“No one has asked young people what they want,” said Tabitha Randell, MBChB, an endocrinologist with Nottingham (England) University Hospitals NHS Trust, who specializes in treating teenagers with type 1 diabetes as they transition to adult care.

Dr. Randell, who has set up a very successful specialist service in her hospital for such patients, said: “We consistently have the best, or the second best, outcomes in this country for our diabetes patients.” She believes this is one of the most important issues in modern endocrinology today.

Speaking at the Diabetes Professional Care conference in London at the end of 2021, and sharing her thoughts afterward with this news organization, she noted that in general there are “virtually no published outcomes” on how best to transition a patient with type 1 diabetes from pediatric to adult care.

“If you actually get them to transition – because some just drop out and disengage and there’s nothing you can do – none of them get lost. Some of them disengage in the adult clinic, but if you’re in the young diabetes service [in England] the rules are that if you miss a diabetes appointment you do not get discharged, as compared with the adult clinic, where if you miss an appointment, you are discharged.”

In the young diabetes clinic, doctors will “carry on trying to contact you, and get you back,” she explained. “And the patients do eventually come back in – it might be a year or 2, but they do come back. We’ve just got to keep them alive in the meantime!”

This issue needs tackling all over the world. Dr. Randell said she’s not aware of any one country – although there may be “pockets” of good care within a given country – that is doing this perfectly.

Across the pond, Grazia Aleppo, MD, division of endocrinology at Northwestern University, Chicago, agreed that transitioning pediatric patients with type 1 diabetes to adult care presents “unique challenges.”
 

Challenges when transitioning from pediatric to adult care

During childhood, type 1 diabetes management is largely supervised by patients’ parents and members of the pediatric diabetes care team, which may include diabetes educators, psychologists, or social workers, as well as pediatric endocrinologists.

When the patient with type 1 diabetes becomes a young adult and takes over management of their own health, Dr. Aleppo said, the care team may diminish along with the time spent in provider visits.

The adult endocrinology setting focuses more on self-management and autonomous functioning of the individual with diabetes.

Adult appointments are typically shorter, and the patient is usually expected to follow doctors’ suggestions independently, she noted. They are also expected to manage the practical aspects of their diabetes care, including prescriptions, diabetes supplies, laboratory tests, scheduling, and keeping appointments.

At the same time that the emerging adult needs to start asserting independence over their health care, they will also be going through a myriad of other important lifestyle changes, such as attending college, living on their own for the first time, and starting a career.  

“With these fundamental differences and challenges, competing priorities, such as college, work and relationships, medical care may become of secondary importance and patients may become disengaged,” Dr. Aleppo explained.

As Dr. Randell has said, loss to follow-up is a big problem with this patient population, with disengagement from specialist services and worsening A1c across the transition, Dr. Aleppo noted. This makes addressing these patients’ specific needs extremely important.
 

Engage with kid, not disease; don’t palm them off on new recruits

“The really key thing these kids say is, ‘I do not want to be a disease,’” Dr. Randell said. “They want you to know that they are a person. Engage these kids!” she suggested. “Ask them: ‘How is your exam revision going?’ Find something positive to say, even if it’s just: ‘I’m glad you came today.’ ”

“If the first thing that you do is tell them off [for poor diabetes care], you are never going to see them again,” she cautioned.

Dr. Randell also said that role models with type 1 diabetes, such as Lila Moss – daughter of British supermodel Kate Moss – who was recently pictured wearing an insulin pump on her leg on the catwalk, are helping youngsters not feel so self-conscious about their diabetes.

“Let them know it’s not the end of the world, having [type 1] diabetes,” she emphasized.  

And Partha Kar, MBBS, OBE, national specialty advisor, diabetes with NHS England, agreed wholeheartedly with Dr. Randall.

Reminiscing about his early days as a newly qualified endocrinologist, Dr. Kar, who works at Portsmouth (England) Hospital NHS Trust, noted that as a new member of staff he was given the youth with type 1 diabetes – those getting ready to transition to adult care – to look after.

But this is the exact opposite of what should be happening, he emphasized. “If you don’t think transition care is important, you shouldn’t be treating type 1 diabetes.”

He believes that every diabetes center “must have a young-adult team lead” and this job must not be given to the least experienced member of staff.

This lead “doesn’t need to be a doctor,” Dr. Kar stressed. “It can be a psychologist, or a diabetes nurse, or a pharmacist, or a dietician.”

In short, it must be someone experienced who loves working with this age group.  

Dr. Randell agreed: “Make sure the team is interested in young people. It shouldn’t be the last person in who gets the job no one else wants.” Teens “are my favorite group to work with. They don’t take any nonsense.”

And she explained: “Young people like to get to know the person who’s going to take care of them. So, stay with them for their young adult years.” This can be “quite a fluid period,” with it normally extending to age 25, but in some cases, “it can be up to 32 years old.”
 

Preparing for the transition

To ease pediatric patients into the transition to adult care, Dr. Aleppo recommended that the pediatric diabetes team provide enough time so that any concerns the patient and their family may have can be addressed.

This should also include transferring management responsibilities to the young adult rather than their parent.

The pediatric provider should discuss with the patient available potential adult colleagues, personalizing these options to their needs, she said.

And the adult and pediatric clinicians should collaborate and provide important information beyond medical records or health summaries.

Adult providers should guide young adults on how to navigate the new practices, from scheduling follow-up appointments to policies regarding medication refills or supplies, to providing information about urgent numbers or email addresses for after-hours communications.

Dr. Kar reiterated that there are too few published outcomes in this patient group to guide the establishment of good transition services.

“Without data, we are dead on the ground. Without data, it’s all conjecture, anecdotes,” he said.

What he does know is that, in the latest national type 1 diabetes audit for England, “Diabetic ketoacidosis admissions ... are up in this age group,” which suggests these patients are not receiving adequate care.
 

Be a guide, not a gatekeeper

Dr. Kar stressed that, of the 8,760 hours in a year, the average patient with type 1 diabetes in the United Kingdom gets just “1-2 hours with you as a clinician, based on four appointments per year of 30 minutes each.”

“So you spend 0.02% of their time with individuals with type 1 diabetes. So, what’s the one thing you can do with that minimal contact? Be nice!”

Dr. Kar said he always has his email open to his adult patients and they are very respectful of his time. “They don’t email you at 1 a.m. That means every one of my patients has got support [from me]. Don’t be a barrier.”

“We have to fundamentally change the narrative. Doctors must have more empathy,” he said, stating that the one thing adolescents have constantly given feedback on has been, “Why don’t appointments start with: ‘How are you?’ 

“For a teenager, if you throw type 1 diabetes into the loop, it’s not easy,” he stressed. “Talk to them about something else. As a clinician, be a guide, not a gatekeeper. Give people the tools to self-manage better.”

Adult providers can meet these young adult patients “at their level,” Dr. Aleppo agreed.

“Pay attention to their immediate needs and focus on their present circumstances – whether how to get through their next semester in college, navigating job interviews, or handling having diabetes in the workplace.”

Paying attention to the mental health needs of these young patients is equally “paramount,” Dr. Aleppo said.

While access to mental health professionals may be challenging in the adult setting, providers should bring it up with their patients and offer counseling referrals.

“Diabetes impacts everything, and office appointments and conversations carry weight on these patients’ lives as a whole, not just on their diabetes,” she stressed. “A patient told me recently: ‘We’re learning to be adults,’ which can be hard enough, and with diabetes it can be even more challenging. Adult providers need to be aware of the patient’s ‘diabetes language’ in that often it is not what a patient is saying, rather how they are saying it that gives us information on what they truly need.

“As adult providers, we need to also train and teach our young patients to advocate for themselves on where to find resources that can help them navigate adulthood with diabetes,” she added.

One particularly helpful resource in the United States is the College Diabetes Network, a not-for-profit organization whose mission is to equip young adults with type 1 diabetes to successfully manage the challenging transition to independence at college and beyond.

“The sweetest thing that can happen to us as adult diabetes providers is when a patient – seen as an emerging adult during college – returns to your practice 10 years later after moving back and seeks you out for their diabetes care because of the relationship and trust you developed in those transitioning years,” Dr. Aleppo said.

Another resource is a freely available comic book series cocreated by Dr. Kar and colleague Mayank Patel, MBBS, an endocrinologist from University Hospital Southampton NHS Foundation Trust.

As detailed by this news organization in 2021, the series consists of three volumes: the first, Type 1: Origins, focuses on actual experiences of patients who have type 1 diabetes; the second, Type 1: Attack of the Ketones, is aimed at professionals who may provide care but have limited understanding of type 1 diabetes; and the third, Type 1 Mission 3: S.T.I.G.M.A., addresses the stigmas and misconceptions that patients with type 1 diabetes may face.

The idea for the first comic was inspired by a patient who compared having diabetes to being like the Marvel character The Hulk, said Dr. Kar, and has been expanded to include the additional volumes.

Dr. Kar and Dr. Patel have also just launched the fourth comic in the series, Type 1: Generations, to mark the 100-year anniversary since insulin was first given to a human.
 

“This is high priority”  

Dr. Kar said the NHS in England has just appointed a national lead for type 1 diabetes in youth, Fulya Mehta, MD, of Alder Hey Children’s NHS Foundation Trust, Liverpool, England.

“If you have a plan, bring it to us,” he told the audience at the DPC conference, and “tell us, what is the one thing you would change? This is not a session we are doing just to tick a box. This is high priority.

“Encourage your colleagues to think about transition services. This is an absolute priority. We will be asking every center [in England] who is your transitioning lead?”

And he once again stressed that “a lead of transition service does not have to be a medic. This should be a multidisciplinary team. But they do need to be comfortable in that space. To that teenager, your job title means nothing. Give them time and space.”

Dr. Randell summed it up: “If we can work together, it’s only going to result in better outcomes. We need to blaze the trail for young people.”

Dr. Aleppo has reported serving as a consultant to Dexcom and Insulet and receiving support to Northwestern University from AstraZeneca, Dexcom, Eli Lilly, Fractyl Health, Insulet, and Novo Nordisk. Dr. Randell and Dr. Kar have no conflicts of interest.

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

“No one has asked young people what they want,” said Tabitha Randell, MBChB, an endocrinologist with Nottingham (England) University Hospitals NHS Trust, who specializes in treating teenagers with type 1 diabetes as they transition to adult care.

Dr. Randell, who has set up a very successful specialist service in her hospital for such patients, said: “We consistently have the best, or the second best, outcomes in this country for our diabetes patients.” She believes this is one of the most important issues in modern endocrinology today.

Speaking at the Diabetes Professional Care conference in London at the end of 2021, and sharing her thoughts afterward with this news organization, she noted that in general there are “virtually no published outcomes” on how best to transition a patient with type 1 diabetes from pediatric to adult care.

“If you actually get them to transition – because some just drop out and disengage and there’s nothing you can do – none of them get lost. Some of them disengage in the adult clinic, but if you’re in the young diabetes service [in England] the rules are that if you miss a diabetes appointment you do not get discharged, as compared with the adult clinic, where if you miss an appointment, you are discharged.”

In the young diabetes clinic, doctors will “carry on trying to contact you, and get you back,” she explained. “And the patients do eventually come back in – it might be a year or 2, but they do come back. We’ve just got to keep them alive in the meantime!”

This issue needs tackling all over the world. Dr. Randell said she’s not aware of any one country – although there may be “pockets” of good care within a given country – that is doing this perfectly.

Across the pond, Grazia Aleppo, MD, division of endocrinology at Northwestern University, Chicago, agreed that transitioning pediatric patients with type 1 diabetes to adult care presents “unique challenges.”
 

Challenges when transitioning from pediatric to adult care

During childhood, type 1 diabetes management is largely supervised by patients’ parents and members of the pediatric diabetes care team, which may include diabetes educators, psychologists, or social workers, as well as pediatric endocrinologists.

When the patient with type 1 diabetes becomes a young adult and takes over management of their own health, Dr. Aleppo said, the care team may diminish along with the time spent in provider visits.

The adult endocrinology setting focuses more on self-management and autonomous functioning of the individual with diabetes.

Adult appointments are typically shorter, and the patient is usually expected to follow doctors’ suggestions independently, she noted. They are also expected to manage the practical aspects of their diabetes care, including prescriptions, diabetes supplies, laboratory tests, scheduling, and keeping appointments.

At the same time that the emerging adult needs to start asserting independence over their health care, they will also be going through a myriad of other important lifestyle changes, such as attending college, living on their own for the first time, and starting a career.  

“With these fundamental differences and challenges, competing priorities, such as college, work and relationships, medical care may become of secondary importance and patients may become disengaged,” Dr. Aleppo explained.

As Dr. Randell has said, loss to follow-up is a big problem with this patient population, with disengagement from specialist services and worsening A1c across the transition, Dr. Aleppo noted. This makes addressing these patients’ specific needs extremely important.
 

Engage with kid, not disease; don’t palm them off on new recruits

“The really key thing these kids say is, ‘I do not want to be a disease,’” Dr. Randell said. “They want you to know that they are a person. Engage these kids!” she suggested. “Ask them: ‘How is your exam revision going?’ Find something positive to say, even if it’s just: ‘I’m glad you came today.’ ”

“If the first thing that you do is tell them off [for poor diabetes care], you are never going to see them again,” she cautioned.

Dr. Randell also said that role models with type 1 diabetes, such as Lila Moss – daughter of British supermodel Kate Moss – who was recently pictured wearing an insulin pump on her leg on the catwalk, are helping youngsters not feel so self-conscious about their diabetes.

“Let them know it’s not the end of the world, having [type 1] diabetes,” she emphasized.  

And Partha Kar, MBBS, OBE, national specialty advisor, diabetes with NHS England, agreed wholeheartedly with Dr. Randall.

Reminiscing about his early days as a newly qualified endocrinologist, Dr. Kar, who works at Portsmouth (England) Hospital NHS Trust, noted that as a new member of staff he was given the youth with type 1 diabetes – those getting ready to transition to adult care – to look after.

But this is the exact opposite of what should be happening, he emphasized. “If you don’t think transition care is important, you shouldn’t be treating type 1 diabetes.”

He believes that every diabetes center “must have a young-adult team lead” and this job must not be given to the least experienced member of staff.

This lead “doesn’t need to be a doctor,” Dr. Kar stressed. “It can be a psychologist, or a diabetes nurse, or a pharmacist, or a dietician.”

In short, it must be someone experienced who loves working with this age group.  

Dr. Randell agreed: “Make sure the team is interested in young people. It shouldn’t be the last person in who gets the job no one else wants.” Teens “are my favorite group to work with. They don’t take any nonsense.”

And she explained: “Young people like to get to know the person who’s going to take care of them. So, stay with them for their young adult years.” This can be “quite a fluid period,” with it normally extending to age 25, but in some cases, “it can be up to 32 years old.”
 

Preparing for the transition

To ease pediatric patients into the transition to adult care, Dr. Aleppo recommended that the pediatric diabetes team provide enough time so that any concerns the patient and their family may have can be addressed.

This should also include transferring management responsibilities to the young adult rather than their parent.

The pediatric provider should discuss with the patient available potential adult colleagues, personalizing these options to their needs, she said.

And the adult and pediatric clinicians should collaborate and provide important information beyond medical records or health summaries.

Adult providers should guide young adults on how to navigate the new practices, from scheduling follow-up appointments to policies regarding medication refills or supplies, to providing information about urgent numbers or email addresses for after-hours communications.

Dr. Kar reiterated that there are too few published outcomes in this patient group to guide the establishment of good transition services.

“Without data, we are dead on the ground. Without data, it’s all conjecture, anecdotes,” he said.

What he does know is that, in the latest national type 1 diabetes audit for England, “Diabetic ketoacidosis admissions ... are up in this age group,” which suggests these patients are not receiving adequate care.
 

Be a guide, not a gatekeeper

Dr. Kar stressed that, of the 8,760 hours in a year, the average patient with type 1 diabetes in the United Kingdom gets just “1-2 hours with you as a clinician, based on four appointments per year of 30 minutes each.”

“So you spend 0.02% of their time with individuals with type 1 diabetes. So, what’s the one thing you can do with that minimal contact? Be nice!”

Dr. Kar said he always has his email open to his adult patients and they are very respectful of his time. “They don’t email you at 1 a.m. That means every one of my patients has got support [from me]. Don’t be a barrier.”

“We have to fundamentally change the narrative. Doctors must have more empathy,” he said, stating that the one thing adolescents have constantly given feedback on has been, “Why don’t appointments start with: ‘How are you?’ 

“For a teenager, if you throw type 1 diabetes into the loop, it’s not easy,” he stressed. “Talk to them about something else. As a clinician, be a guide, not a gatekeeper. Give people the tools to self-manage better.”

Adult providers can meet these young adult patients “at their level,” Dr. Aleppo agreed.

“Pay attention to their immediate needs and focus on their present circumstances – whether how to get through their next semester in college, navigating job interviews, or handling having diabetes in the workplace.”

Paying attention to the mental health needs of these young patients is equally “paramount,” Dr. Aleppo said.

