago,” Ms. Duggan said.
Virtual reality distraction instead of sedation
Henry Huang, MD, anesthesiologist and pain physician at Texas Children’s Hospital, Houston, said a special team there collaborates with the Chariot Program at Stanford (Calif.) University and incorporates virtual reality to distract children from pain and anxiety and harness their imaginations during induction for anesthesia, intravenous placement, and vaccinations.
“At our institution we’ve been recruiting patients to do a proof of concept to do virtual reality distraction for pain procedures, such as nerve blocks or steroid injections,” Dr. Huang said.
Traditionally, kids would have received oral or intravenous sedation to help them cope with the fear and pain.
“We’ve been successful in several cases without relying on any sedation,” he said. “The next target is to expand that to the chronic pain population.”
The distraction techniques are promising for a wide range of ages, he said, and the programming is tailored to the child’s ability to interact with the technology.
He said he is also part of a group promoting use of ultrasound instead of x-rays to guide injections to the spine and chest to reduce children’s exposure to radiation. His group is helping teach these methods to other clinicians nationally.
Dr. Huang said the most important development in chronic pediatric pain has been the growth of rehab centers that include the medical team, and practitioners from psychology as well as occupational and physical therapy.
“More and more hospitals are recognizing the importance of these pain rehab centers,” he said.
The problem, Dr. Huang said, is that these programs have always been resource intensive and involve highly specialized clinicians. The cost and the limited number of specialists make it difficult for widespread rollout.
“That’s always been the challenge from the pediatric pain world,” he said.
Recognizing the complexity of kids’ chronic pain
Angela Garcia, MD, a consulting physician for pediatric rehabilitation medicine at UPMC Children’s Hospital of Pittsburgh said
Techniques such as biofeedback and acupuncture are becoming more mainstream in pediatric chronic care, she said.
At the UPMC clinic, children and their families talk with a care team about their values and what they want to accomplish in managing the child’s pain. They ask what the pain is preventing the child from doing.
“Their goals really are our goals,” she said.
She said she also refers almost all patients to one of the center’s pain psychologists.
“Pain is biopsychosocial,” she said. “We want to make sure we’re addressing how to cope with pain.”
Dr. Garcia said she hopes nutritional therapy is one of the next approaches the clinic will incorporate, particularly surrounding how dietary changes can reduce inflammation “and heal the body from the inside out.”
She said the hospital is also looking at developing an inpatient pain program for kids whose functioning has changed so drastically that they need more intensive therapies.
Whatever the treatment approach, she said, addressing the pain early is critical.
“There is an increased risk of a child with chronic pain becoming an adult with chronic pain,” Dr. Garcia pointed out, “and that can lead to a decrease in the ability to participate in society.”
Ms. Weatherred, Ms. Duggan, Dr. Huang, and Dr. Garcia reported no relevant financial relationships.
PAD procedure overuse: A field in peril or ‘a few bad apples’?
On May 24, the news outlet ProPublica published a scathing investigation of Jeffery Dormu, DO, said to have performed hundreds of “medically unnecessary and invasive vascular procedures” in his Laurel, Md. office, putting patients’ limbs and lives at risk.
On July 15, The New York Times published a broader-based investigation of several vascular specialists said to have performed “risky” procedures on patients with peripheral artery disease (PAD) who subsequently had to have amputations, or died. The focus was mainly on Michigan-based interventional cardiologist Jihad Mustapha, MD.
This follows a 2019 analysis of Medicare claims data that identified outlier physicians with a high early intervention rate for patients newly diagnosed with claudication. According to the American Heart Association statistics, PAD affects approximately 8.5 million U.S. adults age 40 and older (some claim that’s an underestimate); most cases don’t require invasive treatment.
Responding to the Times’ revelations, Joseph L. Mills, MD, president of the Society for Vascular Surgery, wrote on the society’s website: “The overwhelming majority of vascular surgeons, and a vast majority of other specialists that receive some training and play a role in the care of vascular patients, including those trained in vascular medicine, interventional cardiology, and interventional radiology are providing high-quality, evidence-based care with safety and the best patient outcomes in mind.
“This is a complex issue that requires the examination not only of the events detailed in this story ... but of the underlying health care economic, legal and regulatory policies that created fertile soil for this behavior to germinate and take root.”
‘A few bad apples’
“I think it’s a case of a few bad apples,” Sunil V. Rao, MD, director of interventional cardiology at NYU Langone Health, New York, said in an interview. “In general, I think physicians who take care of patients with vascular issues are trying to do the right thing. I think all of us who take care of patients with vascular disease see patients who are very, very complex, and there are going to be some procedures that have complications.
“Without knowing the clinical details, it’s hard to know whether the procedures described in the articles were overuse or unnecessary, or exactly what led to the amputations,” he said. “All we know is that these physicians are outliers in terms of the number of procedures they were billing for.
“But although correlation is not causation, it certainly is cause for concern because you would expect that the use of procedures for specific indications would fall within a certain range,” he added.
Lifestyle changes first
PAD is often asymptomatic or mild, making it difficult to diagnose. Revascularization procedures usually are reserved for the 5%-8% of patients at risk for chronic limb-threatening ischemia (CLTI) or those in whom the cornerstones of PAD treatment – lifestyle changes and, if needed, medication – fail.
Revascularization options include balloon angioplasty or stent placement; atherectomy to remove plaques from the artery; or bypass surgery if a long portion of a leg artery is completely blocked. All carry a risk of long-term adverse outcomes, but the rates are highest for atherectomy.
Lifestyle changes include regular exercise, following a healthy diet, quitting smoking, and controlling diabetes and high blood pressure. When PAD continues or progresses despite these modifications, medications such as antiplatelet agents, antihypertensives, and/or lipid-lowering drugs may be prescribed.
‘Medically unnecessary’
According to the latest American Heart Association/American College of Cardiology guideline on managing patients with lower-extremity PAD, patients should be selected for revascularization based on symptom severity.
Factors to consider include a significant disability as assessed by the patient, and adequacy of response to medical and structured exercise therapy.
There’s the rub regarding the clinicians investigated in the Times and ProPublica. Many patients, apparently, were not encouraged to make lifestyle changes, nor did they receive medication. Instead, they were advised from the get-go to undergo invasive procedures, and often multiple times. Underuse of prevention and lifestyle counseling n the management of PAD has long been a concern.
Furthermore, in at least some cases, patients without any symptoms were encouraged to be screened for blockages that were then treated invasively, according to the Times.
Dr. Dormu, as highlighted in ProPublica, positioned his practice as “life and limb saving.” Yet, in investigative findings that led to a suspension of Dr. Dormu’s license to practice medicine in Maryland, peer reviewers expressed concern regarding his repeated use of invasive and medically unnecessary procedures, exposing patients to “potential risks such as bleeding, infection, blood vessel injuries which could acutely or chronically worsen the patient’s circulation, and limb loss.”
The peer reviewers concurred that Dr. Dormu failed to use conservative management techniques to address the patients’ vascular complaints before resorting to invasive procedures.
Dr. Mustapha is described in the Times as a “high-volume” atherectomy provider. From 2017 to 2021, about half of Medicare’s atherectomy payments – $1.4 billion – went to 200 high-volume providers, with Dr. Mustapha near the top of the list.
Some of Dr. Mustapha’s patients underwent multiple procedures said to help prevent leg amputation, but their legs were amputated anyway, possibly because of the multiple atherectomies, according to the Times.
Judith Lin, MD, MBA, who treated some of Dr. Mustapha’s former patients, was among those who complained about his practice to Michigan’s licensing board. Some of the patients she treated needed amputations; others needed to have leftover wires extracted from their legs.
In 2020, the board investigated Dr. Lin’s complaint and referred it to Michigan’s attorney general, who brought a disciplinary action against Dr. Mustapha. An expert hired by the state to review eight patient cases concluded that Dr. Mustapha’s practice “was characterized by overtreatment and poor documentation.” In some cases, the expert wrote, “unnecessary procedures hastened amputations.”
The statement issued by Dr. Mills, the president of SVS, noted that the society’s practice guideline proposes a threshold of at least 2 years of likely durability for an intervention performed for claudication.
“The growing frequency of multiple, repeated procedures [is] emblematic of poor patient selection and inadequate durability of the chosen procedure, leading to a vicious cycle of repetitive interventions that is not only costly, but also dangerous,” he wrote.
Financial incentives to blame?
In 2008, Medicare created incentives for physicians to perform vascular procedures in offices rather than hospitals, in an effort to reduce medical costs, according to both investigative articles. But the effort backfired.
Before the changes, an office provider inserting a stent could make about $1,700 from Medicare; deploying a balloon could bring in roughly $3,800. By 2011, the payments rose to about $6,400 and $4,800, respectively.
Office-based atherectomies soared when, in 2011, the Centers for Medicare & Medicaid Services started reimbursing $13,500 per procedure, as opposed to roughly $11,450 in a hospital. Atherectomies increased by 60% from 2011 to 2014, and Medicare’s overall costs for peripheral vascular treatments climbed by nearly half a billion dollars.
“The government is really to blame for setting these tremendously high reimbursement values without looking into whether these procedures are helping people or are just worthless procedures or, in fact, are hurting people,” Dipankar Mukherjee, MD, a vascular surgeon and chief of vascular surgery at Inova Fairfax (Va.) Hospital, said in ProPublica.
The result, noted Dr. Rao, is that “there can be perverse or nefarious incentives for doing these procedures. People are incentivized by reimbursement to do something that really falls in the area of clinical judgment and guidelines.”
Major incentives also come from device manufacturers, who often reward physicians who do the most vascular procedures with payments for consulting and other services, according to the Times. In addition, these companies lend money to help physicians or their clinics to finance the purchase of equipment used to perform the procedures.
“Vascular medicine now is the frontier of the Wild West,” Marty Makary, MD, MPH, a professor of surgery and health care quality researcher at Johns Hopkins University, Baltimore, told ProPublica. “People are flying blind walking into the clinics of these doctors with egregious practice patterns, and we know that their pattern is indefensible.”
Recognizing that the situation posed a threat to patients and also damaged the credibility of his specialty, Kim J. Hodgson, MD, a former SVS president, told attendees at the 2021 annual meeting of the SVS, “Somebody has to address what should never have been allowed to get to this level of threat to us and our patients in the first place. We can play whack-a-mole every time the bad actors surface until the cows come home, but that leaves a trail of harmed patients and wasted resources.”
Dr. Hodgson described atherectomy as “a procedure that many believe provides no demonstrable value whatsoever to the patient” and challenged those who disagree to prove it.
Multidisciplinary teams needed
Other experts believe there are times that revascularization procedures, including atherectomy, are appropriate. However, the majority of patients with PAD do not require a procedure, Soo Hyun (Esther) Kim, MD, MPH, director of the Center for Women’s Cardiovascular Health at Atrium Health Sanger Heart and Vascular Institute in Charlotte, N.C., said in an interview. In fact, “many patients do not even know they have leg artery blockages.”
Invasive procedures may well be appropriate for patients with severe PAD, especially those with CLTI, and disparities may be keeping those who truly need such interventions – or for whom they may be at least considered – from accessing them. If PAD is not diagnosed and treated in a timely way, Dr. Kim said, those individuals “do indeed lose their limbs.”
Multidisciplinary teams can help, Dr. Kim said. “Specialists from multiple different training backgrounds [can] take good care of patients with PAD,” she said. This is important when access to a particular type of specialist is limited, and because patients with PAD often have complex medical problems that can benefit from a team approach.
Transcatheter aortic valve replacement heart teams and complex coronary disease heart teams are two examples, Dr. Kim noted. “When a high-stakes procedure is being considered, the patient’s case is reviewed by multiple stakeholders to ensure appropriateness of the procedure and collaboratively evaluate risk.”
Dr. Rao also emphasized a team approach. “PAD does not belong to a single specialty,” he said. The revelations from the Times, ProPublica, and other sources “point to the fact that we all – cardiologists, vascular surgeons, interventional radiologists – should start thinking about how best to police ourselves and also account for the variation in clinical judgment.”
Use of a multidisciplinary team is a “guideline-recommended approach” for coronary artery revascularization, he said, “I think the same should apply for PAD.”
PAD is a sign of systemic atherosclerosis, Dr. Kim noted. “The treatment of PAD includes addressing leg pain and wounds with procedures, but the interventions that will keep people alive are the medications we use to prevent heart attack and stroke. Patients with PAD need to understand that treatment is much more than opening up a blockage in the leg.”
Dr. Rao and Dr. Kim disclosed no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
On May 24, the news outlet ProPublica published a scathing investigation of Jeffery Dormu, DO, said to have performed hundreds of “medically unnecessary and invasive vascular procedures” in his Laurel, Md. office, putting patients’ limbs and lives at risk.
On July 15, The New York Times published a broader-based investigation of several vascular specialists said to have performed “risky” procedures on patients with peripheral artery disease (PAD) who subsequently had to have amputations, or died. The focus was mainly on Michigan-based interventional cardiologist Jihad Mustapha, MD.
This follows a 2019 analysis of Medicare claims data that identified outlier physicians with a high early intervention rate for patients newly diagnosed with claudication. According to the American Heart Association statistics, PAD affects approximately 8.5 million U.S. adults age 40 and older (some claim that’s an underestimate); most cases don’t require invasive treatment.
Responding to the Times’ revelations, Joseph L. Mills, MD, president of the Society for Vascular Surgery, wrote on the society’s website: “The overwhelming majority of vascular surgeons, and a vast majority of other specialists that receive some training and play a role in the care of vascular patients, including those trained in vascular medicine, interventional cardiology, and interventional radiology are providing high-quality, evidence-based care with safety and the best patient outcomes in mind.
“This is a complex issue that requires the examination not only of the events detailed in this story ... but of the underlying health care economic, legal and regulatory policies that created fertile soil for this behavior to germinate and take root.”
‘A few bad apples’
“I think it’s a case of a few bad apples,” Sunil V. Rao, MD, director of interventional cardiology at NYU Langone Health, New York, said in an interview. “In general, I think physicians who take care of patients with vascular issues are trying to do the right thing. I think all of us who take care of patients with vascular disease see patients who are very, very complex, and there are going to be some procedures that have complications.
“Without knowing the clinical details, it’s hard to know whether the procedures described in the articles were overuse or unnecessary, or exactly what led to the amputations,” he said. “All we know is that these physicians are outliers in terms of the number of procedures they were billing for.
“But although correlation is not causation, it certainly is cause for concern because you would expect that the use of procedures for specific indications would fall within a certain range,” he added.
Lifestyle changes first
PAD is often asymptomatic or mild, making it difficult to diagnose. Revascularization procedures usually are reserved for the 5%-8% of patients at risk for chronic limb-threatening ischemia (CLTI) or those in whom the cornerstones of PAD treatment – lifestyle changes and, if needed, medication – fail.
Revascularization options include balloon angioplasty or stent placement; atherectomy to remove plaques from the artery; or bypass surgery if a long portion of a leg artery is completely blocked. All carry a risk of long-term adverse outcomes, but the rates are highest for atherectomy.
Lifestyle changes include regular exercise, following a healthy diet, quitting smoking, and controlling diabetes and high blood pressure. When PAD continues or progresses despite these modifications, medications such as antiplatelet agents, antihypertensives, and/or lipid-lowering drugs may be prescribed.
‘Medically unnecessary’
According to the latest American Heart Association/American College of Cardiology guideline on managing patients with lower-extremity PAD, patients should be selected for revascularization based on symptom severity.
Factors to consider include a significant disability as assessed by the patient, and adequacy of response to medical and structured exercise therapy.
There’s the rub regarding the clinicians investigated in the Times and ProPublica. Many patients, apparently, were not encouraged to make lifestyle changes, nor did they receive medication. Instead, they were advised from the get-go to undergo invasive procedures, and often multiple times. Underuse of prevention and lifestyle counseling n the management of PAD has long been a concern.
Furthermore, in at least some cases, patients without any symptoms were encouraged to be screened for blockages that were then treated invasively, according to the Times.
Dr. Dormu, as highlighted in ProPublica, positioned his practice as “life and limb saving.” Yet, in investigative findings that led to a suspension of Dr. Dormu’s license to practice medicine in Maryland, peer reviewers expressed concern regarding his repeated use of invasive and medically unnecessary procedures, exposing patients to “potential risks such as bleeding, infection, blood vessel injuries which could acutely or chronically worsen the patient’s circulation, and limb loss.”
The peer reviewers concurred that Dr. Dormu failed to use conservative management techniques to address the patients’ vascular complaints before resorting to invasive procedures.
Dr. Mustapha is described in the Times as a “high-volume” atherectomy provider. From 2017 to 2021, about half of Medicare’s atherectomy payments – $1.4 billion – went to 200 high-volume providers, with Dr. Mustapha near the top of the list.
Some of Dr. Mustapha’s patients underwent multiple procedures said to help prevent leg amputation, but their legs were amputated anyway, possibly because of the multiple atherectomies, according to the Times.
Judith Lin, MD, MBA, who treated some of Dr. Mustapha’s former patients, was among those who complained about his practice to Michigan’s licensing board. Some of the patients she treated needed amputations; others needed to have leftover wires extracted from their legs.
In 2020, the board investigated Dr. Lin’s complaint and referred it to Michigan’s attorney general, who brought a disciplinary action against Dr. Mustapha. An expert hired by the state to review eight patient cases concluded that Dr. Mustapha’s practice “was characterized by overtreatment and poor documentation.” In some cases, the expert wrote, “unnecessary procedures hastened amputations.”
The statement issued by Dr. Mills, the president of SVS, noted that the society’s practice guideline proposes a threshold of at least 2 years of likely durability for an intervention performed for claudication.
“The growing frequency of multiple, repeated procedures [is] emblematic of poor patient selection and inadequate durability of the chosen procedure, leading to a vicious cycle of repetitive interventions that is not only costly, but also dangerous,” he wrote.
Financial incentives to blame?
In 2008, Medicare created incentives for physicians to perform vascular procedures in offices rather than hospitals, in an effort to reduce medical costs, according to both investigative articles. But the effort backfired.
Before the changes, an office provider inserting a stent could make about $1,700 from Medicare; deploying a balloon could bring in roughly $3,800. By 2011, the payments rose to about $6,400 and $4,800, respectively.
Office-based atherectomies soared when, in 2011, the Centers for Medicare & Medicaid Services started reimbursing $13,500 per procedure, as opposed to roughly $11,450 in a hospital. Atherectomies increased by 60% from 2011 to 2014, and Medicare’s overall costs for peripheral vascular treatments climbed by nearly half a billion dollars.
“The government is really to blame for setting these tremendously high reimbursement values without looking into whether these procedures are helping people or are just worthless procedures or, in fact, are hurting people,” Dipankar Mukherjee, MD, a vascular surgeon and chief of vascular surgery at Inova Fairfax (Va.) Hospital, said in ProPublica.
The result, noted Dr. Rao, is that “there can be perverse or nefarious incentives for doing these procedures. People are incentivized by reimbursement to do something that really falls in the area of clinical judgment and guidelines.”
Major incentives also come from device manufacturers, who often reward physicians who do the most vascular procedures with payments for consulting and other services, according to the Times. In addition, these companies lend money to help physicians or their clinics to finance the purchase of equipment used to perform the procedures.
“Vascular medicine now is the frontier of the Wild West,” Marty Makary, MD, MPH, a professor of surgery and health care quality researcher at Johns Hopkins University, Baltimore, told ProPublica. “People are flying blind walking into the clinics of these doctors with egregious practice patterns, and we know that their pattern is indefensible.”
Recognizing that the situation posed a threat to patients and also damaged the credibility of his specialty, Kim J. Hodgson, MD, a former SVS president, told attendees at the 2021 annual meeting of the SVS, “Somebody has to address what should never have been allowed to get to this level of threat to us and our patients in the first place. We can play whack-a-mole every time the bad actors surface until the cows come home, but that leaves a trail of harmed patients and wasted resources.”
Dr. Hodgson described atherectomy as “a procedure that many believe provides no demonstrable value whatsoever to the patient” and challenged those who disagree to prove it.
Multidisciplinary teams needed
Other experts believe there are times that revascularization procedures, including atherectomy, are appropriate. However, the majority of patients with PAD do not require a procedure, Soo Hyun (Esther) Kim, MD, MPH, director of the Center for Women’s Cardiovascular Health at Atrium Health Sanger Heart and Vascular Institute in Charlotte, N.C., said in an interview. In fact, “many patients do not even know they have leg artery blockages.”
Invasive procedures may well be appropriate for patients with severe PAD, especially those with CLTI, and disparities may be keeping those who truly need such interventions – or for whom they may be at least considered – from accessing them. If PAD is not diagnosed and treated in a timely way, Dr. Kim said, those individuals “do indeed lose their limbs.”
Multidisciplinary teams can help, Dr. Kim said. “Specialists from multiple different training backgrounds [can] take good care of patients with PAD,” she said. This is important when access to a particular type of specialist is limited, and because patients with PAD often have complex medical problems that can benefit from a team approach.
Transcatheter aortic valve replacement heart teams and complex coronary disease heart teams are two examples, Dr. Kim noted. “When a high-stakes procedure is being considered, the patient’s case is reviewed by multiple stakeholders to ensure appropriateness of the procedure and collaboratively evaluate risk.”
Dr. Rao also emphasized a team approach. “PAD does not belong to a single specialty,” he said. The revelations from the Times, ProPublica, and other sources “point to the fact that we all – cardiologists, vascular surgeons, interventional radiologists – should start thinking about how best to police ourselves and also account for the variation in clinical judgment.”
Use of a multidisciplinary team is a “guideline-recommended approach” for coronary artery revascularization, he said, “I think the same should apply for PAD.”
PAD is a sign of systemic atherosclerosis, Dr. Kim noted. “The treatment of PAD includes addressing leg pain and wounds with procedures, but the interventions that will keep people alive are the medications we use to prevent heart attack and stroke. Patients with PAD need to understand that treatment is much more than opening up a blockage in the leg.”
Dr. Rao and Dr. Kim disclosed no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
On May 24, the news outlet ProPublica published a scathing investigation of Jeffery Dormu, DO, said to have performed hundreds of “medically unnecessary and invasive vascular procedures” in his Laurel, Md. office, putting patients’ limbs and lives at risk.
On July 15, The New York Times published a broader-based investigation of several vascular specialists said to have performed “risky” procedures on patients with peripheral artery disease (PAD) who subsequently had to have amputations, or died. The focus was mainly on Michigan-based interventional cardiologist Jihad Mustapha, MD.
This follows a 2019 analysis of Medicare claims data that identified outlier physicians with a high early intervention rate for patients newly diagnosed with claudication. According to the American Heart Association statistics, PAD affects approximately 8.5 million U.S. adults age 40 and older (some claim that’s an underestimate); most cases don’t require invasive treatment.
Responding to the Times’ revelations, Joseph L. Mills, MD, president of the Society for Vascular Surgery, wrote on the society’s website: “The overwhelming majority of vascular surgeons, and a vast majority of other specialists that receive some training and play a role in the care of vascular patients, including those trained in vascular medicine, interventional cardiology, and interventional radiology are providing high-quality, evidence-based care with safety and the best patient outcomes in mind.
“This is a complex issue that requires the examination not only of the events detailed in this story ... but of the underlying health care economic, legal and regulatory policies that created fertile soil for this behavior to germinate and take root.”
‘A few bad apples’
“I think it’s a case of a few bad apples,” Sunil V. Rao, MD, director of interventional cardiology at NYU Langone Health, New York, said in an interview. “In general, I think physicians who take care of patients with vascular issues are trying to do the right thing. I think all of us who take care of patients with vascular disease see patients who are very, very complex, and there are going to be some procedures that have complications.
“Without knowing the clinical details, it’s hard to know whether the procedures described in the articles were overuse or unnecessary, or exactly what led to the amputations,” he said. “All we know is that these physicians are outliers in terms of the number of procedures they were billing for.
“But although correlation is not causation, it certainly is cause for concern because you would expect that the use of procedures for specific indications would fall within a certain range,” he added.
Lifestyle changes first
PAD is often asymptomatic or mild, making it difficult to diagnose. Revascularization procedures usually are reserved for the 5%-8% of patients at risk for chronic limb-threatening ischemia (CLTI) or those in whom the cornerstones of PAD treatment – lifestyle changes and, if needed, medication – fail.
