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MDs’ One-Word Summary of Long COVID Progress: ‘Frustration’
Stuart Malcolm, MD, a primary care physician who practices in Oregon and northern California, started seeing patients with long COVID early in the pandemic. Back then, he was frustrated by the obstacles and lack of standard diagnostic tests and treatments. Four years later, well, he still is.
“Something I learned the last few years is the logistics to get people care is really, really hard,” he said. “There’s a lot of frustration. It’s mostly frustration.”
For long COVID doctors and patients, there has been little to no progress addressing the challenges, leaving many discouraged. Researchers and clinicians now have a greater understanding of what health agencies formally call post-COVID condition, but the wide spectrum of symptoms, slow progress in launching pharmacologic clinical trials, and the research toward understanding the underlying causes mean standardized diagnostic tests and definitive treatments remain elusive.
“The frustration is that we aren’t able to help everyone with our current knowledge base. And I think the frustration lies not just with us physicians but also with patients because they’re at the point where if they tried everything, literally everything and haven’t gotten better,” said Zijian Chen, MD, director of the Mount Sinai Center for Post-COVID Care in New York City.
Wanted: More Funding, More Doctors, More Clinics
Between 10% and 20% of the estimated hundreds of millions of people infected worldwide with SARS-CoV-2 in the first 2 years went on to develop long-term symptoms. While many recover over time, doctors who have treated long COVID since 2020 said they see some patients still wrestling with the condition after 4 years.
The latest Centers for Disease Control and Prevention Household Pulse Survey, taken between March 5 and April 1, 2024, estimated that nearly 7% of the adult population — more than 18 million people — currently have long COVID. Data from other countries also suggest that millions have been living with long COVID for years now, and hundreds of thousands have seen their day-to-day activities significantly affected.
There is an urgent need for more funding, long COVID clinicians, multidisciplinary clinics, and education for non–long COVID physicians and specialists, doctors said. Instead, funding remains limited, clinics are closing, wait times are “horrendously long,” patients are left in limbo, and physicians are burning out.
“What’s changed in some ways is that there’s even less access to COVID rehab, which sounds crazy because there was very little to begin with,” said Alexandra Rendely, MD, a physical medicine and rehabilitation physician with the interdisciplinary Toronto Rehab, a part of the University Health Network of teaching hospitals in Toronto, Ontario, Canada.
“Patients are still being diagnosed every day, yet the resources available are becoming less and less.”
COVID-19 money earmarked during the pandemic was mostly limited to temporary emergency measures. As those funds dwindled, governments and institutions have decreased financial support. The Long COVID Moonshot campaign, organized by patients with long COVID, is pushing Congress to support $1 billion in annual research funding to close the financial chasm.
The Clinical Trial Conundrum
While long COVID clinics have come a long way in helping patients, gaps remain. Doctors may be unwilling to prescribe off-label treatments without proper clinical trials due to the potential risks and liabilities involved or due to the controversial or unconventional nature of the therapies, said Dr. Malcolm, who left his primary care practice more than 2 years ago to focus on long COVID.
In the absence of standard treatments, Dr. Malcolm and other doctors said they must take a trial-and-error approach in treating patients with long COVID that centers on addressing symptoms and not the underlying condition.
“There are actually a lot of treatments and a lot of them are not curative, but they can help people,” he said.
Dr. Malcolm, who is a medical director at Real Time Health Monitoring, a private clinic in the San Francisco Bay Area that specializes in long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), said it was important for him to be with a clinical team that understood and was supportive of his treatment decisions and was able to offer clinical support for those treatments if needed.
For physicians looking for clinical data before prescribing certain medications, the wait may be long. More than $1.5 billion in US federal funding has been earmarked to study long COVID, but the National Institutes of Health (NIH) has faced criticism from patients and scientists alike for its slow progress and emphasis on observational studies instead of research that could unravel the biological roots of long COVID. Among the clinical trials announced by the NIH’s RECOVER initiative, only a handful involve studying pharmaceutical treatments.
A 2023 editorial published in The Lancet called out the “dismal state of clinical research relative to the substantial burden of [long COVID]” and said, “we are clearly lacking tested pharmacological interventions that treat the underlying pathophysiology.” At the time of publication, it noted that of the 386 long COVID trials listed on ClinicalTrials.gov, only 12 were actually testing pharmacologic interventions.
There are also diagnostic and insurance barriers. The specialized tests that can detect long COVID anomalies are neither commonly known by primary care practitioners nor easily requested at the local lab, can be expensive, and are typically not covered by insurance, Dr. Malcolm explained.
Patients with long COVID also have the added barrier of being unable to advocate as easily because of their energy limitations, doctors said. Patients may appear outwardly fine, but fatigue and brain fog are among the many problems that cannot be measured in appearances. The condition has upended lives, some losing jobs, even homes, and the mental toll is why there is a “not insignificant” suicide rate.
One Patient’s 4-Year Journey
Charlie McCone, 34, used to be a tennis player and an active musician. But he’s spent the past 4 years mostly housebound, grappling with the aftermath of a SARS-CoV-2 infection he contracted in March 2020. He went from biking daily to work 10 miles and back to having at most 2 hours of energy per day.
In the first year alone, Mr. McCone saw more than two dozen doctors and specialists. The conditions now associated with long COVID, like ME/CFS, mast cell activation syndrome (a condition in which a patient experiences episodes of allergic symptoms such as hives, swelling, low blood pressure, and difficulty breathing), or dysautonomia (conditions that affect the autonomic nervous system, which controls automatic processes in the body) were not on physicians’ radars.
Then in 2021, he became bedbound for more than half a year after a Delta variant reinfection. He developed neurologic symptoms, including incapacitating fatigue, post-exertional malaise (where symptoms worsened after minimal physical or mental activity), left-sided weakness, and cognitive impairment. He stopped working altogether. But the worst was the shortness of breath he felt 24/7, even at rest. A battery of lab tests revealed nothing abnormal. He tried numerous drugs and the classic respiratory treatments.
Mr. McCone eventually connected with Dr. Malcolm over X and developed what he describes as an effective patient-doctor collaboration. When studies came out suggesting microclots were a common issue with patients with long COVID and positive outcomes were reported from anticoagulant therapy, they knew it could be one of the answers.
“After 3 weeks on [the antiplatelet drug], I was like, oh my god, my lungs are finally opening up,” said Mr. McCone. He has taken the medication for more than a year and a half, and some days he doesn’t even think about his respiratory symptoms.
“That trial-and-error process is just really long and hard and costly,” said Dr. Malcolm.
Today, fatigue and cognitive stamina are Mr. McCone’s main challenges, and he is far from recovered.
“[I had a] very fulfilling, happy life and now, it’s hard to think about. I’ve come a long way with my mental health and all this, but I’ve lost 4 years,” Mr. McCone said. “The prospect of me being here when I’m 40 seems very real ... so it’s pretty devastating.”
Lessons Learned, Hope Amid Ongoing Research
Despite the daunting obstacle, doctors said the science has come a long way for a new disease. We now know long COVID is likely caused by a combination of triggers, including viral reservoir in the tissue, inflammation, autoimmunity, and microclots; severity of infection is not necessarily an accurate risk factor predictor — long COVID can strike even those who had a mild infection; upward of 200 symptoms have been identified; and we know more about potential biomarkers that could lead to better diagnostic tools.
Unlike many other diseases and conditions with standard treatment protocols, long COVID treatments are typically aimed at addressing individual symptoms.
“It is very detailed and individualized to the patient’s specific symptoms and to the patient’s specific needs,” Dr. Rendely said. Symptoms can also fluctuate, relapse, or wax and wane, for example, so what ails a patient at their first doctor’s appointment could be completely different at the next appointment 2 months later.
Doctors are still hopeful the RECOVER research, which includes trials that look at autonomic and cognitive dysfunctions, will pave the way for more effective long COVID therapies. In Canada, Dr. Rendely is also eying the RECLAIM trial that is currently testing the effectiveness of pentoxifylline, which helps blood flow, and ibudilast, an anti-inflammatory drug.
Doctors are also hopeful when they see patients who have made “tremendous gains” or even full recoveries through their clinics. “It’s a new diagnosis, so I always tell my patients to think of this as a journey because I’m learning along with you,” said Jai Marathe, MD, an infectious disease physician at Boston Medical Center and an assistant professor of infectious diseases at Boston University Chobanian & Avedisian School of Medicine.
“Now we have 4 years of experience, but at the same time, no two long COVID patients are alike.”
Long COVID has also changed the way physicians view healthcare and how they practice medicine.
“I am a completely different person than I used to be because of this illness, and I don’t even have it. That is how profoundly it has affected how I view the universe,” said Dr. Malcolm. “I’ve been doing this for 4 years, and I’m very hopeful. But I don’t think about this in terms of months anymore. I think about this in terms of years.”
A version of this article first appeared on Medscape.com.
Stuart Malcolm, MD, a primary care physician who practices in Oregon and northern California, started seeing patients with long COVID early in the pandemic. Back then, he was frustrated by the obstacles and lack of standard diagnostic tests and treatments. Four years later, well, he still is.
“Something I learned the last few years is the logistics to get people care is really, really hard,” he said. “There’s a lot of frustration. It’s mostly frustration.”
For long COVID doctors and patients, there has been little to no progress addressing the challenges, leaving many discouraged. Researchers and clinicians now have a greater understanding of what health agencies formally call post-COVID condition, but the wide spectrum of symptoms, slow progress in launching pharmacologic clinical trials, and the research toward understanding the underlying causes mean standardized diagnostic tests and definitive treatments remain elusive.
“The frustration is that we aren’t able to help everyone with our current knowledge base. And I think the frustration lies not just with us physicians but also with patients because they’re at the point where if they tried everything, literally everything and haven’t gotten better,” said Zijian Chen, MD, director of the Mount Sinai Center for Post-COVID Care in New York City.
Wanted: More Funding, More Doctors, More Clinics
Between 10% and 20% of the estimated hundreds of millions of people infected worldwide with SARS-CoV-2 in the first 2 years went on to develop long-term symptoms. While many recover over time, doctors who have treated long COVID since 2020 said they see some patients still wrestling with the condition after 4 years.
The latest Centers for Disease Control and Prevention Household Pulse Survey, taken between March 5 and April 1, 2024, estimated that nearly 7% of the adult population — more than 18 million people — currently have long COVID. Data from other countries also suggest that millions have been living with long COVID for years now, and hundreds of thousands have seen their day-to-day activities significantly affected.
There is an urgent need for more funding, long COVID clinicians, multidisciplinary clinics, and education for non–long COVID physicians and specialists, doctors said. Instead, funding remains limited, clinics are closing, wait times are “horrendously long,” patients are left in limbo, and physicians are burning out.
“What’s changed in some ways is that there’s even less access to COVID rehab, which sounds crazy because there was very little to begin with,” said Alexandra Rendely, MD, a physical medicine and rehabilitation physician with the interdisciplinary Toronto Rehab, a part of the University Health Network of teaching hospitals in Toronto, Ontario, Canada.
“Patients are still being diagnosed every day, yet the resources available are becoming less and less.”
COVID-19 money earmarked during the pandemic was mostly limited to temporary emergency measures. As those funds dwindled, governments and institutions have decreased financial support. The Long COVID Moonshot campaign, organized by patients with long COVID, is pushing Congress to support $1 billion in annual research funding to close the financial chasm.
The Clinical Trial Conundrum
While long COVID clinics have come a long way in helping patients, gaps remain. Doctors may be unwilling to prescribe off-label treatments without proper clinical trials due to the potential risks and liabilities involved or due to the controversial or unconventional nature of the therapies, said Dr. Malcolm, who left his primary care practice more than 2 years ago to focus on long COVID.
In the absence of standard treatments, Dr. Malcolm and other doctors said they must take a trial-and-error approach in treating patients with long COVID that centers on addressing symptoms and not the underlying condition.
“There are actually a lot of treatments and a lot of them are not curative, but they can help people,” he said.
Dr. Malcolm, who is a medical director at Real Time Health Monitoring, a private clinic in the San Francisco Bay Area that specializes in long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), said it was important for him to be with a clinical team that understood and was supportive of his treatment decisions and was able to offer clinical support for those treatments if needed.
For physicians looking for clinical data before prescribing certain medications, the wait may be long. More than $1.5 billion in US federal funding has been earmarked to study long COVID, but the National Institutes of Health (NIH) has faced criticism from patients and scientists alike for its slow progress and emphasis on observational studies instead of research that could unravel the biological roots of long COVID. Among the clinical trials announced by the NIH’s RECOVER initiative, only a handful involve studying pharmaceutical treatments.
A 2023 editorial published in The Lancet called out the “dismal state of clinical research relative to the substantial burden of [long COVID]” and said, “we are clearly lacking tested pharmacological interventions that treat the underlying pathophysiology.” At the time of publication, it noted that of the 386 long COVID trials listed on ClinicalTrials.gov, only 12 were actually testing pharmacologic interventions.
There are also diagnostic and insurance barriers. The specialized tests that can detect long COVID anomalies are neither commonly known by primary care practitioners nor easily requested at the local lab, can be expensive, and are typically not covered by insurance, Dr. Malcolm explained.
Patients with long COVID also have the added barrier of being unable to advocate as easily because of their energy limitations, doctors said. Patients may appear outwardly fine, but fatigue and brain fog are among the many problems that cannot be measured in appearances. The condition has upended lives, some losing jobs, even homes, and the mental toll is why there is a “not insignificant” suicide rate.
One Patient’s 4-Year Journey
Charlie McCone, 34, used to be a tennis player and an active musician. But he’s spent the past 4 years mostly housebound, grappling with the aftermath of a SARS-CoV-2 infection he contracted in March 2020. He went from biking daily to work 10 miles and back to having at most 2 hours of energy per day.
In the first year alone, Mr. McCone saw more than two dozen doctors and specialists. The conditions now associated with long COVID, like ME/CFS, mast cell activation syndrome (a condition in which a patient experiences episodes of allergic symptoms such as hives, swelling, low blood pressure, and difficulty breathing), or dysautonomia (conditions that affect the autonomic nervous system, which controls automatic processes in the body) were not on physicians’ radars.
Then in 2021, he became bedbound for more than half a year after a Delta variant reinfection. He developed neurologic symptoms, including incapacitating fatigue, post-exertional malaise (where symptoms worsened after minimal physical or mental activity), left-sided weakness, and cognitive impairment. He stopped working altogether. But the worst was the shortness of breath he felt 24/7, even at rest. A battery of lab tests revealed nothing abnormal. He tried numerous drugs and the classic respiratory treatments.
Mr. McCone eventually connected with Dr. Malcolm over X and developed what he describes as an effective patient-doctor collaboration. When studies came out suggesting microclots were a common issue with patients with long COVID and positive outcomes were reported from anticoagulant therapy, they knew it could be one of the answers.
“After 3 weeks on [the antiplatelet drug], I was like, oh my god, my lungs are finally opening up,” said Mr. McCone. He has taken the medication for more than a year and a half, and some days he doesn’t even think about his respiratory symptoms.
“That trial-and-error process is just really long and hard and costly,” said Dr. Malcolm.
Today, fatigue and cognitive stamina are Mr. McCone’s main challenges, and he is far from recovered.
“[I had a] very fulfilling, happy life and now, it’s hard to think about. I’ve come a long way with my mental health and all this, but I’ve lost 4 years,” Mr. McCone said. “The prospect of me being here when I’m 40 seems very real ... so it’s pretty devastating.”
Lessons Learned, Hope Amid Ongoing Research
Despite the daunting obstacle, doctors said the science has come a long way for a new disease. We now know long COVID is likely caused by a combination of triggers, including viral reservoir in the tissue, inflammation, autoimmunity, and microclots; severity of infection is not necessarily an accurate risk factor predictor — long COVID can strike even those who had a mild infection; upward of 200 symptoms have been identified; and we know more about potential biomarkers that could lead to better diagnostic tools.
Unlike many other diseases and conditions with standard treatment protocols, long COVID treatments are typically aimed at addressing individual symptoms.
“It is very detailed and individualized to the patient’s specific symptoms and to the patient’s specific needs,” Dr. Rendely said. Symptoms can also fluctuate, relapse, or wax and wane, for example, so what ails a patient at their first doctor’s appointment could be completely different at the next appointment 2 months later.
Doctors are still hopeful the RECOVER research, which includes trials that look at autonomic and cognitive dysfunctions, will pave the way for more effective long COVID therapies. In Canada, Dr. Rendely is also eying the RECLAIM trial that is currently testing the effectiveness of pentoxifylline, which helps blood flow, and ibudilast, an anti-inflammatory drug.
Doctors are also hopeful when they see patients who have made “tremendous gains” or even full recoveries through their clinics. “It’s a new diagnosis, so I always tell my patients to think of this as a journey because I’m learning along with you,” said Jai Marathe, MD, an infectious disease physician at Boston Medical Center and an assistant professor of infectious diseases at Boston University Chobanian & Avedisian School of Medicine.
“Now we have 4 years of experience, but at the same time, no two long COVID patients are alike.”
Long COVID has also changed the way physicians view healthcare and how they practice medicine.
“I am a completely different person than I used to be because of this illness, and I don’t even have it. That is how profoundly it has affected how I view the universe,” said Dr. Malcolm. “I’ve been doing this for 4 years, and I’m very hopeful. But I don’t think about this in terms of months anymore. I think about this in terms of years.”
A version of this article first appeared on Medscape.com.
Stuart Malcolm, MD, a primary care physician who practices in Oregon and northern California, started seeing patients with long COVID early in the pandemic. Back then, he was frustrated by the obstacles and lack of standard diagnostic tests and treatments. Four years later, well, he still is.
“Something I learned the last few years is the logistics to get people care is really, really hard,” he said. “There’s a lot of frustration. It’s mostly frustration.”
For long COVID doctors and patients, there has been little to no progress addressing the challenges, leaving many discouraged. Researchers and clinicians now have a greater understanding of what health agencies formally call post-COVID condition, but the wide spectrum of symptoms, slow progress in launching pharmacologic clinical trials, and the research toward understanding the underlying causes mean standardized diagnostic tests and definitive treatments remain elusive.
“The frustration is that we aren’t able to help everyone with our current knowledge base. And I think the frustration lies not just with us physicians but also with patients because they’re at the point where if they tried everything, literally everything and haven’t gotten better,” said Zijian Chen, MD, director of the Mount Sinai Center for Post-COVID Care in New York City.