While access to mental health professionals may be challenging in the adult setting, providers should bring it up with their patients and offer counseling referrals.

“Diabetes impacts everything, and office appointments and conversations carry weight on these patients’ lives as a whole, not just on their diabetes,” she stressed. “A patient told me recently: ‘We’re learning to be adults,’ which can be hard enough, and with diabetes it can be even more challenging. Adult providers need to be aware of the patient’s ‘diabetes language’ in that often it is not what a patient is saying, rather how they are saying it that gives us information on what they truly need.

“As adult providers, we need to also train and teach our young patients to advocate for themselves on where to find resources that can help them navigate adulthood with diabetes,” she added.

One particularly helpful resource in the United States is the College Diabetes Network, a not-for-profit organization whose mission is to equip young adults with type 1 diabetes to successfully manage the challenging transition to independence at college and beyond.

“The sweetest thing that can happen to us as adult diabetes providers is when a patient – seen as an emerging adult during college – returns to your practice 10 years later after moving back and seeks you out for their diabetes care because of the relationship and trust you developed in those transitioning years,” Dr. Aleppo said.

Another resource is a freely available comic book series cocreated by Dr. Kar and colleague Mayank Patel, MBBS, an endocrinologist from University Hospital Southampton NHS Foundation Trust.

As detailed by this news organization in 2021, the series consists of three volumes: the first, Type 1: Origins, focuses on actual experiences of patients who have type 1 diabetes; the second, Type 1: Attack of the Ketones, is aimed at professionals who may provide care but have limited understanding of type 1 diabetes; and the third, Type 1 Mission 3: S.T.I.G.M.A., addresses the stigmas and misconceptions that patients with type 1 diabetes may face.

The idea for the first comic was inspired by a patient who compared having diabetes to being like the Marvel character The Hulk, said Dr. Kar, and has been expanded to include the additional volumes.

Dr. Kar and Dr. Patel have also just launched the fourth comic in the series, Type 1: Generations, to mark the 100-year anniversary since insulin was first given to a human.
 

“This is high priority”  

Dr. Kar said the NHS in England has just appointed a national lead for type 1 diabetes in youth, Fulya Mehta, MD, of Alder Hey Children’s NHS Foundation Trust, Liverpool, England.

“If you have a plan, bring it to us,” he told the audience at the DPC conference, and “tell us, what is the one thing you would change? This is not a session we are doing just to tick a box. This is high priority.

“Encourage your colleagues to think about transition services. This is an absolute priority. We will be asking every center [in England] who is your transitioning lead?”

And he once again stressed that “a lead of transition service does not have to be a medic. This should be a multidisciplinary team. But they do need to be comfortable in that space. To that teenager, your job title means nothing. Give them time and space.”

Dr. Randell summed it up: “If we can work together, it’s only going to result in better outcomes. We need to blaze the trail for young people.”

Dr. Aleppo has reported serving as a consultant to Dexcom and Insulet and receiving support to Northwestern University from AstraZeneca, Dexcom, Eli Lilly, Fractyl Health, Insulet, and Novo Nordisk. Dr. Randell and Dr. Kar have no conflicts of interest.

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

“No one has asked young people what they want,” said Tabitha Randell, MBChB, an endocrinologist with Nottingham (England) University Hospitals NHS Trust, who specializes in treating teenagers with type 1 diabetes as they transition to adult care.

Dr. Randell, who has set up a very successful specialist service in her hospital for such patients, said: “We consistently have the best, or the second best, outcomes in this country for our diabetes patients.” She believes this is one of the most important issues in modern endocrinology today.

Speaking at the Diabetes Professional Care conference in London at the end of 2021, and sharing her thoughts afterward with this news organization, she noted that in general there are “virtually no published outcomes” on how best to transition a patient with type 1 diabetes from pediatric to adult care.

“If you actually get them to transition – because some just drop out and disengage and there’s nothing you can do – none of them get lost. Some of them disengage in the adult clinic, but if you’re in the young diabetes service [in England] the rules are that if you miss a diabetes appointment you do not get discharged, as compared with the adult clinic, where if you miss an appointment, you are discharged.”

In the young diabetes clinic, doctors will “carry on trying to contact you, and get you back,” she explained. “And the patients do eventually come back in – it might be a year or 2, but they do come back. We’ve just got to keep them alive in the meantime!”

This issue needs tackling all over the world. Dr. Randell said she’s not aware of any one country – although there may be “pockets” of good care within a given country – that is doing this perfectly.

Across the pond, Grazia Aleppo, MD, division of endocrinology at Northwestern University, Chicago, agreed that transitioning pediatric patients with type 1 diabetes to adult care presents “unique challenges.”
 

Challenges when transitioning from pediatric to adult care

During childhood, type 1 diabetes management is largely supervised by patients’ parents and members of the pediatric diabetes care team, which may include diabetes educators, psychologists, or social workers, as well as pediatric endocrinologists.

When the patient with type 1 diabetes becomes a young adult and takes over management of their own health, Dr. Aleppo said, the care team may diminish along with the time spent in provider visits.

The adult endocrinology setting focuses more on self-management and autonomous functioning of the individual with diabetes.

Adult appointments are typically shorter, and the patient is usually expected to follow doctors’ suggestions independently, she noted. They are also expected to manage the practical aspects of their diabetes care, including prescriptions, diabetes supplies, laboratory tests, scheduling, and keeping appointments.

At the same time that the emerging adult needs to start asserting independence over their health care, they will also be going through a myriad of other important lifestyle changes, such as attending college, living on their own for the first time, and starting a career.  

“With these fundamental differences and challenges, competing priorities, such as college, work and relationships, medical care may become of secondary importance and patients may become disengaged,” Dr. Aleppo explained.

As Dr. Randell has said, loss to follow-up is a big problem with this patient population, with disengagement from specialist services and worsening A1c across the transition, Dr. Aleppo noted. This makes addressing these patients’ specific needs extremely important.
 

Engage with kid, not disease; don’t palm them off on new recruits

“The really key thing these kids say is, ‘I do not want to be a disease,’” Dr. Randell said. “They want you to know that they are a person. Engage these kids!” she suggested. “Ask them: ‘How is your exam revision going?’ Find something positive to say, even if it’s just: ‘I’m glad you came today.’ ”

“If the first thing that you do is tell them off [for poor diabetes care], you are never going to see them again,” she cautioned.

Dr. Randell also said that role models with type 1 diabetes, such as Lila Moss – daughter of British supermodel Kate Moss – who was recently pictured wearing an insulin pump on her leg on the catwalk, are helping youngsters not feel so self-conscious about their diabetes.

“Let them know it’s not the end of the world, having [type 1] diabetes,” she emphasized.  

And Partha Kar, MBBS, OBE, national specialty advisor, diabetes with NHS England, agreed wholeheartedly with Dr. Randall.

Reminiscing about his early days as a newly qualified endocrinologist, Dr. Kar, who works at Portsmouth (England) Hospital NHS Trust, noted that as a new member of staff he was given the youth with type 1 diabetes – those getting ready to transition to adult care – to look after.

But this is the exact opposite of what should be happening, he emphasized. “If you don’t think transition care is important, you shouldn’t be treating type 1 diabetes.”

He believes that every diabetes center “must have a young-adult team lead” and this job must not be given to the least experienced member of staff.

This lead “doesn’t need to be a doctor,” Dr. Kar stressed. “It can be a psychologist, or a diabetes nurse, or a pharmacist, or a dietician.”

In short, it must be someone experienced who loves working with this age group.  

Dr. Randell agreed: “Make sure the team is interested in young people. It shouldn’t be the last person in who gets the job no one else wants.” Teens “are my favorite group to work with. They don’t take any nonsense.”

And she explained: “Young people like to get to know the person who’s going to take care of them. So, stay with them for their young adult years.” This can be “quite a fluid period,” with it normally extending to age 25, but in some cases, “it can be up to 32 years old.”
 

Preparing for the transition

To ease pediatric patients into the transition to adult care, Dr. Aleppo recommended that the pediatric diabetes team provide enough time so that any concerns the patient and their family may have can be addressed.

This should also include transferring management responsibilities to the young adult rather than their parent.

The pediatric provider should discuss with the patient available potential adult colleagues, personalizing these options to their needs, she said.

And the adult and pediatric clinicians should collaborate and provide important information beyond medical records or health summaries.

Adult providers should guide young adults on how to navigate the new practices, from scheduling follow-up appointments to policies regarding medication refills or supplies, to providing information about urgent numbers or email addresses for after-hours communications.

Dr. Kar reiterated that there are too few published outcomes in this patient group to guide the establishment of good transition services.

“Without data, we are dead on the ground. Without data, it’s all conjecture, anecdotes,” he said.

What he does know is that, in the latest national type 1 diabetes audit for England, “Diabetic ketoacidosis admissions ... are up in this age group,” which suggests these patients are not receiving adequate care.
 

Be a guide, not a gatekeeper

Dr. Kar stressed that, of the 8,760 hours in a year, the average patient with type 1 diabetes in the United Kingdom gets just “1-2 hours with you as a clinician, based on four appointments per year of 30 minutes each.”

“So you spend 0.02% of their time with individuals with type 1 diabetes. So, what’s the one thing you can do with that minimal contact? Be nice!”

Dr. Kar said he always has his email open to his adult patients and they are very respectful of his time. “They don’t email you at 1 a.m. That means every one of my patients has got support [from me]. Don’t be a barrier.”

“We have to fundamentally change the narrative. Doctors must have more empathy,” he said, stating that the one thing adolescents have constantly given feedback on has been, “Why don’t appointments start with: ‘How are you?’ 

“For a teenager, if you throw type 1 diabetes into the loop, it’s not easy,” he stressed. “Talk to them about something else. As a clinician, be a guide, not a gatekeeper. Give people the tools to self-manage better.”

Adult providers can meet these young adult patients “at their level,” Dr. Aleppo agreed.

“Pay attention to their immediate needs and focus on their present circumstances – whether how to get through their next semester in college, navigating job interviews, or handling having diabetes in the workplace.”

Paying attention to the mental health needs of these young patients is equally “paramount,” Dr. Aleppo said.

While access to mental health professionals may be challenging in the adult setting, providers should bring it up with their patients and offer counseling referrals.

“Diabetes impacts everything, and office appointments and conversations carry weight on these patients’ lives as a whole, not just on their diabetes,” she stressed. “A patient told me recently: ‘We’re learning to be adults,’ which can be hard enough, and with diabetes it can be even more challenging. Adult providers need to be aware of the patient’s ‘diabetes language’ in that often it is not what a patient is saying, rather how they are saying it that gives us information on what they truly need.

“As adult providers, we need to also train and teach our young patients to advocate for themselves on where to find resources that can help them navigate adulthood with diabetes,” she added.

One particularly helpful resource in the United States is the College Diabetes Network, a not-for-profit organization whose mission is to equip young adults with type 1 diabetes to successfully manage the challenging transition to independence at college and beyond.

“The sweetest thing that can happen to us as adult diabetes providers is when a patient – seen as an emerging adult during college – returns to your practice 10 years later after moving back and seeks you out for their diabetes care because of the relationship and trust you developed in those transitioning years,” Dr. Aleppo said.

Another resource is a freely available comic book series cocreated by Dr. Kar and colleague Mayank Patel, MBBS, an endocrinologist from University Hospital Southampton NHS Foundation Trust.

As detailed by this news organization in 2021, the series consists of three volumes: the first, Type 1: Origins, focuses on actual experiences of patients who have type 1 diabetes; the second, Type 1: Attack of the Ketones, is aimed at professionals who may provide care but have limited understanding of type 1 diabetes; and the third, Type 1 Mission 3: S.T.I.G.M.A., addresses the stigmas and misconceptions that patients with type 1 diabetes may face.

The idea for the first comic was inspired by a patient who compared having diabetes to being like the Marvel character The Hulk, said Dr. Kar, and has been expanded to include the additional volumes.

Dr. Kar and Dr. Patel have also just launched the fourth comic in the series, Type 1: Generations, to mark the 100-year anniversary since insulin was first given to a human.
 

“This is high priority”  

Dr. Kar said the NHS in England has just appointed a national lead for type 1 diabetes in youth, Fulya Mehta, MD, of Alder Hey Children’s NHS Foundation Trust, Liverpool, England.

“If you have a plan, bring it to us,” he told the audience at the DPC conference, and “tell us, what is the one thing you would change? This is not a session we are doing just to tick a box. This is high priority.

“Encourage your colleagues to think about transition services. This is an absolute priority. We will be asking every center [in England] who is your transitioning lead?”

And he once again stressed that “a lead of transition service does not have to be a medic. This should be a multidisciplinary team. But they do need to be comfortable in that space. To that teenager, your job title means nothing. Give them time and space.”

Dr. Randell summed it up: “If we can work together, it’s only going to result in better outcomes. We need to blaze the trail for young people.”

Dr. Aleppo has reported serving as a consultant to Dexcom and Insulet and receiving support to Northwestern University from AstraZeneca, Dexcom, Eli Lilly, Fractyl Health, Insulet, and Novo Nordisk. Dr. Randell and Dr. Kar have no conflicts of interest.

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

Abraxane, a chemotherapy treatment for advanced pancreatic cancer, advanced non–small cell lung cancer and metastatic breast cancer, is on allocation through early March because of manufacturing delays, forcing physicians to find alternatives for a drug once lauded for being easier to tolerate.

Abraxane (Bristol-Myers Squibb) is a paclitaxel albumin-bound injectable. It is different from alternative chemotherapy treatments like Taxol (paclitaxel) because it doesn’t use the solvents that can make Taxol difficult to tolerate. It was described as a “next-generation taxane” because it didn’t rely on solvents. It was approved in 2005 for metastatic breast cancer, then in 2012 for advanced non–small cell lung cancer, in 2013 for late-stage pancreatic cancer and in 2019 for people with PD-L1–positive metastatic triple-negative breast cancer.

The shortage, which was announced on Oct. 5, 2021, by the Food and Drug Administration, has led to some difficult decisions for patients and physicians. How long the shortage will last isn’t clear.

“I printed out [an] allotment sheet 2 days ago, and all it says [for Abraxane] is allocated,” said Kathy Oubre, MS, CEO of Pontchartrain Cancer Center, Hammond, La. “Everyone is keeping what they’ve got for their own patients, so there really isn’t anything available.”

The Pontchartrain Cancer Center sent two patients to the University of Texas MD Anderson Cancer Center, Houston, for continued treatment with Abraxane, but that option is costly and time consuming for patients. The two patients had the means to travel, but Ms. Oubre said that many others cannot afford to travel for treatment. “Everyone has patients who are living paycheck to paycheck who certainly couldn’t afford to do that. There are going to be patients across the nation that are not going to be able to have care as a result of these things.”

The supply problems are causing difficult decisions for physicians, who may have to switch a patient from an unavailable drug to an alternative that isn’t as effective, Ms. Oubre said. “I can’t imagine the stress and the sadness that the physicians have to feel when they have to go explain that to a patient. That runs counter to everything they are as physicians.”

Other strategies include chemo holidays and rounding down doses in patients with metastatic cancer, according to Camille Hill, PharmD, vice president of oncology pharmacy services, West Cancer Center, Germantown, Tenn.

Shortages and allocations are growing at an alarming rate, Ms. Oubre said. In her 15 years of working in the industry, “I don’t recall it ever being this challenging.” During a Zoom interview, she held up a lengthy list of drugs on allocation or unavailable that her pharmacy group purchasing organization sent her the previous week. “I don’t ever recall getting this kind of list. Every 3 days, I’m getting this. If it were just that one product, I can live with that. We figure it out. But it’s bigger than that.”

Worker shortages are exacerbating the issue. Ms. Oubre received a letter from a drug company describing its employee issues, which included chemists, plant workers, and loading dock staff. On top of that, delivery companies are experiencing staff shortages, which can result in more delays and complicate matters further. “It’s just compounding. These things can get really difficult very quickly. I don’t want to say we’re in crisis, and we’re not rationing care. We’re not in those buckets yet. But I would say that if these things don’t get better, it’s the first time in my work career that we are having those conversations of: ‘How we are going to plan for that it does come to that?’ ” she said.

“In general, with the pandemic, we have seen all sorts of just disruptions to the supply chain. So, I think you just do your best, you find alternatives for those patients that you can, and you come up with strategies. I don’t know that for Abraxane, or any other product, that I’d be particularly confident that we may not see another shortage,” Dr. Hill said.

Abraxane, a chemotherapy treatment for advanced pancreatic cancer, advanced non–small cell lung cancer and metastatic breast cancer, is on allocation through early March because of manufacturing delays, forcing physicians to find alternatives for a drug once lauded for being easier to tolerate.

Abraxane (Bristol-Myers Squibb) is a paclitaxel albumin-bound injectable. It is different from alternative chemotherapy treatments like Taxol (paclitaxel) because it doesn’t use the solvents that can make Taxol difficult to tolerate. It was described as a “next-generation taxane” because it didn’t rely on solvents. It was approved in 2005 for metastatic breast cancer, then in 2012 for advanced non–small cell lung cancer, in 2013 for late-stage pancreatic cancer and in 2019 for people with PD-L1–positive metastatic triple-negative breast cancer.