Revascularization options include balloon angioplasty or stent placement; atherectomy to remove plaques from the artery; or bypass surgery if a long portion of a leg artery is completely blocked. All carry a risk of long-term adverse outcomes, but the rates are highest for atherectomy.
Lifestyle changes include regular exercise, following a healthy diet, quitting smoking, and controlling diabetes and high blood pressure. When PAD continues or progresses despite these modifications, medications such as antiplatelet agents, antihypertensives, and/or lipid-lowering drugs may be prescribed.
‘Medically unnecessary’
According to the latest American Heart Association/American College of Cardiology guideline on managing patients with lower-extremity PAD, patients should be selected for revascularization based on symptom severity.
Factors to consider include a significant disability as assessed by the patient, and adequacy of response to medical and structured exercise therapy.
There’s the rub regarding the clinicians investigated in the Times and ProPublica. Many patients, apparently, were not encouraged to make lifestyle changes, nor did they receive medication. Instead, they were advised from the get-go to undergo invasive procedures, and often multiple times. Underuse of prevention and lifestyle counseling n the management of PAD has long been a concern.
Furthermore, in at least some cases, patients without any symptoms were encouraged to be screened for blockages that were then treated invasively, according to the Times.
Dr. Dormu, as highlighted in ProPublica, positioned his practice as “life and limb saving.” Yet, in investigative findings that led to a suspension of Dr. Dormu’s license to practice medicine in Maryland, peer reviewers expressed concern regarding his repeated use of invasive and medically unnecessary procedures, exposing patients to “potential risks such as bleeding, infection, blood vessel injuries which could acutely or chronically worsen the patient’s circulation, and limb loss.”
The peer reviewers concurred that Dr. Dormu failed to use conservative management techniques to address the patients’ vascular complaints before resorting to invasive procedures.
Dr. Mustapha is described in the Times as a “high-volume” atherectomy provider. From 2017 to 2021, about half of Medicare’s atherectomy payments – $1.4 billion – went to 200 high-volume providers, with Dr. Mustapha near the top of the list.
Some of Dr. Mustapha’s patients underwent multiple procedures said to help prevent leg amputation, but their legs were amputated anyway, possibly because of the multiple atherectomies, according to the Times.
Judith Lin, MD, MBA, who treated some of Dr. Mustapha’s former patients, was among those who complained about his practice to Michigan’s licensing board. Some of the patients she treated needed amputations; others needed to have leftover wires extracted from their legs.
In 2020, the board investigated Dr. Lin’s complaint and referred it to Michigan’s attorney general, who brought a disciplinary action against Dr. Mustapha. An expert hired by the state to review eight patient cases concluded that Dr. Mustapha’s practice “was characterized by overtreatment and poor documentation.” In some cases, the expert wrote, “unnecessary procedures hastened amputations.”
The statement issued by Dr. Mills, the president of SVS, noted that the society’s practice guideline proposes a threshold of at least 2 years of likely durability for an intervention performed for claudication.
“The growing frequency of multiple, repeated procedures [is] emblematic of poor patient selection and inadequate durability of the chosen procedure, leading to a vicious cycle of repetitive interventions that is not only costly, but also dangerous,” he wrote.
Financial incentives to blame?
In 2008, Medicare created incentives for physicians to perform vascular procedures in offices rather than hospitals, in an effort to reduce medical costs, according to both investigative articles. But the effort backfired.
Before the changes, an office provider inserting a stent could make about $1,700 from Medicare; deploying a balloon could bring in roughly $3,800. By 2011, the payments rose to about $6,400 and $4,800, respectively.
Office-based atherectomies soared when, in 2011, the Centers for Medicare & Medicaid Services started reimbursing $13,500 per procedure, as opposed to roughly $11,450 in a hospital. Atherectomies increased by 60% from 2011 to 2014, and Medicare’s overall costs for peripheral vascular treatments climbed by nearly half a billion dollars.
“The government is really to blame for setting these tremendously high reimbursement values without looking into whether these procedures are helping people or are just worthless procedures or, in fact, are hurting people,” Dipankar Mukherjee, MD, a vascular surgeon and chief of vascular surgery at Inova Fairfax (Va.) Hospital, said in ProPublica.
The result, noted Dr. Rao, is that “there can be perverse or nefarious incentives for doing these procedures. People are incentivized by reimbursement to do something that really falls in the area of clinical judgment and guidelines.”
Major incentives also come from device manufacturers, who often reward physicians who do the most vascular procedures with payments for consulting and other services, according to the Times. In addition, these companies lend money to help physicians or their clinics to finance the purchase of equipment used to perform the procedures.
“Vascular medicine now is the frontier of the Wild West,” Marty Makary, MD, MPH, a professor of surgery and health care quality researcher at Johns Hopkins University, Baltimore, told ProPublica. “People are flying blind walking into the clinics of these doctors with egregious practice patterns, and we know that their pattern is indefensible.”
Recognizing that the situation posed a threat to patients and also damaged the credibility of his specialty, Kim J. Hodgson, MD, a former SVS president, told attendees at the 2021 annual meeting of the SVS, “Somebody has to address what should never have been allowed to get to this level of threat to us and our patients in the first place. We can play whack-a-mole every time the bad actors surface until the cows come home, but that leaves a trail of harmed patients and wasted resources.”
Dr. Hodgson described atherectomy as “a procedure that many believe provides no demonstrable value whatsoever to the patient” and challenged those who disagree to prove it.
Multidisciplinary teams needed
Other experts believe there are times that revascularization procedures, including atherectomy, are appropriate. However, the majority of patients with PAD do not require a procedure, Soo Hyun (Esther) Kim, MD, MPH, director of the Center for Women’s Cardiovascular Health at Atrium Health Sanger Heart and Vascular Institute in Charlotte, N.C., said in an interview. In fact, “many patients do not even know they have leg artery blockages.”
Invasive procedures may well be appropriate for patients with severe PAD, especially those with CLTI, and disparities may be keeping those who truly need such interventions – or for whom they may be at least considered – from accessing them. If PAD is not diagnosed and treated in a timely way, Dr. Kim said, those individuals “do indeed lose their limbs.”
Multidisciplinary teams can help, Dr. Kim said. “Specialists from multiple different training backgrounds [can] take good care of patients with PAD,” she said. This is important when access to a particular type of specialist is limited, and because patients with PAD often have complex medical problems that can benefit from a team approach.
Transcatheter aortic valve replacement heart teams and complex coronary disease heart teams are two examples, Dr. Kim noted. “When a high-stakes procedure is being considered, the patient’s case is reviewed by multiple stakeholders to ensure appropriateness of the procedure and collaboratively evaluate risk.”
Dr. Rao also emphasized a team approach. “PAD does not belong to a single specialty,” he said. The revelations from the Times, ProPublica, and other sources “point to the fact that we all – cardiologists, vascular surgeons, interventional radiologists – should start thinking about how best to police ourselves and also account for the variation in clinical judgment.”
Use of a multidisciplinary team is a “guideline-recommended approach” for coronary artery revascularization, he said, “I think the same should apply for PAD.”
PAD is a sign of systemic atherosclerosis, Dr. Kim noted. “The treatment of PAD includes addressing leg pain and wounds with procedures, but the interventions that will keep people alive are the medications we use to prevent heart attack and stroke. Patients with PAD need to understand that treatment is much more than opening up a blockage in the leg.”
Dr. Rao and Dr. Kim disclosed no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
The fertile future of fertility technology
Fifth pregnancy, first baby.
After four pregnancies resulted in losses – and doing things as natural as possible and leaving it up to the birds, bees, and fate – my husband and I decided to explore in vitro fertilization (IVF).
Drugs to direct my follicles to produce more eggs, an egg retrieval procedure, genetic testing of our embryos, a quick procedure to remove a residual uterine septum from my uterus, drugs to thicken my endometrial lining to prepare my body to receive an embryo, an embryo transfer, steroids to suppress my immune system so my body would accept the pregnancy, blood thinner shots to promote blood flow to the baby, and 10 weeks of progesterone in oil shots later and we’re days away from welcoming our first baby into our lives.
In short, there’s more than one way to define “miracle baby.”
Global estimates say 48 million couples and 186 million individuals struggle with infertility. On average, 2 million infants born in the United States each year are conceived through assisted reproductive technology and the demand for treatments like IVF have doubled in the last decade.
Now the need for treatments outweighs clinician availability. “We have about 1,250 practicing fertility physicians in the U.S. to serve the whole country, which is highly inadequate,” said Eduardo Hariton, MD, a reproductive endocrinology physician in San Francisco and managing director of the U.S. Fertility Innovation Fund. “We have people that want to get care waiting 1 to 3 months to be seen.”
Dr. Hariton explains that U.S. IVF clinics are performing around 250,000 to 300,000 IVF cycles per year and need to be doing a million-plus to meet demand. This, plus the cost of fertility treatments – an average IVF cycle runs $23,500 and the majority of patients need multiple cycles to conceive – keeps the barrier to entry high.
Enter technology: New advances are on the way to help the assisted fertility process to run smoother and be less costly. “The field is really coming into an age of great progress and innovation,” added S. Zev Williams, MD, PhD, chief of the division of reproductive endocrinology and Infertility at Columbia University Irving Medical Center, New York City.
I’m personally grateful that such technology exists.
AI will help, of course
Fertility treatments involve endless analysis, diagnosis, and recommendations – dozens if not hundreds of decisions from each physician for each patient. Human action and reaction can affect this process, Dr. Hariton explained.
For example, if he hyperstimulated a woman during the follicle growing stage of her egg retrieval and ended up with eggs too large to retrieve, Dr. Hariton said he may subconsciously be more inclined to be extra cautious with his patients the week after, and vice versa.
This is where AI can help. “Rather than me making decisions from a couple of thousands of cycles of experience, I get to leverage hundreds of thousands of cycles from different providers over different people,” said Dr. Hariton. “I get to use all the data from that patient today – her age, her weight, what happened last cycle, how she’s doing – and make a very objective decision about the optimal time to give that woman or that couple the best outcome possible.”
AI can also assist with tasks like embryo grading. “Once our embryos are made in the lab, we usually have an embryologist looking at those embryos, grading them on a three-variable scale, and then picking the nicest one for transfer,” said Dr. Hariton. Machine learning computer vision software can help doctors select the best embryo.
Many of these AI products are in trials in the United States and some AI-based technology is already being used in fertility labs, especially in other countries. “ALife recently launched a suite of products to help with their decisions during stimulation that can help with the quality KPIs [key performance indicators] in the lab,” said Dr. Hariton. “There’s also a company that does AI-based predictions of success to give patients a better estimate called Univfy.” More AI products are still in development or awaiting Food and Drug Administration clearance.
Robots lend a hand
Like artificial intelligence, robots can be a big help in the IVF lab. Columbia University Fertility Center recently became the first to use an articulated (ART) robot to handle precise and highly repetitive work.
“IVF, from the initial point, involves creating these special plates where embryos can grow, and you do that by making little droplets,” said Dr. Williams. “It’s very time-consuming to create tons of these little droplets for the embryos to grow.” Thus, the lab created a robot to help squirt drops of the media substance required to sustain embryos in a way that is 10 times more precise than that of a trained embryologist.
“It’s a win-win because you allow the robots to do things better than a human can and this allows the humans to do things that a robot just can’t do,” explained Dr. Williams. He and his team began using this technology in the beginning of November 2022.
Dr. Williams sees ART robots being used in many more parts of the fertility treatment journey along the way, like preparing eggs after they are retrieved and performing intracytoplasmic sperm injection (ICSI), with the robot injecting the sperm into the egg.
Launching with the plate making, said Dr. Williams, is a low stakes entry point for robotic technology in the lab. “It allows us to introduce robotics to automate and optimize each step along the way, but to do so in the safest possible way.”
Dr. Williams estimates that robots will have their hands on actual eggs and sperm in 5 years.
Updates in genetic testing
Currently, if a couple wants to have their embryos genetically tested, also known as preimplantation genetic testing, each embryo must be frozen, then a biopsy of that embryo is performed and sent to the lab.
“It takes time to get the results,” said Dr. Williams. “The whole time you’re waiting, you don’t know if you’re going to have any embryos that are transferable or if next month you’re going to have to do another IVF cycle.”
Columbia researchers recently developed a new in-house test that can determine if a fetus or embryo has the right number of chromosomes. This STORK (Short-read Transpore Rapid Karyotyping) can be performed without freezing embryos and sending them out, which Dr. Williams said can save couples money and time, as they won’t necessarily need to do a separate embryo transfer cycle and can transfer an embryo in the same cycle. “You can test in the morning and transfer in the afternoon,” said Dr. Williams.
The test is currently awaiting approval and will first be used to test miscarriage samples to see if embryos were genetically normal or not, which he said should cost around $200 vs. the $2,000 to $4,000 it can cost to have fetal tissue sent to the lab – and insurance doesn’t cover the procedure until after a second or third miscarriage.
This, said Dr. Williams, should be in the field in less than a year, and he estimates that the test will be used for fresh embryos in about a year and a half.
Sperm collection made simpler
Typically, a man delivers a sperm sample in a room at an IVF clinic or by collecting a sample at home and rushing it to the clinic before it degrades, which Dr. Williams said can happen in as little as 15 minutes.
In 2020, Dr. Williams and his team began using a custom at-home sperm collection box that houses sperm in a recyclable foam container that holds a sample cup, which is filled with special sperm-supporting media, at an angle that prevents evaporation and maintains temperature and pH. This allows patients to collect samples in the comfort of their homes and increases the clock to 3 hours.
“It’s great for the patients because it’s much more comfortable,” said Dr. Williams, who notes that having to “perform” on site can be stressful for men. Studies the team has conducted have shown sperm collected in this manner have a better success rate than those collected in the lab, and 90% of Columbia’s Fertility Center patients are now providing sperm samples this way.
Similar innovations to deliver sperm, like Protex, are now on the market, while companies like myLabBox and Legacy are offering at-home sperm testing kits to mail in for a full semen analysis.
At-home monitoring: More and better
Wearable reproductive health devices are also helping more women get pregnant. “I am very excited about biometric data harnessed in wearables to predict periods, ovulation, and fertility,” said Amander Clark, PhD, director of the UCLA Center for Reproductive Science, Health, and Education, Los Angeles.
The Tempdrop Fertility and Ovulation Tracker, for instance, is a wearable sensor with an accompanying charting app that helps a woman identify her most fertile days to conceive. The Bellabeat Ivy is a women’s health smart bracelet with a strong focus on tracking a woman’s cycle and fertility, pregnancy, and postnatal symptoms. And Mirvie, which is currently in development, is a blood test that will be able to predict pregnancy complications earlier.
Physicians are also looking to move as much of the lab experience as they can into a patient’s home, which streamlines processes while offering privacy and comfort. For example, Dr. Hariton, who runs a strategic venture capital fund for physicians, said his team is currently working with a company that does remote ultrasounds.
And Mira, an at-home hormone monitor, uses patented AI algorithms to accurately measure the levels of major reproductive health hormones (E3G, LH, PdG, FSH) in urine, said Meir Olcha, MD, chief medical officer at Sama Fertility. The product recently completed a clinical trial, which showed it was a viable alternative to blood serum for patients undergoing IVF.
Stem cells could make eggs ageless
Research shows that a woman’s egg quality decreases gradually but significantly starting at age 32 and more rapidly after 37. Sperm quality may also decrease with age. A possible workaround: Scientists are actively researching how to create eggs and sperm from stem cells.
“I think getting eggs from stem cells will happen in the future,” said Dr. Hariton, who notes that this type of technology would be a game changer in his clinic. “It will make some of the hardest diagnoses that I have – which is on a daily basis, ‘I’m so sorry, you’re in premature menopause’ or ‘I don’t think we’re going to be successful getting you pregnant with your own eggs; here are some other options like donor eggs’ – much better,” he added. And stem cells are currently being used to research causes of infertility.
Clinics like UCLA have already been making strides. “We are using stem cells to identify new genes required for reproduction and to define the role of these genes in human fertility and infertility,” said Dr. Clark, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, who recently led a study in this arena. “In vitro gametogenesis (IVG), another stem cell technology, is currently used in the research lab to understand causes of infertility.”
These stem cell-based embryo models, she said, can help researchers understand the first few days of embryo development after an embryo implants and be used to provide critical information on causes of early pregnancy loss or birth defects.
A version of this article appeared on Medscape.com.
Fifth pregnancy, first baby.
After four pregnancies resulted in losses – and doing things as natural as possible and leaving it up to the birds, bees, and fate – my husband and I decided to explore in vitro fertilization (IVF).
Drugs to direct my follicles to produce more eggs, an egg retrieval procedure, genetic testing of our embryos, a quick procedure to remove a residual uterine septum from my uterus, drugs to thicken my endometrial lining to prepare my body to receive an embryo, an embryo transfer, steroids to suppress my immune system so my body would accept the pregnancy, blood thinner shots to promote blood flow to the baby, and 10 weeks of progesterone in oil shots later and we’re days away from welcoming our first baby into our lives.
In short, there’s more than one way to define “miracle baby.”
Global estimates say 48 million couples and 186 million individuals struggle with infertility. On average, 2 million infants born in the United States each year are conceived through assisted reproductive technology and the demand for treatments like IVF have doubled in the last decade.
Now the need for treatments outweighs clinician availability. “We have about 1,250 practicing fertility physicians in the U.S. to serve the whole country, which is highly inadequate,” said Eduardo Hariton, MD, a reproductive endocrinology physician in San Francisco and managing director of the U.S. Fertility Innovation Fund. “We have people that want to get care waiting 1 to 3 months to be seen.”
Dr. Hariton explains that U.S. IVF clinics are performing around 250,000 to 300,000 IVF cycles per year and need to be doing a million-plus to meet demand. This, plus the cost of fertility treatments – an average IVF cycle runs $23,500 and the majority of patients need multiple cycles to conceive – keeps the barrier to entry high.
Enter technology: New advances are on the way to help the assisted fertility process to run smoother and be less costly. “The field is really coming into an age of great progress and innovation,” added S. Zev Williams, MD, PhD, chief of the division of reproductive endocrinology and Infertility at Columbia University Irving Medical Center, New York City.
I’m personally grateful that such technology exists.
AI will help, of course
Fertility treatments involve endless analysis, diagnosis, and recommendations – dozens if not hundreds of decisions from each physician for each patient. Human action and reaction can affect this process, Dr. Hariton explained.
For example, if he hyperstimulated a woman during the follicle growing stage of her egg retrieval and ended up with eggs too large to retrieve, Dr. Hariton said he may subconsciously be more inclined to be extra cautious with his patients the week after, and vice versa.
This is where AI can help. “Rather than me making decisions from a couple of thousands of cycles of experience, I get to leverage hundreds of thousands of cycles from different providers over different people,” said Dr. Hariton. “I get to use all the data from that patient today – her age, her weight, what happened last cycle, how she’s doing – and make a very objective decision about the optimal time to give that woman or that couple the best outcome possible.”
AI can also assist with tasks like embryo grading. “Once our embryos are made in the lab, we usually have an embryologist looking at those embryos, grading them on a three-variable scale, and then picking the nicest one for transfer,” said Dr. Hariton. Machine learning computer vision software can help doctors select the best embryo.
Many of these AI products are in trials in the United States and some AI-based technology is already being used in fertility labs, especially in other countries. “ALife recently launched a suite of products to help with their decisions during stimulation that can help with the quality KPIs [key performance indicators] in the lab,” said Dr. Hariton. “There’s also a company that does AI-based predictions of success to give patients a better estimate called Univfy.” More AI products are still in development or awaiting Food and Drug Administration clearance.
Robots lend a hand
Like artificial intelligence, robots can be a big help in the IVF lab. Columbia University Fertility Center recently became the first to use an articulated (ART) robot to handle precise and highly repetitive work.
“IVF, from the initial point, involves creating these special plates where embryos can grow, and you do that by making little droplets,” said Dr. Williams. “It’s very time-consuming to create tons of these little droplets for the embryos to grow.” Thus, the lab created a robot to help squirt drops of the media substance required to sustain embryos in a way that is 10 times more precise than that of a trained embryologist.
“It’s a win-win because you allow the robots to do things better than a human can and this allows the humans to do things that a robot just can’t do,” explained Dr. Williams. He and his team began using this technology in the beginning of November 2022.
Dr. Williams sees ART robots being used in many more parts of the fertility treatment journey along the way, like preparing eggs after they are retrieved and performing intracytoplasmic sperm injection (ICSI), with the robot injecting the sperm into the egg.
Launching with the plate making, said Dr. Williams, is a low stakes entry point for robotic technology in the lab. “It allows us to introduce robotics to automate and optimize each step along the way, but to do so in the safest possible way.”
Dr. Williams estimates that robots will have their hands on actual eggs and sperm in 5 years.
Updates in genetic testing
Currently, if a couple wants to have their embryos genetically tested, also known as preimplantation genetic testing, each embryo must be frozen, then a biopsy of that embryo is performed and sent to the lab.
“It takes time to get the results,” said Dr. Williams. “The whole time you’re waiting, you don’t know if you’re going to have any embryos that are transferable or if next month you’re going to have to do another IVF cycle.”
Columbia researchers recently developed a new in-house test that can determine if a fetus or embryo has the right number of chromosomes. This STORK (Short-read Transpore Rapid Karyotyping) can be performed without freezing embryos and sending them out, which Dr. Williams said can save couples money and time, as they won’t necessarily need to do a separate embryo transfer cycle and can transfer an embryo in the same cycle. “You can test in the morning and transfer in the afternoon,” said Dr. Williams.
The test is currently awaiting approval and will first be used to test miscarriage samples to see if embryos were genetically normal or not, which he said should cost around $200 vs. the $2,000 to $4,000 it can cost to have fetal tissue sent to the lab – and insurance doesn’t cover the procedure until after a second or third miscarriage.
This, said Dr. Williams, should be in the field in less than a year, and he estimates that the test will be used for fresh embryos in about a year and a half.
Sperm collection made simpler
Typically, a man delivers a sperm sample in a room at an IVF clinic or by collecting a sample at home and rushing it to the clinic before it degrades, which Dr. Williams said can happen in as little as 15 minutes.
In 2020, Dr. Williams and his team began using a custom at-home sperm collection box that houses sperm in a recyclable foam container that holds a sample cup, which is filled with special sperm-supporting media, at an angle that prevents evaporation and maintains temperature and pH. This allows patients to collect samples in the comfort of their homes and increases the clock to 3 hours.
“It’s great for the patients because it’s much more comfortable,” said Dr. Williams, who notes that having to “perform” on site can be stressful for men. Studies the team has conducted have shown sperm collected in this manner have a better success rate than those collected in the lab, and 90% of Columbia’s Fertility Center patients are now providing sperm samples this way.
Similar innovations to deliver sperm, like Protex, are now on the market, while companies like myLabBox and Legacy are offering at-home sperm testing kits to mail in for a full semen analysis.
At-home monitoring: More and better
Wearable reproductive health devices are also helping more women get pregnant. “I am very excited about biometric data harnessed in wearables to predict periods, ovulation, and fertility,” said Amander Clark, PhD, director of the UCLA Center for Reproductive Science, Health, and Education, Los Angeles.
The Tempdrop Fertility and Ovulation Tracker, for instance, is a wearable sensor with an accompanying charting app that helps a woman identify her most fertile days to conceive. The Bellabeat Ivy is a women’s health smart bracelet with a strong focus on tracking a woman’s cycle and fertility, pregnancy, and postnatal symptoms. And Mirvie, which is currently in development, is a blood test that will be able to predict pregnancy complications earlier.
Physicians are also looking to move as much of the lab experience as they can into a patient’s home, which streamlines processes while offering privacy and comfort. For example, Dr. Hariton, who runs a strategic venture capital fund for physicians, said his team is currently working with a company that does remote ultrasounds.
And Mira, an at-home hormone monitor, uses patented AI algorithms to accurately measure the levels of major reproductive health hormones (E3G, LH, PdG, FSH) in urine, said Meir Olcha, MD, chief medical officer at Sama Fertility. The product recently completed a clinical trial, which showed it was a viable alternative to blood serum for patients undergoing IVF.
Stem cells could make eggs ageless
Research shows that a woman’s egg quality decreases gradually but significantly starting at age 32 and more rapidly after 37. Sperm quality may also decrease with age. A possible workaround: Scientists are actively researching how to create eggs and sperm from stem cells.