Wanted: More Funding, More Doctors, More Clinics
Between 10% and 20% of the estimated hundreds of millions of people infected worldwide with SARS-CoV-2 in the first 2 years went on to develop long-term symptoms. While many recover over time, doctors who have treated long COVID since 2020 said they see some patients still wrestling with the condition after 4 years.
The latest Centers for Disease Control and Prevention Household Pulse Survey, taken between March 5 and April 1, 2024, estimated that nearly 7% of the adult population — more than 18 million people — currently have long COVID. Data from other countries also suggest that millions have been living with long COVID for years now, and hundreds of thousands have seen their day-to-day activities significantly affected.
There is an urgent need for more funding, long COVID clinicians, multidisciplinary clinics, and education for non–long COVID physicians and specialists, doctors said. Instead, funding remains limited, clinics are closing, wait times are “horrendously long,” patients are left in limbo, and physicians are burning out.
“What’s changed in some ways is that there’s even less access to COVID rehab, which sounds crazy because there was very little to begin with,” said Alexandra Rendely, MD, a physical medicine and rehabilitation physician with the interdisciplinary Toronto Rehab, a part of the University Health Network of teaching hospitals in Toronto, Ontario, Canada.
“Patients are still being diagnosed every day, yet the resources available are becoming less and less.”
COVID-19 money earmarked during the pandemic was mostly limited to temporary emergency measures. As those funds dwindled, governments and institutions have decreased financial support. The Long COVID Moonshot campaign, organized by patients with long COVID, is pushing Congress to support $1 billion in annual research funding to close the financial chasm.
The Clinical Trial Conundrum
While long COVID clinics have come a long way in helping patients, gaps remain. Doctors may be unwilling to prescribe off-label treatments without proper clinical trials due to the potential risks and liabilities involved or due to the controversial or unconventional nature of the therapies, said Dr. Malcolm, who left his primary care practice more than 2 years ago to focus on long COVID.
In the absence of standard treatments, Dr. Malcolm and other doctors said they must take a trial-and-error approach in treating patients with long COVID that centers on addressing symptoms and not the underlying condition.
“There are actually a lot of treatments and a lot of them are not curative, but they can help people,” he said.
Dr. Malcolm, who is a medical director at Real Time Health Monitoring, a private clinic in the San Francisco Bay Area that specializes in long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), said it was important for him to be with a clinical team that understood and was supportive of his treatment decisions and was able to offer clinical support for those treatments if needed.
For physicians looking for clinical data before prescribing certain medications, the wait may be long. More than $1.5 billion in US federal funding has been earmarked to study long COVID, but the National Institutes of Health (NIH) has faced criticism from patients and scientists alike for its slow progress and emphasis on observational studies instead of research that could unravel the biological roots of long COVID. Among the clinical trials announced by the NIH’s RECOVER initiative, only a handful involve studying pharmaceutical treatments.
A 2023 editorial published in The Lancet called out the “dismal state of clinical research relative to the substantial burden of [long COVID]” and said, “we are clearly lacking tested pharmacological interventions that treat the underlying pathophysiology.” At the time of publication, it noted that of the 386 long COVID trials listed on ClinicalTrials.gov, only 12 were actually testing pharmacologic interventions.
There are also diagnostic and insurance barriers. The specialized tests that can detect long COVID anomalies are neither commonly known by primary care practitioners nor easily requested at the local lab, can be expensive, and are typically not covered by insurance, Dr. Malcolm explained.
Patients with long COVID also have the added barrier of being unable to advocate as easily because of their energy limitations, doctors said. Patients may appear outwardly fine, but fatigue and brain fog are among the many problems that cannot be measured in appearances. The condition has upended lives, some losing jobs, even homes, and the mental toll is why there is a “not insignificant” suicide rate.
One Patient’s 4-Year Journey
Charlie McCone, 34, used to be a tennis player and an active musician. But he’s spent the past 4 years mostly housebound, grappling with the aftermath of a SARS-CoV-2 infection he contracted in March 2020. He went from biking daily to work 10 miles and back to having at most 2 hours of energy per day.
In the first year alone, Mr. McCone saw more than two dozen doctors and specialists. The conditions now associated with long COVID, like ME/CFS, mast cell activation syndrome (a condition in which a patient experiences episodes of allergic symptoms such as hives, swelling, low blood pressure, and difficulty breathing), or dysautonomia (conditions that affect the autonomic nervous system, which controls automatic processes in the body) were not on physicians’ radars.
Then in 2021, he became bedbound for more than half a year after a Delta variant reinfection. He developed neurologic symptoms, including incapacitating fatigue, post-exertional malaise (where symptoms worsened after minimal physical or mental activity), left-sided weakness, and cognitive impairment. He stopped working altogether. But the worst was the shortness of breath he felt 24/7, even at rest. A battery of lab tests revealed nothing abnormal. He tried numerous drugs and the classic respiratory treatments.
Mr. McCone eventually connected with Dr. Malcolm over X and developed what he describes as an effective patient-doctor collaboration. When studies came out suggesting microclots were a common issue with patients with long COVID and positive outcomes were reported from anticoagulant therapy, they knew it could be one of the answers.
“After 3 weeks on [the antiplatelet drug], I was like, oh my god, my lungs are finally opening up,” said Mr. McCone. He has taken the medication for more than a year and a half, and some days he doesn’t even think about his respiratory symptoms.
“That trial-and-error process is just really long and hard and costly,” said Dr. Malcolm.
Today, fatigue and cognitive stamina are Mr. McCone’s main challenges, and he is far from recovered.
“[I had a] very fulfilling, happy life and now, it’s hard to think about. I’ve come a long way with my mental health and all this, but I’ve lost 4 years,” Mr. McCone said. “The prospect of me being here when I’m 40 seems very real ... so it’s pretty devastating.”
Lessons Learned, Hope Amid Ongoing Research
Despite the daunting obstacle, doctors said the science has come a long way for a new disease. We now know long COVID is likely caused by a combination of triggers, including viral reservoir in the tissue, inflammation, autoimmunity, and microclots; severity of infection is not necessarily an accurate risk factor predictor — long COVID can strike even those who had a mild infection; upward of 200 symptoms have been identified; and we know more about potential biomarkers that could lead to better diagnostic tools.
Unlike many other diseases and conditions with standard treatment protocols, long COVID treatments are typically aimed at addressing individual symptoms.
“It is very detailed and individualized to the patient’s specific symptoms and to the patient’s specific needs,” Dr. Rendely said. Symptoms can also fluctuate, relapse, or wax and wane, for example, so what ails a patient at their first doctor’s appointment could be completely different at the next appointment 2 months later.
Doctors are still hopeful the RECOVER research, which includes trials that look at autonomic and cognitive dysfunctions, will pave the way for more effective long COVID therapies. In Canada, Dr. Rendely is also eying the RECLAIM trial that is currently testing the effectiveness of pentoxifylline, which helps blood flow, and ibudilast, an anti-inflammatory drug.
Doctors are also hopeful when they see patients who have made “tremendous gains” or even full recoveries through their clinics. “It’s a new diagnosis, so I always tell my patients to think of this as a journey because I’m learning along with you,” said Jai Marathe, MD, an infectious disease physician at Boston Medical Center and an assistant professor of infectious diseases at Boston University Chobanian & Avedisian School of Medicine.
“Now we have 4 years of experience, but at the same time, no two long COVID patients are alike.”
Long COVID has also changed the way physicians view healthcare and how they practice medicine.
“I am a completely different person than I used to be because of this illness, and I don’t even have it. That is how profoundly it has affected how I view the universe,” said Dr. Malcolm. “I’ve been doing this for 4 years, and I’m very hopeful. But I don’t think about this in terms of months anymore. I think about this in terms of years.”
A version of this article first appeared on Medscape.com.
Study Finds Isotretinoin Effective for Acne in Transgender Patients on Hormone Rx
TOPLINE:
, but more information is needed on dosing and barriers to treatment.
METHODOLOGY:
- Acne can be a side effect of masculinizing hormone therapy for transmasculine individuals. While isotretinoin is an effective treatment option for acne, its effectiveness and safety in transgender and gender-diverse individuals are not well understood.
- This retrospective case series included 55 patients (mean age, 25.4 years) undergoing masculinizing hormone therapy at four medical centers, who were prescribed isotretinoin for acne associated with treatment.
- Isotretinoin treatment was started a median of 22.1 months after hormone therapy was initiated and continued for a median of 6 months with a median cumulative dose of 132.7 mg/kg.
- Researchers assessed acne improvement, clearance, recurrence, adverse effects, and reasons for treatment discontinuation.
TAKEAWAY:
- Overall, 48 patients (87.3%) experienced improvement, and 26 (47.3%) achieved clearance during treatment. A higher proportion of patients experienced improvement (97% vs 72.7%) and achieved clearance (63.6% vs 22.7%) with cumulative doses of ≥ 120 mg/kg than those who received cumulative doses < 120 mg/kg.
- The risk for recurrence was 20% (in four patients) among 20 patients who achieved clearance and had any subsequent health care encounters, with a mean follow-up time of 734.3 days.
- Common adverse effects included dryness (80%), joint pain (14.5%), and headaches (10.9%). Other adverse effects included nose bleeds (9.1%) and depression (5.5%).
- Of the 22 patients with a cumulative dose < 120 mg/kg, 14 (63.6%) were lost to follow-up; among those not lost to follow-up, 2 patients discontinued treatment because of transfer of care, 1 because of adverse effects, and 1 because of gender-affirming surgery, with concerns about wound healing.
IN PRACTICE:
“Although isotretinoin appears to be an effective treatment option for acne among individuals undergoing masculinizing hormone therapy, further efforts are needed to understand optimal dosing and treatment barriers to improve outcomes in transgender and gender-diverse individuals receiving testosterone,” the authors concluded.
SOURCE:
The study, led by James Choe, BS, Department of Dermatology, Brigham and Women’s Hospital, Harvard Medical School, Boston, was published online in JAMA Dermatology.
LIMITATIONS:
The study population was limited to four centers, and variability in clinician- and patient-reported acne outcomes and missing information could affect the reliability of data. Because of the small sample size, the association of masculinizing hormone therapy regimens with outcomes could not be evaluated.
DISCLOSURES:
One author is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Three authors reported receiving grants or personal fees from various sources. The other authors declared no conflicts of interest.
A version of this article first appeared on Medscape.com.
TOPLINE:
, but more information is needed on dosing and barriers to treatment.
METHODOLOGY:
- Acne can be a side effect of masculinizing hormone therapy for transmasculine individuals. While isotretinoin is an effective treatment option for acne, its effectiveness and safety in transgender and gender-diverse individuals are not well understood.
- This retrospective case series included 55 patients (mean age, 25.4 years) undergoing masculinizing hormone therapy at four medical centers, who were prescribed isotretinoin for acne associated with treatment.
- Isotretinoin treatment was started a median of 22.1 months after hormone therapy was initiated and continued for a median of 6 months with a median cumulative dose of 132.7 mg/kg.
- Researchers assessed acne improvement, clearance, recurrence, adverse effects, and reasons for treatment discontinuation.
TAKEAWAY:
- Overall, 48 patients (87.3%) experienced improvement, and 26 (47.3%) achieved clearance during treatment. A higher proportion of patients experienced improvement (97% vs 72.7%) and achieved clearance (63.6% vs 22.7%) with cumulative doses of ≥ 120 mg/kg than those who received cumulative doses < 120 mg/kg.
- The risk for recurrence was 20% (in four patients) among 20 patients who achieved clearance and had any subsequent health care encounters, with a mean follow-up time of 734.3 days.
- Common adverse effects included dryness (80%), joint pain (14.5%), and headaches (10.9%). Other adverse effects included nose bleeds (9.1%) and depression (5.5%).
- Of the 22 patients with a cumulative dose < 120 mg/kg, 14 (63.6%) were lost to follow-up; among those not lost to follow-up, 2 patients discontinued treatment because of transfer of care, 1 because of adverse effects, and 1 because of gender-affirming surgery, with concerns about wound healing.
IN PRACTICE:
“Although isotretinoin appears to be an effective treatment option for acne among individuals undergoing masculinizing hormone therapy, further efforts are needed to understand optimal dosing and treatment barriers to improve outcomes in transgender and gender-diverse individuals receiving testosterone,” the authors concluded.
SOURCE:
The study, led by James Choe, BS, Department of Dermatology, Brigham and Women’s Hospital, Harvard Medical School, Boston, was published online in JAMA Dermatology.
LIMITATIONS:
The study population was limited to four centers, and variability in clinician- and patient-reported acne outcomes and missing information could affect the reliability of data. Because of the small sample size, the association of masculinizing hormone therapy regimens with outcomes could not be evaluated.
DISCLOSURES:
One author is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Three authors reported receiving grants or personal fees from various sources. The other authors declared no conflicts of interest.
A version of this article first appeared on Medscape.com.
TOPLINE:
, but more information is needed on dosing and barriers to treatment.
METHODOLOGY:
- Acne can be a side effect of masculinizing hormone therapy for transmasculine individuals. While isotretinoin is an effective treatment option for acne, its effectiveness and safety in transgender and gender-diverse individuals are not well understood.
- This retrospective case series included 55 patients (mean age, 25.4 years) undergoing masculinizing hormone therapy at four medical centers, who were prescribed isotretinoin for acne associated with treatment.
- Isotretinoin treatment was started a median of 22.1 months after hormone therapy was initiated and continued for a median of 6 months with a median cumulative dose of 132.7 mg/kg.
- Researchers assessed acne improvement, clearance, recurrence, adverse effects, and reasons for treatment discontinuation.
TAKEAWAY:
- Overall, 48 patients (87.3%) experienced improvement, and 26 (47.3%) achieved clearance during treatment. A higher proportion of patients experienced improvement (97% vs 72.7%) and achieved clearance (63.6% vs 22.7%) with cumulative doses of ≥ 120 mg/kg than those who received cumulative doses < 120 mg/kg.
- The risk for recurrence was 20% (in four patients) among 20 patients who achieved clearance and had any subsequent health care encounters, with a mean follow-up time of 734.3 days.
- Common adverse effects included dryness (80%), joint pain (14.5%), and headaches (10.9%). Other adverse effects included nose bleeds (9.1%) and depression (5.5%).
- Of the 22 patients with a cumulative dose < 120 mg/kg, 14 (63.6%) were lost to follow-up; among those not lost to follow-up, 2 patients discontinued treatment because of transfer of care, 1 because of adverse effects, and 1 because of gender-affirming surgery, with concerns about wound healing.
IN PRACTICE:
“Although isotretinoin appears to be an effective treatment option for acne among individuals undergoing masculinizing hormone therapy, further efforts are needed to understand optimal dosing and treatment barriers to improve outcomes in transgender and gender-diverse individuals receiving testosterone,” the authors concluded.
SOURCE:
The study, led by James Choe, BS, Department of Dermatology, Brigham and Women’s Hospital, Harvard Medical School, Boston, was published online in JAMA Dermatology.
LIMITATIONS:
The study population was limited to four centers, and variability in clinician- and patient-reported acne outcomes and missing information could affect the reliability of data. Because of the small sample size, the association of masculinizing hormone therapy regimens with outcomes could not be evaluated.
DISCLOSURES:
One author is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Three authors reported receiving grants or personal fees from various sources. The other authors declared no conflicts of interest.
A version of this article first appeared on Medscape.com.
Hidradenitis Suppurativa: Clinical Outcomes for Bimekizumab Positive in Phase 3 Studies
TOPLINE:
, in two phase 3 studies.
METHODOLOGY:
- To assess the efficacy and safety of bimekizumab, an interleukin (IL)-17A and IL-17F antagonist, 320 mg for HS, researchers conducted two 48-week phase 3 trials BE HEARD I (n = 505) and II (n = 509), which enrolled patients with moderate to severe HS and a history of inadequate response to systemic antibiotics.
- Patients were randomly assigned to one of four groups: Bimekizumab every 2 weeks, bimekizumab every 2 weeks for 16 weeks followed by every 4 weeks of dosing, bimekizumab every 4 weeks, or placebo for 16 weeks followed by bimekizumab every 2 weeks.
- The primary outcome was an HS clinical response of at least 50% (HiSCR50) at week 16, defined as at least a 50% reduction in total abscess and inflammatory nodule count.
TAKEAWAY:
- A higher proportion of patients receiving bimekizumab every 2 weeks vs placebo achieved an HiSCR50 response at week 16 in BE HEARD I (48% vs 29%; odds ratio [OR], 2.23; P = .006) and II (52% vs 32%; OR, 2.29; P = .0032) trials.
- Patients receiving bimekizumab every 4 weeks also achieved a higher HiSCR50 response at week 16 vs placebo in the BE HEARD II trial (54% vs 32%; OR, 2.42; P = .0038).
- At week 16, a higher proportion of patients receiving bimekizumab every 2 weeks vs placebo achieved at least a 75% HiSCR (HiSCR75) in both trials, and a higher proportion of those receiving bimekizumab every 4 weeks achieved HiSCR75 in the BE HEARD II trial.
- At week 48, 45%-68% of patients achieved HiSCR50 in both trials.
- Patients who received bimekizumab vs placebo for the initial 16 weeks had greater improvements in patient-reported outcomes, and bimekizumab was well tolerated with a low number of serious or severe treatment-emergent adverse events.
IN PRACTICE:
“Bimekizumab was well tolerated by patients with hidradenitis suppurativa and produced rapid and deep clinically meaningful responses that were maintained up to 48 weeks,” the authors wrote. “These data support the use of bimekizumab as a promising new therapeutic option for patients with moderate to severe hidradenitis suppurativa.”
SOURCE:
Alexa B. Kimball, MD, MPH, from Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, led this study, which was published online in The Lancet.
LIMITATIONS:
The placebo-controlled part of this trial was relatively short at 16 weeks and may affect the interpretation of later efficacy data, there was a lack of an active comparator group, and the efficacy of treatment was evaluated in the presence of rescue treatment with systemic antibiotics.
DISCLOSURES:
The studies were funded by bimekizumab manufacturer UCB Pharma. Seven authors disclosed being current or former employees of UCB Pharma. Other authors reported several ties with many companies, including UCB Pharma.
A version of this article first appeared on Medscape.com.
TOPLINE:
, in two phase 3 studies.
METHODOLOGY:
- To assess the efficacy and safety of bimekizumab, an interleukin (IL)-17A and IL-17F antagonist, 320 mg for HS, researchers conducted two 48-week phase 3 trials BE HEARD I (n = 505) and II (n = 509), which enrolled patients with moderate to severe HS and a history of inadequate response to systemic antibiotics.
