Federal Practitioner

Lucia Agajanian, a 25-year-old freelance film producer in Chicago, doesn’t have a specific primary care doctor, preferring the convenience of visiting a local clinic for flu shots or going online for video visits. “You say what you need, and there’s a 15-minute wait time,” she said, explaining how her appointments usually work. “I really liked that.”

But Olga Lucia Torres, a 52-year-old who teaches narrative medicine classes at Columbia University in New York, misses her longtime primary care doctor, who kept tabs for two decades on her conditions, including lupus and rheumatoid arthritis, and made sure she was up to date on vaccines and screening tests. Two years ago, Torres received a letter informing her that he was changing to a “boutique practice” and would charge a retainer fee of $10,000 for her to stay on as a patient.

“I felt really sad and abandoned,” Ms. Torres said. “This was my PCP. I was like, ‘Dude, I thought we were in this together!’ ”

The two women reflect an ongoing reality: The primary care landscape is changing in ways that could shape patients’ access and quality of care now and for decades to come. A solid and enduring relationship with a primary care doctor – who knows a patient’s history and can monitor new problems – has long been regarded as the bedrock of a quality health care system. But investment in primary care in the U.S. lags behind that of other high-income countries, and America has a smaller share of primary care physicians than most of its European counterparts.

An estimated one-third of all physicians in the U.S. are primary care doctors – who include family medicine physicians, general internists, and pediatricians – according to the Robert Graham Center, a research and analysis organization that studies primary care. Other researchers say the numbers are lower, with the Peterson-KFF Health System Tracker reporting only 12% of U.S. doctors are generalists, compared with 23% in Germany and as many as 45% in the Netherlands.

That means it’s often hard to find a doctor and make an appointment that’s not weeks or months away.

“This is a problem that has been simmering and now beginning to erupt in some communities at a boil. It’s hard to find that front door of the health system,” said Ann Greiner, president and CEO of the Primary Care Collaborative, a nonprofit membership organization.

Today, a smaller percentage of physicians are entering the field than are practicing, suggesting that shortages will worsen over time.

Interest has waned partly because, in the U.S., primary care yields lower salaries than other medical and surgical specialties.

Some doctors now in practice also say they are burned out, facing cumbersome electronic health record systems and limits on appointment times, making it harder to get to know a patient and establish a relationship.

Others are retiring or selling their practices. Hospitals, insurers like Aetna-CVS Health, and other corporate entities like Amazon are on a buying spree, snapping up primary care practices, furthering a move away from the “Marcus Welby, M.D.”-style neighborhood doctor. About 48% of primary care physicians currently work in practices they do not own. Two-thirds of those doctors don’t work for other physicians but are employed by private equity investors or other corporate entities, according to data in the “Primary Care Chartbook,” which is collected and published by the Graham Center.

Patients who seek care at these offices may not be seen by the same doctor at every visit. Indeed, they may not be seen by a doctor at all but by a paraprofessional – a nurse practitioner or a physician assistant, for instance – who works under the doctor’s license. That trend has been accelerated by new state laws – as well as changes in Medicare policy – that loosen the requirements for physician supervisors and billing. And these jobs are expected to be among the decade’s fastest-growing in the health sector.

Overall, demand for primary care is up, spurred partly by record enrollment in Affordable Care Act plans. All those new patients, combined with the low supply of doctors, are contributing to a years-long downward trend in the number of people reporting they have a usual source of care, be it an individual doctor or a specific clinic or practice.

Researchers say that raises questions, including whether people can’t find a primary care doctor, can’t afford one, or simply no longer want an established relationship.

“Is it poor access or problems with the supply of providers? Does it reflect a societal disconnection, a go-it-alone phenomenon?” asked Christopher F. Koller, president of the Milbank Memorial Fund, a foundation whose nonpartisan analyses focus on state health policy.

For patients, frustrating wait times are one result. A recent survey by a physician staffing firm found it now takes an average of 21 days just to get in to see a doctor of family medicine, defined as a subgroup of primary care, which includes general internists and pediatricians. Those physicians are many patients’ first stop for health care. That runs counter to the trend in other countries, where patients complain of months- or years-long waits for elective procedures like hip replacements but generally experience short waits for primary care visits.

Another complication: All these factors are adding urgency to ongoing concerns about attracting new primary care physicians to the specialty.

When she was in medical school, Natalie A. Cameron, MD, specifically chose primary care because she enjoyed forming relationships with patients and because “I’m specifically interested in prevention and women’s health, and you do a lot of that in primary care.” The 33-year-old is currently an instructor of medicine at Northwestern University, Chicago, where she also sees patients at a primary care practice.

Still, she understands why many of her colleagues chose something else. For some, it’s the pay differential. For others, it’s because of primary care’s reputation for involving “a lot of care and paperwork and coordinating a lot of issues that may not just be medical,” Dr. Cameron said.

The million-dollar question, then, is how much does having a usual source of care influence medical outcomes and cost? And for which kinds of patients is having a close relationship with a doctor important? While studies show that many young people value the convenience of visiting urgent care – especially when it takes so long to see a primary care doctor – will their long-term health suffer because of that strategy?

Many patients – particularly the young and generally healthy ones – shrug at the new normal, embracing alternatives that require less waiting. These options are particularly attractive to millennials, who tell focus groups that the convenience of a one-off video call or visit to a big-box store clinic trumps a long-standing relationship with a doctor, especially if they have to wait days, weeks, or longer for a traditional appointment.

“The doctor I have is a family friend, but definitely I would take access and ease over a relationship,” said Matt Degn, 24, who says it can take two to three months to book a routine appointment in Salt Lake City, where he lives.

Patients are increasingly turning to what are dubbed “retail clinics,” such as CVS’ Minute Clinics, which tout “in-person and virtual care 7 days a week.” CVS Health’s more than 1,000 clinics inside stores across the U.S. treated more than 5 million people last year, Creagh Milford, a physician and the company’s senior vice president of retail health, said in a written statement. He cited a recent study by a data products firm showing the use of retail clinics has grown 200% over the past five years.

Health policy experts say increased access to alternatives can be good, but forgoing an ongoing relationship to a regular provider is not, especially as people get older and are more likely to develop chronic conditions or other medical problems.

“There’s a lot of data that show communities with a lot of primary care have better health,” said Mr. Koller.

People with a regular primary care doctor or practice are more likely to get preventive care, such as cancer screenings or flu shots, studies show, and are less likely to die if they do suffer a heart attack.

Physicians who see patients regularly are better able to spot patterns of seemingly minor concerns that could add up to a serious health issue.

“What happens when you go to four different providers on four platforms for urinary tract infections because, well, they are just UTIs,” posed Yalda Jabbarpour, MD, a family physician practicing in Washington, and the director of the Robert Graham Center for Policy Studies. “But actually, you have a large kidney stone that’s causing your UTI or have some sort of immune deficiency like diabetes that’s causing frequent UTIs. But no one tested you.”

Most experts agree that figuring out how to coordinate care amid this changing landscape and make it more accessible without undermining quality – even when different doctors, locations, health systems, and electronic health records are involved – will be as complex as the pressures causing long waits and less interest in today’s primary care market.

And experiences sometimes lead patients to change their minds.

There’s something to be said for establishing a relationship, said Ms. Agajanian, in Chicago. She’s rethinking her decision to cobble together care, rather than have a specific primary care doctor or clinic, following an injury at work last year that led to shoulder surgery.

“As I’m getting older, even though I’m still young,” she said, “I have all these problems with my body, and it would be nice to have a consistent person who knows all my problems to talk with.”

KFF Health News’ Colleen DeGuzman contributed to this report.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF – an independent source of health policy research, polling, and journalism. Learn more about KFF.

Lucia Agajanian, a 25-year-old freelance film producer in Chicago, doesn’t have a specific primary care doctor, preferring the convenience of visiting a local clinic for flu shots or going online for video visits. “You say what you need, and there’s a 15-minute wait time,” she said, explaining how her appointments usually work. “I really liked that.”

But Olga Lucia Torres, a 52-year-old who teaches narrative medicine classes at Columbia University in New York, misses her longtime primary care doctor, who kept tabs for two decades on her conditions, including lupus and rheumatoid arthritis, and made sure she was up to date on vaccines and screening tests. Two years ago, Torres received a letter informing her that he was changing to a “boutique practice” and would charge a retainer fee of $10,000 for her to stay on as a patient.

“I felt really sad and abandoned,” Ms. Torres said. “This was my PCP. I was like, ‘Dude, I thought we were in this together!’ ”

The two women reflect an ongoing reality: The primary care landscape is changing in ways that could shape patients’ access and quality of care now and for decades to come. A solid and enduring relationship with a primary care doctor – who knows a patient’s history and can monitor new problems – has long been regarded as the bedrock of a quality health care system. But investment in primary care in the U.S. lags behind that of other high-income countries, and America has a smaller share of primary care physicians than most of its European counterparts.

An estimated one-third of all physicians in the U.S. are primary care doctors – who include family medicine physicians, general internists, and pediatricians – according to the Robert Graham Center, a research and analysis organization that studies primary care. Other researchers say the numbers are lower, with the Peterson-KFF Health System Tracker reporting only 12% of U.S. doctors are generalists, compared with 23% in Germany and as many as 45% in the Netherlands.

That means it’s often hard to find a doctor and make an appointment that’s not weeks or months away.

“This is a problem that has been simmering and now beginning to erupt in some communities at a boil. It’s hard to find that front door of the health system,” said Ann Greiner, president and CEO of the Primary Care Collaborative, a nonprofit membership organization.

Today, a smaller percentage of physicians are entering the field than are practicing, suggesting that shortages will worsen over time.

Interest has waned partly because, in the U.S., primary care yields lower salaries than other medical and surgical specialties.

Some doctors now in practice also say they are burned out, facing cumbersome electronic health record systems and limits on appointment times, making it harder to get to know a patient and establish a relationship.

Others are retiring or selling their practices. Hospitals, insurers like Aetna-CVS Health, and other corporate entities like Amazon are on a buying spree, snapping up primary care practices, furthering a move away from the “Marcus Welby, M.D.”-style neighborhood doctor. About 48% of primary care physicians currently work in practices they do not own. Two-thirds of those doctors don’t work for other physicians but are employed by private equity investors or other corporate entities, according to data in the “Primary Care Chartbook,” which is collected and published by the Graham Center.

Patients who seek care at these offices may not be seen by the same doctor at every visit. Indeed, they may not be seen by a doctor at all but by a paraprofessional – a nurse practitioner or a physician assistant, for instance – who works under the doctor’s license. That trend has been accelerated by new state laws – as well as changes in Medicare policy – that loosen the requirements for physician supervisors and billing. And these jobs are expected to be among the decade’s fastest-growing in the health sector.

Overall, demand for primary care is up, spurred partly by record enrollment in Affordable Care Act plans. All those new patients, combined with the low supply of doctors, are contributing to a years-long downward trend in the number of people reporting they have a usual source of care, be it an individual doctor or a specific clinic or practice.

Researchers say that raises questions, including whether people can’t find a primary care doctor, can’t afford one, or simply no longer want an established relationship.

“Is it poor access or problems with the supply of providers? Does it reflect a societal disconnection, a go-it-alone phenomenon?” asked Christopher F. Koller, president of the Milbank Memorial Fund, a foundation whose nonpartisan analyses focus on state health policy.

For patients, frustrating wait times are one result. A recent survey by a physician staffing firm found it now takes an average of 21 days just to get in to see a doctor of family medicine, defined as a subgroup of primary care, which includes general internists and pediatricians. Those physicians are many patients’ first stop for health care. That runs counter to the trend in other countries, where patients complain of months- or years-long waits for elective procedures like hip replacements but generally experience short waits for primary care visits.

Another complication: All these factors are adding urgency to ongoing concerns about attracting new primary care physicians to the specialty.

When she was in medical school, Natalie A. Cameron, MD, specifically chose primary care because she enjoyed forming relationships with patients and because “I’m specifically interested in prevention and women’s health, and you do a lot of that in primary care.” The 33-year-old is currently an instructor of medicine at Northwestern University, Chicago, where she also sees patients at a primary care practice.

Still, she understands why many of her colleagues chose something else. For some, it’s the pay differential. For others, it’s because of primary care’s reputation for involving “a lot of care and paperwork and coordinating a lot of issues that may not just be medical,” Dr. Cameron said.

The million-dollar question, then, is how much does having a usual source of care influence medical outcomes and cost? And for which kinds of patients is having a close relationship with a doctor important? While studies show that many young people value the convenience of visiting urgent care – especially when it takes so long to see a primary care doctor – will their long-term health suffer because of that strategy?

Many patients – particularly the young and generally healthy ones – shrug at the new normal, embracing alternatives that require less waiting. These options are particularly attractive to millennials, who tell focus groups that the convenience of a one-off video call or visit to a big-box store clinic trumps a long-standing relationship with a doctor, especially if they have to wait days, weeks, or longer for a traditional appointment.

“The doctor I have is a family friend, but definitely I would take access and ease over a relationship,” said Matt Degn, 24, who says it can take two to three months to book a routine appointment in Salt Lake City, where he lives.

Patients are increasingly turning to what are dubbed “retail clinics,” such as CVS’ Minute Clinics, which tout “in-person and virtual care 7 days a week.” CVS Health’s more than 1,000 clinics inside stores across the U.S. treated more than 5 million people last year, Creagh Milford, a physician and the company’s senior vice president of retail health, said in a written statement. He cited a recent study by a data products firm showing the use of retail clinics has grown 200% over the past five years.