“I think getting eggs from stem cells will happen in the future,” said Dr. Hariton, who notes that this type of technology would be a game changer in his clinic. “It will make some of the hardest diagnoses that I have – which is on a daily basis, ‘I’m so sorry, you’re in premature menopause’ or ‘I don’t think we’re going to be successful getting you pregnant with your own eggs; here are some other options like donor eggs’ – much better,” he added. And stem cells are currently being used to research causes of infertility.
Clinics like UCLA have already been making strides. “We are using stem cells to identify new genes required for reproduction and to define the role of these genes in human fertility and infertility,” said Dr. Clark, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, who recently led a study in this arena. “In vitro gametogenesis (IVG), another stem cell technology, is currently used in the research lab to understand causes of infertility.”
These stem cell-based embryo models, she said, can help researchers understand the first few days of embryo development after an embryo implants and be used to provide critical information on causes of early pregnancy loss or birth defects.
A version of this article appeared on Medscape.com.
Fifth pregnancy, first baby.
After four pregnancies resulted in losses – and doing things as natural as possible and leaving it up to the birds, bees, and fate – my husband and I decided to explore in vitro fertilization (IVF).
Drugs to direct my follicles to produce more eggs, an egg retrieval procedure, genetic testing of our embryos, a quick procedure to remove a residual uterine septum from my uterus, drugs to thicken my endometrial lining to prepare my body to receive an embryo, an embryo transfer, steroids to suppress my immune system so my body would accept the pregnancy, blood thinner shots to promote blood flow to the baby, and 10 weeks of progesterone in oil shots later and we’re days away from welcoming our first baby into our lives.
In short, there’s more than one way to define “miracle baby.”
Global estimates say 48 million couples and 186 million individuals struggle with infertility. On average, 2 million infants born in the United States each year are conceived through assisted reproductive technology and the demand for treatments like IVF have doubled in the last decade.
Now the need for treatments outweighs clinician availability. “We have about 1,250 practicing fertility physicians in the U.S. to serve the whole country, which is highly inadequate,” said Eduardo Hariton, MD, a reproductive endocrinology physician in San Francisco and managing director of the U.S. Fertility Innovation Fund. “We have people that want to get care waiting 1 to 3 months to be seen.”
Dr. Hariton explains that U.S. IVF clinics are performing around 250,000 to 300,000 IVF cycles per year and need to be doing a million-plus to meet demand. This, plus the cost of fertility treatments – an average IVF cycle runs $23,500 and the majority of patients need multiple cycles to conceive – keeps the barrier to entry high.
Enter technology: New advances are on the way to help the assisted fertility process to run smoother and be less costly. “The field is really coming into an age of great progress and innovation,” added S. Zev Williams, MD, PhD, chief of the division of reproductive endocrinology and Infertility at Columbia University Irving Medical Center, New York City.
I’m personally grateful that such technology exists.
AI will help, of course
Fertility treatments involve endless analysis, diagnosis, and recommendations – dozens if not hundreds of decisions from each physician for each patient. Human action and reaction can affect this process, Dr. Hariton explained.
For example, if he hyperstimulated a woman during the follicle growing stage of her egg retrieval and ended up with eggs too large to retrieve, Dr. Hariton said he may subconsciously be more inclined to be extra cautious with his patients the week after, and vice versa.
This is where AI can help. “Rather than me making decisions from a couple of thousands of cycles of experience, I get to leverage hundreds of thousands of cycles from different providers over different people,” said Dr. Hariton. “I get to use all the data from that patient today – her age, her weight, what happened last cycle, how she’s doing – and make a very objective decision about the optimal time to give that woman or that couple the best outcome possible.”
AI can also assist with tasks like embryo grading. “Once our embryos are made in the lab, we usually have an embryologist looking at those embryos, grading them on a three-variable scale, and then picking the nicest one for transfer,” said Dr. Hariton. Machine learning computer vision software can help doctors select the best embryo.
Many of these AI products are in trials in the United States and some AI-based technology is already being used in fertility labs, especially in other countries. “ALife recently launched a suite of products to help with their decisions during stimulation that can help with the quality KPIs [key performance indicators] in the lab,” said Dr. Hariton. “There’s also a company that does AI-based predictions of success to give patients a better estimate called Univfy.” More AI products are still in development or awaiting Food and Drug Administration clearance.
Robots lend a hand
Like artificial intelligence, robots can be a big help in the IVF lab. Columbia University Fertility Center recently became the first to use an articulated (ART) robot to handle precise and highly repetitive work.
“IVF, from the initial point, involves creating these special plates where embryos can grow, and you do that by making little droplets,” said Dr. Williams. “It’s very time-consuming to create tons of these little droplets for the embryos to grow.” Thus, the lab created a robot to help squirt drops of the media substance required to sustain embryos in a way that is 10 times more precise than that of a trained embryologist.
“It’s a win-win because you allow the robots to do things better than a human can and this allows the humans to do things that a robot just can’t do,” explained Dr. Williams. He and his team began using this technology in the beginning of November 2022.
Dr. Williams sees ART robots being used in many more parts of the fertility treatment journey along the way, like preparing eggs after they are retrieved and performing intracytoplasmic sperm injection (ICSI), with the robot injecting the sperm into the egg.
Launching with the plate making, said Dr. Williams, is a low stakes entry point for robotic technology in the lab. “It allows us to introduce robotics to automate and optimize each step along the way, but to do so in the safest possible way.”
Dr. Williams estimates that robots will have their hands on actual eggs and sperm in 5 years.
Updates in genetic testing
Currently, if a couple wants to have their embryos genetically tested, also known as preimplantation genetic testing, each embryo must be frozen, then a biopsy of that embryo is performed and sent to the lab.
“It takes time to get the results,” said Dr. Williams. “The whole time you’re waiting, you don’t know if you’re going to have any embryos that are transferable or if next month you’re going to have to do another IVF cycle.”
Columbia researchers recently developed a new in-house test that can determine if a fetus or embryo has the right number of chromosomes. This STORK (Short-read Transpore Rapid Karyotyping) can be performed without freezing embryos and sending them out, which Dr. Williams said can save couples money and time, as they won’t necessarily need to do a separate embryo transfer cycle and can transfer an embryo in the same cycle. “You can test in the morning and transfer in the afternoon,” said Dr. Williams.
The test is currently awaiting approval and will first be used to test miscarriage samples to see if embryos were genetically normal or not, which he said should cost around $200 vs. the $2,000 to $4,000 it can cost to have fetal tissue sent to the lab – and insurance doesn’t cover the procedure until after a second or third miscarriage.
This, said Dr. Williams, should be in the field in less than a year, and he estimates that the test will be used for fresh embryos in about a year and a half.
Sperm collection made simpler
Typically, a man delivers a sperm sample in a room at an IVF clinic or by collecting a sample at home and rushing it to the clinic before it degrades, which Dr. Williams said can happen in as little as 15 minutes.
In 2020, Dr. Williams and his team began using a custom at-home sperm collection box that houses sperm in a recyclable foam container that holds a sample cup, which is filled with special sperm-supporting media, at an angle that prevents evaporation and maintains temperature and pH. This allows patients to collect samples in the comfort of their homes and increases the clock to 3 hours.
“It’s great for the patients because it’s much more comfortable,” said Dr. Williams, who notes that having to “perform” on site can be stressful for men. Studies the team has conducted have shown sperm collected in this manner have a better success rate than those collected in the lab, and 90% of Columbia’s Fertility Center patients are now providing sperm samples this way.
Similar innovations to deliver sperm, like Protex, are now on the market, while companies like myLabBox and Legacy are offering at-home sperm testing kits to mail in for a full semen analysis.
At-home monitoring: More and better
Wearable reproductive health devices are also helping more women get pregnant. “I am very excited about biometric data harnessed in wearables to predict periods, ovulation, and fertility,” said Amander Clark, PhD, director of the UCLA Center for Reproductive Science, Health, and Education, Los Angeles.
The Tempdrop Fertility and Ovulation Tracker, for instance, is a wearable sensor with an accompanying charting app that helps a woman identify her most fertile days to conceive. The Bellabeat Ivy is a women’s health smart bracelet with a strong focus on tracking a woman’s cycle and fertility, pregnancy, and postnatal symptoms. And Mirvie, which is currently in development, is a blood test that will be able to predict pregnancy complications earlier.
Physicians are also looking to move as much of the lab experience as they can into a patient’s home, which streamlines processes while offering privacy and comfort. For example, Dr. Hariton, who runs a strategic venture capital fund for physicians, said his team is currently working with a company that does remote ultrasounds.
And Mira, an at-home hormone monitor, uses patented AI algorithms to accurately measure the levels of major reproductive health hormones (E3G, LH, PdG, FSH) in urine, said Meir Olcha, MD, chief medical officer at Sama Fertility. The product recently completed a clinical trial, which showed it was a viable alternative to blood serum for patients undergoing IVF.
Stem cells could make eggs ageless
Research shows that a woman’s egg quality decreases gradually but significantly starting at age 32 and more rapidly after 37. Sperm quality may also decrease with age. A possible workaround: Scientists are actively researching how to create eggs and sperm from stem cells.
“I think getting eggs from stem cells will happen in the future,” said Dr. Hariton, who notes that this type of technology would be a game changer in his clinic. “It will make some of the hardest diagnoses that I have – which is on a daily basis, ‘I’m so sorry, you’re in premature menopause’ or ‘I don’t think we’re going to be successful getting you pregnant with your own eggs; here are some other options like donor eggs’ – much better,” he added. And stem cells are currently being used to research causes of infertility.
Clinics like UCLA have already been making strides. “We are using stem cells to identify new genes required for reproduction and to define the role of these genes in human fertility and infertility,” said Dr. Clark, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, who recently led a study in this arena. “In vitro gametogenesis (IVG), another stem cell technology, is currently used in the research lab to understand causes of infertility.”
These stem cell-based embryo models, she said, can help researchers understand the first few days of embryo development after an embryo implants and be used to provide critical information on causes of early pregnancy loss or birth defects.
A version of this article appeared on Medscape.com.
Case series supports targeted drugs in treatment of alopecia in children with AD
in children with AA and concomitant atopy.
It was only a little over a year ago that the JAK inhibitor baricitinib became the first systemic therapy approved by the Food and Drug Administration for AA in adults. In June 2023, the JAK inhibitor ritlecitinib was approved for severe AA in patients as young as 12 years of age, but there is accumulating evidence that dupilumab, which binds to the interleukin-4 receptor, might be an option for even younger children with AA.
Of those who have worked with dupilumab for controlling AA in children, Brittany Craiglow, MD, an adjunct associate professor of dermatology at Yale University, New Haven, Conn., updated a case series at the recent MedscapeLive! Annual Women’s and Pediatric Dermatology Seminar in Baltimore. A series of six children with AA treated with dupilumab was published 2 years ago in JAAD Case Reports.
Even in 2021, her case series was not the first report of benefit from dupilumab in children with AA, but instead contributed to a “growing body of literature” supporting the potential benefit in the setting of concomitant atopy, Dr. Craiglow, one of the authors of the series, said in an interview.
Of the six patients in that series, five had improvement and four had complete regrowth with dupilumab, whether as a monotherapy or in combination with other agents. The children ranged in age from 7 to 12 years. The age range at the time of AA onset was 3-11 years. All had atopic dermatitis (AD) and most had additional atopic conditions, such as food allergies or asthma.
Since publication, Dr. Craiglow has successfully treated many more patients with dupilumab, either as monotherapy or in combination with oral minoxidil, corticosteroids, and/or a topical JAK inhibitor. Dupilumab, which is approved for the treatment of AD in children as young as 6 months of age, has been well tolerated.
“Oral minoxidil is often a great adjuvant treatment in patients with AA and should be used unless there are contraindications,” based on the initial and subsequent experience treating AA with dupilumab, said Dr. Craiglow.
“Topical steroids can be used in combination with dupilumab and minoxidil, but in general dupilumab should not be combined with an oral JAK inhibitor,” she added.
Now, with the approval of ritlecitinib, Dr. Craiglow said this JAK inhibitor will become a first-line therapy in children 12 years or older with severe, persistent AA, but she considers a trial of dupilumab reasonable in younger children, given the controlled studies of safety for atopic diseases.
“I would say that dupilumab could be considered in the following clinical scenarios: children under 12 with AA and concomitant atopy, such as atopic dermatitis, asthma, allergies, and/or elevated IgE; and children over the age of 12 with concomitant atopy who either have a contraindication to a JAK inhibitor or whose families have reservations about or are unwilling to take one,” Dr. Craiglow said.
In older children, she believes that dupilumab has “a much lower chance of being effective” than an oral JAK inhibitor like ritlecitinib, but it circumvents the potential safety issues of JAK inhibitors that have been observed in adults.
With ritlecitinib providing an on-label option for AA in older children, Dr. Craiglow suggested it might be easier to obtain third-party coverage for dupilumab as an alternative to a JAK inhibitor for AA in patients younger than 12, particularly when there is an indication for a concomitant atopic condition and a rationale, such as a concern about relative safety.
Two years ago, when Dr. Craiglow and her coinvestigator published their six-patient case series, a second case series was published about the same time by investigators at the University of Pennsylvania, Philadelphia, in the Journal of the American Academy of Dermatology. This series of 16 pediatric patients with AA on dupilumab was more heterogeneous, but four of six patients with active disease and more than 4 months of follow-up had improvement in AA, including total regrowth. The improvement was concentrated in patients with moderate to severe AD at the time of treatment.
Based on this series, the authors, led by Leslie Castelo-Soccio, MD, PhD, who is now an attending physician in the Dermatology Branch of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Md., concluded that dupilumab “may be a therapeutic option for AA” when traditional therapies have failed, “especially in patients with concurrent AD or asthma, for which the benefits of dupilumab are clear.”
When contacted about where this therapy might fit on the basis of her case series and the update on Dr. Craiglow’s experience, Dr. Castelo-Soccio, like Dr. Craiglow, stressed the importance of employing this therapy selectively.
“I do think that dupilumab is a reasonable option for AA in children with atopy and IgE levels greater than 200 IU/mL, especially if treatment is for atopic dermatitis or asthma as well,” she said.
Many clinicians, including Dr. Craiglow, have experience with oral JAK inhibitors in children younger than 12. Indeed, a recently published case study associated oral abrocitinib, a JAK inhibitor approved for moderate to severe AD in patients ages 12 and older, with hair regrowth in an 11-year-old child who had persistent AA for more than 6 years despite numerous conventional therapies.
However, the advantage of dupilumab in younger children is the greater evidence of safety, providing a level of reassurance for a treatment that is commonly used for severe atopic diseases but does not have a specific indication for AA, according to Dr. Craiglow.
Dr. Craiglow disclosed being a speaker for AbbVie and a speaker and consultant for Eli Lilly, Incyte, Pfizer, Regeneron, and Sanofi Genzyme. Dr. Castelo-Soccio had no disclosures.
in children with AA and concomitant atopy.
It was only a little over a year ago that the JAK inhibitor baricitinib became the first systemic therapy approved by the Food and Drug Administration for AA in adults. In June 2023, the JAK inhibitor ritlecitinib was approved for severe AA in patients as young as 12 years of age, but there is accumulating evidence that dupilumab, which binds to the interleukin-4 receptor, might be an option for even younger children with AA.
Of those who have worked with dupilumab for controlling AA in children, Brittany Craiglow, MD, an adjunct associate professor of dermatology at Yale University, New Haven, Conn., updated a case series at the recent MedscapeLive! Annual Women’s and Pediatric Dermatology Seminar in Baltimore. A series of six children with AA treated with dupilumab was published 2 years ago in JAAD Case Reports.
Even in 2021, her case series was not the first report of benefit from dupilumab in children with AA, but instead contributed to a “growing body of literature” supporting the potential benefit in the setting of concomitant atopy, Dr. Craiglow, one of the authors of the series, said in an interview.
Of the six patients in that series, five had improvement and four had complete regrowth with dupilumab, whether as a monotherapy or in combination with other agents. The children ranged in age from 7 to 12 years. The age range at the time of AA onset was 3-11 years. All had atopic dermatitis (AD) and most had additional atopic conditions, such as food allergies or asthma.
Since publication, Dr. Craiglow has successfully treated many more patients with dupilumab, either as monotherapy or in combination with oral minoxidil, corticosteroids, and/or a topical JAK inhibitor. Dupilumab, which is approved for the treatment of AD in children as young as 6 months of age, has been well tolerated.
“Oral minoxidil is often a great adjuvant treatment in patients with AA and should be used unless there are contraindications,” based on the initial and subsequent experience treating AA with dupilumab, said Dr. Craiglow.
“Topical steroids can be used in combination with dupilumab and minoxidil, but in general dupilumab should not be combined with an oral JAK inhibitor,” she added.
Now, with the approval of ritlecitinib, Dr. Craiglow said this JAK inhibitor will become a first-line therapy in children 12 years or older with severe, persistent AA, but she considers a trial of dupilumab reasonable in younger children, given the controlled studies of safety for atopic diseases.
“I would say that dupilumab could be considered in the following clinical scenarios: children under 12 with AA and concomitant atopy, such as atopic dermatitis, asthma, allergies, and/or elevated IgE; and children over the age of 12 with concomitant atopy who either have a contraindication to a JAK inhibitor or whose families have reservations about or are unwilling to take one,” Dr. Craiglow said.
In older children, she believes that dupilumab has “a much lower chance of being effective” than an oral JAK inhibitor like ritlecitinib, but it circumvents the potential safety issues of JAK inhibitors that have been observed in adults.
With ritlecitinib providing an on-label option for AA in older children, Dr. Craiglow suggested it might be easier to obtain third-party coverage for dupilumab as an alternative to a JAK inhibitor for AA in patients younger than 12, particularly when there is an indication for a concomitant atopic condition and a rationale, such as a concern about relative safety.
Two years ago, when Dr. Craiglow and her coinvestigator published their six-patient case series, a second case series was published about the same time by investigators at the University of Pennsylvania, Philadelphia, in the Journal of the American Academy of Dermatology. This series of 16 pediatric patients with AA on dupilumab was more heterogeneous, but four of six patients with active disease and more than 4 months of follow-up had improvement in AA, including total regrowth. The improvement was concentrated in patients with moderate to severe AD at the time of treatment.
Based on this series, the authors, led by Leslie Castelo-Soccio, MD, PhD, who is now an attending physician in the Dermatology Branch of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Md., concluded that dupilumab “may be a therapeutic option for AA” when traditional therapies have failed, “especially in patients with concurrent AD or asthma, for which the benefits of dupilumab are clear.”
When contacted about where this therapy might fit on the basis of her case series and the update on Dr. Craiglow’s experience, Dr. Castelo-Soccio, like Dr. Craiglow, stressed the importance of employing this therapy selectively.
“I do think that dupilumab is a reasonable option for AA in children with atopy and IgE levels greater than 200 IU/mL, especially if treatment is for atopic dermatitis or asthma as well,” she said.
Many clinicians, including Dr. Craiglow, have experience with oral JAK inhibitors in children younger than 12. Indeed, a recently published case study associated oral abrocitinib, a JAK inhibitor approved for moderate to severe AD in patients ages 12 and older, with hair regrowth in an 11-year-old child who had persistent AA for more than 6 years despite numerous conventional therapies.
However, the advantage of dupilumab in younger children is the greater evidence of safety, providing a level of reassurance for a treatment that is commonly used for severe atopic diseases but does not have a specific indication for AA, according to Dr. Craiglow.
Dr. Craiglow disclosed being a speaker for AbbVie and a speaker and consultant for Eli Lilly, Incyte, Pfizer, Regeneron, and Sanofi Genzyme. Dr. Castelo-Soccio had no disclosures.
in children with AA and concomitant atopy.
It was only a little over a year ago that the JAK inhibitor baricitinib became the first systemic therapy approved by the Food and Drug Administration for AA in adults. In June 2023, the JAK inhibitor ritlecitinib was approved for severe AA in patients as young as 12 years of age, but there is accumulating evidence that dupilumab, which binds to the interleukin-4 receptor, might be an option for even younger children with AA.
Of those who have worked with dupilumab for controlling AA in children, Brittany Craiglow, MD, an adjunct associate professor of dermatology at Yale University, New Haven, Conn., updated a case series at the recent MedscapeLive! Annual Women’s and Pediatric Dermatology Seminar in Baltimore. A series of six children with AA treated with dupilumab was published 2 years ago in JAAD Case Reports.
Even in 2021, her case series was not the first report of benefit from dupilumab in children with AA, but instead contributed to a “growing body of literature” supporting the potential benefit in the setting of concomitant atopy, Dr. Craiglow, one of the authors of the series, said in an interview.
Of the six patients in that series, five had improvement and four had complete regrowth with dupilumab, whether as a monotherapy or in combination with other agents. The children ranged in age from 7 to 12 years. The age range at the time of AA onset was 3-11 years. All had atopic dermatitis (AD) and most had additional atopic conditions, such as food allergies or asthma.
Since publication, Dr. Craiglow has successfully treated many more patients with dupilumab, either as monotherapy or in combination with oral minoxidil, corticosteroids, and/or a topical JAK inhibitor. Dupilumab, which is approved for the treatment of AD in children as young as 6 months of age, has been well tolerated.
“Oral minoxidil is often a great adjuvant treatment in patients with AA and should be used unless there are contraindications,” based on the initial and subsequent experience treating AA with dupilumab, said Dr. Craiglow.
“Topical steroids can be used in combination with dupilumab and minoxidil, but in general dupilumab should not be combined with an oral JAK inhibitor,” she added.
Now, with the approval of ritlecitinib, Dr. Craiglow said this JAK inhibitor will become a first-line therapy in children 12 years or older with severe, persistent AA, but she considers a trial of dupilumab reasonable in younger children, given the controlled studies of safety for atopic diseases.
“I would say that dupilumab could be considered in the following clinical scenarios: children under 12 with AA and concomitant atopy, such as atopic dermatitis, asthma, allergies, and/or elevated IgE; and children over the age of 12 with concomitant atopy who either have a contraindication to a JAK inhibitor or whose families have reservations about or are unwilling to take one,” Dr. Craiglow said.
In older children, she believes that dupilumab has “a much lower chance of being effective” than an oral JAK inhibitor like ritlecitinib, but it circumvents the potential safety issues of JAK inhibitors that have been observed in adults.
With ritlecitinib providing an on-label option for AA in older children, Dr. Craiglow suggested it might be easier to obtain third-party coverage for dupilumab as an alternative to a JAK inhibitor for AA in patients younger than 12, particularly when there is an indication for a concomitant atopic condition and a rationale, such as a concern about relative safety.
Two years ago, when Dr. Craiglow and her coinvestigator published their six-patient case series, a second case series was published about the same time by investigators at the University of Pennsylvania, Philadelphia, in the Journal of the American Academy of Dermatology. This series of 16 pediatric patients with AA on dupilumab was more heterogeneous, but four of six patients with active disease and more than 4 months of follow-up had improvement in AA, including total regrowth. The improvement was concentrated in patients with moderate to severe AD at the time of treatment.
Based on this series, the authors, led by Leslie Castelo-Soccio, MD, PhD, who is now an attending physician in the Dermatology Branch of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Md., concluded that dupilumab “may be a therapeutic option for AA” when traditional therapies have failed, “especially in patients with concurrent AD or asthma, for which the benefits of dupilumab are clear.”
When contacted about where this therapy might fit on the basis of her case series and the update on Dr. Craiglow’s experience, Dr. Castelo-Soccio, like Dr. Craiglow, stressed the importance of employing this therapy selectively.
“I do think that dupilumab is a reasonable option for AA in children with atopy and IgE levels greater than 200 IU/mL, especially if treatment is for atopic dermatitis or asthma as well,” she said.
Many clinicians, including Dr. Craiglow, have experience with oral JAK inhibitors in children younger than 12. Indeed, a recently published case study associated oral abrocitinib, a JAK inhibitor approved for moderate to severe AD in patients ages 12 and older, with hair regrowth in an 11-year-old child who had persistent AA for more than 6 years despite numerous conventional therapies.
However, the advantage of dupilumab in younger children is the greater evidence of safety, providing a level of reassurance for a treatment that is commonly used for severe atopic diseases but does not have a specific indication for AA, according to Dr. Craiglow.
Dr. Craiglow disclosed being a speaker for AbbVie and a speaker and consultant for Eli Lilly, Incyte, Pfizer, Regeneron, and Sanofi Genzyme. Dr. Castelo-Soccio had no disclosures.