- Patients were randomly assigned to one of four groups: Bimekizumab every 2 weeks, bimekizumab every 2 weeks for 16 weeks followed by every 4 weeks of dosing, bimekizumab every 4 weeks, or placebo for 16 weeks followed by bimekizumab every 2 weeks.
- The primary outcome was an HS clinical response of at least 50% (HiSCR50) at week 16, defined as at least a 50% reduction in total abscess and inflammatory nodule count.
TAKEAWAY:
- A higher proportion of patients receiving bimekizumab every 2 weeks vs placebo achieved an HiSCR50 response at week 16 in BE HEARD I (48% vs 29%; odds ratio [OR], 2.23; P = .006) and II (52% vs 32%; OR, 2.29; P = .0032) trials.
- Patients receiving bimekizumab every 4 weeks also achieved a higher HiSCR50 response at week 16 vs placebo in the BE HEARD II trial (54% vs 32%; OR, 2.42; P = .0038).
- At week 16, a higher proportion of patients receiving bimekizumab every 2 weeks vs placebo achieved at least a 75% HiSCR (HiSCR75) in both trials, and a higher proportion of those receiving bimekizumab every 4 weeks achieved HiSCR75 in the BE HEARD II trial.
- At week 48, 45%-68% of patients achieved HiSCR50 in both trials.
- Patients who received bimekizumab vs placebo for the initial 16 weeks had greater improvements in patient-reported outcomes, and bimekizumab was well tolerated with a low number of serious or severe treatment-emergent adverse events.
IN PRACTICE:
“Bimekizumab was well tolerated by patients with hidradenitis suppurativa and produced rapid and deep clinically meaningful responses that were maintained up to 48 weeks,” the authors wrote. “These data support the use of bimekizumab as a promising new therapeutic option for patients with moderate to severe hidradenitis suppurativa.”
SOURCE:
Alexa B. Kimball, MD, MPH, from Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, led this study, which was published online in The Lancet.
LIMITATIONS:
The placebo-controlled part of this trial was relatively short at 16 weeks and may affect the interpretation of later efficacy data, there was a lack of an active comparator group, and the efficacy of treatment was evaluated in the presence of rescue treatment with systemic antibiotics.
DISCLOSURES:
The studies were funded by bimekizumab manufacturer UCB Pharma. Seven authors disclosed being current or former employees of UCB Pharma. Other authors reported several ties with many companies, including UCB Pharma.
A version of this article first appeared on Medscape.com.
TOPLINE:
, in two phase 3 studies.
METHODOLOGY:
- To assess the efficacy and safety of bimekizumab, an interleukin (IL)-17A and IL-17F antagonist, 320 mg for HS, researchers conducted two 48-week phase 3 trials BE HEARD I (n = 505) and II (n = 509), which enrolled patients with moderate to severe HS and a history of inadequate response to systemic antibiotics.
- Patients were randomly assigned to one of four groups: Bimekizumab every 2 weeks, bimekizumab every 2 weeks for 16 weeks followed by every 4 weeks of dosing, bimekizumab every 4 weeks, or placebo for 16 weeks followed by bimekizumab every 2 weeks.
- The primary outcome was an HS clinical response of at least 50% (HiSCR50) at week 16, defined as at least a 50% reduction in total abscess and inflammatory nodule count.
TAKEAWAY:
- A higher proportion of patients receiving bimekizumab every 2 weeks vs placebo achieved an HiSCR50 response at week 16 in BE HEARD I (48% vs 29%; odds ratio [OR], 2.23; P = .006) and II (52% vs 32%; OR, 2.29; P = .0032) trials.
- Patients receiving bimekizumab every 4 weeks also achieved a higher HiSCR50 response at week 16 vs placebo in the BE HEARD II trial (54% vs 32%; OR, 2.42; P = .0038).
- At week 16, a higher proportion of patients receiving bimekizumab every 2 weeks vs placebo achieved at least a 75% HiSCR (HiSCR75) in both trials, and a higher proportion of those receiving bimekizumab every 4 weeks achieved HiSCR75 in the BE HEARD II trial.
- At week 48, 45%-68% of patients achieved HiSCR50 in both trials.
- Patients who received bimekizumab vs placebo for the initial 16 weeks had greater improvements in patient-reported outcomes, and bimekizumab was well tolerated with a low number of serious or severe treatment-emergent adverse events.
IN PRACTICE:
“Bimekizumab was well tolerated by patients with hidradenitis suppurativa and produced rapid and deep clinically meaningful responses that were maintained up to 48 weeks,” the authors wrote. “These data support the use of bimekizumab as a promising new therapeutic option for patients with moderate to severe hidradenitis suppurativa.”
SOURCE:
Alexa B. Kimball, MD, MPH, from Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, led this study, which was published online in The Lancet.
LIMITATIONS:
The placebo-controlled part of this trial was relatively short at 16 weeks and may affect the interpretation of later efficacy data, there was a lack of an active comparator group, and the efficacy of treatment was evaluated in the presence of rescue treatment with systemic antibiotics.
DISCLOSURES:
The studies were funded by bimekizumab manufacturer UCB Pharma. Seven authors disclosed being current or former employees of UCB Pharma. Other authors reported several ties with many companies, including UCB Pharma.
A version of this article first appeared on Medscape.com.
FMT Could Prevent Recurrence of Hepatic Encephalopathy in Patients With Cirrhosis
MILAN — , results of a phase 2 randomized controlled trial show.
“Not only was FMT more beneficial, but also it didn’t matter which route of administration was used — oral or enema — which is good because people don’t really like enemas,” said Jasmohan S. Bajaj, MD, AGAF, professor, School of Medicine, Virginia Commonwealth University, Richmond, and hepatologist at Richmond VA Medical Center.
Donor background (including vegan or omnivore) and dose range also did not affect the efficacy of FMT, Dr. Bajaj said.
Dr. Bajaj presented the findings (Abstract GS-001) at the opening session of the annual European Association for the Study of the Liver (EASL) Congress 2024.
Hepatic encephalopathy is a complication of advanced liver disease that causes a dementia-like state. Standard treatment with lactulose and rifaximin often results in a lack of patient response, meaning the patient is constantly being readmitted to the hospital, Dr. Bajaj said.
“This is a burden for the family as well as the patients,” and is very difficult to manage from a clinical and psychosocial perspective, he said in an interview.
With FMT, “we are transferring an ecosystem of good microbes,” which modifies the gut microbiome in patients with advanced liver disease and reduces associated brain toxicity, Dr. Bajaj explained.
Resetting the Gut
The double-blind, randomized, placebo-controlled trial enrolled a total of 60 patients with cirrhosis who had experienced hepatic encephalopathy. Aged 61-65 years, participants had Model for End-Stage Liver Disease (MELD) scores of 12-13, all were taking lactulose and rifaximin, and all had experienced their last hepatic encephalopathy episode 8-13 months prior.
Participants had similar baseline cognition, Sickness Impact Profile (SIP), and cirrhosis severity. Those with recent infections, taking other antibiotics, with a MELD score > 22, had received a transplant, or were immunosuppressed were excluded.
Study participants were divided into four dose administration groups (n = 15 each): oral and enema active FMT therapy (group 1), oral active FMT and enema placebo (group 2), oral placebo and enema active FMT (group 3), and oral and enema placebo (group 4).
The range of FMT dose frequency was zero (all placebo), or one, two, or three FMT administrations, each given 1 month apart.
Two thirds of those receiving active FMT were given omnivore-donor FMT, and one third were given vegan-donor FMT, in addition to receiving standard of care.
“Colony-forming units were standard and the same whether given via oral capsule or enema,” Dr. Bajaj said. This is “similar to what we used in our phase 1 study.”
Intent-to-treat (ITT) analysis was performed with 6-month data. The primary outcomes were safety and hepatic encephalopathy recurrence defined as ≥ grade 2 on West-Haven criteria. Secondary outcomes included other adverse events, changes in infections, severity of cirrhosis and cognition, and patient-reported outcomes. A statistical regression for hepatic encephalopathy recurrence was also performed. Patients were followed for 6 months or until death.
One Dose of FMT Better Than None
Hepatic encephalopathy recurrence was highest (40%) in group 4 patients, compared with those in group 1 (13%), group 2 (13%), and group 3 (0%), as were liver-related hospitalizations (47% vs 7%-20%).
SIP total/physical and psych scores improved with FMT (P = .003).
When all patients were included in the analysis, the hepatic encephalopathy recurrence was related to dose number (odds radio [OR], 0.27; 95% CI, 0.10-0.79; P = .02), male sex (OR, 0.16; 95% CI, 0.03-0.89; P = .04), and physical SIP (OR, 1.05; 95% CI, 1.01-1.10, P = .05). However, when analyzing results from FMT recipients only, FMT dose, route of administration, and donor source were not found to affect recurrence.
Of those on placebo alone, six patients (40%) had a recurrence, compared with four on FMT (8.8%) in the combined FMT groups.
“As long as a patient received at least one FMT dose, they had a better response than a patient who had none,” Dr. Bajaj said.
Six patients dropped out; two in group 1 died after hepatic encephalopathy and falls, and one in group 2 died after a seizure. Three others did not return for follow-up visits. Four patients developed infections, including spontaneous bacterial peritonitis, cholecystitis, and cellulitis, all unrelated to FMT.
“I think many patients in Western countries are underserved because apart from lactulose and rifaximin, there is little else to give them,” Dr. Bajaj said. “The assumption is because rifaximin kills everything, we shouldn’t give FMT. But here, we administered it to a harsh and hostile wasteland of microbiota, and it still got a toehold and generated a reduction in hepatic encephalopathy.”
He pointed out that in smaller prior studies, the effects lasted up to 1 year.
Setting the Stage for Phase 3 Trials
Dr. Bajaj noted that this phase 2 study sets the stage for larger phase 3 trials in patients not responding to first-line therapy.
“Given how well-tolerated and effective FMT appears to be in these patients, if the larger phase 3 trial shows similar results, I can imagine FMT becoming a standard therapy,” said Colleen R. Kelly, MD, AGAF, gastroenterologist at Brigham and Women’s Hospital and Harvard Medical School, Boston, who was not involved in the study.
This study was built on Dr. Bajaj’s prior work that established the safety of FMT by enema, she added, stressing that this new research was incredibly important in these immunocompromised patients who are at higher risk for infection transmission.
That the administration route doesn’t matter is also an important finding as oral administration is much more feasible than enema, said Dr. Kelly, who went on to point out the importance of finding an alternative to rifaximin and lactulose, which are often poorly tolerated.
The study highlights the central role played by the gut microbiota in dysbiosis in the pathophysiology of hepatic encephalopathy, Dr. Kelly said. “It is another exciting example of how gut microbiota can be manipulated to treat disease.”
Dr. Bajaj and Dr. Kelly report no relevant financial relationships to this study.
A version of this article appeared on Medscape.com.
MILAN — , results of a phase 2 randomized controlled trial show.
“Not only was FMT more beneficial, but also it didn’t matter which route of administration was used — oral or enema — which is good because people don’t really like enemas,” said Jasmohan S. Bajaj, MD, AGAF, professor, School of Medicine, Virginia Commonwealth University, Richmond, and hepatologist at Richmond VA Medical Center.
Donor background (including vegan or omnivore) and dose range also did not affect the efficacy of FMT, Dr. Bajaj said.
Dr. Bajaj presented the findings (Abstract GS-001) at the opening session of the annual European Association for the Study of the Liver (EASL) Congress 2024.
Hepatic encephalopathy is a complication of advanced liver disease that causes a dementia-like state. Standard treatment with lactulose and rifaximin often results in a lack of patient response, meaning the patient is constantly being readmitted to the hospital, Dr. Bajaj said.
“This is a burden for the family as well as the patients,” and is very difficult to manage from a clinical and psychosocial perspective, he said in an interview.
With FMT, “we are transferring an ecosystem of good microbes,” which modifies the gut microbiome in patients with advanced liver disease and reduces associated brain toxicity, Dr. Bajaj explained.
Resetting the Gut
The double-blind, randomized, placebo-controlled trial enrolled a total of 60 patients with cirrhosis who had experienced hepatic encephalopathy. Aged 61-65 years, participants had Model for End-Stage Liver Disease (MELD) scores of 12-13, all were taking lactulose and rifaximin, and all had experienced their last hepatic encephalopathy episode 8-13 months prior.
Participants had similar baseline cognition, Sickness Impact Profile (SIP), and cirrhosis severity. Those with recent infections, taking other antibiotics, with a MELD score > 22, had received a transplant, or were immunosuppressed were excluded.
Study participants were divided into four dose administration groups (n = 15 each): oral and enema active FMT therapy (group 1), oral active FMT and enema placebo (group 2), oral placebo and enema active FMT (group 3), and oral and enema placebo (group 4).
The range of FMT dose frequency was zero (all placebo), or one, two, or three FMT administrations, each given 1 month apart.
Two thirds of those receiving active FMT were given omnivore-donor FMT, and one third were given vegan-donor FMT, in addition to receiving standard of care.
“Colony-forming units were standard and the same whether given via oral capsule or enema,” Dr. Bajaj said. This is “similar to what we used in our phase 1 study.”
Intent-to-treat (ITT) analysis was performed with 6-month data. The primary outcomes were safety and hepatic encephalopathy recurrence defined as ≥ grade 2 on West-Haven criteria. Secondary outcomes included other adverse events, changes in infections, severity of cirrhosis and cognition, and patient-reported outcomes. A statistical regression for hepatic encephalopathy recurrence was also performed. Patients were followed for 6 months or until death.
One Dose of FMT Better Than None
Hepatic encephalopathy recurrence was highest (40%) in group 4 patients, compared with those in group 1 (13%), group 2 (13%), and group 3 (0%), as were liver-related hospitalizations (47% vs 7%-20%).
SIP total/physical and psych scores improved with FMT (P = .003).
When all patients were included in the analysis, the hepatic encephalopathy recurrence was related to dose number (odds radio [OR], 0.27; 95% CI, 0.10-0.79; P = .02), male sex (OR, 0.16; 95% CI, 0.03-0.89; P = .04), and physical SIP (OR, 1.05; 95% CI, 1.01-1.10, P = .05). However, when analyzing results from FMT recipients only, FMT dose, route of administration, and donor source were not found to affect recurrence.
Of those on placebo alone, six patients (40%) had a recurrence, compared with four on FMT (8.8%) in the combined FMT groups.
“As long as a patient received at least one FMT dose, they had a better response than a patient who had none,” Dr. Bajaj said.
Six patients dropped out; two in group 1 died after hepatic encephalopathy and falls, and one in group 2 died after a seizure. Three others did not return for follow-up visits. Four patients developed infections, including spontaneous bacterial peritonitis, cholecystitis, and cellulitis, all unrelated to FMT.
“I think many patients in Western countries are underserved because apart from lactulose and rifaximin, there is little else to give them,” Dr. Bajaj said. “The assumption is because rifaximin kills everything, we shouldn’t give FMT. But here, we administered it to a harsh and hostile wasteland of microbiota, and it still got a toehold and generated a reduction in hepatic encephalopathy.”
He pointed out that in smaller prior studies, the effects lasted up to 1 year.
Setting the Stage for Phase 3 Trials
Dr. Bajaj noted that this phase 2 study sets the stage for larger phase 3 trials in patients not responding to first-line therapy.
“Given how well-tolerated and effective FMT appears to be in these patients, if the larger phase 3 trial shows similar results, I can imagine FMT becoming a standard therapy,” said Colleen R. Kelly, MD, AGAF, gastroenterologist at Brigham and Women’s Hospital and Harvard Medical School, Boston, who was not involved in the study.
This study was built on Dr. Bajaj’s prior work that established the safety of FMT by enema, she added, stressing that this new research was incredibly important in these immunocompromised patients who are at higher risk for infection transmission.
That the administration route doesn’t matter is also an important finding as oral administration is much more feasible than enema, said Dr. Kelly, who went on to point out the importance of finding an alternative to rifaximin and lactulose, which are often poorly tolerated.
The study highlights the central role played by the gut microbiota in dysbiosis in the pathophysiology of hepatic encephalopathy, Dr. Kelly said. “It is another exciting example of how gut microbiota can be manipulated to treat disease.”
Dr. Bajaj and Dr. Kelly report no relevant financial relationships to this study.
A version of this article appeared on Medscape.com.
MILAN — , results of a phase 2 randomized controlled trial show.
“Not only was FMT more beneficial, but also it didn’t matter which route of administration was used — oral or enema — which is good because people don’t really like enemas,” said Jasmohan S. Bajaj, MD, AGAF, professor, School of Medicine, Virginia Commonwealth University, Richmond, and hepatologist at Richmond VA Medical Center.
Donor background (including vegan or omnivore) and dose range also did not affect the efficacy of FMT, Dr. Bajaj said.
Dr. Bajaj presented the findings (Abstract GS-001) at the opening session of the annual European Association for the Study of the Liver (EASL) Congress 2024.
Hepatic encephalopathy is a complication of advanced liver disease that causes a dementia-like state. Standard treatment with lactulose and rifaximin often results in a lack of patient response, meaning the patient is constantly being readmitted to the hospital, Dr. Bajaj said.
“This is a burden for the family as well as the patients,” and is very difficult to manage from a clinical and psychosocial perspective, he said in an interview.
With FMT, “we are transferring an ecosystem of good microbes,” which modifies the gut microbiome in patients with advanced liver disease and reduces associated brain toxicity, Dr. Bajaj explained.
Resetting the Gut
The double-blind, randomized, placebo-controlled trial enrolled a total of 60 patients with cirrhosis who had experienced hepatic encephalopathy. Aged 61-65 years, participants had Model for End-Stage Liver Disease (MELD) scores of 12-13, all were taking lactulose and rifaximin, and all had experienced their last hepatic encephalopathy episode 8-13 months prior.
Participants had similar baseline cognition, Sickness Impact Profile (SIP), and cirrhosis severity. Those with recent infections, taking other antibiotics, with a MELD score > 22, had received a transplant, or were immunosuppressed were excluded.
Study participants were divided into four dose administration groups (n = 15 each): oral and enema active FMT therapy (group 1), oral active FMT and enema placebo (group 2), oral placebo and enema active FMT (group 3), and oral and enema placebo (group 4).
The range of FMT dose frequency was zero (all placebo), or one, two, or three FMT administrations, each given 1 month apart.
Two thirds of those receiving active FMT were given omnivore-donor FMT, and one third were given vegan-donor FMT, in addition to receiving standard of care.
“Colony-forming units were standard and the same whether given via oral capsule or enema,” Dr. Bajaj said. This is “similar to what we used in our phase 1 study.”