Health policy experts say increased access to alternatives can be good, but forgoing an ongoing relationship to a regular provider is not, especially as people get older and are more likely to develop chronic conditions or other medical problems.

“There’s a lot of data that show communities with a lot of primary care have better health,” said Mr. Koller.

People with a regular primary care doctor or practice are more likely to get preventive care, such as cancer screenings or flu shots, studies show, and are less likely to die if they do suffer a heart attack.

Physicians who see patients regularly are better able to spot patterns of seemingly minor concerns that could add up to a serious health issue.

“What happens when you go to four different providers on four platforms for urinary tract infections because, well, they are just UTIs,” posed Yalda Jabbarpour, MD, a family physician practicing in Washington, and the director of the Robert Graham Center for Policy Studies. “But actually, you have a large kidney stone that’s causing your UTI or have some sort of immune deficiency like diabetes that’s causing frequent UTIs. But no one tested you.”

Most experts agree that figuring out how to coordinate care amid this changing landscape and make it more accessible without undermining quality – even when different doctors, locations, health systems, and electronic health records are involved – will be as complex as the pressures causing long waits and less interest in today’s primary care market.

And experiences sometimes lead patients to change their minds.

There’s something to be said for establishing a relationship, said Ms. Agajanian, in Chicago. She’s rethinking her decision to cobble together care, rather than have a specific primary care doctor or clinic, following an injury at work last year that led to shoulder surgery.

“As I’m getting older, even though I’m still young,” she said, “I have all these problems with my body, and it would be nice to have a consistent person who knows all my problems to talk with.”

KFF Health News’ Colleen DeGuzman contributed to this report.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF – an independent source of health policy research, polling, and journalism. Learn more about KFF.

Lucia Agajanian, a 25-year-old freelance film producer in Chicago, doesn’t have a specific primary care doctor, preferring the convenience of visiting a local clinic for flu shots or going online for video visits. “You say what you need, and there’s a 15-minute wait time,” she said, explaining how her appointments usually work. “I really liked that.”

But Olga Lucia Torres, a 52-year-old who teaches narrative medicine classes at Columbia University in New York, misses her longtime primary care doctor, who kept tabs for two decades on her conditions, including lupus and rheumatoid arthritis, and made sure she was up to date on vaccines and screening tests. Two years ago, Torres received a letter informing her that he was changing to a “boutique practice” and would charge a retainer fee of $10,000 for her to stay on as a patient.

“I felt really sad and abandoned,” Ms. Torres said. “This was my PCP. I was like, ‘Dude, I thought we were in this together!’ ”

The two women reflect an ongoing reality: The primary care landscape is changing in ways that could shape patients’ access and quality of care now and for decades to come. A solid and enduring relationship with a primary care doctor – who knows a patient’s history and can monitor new problems – has long been regarded as the bedrock of a quality health care system. But investment in primary care in the U.S. lags behind that of other high-income countries, and America has a smaller share of primary care physicians than most of its European counterparts.

An estimated one-third of all physicians in the U.S. are primary care doctors – who include family medicine physicians, general internists, and pediatricians – according to the Robert Graham Center, a research and analysis organization that studies primary care. Other researchers say the numbers are lower, with the Peterson-KFF Health System Tracker reporting only 12% of U.S. doctors are generalists, compared with 23% in Germany and as many as 45% in the Netherlands.

That means it’s often hard to find a doctor and make an appointment that’s not weeks or months away.

“This is a problem that has been simmering and now beginning to erupt in some communities at a boil. It’s hard to find that front door of the health system,” said Ann Greiner, president and CEO of the Primary Care Collaborative, a nonprofit membership organization.

Today, a smaller percentage of physicians are entering the field than are practicing, suggesting that shortages will worsen over time.

Interest has waned partly because, in the U.S., primary care yields lower salaries than other medical and surgical specialties.

Some doctors now in practice also say they are burned out, facing cumbersome electronic health record systems and limits on appointment times, making it harder to get to know a patient and establish a relationship.

Others are retiring or selling their practices. Hospitals, insurers like Aetna-CVS Health, and other corporate entities like Amazon are on a buying spree, snapping up primary care practices, furthering a move away from the “Marcus Welby, M.D.”-style neighborhood doctor. About 48% of primary care physicians currently work in practices they do not own. Two-thirds of those doctors don’t work for other physicians but are employed by private equity investors or other corporate entities, according to data in the “Primary Care Chartbook,” which is collected and published by the Graham Center.

Patients who seek care at these offices may not be seen by the same doctor at every visit. Indeed, they may not be seen by a doctor at all but by a paraprofessional – a nurse practitioner or a physician assistant, for instance – who works under the doctor’s license. That trend has been accelerated by new state laws – as well as changes in Medicare policy – that loosen the requirements for physician supervisors and billing. And these jobs are expected to be among the decade’s fastest-growing in the health sector.

Overall, demand for primary care is up, spurred partly by record enrollment in Affordable Care Act plans. All those new patients, combined with the low supply of doctors, are contributing to a years-long downward trend in the number of people reporting they have a usual source of care, be it an individual doctor or a specific clinic or practice.

Researchers say that raises questions, including whether people can’t find a primary care doctor, can’t afford one, or simply no longer want an established relationship.

“Is it poor access or problems with the supply of providers? Does it reflect a societal disconnection, a go-it-alone phenomenon?” asked Christopher F. Koller, president of the Milbank Memorial Fund, a foundation whose nonpartisan analyses focus on state health policy.

For patients, frustrating wait times are one result. A recent survey by a physician staffing firm found it now takes an average of 21 days just to get in to see a doctor of family medicine, defined as a subgroup of primary care, which includes general internists and pediatricians. Those physicians are many patients’ first stop for health care. That runs counter to the trend in other countries, where patients complain of months- or years-long waits for elective procedures like hip replacements but generally experience short waits for primary care visits.

Another complication: All these factors are adding urgency to ongoing concerns about attracting new primary care physicians to the specialty.

When she was in medical school, Natalie A. Cameron, MD, specifically chose primary care because she enjoyed forming relationships with patients and because “I’m specifically interested in prevention and women’s health, and you do a lot of that in primary care.” The 33-year-old is currently an instructor of medicine at Northwestern University, Chicago, where she also sees patients at a primary care practice.

Still, she understands why many of her colleagues chose something else. For some, it’s the pay differential. For others, it’s because of primary care’s reputation for involving “a lot of care and paperwork and coordinating a lot of issues that may not just be medical,” Dr. Cameron said.

The million-dollar question, then, is how much does having a usual source of care influence medical outcomes and cost? And for which kinds of patients is having a close relationship with a doctor important? While studies show that many young people value the convenience of visiting urgent care – especially when it takes so long to see a primary care doctor – will their long-term health suffer because of that strategy?

Many patients – particularly the young and generally healthy ones – shrug at the new normal, embracing alternatives that require less waiting. These options are particularly attractive to millennials, who tell focus groups that the convenience of a one-off video call or visit to a big-box store clinic trumps a long-standing relationship with a doctor, especially if they have to wait days, weeks, or longer for a traditional appointment.

“The doctor I have is a family friend, but definitely I would take access and ease over a relationship,” said Matt Degn, 24, who says it can take two to three months to book a routine appointment in Salt Lake City, where he lives.

Patients are increasingly turning to what are dubbed “retail clinics,” such as CVS’ Minute Clinics, which tout “in-person and virtual care 7 days a week.” CVS Health’s more than 1,000 clinics inside stores across the U.S. treated more than 5 million people last year, Creagh Milford, a physician and the company’s senior vice president of retail health, said in a written statement. He cited a recent study by a data products firm showing the use of retail clinics has grown 200% over the past five years.

Health policy experts say increased access to alternatives can be good, but forgoing an ongoing relationship to a regular provider is not, especially as people get older and are more likely to develop chronic conditions or other medical problems.

“There’s a lot of data that show communities with a lot of primary care have better health,” said Mr. Koller.

People with a regular primary care doctor or practice are more likely to get preventive care, such as cancer screenings or flu shots, studies show, and are less likely to die if they do suffer a heart attack.

Physicians who see patients regularly are better able to spot patterns of seemingly minor concerns that could add up to a serious health issue.

“What happens when you go to four different providers on four platforms for urinary tract infections because, well, they are just UTIs,” posed Yalda Jabbarpour, MD, a family physician practicing in Washington, and the director of the Robert Graham Center for Policy Studies. “But actually, you have a large kidney stone that’s causing your UTI or have some sort of immune deficiency like diabetes that’s causing frequent UTIs. But no one tested you.”

Most experts agree that figuring out how to coordinate care amid this changing landscape and make it more accessible without undermining quality – even when different doctors, locations, health systems, and electronic health records are involved – will be as complex as the pressures causing long waits and less interest in today’s primary care market.

And experiences sometimes lead patients to change their minds.

There’s something to be said for establishing a relationship, said Ms. Agajanian, in Chicago. She’s rethinking her decision to cobble together care, rather than have a specific primary care doctor or clinic, following an injury at work last year that led to shoulder surgery.

“As I’m getting older, even though I’m still young,” she said, “I have all these problems with my body, and it would be nice to have a consistent person who knows all my problems to talk with.”

KFF Health News’ Colleen DeGuzman contributed to this report.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF – an independent source of health policy research, polling, and journalism. Learn more about KFF.

Nearly 40% of girls and young women in the United States may have iron deficiency, which can lead to fatigue and increase the risk of many health problems, according to a new study. 

Researchers also found that 6 in every 100 of the girls and young women had extremely low iron levels, known as iron-deficiency anemia, which impacts the blood’s ability to carry oxygen throughout the body.

The findings suggest that current screening guidelines for iron levels in girls and women may be flawed, resulting in missed chances to get a simple blood test that can diagnose the easy-to-treat condition. Iron supplements are often prescribed as a treatment.

The study was published in JAMA and included 12 years of data for a total of nearly 3,500 girls and women aged 12-21 years.

In addition to shortness of breath and fatigue, other symptoms of iron deficiency anemia are: 

• Pale skin
• Cold hands and feet
• Feeling dizzy or lightheaded
• Unusual cravings for nonfood items such as ice, dirt, or paper.

The Cleveland Clinic says the most common causes of iron-deficiency anemia are those that involve blood loss, including heavy menstrual periods. The body gets iron from food, and not getting enough iron from food, as can happen from eating a vegan or vegetarian diet, can also lead to deficiency. 

In this latest study, researchers found that young women and girls’ likelihood to have iron deficiency or iron-deficiency anemia were significantly linked to race and ethnicity, poverty status, access to sufficient or quality food (also called food insecurity), and body mass index. Black and Hispanic girls and women were more likely to have iron level problems, compared with White girls and women. Black girls and women were four times more likely to have iron-deficiency anemia, compared with White girls and women.

The authors did not discuss potential causes and suggested further study is needed to identify risk factors of iron deficiency in girls and young women.

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

Nearly 40% of girls and young women in the United States may have iron deficiency, which can lead to fatigue and increase the risk of many health problems, according to a new study. 

Researchers also found that 6 in every 100 of the girls and young women had extremely low iron levels, known as iron-deficiency anemia, which impacts the blood’s ability to carry oxygen throughout the body.

The findings suggest that current screening guidelines for iron levels in girls and women may be flawed, resulting in missed chances to get a simple blood test that can diagnose the easy-to-treat condition. Iron supplements are often prescribed as a treatment.

The study was published in JAMA and included 12 years of data for a total of nearly 3,500 girls and women aged 12-21 years.

In addition to shortness of breath and fatigue, other symptoms of iron deficiency anemia are: 

• Pale skin
• Cold hands and feet
• Feeling dizzy or lightheaded
• Unusual cravings for nonfood items such as ice, dirt, or paper.

The Cleveland Clinic says the most common causes of iron-deficiency anemia are those that involve blood loss, including heavy menstrual periods. The body gets iron from food, and not getting enough iron from food, as can happen from eating a vegan or vegetarian diet, can also lead to deficiency. 

In this latest study, researchers found that young women and girls’ likelihood to have iron deficiency or iron-deficiency anemia were significantly linked to race and ethnicity, poverty status, access to sufficient or quality food (also called food insecurity), and body mass index. Black and Hispanic girls and women were more likely to have iron level problems, compared with White girls and women. Black girls and women were four times more likely to have iron-deficiency anemia, compared with White girls and women.

The authors did not discuss potential causes and suggested further study is needed to identify risk factors of iron deficiency in girls and young women.

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

Nearly 40% of girls and young women in the United States may have iron deficiency, which can lead to fatigue and increase the risk of many health problems, according to a new study. 

Researchers also found that 6 in every 100 of the girls and young women had extremely low iron levels, known as iron-deficiency anemia, which impacts the blood’s ability to carry oxygen throughout the body.

The findings suggest that current screening guidelines for iron levels in girls and women may be flawed, resulting in missed chances to get a simple blood test that can diagnose the easy-to-treat condition. Iron supplements are often prescribed as a treatment.

The study was published in JAMA and included 12 years of data for a total of nearly 3,500 girls and women aged 12-21 years.

In addition to shortness of breath and fatigue, other symptoms of iron deficiency anemia are: 

• Pale skin
• Cold hands and feet
• Feeling dizzy or lightheaded
• Unusual cravings for nonfood items such as ice, dirt, or paper.