New Alzheimer’s drugs: Setting realistic expectations
With the Food and Drug Administration’s full stamp of approval in hand, Leqembi (lecanemab) is poised to catapult us into a new era of treatment for Alzheimer’s disease. And now that the donanemab trial data are out, there’s another antiamyloid drug waiting in the wings.
To finally have true disease-modifying therapies for Alzheimer’s disease is a massive step forward for a field that’s been plagued with disappointment. But these drugs come with serious concerns and unknowns. They will require complex decision-making, putting doctors, patients, and their families in a medical quandary.
Striking the right balance between cautious optimism and realistic expectations will be a formidable challenge.
Managing patient and family expectations
These drugs are no magic bullet. They slow down the dementia’s progression, buying patients more time (on the order of months) before they begin to experience significant worsening. We’ll need a lot more information from research and clinical experience before we can understand how meaningful that treatment effect is. Right now, it is unclear whether eligible patients and their families will even perceive tangible differences.
In the CLARITY-AD trial, participants on lecanemab experienced a 27% slowing in the rate of cognitive decline over 18 months. Donanemab was shown to slow decline in memory and cognition by about 35% over the same time frame in the TRAILBLAZER-ALZ 2 trial. That translates to more time for patients and their families to enjoy independence, maintain normal life, and stave off the most distressing parts of the disease.
But what happens after 18 months of treatment – will the treatment effect magnify or dissipate? How much time are we really buying in the long run? Counseling patients and their families is made all the more difficult when the answers to important questions like these remain to be seen.
Only a sliver of Alzheimer’s patients are current candidates
The fact is that most patients living with Alzheimer’s disease will not qualify for treatment with these drugs. Lecanemab is approved for people with early-stage disease, meaning their dementia is mild or they have mild cognitive impairment, which is a precursor to full-blown Alzheimer’s disease. Of the 6 million people in the United States living with Alzheimer’s, about 1.5 million are estimated to fall into that category. We can expect to see a similar qualifier for donanemab if it receives FDA approval, especially because that trial suggested a more pronounced treatment effect for patients in the earliest stages of the disease.
Even if a patient hits the sweet spot where they have just enough cognitive impairment, but not too much, they aren’t technically therapeutic candidates until prerequisite testing confirms amyloid protein accumulation in the brain via PET scan or cerebrospinal fluid analysis.
Even then, the FDA’s boxed warning for lecanemab recommends that patients undergo genetic testing for the apo E4 mutation to identify those at a particularly high risk for severe adverse effects including brain bleeding and swelling. This recommendation is not unreasonable considering that 15% of the Alzheimer’s population has two copies of the apo E4 mutation and fall into that high-risk group.
Significant risks
Antiamyloid drugs are well-known to cause serious side effects. In the lecanemab trial, 13% of participants receiving Leqembi experienced brain swelling (vs. 2% of participants receiving placebo) and 17% of participants had brain bleeding (vs. 9% of participants on placebo). In the donanemab trial, brain bleeding occurred in 31.4% of participants on the drug (vs. 13.6% on placebo) and swelling occurred in 24% (vs. 2.1% receiving placebo). Thankfully, in both trials, most of these adverse events did not produce significant symptoms, but in rare cases these events caused severe or catastrophic neurologic injury, including death.
How can we best guide patients and their families to weigh the uncertain benefits against potentially serious risks? We can start by considering the patient characteristics most likely to portend increased risk for serious side effects: apo E4 mutations, blood thinner use, and the presence of microhemorrhages on brain imaging. But after that, we’re left with a lot of uncertainty in terms of which patients are most likely to see meaningful clinical improvements from the drug and unknown factors that may increase the risks of treatment.
A costly therapy
Medicare plans to cover 80% of lecanemab’s steep cost of $26,500 per year. Still, that will leave many patients with a hefty copay, potentially over $6,000 per year. But that only scratches the surface. Consider the frequent medical visits, repeated brain scans, laboratory tests, and infusion center appointments. It’s been estimated that all-in, the treatment will actually cost about $90,000 per year.
Yes, Medicare will reimburse a large portion of that cost, but it adds up to an estimated $2 billion per year for about 85,000 patients. This will probably spur increases to Medicare premiums, among other economic consequences for the health care system.
We’ll probably have to wait for an FDA approval decision before we know where donanemab will be priced.
Logistical challenges could be a rate-limiting step
Ask anyone who’s tried to see a neurologist recently, and they’ll tell you that the wait for a new patient appointment is months long. The shortage of neurologists in the United States is already a crisis, and there are even fewer cognitive neurologists. How long will patients be forced to wait for their diagnosis?
Many geriatricians will get comfortable prescribing these drugs, but will our already overburdened primary care providers have the bandwidth to do the same? It’s a tall order.
A new world of Alzheimer’s treatments also means that the infrastructure of our health care systems will need to be ramped up. Lecanemab infusions are administered every 2 weeks and donanemab every 4 weeks. Infusion centers will need to accommodate a lot more patients. And those patients will need frequent brain scans, so neuroimaging centers will need to increase their capacity to perform many more brain MRI and PET scans.
Antiamyloid drugs: An exciting first step
The bottom line is that these drugs aren’t the Alzheimer’s holy grail: An accessible treatment that could stop the disease in its tracks or reverse cognitive impairment. They are, however, a very promising breakthrough.
Yes, there are a ton of kinks to work out here, but this is an exciting start. Alzheimer’s research is entering a renaissance era that will hopefully bring more groundbreaking developments. Better biomarkers to facilitate faster, easier diagnosis. More drugs that go beyond amyloid proteins for their therapeutic targets. Treatments for later-stage disease. Drugs that prevent dementia altogether.
Ultimately, these new antiamyloid beta drugs are an exciting indication that we will eventually have a toolkit of Alzheimer’s drugs to choose from. For now, we’ve taken a solid step forward and there is ample reason to be hopeful for the future.
Dr. Croll is assistant professor of neurology at Temple University, Philadelphia. She reported no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
With the Food and Drug Administration’s full stamp of approval in hand, Leqembi (lecanemab) is poised to catapult us into a new era of treatment for Alzheimer’s disease. And now that the donanemab trial data are out, there’s another antiamyloid drug waiting in the wings.
To finally have true disease-modifying therapies for Alzheimer’s disease is a massive step forward for a field that’s been plagued with disappointment. But these drugs come with serious concerns and unknowns. They will require complex decision-making, putting doctors, patients, and their families in a medical quandary.
Striking the right balance between cautious optimism and realistic expectations will be a formidable challenge.
Managing patient and family expectations
These drugs are no magic bullet. They slow down the dementia’s progression, buying patients more time (on the order of months) before they begin to experience significant worsening. We’ll need a lot more information from research and clinical experience before we can understand how meaningful that treatment effect is. Right now, it is unclear whether eligible patients and their families will even perceive tangible differences.
In the CLARITY-AD trial, participants on lecanemab experienced a 27% slowing in the rate of cognitive decline over 18 months. Donanemab was shown to slow decline in memory and cognition by about 35% over the same time frame in the TRAILBLAZER-ALZ 2 trial. That translates to more time for patients and their families to enjoy independence, maintain normal life, and stave off the most distressing parts of the disease.
But what happens after 18 months of treatment – will the treatment effect magnify or dissipate? How much time are we really buying in the long run? Counseling patients and their families is made all the more difficult when the answers to important questions like these remain to be seen.
Only a sliver of Alzheimer’s patients are current candidates
The fact is that most patients living with Alzheimer’s disease will not qualify for treatment with these drugs. Lecanemab is approved for people with early-stage disease, meaning their dementia is mild or they have mild cognitive impairment, which is a precursor to full-blown Alzheimer’s disease. Of the 6 million people in the United States living with Alzheimer’s, about 1.5 million are estimated to fall into that category. We can expect to see a similar qualifier for donanemab if it receives FDA approval, especially because that trial suggested a more pronounced treatment effect for patients in the earliest stages of the disease.
Even if a patient hits the sweet spot where they have just enough cognitive impairment, but not too much, they aren’t technically therapeutic candidates until prerequisite testing confirms amyloid protein accumulation in the brain via PET scan or cerebrospinal fluid analysis.
Even then, the FDA’s boxed warning for lecanemab recommends that patients undergo genetic testing for the apo E4 mutation to identify those at a particularly high risk for severe adverse effects including brain bleeding and swelling. This recommendation is not unreasonable considering that 15% of the Alzheimer’s population has two copies of the apo E4 mutation and fall into that high-risk group.
Significant risks
Antiamyloid drugs are well-known to cause serious side effects. In the lecanemab trial, 13% of participants receiving Leqembi experienced brain swelling (vs. 2% of participants receiving placebo) and 17% of participants had brain bleeding (vs. 9% of participants on placebo). In the donanemab trial, brain bleeding occurred in 31.4% of participants on the drug (vs. 13.6% on placebo) and swelling occurred in 24% (vs. 2.1% receiving placebo). Thankfully, in both trials, most of these adverse events did not produce significant symptoms, but in rare cases these events caused severe or catastrophic neurologic injury, including death.
How can we best guide patients and their families to weigh the uncertain benefits against potentially serious risks? We can start by considering the patient characteristics most likely to portend increased risk for serious side effects: apo E4 mutations, blood thinner use, and the presence of microhemorrhages on brain imaging. But after that, we’re left with a lot of uncertainty in terms of which patients are most likely to see meaningful clinical improvements from the drug and unknown factors that may increase the risks of treatment.
A costly therapy
Medicare plans to cover 80% of lecanemab’s steep cost of $26,500 per year. Still, that will leave many patients with a hefty copay, potentially over $6,000 per year. But that only scratches the surface. Consider the frequent medical visits, repeated brain scans, laboratory tests, and infusion center appointments. It’s been estimated that all-in, the treatment will actually cost about $90,000 per year.
Yes, Medicare will reimburse a large portion of that cost, but it adds up to an estimated $2 billion per year for about 85,000 patients. This will probably spur increases to Medicare premiums, among other economic consequences for the health care system.
We’ll probably have to wait for an FDA approval decision before we know where donanemab will be priced.
Logistical challenges could be a rate-limiting step
Ask anyone who’s tried to see a neurologist recently, and they’ll tell you that the wait for a new patient appointment is months long. The shortage of neurologists in the United States is already a crisis, and there are even fewer cognitive neurologists. How long will patients be forced to wait for their diagnosis?
Many geriatricians will get comfortable prescribing these drugs, but will our already overburdened primary care providers have the bandwidth to do the same? It’s a tall order.
A new world of Alzheimer’s treatments also means that the infrastructure of our health care systems will need to be ramped up. Lecanemab infusions are administered every 2 weeks and donanemab every 4 weeks. Infusion centers will need to accommodate a lot more patients. And those patients will need frequent brain scans, so neuroimaging centers will need to increase their capacity to perform many more brain MRI and PET scans.
Antiamyloid drugs: An exciting first step
The bottom line is that these drugs aren’t the Alzheimer’s holy grail: An accessible treatment that could stop the disease in its tracks or reverse cognitive impairment. They are, however, a very promising breakthrough.
Yes, there are a ton of kinks to work out here, but this is an exciting start. Alzheimer’s research is entering a renaissance era that will hopefully bring more groundbreaking developments. Better biomarkers to facilitate faster, easier diagnosis. More drugs that go beyond amyloid proteins for their therapeutic targets. Treatments for later-stage disease. Drugs that prevent dementia altogether.
Ultimately, these new antiamyloid beta drugs are an exciting indication that we will eventually have a toolkit of Alzheimer’s drugs to choose from. For now, we’ve taken a solid step forward and there is ample reason to be hopeful for the future.
Dr. Croll is assistant professor of neurology at Temple University, Philadelphia. She reported no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
With the Food and Drug Administration’s full stamp of approval in hand, Leqembi (lecanemab) is poised to catapult us into a new era of treatment for Alzheimer’s disease. And now that the donanemab trial data are out, there’s another antiamyloid drug waiting in the wings.
To finally have true disease-modifying therapies for Alzheimer’s disease is a massive step forward for a field that’s been plagued with disappointment. But these drugs come with serious concerns and unknowns. They will require complex decision-making, putting doctors, patients, and their families in a medical quandary.
Striking the right balance between cautious optimism and realistic expectations will be a formidable challenge.
Managing patient and family expectations
These drugs are no magic bullet. They slow down the dementia’s progression, buying patients more time (on the order of months) before they begin to experience significant worsening. We’ll need a lot more information from research and clinical experience before we can understand how meaningful that treatment effect is. Right now, it is unclear whether eligible patients and their families will even perceive tangible differences.
In the CLARITY-AD trial, participants on lecanemab experienced a 27% slowing in the rate of cognitive decline over 18 months. Donanemab was shown to slow decline in memory and cognition by about 35% over the same time frame in the TRAILBLAZER-ALZ 2 trial. That translates to more time for patients and their families to enjoy independence, maintain normal life, and stave off the most distressing parts of the disease.
But what happens after 18 months of treatment – will the treatment effect magnify or dissipate? How much time are we really buying in the long run? Counseling patients and their families is made all the more difficult when the answers to important questions like these remain to be seen.
Only a sliver of Alzheimer’s patients are current candidates
The fact is that most patients living with Alzheimer’s disease will not qualify for treatment with these drugs. Lecanemab is approved for people with early-stage disease, meaning their dementia is mild or they have mild cognitive impairment, which is a precursor to full-blown Alzheimer’s disease. Of the 6 million people in the United States living with Alzheimer’s, about 1.5 million are estimated to fall into that category. We can expect to see a similar qualifier for donanemab if it receives FDA approval, especially because that trial suggested a more pronounced treatment effect for patients in the earliest stages of the disease.
Even if a patient hits the sweet spot where they have just enough cognitive impairment, but not too much, they aren’t technically therapeutic candidates until prerequisite testing confirms amyloid protein accumulation in the brain via PET scan or cerebrospinal fluid analysis.
Even then, the FDA’s boxed warning for lecanemab recommends that patients undergo genetic testing for the apo E4 mutation to identify those at a particularly high risk for severe adverse effects including brain bleeding and swelling. This recommendation is not unreasonable considering that 15% of the Alzheimer’s population has two copies of the apo E4 mutation and fall into that high-risk group.
Significant risks
Antiamyloid drugs are well-known to cause serious side effects. In the lecanemab trial, 13% of participants receiving Leqembi experienced brain swelling (vs. 2% of participants receiving placebo) and 17% of participants had brain bleeding (vs. 9% of participants on placebo). In the donanemab trial, brain bleeding occurred in 31.4% of participants on the drug (vs. 13.6% on placebo) and swelling occurred in 24% (vs. 2.1% receiving placebo). Thankfully, in both trials, most of these adverse events did not produce significant symptoms, but in rare cases these events caused severe or catastrophic neurologic injury, including death.
How can we best guide patients and their families to weigh the uncertain benefits against potentially serious risks? We can start by considering the patient characteristics most likely to portend increased risk for serious side effects: apo E4 mutations, blood thinner use, and the presence of microhemorrhages on brain imaging. But after that, we’re left with a lot of uncertainty in terms of which patients are most likely to see meaningful clinical improvements from the drug and unknown factors that may increase the risks of treatment.
A costly therapy
Medicare plans to cover 80% of lecanemab’s steep cost of $26,500 per year. Still, that will leave many patients with a hefty copay, potentially over $6,000 per year. But that only scratches the surface. Consider the frequent medical visits, repeated brain scans, laboratory tests, and infusion center appointments. It’s been estimated that all-in, the treatment will actually cost about $90,000 per year.
Yes, Medicare will reimburse a large portion of that cost, but it adds up to an estimated $2 billion per year for about 85,000 patients. This will probably spur increases to Medicare premiums, among other economic consequences for the health care system.
We’ll probably have to wait for an FDA approval decision before we know where donanemab will be priced.
Logistical challenges could be a rate-limiting step
Ask anyone who’s tried to see a neurologist recently, and they’ll tell you that the wait for a new patient appointment is months long. The shortage of neurologists in the United States is already a crisis, and there are even fewer cognitive neurologists. How long will patients be forced to wait for their diagnosis?
Many geriatricians will get comfortable prescribing these drugs, but will our already overburdened primary care providers have the bandwidth to do the same? It’s a tall order.
A new world of Alzheimer’s treatments also means that the infrastructure of our health care systems will need to be ramped up. Lecanemab infusions are administered every 2 weeks and donanemab every 4 weeks. Infusion centers will need to accommodate a lot more patients. And those patients will need frequent brain scans, so neuroimaging centers will need to increase their capacity to perform many more brain MRI and PET scans.
Antiamyloid drugs: An exciting first step
The bottom line is that these drugs aren’t the Alzheimer’s holy grail: An accessible treatment that could stop the disease in its tracks or reverse cognitive impairment. They are, however, a very promising breakthrough.
Yes, there are a ton of kinks to work out here, but this is an exciting start. Alzheimer’s research is entering a renaissance era that will hopefully bring more groundbreaking developments. Better biomarkers to facilitate faster, easier diagnosis. More drugs that go beyond amyloid proteins for their therapeutic targets. Treatments for later-stage disease. Drugs that prevent dementia altogether.
Ultimately, these new antiamyloid beta drugs are an exciting indication that we will eventually have a toolkit of Alzheimer’s drugs to choose from. For now, we’ve taken a solid step forward and there is ample reason to be hopeful for the future.
Dr. Croll is assistant professor of neurology at Temple University, Philadelphia. She reported no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
Could GLP-1 receptor agonists ease knee osteoarthritis pain, slow progression?
Could glucagon-like peptide-1 receptor agonists, such as liraglutide and semaglutide, also be potential disease-modifying treatments for knee osteoarthritis (KOA)?
Weight loss is recommended for patients with KOA, and GLP-1 receptor agonists are approved for weight loss. New early research suggests these drugs might have a disease-modifying effect for KOA.
Three recently published studies investigated this:
- The LOSEIT phase 4, randomized controlled trial of liraglutide vs. placebo in patients with obesity/overweight and KOA.
- A large observational study out of China in patients with KOA and type 2 diabetes.
- A preclinical trial of liraglutide in mouse models of KOA.
The preclinical trial and the observational study report promising results, and the lack of KOA pain relief in patients in the phase 4 trial may be explained by the trial design. Three other trials are in the works.
This news organization invited two researchers and two outside experts to discuss these studies and potential future treatment of KOA with GLP-1 receptor agonists.
The big picture, as seen by two experts
The GLP-1 receptor agonists liraglutide (Victoza) and semaglutide (Ozempic) are approved for type 2 diabetes, and, in higher doses, liraglutide (Saxenda) and semaglutide (Wegovy) are approved for weight loss in patients with obesity (or overweight with comorbidities), and given as weekly injections.
Victoza and Saxenda are expected to come off patent in December 2023, and in 2026, respectively.
Lauren King, MD, PhD, a rheumatologist and clinician scientist who was not involved with the recent investigational studies of GLP-1 receptor agonists for KOA, noted that obesity is the most important, guideline-recommended, modifiable risk factor for KOA.
“In people with overweight and obesity, losing weight can improve knee osteoarthritis symptoms, and some evidence supports that it may also slow joint structural changes,” Dr. King, of the department of medicine at the University of Toronto, said in an interview.
Large trials of GLP-1 receptor agonists in people with overweight and obesity, such as the STEP trials of semaglutide, she noted, “provide evidence that these medications are safe and effective, facilitating clinically relevant and sustained weight loss.”
Further research is needed, she said, to better understand disease-modifying effects of GLP-1 receptor agonists in patients with KOA.
Similarly, W. Timothy Garvey, MD, professor in the department of nutrition sciences at the University of Alabama at Birmingham and director of the UAB Diabetes Research Center, who was not involved with this research, noted that weight loss improves KOA symptoms.
Dr. Garvey was lead investigator in the STEP 5 trial of semaglutide and lead author of the American Association of Clinical Endocrinologists 2016 Obesity Management guidelines.
“The question is whether these GLP-1 receptor agonists have anything to offer over and above weight loss per se, and we don’t know for sure,” he said.
They “do have anti-inflammatory actions,” and “there are GLP-1 receptors in locations where you think GLP-1 receptor agonism may help inflammation in the knee, in joints, and in other tissues,” he noted.
He looks forward to results of the phase 3 trial of semaglutide in patients with KOA, expected this fall.
Three published studies
LOSEIT: RCT of liraglutide for pain and weight control in KOA
Henrik Rindel Gudbergsen, MD, PhD, and colleagues published results of the only randomized controlled trial of a GLP-1 receptor agonist (liraglutide, Saxenda) vs. placebo in patients with overweight/obesity and KOA, the LOSEIT trial.
All patients first entered an 8-week, pre-randomization phase where they had strict caloric restriction (and ate meal replacements) and lost at least 5% of their initial weight. They also had less knee pain at the end of this phase.
Then they were randomly assigned to receive 3 mg liraglutide or placebo daily injections for 1 year.
From randomization until week 52, the liraglutide group had greater mean weight loss than the placebo group (but this was < 5% of their weight). They did not have greater reduction in knee pain than patients in the placebo group.
“Our interpretation was that dieting results in weight loss and diminishes knee pain (which we knew), and that the impact of liraglutide following severe calorie restriction and weight loss and improvement of pain was limited,” Dr. Gudbergsen, a physician and associate professor at The Parker Institute, University of Copenhagen, told this news organization.
“That was the surprise for us as investigators,” he said, “and, I assume, why Novo Nordisk is now pursuing the investigation of semaglutide for KOA, as this is expected to create a larger effect on body weight and knee symptoms.”
The weight loss was about 12.5 kg (27.5 pounds) prior to randomization, and the subsequent weight loss with liraglutide was about 2.8 kg (6 pounds; about 4% of their weight). “Thus, it could seem that the participants’ potential for weight loss as well as symptom reduction was fully exploited in the pre–random assignment dietary intervention period,” according to the researchers.
“It seems highly relevant to use liraglutide or semaglutide for patients impacted by obesity and KOA, as it is in line with guidelines suggesting weight loss for this group,” Dr. Gudbergsen said. “However, whether liraglutide and/or semaglutide, acting via an anti-inflammatory effect, for example, has an added positive impact on cartilage quality remains to be clarified,” he said.
Others who were not involved in this study suggest that the lack of pain-reduction benefit with liraglutide vs. placebo can be explained by the short-term use of liraglutide (1 year), small weight loss (< 5%), and systemic rather than intraarticular injection.
The LOSEIT trial design “is problematic and could not provide a confirmative conclusion,” Hongyi Zhu, MD, PhD, Shanghai Sixth People’s Hospital, China, and colleagues wrote in their observational study. The small weight loss of < 5% in the liraglutide group may explain why the pain relief was not better than with placebo. A longer study duration with significant weight loss/maintenance may be needed, they noted.
Francis Berenbaum, MD, PhD, senior author of a preclinical study of liraglutide, said that in the LOSEIT trial, “daily systemic injections of liraglutide did not ameliorate OA-related pain, probably because of poor access and hence poor local concentrations of liraglutide in the knee joint.”
Dr. Berenbaum is professor of rheumatology at Sorbonne University and director of the department of rheumatology at AP-HP Saint-Antoine Hospital in Paris. He is cofounder and CEO of 4Moving Biotech (a subsidiary of 4P Pharma, an innovator accelerator biotech company), which is testing liraglutide for KOA.
In experiments in mice, systemic injections of liraglutide did not lead to high enough concentration in synovial fluid to show efficacy for pain relief, he told this news organization. “In order to get the direct effect of liraglutide, it should be injected intraarticularly,” he said.
Observational study of patients with diabetes and KOA
Dr. Zhu and colleagues recently published results of the first clinical investigation of long-term effects of GLP-1 receptor agonists on KOA in patients with comorbid type 2 diabetes.
They analyzed data from a subset of patients with KOA and type 2 diabetes from the Shanghai Osteoarthritis Cohort, including 233 patients who received a GLP-1 receptor agonist (semaglutide, liraglutide, or dulaglutide [Trulicity]) for at least 2 years and 1,574 patients who did not receive this therapy.
The patients had a mean weight of 66 kg (145 pounds), a mean body mass index of 27 kg/m2, and a mean A1c of 7.3%.
“According to conventional wisdom, a weight change greater than 5% is considered clinically relevant for KOA,” the researchers wrote. They found that patients had substantial weight loss after GLP-1 receptor agonist therapy.
The primary outcome, the incidence of knee surgery, was lower in the patients who received a GLP-1 receptor agonist than in the other patients (1.7% vs. 5.9%; adjusted P = .014).