Intent-to-treat (ITT) analysis was performed with 6-month data. The primary outcomes were safety and hepatic encephalopathy recurrence defined as ≥ grade 2 on West-Haven criteria. Secondary outcomes included other adverse events, changes in infections, severity of cirrhosis and cognition, and patient-reported outcomes. A statistical regression for hepatic encephalopathy recurrence was also performed. Patients were followed for 6 months or until death.
One Dose of FMT Better Than None
Hepatic encephalopathy recurrence was highest (40%) in group 4 patients, compared with those in group 1 (13%), group 2 (13%), and group 3 (0%), as were liver-related hospitalizations (47% vs 7%-20%).
SIP total/physical and psych scores improved with FMT (P = .003).
When all patients were included in the analysis, the hepatic encephalopathy recurrence was related to dose number (odds radio [OR], 0.27; 95% CI, 0.10-0.79; P = .02), male sex (OR, 0.16; 95% CI, 0.03-0.89; P = .04), and physical SIP (OR, 1.05; 95% CI, 1.01-1.10, P = .05). However, when analyzing results from FMT recipients only, FMT dose, route of administration, and donor source were not found to affect recurrence.
Of those on placebo alone, six patients (40%) had a recurrence, compared with four on FMT (8.8%) in the combined FMT groups.
“As long as a patient received at least one FMT dose, they had a better response than a patient who had none,” Dr. Bajaj said.
Six patients dropped out; two in group 1 died after hepatic encephalopathy and falls, and one in group 2 died after a seizure. Three others did not return for follow-up visits. Four patients developed infections, including spontaneous bacterial peritonitis, cholecystitis, and cellulitis, all unrelated to FMT.
“I think many patients in Western countries are underserved because apart from lactulose and rifaximin, there is little else to give them,” Dr. Bajaj said. “The assumption is because rifaximin kills everything, we shouldn’t give FMT. But here, we administered it to a harsh and hostile wasteland of microbiota, and it still got a toehold and generated a reduction in hepatic encephalopathy.”
He pointed out that in smaller prior studies, the effects lasted up to 1 year.
Setting the Stage for Phase 3 Trials
Dr. Bajaj noted that this phase 2 study sets the stage for larger phase 3 trials in patients not responding to first-line therapy.
“Given how well-tolerated and effective FMT appears to be in these patients, if the larger phase 3 trial shows similar results, I can imagine FMT becoming a standard therapy,” said Colleen R. Kelly, MD, AGAF, gastroenterologist at Brigham and Women’s Hospital and Harvard Medical School, Boston, who was not involved in the study.
This study was built on Dr. Bajaj’s prior work that established the safety of FMT by enema, she added, stressing that this new research was incredibly important in these immunocompromised patients who are at higher risk for infection transmission.
That the administration route doesn’t matter is also an important finding as oral administration is much more feasible than enema, said Dr. Kelly, who went on to point out the importance of finding an alternative to rifaximin and lactulose, which are often poorly tolerated.
The study highlights the central role played by the gut microbiota in dysbiosis in the pathophysiology of hepatic encephalopathy, Dr. Kelly said. “It is another exciting example of how gut microbiota can be manipulated to treat disease.”
Dr. Bajaj and Dr. Kelly report no relevant financial relationships to this study.
A version of this article appeared on Medscape.com.
FROM EASL 2024
The Positive Effects of Exercise in MS
NASHVILLE, TENNESSEE — Exercise has a long history in multiple sclerosis (MS). In 1838, the Scottish physician John Abercrombie reported that a patient with “a diminution of muscular power,” who could walk but only unsteadily, decided after various failed treatments like “evacuations and spare diet” to try “violent exercise.” He walked 5-6 miles on a warm evening, as quickly as he was able, and returned home “much fatigued, and considerably heated. Next morning he had severe pains in the calves of his legs, but his other complaints were much diminished, and in a few days disappeared. He has ever since enjoyed good health,” Dr. Abercrombie was quoted in Multiple Sclerosis: The History of a Disease by T. Jock Murray.
The first randomized, controlled trial of an exercise intervention for MS didn’t appear in the literature until 1988, but more than 200 have been published in the years since, according to Robert Motl, PhD, who spoke about exercise interventions for MS at the annual meeting of the Consortium of Multiple Sclerosis Centers.
In fact, the evidence shows that exercise can improve walking performance and quality of life. “When we look at what we might call the unseen symptoms, we can see the exercise training is very effective at reducing fatigue in people with MS. It’s very effective at reducing depressive mood in individuals living with MS. There is moderate evidence that it can improve mobility, particularly lower extremity mobility and walking performance in individuals living with multiple sclerosis, as well as balance. And lastly, we see consistent evidence that exercise training can improve quality of life,” said Dr. Motl, who is a professor of kinesiology and nutrition at University of Illinois, Chicago.
There is less evidence that exercise training helps mobility, anxiety, pain, and participation, he said.
Dr. Motl showed the results of various meta-analyses that he co-authored of randomized, controlled trials (RCTs) of exercise training. One meta-analysis of 20 trials that examined the effect on fitness found an effect size of 0.47, which was about one-half of a standard deviation, and is considered to be a clinically meaningful effect. There was also about a 20% improvement in aerobic capacity, and this improves the capacity for maintaining independence, according to Dr. Motl. “That’s huge as individuals who are living with MS over a long-term period of time are aging with this chronic disease and independence does become an issue later in life. We maybe can forestall some of that,” he said.
Another meta-analysis of 17 RCTs examining exercise training and fatigue found a similar effect size of 0.452. When the authors limited the analysis to studies that used the Fatigue Severity Score and its benchmark of clinically significant fatigue of 4.0, “they were able to reduce the mean fatigue severity score below 4.0, meaning you’re taking individuals who have severe fatigue and reducing their fatigue below a threshold of severity that impacts everyday life. So this is something that is clinically meaningful and relevant to the lives of individuals with MS,” he said.
With respect to depression, a meta-analysis of 14 randomized, controlled trials found an effect size of 0.55 standard deviations. The researchers found that the effect size was associated with the number of days per week: The effect was size was doubled among individuals who exercised 3 or more times per week. Another meta-analysis of walking found an average 2-second improvement in walking speed and about a 40-meter improvement in walking endurance. “I believe that’s pretty comparable to what you see with Ampyra (dalfampridine) and its effects on walking speeds, so we’re seeing something that’s as good as a pharmacological agent for managing walking in MS,” said Dr. Motl.
Another meta-analysis of health-related quality of life found that the effect on the physical domain was about twice as large as the effect on mental health–related quality of life. “I think that makes sense because when you are engaging in exercise, it’s a physically invoking stimulus. As you see adaptations, your perceptions of your physical health improve,” said Dr. Motl.
Dr. Motl also addressed safety. There have been some concerns that exercise could lead to temporary worsening of symptoms, “but it was blown up into a major, major problem when it is only 5% of individuals who have these sorts of severe problems,” said Dr. Motl. A systematic review in 2023 found an adverse event rate of 1.2% in the control groups and 2.0% in the exercise groups. This was about the same rates that are seen in the general population, according to Dr. Motl. A consistent adverse event was lower back pain, but further analysis showed it was only reported with resistance training. “The beauty of that is that we have incredible people in the field of MS, who know how to deliver resistance training more safely. And if we do that more effectively, we can avoid this very common injury with exercise training,” said Dr. Motl.
The review also found a 25% reduction in relapses. “It was very interesting. I don’t know if we want to say exercise is a disease-modifying behavior yet, but that effect at the time that these studies were done was about the same as some of the early disease-modifying therapies, showing the same degree of reduction of relapse rate,” said Dr. Motl.
Dr. Motl also discussed updated guidelines for exercise in patients with mild to moderate MS, as well as Parkinson’s disease and stroke survivors. The general advice is for 2-3 days of moderate aerobic exercise per week, beginning at 10 minutes and gradually increasing to 30 minutes per session. The newer guidelines added an option for advanced aerobic exercise, which can be up to 5 times per week and up to 40 minutes per session. Activities include ergometry, walking, aquatics, and elliptical machines for general aerobic exercise, while advanced exercise can also include running or road cycling. Resistance exercise can be done 2-3 times per week with 1-3 sets of 8-15 repetitions, with a total of 5-10 exercises. The authors recommend weight machines, free weights, or resistance bands.
Dr. Motl has received funding from the Department of Defense, National Institutes of Health, Patient-Centered Outcomes Research Institute, National Multiple Sclerosis Society, and Bristol Myers Squibb Foundation.
NASHVILLE, TENNESSEE — Exercise has a long history in multiple sclerosis (MS). In 1838, the Scottish physician John Abercrombie reported that a patient with “a diminution of muscular power,” who could walk but only unsteadily, decided after various failed treatments like “evacuations and spare diet” to try “violent exercise.” He walked 5-6 miles on a warm evening, as quickly as he was able, and returned home “much fatigued, and considerably heated. Next morning he had severe pains in the calves of his legs, but his other complaints were much diminished, and in a few days disappeared. He has ever since enjoyed good health,” Dr. Abercrombie was quoted in Multiple Sclerosis: The History of a Disease by T. Jock Murray.
The first randomized, controlled trial of an exercise intervention for MS didn’t appear in the literature until 1988, but more than 200 have been published in the years since, according to Robert Motl, PhD, who spoke about exercise interventions for MS at the annual meeting of the Consortium of Multiple Sclerosis Centers.
In fact, the evidence shows that exercise can improve walking performance and quality of life. “When we look at what we might call the unseen symptoms, we can see the exercise training is very effective at reducing fatigue in people with MS. It’s very effective at reducing depressive mood in individuals living with MS. There is moderate evidence that it can improve mobility, particularly lower extremity mobility and walking performance in individuals living with multiple sclerosis, as well as balance. And lastly, we see consistent evidence that exercise training can improve quality of life,” said Dr. Motl, who is a professor of kinesiology and nutrition at University of Illinois, Chicago.
There is less evidence that exercise training helps mobility, anxiety, pain, and participation, he said.
Dr. Motl showed the results of various meta-analyses that he co-authored of randomized, controlled trials (RCTs) of exercise training. One meta-analysis of 20 trials that examined the effect on fitness found an effect size of 0.47, which was about one-half of a standard deviation, and is considered to be a clinically meaningful effect. There was also about a 20% improvement in aerobic capacity, and this improves the capacity for maintaining independence, according to Dr. Motl. “That’s huge as individuals who are living with MS over a long-term period of time are aging with this chronic disease and independence does become an issue later in life. We maybe can forestall some of that,” he said.
Another meta-analysis of 17 RCTs examining exercise training and fatigue found a similar effect size of 0.452. When the authors limited the analysis to studies that used the Fatigue Severity Score and its benchmark of clinically significant fatigue of 4.0, “they were able to reduce the mean fatigue severity score below 4.0, meaning you’re taking individuals who have severe fatigue and reducing their fatigue below a threshold of severity that impacts everyday life. So this is something that is clinically meaningful and relevant to the lives of individuals with MS,” he said.
With respect to depression, a meta-analysis of 14 randomized, controlled trials found an effect size of 0.55 standard deviations. The researchers found that the effect size was associated with the number of days per week: The effect was size was doubled among individuals who exercised 3 or more times per week. Another meta-analysis of walking found an average 2-second improvement in walking speed and about a 40-meter improvement in walking endurance. “I believe that’s pretty comparable to what you see with Ampyra (dalfampridine) and its effects on walking speeds, so we’re seeing something that’s as good as a pharmacological agent for managing walking in MS,” said Dr. Motl.
Another meta-analysis of health-related quality of life found that the effect on the physical domain was about twice as large as the effect on mental health–related quality of life. “I think that makes sense because when you are engaging in exercise, it’s a physically invoking stimulus. As you see adaptations, your perceptions of your physical health improve,” said Dr. Motl.
Dr. Motl also addressed safety. There have been some concerns that exercise could lead to temporary worsening of symptoms, “but it was blown up into a major, major problem when it is only 5% of individuals who have these sorts of severe problems,” said Dr. Motl. A systematic review in 2023 found an adverse event rate of 1.2% in the control groups and 2.0% in the exercise groups. This was about the same rates that are seen in the general population, according to Dr. Motl. A consistent adverse event was lower back pain, but further analysis showed it was only reported with resistance training. “The beauty of that is that we have incredible people in the field of MS, who know how to deliver resistance training more safely. And if we do that more effectively, we can avoid this very common injury with exercise training,” said Dr. Motl.
The review also found a 25% reduction in relapses. “It was very interesting. I don’t know if we want to say exercise is a disease-modifying behavior yet, but that effect at the time that these studies were done was about the same as some of the early disease-modifying therapies, showing the same degree of reduction of relapse rate,” said Dr. Motl.
Dr. Motl also discussed updated guidelines for exercise in patients with mild to moderate MS, as well as Parkinson’s disease and stroke survivors. The general advice is for 2-3 days of moderate aerobic exercise per week, beginning at 10 minutes and gradually increasing to 30 minutes per session. The newer guidelines added an option for advanced aerobic exercise, which can be up to 5 times per week and up to 40 minutes per session. Activities include ergometry, walking, aquatics, and elliptical machines for general aerobic exercise, while advanced exercise can also include running or road cycling. Resistance exercise can be done 2-3 times per week with 1-3 sets of 8-15 repetitions, with a total of 5-10 exercises. The authors recommend weight machines, free weights, or resistance bands.
Dr. Motl has received funding from the Department of Defense, National Institutes of Health, Patient-Centered Outcomes Research Institute, National Multiple Sclerosis Society, and Bristol Myers Squibb Foundation.
NASHVILLE, TENNESSEE — Exercise has a long history in multiple sclerosis (MS). In 1838, the Scottish physician John Abercrombie reported that a patient with “a diminution of muscular power,” who could walk but only unsteadily, decided after various failed treatments like “evacuations and spare diet” to try “violent exercise.” He walked 5-6 miles on a warm evening, as quickly as he was able, and returned home “much fatigued, and considerably heated. Next morning he had severe pains in the calves of his legs, but his other complaints were much diminished, and in a few days disappeared. He has ever since enjoyed good health,” Dr. Abercrombie was quoted in Multiple Sclerosis: The History of a Disease by T. Jock Murray.
The first randomized, controlled trial of an exercise intervention for MS didn’t appear in the literature until 1988, but more than 200 have been published in the years since, according to Robert Motl, PhD, who spoke about exercise interventions for MS at the annual meeting of the Consortium of Multiple Sclerosis Centers.
In fact, the evidence shows that exercise can improve walking performance and quality of life. “When we look at what we might call the unseen symptoms, we can see the exercise training is very effective at reducing fatigue in people with MS. It’s very effective at reducing depressive mood in individuals living with MS. There is moderate evidence that it can improve mobility, particularly lower extremity mobility and walking performance in individuals living with multiple sclerosis, as well as balance. And lastly, we see consistent evidence that exercise training can improve quality of life,” said Dr. Motl, who is a professor of kinesiology and nutrition at University of Illinois, Chicago.
There is less evidence that exercise training helps mobility, anxiety, pain, and participation, he said.
Dr. Motl showed the results of various meta-analyses that he co-authored of randomized, controlled trials (RCTs) of exercise training. One meta-analysis of 20 trials that examined the effect on fitness found an effect size of 0.47, which was about one-half of a standard deviation, and is considered to be a clinically meaningful effect. There was also about a 20% improvement in aerobic capacity, and this improves the capacity for maintaining independence, according to Dr. Motl. “That’s huge as individuals who are living with MS over a long-term period of time are aging with this chronic disease and independence does become an issue later in life. We maybe can forestall some of that,” he said.
Another meta-analysis of 17 RCTs examining exercise training and fatigue found a similar effect size of 0.452. When the authors limited the analysis to studies that used the Fatigue Severity Score and its benchmark of clinically significant fatigue of 4.0, “they were able to reduce the mean fatigue severity score below 4.0, meaning you’re taking individuals who have severe fatigue and reducing their fatigue below a threshold of severity that impacts everyday life. So this is something that is clinically meaningful and relevant to the lives of individuals with MS,” he said.
With respect to depression, a meta-analysis of 14 randomized, controlled trials found an effect size of 0.55 standard deviations. The researchers found that the effect size was associated with the number of days per week: The effect was size was doubled among individuals who exercised 3 or more times per week. Another meta-analysis of walking found an average 2-second improvement in walking speed and about a 40-meter improvement in walking endurance. “I believe that’s pretty comparable to what you see with Ampyra (dalfampridine) and its effects on walking speeds, so we’re seeing something that’s as good as a pharmacological agent for managing walking in MS,” said Dr. Motl.
Another meta-analysis of health-related quality of life found that the effect on the physical domain was about twice as large as the effect on mental health–related quality of life. “I think that makes sense because when you are engaging in exercise, it’s a physically invoking stimulus. As you see adaptations, your perceptions of your physical health improve,” said Dr. Motl.
Dr. Motl also addressed safety. There have been some concerns that exercise could lead to temporary worsening of symptoms, “but it was blown up into a major, major problem when it is only 5% of individuals who have these sorts of severe problems,” said Dr. Motl. A systematic review in 2023 found an adverse event rate of 1.2% in the control groups and 2.0% in the exercise groups. This was about the same rates that are seen in the general population, according to Dr. Motl. A consistent adverse event was lower back pain, but further analysis showed it was only reported with resistance training. “The beauty of that is that we have incredible people in the field of MS, who know how to deliver resistance training more safely. And if we do that more effectively, we can avoid this very common injury with exercise training,” said Dr. Motl.
The review also found a 25% reduction in relapses. “It was very interesting. I don’t know if we want to say exercise is a disease-modifying behavior yet, but that effect at the time that these studies were done was about the same as some of the early disease-modifying therapies, showing the same degree of reduction of relapse rate,” said Dr. Motl.
Dr. Motl also discussed updated guidelines for exercise in patients with mild to moderate MS, as well as Parkinson’s disease and stroke survivors. The general advice is for 2-3 days of moderate aerobic exercise per week, beginning at 10 minutes and gradually increasing to 30 minutes per session. The newer guidelines added an option for advanced aerobic exercise, which can be up to 5 times per week and up to 40 minutes per session. Activities include ergometry, walking, aquatics, and elliptical machines for general aerobic exercise, while advanced exercise can also include running or road cycling. Resistance exercise can be done 2-3 times per week with 1-3 sets of 8-15 repetitions, with a total of 5-10 exercises. The authors recommend weight machines, free weights, or resistance bands.
Dr. Motl has received funding from the Department of Defense, National Institutes of Health, Patient-Centered Outcomes Research Institute, National Multiple Sclerosis Society, and Bristol Myers Squibb Foundation.