The shortage, which was announced on Oct. 5, 2021, by the Food and Drug Administration, has led to some difficult decisions for patients and physicians. How long the shortage will last isn’t clear.

“I printed out [an] allotment sheet 2 days ago, and all it says [for Abraxane] is allocated,” said Kathy Oubre, MS, CEO of Pontchartrain Cancer Center, Hammond, La. “Everyone is keeping what they’ve got for their own patients, so there really isn’t anything available.”

The Pontchartrain Cancer Center sent two patients to the University of Texas MD Anderson Cancer Center, Houston, for continued treatment with Abraxane, but that option is costly and time consuming for patients. The two patients had the means to travel, but Ms. Oubre said that many others cannot afford to travel for treatment. “Everyone has patients who are living paycheck to paycheck who certainly couldn’t afford to do that. There are going to be patients across the nation that are not going to be able to have care as a result of these things.”

The supply problems are causing difficult decisions for physicians, who may have to switch a patient from an unavailable drug to an alternative that isn’t as effective, Ms. Oubre said. “I can’t imagine the stress and the sadness that the physicians have to feel when they have to go explain that to a patient. That runs counter to everything they are as physicians.”

Other strategies include chemo holidays and rounding down doses in patients with metastatic cancer, according to Camille Hill, PharmD, vice president of oncology pharmacy services, West Cancer Center, Germantown, Tenn.

Shortages and allocations are growing at an alarming rate, Ms. Oubre said. In her 15 years of working in the industry, “I don’t recall it ever being this challenging.” During a Zoom interview, she held up a lengthy list of drugs on allocation or unavailable that her pharmacy group purchasing organization sent her the previous week. “I don’t ever recall getting this kind of list. Every 3 days, I’m getting this. If it were just that one product, I can live with that. We figure it out. But it’s bigger than that.”

Worker shortages are exacerbating the issue. Ms. Oubre received a letter from a drug company describing its employee issues, which included chemists, plant workers, and loading dock staff. On top of that, delivery companies are experiencing staff shortages, which can result in more delays and complicate matters further. “It’s just compounding. These things can get really difficult very quickly. I don’t want to say we’re in crisis, and we’re not rationing care. We’re not in those buckets yet. But I would say that if these things don’t get better, it’s the first time in my work career that we are having those conversations of: ‘How we are going to plan for that it does come to that?’ ” she said.

“In general, with the pandemic, we have seen all sorts of just disruptions to the supply chain. So, I think you just do your best, you find alternatives for those patients that you can, and you come up with strategies. I don’t know that for Abraxane, or any other product, that I’d be particularly confident that we may not see another shortage,” Dr. Hill said.

Abraxane, a chemotherapy treatment for advanced pancreatic cancer, advanced non–small cell lung cancer and metastatic breast cancer, is on allocation through early March because of manufacturing delays, forcing physicians to find alternatives for a drug once lauded for being easier to tolerate.

Abraxane (Bristol-Myers Squibb) is a paclitaxel albumin-bound injectable. It is different from alternative chemotherapy treatments like Taxol (paclitaxel) because it doesn’t use the solvents that can make Taxol difficult to tolerate. It was described as a “next-generation taxane” because it didn’t rely on solvents. It was approved in 2005 for metastatic breast cancer, then in 2012 for advanced non–small cell lung cancer, in 2013 for late-stage pancreatic cancer and in 2019 for people with PD-L1–positive metastatic triple-negative breast cancer.

The shortage, which was announced on Oct. 5, 2021, by the Food and Drug Administration, has led to some difficult decisions for patients and physicians. How long the shortage will last isn’t clear.

“I printed out [an] allotment sheet 2 days ago, and all it says [for Abraxane] is allocated,” said Kathy Oubre, MS, CEO of Pontchartrain Cancer Center, Hammond, La. “Everyone is keeping what they’ve got for their own patients, so there really isn’t anything available.”

The Pontchartrain Cancer Center sent two patients to the University of Texas MD Anderson Cancer Center, Houston, for continued treatment with Abraxane, but that option is costly and time consuming for patients. The two patients had the means to travel, but Ms. Oubre said that many others cannot afford to travel for treatment. “Everyone has patients who are living paycheck to paycheck who certainly couldn’t afford to do that. There are going to be patients across the nation that are not going to be able to have care as a result of these things.”

The supply problems are causing difficult decisions for physicians, who may have to switch a patient from an unavailable drug to an alternative that isn’t as effective, Ms. Oubre said. “I can’t imagine the stress and the sadness that the physicians have to feel when they have to go explain that to a patient. That runs counter to everything they are as physicians.”

Other strategies include chemo holidays and rounding down doses in patients with metastatic cancer, according to Camille Hill, PharmD, vice president of oncology pharmacy services, West Cancer Center, Germantown, Tenn.

Shortages and allocations are growing at an alarming rate, Ms. Oubre said. In her 15 years of working in the industry, “I don’t recall it ever being this challenging.” During a Zoom interview, she held up a lengthy list of drugs on allocation or unavailable that her pharmacy group purchasing organization sent her the previous week. “I don’t ever recall getting this kind of list. Every 3 days, I’m getting this. If it were just that one product, I can live with that. We figure it out. But it’s bigger than that.”

Worker shortages are exacerbating the issue. Ms. Oubre received a letter from a drug company describing its employee issues, which included chemists, plant workers, and loading dock staff. On top of that, delivery companies are experiencing staff shortages, which can result in more delays and complicate matters further. “It’s just compounding. These things can get really difficult very quickly. I don’t want to say we’re in crisis, and we’re not rationing care. We’re not in those buckets yet. But I would say that if these things don’t get better, it’s the first time in my work career that we are having those conversations of: ‘How we are going to plan for that it does come to that?’ ” she said.

“In general, with the pandemic, we have seen all sorts of just disruptions to the supply chain. So, I think you just do your best, you find alternatives for those patients that you can, and you come up with strategies. I don’t know that for Abraxane, or any other product, that I’d be particularly confident that we may not see another shortage,” Dr. Hill said.

Early in December 2021 a jury awarded a couple $10 million in a case involving a hysterectomy that went badly wrong, according to a story in the New York Post, among other news sites.

In October 2018, Michele Nugent, 41, of New York, underwent the procedure at Richmond University Medical Center. After giving birth to four children via cesarean delivery, she had developed scarring and was suffering from excessive and painful uterine bleeding.

A few days after her hysterectomy, however, Ms. Nugent experienced what she described as the worst pain of her life, along with nausea, vomiting, and urinary leakage. She was brought to the hospital emergency department, where she was reportedly told by staff there that her symptoms were normal complications of her surgery and that the treating gynecologist, Eli Serur, MD, would soon be in touch.

Despite these reassurances, Ms. Nugent’s postsurgical issues continued to worsen over the next 10 days. Among other things, she lost almost complete control of her bladder, which required her to wear adult diapers. Still, her doctor’s office told her to put off visiting until her next scheduled appointment.

At that meeting, which took place 13 days after Ms. Nugent’s surgery, Dr. Serur diagnosed a urinary tract infection and placed her on antibiotics. He also encouraged her to return to work the following week.

Ms. Nugent’s problems persisted, however. At an office meeting that included 20 men, she suddenly lost complete control of her bladder, despite going regularly to the bathroom and wearing adult diapers. “Out of nowhere,” she testified at trial, “I urinated all over myself and had to leave.” The experience left her humiliated and embarrassed.

Several weeks later, Ms. Nugent consulted with a urologist, who soon repaired the cause of her urinary problems – a fistula between her vagina and bladder.

Though successful, the procedure still left Ms. Nugent with, what are for now at least, intractable symptoms. At night, she’s forced to make multiple trips to the bathroom, and sex with her husband has become all but impossible because of the pain it elicits.

In reaching its verdict, the jury of four women and two men faulted Dr. Serur for not only performing a faulty surgery but for failing to identify and correct his mistake. In so doing, it concluded, he had departed “from good and accepted medical practice.”

Jurors divided the $10 million judgment against him into two parts: $6.5 million for Ms. Nugent’s past and future suffering, and $3.5 million to her husband for his past and future loss of consortium – that is, his loss of intimacy with his wife.

As for the medical center, the Nugents agreed to dismiss it from the case prior to trial.
 

Physician accused of gross negligence finally surrenders his license

A California doctor under investigation multiple times during the past 2 decades has surrendered his medical license, as a story reported by Valley Public Radio indicates.

Since 1999, the Medical Board of California has opened three investigations against Bakersfield ob.gyn. Arthur Park, MD, each involving accusations of gross negligence “following the deaths of mothers and/or their babies during childbirth.” In 2000, and again in 2020, the board voted that Dr. Park should lose his license but then suspended its decision, which enabled Dr. Park to continue practicing under probation and on condition that he complete remedial education.

Early in 2021, however, the board filed yet another accusation against him, this one involving the 2019 death of Demi Dominguez and her newborn baby. According to the accusation, Ms. Dominguez died of preeclampsia because Dr. Park and a colleague failed to treat her high blood pressure prior to delivery. While doctors attempted to resuscitate her, Ms. Dominguez’s son was delivered by emergency cesarean but died only a few hours later. The board said that Dr. Park was “grossly negligent in his care and treatment” and that his actions constituted “an extreme departure from the applicable standard of care.”

Early in December 2021, even before the board and attorney general’s office had completed their investigations, Dr. Park agreed to surrender his medical license.

Patient advocates were pleased by the doctor’s decision but also disappointed that he’d no longer be compelled to stand before a judge, as he had been scheduled to do in connection with the Dominguez case.

A review of public records by Valley Public Radio indicates that – between the various board accusations against him and an additional nine lawsuits alleging malpractice and other issues – at least two mothers and five children have died while under Dr. Park’s care. Others whose delivery he oversaw claim their children were permanently injured during childbirth.

Although Dr. Park will be eligible to reapply for his license after 2 years, a representative of his medical office said Dr. Park had decided to retire from practicing medicine.
 

Delayed cancer diagnosis prompts med-mal suit

An Illinois woman who claims her doctor and his staff failed to follow up on her abnormal Pap smear has filed a malpractice suit against them and their medical group, reports a story in the Madison-St. Clair Record.

In early 2019, Lisa Albright visited the medical group after she had experienced pain during intercourse. A family nurse practitioner at the practice performed a Pap smear, and Ms. Albright was instructed to wait a few days and check her patient portal for the results. In her suit, Ms. Albright claims those results were abnormal. Despite this, neither the nurse practitioner nor anyone else at the practice scheduled a follow-up test or other diagnostic assessment.

Approximately 5 months later, Ms. Albright consulted a new physician, whose follow-up testing indicated that Ms. Albright had a cervical squamous cell carcinoma.

Ms. Albright’s suit alleges that the diagnostic delay has, among other things, caused her to undergo multiple surgical procedures and treatments, face a shorter life expectancy, and endure a loss in the quality of her life.

At press time, Ms. Albright and her legal representative have not yet determined the amount they will ask for – it will be set after the severity and permanency of Ms. Albright’s injuries have been more thoroughly investigated. But it’s expected that they will seek damages, along with all legal and court expenses.

The defendants haven’t responded to the plaintiff’s suit.

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

Early in December 2021 a jury awarded a couple $10 million in a case involving a hysterectomy that went badly wrong, according to a story in the New York Post, among other news sites.

In October 2018, Michele Nugent, 41, of New York, underwent the procedure at Richmond University Medical Center. After giving birth to four children via cesarean delivery, she had developed scarring and was suffering from excessive and painful uterine bleeding.

A few days after her hysterectomy, however, Ms. Nugent experienced what she described as the worst pain of her life, along with nausea, vomiting, and urinary leakage. She was brought to the hospital emergency department, where she was reportedly told by staff there that her symptoms were normal complications of her surgery and that the treating gynecologist, Eli Serur, MD, would soon be in touch.

Despite these reassurances, Ms. Nugent’s postsurgical issues continued to worsen over the next 10 days. Among other things, she lost almost complete control of her bladder, which required her to wear adult diapers. Still, her doctor’s office told her to put off visiting until her next scheduled appointment.

At that meeting, which took place 13 days after Ms. Nugent’s surgery, Dr. Serur diagnosed a urinary tract infection and placed her on antibiotics. He also encouraged her to return to work the following week.

Ms. Nugent’s problems persisted, however. At an office meeting that included 20 men, she suddenly lost complete control of her bladder, despite going regularly to the bathroom and wearing adult diapers. “Out of nowhere,” she testified at trial, “I urinated all over myself and had to leave.” The experience left her humiliated and embarrassed.

Several weeks later, Ms. Nugent consulted with a urologist, who soon repaired the cause of her urinary problems – a fistula between her vagina and bladder.

Though successful, the procedure still left Ms. Nugent with, what are for now at least, intractable symptoms. At night, she’s forced to make multiple trips to the bathroom, and sex with her husband has become all but impossible because of the pain it elicits.

In reaching its verdict, the jury of four women and two men faulted Dr. Serur for not only performing a faulty surgery but for failing to identify and correct his mistake. In so doing, it concluded, he had departed “from good and accepted medical practice.”

Jurors divided the $10 million judgment against him into two parts: $6.5 million for Ms. Nugent’s past and future suffering, and $3.5 million to her husband for his past and future loss of consortium – that is, his loss of intimacy with his wife.

As for the medical center, the Nugents agreed to dismiss it from the case prior to trial.
 

Physician accused of gross negligence finally surrenders his license

A California doctor under investigation multiple times during the past 2 decades has surrendered his medical license, as a story reported by Valley Public Radio indicates.

Since 1999, the Medical Board of California has opened three investigations against Bakersfield ob.gyn. Arthur Park, MD, each involving accusations of gross negligence “following the deaths of mothers and/or their babies during childbirth.” In 2000, and again in 2020, the board voted that Dr. Park should lose his license but then suspended its decision, which enabled Dr. Park to continue practicing under probation and on condition that he complete remedial education.

Early in 2021, however, the board filed yet another accusation against him, this one involving the 2019 death of Demi Dominguez and her newborn baby. According to the accusation, Ms. Dominguez died of preeclampsia because Dr. Park and a colleague failed to treat her high blood pressure prior to delivery. While doctors attempted to resuscitate her, Ms. Dominguez’s son was delivered by emergency cesarean but died only a few hours later. The board said that Dr. Park was “grossly negligent in his care and treatment” and that his actions constituted “an extreme departure from the applicable standard of care.”

Early in December 2021, even before the board and attorney general’s office had completed their investigations, Dr. Park agreed to surrender his medical license.

Patient advocates were pleased by the doctor’s decision but also disappointed that he’d no longer be compelled to stand before a judge, as he had been scheduled to do in connection with the Dominguez case.

A review of public records by Valley Public Radio indicates that – between the various board accusations against him and an additional nine lawsuits alleging malpractice and other issues – at least two mothers and five children have died while under Dr. Park’s care. Others whose delivery he oversaw claim their children were permanently injured during childbirth.

Although Dr. Park will be eligible to reapply for his license after 2 years, a representative of his medical office said Dr. Park had decided to retire from practicing medicine.
 

Delayed cancer diagnosis prompts med-mal suit

An Illinois woman who claims her doctor and his staff failed to follow up on her abnormal Pap smear has filed a malpractice suit against them and their medical group, reports a story in the Madison-St. Clair Record.

In early 2019, Lisa Albright visited the medical group after she had experienced pain during intercourse. A family nurse practitioner at the practice performed a Pap smear, and Ms. Albright was instructed to wait a few days and check her patient portal for the results. In her suit, Ms. Albright claims those results were abnormal. Despite this, neither the nurse practitioner nor anyone else at the practice scheduled a follow-up test or other diagnostic assessment.

Approximately 5 months later, Ms. Albright consulted a new physician, whose follow-up testing indicated that Ms. Albright had a cervical squamous cell carcinoma.

Ms. Albright’s suit alleges that the diagnostic delay has, among other things, caused her to undergo multiple surgical procedures and treatments, face a shorter life expectancy, and endure a loss in the quality of her life.

At press time, Ms. Albright and her legal representative have not yet determined the amount they will ask for – it will be set after the severity and permanency of Ms. Albright’s injuries have been more thoroughly investigated. But it’s expected that they will seek damages, along with all legal and court expenses.

The defendants haven’t responded to the plaintiff’s suit.

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

Early in December 2021 a jury awarded a couple $10 million in a case involving a hysterectomy that went badly wrong, according to a story in the New York Post, among other news sites.

In October 2018, Michele Nugent, 41, of New York, underwent the procedure at Richmond University Medical Center. After giving birth to four children via cesarean delivery, she had developed scarring and was suffering from excessive and painful uterine bleeding.

A few days after her hysterectomy, however, Ms. Nugent experienced what she described as the worst pain of her life, along with nausea, vomiting, and urinary leakage. She was brought to the hospital emergency department, where she was reportedly told by staff there that her symptoms were normal complications of her surgery and that the treating gynecologist, Eli Serur, MD, would soon be in touch.