Patients who received a GLP-1 receptor agonist also had greater improvements in secondary outcomes than did other patients, including pain subscale scores and cartilage-loss velocity of the medial femorotibial joint in patients with predominantly lateral OA.
“The effects of GLP-1 receptor agonists on arthritic knees were largely mediated by weight loss instead of glycemic control,” Dr. Zhu and colleagues reported.
They concluded that with long-enough treatment, “GLP-1 receptor agonist therapies might be disease-modifying for KOA patients with comorbid [type 2 diabetes mellitus].”
They called for further research to elucidate the effects of GLP-1 receptor agonists on the disease process, joint structure, and patient-reported outcomes of OA.
Dr. Garvey noted that “whether your BMI is 30 or 40, if there are complications, that tells you that degree of adiposity is sufficient to impair health.” So, if a patient in southeast China has a BMI of 27 kg/m2 and has osteoarthritis, he or she could still benefit from weight loss, he said.
Liraglutide and pain-related behavior in mouse models of OA
Dr. Berenbaum and colleagues reported that liraglutide alleviated pain-related behavior in sodium monoiodoacetate mouse models of KOA.
In addition, liraglutide had anti-inflammatory and anticatabolic effects in synovial fluid from the knees of six patients with OA of varying severity.
The researchers analyzed generic liraglutide (from Hybio Pharmaceuticals, Shenzhen, China) and nongeneric liraglutide (from Novo Nordisk, Bagsværd, Denmark).
They found that “when injected intra-articularly, liraglutide blunts the inflammatory process that is present in OA synovial tissue, explaining the acute analgesic effect,” Dr. Berenbaum said.
“Liraglutide could be a game-changer,” he said, “by demonstrating not only an effect on joint structures like synovial tissue and cartilage, but also on symptoms in a short-term period.”
Dr. Garvey said the symptom improvements after intrasynovial infusion of liraglutide in this trial were “impressive.” This study “adds credence to the hypothesis that these GLP-1 receptor agonists could have effects above and beyond weight loss,” he said.
Two trials near completion, one is upcoming
Phase 1 and 2 trials of 4P-004
“We are now in a phase 1 clinical trial [of 4P-004/liraglutide] in patients suffering from knee OA and should start a large phase 2 trial next year,” said Dr. Berenbaum.
The phase 1 LASARE trial, sponsored by 4Moving Biotech, planned to enroll 32 patients with KOA.
The primary outcome is safety and tolerability of single IA administration of 4P-004 at escalating doses in patients with KOA. Secondary outcomes include plasma concentration of liraglutide when administered this way.
Phase 3 trial of semaglutide for KOA
Novo Nordisk is performing a phase 3 study, “Effect of Subcutaneous Semaglutide 2.4 mg Once-weekly Compared to Placebo in Subjects With Obesity and Knee Osteoarthritis,” with an expected enrollment of 407 patients with KOA and estimated trial completion in September.
Eligible patients were aged 18 and older, with BMI > 30 kg/m2 and KOA with Kellgren-Lawrence grades 2 or 3. The co-primary outcomes are change in body weight and change in WOMAC pain score, from baseline to 68 weeks.
The LOSEIT trial was supported by Novo Nordisk and the Cambridge Weight Plan. The observational study in China was supported by the Shanghai Shenkang Hospital Development Centre, the Clinical Research Plan of SHDC, and the National Natural Science Foundation of China. The preclinical trial was supported by 4P Pharma/4Moving Biotech.
Dr. Berenbaum is CEO of 4Moving Biotech and chair of the scientific advisory board of 4P Pharma. He has received personal fees from 4P Pharma as well as numerous other pharmaceutical companies. Dr. Garvey has reported being a consultant to Boehringer Ingelheim, Novo Nordisk, Eli Lilly, Merck, Fractyl Health, and Alnylam Pharmaceuticals, and reported being an investigator for studies sponsored by Novo Nordisk, Eli Lilly, Pfizer, and Epitomee. Dr. Gudbergsen, Dr. King, and Dr. Zhu report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Could glucagon-like peptide-1 receptor agonists, such as liraglutide and semaglutide, also be potential disease-modifying treatments for knee osteoarthritis (KOA)?
Weight loss is recommended for patients with KOA, and GLP-1 receptor agonists are approved for weight loss. New early research suggests these drugs might have a disease-modifying effect for KOA.
Three recently published studies investigated this:
- The LOSEIT phase 4, randomized controlled trial of liraglutide vs. placebo in patients with obesity/overweight and KOA.
- A large observational study out of China in patients with KOA and type 2 diabetes.
- A preclinical trial of liraglutide in mouse models of KOA.
The preclinical trial and the observational study report promising results, and the lack of KOA pain relief in patients in the phase 4 trial may be explained by the trial design. Three other trials are in the works.
This news organization invited two researchers and two outside experts to discuss these studies and potential future treatment of KOA with GLP-1 receptor agonists.
The big picture, as seen by two experts
The GLP-1 receptor agonists liraglutide (Victoza) and semaglutide (Ozempic) are approved for type 2 diabetes, and, in higher doses, liraglutide (Saxenda) and semaglutide (Wegovy) are approved for weight loss in patients with obesity (or overweight with comorbidities), and given as weekly injections.
Victoza and Saxenda are expected to come off patent in December 2023, and in 2026, respectively.
Lauren King, MD, PhD, a rheumatologist and clinician scientist who was not involved with the recent investigational studies of GLP-1 receptor agonists for KOA, noted that obesity is the most important, guideline-recommended, modifiable risk factor for KOA.
“In people with overweight and obesity, losing weight can improve knee osteoarthritis symptoms, and some evidence supports that it may also slow joint structural changes,” Dr. King, of the department of medicine at the University of Toronto, said in an interview.
Large trials of GLP-1 receptor agonists in people with overweight and obesity, such as the STEP trials of semaglutide, she noted, “provide evidence that these medications are safe and effective, facilitating clinically relevant and sustained weight loss.”
Further research is needed, she said, to better understand disease-modifying effects of GLP-1 receptor agonists in patients with KOA.
Similarly, W. Timothy Garvey, MD, professor in the department of nutrition sciences at the University of Alabama at Birmingham and director of the UAB Diabetes Research Center, who was not involved with this research, noted that weight loss improves KOA symptoms.
Dr. Garvey was lead investigator in the STEP 5 trial of semaglutide and lead author of the American Association of Clinical Endocrinologists 2016 Obesity Management guidelines.
“The question is whether these GLP-1 receptor agonists have anything to offer over and above weight loss per se, and we don’t know for sure,” he said.
They “do have anti-inflammatory actions,” and “there are GLP-1 receptors in locations where you think GLP-1 receptor agonism may help inflammation in the knee, in joints, and in other tissues,” he noted.
He looks forward to results of the phase 3 trial of semaglutide in patients with KOA, expected this fall.
Three published studies
LOSEIT: RCT of liraglutide for pain and weight control in KOA
Henrik Rindel Gudbergsen, MD, PhD, and colleagues published results of the only randomized controlled trial of a GLP-1 receptor agonist (liraglutide, Saxenda) vs. placebo in patients with overweight/obesity and KOA, the LOSEIT trial.
All patients first entered an 8-week, pre-randomization phase where they had strict caloric restriction (and ate meal replacements) and lost at least 5% of their initial weight. They also had less knee pain at the end of this phase.
Then they were randomly assigned to receive 3 mg liraglutide or placebo daily injections for 1 year.
From randomization until week 52, the liraglutide group had greater mean weight loss than the placebo group (but this was < 5% of their weight). They did not have greater reduction in knee pain than patients in the placebo group.
“Our interpretation was that dieting results in weight loss and diminishes knee pain (which we knew), and that the impact of liraglutide following severe calorie restriction and weight loss and improvement of pain was limited,” Dr. Gudbergsen, a physician and associate professor at The Parker Institute, University of Copenhagen, told this news organization.
“That was the surprise for us as investigators,” he said, “and, I assume, why Novo Nordisk is now pursuing the investigation of semaglutide for KOA, as this is expected to create a larger effect on body weight and knee symptoms.”
The weight loss was about 12.5 kg (27.5 pounds) prior to randomization, and the subsequent weight loss with liraglutide was about 2.8 kg (6 pounds; about 4% of their weight). “Thus, it could seem that the participants’ potential for weight loss as well as symptom reduction was fully exploited in the pre–random assignment dietary intervention period,” according to the researchers.
“It seems highly relevant to use liraglutide or semaglutide for patients impacted by obesity and KOA, as it is in line with guidelines suggesting weight loss for this group,” Dr. Gudbergsen said. “However, whether liraglutide and/or semaglutide, acting via an anti-inflammatory effect, for example, has an added positive impact on cartilage quality remains to be clarified,” he said.
Others who were not involved in this study suggest that the lack of pain-reduction benefit with liraglutide vs. placebo can be explained by the short-term use of liraglutide (1 year), small weight loss (< 5%), and systemic rather than intraarticular injection.
The LOSEIT trial design “is problematic and could not provide a confirmative conclusion,” Hongyi Zhu, MD, PhD, Shanghai Sixth People’s Hospital, China, and colleagues wrote in their observational study. The small weight loss of < 5% in the liraglutide group may explain why the pain relief was not better than with placebo. A longer study duration with significant weight loss/maintenance may be needed, they noted.
Francis Berenbaum, MD, PhD, senior author of a preclinical study of liraglutide, said that in the LOSEIT trial, “daily systemic injections of liraglutide did not ameliorate OA-related pain, probably because of poor access and hence poor local concentrations of liraglutide in the knee joint.”
Dr. Berenbaum is professor of rheumatology at Sorbonne University and director of the department of rheumatology at AP-HP Saint-Antoine Hospital in Paris. He is cofounder and CEO of 4Moving Biotech (a subsidiary of 4P Pharma, an innovator accelerator biotech company), which is testing liraglutide for KOA.
In experiments in mice, systemic injections of liraglutide did not lead to high enough concentration in synovial fluid to show efficacy for pain relief, he told this news organization. “In order to get the direct effect of liraglutide, it should be injected intraarticularly,” he said.
Observational study of patients with diabetes and KOA
Dr. Zhu and colleagues recently published results of the first clinical investigation of long-term effects of GLP-1 receptor agonists on KOA in patients with comorbid type 2 diabetes.
They analyzed data from a subset of patients with KOA and type 2 diabetes from the Shanghai Osteoarthritis Cohort, including 233 patients who received a GLP-1 receptor agonist (semaglutide, liraglutide, or dulaglutide [Trulicity]) for at least 2 years and 1,574 patients who did not receive this therapy.
The patients had a mean weight of 66 kg (145 pounds), a mean body mass index of 27 kg/m2, and a mean A1c of 7.3%.
“According to conventional wisdom, a weight change greater than 5% is considered clinically relevant for KOA,” the researchers wrote. They found that patients had substantial weight loss after GLP-1 receptor agonist therapy.
The primary outcome, the incidence of knee surgery, was lower in the patients who received a GLP-1 receptor agonist than in the other patients (1.7% vs. 5.9%; adjusted P = .014).
Patients who received a GLP-1 receptor agonist also had greater improvements in secondary outcomes than did other patients, including pain subscale scores and cartilage-loss velocity of the medial femorotibial joint in patients with predominantly lateral OA.
“The effects of GLP-1 receptor agonists on arthritic knees were largely mediated by weight loss instead of glycemic control,” Dr. Zhu and colleagues reported.
They concluded that with long-enough treatment, “GLP-1 receptor agonist therapies might be disease-modifying for KOA patients with comorbid [type 2 diabetes mellitus].”
They called for further research to elucidate the effects of GLP-1 receptor agonists on the disease process, joint structure, and patient-reported outcomes of OA.
Dr. Garvey noted that “whether your BMI is 30 or 40, if there are complications, that tells you that degree of adiposity is sufficient to impair health.” So, if a patient in southeast China has a BMI of 27 kg/m2 and has osteoarthritis, he or she could still benefit from weight loss, he said.
Liraglutide and pain-related behavior in mouse models of OA
Dr. Berenbaum and colleagues reported that liraglutide alleviated pain-related behavior in sodium monoiodoacetate mouse models of KOA.
In addition, liraglutide had anti-inflammatory and anticatabolic effects in synovial fluid from the knees of six patients with OA of varying severity.
The researchers analyzed generic liraglutide (from Hybio Pharmaceuticals, Shenzhen, China) and nongeneric liraglutide (from Novo Nordisk, Bagsværd, Denmark).
They found that “when injected intra-articularly, liraglutide blunts the inflammatory process that is present in OA synovial tissue, explaining the acute analgesic effect,” Dr. Berenbaum said.
“Liraglutide could be a game-changer,” he said, “by demonstrating not only an effect on joint structures like synovial tissue and cartilage, but also on symptoms in a short-term period.”
Dr. Garvey said the symptom improvements after intrasynovial infusion of liraglutide in this trial were “impressive.” This study “adds credence to the hypothesis that these GLP-1 receptor agonists could have effects above and beyond weight loss,” he said.
Two trials near completion, one is upcoming
Phase 1 and 2 trials of 4P-004
“We are now in a phase 1 clinical trial [of 4P-004/liraglutide] in patients suffering from knee OA and should start a large phase 2 trial next year,” said Dr. Berenbaum.
The phase 1 LASARE trial, sponsored by 4Moving Biotech, planned to enroll 32 patients with KOA.
The primary outcome is safety and tolerability of single IA administration of 4P-004 at escalating doses in patients with KOA. Secondary outcomes include plasma concentration of liraglutide when administered this way.
Phase 3 trial of semaglutide for KOA
Novo Nordisk is performing a phase 3 study, “Effect of Subcutaneous Semaglutide 2.4 mg Once-weekly Compared to Placebo in Subjects With Obesity and Knee Osteoarthritis,” with an expected enrollment of 407 patients with KOA and estimated trial completion in September.
Eligible patients were aged 18 and older, with BMI > 30 kg/m2 and KOA with Kellgren-Lawrence grades 2 or 3. The co-primary outcomes are change in body weight and change in WOMAC pain score, from baseline to 68 weeks.
The LOSEIT trial was supported by Novo Nordisk and the Cambridge Weight Plan. The observational study in China was supported by the Shanghai Shenkang Hospital Development Centre, the Clinical Research Plan of SHDC, and the National Natural Science Foundation of China. The preclinical trial was supported by 4P Pharma/4Moving Biotech.
Dr. Berenbaum is CEO of 4Moving Biotech and chair of the scientific advisory board of 4P Pharma. He has received personal fees from 4P Pharma as well as numerous other pharmaceutical companies. Dr. Garvey has reported being a consultant to Boehringer Ingelheim, Novo Nordisk, Eli Lilly, Merck, Fractyl Health, and Alnylam Pharmaceuticals, and reported being an investigator for studies sponsored by Novo Nordisk, Eli Lilly, Pfizer, and Epitomee. Dr. Gudbergsen, Dr. King, and Dr. Zhu report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Could glucagon-like peptide-1 receptor agonists, such as liraglutide and semaglutide, also be potential disease-modifying treatments for knee osteoarthritis (KOA)?
Weight loss is recommended for patients with KOA, and GLP-1 receptor agonists are approved for weight loss. New early research suggests these drugs might have a disease-modifying effect for KOA.
Three recently published studies investigated this:
- The LOSEIT phase 4, randomized controlled trial of liraglutide vs. placebo in patients with obesity/overweight and KOA.
- A large observational study out of China in patients with KOA and type 2 diabetes.
- A preclinical trial of liraglutide in mouse models of KOA.
The preclinical trial and the observational study report promising results, and the lack of KOA pain relief in patients in the phase 4 trial may be explained by the trial design. Three other trials are in the works.
This news organization invited two researchers and two outside experts to discuss these studies and potential future treatment of KOA with GLP-1 receptor agonists.
The big picture, as seen by two experts
The GLP-1 receptor agonists liraglutide (Victoza) and semaglutide (Ozempic) are approved for type 2 diabetes, and, in higher doses, liraglutide (Saxenda) and semaglutide (Wegovy) are approved for weight loss in patients with obesity (or overweight with comorbidities), and given as weekly injections.
Victoza and Saxenda are expected to come off patent in December 2023, and in 2026, respectively.
Lauren King, MD, PhD, a rheumatologist and clinician scientist who was not involved with the recent investigational studies of GLP-1 receptor agonists for KOA, noted that obesity is the most important, guideline-recommended, modifiable risk factor for KOA.
“In people with overweight and obesity, losing weight can improve knee osteoarthritis symptoms, and some evidence supports that it may also slow joint structural changes,” Dr. King, of the department of medicine at the University of Toronto, said in an interview.
Large trials of GLP-1 receptor agonists in people with overweight and obesity, such as the STEP trials of semaglutide, she noted, “provide evidence that these medications are safe and effective, facilitating clinically relevant and sustained weight loss.”
Further research is needed, she said, to better understand disease-modifying effects of GLP-1 receptor agonists in patients with KOA.
Similarly, W. Timothy Garvey, MD, professor in the department of nutrition sciences at the University of Alabama at Birmingham and director of the UAB Diabetes Research Center, who was not involved with this research, noted that weight loss improves KOA symptoms.
Dr. Garvey was lead investigator in the STEP 5 trial of semaglutide and lead author of the American Association of Clinical Endocrinologists 2016 Obesity Management guidelines.
“The question is whether these GLP-1 receptor agonists have anything to offer over and above weight loss per se, and we don’t know for sure,” he said.
They “do have anti-inflammatory actions,” and “there are GLP-1 receptors in locations where you think GLP-1 receptor agonism may help inflammation in the knee, in joints, and in other tissues,” he noted.
He looks forward to results of the phase 3 trial of semaglutide in patients with KOA, expected this fall.
Three published studies
LOSEIT: RCT of liraglutide for pain and weight control in KOA
Henrik Rindel Gudbergsen, MD, PhD, and colleagues published results of the only randomized controlled trial of a GLP-1 receptor agonist (liraglutide, Saxenda) vs. placebo in patients with overweight/obesity and KOA, the LOSEIT trial.
All patients first entered an 8-week, pre-randomization phase where they had strict caloric restriction (and ate meal replacements) and lost at least 5% of their initial weight. They also had less knee pain at the end of this phase.
Then they were randomly assigned to receive 3 mg liraglutide or placebo daily injections for 1 year.
From randomization until week 52, the liraglutide group had greater mean weight loss than the placebo group (but this was < 5% of their weight). They did not have greater reduction in knee pain than patients in the placebo group.
“Our interpretation was that dieting results in weight loss and diminishes knee pain (which we knew), and that the impact of liraglutide following severe calorie restriction and weight loss and improvement of pain was limited,” Dr. Gudbergsen, a physician and associate professor at The Parker Institute, University of Copenhagen, told this news organization.
“That was the surprise for us as investigators,” he said, “and, I assume, why Novo Nordisk is now pursuing the investigation of semaglutide for KOA, as this is expected to create a larger effect on body weight and knee symptoms.”
The weight loss was about 12.5 kg (27.5 pounds) prior to randomization, and the subsequent weight loss with liraglutide was about 2.8 kg (6 pounds; about 4% of their weight). “Thus, it could seem that the participants’ potential for weight loss as well as symptom reduction was fully exploited in the pre–random assignment dietary intervention period,” according to the researchers.
“It seems highly relevant to use liraglutide or semaglutide for patients impacted by obesity and KOA, as it is in line with guidelines suggesting weight loss for this group,” Dr. Gudbergsen said. “However, whether liraglutide and/or semaglutide, acting via an anti-inflammatory effect, for example, has an added positive impact on cartilage quality remains to be clarified,” he said.
Others who were not involved in this study suggest that the lack of pain-reduction benefit with liraglutide vs. placebo can be explained by the short-term use of liraglutide (1 year), small weight loss (< 5%), and systemic rather than intraarticular injection.
The LOSEIT trial design “is problematic and could not provide a confirmative conclusion,” Hongyi Zhu, MD, PhD, Shanghai Sixth People’s Hospital, China, and colleagues wrote in their observational study. The small weight loss of < 5% in the liraglutide group may explain why the pain relief was not better than with placebo. A longer study duration with significant weight loss/maintenance may be needed, they noted.
Francis Berenbaum, MD, PhD, senior author of a preclinical study of liraglutide, said that in the LOSEIT trial, “daily systemic injections of liraglutide did not ameliorate OA-related pain, probably because of poor access and hence poor local concentrations of liraglutide in the knee joint.”
Dr. Berenbaum is professor of rheumatology at Sorbonne University and director of the department of rheumatology at AP-HP Saint-Antoine Hospital in Paris. He is cofounder and CEO of 4Moving Biotech (a subsidiary of 4P Pharma, an innovator accelerator biotech company), which is testing liraglutide for KOA.
In experiments in mice, systemic injections of liraglutide did not lead to high enough concentration in synovial fluid to show efficacy for pain relief, he told this news organization. “In order to get the direct effect of liraglutide, it should be injected intraarticularly,” he said.
Observational study of patients with diabetes and KOA
Dr. Zhu and colleagues recently published results of the first clinical investigation of long-term effects of GLP-1 receptor agonists on KOA in patients with comorbid type 2 diabetes.
They analyzed data from a subset of patients with KOA and type 2 diabetes from the Shanghai Osteoarthritis Cohort, including 233 patients who received a GLP-1 receptor agonist (semaglutide, liraglutide, or dulaglutide [Trulicity]) for at least 2 years and 1,574 patients who did not receive this therapy.
The patients had a mean weight of 66 kg (145 pounds), a mean body mass index of 27 kg/m2, and a mean A1c of 7.3%.
“According to conventional wisdom, a weight change greater than 5% is considered clinically relevant for KOA,” the researchers wrote. They found that patients had substantial weight loss after GLP-1 receptor agonist therapy.
The primary outcome, the incidence of knee surgery, was lower in the patients who received a GLP-1 receptor agonist than in the other patients (1.7% vs. 5.9%; adjusted P = .014).
Patients who received a GLP-1 receptor agonist also had greater improvements in secondary outcomes than did other patients, including pain subscale scores and cartilage-loss velocity of the medial femorotibial joint in patients with predominantly lateral OA.
“The effects of GLP-1 receptor agonists on arthritic knees were largely mediated by weight loss instead of glycemic control,” Dr. Zhu and colleagues reported.
They concluded that with long-enough treatment, “GLP-1 receptor agonist therapies might be disease-modifying for KOA patients with comorbid [type 2 diabetes mellitus].”
They called for further research to elucidate the effects of GLP-1 receptor agonists on the disease process, joint structure, and patient-reported outcomes of OA.
Dr. Garvey noted that “whether your BMI is 30 or 40, if there are complications, that tells you that degree of adiposity is sufficient to impair health.” So, if a patient in southeast China has a BMI of 27 kg/m2 and has osteoarthritis, he or she could still benefit from weight loss, he said.
Liraglutide and pain-related behavior in mouse models of OA
Dr. Berenbaum and colleagues reported that liraglutide alleviated pain-related behavior in sodium monoiodoacetate mouse models of KOA.
In addition, liraglutide had anti-inflammatory and anticatabolic effects in synovial fluid from the knees of six patients with OA of varying severity.
The researchers analyzed generic liraglutide (from Hybio Pharmaceuticals, Shenzhen, China) and nongeneric liraglutide (from Novo Nordisk, Bagsværd, Denmark).
They found that “when injected intra-articularly, liraglutide blunts the inflammatory process that is present in OA synovial tissue, explaining the acute analgesic effect,” Dr. Berenbaum said.
“Liraglutide could be a game-changer,” he said, “by demonstrating not only an effect on joint structures like synovial tissue and cartilage, but also on symptoms in a short-term period.”
Dr. Garvey said the symptom improvements after intrasynovial infusion of liraglutide in this trial were “impressive.” This study “adds credence to the hypothesis that these GLP-1 receptor agonists could have effects above and beyond weight loss,” he said.
Two trials near completion, one is upcoming
Phase 1 and 2 trials of 4P-004
“We are now in a phase 1 clinical trial [of 4P-004/liraglutide] in patients suffering from knee OA and should start a large phase 2 trial next year,” said Dr. Berenbaum.
The phase 1 LASARE trial, sponsored by 4Moving Biotech, planned to enroll 32 patients with KOA.
The primary outcome is safety and tolerability of single IA administration of 4P-004 at escalating doses in patients with KOA. Secondary outcomes include plasma concentration of liraglutide when administered this way.