The Cleveland Clinic says the most common causes of iron-deficiency anemia are those that involve blood loss, including heavy menstrual periods. The body gets iron from food, and not getting enough iron from food, as can happen from eating a vegan or vegetarian diet, can also lead to deficiency. 

In this latest study, researchers found that young women and girls’ likelihood to have iron deficiency or iron-deficiency anemia were significantly linked to race and ethnicity, poverty status, access to sufficient or quality food (also called food insecurity), and body mass index. Black and Hispanic girls and women were more likely to have iron level problems, compared with White girls and women. Black girls and women were four times more likely to have iron-deficiency anemia, compared with White girls and women.

The authors did not discuss potential causes and suggested further study is needed to identify risk factors of iron deficiency in girls and young women.

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

Abdominal pain has always been among the most common complaints fielded by primary care pediatricians. Much has been written about how we clinicians should respond when one of these patients presents in our office. Obviously, we start with a good history and physical exam and then progress to whatever laboratory or imaging tests we believe will yield the most accurate diagnosis in the shortest amount of time and with the minimum risk to the patient.

However, the number of children complaining of abdominal pain who arrive at clinicians’ offices is but a mere fraction of the youngsters who have shared the complaint with their parents or caregivers. Little has been written about what is going on beneath the surface of this monstrous iceberg of pediatric abdominal pain.

A recent poll commissioned by C.S. Mott Children’s Hospital at the University of Michigan attempts to determine how Doctor Moms and Dads are handling their children’s belly pain complaints on what is truly the frontline of health care. Using a national panel of more than 2,000 parents, the investigators reviewed the responses of more than 1,000 individuals who had at least one child age 3-10.

Seventeen percent of the parents reported that their children complained of abdominal pain at least once a month. Only a bit more than 50% of these parents say they have discussed this frequent pain with their children’s providers. Less than a third of parents reported their children complain of abdominal pain only a few times a year and half the parents responded that their children rarely or never complained of a bellyache.

The survey drilled a little deeper and discovered that for the most part, parents took a thoughtful history and did a reasonably focused physical exam. More than a third of respondents felt “very confident” in their ability to recognize a serious problem. A third of parents reported that they would treat the symptoms with an over-the-counter product.

About a quarter of the parents attributed their children’s complaints to anxiety or to gain attention. In these situations, more than half of the parents said they would talk to the child about his/her concerns and/or suggest relaxation techniques or employ distraction. Only a few would allow the child to stay home from school or miss other activities. In general, it feels like Dr. Moms and Dads in the trenches are doing a pretty good job evaluating, triaging, and managing most children with abdominal pain. At least in my experience, unfortunate outcomes of pediatric abdominal pain as the result of home mismanagement are rare.

This is a nice little survey, but I don’t think it tells us much we haven’t already suspected. What we really want to know more about are those exceedingly rare but avoidable situations when parents have not managed their children’s belly pain well and the results have been tragic. Why did they wait so long to call the physician? What signs did they miss? What symptoms did they ignore or discount? Are there patterns we can better address with education?

Just as in cases of Sudden Unexplained Infant Death, investigating with sensitivity can be extremely difficult. Interviewing parents who are still processing the unexpected death of their child is something that must be done without the slightest hint of assessing blame. Sometimes that is just plain impossible. Fortunately, these cases are rare.

If we are considering launching the study that I have proposed, we must also embark on a parallel study that asks what are the systemic conditions that may have led to the tragic mismanagement of pediatric abdominal pain? When parents have been alert to children’s complaints and appearance and attempted to seek medical care, what impediments did they encounter? Was there a triage nurse or on call physician who didn’t listen, or failed to ask the right questions? Was the emergency room just too busy to allow a proper evaluation? Was there a communication problem? And, of course, there is always the money. Did the parents’ concern about paying for the evaluation blind them to their instinct to call? These are not easy questions to ask ourselves but they must be asked if we wish to bring our failure rate closer to zero and retain the trust of our patients.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.

Abdominal pain has always been among the most common complaints fielded by primary care pediatricians. Much has been written about how we clinicians should respond when one of these patients presents in our office. Obviously, we start with a good history and physical exam and then progress to whatever laboratory or imaging tests we believe will yield the most accurate diagnosis in the shortest amount of time and with the minimum risk to the patient.

However, the number of children complaining of abdominal pain who arrive at clinicians’ offices is but a mere fraction of the youngsters who have shared the complaint with their parents or caregivers. Little has been written about what is going on beneath the surface of this monstrous iceberg of pediatric abdominal pain.

A recent poll commissioned by C.S. Mott Children’s Hospital at the University of Michigan attempts to determine how Doctor Moms and Dads are handling their children’s belly pain complaints on what is truly the frontline of health care. Using a national panel of more than 2,000 parents, the investigators reviewed the responses of more than 1,000 individuals who had at least one child age 3-10.

Seventeen percent of the parents reported that their children complained of abdominal pain at least once a month. Only a bit more than 50% of these parents say they have discussed this frequent pain with their children’s providers. Less than a third of parents reported their children complain of abdominal pain only a few times a year and half the parents responded that their children rarely or never complained of a bellyache.

The survey drilled a little deeper and discovered that for the most part, parents took a thoughtful history and did a reasonably focused physical exam. More than a third of respondents felt “very confident” in their ability to recognize a serious problem. A third of parents reported that they would treat the symptoms with an over-the-counter product.

About a quarter of the parents attributed their children’s complaints to anxiety or to gain attention. In these situations, more than half of the parents said they would talk to the child about his/her concerns and/or suggest relaxation techniques or employ distraction. Only a few would allow the child to stay home from school or miss other activities. In general, it feels like Dr. Moms and Dads in the trenches are doing a pretty good job evaluating, triaging, and managing most children with abdominal pain. At least in my experience, unfortunate outcomes of pediatric abdominal pain as the result of home mismanagement are rare.

This is a nice little survey, but I don’t think it tells us much we haven’t already suspected. What we really want to know more about are those exceedingly rare but avoidable situations when parents have not managed their children’s belly pain well and the results have been tragic. Why did they wait so long to call the physician? What signs did they miss? What symptoms did they ignore or discount? Are there patterns we can better address with education?

Just as in cases of Sudden Unexplained Infant Death, investigating with sensitivity can be extremely difficult. Interviewing parents who are still processing the unexpected death of their child is something that must be done without the slightest hint of assessing blame. Sometimes that is just plain impossible. Fortunately, these cases are rare.

If we are considering launching the study that I have proposed, we must also embark on a parallel study that asks what are the systemic conditions that may have led to the tragic mismanagement of pediatric abdominal pain? When parents have been alert to children’s complaints and appearance and attempted to seek medical care, what impediments did they encounter? Was there a triage nurse or on call physician who didn’t listen, or failed to ask the right questions? Was the emergency room just too busy to allow a proper evaluation? Was there a communication problem? And, of course, there is always the money. Did the parents’ concern about paying for the evaluation blind them to their instinct to call? These are not easy questions to ask ourselves but they must be asked if we wish to bring our failure rate closer to zero and retain the trust of our patients.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.

Abdominal pain has always been among the most common complaints fielded by primary care pediatricians. Much has been written about how we clinicians should respond when one of these patients presents in our office. Obviously, we start with a good history and physical exam and then progress to whatever laboratory or imaging tests we believe will yield the most accurate diagnosis in the shortest amount of time and with the minimum risk to the patient.

However, the number of children complaining of abdominal pain who arrive at clinicians’ offices is but a mere fraction of the youngsters who have shared the complaint with their parents or caregivers. Little has been written about what is going on beneath the surface of this monstrous iceberg of pediatric abdominal pain.

A recent poll commissioned by C.S. Mott Children’s Hospital at the University of Michigan attempts to determine how Doctor Moms and Dads are handling their children’s belly pain complaints on what is truly the frontline of health care. Using a national panel of more than 2,000 parents, the investigators reviewed the responses of more than 1,000 individuals who had at least one child age 3-10.

Seventeen percent of the parents reported that their children complained of abdominal pain at least once a month. Only a bit more than 50% of these parents say they have discussed this frequent pain with their children’s providers. Less than a third of parents reported their children complain of abdominal pain only a few times a year and half the parents responded that their children rarely or never complained of a bellyache.

The survey drilled a little deeper and discovered that for the most part, parents took a thoughtful history and did a reasonably focused physical exam. More than a third of respondents felt “very confident” in their ability to recognize a serious problem. A third of parents reported that they would treat the symptoms with an over-the-counter product.

About a quarter of the parents attributed their children’s complaints to anxiety or to gain attention. In these situations, more than half of the parents said they would talk to the child about his/her concerns and/or suggest relaxation techniques or employ distraction. Only a few would allow the child to stay home from school or miss other activities. In general, it feels like Dr. Moms and Dads in the trenches are doing a pretty good job evaluating, triaging, and managing most children with abdominal pain. At least in my experience, unfortunate outcomes of pediatric abdominal pain as the result of home mismanagement are rare.

This is a nice little survey, but I don’t think it tells us much we haven’t already suspected. What we really want to know more about are those exceedingly rare but avoidable situations when parents have not managed their children’s belly pain well and the results have been tragic. Why did they wait so long to call the physician? What signs did they miss? What symptoms did they ignore or discount? Are there patterns we can better address with education?

Just as in cases of Sudden Unexplained Infant Death, investigating with sensitivity can be extremely difficult. Interviewing parents who are still processing the unexpected death of their child is something that must be done without the slightest hint of assessing blame. Sometimes that is just plain impossible. Fortunately, these cases are rare.

If we are considering launching the study that I have proposed, we must also embark on a parallel study that asks what are the systemic conditions that may have led to the tragic mismanagement of pediatric abdominal pain? When parents have been alert to children’s complaints and appearance and attempted to seek medical care, what impediments did they encounter? Was there a triage nurse or on call physician who didn’t listen, or failed to ask the right questions? Was the emergency room just too busy to allow a proper evaluation? Was there a communication problem? And, of course, there is always the money. Did the parents’ concern about paying for the evaluation blind them to their instinct to call? These are not easy questions to ask ourselves but they must be asked if we wish to bring our failure rate closer to zero and retain the trust of our patients.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.

This transcript has been edited for clarity.

Those of us in the field of diabetes have long wanted to cure type 1 diabetes, and there are little steps making me feel like this might be a possibility. One of those steps is that a company named Vertex – I’m actually on the steering committee for Vertex in terms of this project – has made beta cells from stem cells. Now, instead of waiting for a cadaveric donor, we can make little beta cells. They started giving them to people in human trials. The Food and Drug Administration has been cautious because it’s new, and I get that.

In the first part of these trials, we could only give half a dose of these beta cells. The doses were determined based on what we know from giving beta-cell transplants from cadaveric donors. We gave half a dose of these stem cell–derived beta cells to two people who were having episodes of severe hypoglycemia.

In patient 1, these beta cells worked incredibly well. He became insulin independent, and now after over a year, he’s basically free of his type 1 diabetes. Patient 2 received half a dose, and she did get some activity of the beta cells, but not enough to achieve insulin independence, so she got a second dose. Shortly after the second dose, she decided she didn’t want to participate in the trial anymore and she was lost to follow-up.

Patient 2 didn’t get the same response as patient 1, but then we moved on to four more patients who got a full dose to start with. Now, there’s a total of six patients. Of those additional four patients, one of them has now been followed for a year. Just like patient 1, he’s off insulin. It’s as though his body has normal beta cells and he’s doing great. For the next three patients, we don’t have enough follow-up data to tell you what’s going to happen to them at a year.

I can tell you that, in all six patients, the beta cells worked. They basically were producing insulin, they had positive C-peptide levels, and it showed that these beta cells work when given to human beings. Now the trial is going to start giving more patients these stem cell–derived beta cells.

One of the things that’s important to realize is that this is a very small sample size, at just six individuals. Even within those six individuals, there was variation in terms of the response to the treatment. Probably, just like with all things in medicine, there will be different doses, different ways in which people do respond, people who get off of insulin completely, and people who may require some ongoing insulin therapy. I have no idea what this is going to look like as we test this in more people.

Everybody did start making C-peptide, they were having an effect of these beta cells, and it was working. We’ll have to see how well it works, how well it works in whom, and how we’re going to be able to use these types of therapies in the future.

In terms of side effects, they were really related to immunosuppression. There were no real surprises, but again, this is a very small sample size.

In summary, I think this is really hopeful. I don’t like to give false hope, but each step of this development process has shown that these beta cells derived from stem cells do seem to work in human beings as native beta cells might. Hopefully, this portends a future of newer therapies in the treatment of people with type 1 diabetes. Thank you.
 

Dr. Peters is professor of medicine at the University of Southern California, Los Angeles, and director of the USC clinical diabetes programs. She has published more than 200 articles, reviews, and abstracts, and three books, on diabetes, and has been an investigator for more than 40 research studies. She has spoken internationally at over 400 programs and serves on many committees of several professional organizations She disclosed ties with Abbott Diabetes Care, AstraZeneca, Becton Dickinson, Boehringer Ingelheim Pharmaceuticals, Dexcom, Eli Lilly, Lexicon Pharmaceuticals, Livongo, MannKind Corporation, Medscape, Merck, Novo Nordisk, Omada Health, OptumHealth, Sanofi, and Zafgen.