Despite these reassurances, Ms. Nugent’s postsurgical issues continued to worsen over the next 10 days. Among other things, she lost almost complete control of her bladder, which required her to wear adult diapers. Still, her doctor’s office told her to put off visiting until her next scheduled appointment.

At that meeting, which took place 13 days after Ms. Nugent’s surgery, Dr. Serur diagnosed a urinary tract infection and placed her on antibiotics. He also encouraged her to return to work the following week.

Ms. Nugent’s problems persisted, however. At an office meeting that included 20 men, she suddenly lost complete control of her bladder, despite going regularly to the bathroom and wearing adult diapers. “Out of nowhere,” she testified at trial, “I urinated all over myself and had to leave.” The experience left her humiliated and embarrassed.

Several weeks later, Ms. Nugent consulted with a urologist, who soon repaired the cause of her urinary problems – a fistula between her vagina and bladder.

Though successful, the procedure still left Ms. Nugent with, what are for now at least, intractable symptoms. At night, she’s forced to make multiple trips to the bathroom, and sex with her husband has become all but impossible because of the pain it elicits.

In reaching its verdict, the jury of four women and two men faulted Dr. Serur for not only performing a faulty surgery but for failing to identify and correct his mistake. In so doing, it concluded, he had departed “from good and accepted medical practice.”

Jurors divided the $10 million judgment against him into two parts: $6.5 million for Ms. Nugent’s past and future suffering, and $3.5 million to her husband for his past and future loss of consortium – that is, his loss of intimacy with his wife.

As for the medical center, the Nugents agreed to dismiss it from the case prior to trial.
 

Physician accused of gross negligence finally surrenders his license

A California doctor under investigation multiple times during the past 2 decades has surrendered his medical license, as a story reported by Valley Public Radio indicates.

Since 1999, the Medical Board of California has opened three investigations against Bakersfield ob.gyn. Arthur Park, MD, each involving accusations of gross negligence “following the deaths of mothers and/or their babies during childbirth.” In 2000, and again in 2020, the board voted that Dr. Park should lose his license but then suspended its decision, which enabled Dr. Park to continue practicing under probation and on condition that he complete remedial education.

Early in 2021, however, the board filed yet another accusation against him, this one involving the 2019 death of Demi Dominguez and her newborn baby. According to the accusation, Ms. Dominguez died of preeclampsia because Dr. Park and a colleague failed to treat her high blood pressure prior to delivery. While doctors attempted to resuscitate her, Ms. Dominguez’s son was delivered by emergency cesarean but died only a few hours later. The board said that Dr. Park was “grossly negligent in his care and treatment” and that his actions constituted “an extreme departure from the applicable standard of care.”

Early in December 2021, even before the board and attorney general’s office had completed their investigations, Dr. Park agreed to surrender his medical license.

Patient advocates were pleased by the doctor’s decision but also disappointed that he’d no longer be compelled to stand before a judge, as he had been scheduled to do in connection with the Dominguez case.

A review of public records by Valley Public Radio indicates that – between the various board accusations against him and an additional nine lawsuits alleging malpractice and other issues – at least two mothers and five children have died while under Dr. Park’s care. Others whose delivery he oversaw claim their children were permanently injured during childbirth.

Although Dr. Park will be eligible to reapply for his license after 2 years, a representative of his medical office said Dr. Park had decided to retire from practicing medicine.
 

Delayed cancer diagnosis prompts med-mal suit

An Illinois woman who claims her doctor and his staff failed to follow up on her abnormal Pap smear has filed a malpractice suit against them and their medical group, reports a story in the Madison-St. Clair Record.

In early 2019, Lisa Albright visited the medical group after she had experienced pain during intercourse. A family nurse practitioner at the practice performed a Pap smear, and Ms. Albright was instructed to wait a few days and check her patient portal for the results. In her suit, Ms. Albright claims those results were abnormal. Despite this, neither the nurse practitioner nor anyone else at the practice scheduled a follow-up test or other diagnostic assessment.

Approximately 5 months later, Ms. Albright consulted a new physician, whose follow-up testing indicated that Ms. Albright had a cervical squamous cell carcinoma.

Ms. Albright’s suit alleges that the diagnostic delay has, among other things, caused her to undergo multiple surgical procedures and treatments, face a shorter life expectancy, and endure a loss in the quality of her life.

At press time, Ms. Albright and her legal representative have not yet determined the amount they will ask for – it will be set after the severity and permanency of Ms. Albright’s injuries have been more thoroughly investigated. But it’s expected that they will seek damages, along with all legal and court expenses.

The defendants haven’t responded to the plaintiff’s suit.

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

Postacute COVID syndrome (PACS), an ongoing inflammatory state following infection with SARS-CoV-2, is associated with greater risk of metabolic-associated fatty liver disease (MAFLD), according to an analysis of patients at a single clinic in Canada published in Open Forum Infectious Diseases.

MAFLD, also known as nonalcoholic fatty liver disease (NAFLD), is considered an indicator of general health and is in turn linked to greater risk of cardiovascular complications and mortality. It may be a multisystem disorder with various underlying causes.

Postacute COVID syndrome (PACS), an ongoing inflammatory state following infection with SARS-CoV-2, is associated with greater risk of metabolic-associated fatty liver disease (MAFLD), according to an analysis of patients at a single clinic in Canada published in Open Forum Infectious Diseases.

MAFLD, also known as nonalcoholic fatty liver disease (NAFLD), is considered an indicator of general health and is in turn linked to greater risk of cardiovascular complications and mortality. It may be a multisystem disorder with various underlying causes.

Postacute COVID syndrome (PACS), an ongoing inflammatory state following infection with SARS-CoV-2, is associated with greater risk of metabolic-associated fatty liver disease (MAFLD), according to an analysis of patients at a single clinic in Canada published in Open Forum Infectious Diseases.

MAFLD, also known as nonalcoholic fatty liver disease (NAFLD), is considered an indicator of general health and is in turn linked to greater risk of cardiovascular complications and mortality. It may be a multisystem disorder with various underlying causes.

Use of a novel oral Janus kinase 1 inhibitor known as INCB054707 by patients with moderate to severe hidradenitis suppurativa (HS) produced rapid and dose-dependent clinical responses, results from two small phase 2 studies showed.

“INCB054707 is an oral, small-molecule JAK1 inhibitor with approximately 52-fold greater selectivity for JAK1 versus JAK2,” researchers led by Afsaneh Alavi, MD, of the Mayo Clinic in Rochester, Minn., wrote in an article published recently in the British Journal of Dermatology. “Specifically targeting JAK1, a critical regulator of proinflammatory cytokine signaling implicated in several immune-related diseases, may reduce cytokine signaling involved in HS pathogenesis while limiting JAK2-mediated cytopenias.”

For the first study, 10 patients received 15 mg INCB054707 once daily for 8 weeks (NCT03569371). For the second study, 35 patients were randomized to 30, 60, or 90 mg INCB054707 once daily or placebo (3:1 within each cohort) for 8 weeks (NCT03607487). Eligibility criteria for both studies included patients with Hurley stage II/III HS who aged 18-75 years with lesions present in two or more anatomic locations, and a total abscess and inflammatory nodule count of three or more. The primary endpoint for both studies was safety and tolerability. Secondary endpoints included HS Clinical Response (HiSCR) and other efficacy measures.

The researchers reported that 30% of patients in study 1 and 42.3% of patients who received INCB054707 in study 2 experienced one or more treatment-emergent adverse event, most commonly upper respiratory tract infection. Among evaluable patients, 3 patients (42.9%) in study 1 and 17 patients in study 2 (65.4%) achieved HiSCR at week 8, compared with 57.1% of those in the placebo group. By dosing, 55.6% in the 30-mg group achieved HiSCR at week 8, compared with 55.6% in the 60-mg group and 87.5% in the 90-mg group.

“In conclusion, safety and efficacy findings from these two phase 2 studies establish proof of concept for the JAK1 inhibitor INCB054707 in the treatment of moderate to severe HS,” the authors wrote. “A phase 2, dose-ranging, placebo-controlled study exploring three dose levels and including approximately 200 patients is ongoing (NCT04476043) and expected to provide additional evidence of the safety and efficacy profile of INCB054707 in patients with HS.”

INCB054707 is being developed by Incyte Corporation. Dr. Alavi disclosed that she has received honoraria as a consultant or advisory board participant from AbbVie, Janssen, Novartis, Boehringer Ingelheim, InflaRX, and UCB, and received honoraria as an investigator for Boehringer Ingelheim and Processa.

Use of a novel oral Janus kinase 1 inhibitor known as INCB054707 by patients with moderate to severe hidradenitis suppurativa (HS) produced rapid and dose-dependent clinical responses, results from two small phase 2 studies showed.

“INCB054707 is an oral, small-molecule JAK1 inhibitor with approximately 52-fold greater selectivity for JAK1 versus JAK2,” researchers led by Afsaneh Alavi, MD, of the Mayo Clinic in Rochester, Minn., wrote in an article published recently in the British Journal of Dermatology. “Specifically targeting JAK1, a critical regulator of proinflammatory cytokine signaling implicated in several immune-related diseases, may reduce cytokine signaling involved in HS pathogenesis while limiting JAK2-mediated cytopenias.”

For the first study, 10 patients received 15 mg INCB054707 once daily for 8 weeks (NCT03569371). For the second study, 35 patients were randomized to 30, 60, or 90 mg INCB054707 once daily or placebo (3:1 within each cohort) for 8 weeks (NCT03607487). Eligibility criteria for both studies included patients with Hurley stage II/III HS who aged 18-75 years with lesions present in two or more anatomic locations, and a total abscess and inflammatory nodule count of three or more. The primary endpoint for both studies was safety and tolerability. Secondary endpoints included HS Clinical Response (HiSCR) and other efficacy measures.

The researchers reported that 30% of patients in study 1 and 42.3% of patients who received INCB054707 in study 2 experienced one or more treatment-emergent adverse event, most commonly upper respiratory tract infection. Among evaluable patients, 3 patients (42.9%) in study 1 and 17 patients in study 2 (65.4%) achieved HiSCR at week 8, compared with 57.1% of those in the placebo group. By dosing, 55.6% in the 30-mg group achieved HiSCR at week 8, compared with 55.6% in the 60-mg group and 87.5% in the 90-mg group.

“In conclusion, safety and efficacy findings from these two phase 2 studies establish proof of concept for the JAK1 inhibitor INCB054707 in the treatment of moderate to severe HS,” the authors wrote. “A phase 2, dose-ranging, placebo-controlled study exploring three dose levels and including approximately 200 patients is ongoing (NCT04476043) and expected to provide additional evidence of the safety and efficacy profile of INCB054707 in patients with HS.”

INCB054707 is being developed by Incyte Corporation. Dr. Alavi disclosed that she has received honoraria as a consultant or advisory board participant from AbbVie, Janssen, Novartis, Boehringer Ingelheim, InflaRX, and UCB, and received honoraria as an investigator for Boehringer Ingelheim and Processa.

Use of a novel oral Janus kinase 1 inhibitor known as INCB054707 by patients with moderate to severe hidradenitis suppurativa (HS) produced rapid and dose-dependent clinical responses, results from two small phase 2 studies showed.

“INCB054707 is an oral, small-molecule JAK1 inhibitor with approximately 52-fold greater selectivity for JAK1 versus JAK2,” researchers led by Afsaneh Alavi, MD, of the Mayo Clinic in Rochester, Minn., wrote in an article published recently in the British Journal of Dermatology. “Specifically targeting JAK1, a critical regulator of proinflammatory cytokine signaling implicated in several immune-related diseases, may reduce cytokine signaling involved in HS pathogenesis while limiting JAK2-mediated cytopenias.”

For the first study, 10 patients received 15 mg INCB054707 once daily for 8 weeks (NCT03569371). For the second study, 35 patients were randomized to 30, 60, or 90 mg INCB054707 once daily or placebo (3:1 within each cohort) for 8 weeks (NCT03607487). Eligibility criteria for both studies included patients with Hurley stage II/III HS who aged 18-75 years with lesions present in two or more anatomic locations, and a total abscess and inflammatory nodule count of three or more. The primary endpoint for both studies was safety and tolerability. Secondary endpoints included HS Clinical Response (HiSCR) and other efficacy measures.

The researchers reported that 30% of patients in study 1 and 42.3% of patients who received INCB054707 in study 2 experienced one or more treatment-emergent adverse event, most commonly upper respiratory tract infection. Among evaluable patients, 3 patients (42.9%) in study 1 and 17 patients in study 2 (65.4%) achieved HiSCR at week 8, compared with 57.1% of those in the placebo group. By dosing, 55.6% in the 30-mg group achieved HiSCR at week 8, compared with 55.6% in the 60-mg group and 87.5% in the 90-mg group.

“In conclusion, safety and efficacy findings from these two phase 2 studies establish proof of concept for the JAK1 inhibitor INCB054707 in the treatment of moderate to severe HS,” the authors wrote. “A phase 2, dose-ranging, placebo-controlled study exploring three dose levels and including approximately 200 patients is ongoing (NCT04476043) and expected to provide additional evidence of the safety and efficacy profile of INCB054707 in patients with HS.”

INCB054707 is being developed by Incyte Corporation. Dr. Alavi disclosed that she has received honoraria as a consultant or advisory board participant from AbbVie, Janssen, Novartis, Boehringer Ingelheim, InflaRX, and UCB, and received honoraria as an investigator for Boehringer Ingelheim and Processa.

More than a month after the launch of the new iPLEDGE Risk Evaluation and Mitigation Strategy (REMS) program, the operation still isn’t running smoothly for many dermatologists, pharmacists, and patients. That’s despite improvement attempts by the Food and Drug Administration, which mandates the program to prevent fetal exposure to the teratogenic effects of isotretinoin, and by the American Academy of Dermatology Association, whose members have repeatedly asked the FDA for meetings to discuss solutions. The AADA is the legislative and advocacy arm of AAD.

When the new program launched Dec. 13, 2021, the website crashed repeatedly, with physicians and patients complaining they got locked out or bounced off the platform when they tried to follow instructions to enter information. Hold times to talk to a live person stretched to hours.

The latest improvement attempt, announced Jan. 14 by the FDA, is a tool created by the Isotretinoin Products Manufacturers Group, the manufacturers responsible for the FDA-mandated REMS program. It is meant to allow prescribers and designees to send log-in links directly to patients’ email accounts through the iPLEDGE REMS portal, bypassing the troublesome call center.

And it’s not the answer, dermatologists said.

“The new tool does not solve issues such as prescribers or pharmacies not being able to access the site, unacceptably long call center wait times, inefficiencies caused by frequent attestation requirements for those who cannot become pregnant, patients becoming ‘locked out’ because they missed a window period through no fault of their own, among others,” said John Barbieri, MD, MBA, director of the Advanced Acne Therapeutics Clinic at Brigham and Women’s Hospital and instructor in dermatology at Harvard Medical School, both in Boston.

The day after the FDA update about the new tool, Klint Peebles, MD, a dermatologist at Kaiser Permanente in Washington, D.C., tweeted: “Lip service and empty words.” He noted that the situation has been “disastrous from the start” as the new platform launched.

Under the iPLEDGE program in place for the acne drug, physicians, patients, and pharmacies prescribing, using, or dispensing the drug must all be registered, with requirements that include the use of two forms of an effective contraceptive and regular pregnancy testing for patients who can become pregnant.

The aim of the new gender-neutral approach to the risk mitigation program is to make the experience more inclusive for transgender patients. The previous three risk categories (females of reproductive potential, females not of reproductive potential, and males) are now reduced to just two (those capable of getting pregnant and those not capable of getting pregnant).

The problem is  the execution of the new platform. The transition from the old website to the new was done quickly. By most accounts, the Dec. 13 rollout was chaotic, a failure, and disastrous, triggering numerous expressions of frustration on Twitter and other social media, with some calling for the program to be halted until the bugs could be worked out.

“While the new gender-neutral categories are a welcome improvement to the system, the new categorization approach was not the underlying reason for the new platform and its failed rollout, which was instead due to a change in vendor,” Dr. Barbieri told this news organization.

AADA: More recent efforts to improve the system

“We have a letter to the FDA asking for a stakeholders meeting to include us, the IPMG, and pharmacists because there are ongoing problems, though there have been some improvement in terms of certain elements,” Ilona Frieden, MD, chair of the AADA’s iPLEDGE Workgroup and professor of dermatology at the University of California, San Francisco, said in an interview shortly after the FDA posted the update on the new tool. “That said, there are many patients who have not gotten isotretinoin during the 1 month since the roll-out of the new platform.”

What still needs to be fixed? “We have ongoing concerns about the lack of transparency of the IPMG, about call center wait times, actual number of prescriptions on the hands of patients compared to the previous month, and those patients who can get pregnant who – despite complying with all of the REMS requirements – are being locked out because of the lack of timely attestation to their negative pregnancy status due to the website, not the patients themselves,” Dr. Frieden told this news organization.