Phase 3 trial of semaglutide for KOA
Novo Nordisk is performing a phase 3 study, “Effect of Subcutaneous Semaglutide 2.4 mg Once-weekly Compared to Placebo in Subjects With Obesity and Knee Osteoarthritis,” with an expected enrollment of 407 patients with KOA and estimated trial completion in September.
Eligible patients were aged 18 and older, with BMI > 30 kg/m2 and KOA with Kellgren-Lawrence grades 2 or 3. The co-primary outcomes are change in body weight and change in WOMAC pain score, from baseline to 68 weeks.
The LOSEIT trial was supported by Novo Nordisk and the Cambridge Weight Plan. The observational study in China was supported by the Shanghai Shenkang Hospital Development Centre, the Clinical Research Plan of SHDC, and the National Natural Science Foundation of China. The preclinical trial was supported by 4P Pharma/4Moving Biotech.
Dr. Berenbaum is CEO of 4Moving Biotech and chair of the scientific advisory board of 4P Pharma. He has received personal fees from 4P Pharma as well as numerous other pharmaceutical companies. Dr. Garvey has reported being a consultant to Boehringer Ingelheim, Novo Nordisk, Eli Lilly, Merck, Fractyl Health, and Alnylam Pharmaceuticals, and reported being an investigator for studies sponsored by Novo Nordisk, Eli Lilly, Pfizer, and Epitomee. Dr. Gudbergsen, Dr. King, and Dr. Zhu report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Kidnapping and treatment risks come with medical tourism
In March 2023, four “medical tourists” from South Carolina who were seeking health care in Mexico were kidnapped by a drug cartel. Two were killed when they were trapped in a shootout. One of them was scheduled for tummy-tuck surgery, and others were seeking cheaper prescription drugs.
The news reached Bruce Hermann, MD, a plastic surgeon in Denton, Tex., who brought up the incident in a segment of his podcast, “Nip Talk,” in which he talked about the risks of medical tourism. But violence in foreign countries isn’t Dr. Hermann’s primary concern.
“Being the victim of a crime is lower down the risk strata,” Dr. Hermann said in an interview. “A bigger concern is the lack of regulations of doctors and facilities in countries like Mexico.”
Some employers pay for treatment at certain foreign clinics, and Blue Shield of California’s HMO plan, Access Baja, covers care in certain clinics in Mexico’s Baja peninsula. But U.S. health insurance generally does not cover medical tourism.
Despite its popularity, medical tourism is not siphoning off a significant number of patients from U.S. doctors, with the possible exception of plastic surgery. One study found that medical tourism accounts for less than 2% of U.S. spending on noncosmetic health care.
Still, as many as 1.2 million Americans travel to Mexico each year seeking health care at lower costs, particularly dental care, bariatric surgery, and cosmetic procedures.
Physicians such as Dr. Hermann see the results when things go awry. Dr. Hermann said when he takes calls at a nearby level II trauma center, he sees, on average, one patient a month with complications from plastic surgeries performed abroad.
Patients tell Dr. Hermann they often had little preoperative time with the surgeons, and some may not even see their surgeon. They have to fly back home just days after their procedures, so complications that typically arise later are missed, he said.
Who opts for medical tourism?
There are few statistics on the number of medical tourists or the clinical problems they have. Josef Woodman, CEO of Patients Beyond Borders, a medical tourism consultancy in Chapel Hill, N.C., has developed a profile of medical tourism that is based on his close contacts within the industry.
Mr. Woodman said the vast majority of U.S. medical tourists go to Mexico, which accounts for an estimated 1 million to 1.2 million medical visitors a year. He said Costa Rica is another popular destination, followed by other Latin American countries and some in the Far East, the Middle East, and Eastern Europe.
Mr. Woodman estimates that dental treatments make up 65% of all medical tourism. Cosmetic procedures come in a distant second, at 15%. Cosmetic procedures can be expensive and are rarely covered by insurance. They can be performed at half the price abroad, he said.
According to Mr. Woodman, other significant fields for medical tourism are orthopedics, which accounts for 5% of all visits, and bariatrics, with 3%-5%. Hip and knee replacements are expensive, and in the case of bariatrics, U.S. insurers often deny coverage, he said.
People also go abroad for fertility care and organ transplants, and one Jamaica company even offered dialysis vacations for U.S. tourists.
On the other hand, medical tourism does not work well for cancer treatments, because cancer care involves long periods of treatment and cannot be completed in a trip or two, Mr. Woodman said. “The media also plays up major procedures like open heart surgery, but they are in fact very rare,” he added.
What patients are looking for
Medical tourists typically pay 50%-70% of what they would pay in the United States for the same procedure, Mr. Woodman said. Prices are lower because patients pay in cash, foreign wages are lower, and expenses such as malpractice insurance are much lower.
“Many medical tourists don’t have a choice,” Mr. Woodman said. “They cannot afford the U.S. price for the procedure, even if they have health insurance, because they often have a high deductible.” In one study, the majority of medical tourists to Mexico reported an income of $25,000-$50,000 a year.
That same study also found that the average age of medical tourists was just under 65. These older patients often come for a vacation. “A sizable number of medical tourists are looking for surgery plus a vacation, a tummy-tuck and a stay at an all-inclusive resort,” said Steven P. Davison, MD, DDS, a plastic surgeon in Washington, who has written on the phenomenon.
Another large group of medical tourists are immigrants to the United States who want surgery in their country of origin, such as Brazil or Iran, Dr. Davison said, perhaps because they feel more comfortable or have family members who can act as caregivers. He said some go to places that have expertise in a certain procedure.
“For instance, they get their hair transplantation done in Turkey because surgeons there have the expertise and it’s cheaper than in the United States,” Dr. Davison said.
Arranging the trip
Patients often find foreign providers through online brokers who can arrange the flight, hotel, clinic, and physician. Brokers are not unbiased because they are usually paid by the clinic. Mr. Woodman said this arrangement works when the broker can offer a wide variety of options but not when the broker represents just a few clinics.
Mr. Woodman said patients could conceivably make their own arrangements without a broker, and some do so. “All the tools are on the Internet,” he said. “However, many people don’t trust themselves to do this work.”
Even for patients who depend on brokers, Mr. Woodman advises verifying the quality of the clinic and its doctors before signing on. Most countries have online lists of registered doctors, and patients seeking health care can research complaints against a doctor.
There is no insurance that patients can have to guard against the risks of medical tourism, Mr. Woodman said. “When you could get it, it was prohibitively expensive,” he said. “You can get travel insurance, but that just covers peripheral problems, such as flight cancellations, accidental injury, and emergency care. It has nothing to do with problems stemming from planned procedures.
“Some clinics and hospitals serving medical tourists provide warranties on their work,” he added. “However, plastic surgery clinics are less likely to offer warranties, because patients are so frequently dissatisfied.”
How things can go wrong
Mr. Woodman said medical tourists may often receive substandard care when they select a provider who offers unusually large savings, such as 80% off the U.S. price. “Those providers are likely cutting corners to get that kind of savings, and you should stay away from them,” he said.
Even when receiving care at an excellent clinic, patients can get infections if postoperative requirements are not followed, according to Darrick E. Antell, MD, a plastic surgeon in New York, who has treated medical tourists upon their return to the United States.
Typically, patients are told to stay in their rooms for several days after the operation, but friends may push them to go out and have fun, he said. Sometimes patients choose exotic arrangements that place them at risk for infection. As an example, Dr. Antell mentioned a broker who offered a safari in combination with plastic surgery.
Dr. Hermann said tummy tucks are riskier because they involve large incisions, and many such patients are overweight, which impairs healing. “Tummy-tuck patients need a longer recovery, and they often have more discomfort than other plastic surgery,” he said.
When things go wrong, medical tourists have few legal protections. “They usually don’t go to an accredited facility, there is no credentialing of physicians, and their ability to sue is extremely limited,” Dr. Davison said. Patients would have to return to the foreign country and hire a lawyer there, and even then, it is harder to win a case and to receive an award as high as in the United States, he added.
Dealing with follow-ups
An inherent flaw with medical tourism, Dr. Antell said, is that patients typically go back home before postoperative care is fully completed. “They may stay just a few days after surgery, and then fly back home,” he said.
“Patients who have complex operations abroad should stay for 8-10 days to have a proper follow-up,” he said. “But they fly back early, which can also lead to getting pulmonary embolisms on the flight.
“A checkup right after surgery doesn’t uncover many complications, because these tend to occur 7-12 days after surgery,” Dr. Hermann said.
“If they come to me within 3 months after surgery, I charge an upfront fee just to see them, because it takes an hour of my time,” Dr. Davison said. “Then I will take care of acute emergency, such as taking out an infected implant.”
Hermann said many patients wait too long to have their complications treated in the United States. “They may first try calling their doctor in Mexico, who tells them to take some antibiotics or something,” he said. “So when they finally do seek care, the infection is pretty far along.”
What U.S. doctors can do
Patients rarely tell their U.S. doctors that they are planning a trip to a foreign country to undergo medical treatment, even though they have to request a copy of their medical records for the foreign doctor, Mr. Woodman said.
Dr. Hermann said only one of his patients told him she was planning to go aboard for plastic surgery. “She was a young mom, and I tried to talk her out of it,” he said. “I don’t know what happened because she didn’t come back.”
Dr. Hermann said doctors should assume that they won’t be able to change their patients’ minds, and they should try to help their patients make the best of it.
“They should insist on seeing the doctor ahead of time and make sure they get along with them,” he said. “Ask for credentialing of the doctor and the facility, and stay there several weeks post op. But they’re probably not going to do all of this.”
A version of this article first appeared on Medscape.com.
In March 2023, four “medical tourists” from South Carolina who were seeking health care in Mexico were kidnapped by a drug cartel. Two were killed when they were trapped in a shootout. One of them was scheduled for tummy-tuck surgery, and others were seeking cheaper prescription drugs.
The news reached Bruce Hermann, MD, a plastic surgeon in Denton, Tex., who brought up the incident in a segment of his podcast, “Nip Talk,” in which he talked about the risks of medical tourism. But violence in foreign countries isn’t Dr. Hermann’s primary concern.
“Being the victim of a crime is lower down the risk strata,” Dr. Hermann said in an interview. “A bigger concern is the lack of regulations of doctors and facilities in countries like Mexico.”
Some employers pay for treatment at certain foreign clinics, and Blue Shield of California’s HMO plan, Access Baja, covers care in certain clinics in Mexico’s Baja peninsula. But U.S. health insurance generally does not cover medical tourism.
Despite its popularity, medical tourism is not siphoning off a significant number of patients from U.S. doctors, with the possible exception of plastic surgery. One study found that medical tourism accounts for less than 2% of U.S. spending on noncosmetic health care.
Still, as many as 1.2 million Americans travel to Mexico each year seeking health care at lower costs, particularly dental care, bariatric surgery, and cosmetic procedures.
Physicians such as Dr. Hermann see the results when things go awry. Dr. Hermann said when he takes calls at a nearby level II trauma center, he sees, on average, one patient a month with complications from plastic surgeries performed abroad.
Patients tell Dr. Hermann they often had little preoperative time with the surgeons, and some may not even see their surgeon. They have to fly back home just days after their procedures, so complications that typically arise later are missed, he said.
Who opts for medical tourism?
There are few statistics on the number of medical tourists or the clinical problems they have. Josef Woodman, CEO of Patients Beyond Borders, a medical tourism consultancy in Chapel Hill, N.C., has developed a profile of medical tourism that is based on his close contacts within the industry.
Mr. Woodman said the vast majority of U.S. medical tourists go to Mexico, which accounts for an estimated 1 million to 1.2 million medical visitors a year. He said Costa Rica is another popular destination, followed by other Latin American countries and some in the Far East, the Middle East, and Eastern Europe.
Mr. Woodman estimates that dental treatments make up 65% of all medical tourism. Cosmetic procedures come in a distant second, at 15%. Cosmetic procedures can be expensive and are rarely covered by insurance. They can be performed at half the price abroad, he said.
According to Mr. Woodman, other significant fields for medical tourism are orthopedics, which accounts for 5% of all visits, and bariatrics, with 3%-5%. Hip and knee replacements are expensive, and in the case of bariatrics, U.S. insurers often deny coverage, he said.
People also go abroad for fertility care and organ transplants, and one Jamaica company even offered dialysis vacations for U.S. tourists.
On the other hand, medical tourism does not work well for cancer treatments, because cancer care involves long periods of treatment and cannot be completed in a trip or two, Mr. Woodman said. “The media also plays up major procedures like open heart surgery, but they are in fact very rare,” he added.
What patients are looking for
Medical tourists typically pay 50%-70% of what they would pay in the United States for the same procedure, Mr. Woodman said. Prices are lower because patients pay in cash, foreign wages are lower, and expenses such as malpractice insurance are much lower.
“Many medical tourists don’t have a choice,” Mr. Woodman said. “They cannot afford the U.S. price for the procedure, even if they have health insurance, because they often have a high deductible.” In one study, the majority of medical tourists to Mexico reported an income of $25,000-$50,000 a year.
That same study also found that the average age of medical tourists was just under 65. These older patients often come for a vacation. “A sizable number of medical tourists are looking for surgery plus a vacation, a tummy-tuck and a stay at an all-inclusive resort,” said Steven P. Davison, MD, DDS, a plastic surgeon in Washington, who has written on the phenomenon.
Another large group of medical tourists are immigrants to the United States who want surgery in their country of origin, such as Brazil or Iran, Dr. Davison said, perhaps because they feel more comfortable or have family members who can act as caregivers. He said some go to places that have expertise in a certain procedure.
“For instance, they get their hair transplantation done in Turkey because surgeons there have the expertise and it’s cheaper than in the United States,” Dr. Davison said.
Arranging the trip
Patients often find foreign providers through online brokers who can arrange the flight, hotel, clinic, and physician. Brokers are not unbiased because they are usually paid by the clinic. Mr. Woodman said this arrangement works when the broker can offer a wide variety of options but not when the broker represents just a few clinics.
Mr. Woodman said patients could conceivably make their own arrangements without a broker, and some do so. “All the tools are on the Internet,” he said. “However, many people don’t trust themselves to do this work.”
Even for patients who depend on brokers, Mr. Woodman advises verifying the quality of the clinic and its doctors before signing on. Most countries have online lists of registered doctors, and patients seeking health care can research complaints against a doctor.
There is no insurance that patients can have to guard against the risks of medical tourism, Mr. Woodman said. “When you could get it, it was prohibitively expensive,” he said. “You can get travel insurance, but that just covers peripheral problems, such as flight cancellations, accidental injury, and emergency care. It has nothing to do with problems stemming from planned procedures.
“Some clinics and hospitals serving medical tourists provide warranties on their work,” he added. “However, plastic surgery clinics are less likely to offer warranties, because patients are so frequently dissatisfied.”
How things can go wrong
Mr. Woodman said medical tourists may often receive substandard care when they select a provider who offers unusually large savings, such as 80% off the U.S. price. “Those providers are likely cutting corners to get that kind of savings, and you should stay away from them,” he said.
Even when receiving care at an excellent clinic, patients can get infections if postoperative requirements are not followed, according to Darrick E. Antell, MD, a plastic surgeon in New York, who has treated medical tourists upon their return to the United States.
Typically, patients are told to stay in their rooms for several days after the operation, but friends may push them to go out and have fun, he said. Sometimes patients choose exotic arrangements that place them at risk for infection. As an example, Dr. Antell mentioned a broker who offered a safari in combination with plastic surgery.
Dr. Hermann said tummy tucks are riskier because they involve large incisions, and many such patients are overweight, which impairs healing. “Tummy-tuck patients need a longer recovery, and they often have more discomfort than other plastic surgery,” he said.
When things go wrong, medical tourists have few legal protections. “They usually don’t go to an accredited facility, there is no credentialing of physicians, and their ability to sue is extremely limited,” Dr. Davison said. Patients would have to return to the foreign country and hire a lawyer there, and even then, it is harder to win a case and to receive an award as high as in the United States, he added.
Dealing with follow-ups
An inherent flaw with medical tourism, Dr. Antell said, is that patients typically go back home before postoperative care is fully completed. “They may stay just a few days after surgery, and then fly back home,” he said.
“Patients who have complex operations abroad should stay for 8-10 days to have a proper follow-up,” he said. “But they fly back early, which can also lead to getting pulmonary embolisms on the flight.
“A checkup right after surgery doesn’t uncover many complications, because these tend to occur 7-12 days after surgery,” Dr. Hermann said.
“If they come to me within 3 months after surgery, I charge an upfront fee just to see them, because it takes an hour of my time,” Dr. Davison said. “Then I will take care of acute emergency, such as taking out an infected implant.”
Hermann said many patients wait too long to have their complications treated in the United States. “They may first try calling their doctor in Mexico, who tells them to take some antibiotics or something,” he said. “So when they finally do seek care, the infection is pretty far along.”
What U.S. doctors can do
Patients rarely tell their U.S. doctors that they are planning a trip to a foreign country to undergo medical treatment, even though they have to request a copy of their medical records for the foreign doctor, Mr. Woodman said.
Dr. Hermann said only one of his patients told him she was planning to go aboard for plastic surgery. “She was a young mom, and I tried to talk her out of it,” he said. “I don’t know what happened because she didn’t come back.”
Dr. Hermann said doctors should assume that they won’t be able to change their patients’ minds, and they should try to help their patients make the best of it.
“They should insist on seeing the doctor ahead of time and make sure they get along with them,” he said. “Ask for credentialing of the doctor and the facility, and stay there several weeks post op. But they’re probably not going to do all of this.”
A version of this article first appeared on Medscape.com.
In March 2023, four “medical tourists” from South Carolina who were seeking health care in Mexico were kidnapped by a drug cartel. Two were killed when they were trapped in a shootout. One of them was scheduled for tummy-tuck surgery, and others were seeking cheaper prescription drugs.
The news reached Bruce Hermann, MD, a plastic surgeon in Denton, Tex., who brought up the incident in a segment of his podcast, “Nip Talk,” in which he talked about the risks of medical tourism. But violence in foreign countries isn’t Dr. Hermann’s primary concern.
“Being the victim of a crime is lower down the risk strata,” Dr. Hermann said in an interview. “A bigger concern is the lack of regulations of doctors and facilities in countries like Mexico.”
Some employers pay for treatment at certain foreign clinics, and Blue Shield of California’s HMO plan, Access Baja, covers care in certain clinics in Mexico’s Baja peninsula. But U.S. health insurance generally does not cover medical tourism.
Despite its popularity, medical tourism is not siphoning off a significant number of patients from U.S. doctors, with the possible exception of plastic surgery. One study found that medical tourism accounts for less than 2% of U.S. spending on noncosmetic health care.
Still, as many as 1.2 million Americans travel to Mexico each year seeking health care at lower costs, particularly dental care, bariatric surgery, and cosmetic procedures.
Physicians such as Dr. Hermann see the results when things go awry. Dr. Hermann said when he takes calls at a nearby level II trauma center, he sees, on average, one patient a month with complications from plastic surgeries performed abroad.
Patients tell Dr. Hermann they often had little preoperative time with the surgeons, and some may not even see their surgeon. They have to fly back home just days after their procedures, so complications that typically arise later are missed, he said.
Who opts for medical tourism?
There are few statistics on the number of medical tourists or the clinical problems they have. Josef Woodman, CEO of Patients Beyond Borders, a medical tourism consultancy in Chapel Hill, N.C., has developed a profile of medical tourism that is based on his close contacts within the industry.
Mr. Woodman said the vast majority of U.S. medical tourists go to Mexico, which accounts for an estimated 1 million to 1.2 million medical visitors a year. He said Costa Rica is another popular destination, followed by other Latin American countries and some in the Far East, the Middle East, and Eastern Europe.
Mr. Woodman estimates that dental treatments make up 65% of all medical tourism. Cosmetic procedures come in a distant second, at 15%. Cosmetic procedures can be expensive and are rarely covered by insurance. They can be performed at half the price abroad, he said.
According to Mr. Woodman, other significant fields for medical tourism are orthopedics, which accounts for 5% of all visits, and bariatrics, with 3%-5%. Hip and knee replacements are expensive, and in the case of bariatrics, U.S. insurers often deny coverage, he said.
People also go abroad for fertility care and organ transplants, and one Jamaica company even offered dialysis vacations for U.S. tourists.
On the other hand, medical tourism does not work well for cancer treatments, because cancer care involves long periods of treatment and cannot be completed in a trip or two, Mr. Woodman said. “The media also plays up major procedures like open heart surgery, but they are in fact very rare,” he added.
What patients are looking for
Medical tourists typically pay 50%-70% of what they would pay in the United States for the same procedure, Mr. Woodman said. Prices are lower because patients pay in cash, foreign wages are lower, and expenses such as malpractice insurance are much lower.
“Many medical tourists don’t have a choice,” Mr. Woodman said. “They cannot afford the U.S. price for the procedure, even if they have health insurance, because they often have a high deductible.” In one study, the majority of medical tourists to Mexico reported an income of $25,000-$50,000 a year.
That same study also found that the average age of medical tourists was just under 65. These older patients often come for a vacation. “A sizable number of medical tourists are looking for surgery plus a vacation, a tummy-tuck and a stay at an all-inclusive resort,” said Steven P. Davison, MD, DDS, a plastic surgeon in Washington, who has written on the phenomenon.
Another large group of medical tourists are immigrants to the United States who want surgery in their country of origin, such as Brazil or Iran, Dr. Davison said, perhaps because they feel more comfortable or have family members who can act as caregivers. He said some go to places that have expertise in a certain procedure.
“For instance, they get their hair transplantation done in Turkey because surgeons there have the expertise and it’s cheaper than in the United States,” Dr. Davison said.
Arranging the trip
Patients often find foreign providers through online brokers who can arrange the flight, hotel, clinic, and physician. Brokers are not unbiased because they are usually paid by the clinic. Mr. Woodman said this arrangement works when the broker can offer a wide variety of options but not when the broker represents just a few clinics.
Mr. Woodman said patients could conceivably make their own arrangements without a broker, and some do so. “All the tools are on the Internet,” he said. “However, many people don’t trust themselves to do this work.”
Even for patients who depend on brokers, Mr. Woodman advises verifying the quality of the clinic and its doctors before signing on. Most countries have online lists of registered doctors, and patients seeking health care can research complaints against a doctor.
There is no insurance that patients can have to guard against the risks of medical tourism, Mr. Woodman said. “When you could get it, it was prohibitively expensive,” he said. “You can get travel insurance, but that just covers peripheral problems, such as flight cancellations, accidental injury, and emergency care. It has nothing to do with problems stemming from planned procedures.
“Some clinics and hospitals serving medical tourists provide warranties on their work,” he added. “However, plastic surgery clinics are less likely to offer warranties, because patients are so frequently dissatisfied.”
How things can go wrong
Mr. Woodman said medical tourists may often receive substandard care when they select a provider who offers unusually large savings, such as 80% off the U.S. price. “Those providers are likely cutting corners to get that kind of savings, and you should stay away from them,” he said.
Even when receiving care at an excellent clinic, patients can get infections if postoperative requirements are not followed, according to Darrick E. Antell, MD, a plastic surgeon in New York, who has treated medical tourists upon their return to the United States.
Typically, patients are told to stay in their rooms for several days after the operation, but friends may push them to go out and have fun, he said. Sometimes patients choose exotic arrangements that place them at risk for infection. As an example, Dr. Antell mentioned a broker who offered a safari in combination with plastic surgery.
Dr. Hermann said tummy tucks are riskier because they involve large incisions, and many such patients are overweight, which impairs healing. “Tummy-tuck patients need a longer recovery, and they often have more discomfort than other plastic surgery,” he said.
When things go wrong, medical tourists have few legal protections. “They usually don’t go to an accredited facility, there is no credentialing of physicians, and their ability to sue is extremely limited,” Dr. Davison said. Patients would have to return to the foreign country and hire a lawyer there, and even then, it is harder to win a case and to receive an award as high as in the United States, he added.
Dealing with follow-ups
An inherent flaw with medical tourism, Dr. Antell said, is that patients typically go back home before postoperative care is fully completed. “They may stay just a few days after surgery, and then fly back home,” he said.
“Patients who have complex operations abroad should stay for 8-10 days to have a proper follow-up,” he said. “But they fly back early, which can also lead to getting pulmonary embolisms on the flight.
“A checkup right after surgery doesn’t uncover many complications, because these tend to occur 7-12 days after surgery,” Dr. Hermann said.
“If they come to me within 3 months after surgery, I charge an upfront fee just to see them, because it takes an hour of my time,” Dr. Davison said. “Then I will take care of acute emergency, such as taking out an infected implant.”