FROM CMSC 2024
Congratulations to the 2024 AGA Research Foundation awardees!
The program serves as a catalyst for discovery and career growth among the most promising researchers in gastroenterology and hepatology.
“This year’s awardees are an exceptional group of investigators who are committed to furthering patient care through research,” said Michael Camilleri, MD, AGAF, chair, AGA Research Foundation. “The AGA Research Foundation is proud to fund these investigators and their ongoing efforts to advance GI research at a critical time in their careers. We believe the Foundation’s investment will ultimately enable new discoveries in gastroenterology and hepatology that will benefit patients.”
Treatment options for digestive diseases begin with vigorous research. The AGA Research Foundation supports medical investigators as they advance our understanding of gastrointestinal and liver conditions. Here are this year’s award recipients:
RESEARCH SCHOLAR AWARDS
AGA Research Scholar Award
- Karen Jane Dunbar, PhD, Columbia University, New York, New York
- Aaron Hecht, MD, PhD, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania
- Sarah Maxwell, MD, University of California, San Francisco
- Chung Sang Tse, MD, University of Pennsylvania, Philadelphia, Pennsylvania
- Jason (Yanjia) Zhang, MD, PhD, Boston Children’s Hospital, Massachusetts
AGA-Bristol Myers Squibb Research Scholar Award in Inflammatory Bowel Disease
- Joseph R. Burclaff, PhD, University of North Carolina at Chapel Hill
SPECIALTY AWARDS
AGA-Caroline Craig Augustyn & Damian Augustyn Award in Digestive Cancer
- Swathi Eluri, MD, MSCR, Mayo Clinic, Rochester, Minnesota
AGA-R. Robert & Sally Funderburg Research Award in Gastric Cancer
- Jianwen Que, MD, PhD, Columbia University, New York, New York
AGA-Pfizer Fellowship-to-Faculty Transition Award
- Lianna Wood, MD, PhD, Boston Children’s Hospital, Massachusetts
AGA-Ironwood Fellowship-to-Faculty Transition Award
- ZeNan Li Chang, MD, PhD, Washington University School of Medicine, St. Louis, Missouri
PILOT AWARDS
AGA Pilot Research Award
- Linda C. Cummings, MD, MS, University Hospitals Cleveland Medical Center, Cleveland, Ohio
- Pooja Mehta, MD, MSCS, University of Colorado Denver
- Guilherme Piovezani Ramos, MD, Boston Children’s Hospital
- Simon Schwoerer, PhD, University of Chicago, Illinois
- Yankai Wen, PhD, University of Texas Health Science Center at Houston
AGA-Pfizer Pilot Research Award in Non-Alcoholic Steatohepatitis
- Alice Cheng, PhD, Stanford University, California
- Petra Hirsova, PhD, PharmD, Mayo Clinic, Rochester, Minnesota
- Sarah Maxwell, MD, University of California, San Francisco
AGA-Pfizer Pilot Research Award in Inflammatory Bowel Disease
- David Boone, PhD, Indiana University, Indianapolis, Indiana
- Sara Chloe Di Rienzi, PhD, Baylor College of Medicine, Houston, Texas
- Jared Andrew Sninsky, MD, MSCR, Baylor College of Medicine, Houston, Texas
UNDERGRADUATE RESEARCH AWARDS
AGA-Aman Armaan Ahmed Family Surf for Success Program
- Eli Burstein, Yeshiva University, New York, New York
- Chloe Carlisle, University of Florida, Gainesville, Florida
- Adna Hassan, University of Minnesota Rochester
- Nicole Rodriguez Hilario, Barry University, Miami, Florida
- Maryam Jimoh, College of Wooster, Wooster, Ohio
- Viktoriya Kalinina, Brandeis University, Waltham, Massachusetts
AGA-Dr. Harvey Young Education & Development Foundation’s Young Guts Scholar Program
- Rafaella Lavalle Lacerda de Almeida, Michigan State University, East Lansing, Michigan
- Lara Cheesman, John’s Hopkins University School of Medicine, Baltimore, Maryland
- Cass Condray, University of Oklahoma, Norman, Oklahoma
- Daniel Juarez, Columbia University, New York, New York
- Jason Lin, University of Michigan, Ann Arbor, Michigan
- Riya Malhotra, Case Western Reserve University, Cleveland, Ohio
- Brian Nguyen, Brown University, Providence, Rhode Island
- Mahmoud (Moudy) Salem, Stony Brook University, Stony Brook, New York
ABSTRACT AWARDS
AGA Fellow Abstract of the Year Award
- Andrea Tou, MD Children’s Hospital of Philadelphia, Pennsylvania
AGA Fellow Abstract Awards
- Manik Aggarwal, MBBS, Mayo Clinic, Rochester, Minnesota
- Kole Buckley, PhD, University of Pennsylvania, Philadelphia, Pennsylvania
- Jane Ha, MD, Massachusetts General Hospital, Boston, Massachusetts
- Brent Hiramoto, MD, Brigham and Women’s Hospital, Boston, Massachusetts
- Md Obaidul Islam, PhD, University of Miami, Coral Gables, Florida
- Kanak Kennedy, MD, MPH, Children’s Hospital of Philadelphia, Pennsylvania
- Hanseul Kim, PhD, MS, Massachusetts General Hospital, Boston, Massachusetts
- Chiraag Kulkarni, MD, Stanford University, Stanford, California
- Su-Hyung Lee, PhD, DVM, Vanderbilt University Medical Center, Nashville, Tennessee
- Caroline Muiler, PhD, Yale School of Medicine, New Haven, Connecticut
- Sarah Najjar, PhD, New York University, New York, New York
- Ronaldo Panganiban, MD, PhD Penn State Hershey Medical Center, Hershey, Pennsylvania
- Perseus Patel, MD, Stanford University, California
- Hassan Sinan, MD, Johns Hopkins University, Baltimore, Maryland
- Patricia Snarski, PhD, Tulane University, New Orleans, Louisiana
- Fernando Vicentini, PhD, MS, McMaster University, Hamilton, Ontario, Canada
- Remington Winter, MD, University of Manitoba – Health Sciences Centre, Winnipeg, Manitoba, Canada
- Tiaosi Xing, PhD, MBBS, MS, Penn State College of Medicine, Hershey, Pennsylvania
AGA Student Abstract of the Year Award
- Jazmyne Jackson, Temple University, Philadelphia, Pennsylvania
AGA Student Abstract Award
- Valentina Alvarez, University of Washington School of Medicine, Seattle, Washington
- Yasaman Bahojb Habibyan, MS, University of Calgary, Alberta, Canada
- Tessa Herman, MD, University of Minnesota, Minneapolis-Saint Paul, Minnesota
- Jason Jin, Yale School of Medicine, New Haven, Connecticut
- Frederikke Larsen, Western University, London, Ontario, Canada
- Kara McNamara, Vanderbilt University, Nashville, Tennessee
- Julia Sessions, MD, University of Virginia, Charlottesville, Virginia
- Scott Silvey, MS, Virginia Commonwealth University, Richmond, Virginia
- Vijaya Sundaram, Marshall University School of Medicine, Huntington, West Virginia
- Kafayat Yusuf, MS, University of Kansas Medical Center, Kansas City, Kansas
AGA–Eric Esrailian Student Abstract Prize
- Brent Gawey, MD, MS, Mayo Clinic, Rochester, Minnesota
- Fei Li, MBBS, MS, University of Michigan, Ann Arbor, Michigan
- Emily Wong, University of Toronto, Ontario, Canada
- Jordan Woodard, MD, Prisma Health – Upstate, Greenville, South Carolina
AGA–Radhika Srinivasan Student Abstract Prize
- Raz Abdulqadir, MS, Penn State College of Medicine, Hershey, Pennsylvania
- Rebecca Ekeanyanwu, MHS, Meharry Medical College, Nashville, Tennessee
- Jared Morris, MD, University of Manitoba, Winnipeg City, Manitoba, Canada
- Rachel Stubler, Medical University of South Carolina, Charleston, South Carolina
AGA Abstract Award for Health Disparities Research
- Saqr Alsakarneh, MD University of Missouri-Kansas City
- Marco Noriega, MD, Beth Israel Deaconess Medical Center, Boston, Massachusetts
- Temitope Olasehinde, MD, University Hospitals/Case Western Reserve University, Cleveland, Ohio
- Gabrielle Waclawik, MD, MPH, University of Wisconsin, Madison, Wisconsin
AGA-Moti L. & Kamla Rustgi International Travel Award
- W. Keith Tan, MBChB, University of Cambridge, Cambridge, England
- Elsa van Liere, MD Amsterdam Universitair Medische Centra, Amsterdam, Netherlands
The program serves as a catalyst for discovery and career growth among the most promising researchers in gastroenterology and hepatology.
“This year’s awardees are an exceptional group of investigators who are committed to furthering patient care through research,” said Michael Camilleri, MD, AGAF, chair, AGA Research Foundation. “The AGA Research Foundation is proud to fund these investigators and their ongoing efforts to advance GI research at a critical time in their careers. We believe the Foundation’s investment will ultimately enable new discoveries in gastroenterology and hepatology that will benefit patients.”
Treatment options for digestive diseases begin with vigorous research. The AGA Research Foundation supports medical investigators as they advance our understanding of gastrointestinal and liver conditions. Here are this year’s award recipients:
RESEARCH SCHOLAR AWARDS
AGA Research Scholar Award
- Karen Jane Dunbar, PhD, Columbia University, New York, New York
- Aaron Hecht, MD, PhD, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania
- Sarah Maxwell, MD, University of California, San Francisco
- Chung Sang Tse, MD, University of Pennsylvania, Philadelphia, Pennsylvania
- Jason (Yanjia) Zhang, MD, PhD, Boston Children’s Hospital, Massachusetts
AGA-Bristol Myers Squibb Research Scholar Award in Inflammatory Bowel Disease
- Joseph R. Burclaff, PhD, University of North Carolina at Chapel Hill
SPECIALTY AWARDS
AGA-Caroline Craig Augustyn & Damian Augustyn Award in Digestive Cancer
- Swathi Eluri, MD, MSCR, Mayo Clinic, Rochester, Minnesota
AGA-R. Robert & Sally Funderburg Research Award in Gastric Cancer
- Jianwen Que, MD, PhD, Columbia University, New York, New York
AGA-Pfizer Fellowship-to-Faculty Transition Award
- Lianna Wood, MD, PhD, Boston Children’s Hospital, Massachusetts
AGA-Ironwood Fellowship-to-Faculty Transition Award
- ZeNan Li Chang, MD, PhD, Washington University School of Medicine, St. Louis, Missouri
PILOT AWARDS
AGA Pilot Research Award
- Linda C. Cummings, MD, MS, University Hospitals Cleveland Medical Center, Cleveland, Ohio
- Pooja Mehta, MD, MSCS, University of Colorado Denver
- Guilherme Piovezani Ramos, MD, Boston Children’s Hospital
- Simon Schwoerer, PhD, University of Chicago, Illinois
- Yankai Wen, PhD, University of Texas Health Science Center at Houston
AGA-Pfizer Pilot Research Award in Non-Alcoholic Steatohepatitis
- Alice Cheng, PhD, Stanford University, California
- Petra Hirsova, PhD, PharmD, Mayo Clinic, Rochester, Minnesota
- Sarah Maxwell, MD, University of California, San Francisco
AGA-Pfizer Pilot Research Award in Inflammatory Bowel Disease
- David Boone, PhD, Indiana University, Indianapolis, Indiana
- Sara Chloe Di Rienzi, PhD, Baylor College of Medicine, Houston, Texas
- Jared Andrew Sninsky, MD, MSCR, Baylor College of Medicine, Houston, Texas
UNDERGRADUATE RESEARCH AWARDS
AGA-Aman Armaan Ahmed Family Surf for Success Program
- Eli Burstein, Yeshiva University, New York, New York
- Chloe Carlisle, University of Florida, Gainesville, Florida
- Adna Hassan, University of Minnesota Rochester
- Nicole Rodriguez Hilario, Barry University, Miami, Florida
- Maryam Jimoh, College of Wooster, Wooster, Ohio
- Viktoriya Kalinina, Brandeis University, Waltham, Massachusetts
AGA-Dr. Harvey Young Education & Development Foundation’s Young Guts Scholar Program
- Rafaella Lavalle Lacerda de Almeida, Michigan State University, East Lansing, Michigan
- Lara Cheesman, John’s Hopkins University School of Medicine, Baltimore, Maryland
- Cass Condray, University of Oklahoma, Norman, Oklahoma
- Daniel Juarez, Columbia University, New York, New York
- Jason Lin, University of Michigan, Ann Arbor, Michigan
- Riya Malhotra, Case Western Reserve University, Cleveland, Ohio
- Brian Nguyen, Brown University, Providence, Rhode Island
- Mahmoud (Moudy) Salem, Stony Brook University, Stony Brook, New York
ABSTRACT AWARDS
AGA Fellow Abstract of the Year Award
- Andrea Tou, MD Children’s Hospital of Philadelphia, Pennsylvania
AGA Fellow Abstract Awards
- Manik Aggarwal, MBBS, Mayo Clinic, Rochester, Minnesota
- Kole Buckley, PhD, University of Pennsylvania, Philadelphia, Pennsylvania
- Jane Ha, MD, Massachusetts General Hospital, Boston, Massachusetts
- Brent Hiramoto, MD, Brigham and Women’s Hospital, Boston, Massachusetts
- Md Obaidul Islam, PhD, University of Miami, Coral Gables, Florida
- Kanak Kennedy, MD, MPH, Children’s Hospital of Philadelphia, Pennsylvania
- Hanseul Kim, PhD, MS, Massachusetts General Hospital, Boston, Massachusetts
- Chiraag Kulkarni, MD, Stanford University, Stanford, California
- Su-Hyung Lee, PhD, DVM, Vanderbilt University Medical Center, Nashville, Tennessee
- Caroline Muiler, PhD, Yale School of Medicine, New Haven, Connecticut
- Sarah Najjar, PhD, New York University, New York, New York
- Ronaldo Panganiban, MD, PhD Penn State Hershey Medical Center, Hershey, Pennsylvania
- Perseus Patel, MD, Stanford University, California
- Hassan Sinan, MD, Johns Hopkins University, Baltimore, Maryland
- Patricia Snarski, PhD, Tulane University, New Orleans, Louisiana
- Fernando Vicentini, PhD, MS, McMaster University, Hamilton, Ontario, Canada
- Remington Winter, MD, University of Manitoba – Health Sciences Centre, Winnipeg, Manitoba, Canada
- Tiaosi Xing, PhD, MBBS, MS, Penn State College of Medicine, Hershey, Pennsylvania
AGA Student Abstract of the Year Award
- Jazmyne Jackson, Temple University, Philadelphia, Pennsylvania
AGA Student Abstract Award
- Valentina Alvarez, University of Washington School of Medicine, Seattle, Washington
- Yasaman Bahojb Habibyan, MS, University of Calgary, Alberta, Canada
- Tessa Herman, MD, University of Minnesota, Minneapolis-Saint Paul, Minnesota
- Jason Jin, Yale School of Medicine, New Haven, Connecticut
- Frederikke Larsen, Western University, London, Ontario, Canada
- Kara McNamara, Vanderbilt University, Nashville, Tennessee
- Julia Sessions, MD, University of Virginia, Charlottesville, Virginia
- Scott Silvey, MS, Virginia Commonwealth University, Richmond, Virginia
- Vijaya Sundaram, Marshall University School of Medicine, Huntington, West Virginia
- Kafayat Yusuf, MS, University of Kansas Medical Center, Kansas City, Kansas
AGA–Eric Esrailian Student Abstract Prize
- Brent Gawey, MD, MS, Mayo Clinic, Rochester, Minnesota
- Fei Li, MBBS, MS, University of Michigan, Ann Arbor, Michigan
- Emily Wong, University of Toronto, Ontario, Canada
- Jordan Woodard, MD, Prisma Health – Upstate, Greenville, South Carolina
AGA–Radhika Srinivasan Student Abstract Prize
- Raz Abdulqadir, MS, Penn State College of Medicine, Hershey, Pennsylvania
- Rebecca Ekeanyanwu, MHS, Meharry Medical College, Nashville, Tennessee
- Jared Morris, MD, University of Manitoba, Winnipeg City, Manitoba, Canada
- Rachel Stubler, Medical University of South Carolina, Charleston, South Carolina
AGA Abstract Award for Health Disparities Research
- Saqr Alsakarneh, MD University of Missouri-Kansas City
- Marco Noriega, MD, Beth Israel Deaconess Medical Center, Boston, Massachusetts
- Temitope Olasehinde, MD, University Hospitals/Case Western Reserve University, Cleveland, Ohio
- Gabrielle Waclawik, MD, MPH, University of Wisconsin, Madison, Wisconsin
AGA-Moti L. & Kamla Rustgi International Travel Award
- W. Keith Tan, MBChB, University of Cambridge, Cambridge, England
- Elsa van Liere, MD Amsterdam Universitair Medische Centra, Amsterdam, Netherlands
The program serves as a catalyst for discovery and career growth among the most promising researchers in gastroenterology and hepatology.
“This year’s awardees are an exceptional group of investigators who are committed to furthering patient care through research,” said Michael Camilleri, MD, AGAF, chair, AGA Research Foundation. “The AGA Research Foundation is proud to fund these investigators and their ongoing efforts to advance GI research at a critical time in their careers. We believe the Foundation’s investment will ultimately enable new discoveries in gastroenterology and hepatology that will benefit patients.”