“We are continuing to advocate to have decreased attestation requirements for individuals who cannot become pregnant – because this will improve the efficiency of the system for those patients for whom the REMS program goals are truly intended – those who can become pregnant, since the primary aim of the REMS program is to minimize fetal exposure.”

An AADA spokesperson said that the IPMG has invited the AADA to a joint stakeholders meeting on Jan. 26, along with representatives from the FDA and pharmacy industry.
 

Spotty progress

“The iPLEDGE situation is as frustrating as ever,” said Neil S. Goldberg, MD, a dermatologist in Westchester County, New York, after the FDA’s Jan. 14 update was released. “It’s like they never tested the new website before deploying it.”

Among the issues he has experienced in his practice, he said, is an instance in which iPLEDGE swapped the first names of a mother and daughter, so it was impossible to fill the prescription. “It happened twice in the same day,” Dr. Goldberg said. The patient had to call iPLEDGE to fix this, but the call center wasn’t taking calls.

In today’s technology environment, he said, it’s hard to believe that “we have to put up with this.”

Some have seen success. ‘’The tool is working fine on our end,” said Mitesh Patel, PharmD, pharmacy manager at Sunshine Pharmacy in White Plains, N.Y. However, he added that some doctors and patients are still having issues. He encourages dermatologists still having issues with the system to reach out to independent pharmacies that have processed iPLEDGE prescriptions and ‘’lean on them to assist.”

This news organization contacted CVS and Walgreens about how the system is working at their locations, but has not yet received a response. 

Dr. Goldberg, Dr. Frieden, Dr. Barbieri, and Dr. Peebles have disclosed no relevant financial relationships.

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

This story was updated on 1/24/22.

More than a month after the launch of the new iPLEDGE Risk Evaluation and Mitigation Strategy (REMS) program, the operation still isn’t running smoothly for many dermatologists, pharmacists, and patients. That’s despite improvement attempts by the Food and Drug Administration, which mandates the program to prevent fetal exposure to the teratogenic effects of isotretinoin, and by the American Academy of Dermatology Association, whose members have repeatedly asked the FDA for meetings to discuss solutions. The AADA is the legislative and advocacy arm of AAD.

When the new program launched Dec. 13, 2021, the website crashed repeatedly, with physicians and patients complaining they got locked out or bounced off the platform when they tried to follow instructions to enter information. Hold times to talk to a live person stretched to hours.

The latest improvement attempt, announced Jan. 14 by the FDA, is a tool created by the Isotretinoin Products Manufacturers Group, the manufacturers responsible for the FDA-mandated REMS program. It is meant to allow prescribers and designees to send log-in links directly to patients’ email accounts through the iPLEDGE REMS portal, bypassing the troublesome call center.

And it’s not the answer, dermatologists said.

“The new tool does not solve issues such as prescribers or pharmacies not being able to access the site, unacceptably long call center wait times, inefficiencies caused by frequent attestation requirements for those who cannot become pregnant, patients becoming ‘locked out’ because they missed a window period through no fault of their own, among others,” said John Barbieri, MD, MBA, director of the Advanced Acne Therapeutics Clinic at Brigham and Women’s Hospital and instructor in dermatology at Harvard Medical School, both in Boston.

The day after the FDA update about the new tool, Klint Peebles, MD, a dermatologist at Kaiser Permanente in Washington, D.C., tweeted: “Lip service and empty words.” He noted that the situation has been “disastrous from the start” as the new platform launched.

Under the iPLEDGE program in place for the acne drug, physicians, patients, and pharmacies prescribing, using, or dispensing the drug must all be registered, with requirements that include the use of two forms of an effective contraceptive and regular pregnancy testing for patients who can become pregnant.

The aim of the new gender-neutral approach to the risk mitigation program is to make the experience more inclusive for transgender patients. The previous three risk categories (females of reproductive potential, females not of reproductive potential, and males) are now reduced to just two (those capable of getting pregnant and those not capable of getting pregnant).

The problem is  the execution of the new platform. The transition from the old website to the new was done quickly. By most accounts, the Dec. 13 rollout was chaotic, a failure, and disastrous, triggering numerous expressions of frustration on Twitter and other social media, with some calling for the program to be halted until the bugs could be worked out.

“While the new gender-neutral categories are a welcome improvement to the system, the new categorization approach was not the underlying reason for the new platform and its failed rollout, which was instead due to a change in vendor,” Dr. Barbieri told this news organization.

AADA: More recent efforts to improve the system

“We have a letter to the FDA asking for a stakeholders meeting to include us, the IPMG, and pharmacists because there are ongoing problems, though there have been some improvement in terms of certain elements,” Ilona Frieden, MD, chair of the AADA’s iPLEDGE Workgroup and professor of dermatology at the University of California, San Francisco, said in an interview shortly after the FDA posted the update on the new tool. “That said, there are many patients who have not gotten isotretinoin during the 1 month since the roll-out of the new platform.”

What still needs to be fixed? “We have ongoing concerns about the lack of transparency of the IPMG, about call center wait times, actual number of prescriptions on the hands of patients compared to the previous month, and those patients who can get pregnant who – despite complying with all of the REMS requirements – are being locked out because of the lack of timely attestation to their negative pregnancy status due to the website, not the patients themselves,” Dr. Frieden told this news organization.

“We are continuing to advocate to have decreased attestation requirements for individuals who cannot become pregnant – because this will improve the efficiency of the system for those patients for whom the REMS program goals are truly intended – those who can become pregnant, since the primary aim of the REMS program is to minimize fetal exposure.”

An AADA spokesperson said that the IPMG has invited the AADA to a joint stakeholders meeting on Jan. 26, along with representatives from the FDA and pharmacy industry.
 

Spotty progress

“The iPLEDGE situation is as frustrating as ever,” said Neil S. Goldberg, MD, a dermatologist in Westchester County, New York, after the FDA’s Jan. 14 update was released. “It’s like they never tested the new website before deploying it.”

Among the issues he has experienced in his practice, he said, is an instance in which iPLEDGE swapped the first names of a mother and daughter, so it was impossible to fill the prescription. “It happened twice in the same day,” Dr. Goldberg said. The patient had to call iPLEDGE to fix this, but the call center wasn’t taking calls.

In today’s technology environment, he said, it’s hard to believe that “we have to put up with this.”

Some have seen success. ‘’The tool is working fine on our end,” said Mitesh Patel, PharmD, pharmacy manager at Sunshine Pharmacy in White Plains, N.Y. However, he added that some doctors and patients are still having issues. He encourages dermatologists still having issues with the system to reach out to independent pharmacies that have processed iPLEDGE prescriptions and ‘’lean on them to assist.”

This news organization contacted CVS and Walgreens about how the system is working at their locations, but has not yet received a response. 

Dr. Goldberg, Dr. Frieden, Dr. Barbieri, and Dr. Peebles have disclosed no relevant financial relationships.

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

This story was updated on 1/24/22.

More than a month after the launch of the new iPLEDGE Risk Evaluation and Mitigation Strategy (REMS) program, the operation still isn’t running smoothly for many dermatologists, pharmacists, and patients. That’s despite improvement attempts by the Food and Drug Administration, which mandates the program to prevent fetal exposure to the teratogenic effects of isotretinoin, and by the American Academy of Dermatology Association, whose members have repeatedly asked the FDA for meetings to discuss solutions. The AADA is the legislative and advocacy arm of AAD.

When the new program launched Dec. 13, 2021, the website crashed repeatedly, with physicians and patients complaining they got locked out or bounced off the platform when they tried to follow instructions to enter information. Hold times to talk to a live person stretched to hours.

The latest improvement attempt, announced Jan. 14 by the FDA, is a tool created by the Isotretinoin Products Manufacturers Group, the manufacturers responsible for the FDA-mandated REMS program. It is meant to allow prescribers and designees to send log-in links directly to patients’ email accounts through the iPLEDGE REMS portal, bypassing the troublesome call center.

And it’s not the answer, dermatologists said.

“The new tool does not solve issues such as prescribers or pharmacies not being able to access the site, unacceptably long call center wait times, inefficiencies caused by frequent attestation requirements for those who cannot become pregnant, patients becoming ‘locked out’ because they missed a window period through no fault of their own, among others,” said John Barbieri, MD, MBA, director of the Advanced Acne Therapeutics Clinic at Brigham and Women’s Hospital and instructor in dermatology at Harvard Medical School, both in Boston.

The day after the FDA update about the new tool, Klint Peebles, MD, a dermatologist at Kaiser Permanente in Washington, D.C., tweeted: “Lip service and empty words.” He noted that the situation has been “disastrous from the start” as the new platform launched.

Under the iPLEDGE program in place for the acne drug, physicians, patients, and pharmacies prescribing, using, or dispensing the drug must all be registered, with requirements that include the use of two forms of an effective contraceptive and regular pregnancy testing for patients who can become pregnant.

The aim of the new gender-neutral approach to the risk mitigation program is to make the experience more inclusive for transgender patients. The previous three risk categories (females of reproductive potential, females not of reproductive potential, and males) are now reduced to just two (those capable of getting pregnant and those not capable of getting pregnant).

The problem is  the execution of the new platform. The transition from the old website to the new was done quickly. By most accounts, the Dec. 13 rollout was chaotic, a failure, and disastrous, triggering numerous expressions of frustration on Twitter and other social media, with some calling for the program to be halted until the bugs could be worked out.

“While the new gender-neutral categories are a welcome improvement to the system, the new categorization approach was not the underlying reason for the new platform and its failed rollout, which was instead due to a change in vendor,” Dr. Barbieri told this news organization.

AADA: More recent efforts to improve the system

“We have a letter to the FDA asking for a stakeholders meeting to include us, the IPMG, and pharmacists because there are ongoing problems, though there have been some improvement in terms of certain elements,” Ilona Frieden, MD, chair of the AADA’s iPLEDGE Workgroup and professor of dermatology at the University of California, San Francisco, said in an interview shortly after the FDA posted the update on the new tool. “That said, there are many patients who have not gotten isotretinoin during the 1 month since the roll-out of the new platform.”

What still needs to be fixed? “We have ongoing concerns about the lack of transparency of the IPMG, about call center wait times, actual number of prescriptions on the hands of patients compared to the previous month, and those patients who can get pregnant who – despite complying with all of the REMS requirements – are being locked out because of the lack of timely attestation to their negative pregnancy status due to the website, not the patients themselves,” Dr. Frieden told this news organization.

“We are continuing to advocate to have decreased attestation requirements for individuals who cannot become pregnant – because this will improve the efficiency of the system for those patients for whom the REMS program goals are truly intended – those who can become pregnant, since the primary aim of the REMS program is to minimize fetal exposure.”

An AADA spokesperson said that the IPMG has invited the AADA to a joint stakeholders meeting on Jan. 26, along with representatives from the FDA and pharmacy industry.
 

Spotty progress

“The iPLEDGE situation is as frustrating as ever,” said Neil S. Goldberg, MD, a dermatologist in Westchester County, New York, after the FDA’s Jan. 14 update was released. “It’s like they never tested the new website before deploying it.”

Among the issues he has experienced in his practice, he said, is an instance in which iPLEDGE swapped the first names of a mother and daughter, so it was impossible to fill the prescription. “It happened twice in the same day,” Dr. Goldberg said. The patient had to call iPLEDGE to fix this, but the call center wasn’t taking calls.

In today’s technology environment, he said, it’s hard to believe that “we have to put up with this.”

Some have seen success. ‘’The tool is working fine on our end,” said Mitesh Patel, PharmD, pharmacy manager at Sunshine Pharmacy in White Plains, N.Y. However, he added that some doctors and patients are still having issues. He encourages dermatologists still having issues with the system to reach out to independent pharmacies that have processed iPLEDGE prescriptions and ‘’lean on them to assist.”

This news organization contacted CVS and Walgreens about how the system is working at their locations, but has not yet received a response. 

Dr. Goldberg, Dr. Frieden, Dr. Barbieri, and Dr. Peebles have disclosed no relevant financial relationships.

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

This story was updated on 1/24/22.

An intensive weight-loss intervention prior to conception had no effect on birth rates in women with obesity and unexplained infertility, compared with a standard weight-maintenance program, based on data from nearly 400 women.

Obese women experiencing infertility are often counseled to lose weight before attempting fertility treatments in order to improve outcomes based on epidemiologic evidence of an association between obesity and infertility, but data to support this advice are limited, wrote Richard S. Legro, MD, of Penn State University, Hershey, and colleagues.

The researchers proposed that a more intensive preconception weight loss intervention followed by infertility treatment would be more likely to yield a healthy live birth, compared with a standard weight maintenance intervention.

In an open-label study published in PLOS Medicine, the researchers randomized 379 women at nine academic centers to a standard lifestyle group that followed a weight-maintenance plan focused on physical activity, but not weight loss; or an intensive intervention of diet and medication with a target weight loss of 7%. Both interventions lasted for 16 weeks between July 2015 and July 2018. After the interventions, patients in both groups underwent standardized empiric fertility treatment with three cycles of ovarian stimulation and intrauterine insemination.

The primary outcome was a live birth at 37 weeks’ gestation or later, with no congenital abnormalities and a birth weight between 2,500 g and 4,000 g. Baseline characteristics including age, education level, race, and body mass index (BMI) were similar between the groups.

The incidence of healthy live births was not significantly different between the standard treatment and intensive treatment groups (15.2% vs. 12.2%; P = 0.40) by the final follow-up time of September 2019. However, women in the intensive group had significantly greater weight loss, compared with the standard group (–6.6% vs. –0.3%; P < .001). Women in the intensive group also showed improvements in metabolic health. Notably, the incidence of metabolic syndrome dropped from 53.6% to 49.4% in the standard group, compared with a decrease from 52.8% to 32.2% in the intensive group over the 16-week study period, the researchers wrote.

Gastrointestinal side effects were significantly more common in the intensive group, but these were consistent with documented side effects of the weight loss medication used (Orlistat).

First-trimester pregnancy loss was higher in the intensive group, compared with the standard group (33.3% vs. 23.7%), but the difference was not significant. Most pregnancy complications, including preterm labor, premature rupture of membranes, preeclampsia, and gestational diabetes had nonsignificant improvements in the intensive group, compared with the standard group. Similarly, nonsignificant improvements were noted in the intervention group for intrauterine growth restriction and admission to the neonatal ICU.

Limitations of the study included the relatively small number of pregnancies, which prevented assessment of rare complications in subgroups, and the challenge of matching control interventions, the researchers noted.

However, the results were strengthened by the focus on women with unexplained infertility, the inclusion of a comparison group, and the collection of data on complications after conception, they wrote.

Avenues for future research include interventions of different duration and intensity prior to conception, which may improve outcomes, the researchers said in their discussion of the findings. “A period of weight stabilization and maintenance after a weight-loss intervention prior to commencing infertility therapy is worth exploring,” they noted, but couples eager to conceive may be reluctant to wait for a weight-loss intervention, they added.

“Our findings directly impact current standards of clinical care, where women who are obese with unexplained infertility are to our knowledge routinely counseled to lose weight prior to initiation of infertility treatment,” they concluded.
 

Data may inform patient discussions

The current study is important because a large amount of previous research has shown an association between obesity and decreased fecundity in women and men, Mark P. Trolice, MD, of the University of Central Florida, Orlando, and director of the IVF Center in Winter Park, Fla., said in an interview.

According to the Centers for Disease Control and Prevention, the prevalence of obesity in the United States remains more than 40%, said Dr. Trolice. “Patients and physicians would benefit from clarity of obesity’s effect, if any, on reproduction,” he noted.

In contrast to the authors’ hypothesis, “the study did not find a difference in the live birth rate following up to three cycles of intrauterine insemination (IUI) between an intensive weight loss group [and] women who exercised without weight loss,” said Dr. Trolice. “Prior to this study, many reports suggested a decline in fertility with elevations in BMI, particularly during fertility treatment,” he added.

The take-home message from the current study is a that an elevated BMI, while possibly increasing the risks of metabolic disorders, did not appear to impact fecundity, he said.

The authors therefore concluded, “There is not strong evidence to recommend weight loss prior to conception in women who are obese with unexplained infertility,” Dr. Trolice said.

Regardless of the potential effect of preconception weight loss on fertility, barriers to starting a weight loss program include a woman’s eagerness to move forward with fertility treatments without waiting for weight loss, Dr. Trolice noted. “By the time a woman reaches an infertility specialist, she has been trying to conceive for at least 1 year,” he said. “At the initial consultation, these patients are anxious to undergo necessary additional diagnostic testing followed by treatment. Consequently, initiation of a weight-loss program is viewed as a delay toward the goal of family building,” he explained.

“More research is needed to demonstrate the safety of intensive weight loss preconception,” said Dr. Trolice. However, he said, “the issue of elevated BMI and increased risk of pregnancy complications remains, but this study provides important information for providers regarding counseling their patients desiring pregnancy.”