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

This transcript has been edited for clarity.

Those of us in the field of diabetes have long wanted to cure type 1 diabetes, and there are little steps making me feel like this might be a possibility. One of those steps is that a company named Vertex – I’m actually on the steering committee for Vertex in terms of this project – has made beta cells from stem cells. Now, instead of waiting for a cadaveric donor, we can make little beta cells. They started giving them to people in human trials. The Food and Drug Administration has been cautious because it’s new, and I get that.

In the first part of these trials, we could only give half a dose of these beta cells. The doses were determined based on what we know from giving beta-cell transplants from cadaveric donors. We gave half a dose of these stem cell–derived beta cells to two people who were having episodes of severe hypoglycemia.

In patient 1, these beta cells worked incredibly well. He became insulin independent, and now after over a year, he’s basically free of his type 1 diabetes. Patient 2 received half a dose, and she did get some activity of the beta cells, but not enough to achieve insulin independence, so she got a second dose. Shortly after the second dose, she decided she didn’t want to participate in the trial anymore and she was lost to follow-up.

Patient 2 didn’t get the same response as patient 1, but then we moved on to four more patients who got a full dose to start with. Now, there’s a total of six patients. Of those additional four patients, one of them has now been followed for a year. Just like patient 1, he’s off insulin. It’s as though his body has normal beta cells and he’s doing great. For the next three patients, we don’t have enough follow-up data to tell you what’s going to happen to them at a year.

I can tell you that, in all six patients, the beta cells worked. They basically were producing insulin, they had positive C-peptide levels, and it showed that these beta cells work when given to human beings. Now the trial is going to start giving more patients these stem cell–derived beta cells.

One of the things that’s important to realize is that this is a very small sample size, at just six individuals. Even within those six individuals, there was variation in terms of the response to the treatment. Probably, just like with all things in medicine, there will be different doses, different ways in which people do respond, people who get off of insulin completely, and people who may require some ongoing insulin therapy. I have no idea what this is going to look like as we test this in more people.

Everybody did start making C-peptide, they were having an effect of these beta cells, and it was working. We’ll have to see how well it works, how well it works in whom, and how we’re going to be able to use these types of therapies in the future.

In terms of side effects, they were really related to immunosuppression. There were no real surprises, but again, this is a very small sample size.

In summary, I think this is really hopeful. I don’t like to give false hope, but each step of this development process has shown that these beta cells derived from stem cells do seem to work in human beings as native beta cells might. Hopefully, this portends a future of newer therapies in the treatment of people with type 1 diabetes. Thank you.
 

Dr. Peters is professor of medicine at the University of Southern California, Los Angeles, and director of the USC clinical diabetes programs. She has published more than 200 articles, reviews, and abstracts, and three books, on diabetes, and has been an investigator for more than 40 research studies. She has spoken internationally at over 400 programs and serves on many committees of several professional organizations She disclosed ties with Abbott Diabetes Care, AstraZeneca, Becton Dickinson, Boehringer Ingelheim Pharmaceuticals, Dexcom, Eli Lilly, Lexicon Pharmaceuticals, Livongo, MannKind Corporation, Medscape, Merck, Novo Nordisk, Omada Health, OptumHealth, Sanofi, and Zafgen.

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

This transcript has been edited for clarity.

Those of us in the field of diabetes have long wanted to cure type 1 diabetes, and there are little steps making me feel like this might be a possibility. One of those steps is that a company named Vertex – I’m actually on the steering committee for Vertex in terms of this project – has made beta cells from stem cells. Now, instead of waiting for a cadaveric donor, we can make little beta cells. They started giving them to people in human trials. The Food and Drug Administration has been cautious because it’s new, and I get that.

In the first part of these trials, we could only give half a dose of these beta cells. The doses were determined based on what we know from giving beta-cell transplants from cadaveric donors. We gave half a dose of these stem cell–derived beta cells to two people who were having episodes of severe hypoglycemia.

In patient 1, these beta cells worked incredibly well. He became insulin independent, and now after over a year, he’s basically free of his type 1 diabetes. Patient 2 received half a dose, and she did get some activity of the beta cells, but not enough to achieve insulin independence, so she got a second dose. Shortly after the second dose, she decided she didn’t want to participate in the trial anymore and she was lost to follow-up.

Patient 2 didn’t get the same response as patient 1, but then we moved on to four more patients who got a full dose to start with. Now, there’s a total of six patients. Of those additional four patients, one of them has now been followed for a year. Just like patient 1, he’s off insulin. It’s as though his body has normal beta cells and he’s doing great. For the next three patients, we don’t have enough follow-up data to tell you what’s going to happen to them at a year.

I can tell you that, in all six patients, the beta cells worked. They basically were producing insulin, they had positive C-peptide levels, and it showed that these beta cells work when given to human beings. Now the trial is going to start giving more patients these stem cell–derived beta cells.

One of the things that’s important to realize is that this is a very small sample size, at just six individuals. Even within those six individuals, there was variation in terms of the response to the treatment. Probably, just like with all things in medicine, there will be different doses, different ways in which people do respond, people who get off of insulin completely, and people who may require some ongoing insulin therapy. I have no idea what this is going to look like as we test this in more people.

Everybody did start making C-peptide, they were having an effect of these beta cells, and it was working. We’ll have to see how well it works, how well it works in whom, and how we’re going to be able to use these types of therapies in the future.

In terms of side effects, they were really related to immunosuppression. There were no real surprises, but again, this is a very small sample size.

In summary, I think this is really hopeful. I don’t like to give false hope, but each step of this development process has shown that these beta cells derived from stem cells do seem to work in human beings as native beta cells might. Hopefully, this portends a future of newer therapies in the treatment of people with type 1 diabetes. Thank you.
 

Dr. Peters is professor of medicine at the University of Southern California, Los Angeles, and director of the USC clinical diabetes programs. She has published more than 200 articles, reviews, and abstracts, and three books, on diabetes, and has been an investigator for more than 40 research studies. She has spoken internationally at over 400 programs and serves on many committees of several professional organizations She disclosed ties with Abbott Diabetes Care, AstraZeneca, Becton Dickinson, Boehringer Ingelheim Pharmaceuticals, Dexcom, Eli Lilly, Lexicon Pharmaceuticals, Livongo, MannKind Corporation, Medscape, Merck, Novo Nordisk, Omada Health, OptumHealth, Sanofi, and Zafgen.

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

The Food and Drug Administration has approved donislecel (Lantidra, CellTrans), a pancreatic islet cell therapy developed from cadaver donors, for the treatment of people with type 1 diabetes who are unable to achieve target glucose levels owing to severe hypoglycemic episodes.

The product is given as a single infusion via the hepatic portal vein into the liver. A second infusion is given if necessary. Immunosuppression is required to maintain cell viability, just as it is required to support a transplanted kidney or other organ, as these all represent “foreign” tissues to the recipient.

“Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in an FDA statement.

The product was approved despite concerns from the American Society of Transplant Surgeons, the American Society of Transplantation, and an organization of more than 50 transplant surgeons – the Islets for U.S. Collaborative – whose members argue that cadaver-derived (allogeneic) pancreatic islets should be regulated as transplanted organs rather than as biologic drugs, as is done in many other parts of the world.

Lantidra differs from stem cell therapy being developed by Vertex Pharmaceuticals. In the latter, beta cells are grown from allogeneic stem cells using a proprietary technology. So far, six patients have received the therapy, and it has been successful in all of them to varying degrees, as reported at last week’s American Diabetes Association meeting. So while this is a promising technology, with talk of a “cure” for type 1 diabetes, it’s important to remember that this is very early in the development phase, says Anne Peters, MD, of the University of California, Los Angeles.
 

Approval based on small studies, with adverse events

The approval of Lantidra, following a 12-4 vote in favor by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in April 2021, was based on two nonrandomized, single-arm studies that included a total of 30 individuals with type 1 diabetes who had hypoglycemic unawareness and who received between one and three infusions of donislecel.

Insulin independence was achieved at 1 year by 21 participants; 11 were still insulin independent at 5 years, and 10 remained so more than 5 years. Five participants were unable to discontinue insulin treatment at all.

Adverse events included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most of the participants experienced at least one serious adverse reaction related to the method of infusion and/or the use of immunosuppression. Some of these reactions required discontinuation of the immunosuppressive medications, resulting in the loss of islet cell function and return to insulin dependence.

“These adverse events should be considered when assessing the benefits and risks of Lantidra for each patient. Lantidra is approved with patient-directed labeling to inform patients with type 1 diabetes about benefits and risks of Lantidra,” according to the FDA statement.
 

U.S. transplant physicians had expressed concern, bill introduced

The transplant surgery organizations had written letters to the FDA, as well as to several other government agencies, to ask that the regulatory framework for Lantidra be shifted from the FDA to the Organ Procurement and Transplantation Network and the United Network for Organ Sharing.

They also wrote to members of Congress. On June 22, 2023, U.S. Senators Mike Lee (R-UT), Ted Budd (R-NC), and Marsha Blackburn (R-TN) introduced the Islet Transplantation Bill, which would shift the regulatory framework for cadaveric islets from that of biologic drugs to transplanted organs.

Asked for comment, Piotr Witkowski, MD, PhD, the leader of the Islets for U.S. Collaborative, told this news organization: “We were really happy about the introduction of the islet bill. Now, we’re concerned about negative downstream effects of granting a licence to a private company for distribution of the cadaveric islets.”

During the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee’s discussion in 2021, several panel members noted that the target patient population for this treatment with the current indication will likely be smaller today than it was when the two studies were initiated, in 2004 and 2007, given current automated diabetes technology – such as insulin pumps, continuous glucose monitors, and hybrid closed-loop systems in which the two are linked together as a so-called artificial pancreas – that reduces hypoglycemia risk.

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

The Food and Drug Administration has approved donislecel (Lantidra, CellTrans), a pancreatic islet cell therapy developed from cadaver donors, for the treatment of people with type 1 diabetes who are unable to achieve target glucose levels owing to severe hypoglycemic episodes.

The product is given as a single infusion via the hepatic portal vein into the liver. A second infusion is given if necessary. Immunosuppression is required to maintain cell viability, just as it is required to support a transplanted kidney or other organ, as these all represent “foreign” tissues to the recipient.

“Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in an FDA statement.

The product was approved despite concerns from the American Society of Transplant Surgeons, the American Society of Transplantation, and an organization of more than 50 transplant surgeons – the Islets for U.S. Collaborative – whose members argue that cadaver-derived (allogeneic) pancreatic islets should be regulated as transplanted organs rather than as biologic drugs, as is done in many other parts of the world.

Lantidra differs from stem cell therapy being developed by Vertex Pharmaceuticals. In the latter, beta cells are grown from allogeneic stem cells using a proprietary technology. So far, six patients have received the therapy, and it has been successful in all of them to varying degrees, as reported at last week’s American Diabetes Association meeting. So while this is a promising technology, with talk of a “cure” for type 1 diabetes, it’s important to remember that this is very early in the development phase, says Anne Peters, MD, of the University of California, Los Angeles.
 

Approval based on small studies, with adverse events

The approval of Lantidra, following a 12-4 vote in favor by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in April 2021, was based on two nonrandomized, single-arm studies that included a total of 30 individuals with type 1 diabetes who had hypoglycemic unawareness and who received between one and three infusions of donislecel.

Insulin independence was achieved at 1 year by 21 participants; 11 were still insulin independent at 5 years, and 10 remained so more than 5 years. Five participants were unable to discontinue insulin treatment at all.

Adverse events included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most of the participants experienced at least one serious adverse reaction related to the method of infusion and/or the use of immunosuppression. Some of these reactions required discontinuation of the immunosuppressive medications, resulting in the loss of islet cell function and return to insulin dependence.

“These adverse events should be considered when assessing the benefits and risks of Lantidra for each patient. Lantidra is approved with patient-directed labeling to inform patients with type 1 diabetes about benefits and risks of Lantidra,” according to the FDA statement.
 

U.S. transplant physicians had expressed concern, bill introduced

The transplant surgery organizations had written letters to the FDA, as well as to several other government agencies, to ask that the regulatory framework for Lantidra be shifted from the FDA to the Organ Procurement and Transplantation Network and the United Network for Organ Sharing.

They also wrote to members of Congress. On June 22, 2023, U.S. Senators Mike Lee (R-UT), Ted Budd (R-NC), and Marsha Blackburn (R-TN) introduced the Islet Transplantation Bill, which would shift the regulatory framework for cadaveric islets from that of biologic drugs to transplanted organs.

Asked for comment, Piotr Witkowski, MD, PhD, the leader of the Islets for U.S. Collaborative, told this news organization: “We were really happy about the introduction of the islet bill. Now, we’re concerned about negative downstream effects of granting a licence to a private company for distribution of the cadaveric islets.”

During the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee’s discussion in 2021, several panel members noted that the target patient population for this treatment with the current indication will likely be smaller today than it was when the two studies were initiated, in 2004 and 2007, given current automated diabetes technology – such as insulin pumps, continuous glucose monitors, and hybrid closed-loop systems in which the two are linked together as a so-called artificial pancreas – that reduces hypoglycemia risk.

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

The Food and Drug Administration has approved donislecel (Lantidra, CellTrans), a pancreatic islet cell therapy developed from cadaver donors, for the treatment of people with type 1 diabetes who are unable to achieve target glucose levels owing to severe hypoglycemic episodes.