Treatment options for digestive diseases begin with vigorous research. The AGA Research Foundation supports medical investigators as they advance our understanding of gastrointestinal and liver conditions. Here are this year’s award recipients:
RESEARCH SCHOLAR AWARDS
AGA Research Scholar Award
- Karen Jane Dunbar, PhD, Columbia University, New York, New York
- Aaron Hecht, MD, PhD, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania
- Sarah Maxwell, MD, University of California, San Francisco
- Chung Sang Tse, MD, University of Pennsylvania, Philadelphia, Pennsylvania
- Jason (Yanjia) Zhang, MD, PhD, Boston Children’s Hospital, Massachusetts
AGA-Bristol Myers Squibb Research Scholar Award in Inflammatory Bowel Disease
- Joseph R. Burclaff, PhD, University of North Carolina at Chapel Hill
SPECIALTY AWARDS
AGA-Caroline Craig Augustyn & Damian Augustyn Award in Digestive Cancer
- Swathi Eluri, MD, MSCR, Mayo Clinic, Rochester, Minnesota
AGA-R. Robert & Sally Funderburg Research Award in Gastric Cancer
- Jianwen Que, MD, PhD, Columbia University, New York, New York
AGA-Pfizer Fellowship-to-Faculty Transition Award
- Lianna Wood, MD, PhD, Boston Children’s Hospital, Massachusetts
AGA-Ironwood Fellowship-to-Faculty Transition Award
- ZeNan Li Chang, MD, PhD, Washington University School of Medicine, St. Louis, Missouri
PILOT AWARDS
AGA Pilot Research Award
- Linda C. Cummings, MD, MS, University Hospitals Cleveland Medical Center, Cleveland, Ohio
- Pooja Mehta, MD, MSCS, University of Colorado Denver
- Guilherme Piovezani Ramos, MD, Boston Children’s Hospital
- Simon Schwoerer, PhD, University of Chicago, Illinois
- Yankai Wen, PhD, University of Texas Health Science Center at Houston
AGA-Pfizer Pilot Research Award in Non-Alcoholic Steatohepatitis
- Alice Cheng, PhD, Stanford University, California
- Petra Hirsova, PhD, PharmD, Mayo Clinic, Rochester, Minnesota
- Sarah Maxwell, MD, University of California, San Francisco
AGA-Pfizer Pilot Research Award in Inflammatory Bowel Disease
- David Boone, PhD, Indiana University, Indianapolis, Indiana
- Sara Chloe Di Rienzi, PhD, Baylor College of Medicine, Houston, Texas
- Jared Andrew Sninsky, MD, MSCR, Baylor College of Medicine, Houston, Texas
UNDERGRADUATE RESEARCH AWARDS
AGA-Aman Armaan Ahmed Family Surf for Success Program
- Eli Burstein, Yeshiva University, New York, New York
- Chloe Carlisle, University of Florida, Gainesville, Florida
- Adna Hassan, University of Minnesota Rochester
- Nicole Rodriguez Hilario, Barry University, Miami, Florida
- Maryam Jimoh, College of Wooster, Wooster, Ohio
- Viktoriya Kalinina, Brandeis University, Waltham, Massachusetts
AGA-Dr. Harvey Young Education & Development Foundation’s Young Guts Scholar Program
- Rafaella Lavalle Lacerda de Almeida, Michigan State University, East Lansing, Michigan
- Lara Cheesman, John’s Hopkins University School of Medicine, Baltimore, Maryland
- Cass Condray, University of Oklahoma, Norman, Oklahoma
- Daniel Juarez, Columbia University, New York, New York
- Jason Lin, University of Michigan, Ann Arbor, Michigan
- Riya Malhotra, Case Western Reserve University, Cleveland, Ohio
- Brian Nguyen, Brown University, Providence, Rhode Island
- Mahmoud (Moudy) Salem, Stony Brook University, Stony Brook, New York
ABSTRACT AWARDS
AGA Fellow Abstract of the Year Award
- Andrea Tou, MD Children’s Hospital of Philadelphia, Pennsylvania
AGA Fellow Abstract Awards
- Manik Aggarwal, MBBS, Mayo Clinic, Rochester, Minnesota
- Kole Buckley, PhD, University of Pennsylvania, Philadelphia, Pennsylvania
- Jane Ha, MD, Massachusetts General Hospital, Boston, Massachusetts
- Brent Hiramoto, MD, Brigham and Women’s Hospital, Boston, Massachusetts
- Md Obaidul Islam, PhD, University of Miami, Coral Gables, Florida
- Kanak Kennedy, MD, MPH, Children’s Hospital of Philadelphia, Pennsylvania
- Hanseul Kim, PhD, MS, Massachusetts General Hospital, Boston, Massachusetts
- Chiraag Kulkarni, MD, Stanford University, Stanford, California
- Su-Hyung Lee, PhD, DVM, Vanderbilt University Medical Center, Nashville, Tennessee
- Caroline Muiler, PhD, Yale School of Medicine, New Haven, Connecticut
- Sarah Najjar, PhD, New York University, New York, New York
- Ronaldo Panganiban, MD, PhD Penn State Hershey Medical Center, Hershey, Pennsylvania
- Perseus Patel, MD, Stanford University, California
- Hassan Sinan, MD, Johns Hopkins University, Baltimore, Maryland
- Patricia Snarski, PhD, Tulane University, New Orleans, Louisiana
- Fernando Vicentini, PhD, MS, McMaster University, Hamilton, Ontario, Canada
- Remington Winter, MD, University of Manitoba – Health Sciences Centre, Winnipeg, Manitoba, Canada
- Tiaosi Xing, PhD, MBBS, MS, Penn State College of Medicine, Hershey, Pennsylvania
AGA Student Abstract of the Year Award
- Jazmyne Jackson, Temple University, Philadelphia, Pennsylvania
AGA Student Abstract Award
- Valentina Alvarez, University of Washington School of Medicine, Seattle, Washington
- Yasaman Bahojb Habibyan, MS, University of Calgary, Alberta, Canada
- Tessa Herman, MD, University of Minnesota, Minneapolis-Saint Paul, Minnesota
- Jason Jin, Yale School of Medicine, New Haven, Connecticut
- Frederikke Larsen, Western University, London, Ontario, Canada
- Kara McNamara, Vanderbilt University, Nashville, Tennessee
- Julia Sessions, MD, University of Virginia, Charlottesville, Virginia
- Scott Silvey, MS, Virginia Commonwealth University, Richmond, Virginia
- Vijaya Sundaram, Marshall University School of Medicine, Huntington, West Virginia
- Kafayat Yusuf, MS, University of Kansas Medical Center, Kansas City, Kansas
AGA–Eric Esrailian Student Abstract Prize
- Brent Gawey, MD, MS, Mayo Clinic, Rochester, Minnesota
- Fei Li, MBBS, MS, University of Michigan, Ann Arbor, Michigan
- Emily Wong, University of Toronto, Ontario, Canada
- Jordan Woodard, MD, Prisma Health – Upstate, Greenville, South Carolina
AGA–Radhika Srinivasan Student Abstract Prize
- Raz Abdulqadir, MS, Penn State College of Medicine, Hershey, Pennsylvania
- Rebecca Ekeanyanwu, MHS, Meharry Medical College, Nashville, Tennessee
- Jared Morris, MD, University of Manitoba, Winnipeg City, Manitoba, Canada
- Rachel Stubler, Medical University of South Carolina, Charleston, South Carolina
AGA Abstract Award for Health Disparities Research
- Saqr Alsakarneh, MD University of Missouri-Kansas City
- Marco Noriega, MD, Beth Israel Deaconess Medical Center, Boston, Massachusetts
- Temitope Olasehinde, MD, University Hospitals/Case Western Reserve University, Cleveland, Ohio
- Gabrielle Waclawik, MD, MPH, University of Wisconsin, Madison, Wisconsin
AGA-Moti L. & Kamla Rustgi International Travel Award
- W. Keith Tan, MBChB, University of Cambridge, Cambridge, England
- Elsa van Liere, MD Amsterdam Universitair Medische Centra, Amsterdam, Netherlands
FIB-4 Index Misclassifies Many Patients
, potentially impacting clinical decisions, according to investigators.
These findings call for a cautious interpretation of low-risk FIB-4 results among patients at greatest risk of misclassification, and/or use of alternative assessment strategies, reported Mazen Noureddin, MD, MHSc, of Houston Methodist Hospital, and coauthors.
“Currently, the AGA/AASLD Pathways recommends identifying patients at risk for metabolic dysfunction-associated steatotic liver disease (MASLD), then using sequential testing with FIB-4 followed by FibroScan to risk-stratify patients,” the investigators wrote in Clinical Gastroenterology and Hepatology.
Yet the performance of the FIB-4 index in this context remains unclear.
“Previous studies have shown FIB-4 to have low accuracy for screening liver fibrosis, especially among obese and diabetic patients,” the investigators wrote. “Thus, there is a concern that classifying patients with FIB-4 can lead to misclassification and missed diagnosis.”
To explore this concern, Dr. Noureddin and colleagues turned to data from the 2017-2020 National Health and Nutrition Examination Surveys, including 5285 subjects at risk for MASLD. Exclusions were made for those with excessive alcohol intake or other liver diseases, resulting in a final cohort of 3741 individuals.
All subjects were classified as low-, indeterminate-, or high-risk for advanced liver fibrosis based on FIB-4 scores. These scores were then compared with liver stiffness measurements (LSM) obtained through transient elastography (FibroScan).
Out of 2776 subjects classified as low-risk by FIB-4, 277 (10%) were reclassified as higher risk by LSM, including 75 (2.7%) who were found to be at high risk. Out of 879 subjects with indeterminate FIB-4 scores, 37 (4.2%) were at high risk according to LSM. Finally, among the 86 subjects classified as high risk by FIB-4, 68 (79.1%) were reclassified as lower risk by LSM, including 54 (62.8%) who were deemed low risk.
Subjects misclassified as low risk by FIB-4 were typically older and had higher waist circumferences, body mass indices, glycohemoglobin A1c levels, fasting glucose levels, liver enzyme levels, diastolic blood pressures, controlled attenuation parameter scores, white blood cell counts, and alkaline phosphatase levels, but lower high-density lipoprotein and albumin levels (all P less than .05). They were also more likely to have prediabetes or diabetes.
“[I]t is important to acknowledge that 10% of the subjects were misclassified as low risk by FIB-4,” Dr. Noureddin and colleagues wrote, including 2.7% of patients who were actually high risk. “This misclassification of high-risk patients can lead to missed diagnoses, delaying crucial medical treatments or lifestyle interventions.”
They therefore suggested cautious interpretation of low-risk FIB-4 results among patients with factors predicting misclassification, or even use of alternative diagnostic strategies.
“Some possible alternatives to FIB-4 include new serum tests such NIS-2+, MASEF, SAFE score, and machine learning methods,” Dr. Noureddin and colleagues wrote. “However, additional confirmatory and cost-effective studies are required to validate the effectiveness of these tests, including studies conducted on the general population.”
The investigators disclosed relationships with AbbVie, Corcept, Galectin, and others.
, potentially impacting clinical decisions, according to investigators.
These findings call for a cautious interpretation of low-risk FIB-4 results among patients at greatest risk of misclassification, and/or use of alternative assessment strategies, reported Mazen Noureddin, MD, MHSc, of Houston Methodist Hospital, and coauthors.
“Currently, the AGA/AASLD Pathways recommends identifying patients at risk for metabolic dysfunction-associated steatotic liver disease (MASLD), then using sequential testing with FIB-4 followed by FibroScan to risk-stratify patients,” the investigators wrote in Clinical Gastroenterology and Hepatology.
Yet the performance of the FIB-4 index in this context remains unclear.
“Previous studies have shown FIB-4 to have low accuracy for screening liver fibrosis, especially among obese and diabetic patients,” the investigators wrote. “Thus, there is a concern that classifying patients with FIB-4 can lead to misclassification and missed diagnosis.”
To explore this concern, Dr. Noureddin and colleagues turned to data from the 2017-2020 National Health and Nutrition Examination Surveys, including 5285 subjects at risk for MASLD. Exclusions were made for those with excessive alcohol intake or other liver diseases, resulting in a final cohort of 3741 individuals.
All subjects were classified as low-, indeterminate-, or high-risk for advanced liver fibrosis based on FIB-4 scores. These scores were then compared with liver stiffness measurements (LSM) obtained through transient elastography (FibroScan).
Out of 2776 subjects classified as low-risk by FIB-4, 277 (10%) were reclassified as higher risk by LSM, including 75 (2.7%) who were found to be at high risk. Out of 879 subjects with indeterminate FIB-4 scores, 37 (4.2%) were at high risk according to LSM. Finally, among the 86 subjects classified as high risk by FIB-4, 68 (79.1%) were reclassified as lower risk by LSM, including 54 (62.8%) who were deemed low risk.
Subjects misclassified as low risk by FIB-4 were typically older and had higher waist circumferences, body mass indices, glycohemoglobin A1c levels, fasting glucose levels, liver enzyme levels, diastolic blood pressures, controlled attenuation parameter scores, white blood cell counts, and alkaline phosphatase levels, but lower high-density lipoprotein and albumin levels (all P less than .05). They were also more likely to have prediabetes or diabetes.
“[I]t is important to acknowledge that 10% of the subjects were misclassified as low risk by FIB-4,” Dr. Noureddin and colleagues wrote, including 2.7% of patients who were actually high risk. “This misclassification of high-risk patients can lead to missed diagnoses, delaying crucial medical treatments or lifestyle interventions.”
They therefore suggested cautious interpretation of low-risk FIB-4 results among patients with factors predicting misclassification, or even use of alternative diagnostic strategies.
“Some possible alternatives to FIB-4 include new serum tests such NIS-2+, MASEF, SAFE score, and machine learning methods,” Dr. Noureddin and colleagues wrote. “However, additional confirmatory and cost-effective studies are required to validate the effectiveness of these tests, including studies conducted on the general population.”
The investigators disclosed relationships with AbbVie, Corcept, Galectin, and others.
, potentially impacting clinical decisions, according to investigators.
These findings call for a cautious interpretation of low-risk FIB-4 results among patients at greatest risk of misclassification, and/or use of alternative assessment strategies, reported Mazen Noureddin, MD, MHSc, of Houston Methodist Hospital, and coauthors.
“Currently, the AGA/AASLD Pathways recommends identifying patients at risk for metabolic dysfunction-associated steatotic liver disease (MASLD), then using sequential testing with FIB-4 followed by FibroScan to risk-stratify patients,” the investigators wrote in Clinical Gastroenterology and Hepatology.
Yet the performance of the FIB-4 index in this context remains unclear.
“Previous studies have shown FIB-4 to have low accuracy for screening liver fibrosis, especially among obese and diabetic patients,” the investigators wrote. “Thus, there is a concern that classifying patients with FIB-4 can lead to misclassification and missed diagnosis.”
To explore this concern, Dr. Noureddin and colleagues turned to data from the 2017-2020 National Health and Nutrition Examination Surveys, including 5285 subjects at risk for MASLD. Exclusions were made for those with excessive alcohol intake or other liver diseases, resulting in a final cohort of 3741 individuals.
All subjects were classified as low-, indeterminate-, or high-risk for advanced liver fibrosis based on FIB-4 scores. These scores were then compared with liver stiffness measurements (LSM) obtained through transient elastography (FibroScan).
Out of 2776 subjects classified as low-risk by FIB-4, 277 (10%) were reclassified as higher risk by LSM, including 75 (2.7%) who were found to be at high risk. Out of 879 subjects with indeterminate FIB-4 scores, 37 (4.2%) were at high risk according to LSM. Finally, among the 86 subjects classified as high risk by FIB-4, 68 (79.1%) were reclassified as lower risk by LSM, including 54 (62.8%) who were deemed low risk.
Subjects misclassified as low risk by FIB-4 were typically older and had higher waist circumferences, body mass indices, glycohemoglobin A1c levels, fasting glucose levels, liver enzyme levels, diastolic blood pressures, controlled attenuation parameter scores, white blood cell counts, and alkaline phosphatase levels, but lower high-density lipoprotein and albumin levels (all P less than .05). They were also more likely to have prediabetes or diabetes.
“[I]t is important to acknowledge that 10% of the subjects were misclassified as low risk by FIB-4,” Dr. Noureddin and colleagues wrote, including 2.7% of patients who were actually high risk. “This misclassification of high-risk patients can lead to missed diagnoses, delaying crucial medical treatments or lifestyle interventions.”
They therefore suggested cautious interpretation of low-risk FIB-4 results among patients with factors predicting misclassification, or even use of alternative diagnostic strategies.
“Some possible alternatives to FIB-4 include new serum tests such NIS-2+, MASEF, SAFE score, and machine learning methods,” Dr. Noureddin and colleagues wrote. “However, additional confirmatory and cost-effective studies are required to validate the effectiveness of these tests, including studies conducted on the general population.”
The investigators disclosed relationships with AbbVie, Corcept, Galectin, and others.
FROM CLINICAL GASTROENTEROLOGY AND HEPATOLOGY
Introducing the 119th AGA President: Dr. Maria T. Abreu
She currently serves as the Martin Kalser Endowed Chair of Gastroenterology; professor of medicine, microbiology, and immunology; and director of the Crohn’s and Colitis Center at the University of Miami. Dr. Abreu is the fifth woman to lead AGA as president.
Born in New York and raised in New Jersey, Dr. Abreu grew up surrounded by a strong, tight-knit Cuban community. Her family moved to Miami when she was in the ninth grade. She later entered the 6-year medical program at the University of Miami, which was the beginning of her unparalleled academic and professional excellence in medicine.
Dr. Abreu is a leader in inflammatory bowel disease patient care, and she was honored by the prestigious Sherman Prize in 2019. Her service to AGA is lengthy and begins when she took on the role of fellow representative for the research grant committee. She has since sat on both the government advocacy and diversity committees. She also served as the chair of the Immunology, Microbiology and Inflammatory Bowel Diseases Section of the AGA Council, and later as chair of the full AGA Council. While chair she developed a more streamlined in-person planning committee meeting to better organize DDW.
When asked about goals for her presidency, Dr. Abreu wants to make DDW a better experience for the modern gastroenterologist. This includes finding that perfect balance between digesting the latest education and science with networking and socializing. She plans to collaborate with the presidents of the other societies to make this come to fruition.
Perhaps the area that Dr. Abreu is most passionate about is welcoming and fostering the growth of women in gastroenterology. She wants to support women who want to succeed in academics and in practice, who want ergonomics to match their work needs, and who want to have families.
“Maria is the ultimate ‘triple threat’: master scientist, master clinician, and devoted mentor. She has not only been a major player advancing knowledge in IBD, but also motivating and pushing others to develop successful careers,” said Andres Yarur, MD, AGAF, associate professor of medicine at Cedars-Sinai Medical Center. “Her work, brilliance, passion, and charm inspire all of us and will continue to inspire many generations to come.”
She currently serves as the Martin Kalser Endowed Chair of Gastroenterology; professor of medicine, microbiology, and immunology; and director of the Crohn’s and Colitis Center at the University of Miami. Dr. Abreu is the fifth woman to lead AGA as president.
Born in New York and raised in New Jersey, Dr. Abreu grew up surrounded by a strong, tight-knit Cuban community. Her family moved to Miami when she was in the ninth grade. She later entered the 6-year medical program at the University of Miami, which was the beginning of her unparalleled academic and professional excellence in medicine.