The study was supported by multiple grants from the National Institutes of Health through the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Center for Advancing Translational Sciences. Nutrisystem provided discounted coupons for food allotments in the standardized treatment group, and FitBit provided the study organizers with discounted Fitbits for activity monitoring. Lead author Dr. Legro disclosed consulting fees from InSupp, Ferring, Bayer, Abbvie and Fractyl, and research sponsorship from Guerbet and the National Institutes of Health. Dr. Trolice had no financial conflicts to disclose and serves on the Editorial Advisory Board of Ob.Gyn News.

An intensive weight-loss intervention prior to conception had no effect on birth rates in women with obesity and unexplained infertility, compared with a standard weight-maintenance program, based on data from nearly 400 women.

Obese women experiencing infertility are often counseled to lose weight before attempting fertility treatments in order to improve outcomes based on epidemiologic evidence of an association between obesity and infertility, but data to support this advice are limited, wrote Richard S. Legro, MD, of Penn State University, Hershey, and colleagues.

The researchers proposed that a more intensive preconception weight loss intervention followed by infertility treatment would be more likely to yield a healthy live birth, compared with a standard weight maintenance intervention.

In an open-label study published in PLOS Medicine, the researchers randomized 379 women at nine academic centers to a standard lifestyle group that followed a weight-maintenance plan focused on physical activity, but not weight loss; or an intensive intervention of diet and medication with a target weight loss of 7%. Both interventions lasted for 16 weeks between July 2015 and July 2018. After the interventions, patients in both groups underwent standardized empiric fertility treatment with three cycles of ovarian stimulation and intrauterine insemination.

The primary outcome was a live birth at 37 weeks’ gestation or later, with no congenital abnormalities and a birth weight between 2,500 g and 4,000 g. Baseline characteristics including age, education level, race, and body mass index (BMI) were similar between the groups.

The incidence of healthy live births was not significantly different between the standard treatment and intensive treatment groups (15.2% vs. 12.2%; P = 0.40) by the final follow-up time of September 2019. However, women in the intensive group had significantly greater weight loss, compared with the standard group (–6.6% vs. –0.3%; P < .001). Women in the intensive group also showed improvements in metabolic health. Notably, the incidence of metabolic syndrome dropped from 53.6% to 49.4% in the standard group, compared with a decrease from 52.8% to 32.2% in the intensive group over the 16-week study period, the researchers wrote.

Gastrointestinal side effects were significantly more common in the intensive group, but these were consistent with documented side effects of the weight loss medication used (Orlistat).

First-trimester pregnancy loss was higher in the intensive group, compared with the standard group (33.3% vs. 23.7%), but the difference was not significant. Most pregnancy complications, including preterm labor, premature rupture of membranes, preeclampsia, and gestational diabetes had nonsignificant improvements in the intensive group, compared with the standard group. Similarly, nonsignificant improvements were noted in the intervention group for intrauterine growth restriction and admission to the neonatal ICU.

Limitations of the study included the relatively small number of pregnancies, which prevented assessment of rare complications in subgroups, and the challenge of matching control interventions, the researchers noted.

However, the results were strengthened by the focus on women with unexplained infertility, the inclusion of a comparison group, and the collection of data on complications after conception, they wrote.

Avenues for future research include interventions of different duration and intensity prior to conception, which may improve outcomes, the researchers said in their discussion of the findings. “A period of weight stabilization and maintenance after a weight-loss intervention prior to commencing infertility therapy is worth exploring,” they noted, but couples eager to conceive may be reluctant to wait for a weight-loss intervention, they added.

“Our findings directly impact current standards of clinical care, where women who are obese with unexplained infertility are to our knowledge routinely counseled to lose weight prior to initiation of infertility treatment,” they concluded.
 

Data may inform patient discussions

The current study is important because a large amount of previous research has shown an association between obesity and decreased fecundity in women and men, Mark P. Trolice, MD, of the University of Central Florida, Orlando, and director of the IVF Center in Winter Park, Fla., said in an interview.

According to the Centers for Disease Control and Prevention, the prevalence of obesity in the United States remains more than 40%, said Dr. Trolice. “Patients and physicians would benefit from clarity of obesity’s effect, if any, on reproduction,” he noted.

In contrast to the authors’ hypothesis, “the study did not find a difference in the live birth rate following up to three cycles of intrauterine insemination (IUI) between an intensive weight loss group [and] women who exercised without weight loss,” said Dr. Trolice. “Prior to this study, many reports suggested a decline in fertility with elevations in BMI, particularly during fertility treatment,” he added.

The take-home message from the current study is a that an elevated BMI, while possibly increasing the risks of metabolic disorders, did not appear to impact fecundity, he said.

The authors therefore concluded, “There is not strong evidence to recommend weight loss prior to conception in women who are obese with unexplained infertility,” Dr. Trolice said.

Regardless of the potential effect of preconception weight loss on fertility, barriers to starting a weight loss program include a woman’s eagerness to move forward with fertility treatments without waiting for weight loss, Dr. Trolice noted. “By the time a woman reaches an infertility specialist, she has been trying to conceive for at least 1 year,” he said. “At the initial consultation, these patients are anxious to undergo necessary additional diagnostic testing followed by treatment. Consequently, initiation of a weight-loss program is viewed as a delay toward the goal of family building,” he explained.

“More research is needed to demonstrate the safety of intensive weight loss preconception,” said Dr. Trolice. However, he said, “the issue of elevated BMI and increased risk of pregnancy complications remains, but this study provides important information for providers regarding counseling their patients desiring pregnancy.”

The study was supported by multiple grants from the National Institutes of Health through the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Center for Advancing Translational Sciences. Nutrisystem provided discounted coupons for food allotments in the standardized treatment group, and FitBit provided the study organizers with discounted Fitbits for activity monitoring. Lead author Dr. Legro disclosed consulting fees from InSupp, Ferring, Bayer, Abbvie and Fractyl, and research sponsorship from Guerbet and the National Institutes of Health. Dr. Trolice had no financial conflicts to disclose and serves on the Editorial Advisory Board of Ob.Gyn News.

An intensive weight-loss intervention prior to conception had no effect on birth rates in women with obesity and unexplained infertility, compared with a standard weight-maintenance program, based on data from nearly 400 women.

Obese women experiencing infertility are often counseled to lose weight before attempting fertility treatments in order to improve outcomes based on epidemiologic evidence of an association between obesity and infertility, but data to support this advice are limited, wrote Richard S. Legro, MD, of Penn State University, Hershey, and colleagues.

The researchers proposed that a more intensive preconception weight loss intervention followed by infertility treatment would be more likely to yield a healthy live birth, compared with a standard weight maintenance intervention.

In an open-label study published in PLOS Medicine, the researchers randomized 379 women at nine academic centers to a standard lifestyle group that followed a weight-maintenance plan focused on physical activity, but not weight loss; or an intensive intervention of diet and medication with a target weight loss of 7%. Both interventions lasted for 16 weeks between July 2015 and July 2018. After the interventions, patients in both groups underwent standardized empiric fertility treatment with three cycles of ovarian stimulation and intrauterine insemination.

The primary outcome was a live birth at 37 weeks’ gestation or later, with no congenital abnormalities and a birth weight between 2,500 g and 4,000 g. Baseline characteristics including age, education level, race, and body mass index (BMI) were similar between the groups.

The incidence of healthy live births was not significantly different between the standard treatment and intensive treatment groups (15.2% vs. 12.2%; P = 0.40) by the final follow-up time of September 2019. However, women in the intensive group had significantly greater weight loss, compared with the standard group (–6.6% vs. –0.3%; P < .001). Women in the intensive group also showed improvements in metabolic health. Notably, the incidence of metabolic syndrome dropped from 53.6% to 49.4% in the standard group, compared with a decrease from 52.8% to 32.2% in the intensive group over the 16-week study period, the researchers wrote.

Gastrointestinal side effects were significantly more common in the intensive group, but these were consistent with documented side effects of the weight loss medication used (Orlistat).

First-trimester pregnancy loss was higher in the intensive group, compared with the standard group (33.3% vs. 23.7%), but the difference was not significant. Most pregnancy complications, including preterm labor, premature rupture of membranes, preeclampsia, and gestational diabetes had nonsignificant improvements in the intensive group, compared with the standard group. Similarly, nonsignificant improvements were noted in the intervention group for intrauterine growth restriction and admission to the neonatal ICU.

Limitations of the study included the relatively small number of pregnancies, which prevented assessment of rare complications in subgroups, and the challenge of matching control interventions, the researchers noted.

However, the results were strengthened by the focus on women with unexplained infertility, the inclusion of a comparison group, and the collection of data on complications after conception, they wrote.

Avenues for future research include interventions of different duration and intensity prior to conception, which may improve outcomes, the researchers said in their discussion of the findings. “A period of weight stabilization and maintenance after a weight-loss intervention prior to commencing infertility therapy is worth exploring,” they noted, but couples eager to conceive may be reluctant to wait for a weight-loss intervention, they added.

“Our findings directly impact current standards of clinical care, where women who are obese with unexplained infertility are to our knowledge routinely counseled to lose weight prior to initiation of infertility treatment,” they concluded.
 

Data may inform patient discussions

The current study is important because a large amount of previous research has shown an association between obesity and decreased fecundity in women and men, Mark P. Trolice, MD, of the University of Central Florida, Orlando, and director of the IVF Center in Winter Park, Fla., said in an interview.

According to the Centers for Disease Control and Prevention, the prevalence of obesity in the United States remains more than 40%, said Dr. Trolice. “Patients and physicians would benefit from clarity of obesity’s effect, if any, on reproduction,” he noted.

In contrast to the authors’ hypothesis, “the study did not find a difference in the live birth rate following up to three cycles of intrauterine insemination (IUI) between an intensive weight loss group [and] women who exercised without weight loss,” said Dr. Trolice. “Prior to this study, many reports suggested a decline in fertility with elevations in BMI, particularly during fertility treatment,” he added.

The take-home message from the current study is a that an elevated BMI, while possibly increasing the risks of metabolic disorders, did not appear to impact fecundity, he said.

The authors therefore concluded, “There is not strong evidence to recommend weight loss prior to conception in women who are obese with unexplained infertility,” Dr. Trolice said.

Regardless of the potential effect of preconception weight loss on fertility, barriers to starting a weight loss program include a woman’s eagerness to move forward with fertility treatments without waiting for weight loss, Dr. Trolice noted. “By the time a woman reaches an infertility specialist, she has been trying to conceive for at least 1 year,” he said. “At the initial consultation, these patients are anxious to undergo necessary additional diagnostic testing followed by treatment. Consequently, initiation of a weight-loss program is viewed as a delay toward the goal of family building,” he explained.

“More research is needed to demonstrate the safety of intensive weight loss preconception,” said Dr. Trolice. However, he said, “the issue of elevated BMI and increased risk of pregnancy complications remains, but this study provides important information for providers regarding counseling their patients desiring pregnancy.”

The study was supported by multiple grants from the National Institutes of Health through the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Center for Advancing Translational Sciences. Nutrisystem provided discounted coupons for food allotments in the standardized treatment group, and FitBit provided the study organizers with discounted Fitbits for activity monitoring. Lead author Dr. Legro disclosed consulting fees from InSupp, Ferring, Bayer, Abbvie and Fractyl, and research sponsorship from Guerbet and the National Institutes of Health. Dr. Trolice had no financial conflicts to disclose and serves on the Editorial Advisory Board of Ob.Gyn News.

“Turns out smarter kids are made, not born.” The headline of the article leapt off the computer screen. Although I realize that it has limits when it comes to dissuading vaccine refusers, I believe that education is a critical element in the success of individuals and the societies they inhabit. However, I must admit to a bias based on my observations that, in general, cognitive skill is inherited. This is an opinion I suspect I share with most folks. You can understand why the article I discovered describing a recent study by several Harvard-based researchers caught my attention.

The study involved 33 mothers and their 1-year-old children. The researchers found that infants whose mothers were stressed and had a “fixed mindset” had lower brain activity than the infants of stressed mothers who held a “growth mindset.” You may be on top of the education literature but I had to do some heavy Googling to learn what was up with growth and fixed mindsets. Was this just a new riff on the whole mindfulness thing?

I quickly learned that in 2006 Carol Dweck, PhD, a psychologist now at Stanford, published a book titled “Mindset” (New York: Penguin Random House) in which she described individuals with a “fixed mindset” who believe that their personality or intelligence will not change over time. On the other hand, individuals with a “growth mindset” view their intelligence and personality as malleable. Her observations have spread across the education and self-help literature like a wildfire that has somehow been roaring along under my radar. I guess I have noticed a subtle change in emphasis when I hear some parents and educators praising a child’s effort in situations in which I might have expected them to say, “You’re so smart.” But, in general I have been clueless.

My initial impression was that this mindset stuff was just coining new buzz words to differentiate optimists from pessimists. But, here I am again revealing a fixed mindset bias. I probably should have said that someone demonstrating a growth mindset approach is “exercising optimism” instead of implying that they were simply born with a sunny disposition.

The growth mindset revolution has not been without skeptics and critics, which is not surprising because educators have a history of jumping on bandwagons before all the wheels have been completely tightened. However, the mindset approach does have some merit, especially for individuals in the center of the bell-shaped curve. We all know of individuals who have failed to meet or have exceeded what would seem to be rational expectations. It is likely that the degree to which a growth mindset approach was applied may be the explanation.

Which brings me to the question of whether we as pediatricians should be more careful of how we choose our words when talking to patients and parents. If the results of the study that alerted me to the growth mindset are reproducible, maybe we should be spending more time with new parents (all of whom are stressed by definition), helping them discover ways in which they can improve the situation they find themselves in by praising them for their efforts at parenting.

Should we be modeling growth mindset language by using it when we interact with our patients? For example, not just complimenting a child on the acquisition of a skill but adding that we were even more impressed by the effort required to acquire it. When we hear a parent clearly expressing a fixed mindset in describing their child should we correct them on the spot or make an appointment to discuss how adopting a growth mindset might help their child meet or exceed his or her potential?

Most smart children may be born that way, but there are always opportunities for improvement, and our patients and their parents need to believe that.
 

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 [email protected].

“Turns out smarter kids are made, not born.” The headline of the article leapt off the computer screen. Although I realize that it has limits when it comes to dissuading vaccine refusers, I believe that education is a critical element in the success of individuals and the societies they inhabit. However, I must admit to a bias based on my observations that, in general, cognitive skill is inherited. This is an opinion I suspect I share with most folks. You can understand why the article I discovered describing a recent study by several Harvard-based researchers caught my attention.

The study involved 33 mothers and their 1-year-old children. The researchers found that infants whose mothers were stressed and had a “fixed mindset” had lower brain activity than the infants of stressed mothers who held a “growth mindset.” You may be on top of the education literature but I had to do some heavy Googling to learn what was up with growth and fixed mindsets. Was this just a new riff on the whole mindfulness thing?

I quickly learned that in 2006 Carol Dweck, PhD, a psychologist now at Stanford, published a book titled “Mindset” (New York: Penguin Random House) in which she described individuals with a “fixed mindset” who believe that their personality or intelligence will not change over time. On the other hand, individuals with a “growth mindset” view their intelligence and personality as malleable. Her observations have spread across the education and self-help literature like a wildfire that has somehow been roaring along under my radar. I guess I have noticed a subtle change in emphasis when I hear some parents and educators praising a child’s effort in situations in which I might have expected them to say, “You’re so smart.” But, in general I have been clueless.

My initial impression was that this mindset stuff was just coining new buzz words to differentiate optimists from pessimists. But, here I am again revealing a fixed mindset bias. I probably should have said that someone demonstrating a growth mindset approach is “exercising optimism” instead of implying that they were simply born with a sunny disposition.

The growth mindset revolution has not been without skeptics and critics, which is not surprising because educators have a history of jumping on bandwagons before all the wheels have been completely tightened. However, the mindset approach does have some merit, especially for individuals in the center of the bell-shaped curve. We all know of individuals who have failed to meet or have exceeded what would seem to be rational expectations. It is likely that the degree to which a growth mindset approach was applied may be the explanation.

Which brings me to the question of whether we as pediatricians should be more careful of how we choose our words when talking to patients and parents. If the results of the study that alerted me to the growth mindset are reproducible, maybe we should be spending more time with new parents (all of whom are stressed by definition), helping them discover ways in which they can improve the situation they find themselves in by praising them for their efforts at parenting.

Should we be modeling growth mindset language by using it when we interact with our patients? For example, not just complimenting a child on the acquisition of a skill but adding that we were even more impressed by the effort required to acquire it. When we hear a parent clearly expressing a fixed mindset in describing their child should we correct them on the spot or make an appointment to discuss how adopting a growth mindset might help their child meet or exceed his or her potential?

Most smart children may be born that way, but there are always opportunities for improvement, and our patients and their parents need to believe that.
 

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 [email protected].

“Turns out smarter kids are made, not born.” The headline of the article leapt off the computer screen. Although I realize that it has limits when it comes to dissuading vaccine refusers, I believe that education is a critical element in the success of individuals and the societies they inhabit. However, I must admit to a bias based on my observations that, in general, cognitive skill is inherited. This is an opinion I suspect I share with most folks. You can understand why the article I discovered describing a recent study by several Harvard-based researchers caught my attention.