The product is given as a single infusion via the hepatic portal vein into the liver. A second infusion is given if necessary. Immunosuppression is required to maintain cell viability, just as it is required to support a transplanted kidney or other organ, as these all represent “foreign” tissues to the recipient.

“Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in an FDA statement.

The product was approved despite concerns from the American Society of Transplant Surgeons, the American Society of Transplantation, and an organization of more than 50 transplant surgeons – the Islets for U.S. Collaborative – whose members argue that cadaver-derived (allogeneic) pancreatic islets should be regulated as transplanted organs rather than as biologic drugs, as is done in many other parts of the world.

Lantidra differs from stem cell therapy being developed by Vertex Pharmaceuticals. In the latter, beta cells are grown from allogeneic stem cells using a proprietary technology. So far, six patients have received the therapy, and it has been successful in all of them to varying degrees, as reported at last week’s American Diabetes Association meeting. So while this is a promising technology, with talk of a “cure” for type 1 diabetes, it’s important to remember that this is very early in the development phase, says Anne Peters, MD, of the University of California, Los Angeles.
 

Approval based on small studies, with adverse events

The approval of Lantidra, following a 12-4 vote in favor by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in April 2021, was based on two nonrandomized, single-arm studies that included a total of 30 individuals with type 1 diabetes who had hypoglycemic unawareness and who received between one and three infusions of donislecel.

Insulin independence was achieved at 1 year by 21 participants; 11 were still insulin independent at 5 years, and 10 remained so more than 5 years. Five participants were unable to discontinue insulin treatment at all.

Adverse events included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most of the participants experienced at least one serious adverse reaction related to the method of infusion and/or the use of immunosuppression. Some of these reactions required discontinuation of the immunosuppressive medications, resulting in the loss of islet cell function and return to insulin dependence.

“These adverse events should be considered when assessing the benefits and risks of Lantidra for each patient. Lantidra is approved with patient-directed labeling to inform patients with type 1 diabetes about benefits and risks of Lantidra,” according to the FDA statement.
 

U.S. transplant physicians had expressed concern, bill introduced

The transplant surgery organizations had written letters to the FDA, as well as to several other government agencies, to ask that the regulatory framework for Lantidra be shifted from the FDA to the Organ Procurement and Transplantation Network and the United Network for Organ Sharing.

They also wrote to members of Congress. On June 22, 2023, U.S. Senators Mike Lee (R-UT), Ted Budd (R-NC), and Marsha Blackburn (R-TN) introduced the Islet Transplantation Bill, which would shift the regulatory framework for cadaveric islets from that of biologic drugs to transplanted organs.

Asked for comment, Piotr Witkowski, MD, PhD, the leader of the Islets for U.S. Collaborative, told this news organization: “We were really happy about the introduction of the islet bill. Now, we’re concerned about negative downstream effects of granting a licence to a private company for distribution of the cadaveric islets.”

During the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee’s discussion in 2021, several panel members noted that the target patient population for this treatment with the current indication will likely be smaller today than it was when the two studies were initiated, in 2004 and 2007, given current automated diabetes technology – such as insulin pumps, continuous glucose monitors, and hybrid closed-loop systems in which the two are linked together as a so-called artificial pancreas – that reduces hypoglycemia risk.

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

Offering a blood test to people who have declined both a colonoscopy and a fecal immunochemical test (FIT) increased colorectal cancer (CRC) screening by 7.5% without decreasing use of the preferred first-line options, researchers report.

However, the number of people in the study who subsequently underwent timely colonoscopy after a positive blood test did not increase, signaling a continuing challenge in CRC prevention and treatment.

“The main message is that the blood test can do what it’s meant to do, which is increase screening uptake,” first author Peter Liang, MD, MPH, told this news organization. Dr. Liang is a gastroenterologist and researcher at NYU Langone Health and the VA New York Harbor Health Care System in New York.

The study was published online in Clinical Gastroenterology and Hepatology.

In the United States, the rate of use of first-line screening has for years been stuck at about 70% or lower for eligible people, Dr. Liang said. A different modality is needed to help raise the numbers.

The blood test is easy to perform and requires only a few tubes of blood, he noted. No diet restrictions, test prep, or contact with stool is necessary.

We are all searching for ways to get that first-line screening rate up from 60% to 70% to 80% to 90%, noted Folasade P. May, MD, PhD, who was not involved in the study.

“A lot of people think these blood tests are the promised land,” said Dr. May, associate professor of medicine at the University of California, Los Angeles, and director of the gastroenterology quality improvement program at UCLA Health. “We want to see that, when we offer these blood tests, the uptake is 20%-25% higher, which would get us closer to the national goal of 80% screened.”
 

Blood test as a second-line screening option

The study enrolled 359 veterans at a Veterans Affairs medical center. Participants were 50-75 years old and were eligible for screening but had declined a colonoscopy and a stool test within the previous 6 months.

They were randomly assigned to one of two groups. The control group received a letter and telephone outreach in which participants were again offered screening with colonoscopy or FIT only. The intervention group was additionally offered the blood test as a second-line option.

The primary outcome was completion of any screening test within 6 months. The secondary outcome was completion of a full screening strategy within 6 months, including colonoscopy for those with a positive noninvasive test result.

Of the people who had declined first-line tests and were reoffered first-line tests and the blood test, 17.1% completed screening within 6 months, compared with 9.6% of those who were only reoffered the first-line tests. The uptake of colonoscopy and FIT was similar between the two groups. The full-screening strategy was completed by 14.9% in the intervention group, compared with 9% in the control group.

At first glance, the results for uptake seem a bit disappointing, Dr. May said. However, the numbers in this study may not reflect the true potential of the blood tests – which are relatively new and have not yet been incorporated into routine care – because they had to be conducted in a separate appointment at a lab, she said.

If blood tests for CRC were part of the workflow, Dr. May explained, patients could undergo them with a routine blood draw already scheduled to check for diabetes or high cholesterol, for instance, and the numbers presumably would go up.

“I think this study underestimates the proportion of people who will participate,” she said. “We need a study that tests this strategy in a more real-world scenario.”

Nonetheless, “This is the first trial that’s looking at this question, and it’s an important question,” Dr. May added.

Dr. Liang and colleagues acknowledge that a limitation of the study is that it was performed in only one VA center among an older, predominantly male population, so it will be important to make the comparisons in more diverse study populations.
 

Blood test reliability

The septin 9 blood test is the only blood test approved by the U.S. Food and Drug Administration for CRC screening and is indicated for those who have declined first-line tests. However, the extent of usage of the blood test in this context is unclear, as it has only been approved since 2016.

Because the blood test is not covered by Medicare, Dr. Liang said, accessibility has been limited. One of the reasons for the lack of coverage is that the blood tests are less reliable than first-line tests, he said.

The test detects methylated septin 9 DNA, a biomarker for CRC. The FDA-approved version of the test has a sensitivity of 68% and a specificity of 79% for CRC.

Dr. May said she’d have more confidence in the blood tests if those numbers were higher, at 80%-90%.

Dr. Liang told this news organization that previous research has compared test use when colonoscopy, FIT, and a blood test are considered equally, but because the blood test is indicated only after a person declines first-line screening, his team designed an approach in which the blood test was a second-line option for its target population.

Other blood tests now on the market or under development appear to have higher sensitivity and specificity, he added.

“We think our results are actually applicable to a blood test in general,” Dr. Liang said.

Blood tests are only the first step, though. Getting people who screen positive to follow up with a diagnostic colonoscopy is critical, Dr. May and the authors agree.

“That’s something we, as a nation, just haven’t figured out,” Dr. May said. “It has to become a priority.”

The study was supported in part by the Veterans Health Administration and was funded by Epigenomics and a grant from the New York Society for Gastrointestinal Endoscopy’s Florence Lefcourt Endoscopy Research Award to Dr. Laing, who is also supported by the National Cancer Institute. Dr. Liang has received research support from Epigenomics and Freenome and is on the advisory board for Guardant Health. The remaining authors disclosed no relevant financial relationships. Dr. May is a consultant for Exact Sciences and Geneoscopy, both of which are developing stool tests, and for Freenome, which is developing stool and blood tests.

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

Offering a blood test to people who have declined both a colonoscopy and a fecal immunochemical test (FIT) increased colorectal cancer (CRC) screening by 7.5% without decreasing use of the preferred first-line options, researchers report.

However, the number of people in the study who subsequently underwent timely colonoscopy after a positive blood test did not increase, signaling a continuing challenge in CRC prevention and treatment.

“The main message is that the blood test can do what it’s meant to do, which is increase screening uptake,” first author Peter Liang, MD, MPH, told this news organization. Dr. Liang is a gastroenterologist and researcher at NYU Langone Health and the VA New York Harbor Health Care System in New York.

The study was published online in Clinical Gastroenterology and Hepatology.

In the United States, the rate of use of first-line screening has for years been stuck at about 70% or lower for eligible people, Dr. Liang said. A different modality is needed to help raise the numbers.

The blood test is easy to perform and requires only a few tubes of blood, he noted. No diet restrictions, test prep, or contact with stool is necessary.

We are all searching for ways to get that first-line screening rate up from 60% to 70% to 80% to 90%, noted Folasade P. May, MD, PhD, who was not involved in the study.

“A lot of people think these blood tests are the promised land,” said Dr. May, associate professor of medicine at the University of California, Los Angeles, and director of the gastroenterology quality improvement program at UCLA Health. “We want to see that, when we offer these blood tests, the uptake is 20%-25% higher, which would get us closer to the national goal of 80% screened.”
 

Blood test as a second-line screening option

The study enrolled 359 veterans at a Veterans Affairs medical center. Participants were 50-75 years old and were eligible for screening but had declined a colonoscopy and a stool test within the previous 6 months.

They were randomly assigned to one of two groups. The control group received a letter and telephone outreach in which participants were again offered screening with colonoscopy or FIT only. The intervention group was additionally offered the blood test as a second-line option.

The primary outcome was completion of any screening test within 6 months. The secondary outcome was completion of a full screening strategy within 6 months, including colonoscopy for those with a positive noninvasive test result.

Of the people who had declined first-line tests and were reoffered first-line tests and the blood test, 17.1% completed screening within 6 months, compared with 9.6% of those who were only reoffered the first-line tests. The uptake of colonoscopy and FIT was similar between the two groups. The full-screening strategy was completed by 14.9% in the intervention group, compared with 9% in the control group.

At first glance, the results for uptake seem a bit disappointing, Dr. May said. However, the numbers in this study may not reflect the true potential of the blood tests – which are relatively new and have not yet been incorporated into routine care – because they had to be conducted in a separate appointment at a lab, she said.

If blood tests for CRC were part of the workflow, Dr. May explained, patients could undergo them with a routine blood draw already scheduled to check for diabetes or high cholesterol, for instance, and the numbers presumably would go up.

“I think this study underestimates the proportion of people who will participate,” she said. “We need a study that tests this strategy in a more real-world scenario.”

Nonetheless, “This is the first trial that’s looking at this question, and it’s an important question,” Dr. May added.

Dr. Liang and colleagues acknowledge that a limitation of the study is that it was performed in only one VA center among an older, predominantly male population, so it will be important to make the comparisons in more diverse study populations.
 

Blood test reliability

The septin 9 blood test is the only blood test approved by the U.S. Food and Drug Administration for CRC screening and is indicated for those who have declined first-line tests. However, the extent of usage of the blood test in this context is unclear, as it has only been approved since 2016.

Because the blood test is not covered by Medicare, Dr. Liang said, accessibility has been limited. One of the reasons for the lack of coverage is that the blood tests are less reliable than first-line tests, he said.

The test detects methylated septin 9 DNA, a biomarker for CRC. The FDA-approved version of the test has a sensitivity of 68% and a specificity of 79% for CRC.

Dr. May said she’d have more confidence in the blood tests if those numbers were higher, at 80%-90%.

Dr. Liang told this news organization that previous research has compared test use when colonoscopy, FIT, and a blood test are considered equally, but because the blood test is indicated only after a person declines first-line screening, his team designed an approach in which the blood test was a second-line option for its target population.

Other blood tests now on the market or under development appear to have higher sensitivity and specificity, he added.

“We think our results are actually applicable to a blood test in general,” Dr. Liang said.

Blood tests are only the first step, though. Getting people who screen positive to follow up with a diagnostic colonoscopy is critical, Dr. May and the authors agree.

“That’s something we, as a nation, just haven’t figured out,” Dr. May said. “It has to become a priority.”

The study was supported in part by the Veterans Health Administration and was funded by Epigenomics and a grant from the New York Society for Gastrointestinal Endoscopy’s Florence Lefcourt Endoscopy Research Award to Dr. Laing, who is also supported by the National Cancer Institute. Dr. Liang has received research support from Epigenomics and Freenome and is on the advisory board for Guardant Health. The remaining authors disclosed no relevant financial relationships. Dr. May is a consultant for Exact Sciences and Geneoscopy, both of which are developing stool tests, and for Freenome, which is developing stool and blood tests.

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

Offering a blood test to people who have declined both a colonoscopy and a fecal immunochemical test (FIT) increased colorectal cancer (CRC) screening by 7.5% without decreasing use of the preferred first-line options, researchers report.

However, the number of people in the study who subsequently underwent timely colonoscopy after a positive blood test did not increase, signaling a continuing challenge in CRC prevention and treatment.