Dr. Abreu is a leader in inflammatory bowel disease patient care, and she was honored by the prestigious Sherman Prize in 2019. Her service to AGA is lengthy and begins when she took on the role of fellow representative for the research grant committee. She has since sat on both the government advocacy and diversity committees. She also served as the chair of the Immunology, Microbiology and Inflammatory Bowel Diseases Section of the AGA Council, and later as chair of the full AGA Council. While chair she developed a more streamlined in-person planning committee meeting to better organize DDW.
When asked about goals for her presidency, Dr. Abreu wants to make DDW a better experience for the modern gastroenterologist. This includes finding that perfect balance between digesting the latest education and science with networking and socializing. She plans to collaborate with the presidents of the other societies to make this come to fruition.
Perhaps the area that Dr. Abreu is most passionate about is welcoming and fostering the growth of women in gastroenterology. She wants to support women who want to succeed in academics and in practice, who want ergonomics to match their work needs, and who want to have families.
“Maria is the ultimate ‘triple threat’: master scientist, master clinician, and devoted mentor. She has not only been a major player advancing knowledge in IBD, but also motivating and pushing others to develop successful careers,” said Andres Yarur, MD, AGAF, associate professor of medicine at Cedars-Sinai Medical Center. “Her work, brilliance, passion, and charm inspire all of us and will continue to inspire many generations to come.”
She currently serves as the Martin Kalser Endowed Chair of Gastroenterology; professor of medicine, microbiology, and immunology; and director of the Crohn’s and Colitis Center at the University of Miami. Dr. Abreu is the fifth woman to lead AGA as president.
Born in New York and raised in New Jersey, Dr. Abreu grew up surrounded by a strong, tight-knit Cuban community. Her family moved to Miami when she was in the ninth grade. She later entered the 6-year medical program at the University of Miami, which was the beginning of her unparalleled academic and professional excellence in medicine.
Dr. Abreu is a leader in inflammatory bowel disease patient care, and she was honored by the prestigious Sherman Prize in 2019. Her service to AGA is lengthy and begins when she took on the role of fellow representative for the research grant committee. She has since sat on both the government advocacy and diversity committees. She also served as the chair of the Immunology, Microbiology and Inflammatory Bowel Diseases Section of the AGA Council, and later as chair of the full AGA Council. While chair she developed a more streamlined in-person planning committee meeting to better organize DDW.
When asked about goals for her presidency, Dr. Abreu wants to make DDW a better experience for the modern gastroenterologist. This includes finding that perfect balance between digesting the latest education and science with networking and socializing. She plans to collaborate with the presidents of the other societies to make this come to fruition.
Perhaps the area that Dr. Abreu is most passionate about is welcoming and fostering the growth of women in gastroenterology. She wants to support women who want to succeed in academics and in practice, who want ergonomics to match their work needs, and who want to have families.
“Maria is the ultimate ‘triple threat’: master scientist, master clinician, and devoted mentor. She has not only been a major player advancing knowledge in IBD, but also motivating and pushing others to develop successful careers,” said Andres Yarur, MD, AGAF, associate professor of medicine at Cedars-Sinai Medical Center. “Her work, brilliance, passion, and charm inspire all of us and will continue to inspire many generations to come.”
Chemo May Benefit Some Older Patients With Metastatic Pancreatic Cancer
TOPLINE:
METHODOLOGY:
Pancreatic cancer is most often diagnosed in adults aged 65 years or older. Providing cancer treatment for this older, often vulnerable, population comes with significant challenges and can lead to worse survival.
To examine real-world outcomes of older adults with untreated metastatic pancreatic cancer, researchers recruited patients aged 70 years or older and performed a geriatric assessment to identify comorbidities, cognitive issues, and other geriatric abnormalities.
Those who were deemed “fit” (ie, with no geriatric abnormalities) were assigned to receive off-study standard-of-care treatment, whereas those classified as “frail” (ie, with severe abnormalities) received off-study supportive care.
The remaining 176 “vulnerable” patients with mild to moderate geriatric abnormalities completed a geriatric and quality-of-life assessment and were then randomly assigned to receive either dose-reduced 5-fluorouracil (5-FU), leucovorin plus liposomal irinotecan (n = 88) or modified gemcitabine plus nab-paclitaxel (n = 88) every 2 weeks. Ultimately, 79 patients started the 5-FU combination and 75 received gemcitabine plus nab-paclitaxel. Patients were assessed every 8 weeks until disease progression or intolerance.
Overall, patients had a median age of 77 years; 61.9% were aged 75 years or older. About half were female, and 81.5% were White. The majority (87.5%) had a performance status of 0 or 1.
TAKEAWAY:
- Median overall survival was 4.7 months in the gemcitabine plus nab-paclitaxel arm and 4.4 months in the 5-FU combination group, with no significant survival difference observed between the two arms (P = .72).
- When the overall survival analysis was restricted to patients who received at least 4 weeks, or two cycles, of treatment (about 62% of patients), the median overall survival across the two treatment arms reached 8.0 months, in line with expectations for these regimens.
- Patient stratification revealed that those with a performance status of 2 had significantly worse overall survival than those with a status of 0: 1.4 months vs 6.9 months, respectively (hazard ratio [HR], 2.77; P < .001). A similar divide was seen when patients were stratified by physical/functional status and well-being. Age, however, did not significantly influence the results.
- Overall, more than half of patients experienced grade 3 or higher adverse events. Just over 38% of patients received only one to three cycles of therapy, whereas 26% remained on treatment for 12 or more cycles. The adverse event rates were similar between the two regimens, but the toxicity profile was slightly different — the researchers, for instance, observed more peripheral neuropathy with gemcitabine plus nab-paclitaxel and more diarrhea in the 5-FU combination arm.
IN PRACTICE:
- Overall, the “survival outcomes among vulnerable older patients were lower than expected, with high percentage of patients not able to start treatment, or complete one month of therapy due to clinical deterioration,” said study presenter Efrat Dotan, MD, chief, Division of Gastrointestinal Medical Oncology, Fox Chase Cancer Center, Philadelphia.
- “For vulnerable older adults who can tolerate treatment, these two regimens provide clinicians with options for tailoring therapy based on toxicity profile,” Dr. Dotan added. But “tools are needed to better identify patients who can benefit from treatment.”
- The results underline the need to perform geriatric assessments, as opposed to merely looking at performance status, commented David F. Chang, PhD, MS, MBBS, professor of Surgical Oncology, University of Glasgow, Scotland, who was not involved in the study.
SOURCE:
The research, presented at the 2024 annual meeting of the American Society of Clinical Oncology, was funded by the National Cancer Institute and the Eastern Cooperative Oncology Group.
LIMITATIONS:
Dr. Chang noted that the study did not reveal which treatment regimen was more effective.
DISCLOSURES:
Dr. Dotan declared relationships with Agenus, Amgen, G1 Therapeutics, Incyte, Olympus, and Taiho Pharmaceutical and institutional relationships with Dragonfly Therapeutics, Gilead Sciences, Ipsen, Kinnate Biopharma, Leap Therapeutics, Lilly, Lutris, NGM Biopharmaceuticals, Relay Therapeutics, and Zymeworks. Dr. Chang declared relationships with Immodulon Therapeutics and Mylan and institutional relationships with AstraZeneca, BMS GmbH & Co. KG, Immodulon Therapeutics, and Merck.
A version of this article appeared on Medscape.com.
TOPLINE:
METHODOLOGY:
Pancreatic cancer is most often diagnosed in adults aged 65 years or older. Providing cancer treatment for this older, often vulnerable, population comes with significant challenges and can lead to worse survival.
To examine real-world outcomes of older adults with untreated metastatic pancreatic cancer, researchers recruited patients aged 70 years or older and performed a geriatric assessment to identify comorbidities, cognitive issues, and other geriatric abnormalities.
Those who were deemed “fit” (ie, with no geriatric abnormalities) were assigned to receive off-study standard-of-care treatment, whereas those classified as “frail” (ie, with severe abnormalities) received off-study supportive care.
The remaining 176 “vulnerable” patients with mild to moderate geriatric abnormalities completed a geriatric and quality-of-life assessment and were then randomly assigned to receive either dose-reduced 5-fluorouracil (5-FU), leucovorin plus liposomal irinotecan (n = 88) or modified gemcitabine plus nab-paclitaxel (n = 88) every 2 weeks. Ultimately, 79 patients started the 5-FU combination and 75 received gemcitabine plus nab-paclitaxel. Patients were assessed every 8 weeks until disease progression or intolerance.
Overall, patients had a median age of 77 years; 61.9% were aged 75 years or older. About half were female, and 81.5% were White. The majority (87.5%) had a performance status of 0 or 1.
TAKEAWAY:
- Median overall survival was 4.7 months in the gemcitabine plus nab-paclitaxel arm and 4.4 months in the 5-FU combination group, with no significant survival difference observed between the two arms (P = .72).
- When the overall survival analysis was restricted to patients who received at least 4 weeks, or two cycles, of treatment (about 62% of patients), the median overall survival across the two treatment arms reached 8.0 months, in line with expectations for these regimens.
- Patient stratification revealed that those with a performance status of 2 had significantly worse overall survival than those with a status of 0: 1.4 months vs 6.9 months, respectively (hazard ratio [HR], 2.77; P < .001). A similar divide was seen when patients were stratified by physical/functional status and well-being. Age, however, did not significantly influence the results.
- Overall, more than half of patients experienced grade 3 or higher adverse events. Just over 38% of patients received only one to three cycles of therapy, whereas 26% remained on treatment for 12 or more cycles. The adverse event rates were similar between the two regimens, but the toxicity profile was slightly different — the researchers, for instance, observed more peripheral neuropathy with gemcitabine plus nab-paclitaxel and more diarrhea in the 5-FU combination arm.
IN PRACTICE:
- Overall, the “survival outcomes among vulnerable older patients were lower than expected, with high percentage of patients not able to start treatment, or complete one month of therapy due to clinical deterioration,” said study presenter Efrat Dotan, MD, chief, Division of Gastrointestinal Medical Oncology, Fox Chase Cancer Center, Philadelphia.
- “For vulnerable older adults who can tolerate treatment, these two regimens provide clinicians with options for tailoring therapy based on toxicity profile,” Dr. Dotan added. But “tools are needed to better identify patients who can benefit from treatment.”
- The results underline the need to perform geriatric assessments, as opposed to merely looking at performance status, commented David F. Chang, PhD, MS, MBBS, professor of Surgical Oncology, University of Glasgow, Scotland, who was not involved in the study.
SOURCE:
The research, presented at the 2024 annual meeting of the American Society of Clinical Oncology, was funded by the National Cancer Institute and the Eastern Cooperative Oncology Group.
LIMITATIONS:
Dr. Chang noted that the study did not reveal which treatment regimen was more effective.
DISCLOSURES:
Dr. Dotan declared relationships with Agenus, Amgen, G1 Therapeutics, Incyte, Olympus, and Taiho Pharmaceutical and institutional relationships with Dragonfly Therapeutics, Gilead Sciences, Ipsen, Kinnate Biopharma, Leap Therapeutics, Lilly, Lutris, NGM Biopharmaceuticals, Relay Therapeutics, and Zymeworks. Dr. Chang declared relationships with Immodulon Therapeutics and Mylan and institutional relationships with AstraZeneca, BMS GmbH & Co. KG, Immodulon Therapeutics, and Merck.
A version of this article appeared on Medscape.com.
TOPLINE:
METHODOLOGY:
Pancreatic cancer is most often diagnosed in adults aged 65 years or older. Providing cancer treatment for this older, often vulnerable, population comes with significant challenges and can lead to worse survival.
To examine real-world outcomes of older adults with untreated metastatic pancreatic cancer, researchers recruited patients aged 70 years or older and performed a geriatric assessment to identify comorbidities, cognitive issues, and other geriatric abnormalities.
Those who were deemed “fit” (ie, with no geriatric abnormalities) were assigned to receive off-study standard-of-care treatment, whereas those classified as “frail” (ie, with severe abnormalities) received off-study supportive care.
The remaining 176 “vulnerable” patients with mild to moderate geriatric abnormalities completed a geriatric and quality-of-life assessment and were then randomly assigned to receive either dose-reduced 5-fluorouracil (5-FU), leucovorin plus liposomal irinotecan (n = 88) or modified gemcitabine plus nab-paclitaxel (n = 88) every 2 weeks. Ultimately, 79 patients started the 5-FU combination and 75 received gemcitabine plus nab-paclitaxel. Patients were assessed every 8 weeks until disease progression or intolerance.
Overall, patients had a median age of 77 years; 61.9% were aged 75 years or older. About half were female, and 81.5% were White. The majority (87.5%) had a performance status of 0 or 1.
TAKEAWAY:
- Median overall survival was 4.7 months in the gemcitabine plus nab-paclitaxel arm and 4.4 months in the 5-FU combination group, with no significant survival difference observed between the two arms (P = .72).
- When the overall survival analysis was restricted to patients who received at least 4 weeks, or two cycles, of treatment (about 62% of patients), the median overall survival across the two treatment arms reached 8.0 months, in line with expectations for these regimens.
- Patient stratification revealed that those with a performance status of 2 had significantly worse overall survival than those with a status of 0: 1.4 months vs 6.9 months, respectively (hazard ratio [HR], 2.77; P < .001). A similar divide was seen when patients were stratified by physical/functional status and well-being. Age, however, did not significantly influence the results.
- Overall, more than half of patients experienced grade 3 or higher adverse events. Just over 38% of patients received only one to three cycles of therapy, whereas 26% remained on treatment for 12 or more cycles. The adverse event rates were similar between the two regimens, but the toxicity profile was slightly different — the researchers, for instance, observed more peripheral neuropathy with gemcitabine plus nab-paclitaxel and more diarrhea in the 5-FU combination arm.
IN PRACTICE:
- Overall, the “survival outcomes among vulnerable older patients were lower than expected, with high percentage of patients not able to start treatment, or complete one month of therapy due to clinical deterioration,” said study presenter Efrat Dotan, MD, chief, Division of Gastrointestinal Medical Oncology, Fox Chase Cancer Center, Philadelphia.
- “For vulnerable older adults who can tolerate treatment, these two regimens provide clinicians with options for tailoring therapy based on toxicity profile,” Dr. Dotan added. But “tools are needed to better identify patients who can benefit from treatment.”
- The results underline the need to perform geriatric assessments, as opposed to merely looking at performance status, commented David F. Chang, PhD, MS, MBBS, professor of Surgical Oncology, University of Glasgow, Scotland, who was not involved in the study.
SOURCE:
The research, presented at the 2024 annual meeting of the American Society of Clinical Oncology, was funded by the National Cancer Institute and the Eastern Cooperative Oncology Group.
LIMITATIONS:
Dr. Chang noted that the study did not reveal which treatment regimen was more effective.
DISCLOSURES:
Dr. Dotan declared relationships with Agenus, Amgen, G1 Therapeutics, Incyte, Olympus, and Taiho Pharmaceutical and institutional relationships with Dragonfly Therapeutics, Gilead Sciences, Ipsen, Kinnate Biopharma, Leap Therapeutics, Lilly, Lutris, NGM Biopharmaceuticals, Relay Therapeutics, and Zymeworks. Dr. Chang declared relationships with Immodulon Therapeutics and Mylan and institutional relationships with AstraZeneca, BMS GmbH & Co. KG, Immodulon Therapeutics, and Merck.
A version of this article appeared on Medscape.com.
EULAR 2024 Preview: Therapeutics in Development Take Center Stage
The European Alliance of Associations for Rheumatology (EULAR) 2024 European Congress of Rheumatology annual meeting is about to take place in Vienna, Austria. From June 12 to 15, some of the world’s leading researchers and clinicians will convene to present and learn about data on some of the new and innovative treatments for people with rheumatic and musculoskeletal diseases (RMDs) as well as to discuss how to use and optimize existing approaches.
Ahead of the Congress, this news organization asked the Congress Committee’s Scientific Programme Chair Caroline Ospelt, MD, PhD, and Abstract Chair Christian Dejaco, MD, PhD, MBA, to discuss some of their highlights of this year’s meeting.
From Bench to Bedside
“For me, the beauty at EULAR is really that you have the latest on basic research, how this can be translated in clinical trials, and then the last step would be how EULAR recommends it to be used in clinical practice,” Dr. Ospelt, professor of experimental rheumatology at University Hospital Zurich, said in an interview.
“So, if you go to EULAR continuously, you can actually follow the whole story of how novelty comes into clinical practice,” she added.
In a separate interview, Dr. Dejaco, a consultant rheumatologist and associate professor at the Medical University of Graz in Austria, said: “There are several new drug trials that are going to be presented.”
One of his highlights on the use of new drugs for the treatment of giant cell arteritis will be the phase 3 SELECT-GCA trial of the Janus kinase (JAK) inhibitor upadacitinib (LBA0001).
“It’s a trial that hopefully will lead to the approval of this drug in this indication,” Dr. Dejaco said.
Late-Breaking Abstracts
Dr. Ospelt noted: “We had a lot of good late-breaking abstracts this year.”
Some of these include:
- Real-world data on the comparative effectiveness of five different classes of drugs used to treat psoriatic arthritis (PsA; LBA0002)
- The 16-week results of a phase 2b/3 study with the novel interleukin (IL)–17A inhibitor izokibep in people with PsA (LBA0005)
- Data from the COSPIRIT-JIA trial on the efficacy and safety of ixekizumab (Taltz) in juvenile idiopathic arthritis (LBA0009)
- Phase 2 data on the safety and efficacy of the CD38-targeting monoclonal antibody daratumumab in systemic lupus erythematosus (LBA0007)
- Results of the phase 2 DAHLIAS study of the anti–neonatal Fc receptor monoclonal antibody nipocalimab in people with primary Sjögren disease (LBA0010)
- Safety and immunogenicity data from a phase 1 study of an active anti–IL-6 immunotherapy in people with knee osteoarthritis (LBA0011)
The latter is “really interesting,” Dr. Ospelt said. As of now, there is no approved treatment for osteoarthritis, and there is no immunotherapy, “so this would be the first.”
But it’s not just the late-breaker abstracts to look out for. Dr. Dejaco highlighted two abstracts that will be presented during the Abstract Plenary:
- A phase 3 study of a new selective JAK1 inhibitor, SHR0302, in rheumatoid arthritis (OP0037)
- A multi-omics analysis and targeted gene-editing study in people with , which causes inflammatory and hematologic changes (OP0073)
Of the latter, he said, “this disease is still incompletely understood, and this abstract really helps to better understand the mechanisms underlying this disease.”