The study involved 33 mothers and their 1-year-old children. The researchers found that infants whose mothers were stressed and had a “fixed mindset” had lower brain activity than the infants of stressed mothers who held a “growth mindset.” You may be on top of the education literature but I had to do some heavy Googling to learn what was up with growth and fixed mindsets. Was this just a new riff on the whole mindfulness thing?

I quickly learned that in 2006 Carol Dweck, PhD, a psychologist now at Stanford, published a book titled “Mindset” (New York: Penguin Random House) in which she described individuals with a “fixed mindset” who believe that their personality or intelligence will not change over time. On the other hand, individuals with a “growth mindset” view their intelligence and personality as malleable. Her observations have spread across the education and self-help literature like a wildfire that has somehow been roaring along under my radar. I guess I have noticed a subtle change in emphasis when I hear some parents and educators praising a child’s effort in situations in which I might have expected them to say, “You’re so smart.” But, in general I have been clueless.

My initial impression was that this mindset stuff was just coining new buzz words to differentiate optimists from pessimists. But, here I am again revealing a fixed mindset bias. I probably should have said that someone demonstrating a growth mindset approach is “exercising optimism” instead of implying that they were simply born with a sunny disposition.

The growth mindset revolution has not been without skeptics and critics, which is not surprising because educators have a history of jumping on bandwagons before all the wheels have been completely tightened. However, the mindset approach does have some merit, especially for individuals in the center of the bell-shaped curve. We all know of individuals who have failed to meet or have exceeded what would seem to be rational expectations. It is likely that the degree to which a growth mindset approach was applied may be the explanation.

Which brings me to the question of whether we as pediatricians should be more careful of how we choose our words when talking to patients and parents. If the results of the study that alerted me to the growth mindset are reproducible, maybe we should be spending more time with new parents (all of whom are stressed by definition), helping them discover ways in which they can improve the situation they find themselves in by praising them for their efforts at parenting.

Should we be modeling growth mindset language by using it when we interact with our patients? For example, not just complimenting a child on the acquisition of a skill but adding that we were even more impressed by the effort required to acquire it. When we hear a parent clearly expressing a fixed mindset in describing their child should we correct them on the spot or make an appointment to discuss how adopting a growth mindset might help their child meet or exceed his or her potential?

Most smart children may be born that way, but there are always opportunities for improvement, and our patients and their parents need to believe that.
 

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 [email protected].

Two close colleagues at New York’s Memorial Sloan Kettering Cancer Center, world leaders in hematopoietic stem cell transplantation (HSCT) who were both promoted days after COVID-19 locked down the city in 2020, were too busy battling the pandemic’s impact on patients in the summer of 2021 to notice their latest shared career milestone.

On July 29, 2021, Sergio Giralt, MD, deputy division head of the division of hematologic malignancies and Miguel-Angel Perales, MD, chief of the adult bone marrow transplant service at MSKCC, published their 100th peer-reviewed paper as coauthors. Listing hundreds of such articles on a CV is standard for top-tier physicians, but the pair had gone one better: 100 publications written together in 10 years.

Their centenary article hit scientific newsstands almost exactly a decade after their first joint paper, which appeared in September 2011, not long after they met.

Born in Cuba, Dr. Giralt grew up in Venezuela. From the age of 14, he knew that medicine was his path, and in 1984 he earned a medical degree from the Universidad Central de Venezuela, Caracas. Next came a research position at Harvard Medical School, a residency at the Good Samaritan Hospital, Cincinnati, and a fellowship at the University of Texas MD Anderson Cancer Center, Houston. Dr. Giralt arrived at MSKCC in 2010 as the new chief of the adult bone marrow transplant service. There he was introduced to a new colleague, Dr. Perales. They soon learned that in addition to expertise in hematology, they had second language in common: Spanish.

Dr. Giralt said: “We both have a Spanish background and in a certain sense, there was an affinity there. ... We both have shared experiences.”

Dr. Perales was brought up in Belgium, a European nation with three official languages: French, Dutch, and German. He speaks five tongues in all and learned Spanish from his father, who came from Spain.

Courtesy MSKCC

Fluency in Spanish enables both physicians to take care of the many New Yorkers who are more comfortable in that language – especially when navigating cancer treatment. However, both Dr. Giralt and Dr. Perales said that a second language is more than a professional tool. They described the enjoyable change of persona that happens when they switch to Spanish.

“People who are multilingual have different roles [as much as] different languages,” said Dr. Perales. “When I’m in Spanish, part of my brain is [thinking back to] summer vacations and hanging out with my cousins.”

When it comes to clinical science, however, English is the language of choice.
 

Global leaders in HSCT

Dr. Giralt and Dr. Perales are known worldwide in the field of allogeneic HSCT, a potentially curative treatment for an elongating list of both malignant and nonmalignant diseases.

In 1973, MSKCC conducted the first bone-marrow transplant from an unrelated donor. Fifty years on, medical oncologists in the United States conduct approximately 8,500 allogeneic transplants each year, 72% to treat acute leukemias or myelodysplastic syndrome (MDS).

However, stripping the immune system with intensive chemotherapy ‘conditioning,’ then rebuilding it with non-diseased donor hematopoietic cells is a hazardous undertaking. Older patients are less likely to survive the intensive conditioning, so historically have missed out. Also, even with a good human leukocyte antigen (HLA) match, the recipient needs often brutal immunosuppression.

Since Dr. Giralt and Dr. Perales began their partnership in 2010, the goals of their work have not changed: to develop safer, lower-intensity transplantation suitable for older, more vulnerable patients and reduce fearsome posttransplant sequelae such as graft-versus-host disease (GVHD).

Dr. Giralt’s publication list spans more than 600 peer-reviewed papers, articles and book chapters, almost exclusively on HSCT. Dr. Perales has more than 300 publication credits on the topic.

The two paired up on their first paper just months after Dr. Giralt arrived at MSKCC. That article, published in Biology of Blood and Marrow Transplantation, compared umbilical cord blood for HSCT with donor blood in 367 people with a variety of hematologic malignancies, including acute and chronic leukemias, MDS, and lymphoma.

Courtesy MSKCC

The MSKCC team found that transplant-related mortality in the first 180 days was higher for the cord blood (21%), but thereafter mortality and relapse were much lower than for donated blood, with the result that 2-year progression-free survival of 55% was similar. Dr. Perales, Dr. Giralt and their coauthors concluded that the data provided “strong support” for further work on cord blood as an alternative stem-cell source.

During their first decade of collaboration, Dr. Giralt and Dr. Perales worked on any promising avenue that could improve outcomes and the experience of HSCT recipients, including reduced-intensity conditioning regimens to allow older adults to benefit from curative HSCT and donor T-cell depletion by CD34 selection, to reduce graft-versus-host disease (GVHD).

The CD34 protein is typically found on the surface of early stage and highly active stem cell types. Selecting these cell types using a range of techniques can eliminate many other potentially interfering or inactive cells. This enriches the transplant population with the most effective cells and can lower the risk of GVHD.

The 100th paper on which Dr. Giralt and Dr. Perales were coauthors was published in Blood Advances on July 27, 2021. The retrospective study examined the fate of 58 MSKCC patients with a rare form of chronic lymphocytic leukemia, CLL with Richter’s transformation (CLL-RT). It was the largest such study to date of this rare disease.
M.D. Anderson Cancer Center had shown in 2006 that, despite chemotherapy, overall survival in patients with CLL-RT was approximately 8 months. HSCT improved survival dramatically (75% at 3 years; n = 7). However, with the advent of novel targeted drugs for CLL such as ibrutinib (Imbruvica), venetoclax (Venclexta), or idelalisib (Zydelig), the MSKCC team asked themselves: What was the role of reduced-intensive conditioning HSCT? Was it even safe? Among other findings, Dr. Giralt and Dr. Perales’ 100th paper showed that reduced-intensity HSCT remained a viable alternative after a CLL-RT patient progressed on a novel agent.

Impact of the pandemic

When COVID-19 hit, the team lost many research staff and developed a huge backlog, said Dr. Giralt. He and Dr. Perales realized that they needed to be “thoughtful and careful” about which studies to continue. “For example, the CD-34 selection trials we did not close because these are our workhorse trials,” Dr. Giralt said. “We have people we need to treat, and some of the patients that we need to treat can only be treated on trial.”

The team was also able to pivot some of their work into COVID 19 itself, and they collected crucial information on HSCT in recovered COVID-19 patients, as an example.

“We were living through a critical time, but that doesn’t mean we [aren’t] obligated to continue our mission, our research mission,” said Dr. Giralt. “It really is team science. The way we look at it ... there’s a common thread: We both like to do allogeneic transplant, and we both believe in trying to make CD-34 selection better. So we’re both very much [working on] how can we improve what we call ‘the Memorial way’ of doing transplants. Where we separate is, Miguel does primarily lymphoma. He doesn’t do myeloma [like me]. So in those two areas, we’re helping develop the junior faculty in a different way.”
 

Something more in common

Right from the start, Dr. Perales and Dr. Giralt also shared a commitment to mentoring. Since 2010, Dr. Perales has mentored 22 up-and-coming junior faculty, including 10 from Europe (8 from Spain) and 2 from Latin America.

“[It makes] the research enterprise much more productive but [these young scientists] really increase the visibility of the program,” said Dr. Giralt.

He cited Dr. Perales’ track record of mentoring as one of the reasons for his promotion to chief of the adult bone marrow transplant service. In March 2020, Dr. Perales seamlessly stepped into Dr. Giralt’s shoes, while Dr. Giralt moved on to his present role as deputy division head of the division of hematologic malignancies.

Dr. Perales said: “The key aspect [of these promotions] is the fantastic working relationship that we’ve had over the years. ... I consider Sergio my mentor, but also a good friend and colleague. And so I think it’s this ability that we’ve had to work together and that relationship of trust, which has been key.”

“Sergio is somebody who lifts people up,” Dr. Perales added. “Many people will tell you that Sergio has helped them in their career. ... And I think that’s a lesson I’ve learned from him: training the next generation. And [that’s] not just in the U.S., but outside. I think that’s a key role that we have. And our responsibility.”

Asked to comment on their 100th-paper milestone, Dr. Perales firmly turned the spotlight from himself and Dr. Giralt to the junior investigators who have passed through the doors of the bone-marrow transplant program: “This body of work represents not just our collaboration but also the many contributions of our team at MSK ... and beyond MSK.”

This article was updated 1/26/22.

Two close colleagues at New York’s Memorial Sloan Kettering Cancer Center, world leaders in hematopoietic stem cell transplantation (HSCT) who were both promoted days after COVID-19 locked down the city in 2020, were too busy battling the pandemic’s impact on patients in the summer of 2021 to notice their latest shared career milestone.

On July 29, 2021, Sergio Giralt, MD, deputy division head of the division of hematologic malignancies and Miguel-Angel Perales, MD, chief of the adult bone marrow transplant service at MSKCC, published their 100th peer-reviewed paper as coauthors. Listing hundreds of such articles on a CV is standard for top-tier physicians, but the pair had gone one better: 100 publications written together in 10 years.

Their centenary article hit scientific newsstands almost exactly a decade after their first joint paper, which appeared in September 2011, not long after they met.

Born in Cuba, Dr. Giralt grew up in Venezuela. From the age of 14, he knew that medicine was his path, and in 1984 he earned a medical degree from the Universidad Central de Venezuela, Caracas. Next came a research position at Harvard Medical School, a residency at the Good Samaritan Hospital, Cincinnati, and a fellowship at the University of Texas MD Anderson Cancer Center, Houston. Dr. Giralt arrived at MSKCC in 2010 as the new chief of the adult bone marrow transplant service. There he was introduced to a new colleague, Dr. Perales. They soon learned that in addition to expertise in hematology, they had second language in common: Spanish.

Dr. Giralt said: “We both have a Spanish background and in a certain sense, there was an affinity there. ... We both have shared experiences.”

Dr. Perales was brought up in Belgium, a European nation with three official languages: French, Dutch, and German. He speaks five tongues in all and learned Spanish from his father, who came from Spain.

Courtesy MSKCC

Fluency in Spanish enables both physicians to take care of the many New Yorkers who are more comfortable in that language – especially when navigating cancer treatment. However, both Dr. Giralt and Dr. Perales said that a second language is more than a professional tool. They described the enjoyable change of persona that happens when they switch to Spanish.

“People who are multilingual have different roles [as much as] different languages,” said Dr. Perales. “When I’m in Spanish, part of my brain is [thinking back to] summer vacations and hanging out with my cousins.”

When it comes to clinical science, however, English is the language of choice.
 

Global leaders in HSCT

Dr. Giralt and Dr. Perales are known worldwide in the field of allogeneic HSCT, a potentially curative treatment for an elongating list of both malignant and nonmalignant diseases.

In 1973, MSKCC conducted the first bone-marrow transplant from an unrelated donor. Fifty years on, medical oncologists in the United States conduct approximately 8,500 allogeneic transplants each year, 72% to treat acute leukemias or myelodysplastic syndrome (MDS).

However, stripping the immune system with intensive chemotherapy ‘conditioning,’ then rebuilding it with non-diseased donor hematopoietic cells is a hazardous undertaking. Older patients are less likely to survive the intensive conditioning, so historically have missed out. Also, even with a good human leukocyte antigen (HLA) match, the recipient needs often brutal immunosuppression.

Since Dr. Giralt and Dr. Perales began their partnership in 2010, the goals of their work have not changed: to develop safer, lower-intensity transplantation suitable for older, more vulnerable patients and reduce fearsome posttransplant sequelae such as graft-versus-host disease (GVHD).

Dr. Giralt’s publication list spans more than 600 peer-reviewed papers, articles and book chapters, almost exclusively on HSCT. Dr. Perales has more than 300 publication credits on the topic.

The two paired up on their first paper just months after Dr. Giralt arrived at MSKCC. That article, published in Biology of Blood and Marrow Transplantation, compared umbilical cord blood for HSCT with donor blood in 367 people with a variety of hematologic malignancies, including acute and chronic leukemias, MDS, and lymphoma.

Courtesy MSKCC

The MSKCC team found that transplant-related mortality in the first 180 days was higher for the cord blood (21%), but thereafter mortality and relapse were much lower than for donated blood, with the result that 2-year progression-free survival of 55% was similar. Dr. Perales, Dr. Giralt and their coauthors concluded that the data provided “strong support” for further work on cord blood as an alternative stem-cell source.

During their first decade of collaboration, Dr. Giralt and Dr. Perales worked on any promising avenue that could improve outcomes and the experience of HSCT recipients, including reduced-intensity conditioning regimens to allow older adults to benefit from curative HSCT and donor T-cell depletion by CD34 selection, to reduce graft-versus-host disease (GVHD).

The CD34 protein is typically found on the surface of early stage and highly active stem cell types. Selecting these cell types using a range of techniques can eliminate many other potentially interfering or inactive cells. This enriches the transplant population with the most effective cells and can lower the risk of GVHD.

The 100th paper on which Dr. Giralt and Dr. Perales were coauthors was published in Blood Advances on July 27, 2021. The retrospective study examined the fate of 58 MSKCC patients with a rare form of chronic lymphocytic leukemia, CLL with Richter’s transformation (CLL-RT). It was the largest such study to date of this rare disease.
M.D. Anderson Cancer Center had shown in 2006 that, despite chemotherapy, overall survival in patients with CLL-RT was approximately 8 months. HSCT improved survival dramatically (75% at 3 years; n = 7). However, with the advent of novel targeted drugs for CLL such as ibrutinib (Imbruvica), venetoclax (Venclexta), or idelalisib (Zydelig), the MSKCC team asked themselves: What was the role of reduced-intensive conditioning HSCT? Was it even safe? Among other findings, Dr. Giralt and Dr. Perales’ 100th paper showed that reduced-intensity HSCT remained a viable alternative after a CLL-RT patient progressed on a novel agent.

Impact of the pandemic

When COVID-19 hit, the team lost many research staff and developed a huge backlog, said Dr. Giralt. He and Dr. Perales realized that they needed to be “thoughtful and careful” about which studies to continue. “For example, the CD-34 selection trials we did not close because these are our workhorse trials,” Dr. Giralt said. “We have people we need to treat, and some of the patients that we need to treat can only be treated on trial.”

The team was also able to pivot some of their work into COVID 19 itself, and they collected crucial information on HSCT in recovered COVID-19 patients, as an example.

“We were living through a critical time, but that doesn’t mean we [aren’t] obligated to continue our mission, our research mission,” said Dr. Giralt. “It really is team science. The way we look at it ... there’s a common thread: We both like to do allogeneic transplant, and we both believe in trying to make CD-34 selection better. So we’re both very much [working on] how can we improve what we call ‘the Memorial way’ of doing transplants. Where we separate is, Miguel does primarily lymphoma. He doesn’t do myeloma [like me]. So in those two areas, we’re helping develop the junior faculty in a different way.”
 