“The main message is that the blood test can do what it’s meant to do, which is increase screening uptake,” first author Peter Liang, MD, MPH, told this news organization. Dr. Liang is a gastroenterologist and researcher at NYU Langone Health and the VA New York Harbor Health Care System in New York.

The study was published online in Clinical Gastroenterology and Hepatology.

In the United States, the rate of use of first-line screening has for years been stuck at about 70% or lower for eligible people, Dr. Liang said. A different modality is needed to help raise the numbers.

The blood test is easy to perform and requires only a few tubes of blood, he noted. No diet restrictions, test prep, or contact with stool is necessary.

We are all searching for ways to get that first-line screening rate up from 60% to 70% to 80% to 90%, noted Folasade P. May, MD, PhD, who was not involved in the study.

“A lot of people think these blood tests are the promised land,” said Dr. May, associate professor of medicine at the University of California, Los Angeles, and director of the gastroenterology quality improvement program at UCLA Health. “We want to see that, when we offer these blood tests, the uptake is 20%-25% higher, which would get us closer to the national goal of 80% screened.”
 

Blood test as a second-line screening option

The study enrolled 359 veterans at a Veterans Affairs medical center. Participants were 50-75 years old and were eligible for screening but had declined a colonoscopy and a stool test within the previous 6 months.

They were randomly assigned to one of two groups. The control group received a letter and telephone outreach in which participants were again offered screening with colonoscopy or FIT only. The intervention group was additionally offered the blood test as a second-line option.

The primary outcome was completion of any screening test within 6 months. The secondary outcome was completion of a full screening strategy within 6 months, including colonoscopy for those with a positive noninvasive test result.

Of the people who had declined first-line tests and were reoffered first-line tests and the blood test, 17.1% completed screening within 6 months, compared with 9.6% of those who were only reoffered the first-line tests. The uptake of colonoscopy and FIT was similar between the two groups. The full-screening strategy was completed by 14.9% in the intervention group, compared with 9% in the control group.

At first glance, the results for uptake seem a bit disappointing, Dr. May said. However, the numbers in this study may not reflect the true potential of the blood tests – which are relatively new and have not yet been incorporated into routine care – because they had to be conducted in a separate appointment at a lab, she said.

If blood tests for CRC were part of the workflow, Dr. May explained, patients could undergo them with a routine blood draw already scheduled to check for diabetes or high cholesterol, for instance, and the numbers presumably would go up.

“I think this study underestimates the proportion of people who will participate,” she said. “We need a study that tests this strategy in a more real-world scenario.”

Nonetheless, “This is the first trial that’s looking at this question, and it’s an important question,” Dr. May added.

Dr. Liang and colleagues acknowledge that a limitation of the study is that it was performed in only one VA center among an older, predominantly male population, so it will be important to make the comparisons in more diverse study populations.
 

Blood test reliability

The septin 9 blood test is the only blood test approved by the U.S. Food and Drug Administration for CRC screening and is indicated for those who have declined first-line tests. However, the extent of usage of the blood test in this context is unclear, as it has only been approved since 2016.

Because the blood test is not covered by Medicare, Dr. Liang said, accessibility has been limited. One of the reasons for the lack of coverage is that the blood tests are less reliable than first-line tests, he said.

The test detects methylated septin 9 DNA, a biomarker for CRC. The FDA-approved version of the test has a sensitivity of 68% and a specificity of 79% for CRC.

Dr. May said she’d have more confidence in the blood tests if those numbers were higher, at 80%-90%.

Dr. Liang told this news organization that previous research has compared test use when colonoscopy, FIT, and a blood test are considered equally, but because the blood test is indicated only after a person declines first-line screening, his team designed an approach in which the blood test was a second-line option for its target population.

Other blood tests now on the market or under development appear to have higher sensitivity and specificity, he added.

“We think our results are actually applicable to a blood test in general,” Dr. Liang said.

Blood tests are only the first step, though. Getting people who screen positive to follow up with a diagnostic colonoscopy is critical, Dr. May and the authors agree.

“That’s something we, as a nation, just haven’t figured out,” Dr. May said. “It has to become a priority.”

The study was supported in part by the Veterans Health Administration and was funded by Epigenomics and a grant from the New York Society for Gastrointestinal Endoscopy’s Florence Lefcourt Endoscopy Research Award to Dr. Laing, who is also supported by the National Cancer Institute. Dr. Liang has received research support from Epigenomics and Freenome and is on the advisory board for Guardant Health. The remaining authors disclosed no relevant financial relationships. Dr. May is a consultant for Exact Sciences and Geneoscopy, both of which are developing stool tests, and for Freenome, which is developing stool and blood tests.

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

Continuing anticoagulation indefinitely in patients with a first unprovoked venous thromboembolism (VTE) may have benefits for certain patients but is unlikely to be cost effective, say authors of a new study.

Continued anticoagulation for such patients “has little chance of improving life expectancy but might provide a mortality benefit in certain subgroups including patients with an initial PE (pulmonary embolism) or those at a very low risk for major bleeding,” wrote the authors, led by Faizan Khan, PhD, with the O’Brien Institute for Public Health, University of Calgary (Alta.).

Therefore, shared decision-making between patients with unprovoked VTE and physicians that includes discussion of preferences and values and use of validated prediction tools is important.

The authors noted that some patients might value avoiding morbidities of recurrent VTE the most and want to have lifelong anticoagulation. Some might be more fearful of major bleeding than VTE repercussions or don’t want the inconveniences of taking anticoagulants for a lifetime.

The findings were published in Annals of Internal Medicine.
 

Current guidelines recommend indefinite anticoagulation

Clinical practice guidelines now recommend indefinite anticoagulation for a first unprovoked VTE.

The authors did a modeling study in a hypothetical cohort of 1,000 patients aged 55 years with a first unprovoked VTE who had completed 3-6 months of initial anticoagulation. The study found indefinite anticoagulation, compared with discontinuing anticoagulation, on average, resulted in 368 fewer recurrent VTE events and 14 fewer fatal PE events.

At the same time, indefinite coagulation in the hypothetical group induced an additional 114 major bleeding events, 30 intracerebral hemorrhages, and 11 fatal bleeding events over 40 years.

As for cost effectiveness, from the perspective of Canada’s health care system, continuing anticoagulation indefinitely, on average, increased costs by $16,014 Canadian dollars per person ($12,140 USD) without improving quality-adjusted life-years (incremental difference, 0.075 per person; 95% uncertainty interval, –0.192 to 0.017).

The authors noted that cost is a prime consideration as the estimated annual health care costs of VTE and its complications is $600 Canadian dollars ($7 billion–$10 billion USD).
 

High probability of small benefit

The authors spelled out the small benefit in patients with an initial PE.

According to the study, indefinite anticoagulation would result in an 80% probability of a marginal added clinical benefit (average increase of 57 days of perfect health over a lifetime) in patients with an initial PE (but with only a 24% chance of being cost effective).

“This high probability of an additional clinical benefit is plausible due to the higher proportion of recurrent VTE events presenting as PE (approximately 70% of episodes) in patients initially presenting with PE, in turn, resulting in a two- to threefold higher case-fatality rate of recurrent VTE in this patient subgroup.”
 

Tools to estimate bleeding risk imprecise

Scott Woller, MD, an internal medicine specialist and chair of medicine at Intermountain Medical Center, Murray, Utah, said in an interview that these results should help physicians’ discuss with their patients about duration of anticoagulation after the treatment phase.

He noted that the authors suggest that a low estimated annual risk for major bleeding should be assumed (< 0.67%) to make the choice for indefinite anticoagulation.

“This is a sticky wicket,” he said, “as tools to estimate bleeding risk among VTE patients are presently imprecise. For these reasons PCPs should take into account patient risk estimates – and the limitations that exist surrounding how we calculate these estimates – in addition to their values and preferences. This is really key in electing duration of anticoagulation.” 

A limitation of the study is that the model assumed that risks for recurrent VTE and major bleeding in clinical trials at 1 year remained constant during extended anticoagulation.

Dr. Woller said about that limitation: “One might argue that this is unlikely; age is a risk factor for major bleeding and therefore risks may be underestimated. However, in the ‘real world’ those that are perceived at lowest risk and demonstrate good tolerance to anticoagulation might likely preferentially continue anticoagulants and therefore risks may be overestimated.”

One coauthor reported being a clinical investigator for trials sponsored by Pfizer and Bristol-Myers Squibb and receiving honoraria from Pfizer, Sanofi and Aspen Pharma. The other authors disclosed no other relevant financial relationships. Dr. Woller is cochair of the CHEST guidelines on the treatment of venous thromboembolic disease.

Continuing anticoagulation indefinitely in patients with a first unprovoked venous thromboembolism (VTE) may have benefits for certain patients but is unlikely to be cost effective, say authors of a new study.

Continued anticoagulation for such patients “has little chance of improving life expectancy but might provide a mortality benefit in certain subgroups including patients with an initial PE (pulmonary embolism) or those at a very low risk for major bleeding,” wrote the authors, led by Faizan Khan, PhD, with the O’Brien Institute for Public Health, University of Calgary (Alta.).

Therefore, shared decision-making between patients with unprovoked VTE and physicians that includes discussion of preferences and values and use of validated prediction tools is important.

The authors noted that some patients might value avoiding morbidities of recurrent VTE the most and want to have lifelong anticoagulation. Some might be more fearful of major bleeding than VTE repercussions or don’t want the inconveniences of taking anticoagulants for a lifetime.

The findings were published in Annals of Internal Medicine.
 

Current guidelines recommend indefinite anticoagulation

Clinical practice guidelines now recommend indefinite anticoagulation for a first unprovoked VTE.

The authors did a modeling study in a hypothetical cohort of 1,000 patients aged 55 years with a first unprovoked VTE who had completed 3-6 months of initial anticoagulation. The study found indefinite anticoagulation, compared with discontinuing anticoagulation, on average, resulted in 368 fewer recurrent VTE events and 14 fewer fatal PE events.

At the same time, indefinite coagulation in the hypothetical group induced an additional 114 major bleeding events, 30 intracerebral hemorrhages, and 11 fatal bleeding events over 40 years.

As for cost effectiveness, from the perspective of Canada’s health care system, continuing anticoagulation indefinitely, on average, increased costs by $16,014 Canadian dollars per person ($12,140 USD) without improving quality-adjusted life-years (incremental difference, 0.075 per person; 95% uncertainty interval, –0.192 to 0.017).

The authors noted that cost is a prime consideration as the estimated annual health care costs of VTE and its complications is $600 Canadian dollars ($7 billion–$10 billion USD).
 

High probability of small benefit

The authors spelled out the small benefit in patients with an initial PE.

According to the study, indefinite anticoagulation would result in an 80% probability of a marginal added clinical benefit (average increase of 57 days of perfect health over a lifetime) in patients with an initial PE (but with only a 24% chance of being cost effective).

“This high probability of an additional clinical benefit is plausible due to the higher proportion of recurrent VTE events presenting as PE (approximately 70% of episodes) in patients initially presenting with PE, in turn, resulting in a two- to threefold higher case-fatality rate of recurrent VTE in this patient subgroup.”
 

Tools to estimate bleeding risk imprecise

Scott Woller, MD, an internal medicine specialist and chair of medicine at Intermountain Medical Center, Murray, Utah, said in an interview that these results should help physicians’ discuss with their patients about duration of anticoagulation after the treatment phase.

He noted that the authors suggest that a low estimated annual risk for major bleeding should be assumed (< 0.67%) to make the choice for indefinite anticoagulation.

“This is a sticky wicket,” he said, “as tools to estimate bleeding risk among VTE patients are presently imprecise. For these reasons PCPs should take into account patient risk estimates – and the limitations that exist surrounding how we calculate these estimates – in addition to their values and preferences. This is really key in electing duration of anticoagulation.” 

A limitation of the study is that the model assumed that risks for recurrent VTE and major bleeding in clinical trials at 1 year remained constant during extended anticoagulation.

Dr. Woller said about that limitation: “One might argue that this is unlikely; age is a risk factor for major bleeding and therefore risks may be underestimated. However, in the ‘real world’ those that are perceived at lowest risk and demonstrate good tolerance to anticoagulation might likely preferentially continue anticoagulants and therefore risks may be overestimated.”

One coauthor reported being a clinical investigator for trials sponsored by Pfizer and Bristol-Myers Squibb and receiving honoraria from Pfizer, Sanofi and Aspen Pharma. The other authors disclosed no other relevant financial relationships. Dr. Woller is cochair of the CHEST guidelines on the treatment of venous thromboembolic disease.

Continuing anticoagulation indefinitely in patients with a first unprovoked venous thromboembolism (VTE) may have benefits for certain patients but is unlikely to be cost effective, say authors of a new study.

Continued anticoagulation for such patients “has little chance of improving life expectancy but might provide a mortality benefit in certain subgroups including patients with an initial PE (pulmonary embolism) or those at a very low risk for major bleeding,” wrote the authors, led by Faizan Khan, PhD, with the O’Brien Institute for Public Health, University of Calgary (Alta.).

Therefore, shared decision-making between patients with unprovoked VTE and physicians that includes discussion of preferences and values and use of validated prediction tools is important.

The authors noted that some patients might value avoiding morbidities of recurrent VTE the most and want to have lifelong anticoagulation. Some might be more fearful of major bleeding than VTE repercussions or don’t want the inconveniences of taking anticoagulants for a lifetime.