One to Watch: CAR T-Cell Therapy
Dr. Ospelt said that the scientific program is about 80% clinical and 20% basic science overall. However, more sessions are being held jointly because data are starting to move from the bench to bedside.
One of the basic science areas that has had “a real buzz” around it and is now producing results in the clinic is the use of chimeric antigen receptor (CAR) T cells. In one of the first, and perhaps aptly titled What Is New, or WIN, sessions of the congress, Georg Schett, MD, vice president of research at Friedrich-Alexander-Universität Erlangen-Nüremberg in Germany, will discuss the use of CAR T-cell therapy for inflammatory RMDs. There are also multiple abstract presentations on this topic.
In-depth tissue analysis and prediction of treatment response is another interesting approach, Dr. Ospelt said. “I think that’s the way to go, that we come from the blood, we go into the tissue.” A “very nice” example of this approach will be presented during the Abstract Plenary session on Wednesday, June 12, looking at how synovial tissue macrophages may be able to give information on likely treatment response in treatment-naive rheumatoid arthritis (OP0062). There are also some further findings related to the tissue biopsy–driven treatment trial R4RA that are being presented at the meeting (OP0218, OP0242, and POS0351).
EULAR Highlighted Sessions
Among the highlighted sessions on the EULAR 2024 website is one on axial involvement in PsA and spondyloarthritis (SpA).
“Axial involvement in psoriatic arthritis and peripheral involvement in axial spondyloarthritis is quite a hot topic at the moment,” Dr. Ospelt said. There are lots of questions: “How connected are they? How different are they? Do we need different treatment for axial involvement compared to peripheral involvement?”
Another EULAR highlighted session is the 75th anniversary of glucocorticoid treatment, during which Past President of EULAR and Emeritus Professor of Rheumatology Josef S. Smolen, MD, will overview the “past, present, and future” of glucocorticoids in RMDs. Consultant rheumatologist Frank Buttgereit, MD, from the German Rheumatism Research Center in Berlin, will discuss the practicalities of using these drugs in clinical practice.
Dr. Dejaco noted: “Glucocorticoids have been one of the most important treatments for a very long time, and they’re still the most important treatment for the acute treatment of systemic inflammatory diseases.”
For a long time, there was no alternative to using steroids, he added, but steroid-sparing options now exist, and there will be data presented on a new type of drug that could potentially be used to control cortisol levels in the body (OP0335).
Recommendations and More
Dr. Ospelt and Dr. Dejaco both pointed out other sessions that are likely to be very popular, such as the first and second EULAR Recommendations sessions, a session on rheumatoid arthritis prevention, as well as the many presentations and sessions on digital health and nonpharmacologic interventions such as exercise.
With over 5242 submitted abstracts, there is going to be no shortage of data being presented at EULAR 2024. Alongside the traditional abstract submission categories, this year there is a new clinical case reports category.
“We had about 578 submissions for that category,” Dr. Dejaco said. There were 3315 abstracts submitted for the clinical research category, 812 for the basic and translational research category, 283 from health professionals in rheumatology, 152 from patient groups, and 102 in the field of pediatric rheumatology.
Join in On-Site, Watch on Demand
EULAR 2024 reverts to an on-site–only meeting this year. Some of the more lighthearted yet educational elements of the program for those attending include the second edition of the EMEUNET Rheumatology Quiz and, new for this year, two escape rooms. These rooms will provide an interactive experience where small teams will have to solve rheumatologic conundrums in order to escape the room within the hour, Dr. Dejaco explained. There will also be a morning run on Friday, June 14. “It’s not a race, it’s simply to meet and run together,” Dr. Dejaco said.
But if you cannot make the congress in person, the EULAR 2024 Livestream will be broadcasting throughout the congress. Anyone registered by June 30 will have on-demand access to the recorded content from June 17 until December 31, 2024.
Abstracts for the meeting will be published as a supplement to Annals of the Rheumatic Diseases, the official journal of EULAR.
Dr. Ospelt reported no relevant financial relationships. Dr. Dejaco has received consulting/speaker fees from AbbVie, Eli Lilly, Janssen, Sparrow, Novartis, Pfizer, Roche, Galapagos, and Sanofi.
A version of this article appeared on Medscape.com.
The European Alliance of Associations for Rheumatology (EULAR) 2024 European Congress of Rheumatology annual meeting is about to take place in Vienna, Austria. From June 12 to 15, some of the world’s leading researchers and clinicians will convene to present and learn about data on some of the new and innovative treatments for people with rheumatic and musculoskeletal diseases (RMDs) as well as to discuss how to use and optimize existing approaches.
Ahead of the Congress, this news organization asked the Congress Committee’s Scientific Programme Chair Caroline Ospelt, MD, PhD, and Abstract Chair Christian Dejaco, MD, PhD, MBA, to discuss some of their highlights of this year’s meeting.
From Bench to Bedside
“For me, the beauty at EULAR is really that you have the latest on basic research, how this can be translated in clinical trials, and then the last step would be how EULAR recommends it to be used in clinical practice,” Dr. Ospelt, professor of experimental rheumatology at University Hospital Zurich, said in an interview.
“So, if you go to EULAR continuously, you can actually follow the whole story of how novelty comes into clinical practice,” she added.
In a separate interview, Dr. Dejaco, a consultant rheumatologist and associate professor at the Medical University of Graz in Austria, said: “There are several new drug trials that are going to be presented.”
One of his highlights on the use of new drugs for the treatment of giant cell arteritis will be the phase 3 SELECT-GCA trial of the Janus kinase (JAK) inhibitor upadacitinib (LBA0001).
“It’s a trial that hopefully will lead to the approval of this drug in this indication,” Dr. Dejaco said.
Late-Breaking Abstracts
Dr. Ospelt noted: “We had a lot of good late-breaking abstracts this year.”
Some of these include:
- Real-world data on the comparative effectiveness of five different classes of drugs used to treat psoriatic arthritis (PsA; LBA0002)
- The 16-week results of a phase 2b/3 study with the novel interleukin (IL)–17A inhibitor izokibep in people with PsA (LBA0005)
- Data from the COSPIRIT-JIA trial on the efficacy and safety of ixekizumab (Taltz) in juvenile idiopathic arthritis (LBA0009)
- Phase 2 data on the safety and efficacy of the CD38-targeting monoclonal antibody daratumumab in systemic lupus erythematosus (LBA0007)
- Results of the phase 2 DAHLIAS study of the anti–neonatal Fc receptor monoclonal antibody nipocalimab in people with primary Sjögren disease (LBA0010)
- Safety and immunogenicity data from a phase 1 study of an active anti–IL-6 immunotherapy in people with knee osteoarthritis (LBA0011)
The latter is “really interesting,” Dr. Ospelt said. As of now, there is no approved treatment for osteoarthritis, and there is no immunotherapy, “so this would be the first.”
But it’s not just the late-breaker abstracts to look out for. Dr. Dejaco highlighted two abstracts that will be presented during the Abstract Plenary:
- A phase 3 study of a new selective JAK1 inhibitor, SHR0302, in rheumatoid arthritis (OP0037)
- A multi-omics analysis and targeted gene-editing study in people with , which causes inflammatory and hematologic changes (OP0073)
Of the latter, he said, “this disease is still incompletely understood, and this abstract really helps to better understand the mechanisms underlying this disease.”
One to Watch: CAR T-Cell Therapy
Dr. Ospelt said that the scientific program is about 80% clinical and 20% basic science overall. However, more sessions are being held jointly because data are starting to move from the bench to bedside.
One of the basic science areas that has had “a real buzz” around it and is now producing results in the clinic is the use of chimeric antigen receptor (CAR) T cells. In one of the first, and perhaps aptly titled What Is New, or WIN, sessions of the congress, Georg Schett, MD, vice president of research at Friedrich-Alexander-Universität Erlangen-Nüremberg in Germany, will discuss the use of CAR T-cell therapy for inflammatory RMDs. There are also multiple abstract presentations on this topic.
In-depth tissue analysis and prediction of treatment response is another interesting approach, Dr. Ospelt said. “I think that’s the way to go, that we come from the blood, we go into the tissue.” A “very nice” example of this approach will be presented during the Abstract Plenary session on Wednesday, June 12, looking at how synovial tissue macrophages may be able to give information on likely treatment response in treatment-naive rheumatoid arthritis (OP0062). There are also some further findings related to the tissue biopsy–driven treatment trial R4RA that are being presented at the meeting (OP0218, OP0242, and POS0351).
EULAR Highlighted Sessions
Among the highlighted sessions on the EULAR 2024 website is one on axial involvement in PsA and spondyloarthritis (SpA).
“Axial involvement in psoriatic arthritis and peripheral involvement in axial spondyloarthritis is quite a hot topic at the moment,” Dr. Ospelt said. There are lots of questions: “How connected are they? How different are they? Do we need different treatment for axial involvement compared to peripheral involvement?”
Another EULAR highlighted session is the 75th anniversary of glucocorticoid treatment, during which Past President of EULAR and Emeritus Professor of Rheumatology Josef S. Smolen, MD, will overview the “past, present, and future” of glucocorticoids in RMDs. Consultant rheumatologist Frank Buttgereit, MD, from the German Rheumatism Research Center in Berlin, will discuss the practicalities of using these drugs in clinical practice.
Dr. Dejaco noted: “Glucocorticoids have been one of the most important treatments for a very long time, and they’re still the most important treatment for the acute treatment of systemic inflammatory diseases.”
For a long time, there was no alternative to using steroids, he added, but steroid-sparing options now exist, and there will be data presented on a new type of drug that could potentially be used to control cortisol levels in the body (OP0335).
Recommendations and More
Dr. Ospelt and Dr. Dejaco both pointed out other sessions that are likely to be very popular, such as the first and second EULAR Recommendations sessions, a session on rheumatoid arthritis prevention, as well as the many presentations and sessions on digital health and nonpharmacologic interventions such as exercise.
With over 5242 submitted abstracts, there is going to be no shortage of data being presented at EULAR 2024. Alongside the traditional abstract submission categories, this year there is a new clinical case reports category.
“We had about 578 submissions for that category,” Dr. Dejaco said. There were 3315 abstracts submitted for the clinical research category, 812 for the basic and translational research category, 283 from health professionals in rheumatology, 152 from patient groups, and 102 in the field of pediatric rheumatology.
Join in On-Site, Watch on Demand
EULAR 2024 reverts to an on-site–only meeting this year. Some of the more lighthearted yet educational elements of the program for those attending include the second edition of the EMEUNET Rheumatology Quiz and, new for this year, two escape rooms. These rooms will provide an interactive experience where small teams will have to solve rheumatologic conundrums in order to escape the room within the hour, Dr. Dejaco explained. There will also be a morning run on Friday, June 14. “It’s not a race, it’s simply to meet and run together,” Dr. Dejaco said.
But if you cannot make the congress in person, the EULAR 2024 Livestream will be broadcasting throughout the congress. Anyone registered by June 30 will have on-demand access to the recorded content from June 17 until December 31, 2024.
Abstracts for the meeting will be published as a supplement to Annals of the Rheumatic Diseases, the official journal of EULAR.
Dr. Ospelt reported no relevant financial relationships. Dr. Dejaco has received consulting/speaker fees from AbbVie, Eli Lilly, Janssen, Sparrow, Novartis, Pfizer, Roche, Galapagos, and Sanofi.
A version of this article appeared on Medscape.com.
The European Alliance of Associations for Rheumatology (EULAR) 2024 European Congress of Rheumatology annual meeting is about to take place in Vienna, Austria. From June 12 to 15, some of the world’s leading researchers and clinicians will convene to present and learn about data on some of the new and innovative treatments for people with rheumatic and musculoskeletal diseases (RMDs) as well as to discuss how to use and optimize existing approaches.
Ahead of the Congress, this news organization asked the Congress Committee’s Scientific Programme Chair Caroline Ospelt, MD, PhD, and Abstract Chair Christian Dejaco, MD, PhD, MBA, to discuss some of their highlights of this year’s meeting.
From Bench to Bedside
“For me, the beauty at EULAR is really that you have the latest on basic research, how this can be translated in clinical trials, and then the last step would be how EULAR recommends it to be used in clinical practice,” Dr. Ospelt, professor of experimental rheumatology at University Hospital Zurich, said in an interview.
“So, if you go to EULAR continuously, you can actually follow the whole story of how novelty comes into clinical practice,” she added.
In a separate interview, Dr. Dejaco, a consultant rheumatologist and associate professor at the Medical University of Graz in Austria, said: “There are several new drug trials that are going to be presented.”
One of his highlights on the use of new drugs for the treatment of giant cell arteritis will be the phase 3 SELECT-GCA trial of the Janus kinase (JAK) inhibitor upadacitinib (LBA0001).
“It’s a trial that hopefully will lead to the approval of this drug in this indication,” Dr. Dejaco said.
Late-Breaking Abstracts
Dr. Ospelt noted: “We had a lot of good late-breaking abstracts this year.”
Some of these include:
- Real-world data on the comparative effectiveness of five different classes of drugs used to treat psoriatic arthritis (PsA; LBA0002)
- The 16-week results of a phase 2b/3 study with the novel interleukin (IL)–17A inhibitor izokibep in people with PsA (LBA0005)
- Data from the COSPIRIT-JIA trial on the efficacy and safety of ixekizumab (Taltz) in juvenile idiopathic arthritis (LBA0009)
- Phase 2 data on the safety and efficacy of the CD38-targeting monoclonal antibody daratumumab in systemic lupus erythematosus (LBA0007)
- Results of the phase 2 DAHLIAS study of the anti–neonatal Fc receptor monoclonal antibody nipocalimab in people with primary Sjögren disease (LBA0010)
- Safety and immunogenicity data from a phase 1 study of an active anti–IL-6 immunotherapy in people with knee osteoarthritis (LBA0011)
The latter is “really interesting,” Dr. Ospelt said. As of now, there is no approved treatment for osteoarthritis, and there is no immunotherapy, “so this would be the first.”
But it’s not just the late-breaker abstracts to look out for. Dr. Dejaco highlighted two abstracts that will be presented during the Abstract Plenary:
- A phase 3 study of a new selective JAK1 inhibitor, SHR0302, in rheumatoid arthritis (OP0037)
- A multi-omics analysis and targeted gene-editing study in people with , which causes inflammatory and hematologic changes (OP0073)
Of the latter, he said, “this disease is still incompletely understood, and this abstract really helps to better understand the mechanisms underlying this disease.”
One to Watch: CAR T-Cell Therapy
Dr. Ospelt said that the scientific program is about 80% clinical and 20% basic science overall. However, more sessions are being held jointly because data are starting to move from the bench to bedside.
One of the basic science areas that has had “a real buzz” around it and is now producing results in the clinic is the use of chimeric antigen receptor (CAR) T cells. In one of the first, and perhaps aptly titled What Is New, or WIN, sessions of the congress, Georg Schett, MD, vice president of research at Friedrich-Alexander-Universität Erlangen-Nüremberg in Germany, will discuss the use of CAR T-cell therapy for inflammatory RMDs. There are also multiple abstract presentations on this topic.
In-depth tissue analysis and prediction of treatment response is another interesting approach, Dr. Ospelt said. “I think that’s the way to go, that we come from the blood, we go into the tissue.” A “very nice” example of this approach will be presented during the Abstract Plenary session on Wednesday, June 12, looking at how synovial tissue macrophages may be able to give information on likely treatment response in treatment-naive rheumatoid arthritis (OP0062). There are also some further findings related to the tissue biopsy–driven treatment trial R4RA that are being presented at the meeting (OP0218, OP0242, and POS0351).
EULAR Highlighted Sessions
Among the highlighted sessions on the EULAR 2024 website is one on axial involvement in PsA and spondyloarthritis (SpA).
“Axial involvement in psoriatic arthritis and peripheral involvement in axial spondyloarthritis is quite a hot topic at the moment,” Dr. Ospelt said. There are lots of questions: “How connected are they? How different are they? Do we need different treatment for axial involvement compared to peripheral involvement?”
Another EULAR highlighted session is the 75th anniversary of glucocorticoid treatment, during which Past President of EULAR and Emeritus Professor of Rheumatology Josef S. Smolen, MD, will overview the “past, present, and future” of glucocorticoids in RMDs. Consultant rheumatologist Frank Buttgereit, MD, from the German Rheumatism Research Center in Berlin, will discuss the practicalities of using these drugs in clinical practice.
Dr. Dejaco noted: “Glucocorticoids have been one of the most important treatments for a very long time, and they’re still the most important treatment for the acute treatment of systemic inflammatory diseases.”
For a long time, there was no alternative to using steroids, he added, but steroid-sparing options now exist, and there will be data presented on a new type of drug that could potentially be used to control cortisol levels in the body (OP0335).
Recommendations and More
Dr. Ospelt and Dr. Dejaco both pointed out other sessions that are likely to be very popular, such as the first and second EULAR Recommendations sessions, a session on rheumatoid arthritis prevention, as well as the many presentations and sessions on digital health and nonpharmacologic interventions such as exercise.
With over 5242 submitted abstracts, there is going to be no shortage of data being presented at EULAR 2024. Alongside the traditional abstract submission categories, this year there is a new clinical case reports category.
“We had about 578 submissions for that category,” Dr. Dejaco said. There were 3315 abstracts submitted for the clinical research category, 812 for the basic and translational research category, 283 from health professionals in rheumatology, 152 from patient groups, and 102 in the field of pediatric rheumatology.
Join in On-Site, Watch on Demand
EULAR 2024 reverts to an on-site–only meeting this year. Some of the more lighthearted yet educational elements of the program for those attending include the second edition of the EMEUNET Rheumatology Quiz and, new for this year, two escape rooms. These rooms will provide an interactive experience where small teams will have to solve rheumatologic conundrums in order to escape the room within the hour, Dr. Dejaco explained. There will also be a morning run on Friday, June 14. “It’s not a race, it’s simply to meet and run together,” Dr. Dejaco said.
But if you cannot make the congress in person, the EULAR 2024 Livestream will be broadcasting throughout the congress. Anyone registered by June 30 will have on-demand access to the recorded content from June 17 until December 31, 2024.
Abstracts for the meeting will be published as a supplement to Annals of the Rheumatic Diseases, the official journal of EULAR.
Dr. Ospelt reported no relevant financial relationships. Dr. Dejaco has received consulting/speaker fees from AbbVie, Eli Lilly, Janssen, Sparrow, Novartis, Pfizer, Roche, Galapagos, and Sanofi.
A version of this article appeared on Medscape.com.