Hermann said many patients wait too long to have their complications treated in the United States. “They may first try calling their doctor in Mexico, who tells them to take some antibiotics or something,” he said. “So when they finally do seek care, the infection is pretty far along.”
What U.S. doctors can do
Patients rarely tell their U.S. doctors that they are planning a trip to a foreign country to undergo medical treatment, even though they have to request a copy of their medical records for the foreign doctor, Mr. Woodman said.
Dr. Hermann said only one of his patients told him she was planning to go aboard for plastic surgery. “She was a young mom, and I tried to talk her out of it,” he said. “I don’t know what happened because she didn’t come back.”
Dr. Hermann said doctors should assume that they won’t be able to change their patients’ minds, and they should try to help their patients make the best of it.
“They should insist on seeing the doctor ahead of time and make sure they get along with them,” he said. “Ask for credentialing of the doctor and the facility, and stay there several weeks post op. But they’re probably not going to do all of this.”
A version of this article first appeared on Medscape.com.
Chaperones for physical exams? Doctors and patients weigh in
News of physicians accused or convicted of sexually inappropriate behavior toward patients during medical exams has been frequent recently. And patient advocates have brought up the fact that many patients are uncomfortable during sensitive exams.
As a result, more
For some institutions, the movement has been toward mandating chaperones. Chaperones can be present for any examination of what are often referred to as sensitive areas, such as breasts, genitalia, and the perianal area. For example, Yale University Health requires patients to use a medical staff member as a chaperone for all sensitive exams except in emergency situations. UCLA Health, on the other hand, allows any adult or child aged 12 years or older to decline a chaperone if they wish to do so. Michigan Medicine provides a chaperone on request, although a patient can opt out.
Many physicians fully support the concept. “If a patient requests a chaperone I think we all, as practitioners, should honor that request respectfully,” said Shieva Ghofrany, MD, an ob.gyn. practicing in Stamford, Conn., and cofounder of tribecalledv, a women’s health knowledge platform. “It’s a clear sign a patient wants to ensure that they feel safe and comfortable.”
However, using a chaperone can be challenging for many primary care physicians and specialists in terms of logistics. Should a chaperone’s job be purely observational? Or should the chaperone also be a medical professional who assists during the procedure? How, specifically, should a chaperone perform their duty during an exam? Where should the chaperone stand? What about legal and ethical ramifications?
Who should be a chaperone?
The role of a chaperone is sometimes vaguely defined. It’s logical that a chaperone should have sufficient medical knowledge. Could it be a medical student? Should it be a nurse or another doctor? Would a patient representative suffice even if they aren’t a direct-care clinician?
There are no set standards about who can assume the role. However, the American Medical Association recommends that a patient chaperone be an authorized member of a health care team. For many patients, this is vital.
“As a survivor of sexual assault by an ob.gyn. while I was pregnant, I can assure you that the heinous crime committed against me and hundreds of other women would have been prevented had there been another medical professional in the room,” said Laurie Kanyok, founder of a dance company in New York City, whose former doctor was recently convicted of federal sex abuse charges and is scheduled to be sentenced in July 2023. “The chaperone should be a physician assistant, nurse or medical professional. Qualified professionals better understand the nature and protocols of an exam.”
For children and adolescents, allowing a parent to be a chaperone might be a logical and comforting choice. However, a new British study found that it’s not appropriate.
Study authors Rebecca Jane Moon and Justin Huw Davies looked at pubertal staging–related exams and contend that a chaperone should always be impartial, and a parent’s presence could conceivably open up a physician to false charges of misconduct.
The solution: A parent should have the option be present during any physical exam of their child, with a medical professional additionally acting as chaperone. This can also work for any patient who wishes to bring a trusted friend or family member to their medical appointments.
How does a chaperoned exam work?
A research team from the Medical College of Wisconsin and Louisiana State University Health conducted a recent systematic review of patient and clinician perspectives on carrying out a chaperoned urology exam. The review found that the protocol could use improvement. For example, it was reported that over one-quarter of patients didn’t feel comfortable asking for a chaperone if they were not offered one. “Patients should not have to request this,” said Diana Londoño, MD, a board-certified urologist and assistant clinical professor at City of Hope National Medical Center in Duarte, Calif.
The researchers also found that 93% of female patients preferred a chaperone of the same gender, whereas male patients were split on this issue. Key duties of a chaperone should be to ensure privacy, help interpret instructions from doctor to patient, and continually clarify that consent is being requested as a sensitive exam proceeds, the researchers report. Although clinical practice standards for chaperones aren’t uniform, keeping these important points in mind can easily be adopted by any health care provider.
Many doctors do follow their own set guidelines. “Often, we have our medical assistants be our chaperones,” explained Dr. Ghofrany. “Ideally they ‘room’ the patient – take vitals and ask the patient what specific concerns they would like to address. This helps with exam set-up.
“For example, if a patient has a breast concern versus a concern about their genital area, this would change what drapes are used. The medical assistant would then stand near me if they are helping with a Pap smear or cultures, or they may stand by the patient’s head at the bedside and offer support if needed. Some patients want to hold someone’s hand during an uncomfortable exam.”
The issue of positioning is important. A patient may feel very uneasy if it appears that the chaperone is looking at their body from the doctor’s angle, negating the point of reassurance. The key is to explain before an exam exactly how a chaperone is needed during the exam itself and whether the chaperone is a medical assistant or nurse.
“Chaperones usually stand next to me on either side or on the side of the patient facing me, depending on if they are immediately assisting me or helping the patient,” said Dr. Londoño. If a chaperone will be moving back and forth during the exam, that should be conveyed as well. For virtual visits, a chaperone can act as a third party and be present on a split screen, a process Michigan Medicine uses.
It’s also important to inform patients that a chaperone can step in and stop an exam at any point, both at the patient’s request or because of any observation of inappropriate physician behavior. Understanding this can help reluctant patients feel less worry.
When Christian Miller was diagnosed with a sleep disorder, his doctor suggested having a chaperone present during his physical exams. At first, he was apprehensive but then found the experience to be very positive. “Having someone there with me gave me an extra measure of assurance that nothing untoward would happen,” he said. “I found that having a chaperone was reassuring. The chaperone was not intrusive but stood close enough to ensure everything went smoothly and respectfully.”
Do chaperones help protect physicians legally?
Some states mandate that chaperones be present by patient request during sensitive exams, such as Texas and Oregon. For the most part, though, physicians have no legal obligation to offer chaperones, although it could be in their best interest to do so.
Ob.gyn. is one of the most litigated specialties, for example, so these physicians may find chaperones can play a key role in averting lawsuits. According to data from Physicians’ Insurance, having a reliable witness in an exam room can mitigate claims, and doctors should follow a clear practice policy regarding chaperones for all patients to further reduce liability.
Another advantage to having a chaperone present: protection for a doctor against a problematic patient. The risk for assault or aggression during an exam can of course go both ways. It’s important to be able to prove any patient misconduct through a witness, and a chaperone can lessen the prospect of violence in any form. “Having a chaperone in an exam room is about protecting the doctors as well as the patient,” said Ms. Kanyok.
A chaperone can also defuse ethical dilemmas. Consider a patient who refuses to allow a chaperone in an exam, but a chaperone is required by the physician’s health care organization. Asking a patient to state their reasons for wanting privacy with the chaperone present before the actual exam can help document the patient’s wish respectfully as well as protect a physician and organization from any potential liability if the patient refuses the exam altogether or if an exception for the patient is made.
Making a chaperone policy work best
- Have your staff inform patients of a medical chaperone policy at the time an appointment is made. Have fact sheets available for the patient before appointments fully outlining your policy.
- Inform the chaperone fully about the details of the exam and the patient’s case prior to the exam, in accordance with HIPAA.
- The chaperone can introduce themselves to the patient in the waiting room or exam room before you enter the room. The chaperone can go over the policy again verbally and answer any questions the patient may have initially. You can then clarify whether the patient understands your chaperone policy when you come in.
- Document, document, document. Write down who the chaperone was for each exam in patient notes and note the details of any interactions that are significant, such as patient questions or conflicts.
- Practice respect, above all. “A patient’s safety and level of comfort should be prioritized,” said Ms. Kanyok.
A version of this article first appeared on Medscape.com.
News of physicians accused or convicted of sexually inappropriate behavior toward patients during medical exams has been frequent recently. And patient advocates have brought up the fact that many patients are uncomfortable during sensitive exams.
As a result, more
For some institutions, the movement has been toward mandating chaperones. Chaperones can be present for any examination of what are often referred to as sensitive areas, such as breasts, genitalia, and the perianal area. For example, Yale University Health requires patients to use a medical staff member as a chaperone for all sensitive exams except in emergency situations. UCLA Health, on the other hand, allows any adult or child aged 12 years or older to decline a chaperone if they wish to do so. Michigan Medicine provides a chaperone on request, although a patient can opt out.
Many physicians fully support the concept. “If a patient requests a chaperone I think we all, as practitioners, should honor that request respectfully,” said Shieva Ghofrany, MD, an ob.gyn. practicing in Stamford, Conn., and cofounder of tribecalledv, a women’s health knowledge platform. “It’s a clear sign a patient wants to ensure that they feel safe and comfortable.”
However, using a chaperone can be challenging for many primary care physicians and specialists in terms of logistics. Should a chaperone’s job be purely observational? Or should the chaperone also be a medical professional who assists during the procedure? How, specifically, should a chaperone perform their duty during an exam? Where should the chaperone stand? What about legal and ethical ramifications?
Who should be a chaperone?
The role of a chaperone is sometimes vaguely defined. It’s logical that a chaperone should have sufficient medical knowledge. Could it be a medical student? Should it be a nurse or another doctor? Would a patient representative suffice even if they aren’t a direct-care clinician?
There are no set standards about who can assume the role. However, the American Medical Association recommends that a patient chaperone be an authorized member of a health care team. For many patients, this is vital.
“As a survivor of sexual assault by an ob.gyn. while I was pregnant, I can assure you that the heinous crime committed against me and hundreds of other women would have been prevented had there been another medical professional in the room,” said Laurie Kanyok, founder of a dance company in New York City, whose former doctor was recently convicted of federal sex abuse charges and is scheduled to be sentenced in July 2023. “The chaperone should be a physician assistant, nurse or medical professional. Qualified professionals better understand the nature and protocols of an exam.”
For children and adolescents, allowing a parent to be a chaperone might be a logical and comforting choice. However, a new British study found that it’s not appropriate.
Study authors Rebecca Jane Moon and Justin Huw Davies looked at pubertal staging–related exams and contend that a chaperone should always be impartial, and a parent’s presence could conceivably open up a physician to false charges of misconduct.
The solution: A parent should have the option be present during any physical exam of their child, with a medical professional additionally acting as chaperone. This can also work for any patient who wishes to bring a trusted friend or family member to their medical appointments.
How does a chaperoned exam work?
A research team from the Medical College of Wisconsin and Louisiana State University Health conducted a recent systematic review of patient and clinician perspectives on carrying out a chaperoned urology exam. The review found that the protocol could use improvement. For example, it was reported that over one-quarter of patients didn’t feel comfortable asking for a chaperone if they were not offered one. “Patients should not have to request this,” said Diana Londoño, MD, a board-certified urologist and assistant clinical professor at City of Hope National Medical Center in Duarte, Calif.
The researchers also found that 93% of female patients preferred a chaperone of the same gender, whereas male patients were split on this issue. Key duties of a chaperone should be to ensure privacy, help interpret instructions from doctor to patient, and continually clarify that consent is being requested as a sensitive exam proceeds, the researchers report. Although clinical practice standards for chaperones aren’t uniform, keeping these important points in mind can easily be adopted by any health care provider.
Many doctors do follow their own set guidelines. “Often, we have our medical assistants be our chaperones,” explained Dr. Ghofrany. “Ideally they ‘room’ the patient – take vitals and ask the patient what specific concerns they would like to address. This helps with exam set-up.
“For example, if a patient has a breast concern versus a concern about their genital area, this would change what drapes are used. The medical assistant would then stand near me if they are helping with a Pap smear or cultures, or they may stand by the patient’s head at the bedside and offer support if needed. Some patients want to hold someone’s hand during an uncomfortable exam.”
The issue of positioning is important. A patient may feel very uneasy if it appears that the chaperone is looking at their body from the doctor’s angle, negating the point of reassurance. The key is to explain before an exam exactly how a chaperone is needed during the exam itself and whether the chaperone is a medical assistant or nurse.
“Chaperones usually stand next to me on either side or on the side of the patient facing me, depending on if they are immediately assisting me or helping the patient,” said Dr. Londoño. If a chaperone will be moving back and forth during the exam, that should be conveyed as well. For virtual visits, a chaperone can act as a third party and be present on a split screen, a process Michigan Medicine uses.
It’s also important to inform patients that a chaperone can step in and stop an exam at any point, both at the patient’s request or because of any observation of inappropriate physician behavior. Understanding this can help reluctant patients feel less worry.
When Christian Miller was diagnosed with a sleep disorder, his doctor suggested having a chaperone present during his physical exams. At first, he was apprehensive but then found the experience to be very positive. “Having someone there with me gave me an extra measure of assurance that nothing untoward would happen,” he said. “I found that having a chaperone was reassuring. The chaperone was not intrusive but stood close enough to ensure everything went smoothly and respectfully.”
Do chaperones help protect physicians legally?
Some states mandate that chaperones be present by patient request during sensitive exams, such as Texas and Oregon. For the most part, though, physicians have no legal obligation to offer chaperones, although it could be in their best interest to do so.
Ob.gyn. is one of the most litigated specialties, for example, so these physicians may find chaperones can play a key role in averting lawsuits. According to data from Physicians’ Insurance, having a reliable witness in an exam room can mitigate claims, and doctors should follow a clear practice policy regarding chaperones for all patients to further reduce liability.
Another advantage to having a chaperone present: protection for a doctor against a problematic patient. The risk for assault or aggression during an exam can of course go both ways. It’s important to be able to prove any patient misconduct through a witness, and a chaperone can lessen the prospect of violence in any form. “Having a chaperone in an exam room is about protecting the doctors as well as the patient,” said Ms. Kanyok.
A chaperone can also defuse ethical dilemmas. Consider a patient who refuses to allow a chaperone in an exam, but a chaperone is required by the physician’s health care organization. Asking a patient to state their reasons for wanting privacy with the chaperone present before the actual exam can help document the patient’s wish respectfully as well as protect a physician and organization from any potential liability if the patient refuses the exam altogether or if an exception for the patient is made.
Making a chaperone policy work best
- Have your staff inform patients of a medical chaperone policy at the time an appointment is made. Have fact sheets available for the patient before appointments fully outlining your policy.
- Inform the chaperone fully about the details of the exam and the patient’s case prior to the exam, in accordance with HIPAA.
- The chaperone can introduce themselves to the patient in the waiting room or exam room before you enter the room. The chaperone can go over the policy again verbally and answer any questions the patient may have initially. You can then clarify whether the patient understands your chaperone policy when you come in.
- Document, document, document. Write down who the chaperone was for each exam in patient notes and note the details of any interactions that are significant, such as patient questions or conflicts.
- Practice respect, above all. “A patient’s safety and level of comfort should be prioritized,” said Ms. Kanyok.
A version of this article first appeared on Medscape.com.
News of physicians accused or convicted of sexually inappropriate behavior toward patients during medical exams has been frequent recently. And patient advocates have brought up the fact that many patients are uncomfortable during sensitive exams.
As a result, more
For some institutions, the movement has been toward mandating chaperones. Chaperones can be present for any examination of what are often referred to as sensitive areas, such as breasts, genitalia, and the perianal area. For example, Yale University Health requires patients to use a medical staff member as a chaperone for all sensitive exams except in emergency situations. UCLA Health, on the other hand, allows any adult or child aged 12 years or older to decline a chaperone if they wish to do so. Michigan Medicine provides a chaperone on request, although a patient can opt out.
Many physicians fully support the concept. “If a patient requests a chaperone I think we all, as practitioners, should honor that request respectfully,” said Shieva Ghofrany, MD, an ob.gyn. practicing in Stamford, Conn., and cofounder of tribecalledv, a women’s health knowledge platform. “It’s a clear sign a patient wants to ensure that they feel safe and comfortable.”
However, using a chaperone can be challenging for many primary care physicians and specialists in terms of logistics. Should a chaperone’s job be purely observational? Or should the chaperone also be a medical professional who assists during the procedure? How, specifically, should a chaperone perform their duty during an exam? Where should the chaperone stand? What about legal and ethical ramifications?
Who should be a chaperone?
The role of a chaperone is sometimes vaguely defined. It’s logical that a chaperone should have sufficient medical knowledge. Could it be a medical student? Should it be a nurse or another doctor? Would a patient representative suffice even if they aren’t a direct-care clinician?
There are no set standards about who can assume the role. However, the American Medical Association recommends that a patient chaperone be an authorized member of a health care team. For many patients, this is vital.
“As a survivor of sexual assault by an ob.gyn. while I was pregnant, I can assure you that the heinous crime committed against me and hundreds of other women would have been prevented had there been another medical professional in the room,” said Laurie Kanyok, founder of a dance company in New York City, whose former doctor was recently convicted of federal sex abuse charges and is scheduled to be sentenced in July 2023. “The chaperone should be a physician assistant, nurse or medical professional. Qualified professionals better understand the nature and protocols of an exam.”
For children and adolescents, allowing a parent to be a chaperone might be a logical and comforting choice. However, a new British study found that it’s not appropriate.
Study authors Rebecca Jane Moon and Justin Huw Davies looked at pubertal staging–related exams and contend that a chaperone should always be impartial, and a parent’s presence could conceivably open up a physician to false charges of misconduct.
The solution: A parent should have the option be present during any physical exam of their child, with a medical professional additionally acting as chaperone. This can also work for any patient who wishes to bring a trusted friend or family member to their medical appointments.
How does a chaperoned exam work?
A research team from the Medical College of Wisconsin and Louisiana State University Health conducted a recent systematic review of patient and clinician perspectives on carrying out a chaperoned urology exam. The review found that the protocol could use improvement. For example, it was reported that over one-quarter of patients didn’t feel comfortable asking for a chaperone if they were not offered one. “Patients should not have to request this,” said Diana Londoño, MD, a board-certified urologist and assistant clinical professor at City of Hope National Medical Center in Duarte, Calif.
The researchers also found that 93% of female patients preferred a chaperone of the same gender, whereas male patients were split on this issue. Key duties of a chaperone should be to ensure privacy, help interpret instructions from doctor to patient, and continually clarify that consent is being requested as a sensitive exam proceeds, the researchers report. Although clinical practice standards for chaperones aren’t uniform, keeping these important points in mind can easily be adopted by any health care provider.
Many doctors do follow their own set guidelines. “Often, we have our medical assistants be our chaperones,” explained Dr. Ghofrany. “Ideally they ‘room’ the patient – take vitals and ask the patient what specific concerns they would like to address. This helps with exam set-up.
“For example, if a patient has a breast concern versus a concern about their genital area, this would change what drapes are used. The medical assistant would then stand near me if they are helping with a Pap smear or cultures, or they may stand by the patient’s head at the bedside and offer support if needed. Some patients want to hold someone’s hand during an uncomfortable exam.”
The issue of positioning is important. A patient may feel very uneasy if it appears that the chaperone is looking at their body from the doctor’s angle, negating the point of reassurance. The key is to explain before an exam exactly how a chaperone is needed during the exam itself and whether the chaperone is a medical assistant or nurse.
“Chaperones usually stand next to me on either side or on the side of the patient facing me, depending on if they are immediately assisting me or helping the patient,” said Dr. Londoño. If a chaperone will be moving back and forth during the exam, that should be conveyed as well. For virtual visits, a chaperone can act as a third party and be present on a split screen, a process Michigan Medicine uses.
It’s also important to inform patients that a chaperone can step in and stop an exam at any point, both at the patient’s request or because of any observation of inappropriate physician behavior. Understanding this can help reluctant patients feel less worry.
When Christian Miller was diagnosed with a sleep disorder, his doctor suggested having a chaperone present during his physical exams. At first, he was apprehensive but then found the experience to be very positive. “Having someone there with me gave me an extra measure of assurance that nothing untoward would happen,” he said. “I found that having a chaperone was reassuring. The chaperone was not intrusive but stood close enough to ensure everything went smoothly and respectfully.”
Do chaperones help protect physicians legally?
Some states mandate that chaperones be present by patient request during sensitive exams, such as Texas and Oregon. For the most part, though, physicians have no legal obligation to offer chaperones, although it could be in their best interest to do so.
Ob.gyn. is one of the most litigated specialties, for example, so these physicians may find chaperones can play a key role in averting lawsuits. According to data from Physicians’ Insurance, having a reliable witness in an exam room can mitigate claims, and doctors should follow a clear practice policy regarding chaperones for all patients to further reduce liability.
Another advantage to having a chaperone present: protection for a doctor against a problematic patient. The risk for assault or aggression during an exam can of course go both ways. It’s important to be able to prove any patient misconduct through a witness, and a chaperone can lessen the prospect of violence in any form. “Having a chaperone in an exam room is about protecting the doctors as well as the patient,” said Ms. Kanyok.
A chaperone can also defuse ethical dilemmas. Consider a patient who refuses to allow a chaperone in an exam, but a chaperone is required by the physician’s health care organization. Asking a patient to state their reasons for wanting privacy with the chaperone present before the actual exam can help document the patient’s wish respectfully as well as protect a physician and organization from any potential liability if the patient refuses the exam altogether or if an exception for the patient is made.
Making a chaperone policy work best
- Have your staff inform patients of a medical chaperone policy at the time an appointment is made. Have fact sheets available for the patient before appointments fully outlining your policy.
- Inform the chaperone fully about the details of the exam and the patient’s case prior to the exam, in accordance with HIPAA.
- The chaperone can introduce themselves to the patient in the waiting room or exam room before you enter the room. The chaperone can go over the policy again verbally and answer any questions the patient may have initially. You can then clarify whether the patient understands your chaperone policy when you come in.
- Document, document, document. Write down who the chaperone was for each exam in patient notes and note the details of any interactions that are significant, such as patient questions or conflicts.
- Practice respect, above all. “A patient’s safety and level of comfort should be prioritized,” said Ms. Kanyok.
A version of this article first appeared on Medscape.com.
Over-the-counter switches improve access but come with risks
On July 13, the Food and Drug Administration approved the first over-the-counter (OTC) norgestrel birth control pill (Opill). The daily oral contraceptive was approved for prescription use 5 decades ago, providing regulators with a half-century of data to show that the progestin-only drug can be used safely without a prescription.
The drug is the latest in a series of medications that have made the switch from behind the pharmacy counter to retail shelves.
Why switch?
When a drug manufacturer submits a proposal for a switch to OTC, the key question that the FDA considers is patient safety. Some risks can be mitigated by approving OTC drugs at lower doses than what is available as the prescription version.
“There is no drug that doesn’t have risks,” said Almut G. Winterstein, RPh, PhD, a distinguished professor in pharmaceutical outcomes and policy and director of the Center for Drug Evaluation and Safety at the University of Florida, Gainesville. “Risks are mitigated by putting specific constraints around access to those medications.”
Dr. Winterstein, a former chair of the FDA’s Drug Safety and Risk Management Advisory Committee, said that nonprescription drugs are unnecessary in a functional health care system.
Many patients may struggle with accessing health clinicians, so making medications available OTC fills gaps left by not being able to get a prescription, according to Dr. Winterstein.
A 2012 paper funded by the Consumer Healthcare Products Association (CHPA), the organization representing manufacturers and distributors of OTC medications, estimated that one quarter of people who bought OTC drugs would not otherwise seek treatment if these treatments were available only via prescription. The CHPA notes that the number of those who experience allergies who use nonprescription antihistamines and allergy-relief drugs increased by about 10% between 2009 and 2015.
Cholesterol drugs
Approximately 80 million U.S. adults are eligible for cholesterol-lowering medications, particularly statins, but nearly half don’t take them, according to the Centers for Disease Control and Prevention.
Fear of side effects is the most common reason people might avoid taking these drugs. But eliminating the need for a refill may encourage uptake of the statins.
“It’s refill, refill, refill,” said Allen J. Taylor, MD, chairman of cardiology at MedStar Heart and Vascular Institute, in Washington. “We spend a ton of time refilling statins and it’s a headache for patients, too.”