The findings were published in Annals of Internal Medicine.
 

Current guidelines recommend indefinite anticoagulation

Clinical practice guidelines now recommend indefinite anticoagulation for a first unprovoked VTE.

The authors did a modeling study in a hypothetical cohort of 1,000 patients aged 55 years with a first unprovoked VTE who had completed 3-6 months of initial anticoagulation. The study found indefinite anticoagulation, compared with discontinuing anticoagulation, on average, resulted in 368 fewer recurrent VTE events and 14 fewer fatal PE events.

At the same time, indefinite coagulation in the hypothetical group induced an additional 114 major bleeding events, 30 intracerebral hemorrhages, and 11 fatal bleeding events over 40 years.

As for cost effectiveness, from the perspective of Canada’s health care system, continuing anticoagulation indefinitely, on average, increased costs by $16,014 Canadian dollars per person ($12,140 USD) without improving quality-adjusted life-years (incremental difference, 0.075 per person; 95% uncertainty interval, –0.192 to 0.017).

The authors noted that cost is a prime consideration as the estimated annual health care costs of VTE and its complications is $600 Canadian dollars ($7 billion–$10 billion USD).
 

High probability of small benefit

The authors spelled out the small benefit in patients with an initial PE.

According to the study, indefinite anticoagulation would result in an 80% probability of a marginal added clinical benefit (average increase of 57 days of perfect health over a lifetime) in patients with an initial PE (but with only a 24% chance of being cost effective).

“This high probability of an additional clinical benefit is plausible due to the higher proportion of recurrent VTE events presenting as PE (approximately 70% of episodes) in patients initially presenting with PE, in turn, resulting in a two- to threefold higher case-fatality rate of recurrent VTE in this patient subgroup.”
 

Tools to estimate bleeding risk imprecise

Scott Woller, MD, an internal medicine specialist and chair of medicine at Intermountain Medical Center, Murray, Utah, said in an interview that these results should help physicians’ discuss with their patients about duration of anticoagulation after the treatment phase.

He noted that the authors suggest that a low estimated annual risk for major bleeding should be assumed (< 0.67%) to make the choice for indefinite anticoagulation.

“This is a sticky wicket,” he said, “as tools to estimate bleeding risk among VTE patients are presently imprecise. For these reasons PCPs should take into account patient risk estimates – and the limitations that exist surrounding how we calculate these estimates – in addition to their values and preferences. This is really key in electing duration of anticoagulation.” 

A limitation of the study is that the model assumed that risks for recurrent VTE and major bleeding in clinical trials at 1 year remained constant during extended anticoagulation.

Dr. Woller said about that limitation: “One might argue that this is unlikely; age is a risk factor for major bleeding and therefore risks may be underestimated. However, in the ‘real world’ those that are perceived at lowest risk and demonstrate good tolerance to anticoagulation might likely preferentially continue anticoagulants and therefore risks may be overestimated.”

One coauthor reported being a clinical investigator for trials sponsored by Pfizer and Bristol-Myers Squibb and receiving honoraria from Pfizer, Sanofi and Aspen Pharma. The other authors disclosed no other relevant financial relationships. Dr. Woller is cochair of the CHEST guidelines on the treatment of venous thromboembolic disease.

Patients with head and neck cancer and overweight saw better treatment response and survival after chemoradiation, compared with patients with the same type of cancer but a normal weight, a new study finds.

The findings, published in JAMA Network Open, are the latest to parse the complex relationship between body mass index (BMI) and treatment in cancers that is sometimes called the “obesity paradox.” The researchers compared outcomes among patients with normal weight, overweight, and obesity.

While higher BMI is an established risk factor for many types of cancer and for cancer-specific mortality overall, studies in some cancers have shown that patients with higher BMI do better, possibly because excess BMI acts as a nutrient reserve against treatment-associated weight loss.
 

Methods and results

For their research, Sung Jun Ma, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., and colleagues looked at records for 445 patients (84% men, median age 61) at Dr. Ma’s institution with nonmetastatic head and neck cancer who underwent chemoradiotherapy between 2005 and 2021. Patients were followed up for a median 48 months, and those with underweight at treatment initiation were excluded.

The researchers found that overweight BMI (25-29.9 kg/m²) was associated with improved overall survival at 5 years (71% vs. 58% of patients with normal weight), as well as 5-year progression-free survival (68% vs. 51%). No overall or progression-free survival benefit link was seen in patients with a BMI of 30 or higher, in contrast to some previous studies of patients with head and neck cancers. BMI was not associated with improved survival outcomes among human papillomavirus–positive patients.

Both overweight and obesity were associated with complete response on follow-up PET-CT, with nearly 92% of patients with overweight and 91% of patients with obesity (defined as having a BMI of 30 or higher) seeing a complete metabolic response, compared with 74% of patients with normal weight.

Having an overweight BMI was also associated with improvements in tumor recurrence, with fewer of patients with this type of BMI experiencing 5-year locoregional failure than patients with normal weight (7% vs 26%). Having an obese BMI was not associated with improvements in recurrence. All the reported differences reached statistical significance.

The study authors surmised that the discrepancies between outcomes for patients with overweight and obesity “may be due to a nonlinear association between BMI and survival, with the highest survival seen in the overweight BMI range.”

It was important to note that this study saw no differences in treatment interruptions between the BMI groups that could account for differences in outcomes. Only three patients in the cohort saw their radiotherapy treatment interrupted, Dr. Ma said in an interview.

“If we felt that the obesity paradox happens because people with normal BMI lose too much weight during the treatment course, treatment gets interrupted, and they get worse outcomes from suboptimal treatments, then we would have seen more treatment interruptions among those with normal BMI. However, that was not the case in our study,” he said. Rather, the results point to “a complex interaction among cancer, [a person’s build], and nutritional status.”

Clinicians should be aware, Dr. Ma added, “that the same head and neck cancer may behave more aggressively among patients with normal BMI, compared to others with overweight BMI. Patients with normal BMI may need to be monitored more closely and carefully for potentially worse outcomes.” 

The investigators acknowledged several weaknesses of their study, including its retrospective design, the measure of BMI using cutoffs rather than a continuum, and the collection of BMI information at a single time point. While 84% of patients in the study received cisplatin, the study did not contain information on cumulative cisplatin dose.
 

Importance of nutritional support during treatment highlighted

In an interview, Ari Rosenberg, MD, of the University of Chicago Medicine, commented that the findings highlighted the importance of expert nutritional supportive care during treatment and monitoring for patients with advanced head and neck cancers undergoing chemoradiation.

“Nutritional status is very important both at baseline and during treatment,” Dr. Rosenberg said. “Even small changes in weight or BMI can be a key indicator of supportive care during chemoradiation and represent a biomarker to guide supportive management. ... The take home message is that patients should be treated at centers that have a high volume of advanced head and neck cancer patients, which have all the supportive components and expertise to optimize treatment delivery and maximize survival.”

Dr. Ma and colleagues’ study was funded by the National Cancer Institute Cancer Center. None of its authors declared financial conflicts of interest. Dr. Rosenberg disclosed receiving consulting fees from EMD Serono related to head and neck cancer treatment.
 

Patients with head and neck cancer and overweight saw better treatment response and survival after chemoradiation, compared with patients with the same type of cancer but a normal weight, a new study finds.

The findings, published in JAMA Network Open, are the latest to parse the complex relationship between body mass index (BMI) and treatment in cancers that is sometimes called the “obesity paradox.” The researchers compared outcomes among patients with normal weight, overweight, and obesity.

While higher BMI is an established risk factor for many types of cancer and for cancer-specific mortality overall, studies in some cancers have shown that patients with higher BMI do better, possibly because excess BMI acts as a nutrient reserve against treatment-associated weight loss.
 

Methods and results

For their research, Sung Jun Ma, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., and colleagues looked at records for 445 patients (84% men, median age 61) at Dr. Ma’s institution with nonmetastatic head and neck cancer who underwent chemoradiotherapy between 2005 and 2021. Patients were followed up for a median 48 months, and those with underweight at treatment initiation were excluded.

The researchers found that overweight BMI (25-29.9 kg/m²) was associated with improved overall survival at 5 years (71% vs. 58% of patients with normal weight), as well as 5-year progression-free survival (68% vs. 51%). No overall or progression-free survival benefit link was seen in patients with a BMI of 30 or higher, in contrast to some previous studies of patients with head and neck cancers. BMI was not associated with improved survival outcomes among human papillomavirus–positive patients.

Both overweight and obesity were associated with complete response on follow-up PET-CT, with nearly 92% of patients with overweight and 91% of patients with obesity (defined as having a BMI of 30 or higher) seeing a complete metabolic response, compared with 74% of patients with normal weight.

Having an overweight BMI was also associated with improvements in tumor recurrence, with fewer of patients with this type of BMI experiencing 5-year locoregional failure than patients with normal weight (7% vs 26%). Having an obese BMI was not associated with improvements in recurrence. All the reported differences reached statistical significance.

The study authors surmised that the discrepancies between outcomes for patients with overweight and obesity “may be due to a nonlinear association between BMI and survival, with the highest survival seen in the overweight BMI range.”

It was important to note that this study saw no differences in treatment interruptions between the BMI groups that could account for differences in outcomes. Only three patients in the cohort saw their radiotherapy treatment interrupted, Dr. Ma said in an interview.

“If we felt that the obesity paradox happens because people with normal BMI lose too much weight during the treatment course, treatment gets interrupted, and they get worse outcomes from suboptimal treatments, then we would have seen more treatment interruptions among those with normal BMI. However, that was not the case in our study,” he said. Rather, the results point to “a complex interaction among cancer, [a person’s build], and nutritional status.”

Clinicians should be aware, Dr. Ma added, “that the same head and neck cancer may behave more aggressively among patients with normal BMI, compared to others with overweight BMI. Patients with normal BMI may need to be monitored more closely and carefully for potentially worse outcomes.” 

The investigators acknowledged several weaknesses of their study, including its retrospective design, the measure of BMI using cutoffs rather than a continuum, and the collection of BMI information at a single time point. While 84% of patients in the study received cisplatin, the study did not contain information on cumulative cisplatin dose.
 

Importance of nutritional support during treatment highlighted

In an interview, Ari Rosenberg, MD, of the University of Chicago Medicine, commented that the findings highlighted the importance of expert nutritional supportive care during treatment and monitoring for patients with advanced head and neck cancers undergoing chemoradiation.

“Nutritional status is very important both at baseline and during treatment,” Dr. Rosenberg said. “Even small changes in weight or BMI can be a key indicator of supportive care during chemoradiation and represent a biomarker to guide supportive management. ... The take home message is that patients should be treated at centers that have a high volume of advanced head and neck cancer patients, which have all the supportive components and expertise to optimize treatment delivery and maximize survival.”

Dr. Ma and colleagues’ study was funded by the National Cancer Institute Cancer Center. None of its authors declared financial conflicts of interest. Dr. Rosenberg disclosed receiving consulting fees from EMD Serono related to head and neck cancer treatment.
 

Patients with head and neck cancer and overweight saw better treatment response and survival after chemoradiation, compared with patients with the same type of cancer but a normal weight, a new study finds.

The findings, published in JAMA Network Open, are the latest to parse the complex relationship between body mass index (BMI) and treatment in cancers that is sometimes called the “obesity paradox.” The researchers compared outcomes among patients with normal weight, overweight, and obesity.

While higher BMI is an established risk factor for many types of cancer and for cancer-specific mortality overall, studies in some cancers have shown that patients with higher BMI do better, possibly because excess BMI acts as a nutrient reserve against treatment-associated weight loss.
 

Methods and results

For their research, Sung Jun Ma, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., and colleagues looked at records for 445 patients (84% men, median age 61) at Dr. Ma’s institution with nonmetastatic head and neck cancer who underwent chemoradiotherapy between 2005 and 2021. Patients were followed up for a median 48 months, and those with underweight at treatment initiation were excluded.

The researchers found that overweight BMI (25-29.9 kg/m²) was associated with improved overall survival at 5 years (71% vs. 58% of patients with normal weight), as well as 5-year progression-free survival (68% vs. 51%). No overall or progression-free survival benefit link was seen in patients with a BMI of 30 or higher, in contrast to some previous studies of patients with head and neck cancers. BMI was not associated with improved survival outcomes among human papillomavirus–positive patients.

Both overweight and obesity were associated with complete response on follow-up PET-CT, with nearly 92% of patients with overweight and 91% of patients with obesity (defined as having a BMI of 30 or higher) seeing a complete metabolic response, compared with 74% of patients with normal weight.

Having an overweight BMI was also associated with improvements in tumor recurrence, with fewer of patients with this type of BMI experiencing 5-year locoregional failure than patients with normal weight (7% vs 26%). Having an obese BMI was not associated with improvements in recurrence. All the reported differences reached statistical significance.

The study authors surmised that the discrepancies between outcomes for patients with overweight and obesity “may be due to a nonlinear association between BMI and survival, with the highest survival seen in the overweight BMI range.”

It was important to note that this study saw no differences in treatment interruptions between the BMI groups that could account for differences in outcomes. Only three patients in the cohort saw their radiotherapy treatment interrupted, Dr. Ma said in an interview.