The need to secure regular prescriptions for the drug, “doesn’t put enough trust and faith in pharmacists and doesn’t put enough trust and faith in patients,” Dr. Taylor said.
Moving statins to the front end of a pharmacy might not be the best move given the potential for drug interactions, but a nonprescription behind-the-counter approach could work, according to Dr. Taylor.
“The concerns are modest at most, to where they can be monitored by a pharmacist,” he said. “There’s probably more people that would take a statin if they had that kind of access.”
Many statin manufacturers have attempted to make the prescription-to-OTC switch. In 2005, an FDA advisory panel rejected Merck’s proposal for OTC sales of lovastatin after reviewing a study that found only 55% of OTC purchases would have been medically appropriate.
In 2015, Pfizer pulled its application to make the cholesterol drug atorvastatin available to patients OTC because patients were not using the drug correctly. AstraZeneca is investigating an online platform that would allow patients to self-assess their eligibility for rosuvastatin.
Asthma inhalers
Inhalers are the main rescue therapy for asthma aside from a visit to the ED.
The only inhaler available OTC is epinephrine sold under the brand name Primatene Mist, but this type of medicine device is not recommended as a first-line therapy for acute asthma symptoms, according to the American Medical Association.
“It’s been around for a long time and has stayed over the counter even though newer, safer agents have come onto the market which aren’t available over the counter,” said William B. Feldman, MD, DPhil, MPH, a pulmonologist at Brigham and Women’s Hospital, Boston.
Patients who have a hard time getting to a doctor or patients who lack insurance often face barriers accessing albuterol inhalers and beta agonist–corticosteroid combinations, according to Dr. Feldman. A switch to OTC distribution would widen access.
“What we’re advocating is, if they’re going to have access to Primatene Mist, wouldn’t it be sensible to have access to a safer and more effective therapy?” Dr. Feldman said.
Triptans
Migraines affect an estimated 39 million people in the United States, according to the American Migraine Foundation. Several drugs to treat migraine are available OTC, including nonsteroidal anti-inflammatory drugs, aspirin, and acetaminophen. Triptans, drugs used for the short-term treatment of acute symptoms, are prescription-only in the United States.
But in the United Kingdom, triptans first became available in retail stores in 2006, leading to reduced costs for patients, employers, and the government. One study found that government health expenditures would be reduced by $84 million annually if the OTC switch were made in six European countries.
However, overuse of the drug and potential contraindications have been cited as concerns with OTC access.
For Dr. Winterstein, the decision to switch isn’t just about the freedom to buy a drug; it comes down to weighing potential risks and benefits.
“Drugs are only as good as if they’re used in the context of how they should be used,” Dr. Winterstein said. “It’s not candy.”
A version of this article first appeared on Medscape.com.
On July 13, the Food and Drug Administration approved the first over-the-counter (OTC) norgestrel birth control pill (Opill). The daily oral contraceptive was approved for prescription use 5 decades ago, providing regulators with a half-century of data to show that the progestin-only drug can be used safely without a prescription.
The drug is the latest in a series of medications that have made the switch from behind the pharmacy counter to retail shelves.
Why switch?
When a drug manufacturer submits a proposal for a switch to OTC, the key question that the FDA considers is patient safety. Some risks can be mitigated by approving OTC drugs at lower doses than what is available as the prescription version.
“There is no drug that doesn’t have risks,” said Almut G. Winterstein, RPh, PhD, a distinguished professor in pharmaceutical outcomes and policy and director of the Center for Drug Evaluation and Safety at the University of Florida, Gainesville. “Risks are mitigated by putting specific constraints around access to those medications.”
Dr. Winterstein, a former chair of the FDA’s Drug Safety and Risk Management Advisory Committee, said that nonprescription drugs are unnecessary in a functional health care system.
Many patients may struggle with accessing health clinicians, so making medications available OTC fills gaps left by not being able to get a prescription, according to Dr. Winterstein.
A 2012 paper funded by the Consumer Healthcare Products Association (CHPA), the organization representing manufacturers and distributors of OTC medications, estimated that one quarter of people who bought OTC drugs would not otherwise seek treatment if these treatments were available only via prescription. The CHPA notes that the number of those who experience allergies who use nonprescription antihistamines and allergy-relief drugs increased by about 10% between 2009 and 2015.
Cholesterol drugs
Approximately 80 million U.S. adults are eligible for cholesterol-lowering medications, particularly statins, but nearly half don’t take them, according to the Centers for Disease Control and Prevention.
Fear of side effects is the most common reason people might avoid taking these drugs. But eliminating the need for a refill may encourage uptake of the statins.
“It’s refill, refill, refill,” said Allen J. Taylor, MD, chairman of cardiology at MedStar Heart and Vascular Institute, in Washington. “We spend a ton of time refilling statins and it’s a headache for patients, too.”
The need to secure regular prescriptions for the drug, “doesn’t put enough trust and faith in pharmacists and doesn’t put enough trust and faith in patients,” Dr. Taylor said.
Moving statins to the front end of a pharmacy might not be the best move given the potential for drug interactions, but a nonprescription behind-the-counter approach could work, according to Dr. Taylor.
“The concerns are modest at most, to where they can be monitored by a pharmacist,” he said. “There’s probably more people that would take a statin if they had that kind of access.”
Many statin manufacturers have attempted to make the prescription-to-OTC switch. In 2005, an FDA advisory panel rejected Merck’s proposal for OTC sales of lovastatin after reviewing a study that found only 55% of OTC purchases would have been medically appropriate.
In 2015, Pfizer pulled its application to make the cholesterol drug atorvastatin available to patients OTC because patients were not using the drug correctly. AstraZeneca is investigating an online platform that would allow patients to self-assess their eligibility for rosuvastatin.
Asthma inhalers
Inhalers are the main rescue therapy for asthma aside from a visit to the ED.
The only inhaler available OTC is epinephrine sold under the brand name Primatene Mist, but this type of medicine device is not recommended as a first-line therapy for acute asthma symptoms, according to the American Medical Association.
“It’s been around for a long time and has stayed over the counter even though newer, safer agents have come onto the market which aren’t available over the counter,” said William B. Feldman, MD, DPhil, MPH, a pulmonologist at Brigham and Women’s Hospital, Boston.
Patients who have a hard time getting to a doctor or patients who lack insurance often face barriers accessing albuterol inhalers and beta agonist–corticosteroid combinations, according to Dr. Feldman. A switch to OTC distribution would widen access.
“What we’re advocating is, if they’re going to have access to Primatene Mist, wouldn’t it be sensible to have access to a safer and more effective therapy?” Dr. Feldman said.
Triptans
Migraines affect an estimated 39 million people in the United States, according to the American Migraine Foundation. Several drugs to treat migraine are available OTC, including nonsteroidal anti-inflammatory drugs, aspirin, and acetaminophen. Triptans, drugs used for the short-term treatment of acute symptoms, are prescription-only in the United States.
But in the United Kingdom, triptans first became available in retail stores in 2006, leading to reduced costs for patients, employers, and the government. One study found that government health expenditures would be reduced by $84 million annually if the OTC switch were made in six European countries.
However, overuse of the drug and potential contraindications have been cited as concerns with OTC access.
For Dr. Winterstein, the decision to switch isn’t just about the freedom to buy a drug; it comes down to weighing potential risks and benefits.
“Drugs are only as good as if they’re used in the context of how they should be used,” Dr. Winterstein said. “It’s not candy.”
A version of this article first appeared on Medscape.com.
On July 13, the Food and Drug Administration approved the first over-the-counter (OTC) norgestrel birth control pill (Opill). The daily oral contraceptive was approved for prescription use 5 decades ago, providing regulators with a half-century of data to show that the progestin-only drug can be used safely without a prescription.
The drug is the latest in a series of medications that have made the switch from behind the pharmacy counter to retail shelves.
Why switch?
When a drug manufacturer submits a proposal for a switch to OTC, the key question that the FDA considers is patient safety. Some risks can be mitigated by approving OTC drugs at lower doses than what is available as the prescription version.
“There is no drug that doesn’t have risks,” said Almut G. Winterstein, RPh, PhD, a distinguished professor in pharmaceutical outcomes and policy and director of the Center for Drug Evaluation and Safety at the University of Florida, Gainesville. “Risks are mitigated by putting specific constraints around access to those medications.”
Dr. Winterstein, a former chair of the FDA’s Drug Safety and Risk Management Advisory Committee, said that nonprescription drugs are unnecessary in a functional health care system.
Many patients may struggle with accessing health clinicians, so making medications available OTC fills gaps left by not being able to get a prescription, according to Dr. Winterstein.
A 2012 paper funded by the Consumer Healthcare Products Association (CHPA), the organization representing manufacturers and distributors of OTC medications, estimated that one quarter of people who bought OTC drugs would not otherwise seek treatment if these treatments were available only via prescription. The CHPA notes that the number of those who experience allergies who use nonprescription antihistamines and allergy-relief drugs increased by about 10% between 2009 and 2015.
Cholesterol drugs
Approximately 80 million U.S. adults are eligible for cholesterol-lowering medications, particularly statins, but nearly half don’t take them, according to the Centers for Disease Control and Prevention.
Fear of side effects is the most common reason people might avoid taking these drugs. But eliminating the need for a refill may encourage uptake of the statins.
“It’s refill, refill, refill,” said Allen J. Taylor, MD, chairman of cardiology at MedStar Heart and Vascular Institute, in Washington. “We spend a ton of time refilling statins and it’s a headache for patients, too.”
The need to secure regular prescriptions for the drug, “doesn’t put enough trust and faith in pharmacists and doesn’t put enough trust and faith in patients,” Dr. Taylor said.
Moving statins to the front end of a pharmacy might not be the best move given the potential for drug interactions, but a nonprescription behind-the-counter approach could work, according to Dr. Taylor.
“The concerns are modest at most, to where they can be monitored by a pharmacist,” he said. “There’s probably more people that would take a statin if they had that kind of access.”
Many statin manufacturers have attempted to make the prescription-to-OTC switch. In 2005, an FDA advisory panel rejected Merck’s proposal for OTC sales of lovastatin after reviewing a study that found only 55% of OTC purchases would have been medically appropriate.
In 2015, Pfizer pulled its application to make the cholesterol drug atorvastatin available to patients OTC because patients were not using the drug correctly. AstraZeneca is investigating an online platform that would allow patients to self-assess their eligibility for rosuvastatin.
Asthma inhalers
Inhalers are the main rescue therapy for asthma aside from a visit to the ED.
The only inhaler available OTC is epinephrine sold under the brand name Primatene Mist, but this type of medicine device is not recommended as a first-line therapy for acute asthma symptoms, according to the American Medical Association.
“It’s been around for a long time and has stayed over the counter even though newer, safer agents have come onto the market which aren’t available over the counter,” said William B. Feldman, MD, DPhil, MPH, a pulmonologist at Brigham and Women’s Hospital, Boston.
Patients who have a hard time getting to a doctor or patients who lack insurance often face barriers accessing albuterol inhalers and beta agonist–corticosteroid combinations, according to Dr. Feldman. A switch to OTC distribution would widen access.
“What we’re advocating is, if they’re going to have access to Primatene Mist, wouldn’t it be sensible to have access to a safer and more effective therapy?” Dr. Feldman said.
Triptans
Migraines affect an estimated 39 million people in the United States, according to the American Migraine Foundation. Several drugs to treat migraine are available OTC, including nonsteroidal anti-inflammatory drugs, aspirin, and acetaminophen. Triptans, drugs used for the short-term treatment of acute symptoms, are prescription-only in the United States.
But in the United Kingdom, triptans first became available in retail stores in 2006, leading to reduced costs for patients, employers, and the government. One study found that government health expenditures would be reduced by $84 million annually if the OTC switch were made in six European countries.
However, overuse of the drug and potential contraindications have been cited as concerns with OTC access.
For Dr. Winterstein, the decision to switch isn’t just about the freedom to buy a drug; it comes down to weighing potential risks and benefits.
“Drugs are only as good as if they’re used in the context of how they should be used,” Dr. Winterstein said. “It’s not candy.”
A version of this article first appeared on Medscape.com.
Indian Health Service dermatologist: ‘I saw a real need to be of service’
After completing his dermatology residency at Johns Hopkins Hospital in 2010, Christopher Bengson, MD, MHS, then a Lieutenant Commander in the U.S. Public Health Service, accepted an offer to become a full-time dermatologist at Phoenix Indian Medical Center (PIMC) in Arizona, fulfilling a long desire to provide care for underserved individuals. Thirteen years later, .
As one of the largest hospitals in the IHS system, PIMC provides direct health care services to a population of more than 156,000, including tribal members from The Fort McDowell Yavapai Nation, the Salt River Pima-Maricopa Indian Community, and the San Lucy District of the Tohono O’odham Nation, the Tonto Apache Tribe, the Yavapai-Apache Indian Tribe, and the Yavapai-Prescott Indian Tribe. Dr. Bengson also cares for tribal members who travel to PIMC from as far away as Washington State and Hawaii to receive dermatologic care.
“There is a disproportionate number of Native American patients that come in with severe psoriasis, hidradenitis suppurativa, and dissecting cellulitis of the scalp compared to the general U.S. population, and I’ve been surprised by how many have nonmelanoma skin cancers and autoimmune connective tissue diseases like lupus, as the prevailing sentiment among his patients is that Native people do not get skin cancer,” he said in an interview. “Those who travel great distances are those who come see me for the surgical removal of skin cancers.”
Interesting cases he’s seen in his nearly 13 years on the job include Epstein-Barr virus-induced NK/T-cell lymphoma, anaplastic large cell lymphoma, subcutaneous panniculitis-like T-cell lymphoma, and necrobiotic xanthogranuloma, “tumors that have generally gone to tertiary care facilities for treatment, but we’ve been able to manage here.”
In 2017, Dr. Bengson was appointed as the IHS’s first chief clinical consultant for dermatology, a post that provides him the opportunity to interface with Native people and IHS-affiliated clinicians nationwide regarding skin-related questions and concerns. As the only full-time dermatologist employed by the IHS, he also views his role as providing an opportunity to change the perception that some Native Americans may still hold about federally delivered health care, “where there may be a cultural distrust of government health care in indigenous communities, driven by generational historical traumas that have come out of boarding schools, population relocation to desolate and isolated areas of the country, and contracts that were simply not honored,” he explained.
“While none of these issues are new, what has been great for me is that I’m going on 13 years of being at the same facility, and I’ve treated family members, their kids, and even their grandkids. In some ways the primary barrier of continuity of care – at least at PIMC – has been eliminated by me just being here for a long period of time.”
In Dr. Bengson’s opinion, efforts to improve access to attract more Native Americans to dermatology are laudable, including the American Academy of Dermatology’s Pathways Program, which aims to increase the number of dermatology residents from Black, Latino, and indigenous communities from approximately 100 residents to 250 residents by 2027, or by over 150%, through community-based engagement strategies that begin in high school.
“To have an objective benchmark is encouraging,” he said. However, he encourages dermatology residency program directors to rethink how they recruit Native Americans, many of whom hail from rural areas. “If you’re recruiting primarily from urban settings, you’re very unlikely to include Native Americans as a larger group of minorities,” he said. “When you look at the number of department chairs who are Native American, it’s on the order of 0.1%, [so] it’s no surprise that dermatologists coming out of a residency program don’t want to go to reservations to provide dermatologic care. We pay a lot of lip service to mentorship programs and things like that, but you need a mentor who follows you through the process – and it’s a long process.”
He believes that residency program directors should reconsider the metrics used to select dermatology residents and should consider the degree of adversity that a Native American applicant may have had to overcome to make it to the residency selection committees.
Despite obstacles to attracting young Native Americans to a career in medicine, Dr. Bengson sees encouraging signs ahead. Some of his Native American patients and family members of patients have enrolled in medical school and have asked to rotate with him at PIMC at the premedical and medical student level. “Some have moved on, not necessarily to dermatology, but to other specialties and careers in health care,” he said. “When you have such high rates of obesity, diabetes, hypertension, coronary artery disease, and stroke in Native American communities, nodulocystic acne and other skin conditions that are not threats to life and limb become less of a priority. We need to get more people in the pipeline to deliver medical services even if it may not be in dermatology, as the need for dedicated health care professionals is so great across all disciplines.”
After completing his dermatology residency at Johns Hopkins Hospital in 2010, Christopher Bengson, MD, MHS, then a Lieutenant Commander in the U.S. Public Health Service, accepted an offer to become a full-time dermatologist at Phoenix Indian Medical Center (PIMC) in Arizona, fulfilling a long desire to provide care for underserved individuals. Thirteen years later, .
As one of the largest hospitals in the IHS system, PIMC provides direct health care services to a population of more than 156,000, including tribal members from The Fort McDowell Yavapai Nation, the Salt River Pima-Maricopa Indian Community, and the San Lucy District of the Tohono O’odham Nation, the Tonto Apache Tribe, the Yavapai-Apache Indian Tribe, and the Yavapai-Prescott Indian Tribe. Dr. Bengson also cares for tribal members who travel to PIMC from as far away as Washington State and Hawaii to receive dermatologic care.
“There is a disproportionate number of Native American patients that come in with severe psoriasis, hidradenitis suppurativa, and dissecting cellulitis of the scalp compared to the general U.S. population, and I’ve been surprised by how many have nonmelanoma skin cancers and autoimmune connective tissue diseases like lupus, as the prevailing sentiment among his patients is that Native people do not get skin cancer,” he said in an interview. “Those who travel great distances are those who come see me for the surgical removal of skin cancers.”
Interesting cases he’s seen in his nearly 13 years on the job include Epstein-Barr virus-induced NK/T-cell lymphoma, anaplastic large cell lymphoma, subcutaneous panniculitis-like T-cell lymphoma, and necrobiotic xanthogranuloma, “tumors that have generally gone to tertiary care facilities for treatment, but we’ve been able to manage here.”
In 2017, Dr. Bengson was appointed as the IHS’s first chief clinical consultant for dermatology, a post that provides him the opportunity to interface with Native people and IHS-affiliated clinicians nationwide regarding skin-related questions and concerns. As the only full-time dermatologist employed by the IHS, he also views his role as providing an opportunity to change the perception that some Native Americans may still hold about federally delivered health care, “where there may be a cultural distrust of government health care in indigenous communities, driven by generational historical traumas that have come out of boarding schools, population relocation to desolate and isolated areas of the country, and contracts that were simply not honored,” he explained.
“While none of these issues are new, what has been great for me is that I’m going on 13 years of being at the same facility, and I’ve treated family members, their kids, and even their grandkids. In some ways the primary barrier of continuity of care – at least at PIMC – has been eliminated by me just being here for a long period of time.”
In Dr. Bengson’s opinion, efforts to improve access to attract more Native Americans to dermatology are laudable, including the American Academy of Dermatology’s Pathways Program, which aims to increase the number of dermatology residents from Black, Latino, and indigenous communities from approximately 100 residents to 250 residents by 2027, or by over 150%, through community-based engagement strategies that begin in high school.
“To have an objective benchmark is encouraging,” he said. However, he encourages dermatology residency program directors to rethink how they recruit Native Americans, many of whom hail from rural areas. “If you’re recruiting primarily from urban settings, you’re very unlikely to include Native Americans as a larger group of minorities,” he said. “When you look at the number of department chairs who are Native American, it’s on the order of 0.1%, [so] it’s no surprise that dermatologists coming out of a residency program don’t want to go to reservations to provide dermatologic care. We pay a lot of lip service to mentorship programs and things like that, but you need a mentor who follows you through the process – and it’s a long process.”
He believes that residency program directors should reconsider the metrics used to select dermatology residents and should consider the degree of adversity that a Native American applicant may have had to overcome to make it to the residency selection committees.
Despite obstacles to attracting young Native Americans to a career in medicine, Dr. Bengson sees encouraging signs ahead. Some of his Native American patients and family members of patients have enrolled in medical school and have asked to rotate with him at PIMC at the premedical and medical student level. “Some have moved on, not necessarily to dermatology, but to other specialties and careers in health care,” he said. “When you have such high rates of obesity, diabetes, hypertension, coronary artery disease, and stroke in Native American communities, nodulocystic acne and other skin conditions that are not threats to life and limb become less of a priority. We need to get more people in the pipeline to deliver medical services even if it may not be in dermatology, as the need for dedicated health care professionals is so great across all disciplines.”
After completing his dermatology residency at Johns Hopkins Hospital in 2010, Christopher Bengson, MD, MHS, then a Lieutenant Commander in the U.S. Public Health Service, accepted an offer to become a full-time dermatologist at Phoenix Indian Medical Center (PIMC) in Arizona, fulfilling a long desire to provide care for underserved individuals. Thirteen years later, .
As one of the largest hospitals in the IHS system, PIMC provides direct health care services to a population of more than 156,000, including tribal members from The Fort McDowell Yavapai Nation, the Salt River Pima-Maricopa Indian Community, and the San Lucy District of the Tohono O’odham Nation, the Tonto Apache Tribe, the Yavapai-Apache Indian Tribe, and the Yavapai-Prescott Indian Tribe. Dr. Bengson also cares for tribal members who travel to PIMC from as far away as Washington State and Hawaii to receive dermatologic care.
“There is a disproportionate number of Native American patients that come in with severe psoriasis, hidradenitis suppurativa, and dissecting cellulitis of the scalp compared to the general U.S. population, and I’ve been surprised by how many have nonmelanoma skin cancers and autoimmune connective tissue diseases like lupus, as the prevailing sentiment among his patients is that Native people do not get skin cancer,” he said in an interview. “Those who travel great distances are those who come see me for the surgical removal of skin cancers.”
Interesting cases he’s seen in his nearly 13 years on the job include Epstein-Barr virus-induced NK/T-cell lymphoma, anaplastic large cell lymphoma, subcutaneous panniculitis-like T-cell lymphoma, and necrobiotic xanthogranuloma, “tumors that have generally gone to tertiary care facilities for treatment, but we’ve been able to manage here.”
In 2017, Dr. Bengson was appointed as the IHS’s first chief clinical consultant for dermatology, a post that provides him the opportunity to interface with Native people and IHS-affiliated clinicians nationwide regarding skin-related questions and concerns. As the only full-time dermatologist employed by the IHS, he also views his role as providing an opportunity to change the perception that some Native Americans may still hold about federally delivered health care, “where there may be a cultural distrust of government health care in indigenous communities, driven by generational historical traumas that have come out of boarding schools, population relocation to desolate and isolated areas of the country, and contracts that were simply not honored,” he explained.
“While none of these issues are new, what has been great for me is that I’m going on 13 years of being at the same facility, and I’ve treated family members, their kids, and even their grandkids. In some ways the primary barrier of continuity of care – at least at PIMC – has been eliminated by me just being here for a long period of time.”
In Dr. Bengson’s opinion, efforts to improve access to attract more Native Americans to dermatology are laudable, including the American Academy of Dermatology’s Pathways Program, which aims to increase the number of dermatology residents from Black, Latino, and indigenous communities from approximately 100 residents to 250 residents by 2027, or by over 150%, through community-based engagement strategies that begin in high school.
“To have an objective benchmark is encouraging,” he said. However, he encourages dermatology residency program directors to rethink how they recruit Native Americans, many of whom hail from rural areas. “If you’re recruiting primarily from urban settings, you’re very unlikely to include Native Americans as a larger group of minorities,” he said. “When you look at the number of department chairs who are Native American, it’s on the order of 0.1%, [so] it’s no surprise that dermatologists coming out of a residency program don’t want to go to reservations to provide dermatologic care. We pay a lot of lip service to mentorship programs and things like that, but you need a mentor who follows you through the process – and it’s a long process.”
He believes that residency program directors should reconsider the metrics used to select dermatology residents and should consider the degree of adversity that a Native American applicant may have had to overcome to make it to the residency selection committees.
Despite obstacles to attracting young Native Americans to a career in medicine, Dr. Bengson sees encouraging signs ahead. Some of his Native American patients and family members of patients have enrolled in medical school and have asked to rotate with him at PIMC at the premedical and medical student level. “Some have moved on, not necessarily to dermatology, but to other specialties and careers in health care,” he said. “When you have such high rates of obesity, diabetes, hypertension, coronary artery disease, and stroke in Native American communities, nodulocystic acne and other skin conditions that are not threats to life and limb become less of a priority. We need to get more people in the pipeline to deliver medical services even if it may not be in dermatology, as the need for dedicated health care professionals is so great across all disciplines.”