“If we felt that the obesity paradox happens because people with normal BMI lose too much weight during the treatment course, treatment gets interrupted, and they get worse outcomes from suboptimal treatments, then we would have seen more treatment interruptions among those with normal BMI. However, that was not the case in our study,” he said. Rather, the results point to “a complex interaction among cancer, [a person’s build], and nutritional status.”

Clinicians should be aware, Dr. Ma added, “that the same head and neck cancer may behave more aggressively among patients with normal BMI, compared to others with overweight BMI. Patients with normal BMI may need to be monitored more closely and carefully for potentially worse outcomes.” 

The investigators acknowledged several weaknesses of their study, including its retrospective design, the measure of BMI using cutoffs rather than a continuum, and the collection of BMI information at a single time point. While 84% of patients in the study received cisplatin, the study did not contain information on cumulative cisplatin dose.
 

Importance of nutritional support during treatment highlighted

In an interview, Ari Rosenberg, MD, of the University of Chicago Medicine, commented that the findings highlighted the importance of expert nutritional supportive care during treatment and monitoring for patients with advanced head and neck cancers undergoing chemoradiation.

“Nutritional status is very important both at baseline and during treatment,” Dr. Rosenberg said. “Even small changes in weight or BMI can be a key indicator of supportive care during chemoradiation and represent a biomarker to guide supportive management. ... The take home message is that patients should be treated at centers that have a high volume of advanced head and neck cancer patients, which have all the supportive components and expertise to optimize treatment delivery and maximize survival.”

Dr. Ma and colleagues’ study was funded by the National Cancer Institute Cancer Center. None of its authors declared financial conflicts of interest. Dr. Rosenberg disclosed receiving consulting fees from EMD Serono related to head and neck cancer treatment.
 

The Food and Drug Administration has rejected Intercept Pharmaceutical’s second bid for approval of obeticholic acid (OCA) for treatment of nonalcoholic steatohepatitis (NASH) with stage 2 or 3 fibrosis.

In response, the company has decided to discontinue all NASH-related investment.

Intercept first sought FDA approval for OCA in treatment of NASH in 2019 and received a complete response letter. The company refiled for a new drug application in December 2022. A second resubmission would require a completion of the long-term outcomes phase of an ongoing clinical trial, according to an Intercept press release.

The FDA decision follows the recommendation from May’s FDA Gastrointestinal Drugs Advisory Committee meeting. During the meeting, members voted 15 to 1 to advise deferring approval until clinical outcome data became available. Intercept’s clinical trial data demonstrated that OCA showed moderate benefit over placebo in improving fibrosis in NASH patients, but “there is uncertainty how the magnitude of changes in these surrogate endpoints may translate to meaningful changes in clinical outcomes,” an FDA meeting briefing document stated. There were also notable safety concerns including an increased risk for drug-induced liver injury.

An estimated 16.8 million Americans have NASH, and there are no FDA-approved medications for the condition.

Intercept plans to promptly begin closing out their NASH clinical trial and restructuring to focus on rare and serious liver diseases.

“While this is clearly not the outcome that we have worked toward, I’m proud of the impact that Intercept has made to move the science of NASH forward and bring the field closer to a treatment option,” said Jerry Durso, the president and CEO of Intercept, in a statement. “Intercept thanks the scientists, clinicians, and patients whose contributions to the clinical development of OCA in NASH have significantly advanced the understanding of this deadly disease.”

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

The Food and Drug Administration has rejected Intercept Pharmaceutical’s second bid for approval of obeticholic acid (OCA) for treatment of nonalcoholic steatohepatitis (NASH) with stage 2 or 3 fibrosis.

In response, the company has decided to discontinue all NASH-related investment.

Intercept first sought FDA approval for OCA in treatment of NASH in 2019 and received a complete response letter. The company refiled for a new drug application in December 2022. A second resubmission would require a completion of the long-term outcomes phase of an ongoing clinical trial, according to an Intercept press release.

The FDA decision follows the recommendation from May’s FDA Gastrointestinal Drugs Advisory Committee meeting. During the meeting, members voted 15 to 1 to advise deferring approval until clinical outcome data became available. Intercept’s clinical trial data demonstrated that OCA showed moderate benefit over placebo in improving fibrosis in NASH patients, but “there is uncertainty how the magnitude of changes in these surrogate endpoints may translate to meaningful changes in clinical outcomes,” an FDA meeting briefing document stated. There were also notable safety concerns including an increased risk for drug-induced liver injury.

An estimated 16.8 million Americans have NASH, and there are no FDA-approved medications for the condition.

Intercept plans to promptly begin closing out their NASH clinical trial and restructuring to focus on rare and serious liver diseases.

“While this is clearly not the outcome that we have worked toward, I’m proud of the impact that Intercept has made to move the science of NASH forward and bring the field closer to a treatment option,” said Jerry Durso, the president and CEO of Intercept, in a statement. “Intercept thanks the scientists, clinicians, and patients whose contributions to the clinical development of OCA in NASH have significantly advanced the understanding of this deadly disease.”

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

The Food and Drug Administration has rejected Intercept Pharmaceutical’s second bid for approval of obeticholic acid (OCA) for treatment of nonalcoholic steatohepatitis (NASH) with stage 2 or 3 fibrosis.

In response, the company has decided to discontinue all NASH-related investment.

Intercept first sought FDA approval for OCA in treatment of NASH in 2019 and received a complete response letter. The company refiled for a new drug application in December 2022. A second resubmission would require a completion of the long-term outcomes phase of an ongoing clinical trial, according to an Intercept press release.

The FDA decision follows the recommendation from May’s FDA Gastrointestinal Drugs Advisory Committee meeting. During the meeting, members voted 15 to 1 to advise deferring approval until clinical outcome data became available. Intercept’s clinical trial data demonstrated that OCA showed moderate benefit over placebo in improving fibrosis in NASH patients, but “there is uncertainty how the magnitude of changes in these surrogate endpoints may translate to meaningful changes in clinical outcomes,” an FDA meeting briefing document stated. There were also notable safety concerns including an increased risk for drug-induced liver injury.

An estimated 16.8 million Americans have NASH, and there are no FDA-approved medications for the condition.

Intercept plans to promptly begin closing out their NASH clinical trial and restructuring to focus on rare and serious liver diseases.

“While this is clearly not the outcome that we have worked toward, I’m proud of the impact that Intercept has made to move the science of NASH forward and bring the field closer to a treatment option,” said Jerry Durso, the president and CEO of Intercept, in a statement. “Intercept thanks the scientists, clinicians, and patients whose contributions to the clinical development of OCA in NASH have significantly advanced the understanding of this deadly disease.”

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

The European Society for Medical Oncology (ESMO), in collaboration with the European Hematology Association, has released a tool to help hematologists evaluate the magnitude of clinical benefit expected from new blood cancer treatments.

It consists of 11 2- to 3-page forms with checklists to grade treatment trials on the extent to which they meet efficacy and safety thresholds. Each of the 11 forms covers a specific trial scenario, such as a randomized controlled trial with curative intent or a trial of a therapy that is not likely to be curative with a primary endpoint of overall survival.

Treatments with curative intent are graded A, B, or C, while treatments in the noncurative setting are graded on a descending scale from 5 to 1. Scores of A and B in the curative setting and 5 and 4 in the noncurative setting represent substantial benefit.

On the form for RCTs with curative intent, for instance, a survival improvement of 5% or more garners an A but an improvement of less than 3% gets a C. Scores are also annotated for serious acute and/or persistent toxicity if present.

The tool, dubbed the ESMO-MCBS:H (European Society for Medical Oncology Magnitude of Clinical Benefit Scale: Hematology), is explained in an article published in Annals of Oncology. The evaluation forms are available online.

The idea behind the work is to help health care professionals and others to more “accurately assess the value of and prioritise therapies for patients with blood cancers. For clinicians, ESMO-MCBS:H will aid in their clinical decision-making and in the development of evidence-based practice and guidelines,” ESMO said in a press release.

To develop ESMO-MCBS:H, the group tailored its tool for evaluating solid tumor therapies, the ESMO-MCBS, to account for the sometimes different endpoints used in hematologic malignancy trials and the very indolent nature of some blood cancers, such as follicular lymphoma, which hampers development of mature data.

Specific changes include adding a new evaluation form to grade single-arm trials with curative intent, such as those used for CAR-T-cell therapies; incorporating molecular surrogate endpoints used in CML trials; and adding a way to grade outcomes for indolent cancers, among others.

The development process included applying the solid tumor tool to 80 blood cancer studies to identify shortcomings and improve its applicability. The final tool was field tested with 51 international experts from EHA and ESMO who largely agreed on the reasonableness of the trial scores.

ESMO said it expects ESMO-MCBS:H will be useful. The solid tumor tool, first published in 2015, is used by the World Health Organization to screen medications for its essential medicines list as well as by ESMO to generate guidelines and oncology centers across Europe to help with resource allocation decisions.

The European Society for Medical Oncology (ESMO), in collaboration with the European Hematology Association, has released a tool to help hematologists evaluate the magnitude of clinical benefit expected from new blood cancer treatments.

It consists of 11 2- to 3-page forms with checklists to grade treatment trials on the extent to which they meet efficacy and safety thresholds. Each of the 11 forms covers a specific trial scenario, such as a randomized controlled trial with curative intent or a trial of a therapy that is not likely to be curative with a primary endpoint of overall survival.

Treatments with curative intent are graded A, B, or C, while treatments in the noncurative setting are graded on a descending scale from 5 to 1. Scores of A and B in the curative setting and 5 and 4 in the noncurative setting represent substantial benefit.

On the form for RCTs with curative intent, for instance, a survival improvement of 5% or more garners an A but an improvement of less than 3% gets a C. Scores are also annotated for serious acute and/or persistent toxicity if present.

The tool, dubbed the ESMO-MCBS:H (European Society for Medical Oncology Magnitude of Clinical Benefit Scale: Hematology), is explained in an article published in Annals of Oncology. The evaluation forms are available online.

The idea behind the work is to help health care professionals and others to more “accurately assess the value of and prioritise therapies for patients with blood cancers. For clinicians, ESMO-MCBS:H will aid in their clinical decision-making and in the development of evidence-based practice and guidelines,” ESMO said in a press release.

To develop ESMO-MCBS:H, the group tailored its tool for evaluating solid tumor therapies, the ESMO-MCBS, to account for the sometimes different endpoints used in hematologic malignancy trials and the very indolent nature of some blood cancers, such as follicular lymphoma, which hampers development of mature data.

Specific changes include adding a new evaluation form to grade single-arm trials with curative intent, such as those used for CAR-T-cell therapies; incorporating molecular surrogate endpoints used in CML trials; and adding a way to grade outcomes for indolent cancers, among others.

The development process included applying the solid tumor tool to 80 blood cancer studies to identify shortcomings and improve its applicability. The final tool was field tested with 51 international experts from EHA and ESMO who largely agreed on the reasonableness of the trial scores.

ESMO said it expects ESMO-MCBS:H will be useful. The solid tumor tool, first published in 2015, is used by the World Health Organization to screen medications for its essential medicines list as well as by ESMO to generate guidelines and oncology centers across Europe to help with resource allocation decisions.

The European Society for Medical Oncology (ESMO), in collaboration with the European Hematology Association, has released a tool to help hematologists evaluate the magnitude of clinical benefit expected from new blood cancer treatments.

It consists of 11 2- to 3-page forms with checklists to grade treatment trials on the extent to which they meet efficacy and safety thresholds. Each of the 11 forms covers a specific trial scenario, such as a randomized controlled trial with curative intent or a trial of a therapy that is not likely to be curative with a primary endpoint of overall survival.

Treatments with curative intent are graded A, B, or C, while treatments in the noncurative setting are graded on a descending scale from 5 to 1. Scores of A and B in the curative setting and 5 and 4 in the noncurative setting represent substantial benefit.

On the form for RCTs with curative intent, for instance, a survival improvement of 5% or more garners an A but an improvement of less than 3% gets a C. Scores are also annotated for serious acute and/or persistent toxicity if present.

The tool, dubbed the ESMO-MCBS:H (European Society for Medical Oncology Magnitude of Clinical Benefit Scale: Hematology), is explained in an article published in Annals of Oncology. The evaluation forms are available online.

The idea behind the work is to help health care professionals and others to more “accurately assess the value of and prioritise therapies for patients with blood cancers. For clinicians, ESMO-MCBS:H will aid in their clinical decision-making and in the development of evidence-based practice and guidelines,” ESMO said in a press release.

To develop ESMO-MCBS:H, the group tailored its tool for evaluating solid tumor therapies, the ESMO-MCBS, to account for the sometimes different endpoints used in hematologic malignancy trials and the very indolent nature of some blood cancers, such as follicular lymphoma, which hampers development of mature data.

Specific changes include adding a new evaluation form to grade single-arm trials with curative intent, such as those used for CAR-T-cell therapies; incorporating molecular surrogate endpoints used in CML trials; and adding a way to grade outcomes for indolent cancers, among others.

The development process included applying the solid tumor tool to 80 blood cancer studies to identify shortcomings and improve its applicability. The final tool was field tested with 51 international experts from EHA and ESMO who largely agreed on the reasonableness of the trial scores.

ESMO said it expects ESMO-MCBS:H will be useful. The solid tumor tool, first published in 2015, is used by the World Health Organization to screen medications for its essential medicines list as well as by ESMO to generate guidelines and oncology centers across Europe to help with resource allocation decisions.