Something more in common

Right from the start, Dr. Perales and Dr. Giralt also shared a commitment to mentoring. Since 2010, Dr. Perales has mentored 22 up-and-coming junior faculty, including 10 from Europe (8 from Spain) and 2 from Latin America.

“[It makes] the research enterprise much more productive but [these young scientists] really increase the visibility of the program,” said Dr. Giralt.

He cited Dr. Perales’ track record of mentoring as one of the reasons for his promotion to chief of the adult bone marrow transplant service. In March 2020, Dr. Perales seamlessly stepped into Dr. Giralt’s shoes, while Dr. Giralt moved on to his present role as deputy division head of the division of hematologic malignancies.

Dr. Perales said: “The key aspect [of these promotions] is the fantastic working relationship that we’ve had over the years. ... I consider Sergio my mentor, but also a good friend and colleague. And so I think it’s this ability that we’ve had to work together and that relationship of trust, which has been key.”

“Sergio is somebody who lifts people up,” Dr. Perales added. “Many people will tell you that Sergio has helped them in their career. ... And I think that’s a lesson I’ve learned from him: training the next generation. And [that’s] not just in the U.S., but outside. I think that’s a key role that we have. And our responsibility.”

Asked to comment on their 100th-paper milestone, Dr. Perales firmly turned the spotlight from himself and Dr. Giralt to the junior investigators who have passed through the doors of the bone-marrow transplant program: “This body of work represents not just our collaboration but also the many contributions of our team at MSK ... and beyond MSK.”

This article was updated 1/26/22.

Two close colleagues at New York’s Memorial Sloan Kettering Cancer Center, world leaders in hematopoietic stem cell transplantation (HSCT) who were both promoted days after COVID-19 locked down the city in 2020, were too busy battling the pandemic’s impact on patients in the summer of 2021 to notice their latest shared career milestone.

On July 29, 2021, Sergio Giralt, MD, deputy division head of the division of hematologic malignancies and Miguel-Angel Perales, MD, chief of the adult bone marrow transplant service at MSKCC, published their 100th peer-reviewed paper as coauthors. Listing hundreds of such articles on a CV is standard for top-tier physicians, but the pair had gone one better: 100 publications written together in 10 years.

Their centenary article hit scientific newsstands almost exactly a decade after their first joint paper, which appeared in September 2011, not long after they met.

Born in Cuba, Dr. Giralt grew up in Venezuela. From the age of 14, he knew that medicine was his path, and in 1984 he earned a medical degree from the Universidad Central de Venezuela, Caracas. Next came a research position at Harvard Medical School, a residency at the Good Samaritan Hospital, Cincinnati, and a fellowship at the University of Texas MD Anderson Cancer Center, Houston. Dr. Giralt arrived at MSKCC in 2010 as the new chief of the adult bone marrow transplant service. There he was introduced to a new colleague, Dr. Perales. They soon learned that in addition to expertise in hematology, they had second language in common: Spanish.

Dr. Giralt said: “We both have a Spanish background and in a certain sense, there was an affinity there. ... We both have shared experiences.”

Dr. Perales was brought up in Belgium, a European nation with three official languages: French, Dutch, and German. He speaks five tongues in all and learned Spanish from his father, who came from Spain.

Courtesy MSKCC

Fluency in Spanish enables both physicians to take care of the many New Yorkers who are more comfortable in that language – especially when navigating cancer treatment. However, both Dr. Giralt and Dr. Perales said that a second language is more than a professional tool. They described the enjoyable change of persona that happens when they switch to Spanish.

“People who are multilingual have different roles [as much as] different languages,” said Dr. Perales. “When I’m in Spanish, part of my brain is [thinking back to] summer vacations and hanging out with my cousins.”

When it comes to clinical science, however, English is the language of choice.
 

Global leaders in HSCT

Dr. Giralt and Dr. Perales are known worldwide in the field of allogeneic HSCT, a potentially curative treatment for an elongating list of both malignant and nonmalignant diseases.

In 1973, MSKCC conducted the first bone-marrow transplant from an unrelated donor. Fifty years on, medical oncologists in the United States conduct approximately 8,500 allogeneic transplants each year, 72% to treat acute leukemias or myelodysplastic syndrome (MDS).

However, stripping the immune system with intensive chemotherapy ‘conditioning,’ then rebuilding it with non-diseased donor hematopoietic cells is a hazardous undertaking. Older patients are less likely to survive the intensive conditioning, so historically have missed out. Also, even with a good human leukocyte antigen (HLA) match, the recipient needs often brutal immunosuppression.

Since Dr. Giralt and Dr. Perales began their partnership in 2010, the goals of their work have not changed: to develop safer, lower-intensity transplantation suitable for older, more vulnerable patients and reduce fearsome posttransplant sequelae such as graft-versus-host disease (GVHD).

Dr. Giralt’s publication list spans more than 600 peer-reviewed papers, articles and book chapters, almost exclusively on HSCT. Dr. Perales has more than 300 publication credits on the topic.

The two paired up on their first paper just months after Dr. Giralt arrived at MSKCC. That article, published in Biology of Blood and Marrow Transplantation, compared umbilical cord blood for HSCT with donor blood in 367 people with a variety of hematologic malignancies, including acute and chronic leukemias, MDS, and lymphoma.

Courtesy MSKCC

The MSKCC team found that transplant-related mortality in the first 180 days was higher for the cord blood (21%), but thereafter mortality and relapse were much lower than for donated blood, with the result that 2-year progression-free survival of 55% was similar. Dr. Perales, Dr. Giralt and their coauthors concluded that the data provided “strong support” for further work on cord blood as an alternative stem-cell source.

During their first decade of collaboration, Dr. Giralt and Dr. Perales worked on any promising avenue that could improve outcomes and the experience of HSCT recipients, including reduced-intensity conditioning regimens to allow older adults to benefit from curative HSCT and donor T-cell depletion by CD34 selection, to reduce graft-versus-host disease (GVHD).

The CD34 protein is typically found on the surface of early stage and highly active stem cell types. Selecting these cell types using a range of techniques can eliminate many other potentially interfering or inactive cells. This enriches the transplant population with the most effective cells and can lower the risk of GVHD.

The 100th paper on which Dr. Giralt and Dr. Perales were coauthors was published in Blood Advances on July 27, 2021. The retrospective study examined the fate of 58 MSKCC patients with a rare form of chronic lymphocytic leukemia, CLL with Richter’s transformation (CLL-RT). It was the largest such study to date of this rare disease.
M.D. Anderson Cancer Center had shown in 2006 that, despite chemotherapy, overall survival in patients with CLL-RT was approximately 8 months. HSCT improved survival dramatically (75% at 3 years; n = 7). However, with the advent of novel targeted drugs for CLL such as ibrutinib (Imbruvica), venetoclax (Venclexta), or idelalisib (Zydelig), the MSKCC team asked themselves: What was the role of reduced-intensive conditioning HSCT? Was it even safe? Among other findings, Dr. Giralt and Dr. Perales’ 100th paper showed that reduced-intensity HSCT remained a viable alternative after a CLL-RT patient progressed on a novel agent.

Impact of the pandemic

When COVID-19 hit, the team lost many research staff and developed a huge backlog, said Dr. Giralt. He and Dr. Perales realized that they needed to be “thoughtful and careful” about which studies to continue. “For example, the CD-34 selection trials we did not close because these are our workhorse trials,” Dr. Giralt said. “We have people we need to treat, and some of the patients that we need to treat can only be treated on trial.”

The team was also able to pivot some of their work into COVID 19 itself, and they collected crucial information on HSCT in recovered COVID-19 patients, as an example.

“We were living through a critical time, but that doesn’t mean we [aren’t] obligated to continue our mission, our research mission,” said Dr. Giralt. “It really is team science. The way we look at it ... there’s a common thread: We both like to do allogeneic transplant, and we both believe in trying to make CD-34 selection better. So we’re both very much [working on] how can we improve what we call ‘the Memorial way’ of doing transplants. Where we separate is, Miguel does primarily lymphoma. He doesn’t do myeloma [like me]. So in those two areas, we’re helping develop the junior faculty in a different way.”
 

Something more in common

Right from the start, Dr. Perales and Dr. Giralt also shared a commitment to mentoring. Since 2010, Dr. Perales has mentored 22 up-and-coming junior faculty, including 10 from Europe (8 from Spain) and 2 from Latin America.

“[It makes] the research enterprise much more productive but [these young scientists] really increase the visibility of the program,” said Dr. Giralt.

He cited Dr. Perales’ track record of mentoring as one of the reasons for his promotion to chief of the adult bone marrow transplant service. In March 2020, Dr. Perales seamlessly stepped into Dr. Giralt’s shoes, while Dr. Giralt moved on to his present role as deputy division head of the division of hematologic malignancies.

Dr. Perales said: “The key aspect [of these promotions] is the fantastic working relationship that we’ve had over the years. ... I consider Sergio my mentor, but also a good friend and colleague. And so I think it’s this ability that we’ve had to work together and that relationship of trust, which has been key.”

“Sergio is somebody who lifts people up,” Dr. Perales added. “Many people will tell you that Sergio has helped them in their career. ... And I think that’s a lesson I’ve learned from him: training the next generation. And [that’s] not just in the U.S., but outside. I think that’s a key role that we have. And our responsibility.”

Asked to comment on their 100th-paper milestone, Dr. Perales firmly turned the spotlight from himself and Dr. Giralt to the junior investigators who have passed through the doors of the bone-marrow transplant program: “This body of work represents not just our collaboration but also the many contributions of our team at MSK ... and beyond MSK.”

This article was updated 1/26/22.

In a large, blinded study of peanut-allergic toddlers published in The Lancet, 71% of treated participants could safely consume 5,000 mg of peanut protein – equivalent to nearly 17 peanuts – after 2½ years on oral immunotherapy. Even after stopping maintenance dosing for the next 6 months, more than 1 in 5 maintained that level of protection, and nearly 3 in 5 still met the 600-mg benchmark (about 2 peanuts) set by the phase 3 PALISADE trial of the FDA-approved peanut-flour product, Palforzia.

About 2% of children in the United States are allergic to peanuts, and most will not outgrow this allergy. In addition, other research suggests that the immune system is more malleable during early childhood.

Consistent with this idea, prior research showed that toddlers can succeed with peanut oral immunotherapy (OIT) – a regimen that builds tolerance through small amounts of the allergen consumed daily for months. However, that trial (DEVIL) was small, was conducted at a single site, and had no placebo group.

In contrast, the Peanut Oral Immunotherapy in Children Trial (IMPACT) enrolled 146 children aged 1-3 years at five academic medical centers in the United States – the first placebo-controlled study of OIT in this younger age group.

“This is a well done study,” Jaclyn Bjelac, MD, associate director of the Food Allergy Center of Excellence at the Cleveland Clinic, told this news organization. “We have seen improved outcomes in OIT, both in our own experience and other published studies, so while this is no surprise, the outcomes and large number of participants contribute to this being a really exciting publication.”

The trial was long and demanding for families. Toddlers who reacted to 500 mg or less of peanut protein in an entry food challenge were randomized in a 2:1 ratio to receive daily peanut flour or oat flour placebo. After initial dose escalation (from 0.1 mg to 6 mg) and biweekly buildup to a 2,000-mg target dose by week 30, participants continued with 20,00-mg daily maintenance dosing through week 134 – at which point they underwent a food challenge. They then went off treatment for 26 weeks and had another food challenge (week 160). In addition, participants came in for skin-prick and blood tests at baseline and at weeks 30, 82, 134, and 160.

In the placebo group, only 23 of 50 participants (46%) completed the study. “If you did 2½ years of this and then bombed the food challenge, you probably can guess that you were not on the real thing. And they were still asked to come back in 6 months and do it again. So, sure enough, a big chunk of those people chose not to continue, and you can’t blame them,” said Lancet co-author Edwin Kim, MD, in an interview. Dr. Kim directs the UNC Food Allergy Initiative at the University of North Carolina School of Medicine, Chapel Hill.

There was attrition in the treatment group as well. Among 96 children initially assigned to this arm, 68 (71%) passed the 5,000-mg peanut challenge at week 134 – but 11 withdrew in the study’s off-treatment phase. “It was a very tough decision. How much do you give toward science?” said Dr. Kim. “When push came to shove, some of the families couldn’t pull the trigger to potentially give up what they worked so hard for.”

In the intention-to-treat analysis, 20 of 96 treated participants (21%) could still tolerate 5,000 mg of peanut protein after going off therapy for 6 months. That translates to a 29% remission rate in the per-protocol subset (n = 70) who completed the study. Forty (57%) of these completers safely consumed at least 1,755 mg of peanut (cumulative dose). By comparison, the PALISADE trial of Palforzia used a 10,430-mg cumulative peanut dose to measure treatment efficacy.

On safety, 98% of treated participants – but also 80% of the placebo group – reported reactions, of which 35 were treated with epinephrine in 21 children receiving peanut OIT.

While some have noted that epinephrine use seemed high, Dr. Kim said, “we’re actually OK with that, because we’d much rather they overtreat and make sure that 1-year-old is safe than take any chances.” Overall, the safety profile looks similar to prior OIT studies of older children. “I think it suggests that, yeah, side effects will happen, [but] they’re all manageable, and people are not anaphylaxing left and right.”

On remission and immunologic parameters, benefits seemed stronger in the youngest subset (12 to 24 months), particularly those with lower peanut-specific IgE at baseline. These trends require further analyses, though, given the limited number of participants under 24 months.

Another noteworthy observation from longitudinal peanut-specific IgE trends in the placebo group: “Avoidance may not be benign,” Dr. Kim said. “If you look at their labs, they don’t stay flat. They actually go up.” The results jibe with the long-held idea of an early window of opportunity while a child’s immune system is maturing. “If you can grab this kid when his IgE is 10, versus next year when it might be 50, maybe you’ll get a different treatment effect,” Dr. Kim said. “We don’t know that for sure, but the placebo labs kind of point toward that.”

Beyond the science, there are practical advantages to starting OIT early. “Trying to convince a 9-year-old who’s been petrified of peanuts for their whole life to start doing this every day is not an easy task,” whereas with a 1- or 2-year-old, “you build it into their routine,” Dr. Kim said.

Plus, some say there’s no need for families to wait for regulatory approval of additional commercial products for very young children. Though some have advocated against the use of “grocery store” products, most peanut OIT research “has used the same 12% light roast defatted peanut flour used in IMPACT,” noted Marcus S. Shaker, MD, professor of pediatrics and of medicine at the Dartmouth Geisel School of Medicine and a physician at the Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire. The commercial product (Palforzia) and grocery-store products “come from the exact same source in the U.S.,” he said in an interview. “Both are an option for parents to consider, but a commercial product is not, nor has [it] ever been, a necessity.”

Dr. Bjelac reports no relevant financial relationships. Dr. Kim reports consultancy with Aimmune Therapeutics, Allako, AllerGenis, Belhaven Pharma, DBV Technologies, Duke Clinical Research Institute, and Nutricia; advisory board membership with ALK, DBV Technologies, Kenota Health, and Ukko; and grant support from the NIH’s National Institute of Allergy and Infectious Diseases, National Center for Complementary and Integrative Health and Immune Tolerance Network, Food Allergy Research and Education, and the Wallace Research Foundation. Dr. Shaker has participated in research funded by DBV, is cochair of the AAAAI/ACAAI Joint Task Force on Practice Parameters, is an associate editor at the Annals of Allergy, Asthma, and Immunology, and is an editorial board member of the Journal of Allergy and Clinical Immunology in Practice.

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

In a large, blinded study of peanut-allergic toddlers published in The Lancet, 71% of treated participants could safely consume 5,000 mg of peanut protein – equivalent to nearly 17 peanuts – after 2½ years on oral immunotherapy. Even after stopping maintenance dosing for the next 6 months, more than 1 in 5 maintained that level of protection, and nearly 3 in 5 still met the 600-mg benchmark (about 2 peanuts) set by the phase 3 PALISADE trial of the FDA-approved peanut-flour product, Palforzia.

About 2% of children in the United States are allergic to peanuts, and most will not outgrow this allergy. In addition, other research suggests that the immune system is more malleable during early childhood.

Consistent with this idea, prior research showed that toddlers can succeed with peanut oral immunotherapy (OIT) – a regimen that builds tolerance through small amounts of the allergen consumed daily for months. However, that trial (DEVIL) was small, was conducted at a single site, and had no placebo group.

In contrast, the Peanut Oral Immunotherapy in Children Trial (IMPACT) enrolled 146 children aged 1-3 years at five academic medical centers in the United States – the first placebo-controlled study of OIT in this younger age group.

“This is a well done study,” Jaclyn Bjelac, MD, associate director of the Food Allergy Center of Excellence at the Cleveland Clinic, told this news organization. “We have seen improved outcomes in OIT, both in our own experience and other published studies, so while this is no surprise, the outcomes and large number of participants contribute to this being a really exciting publication.”

The trial was long and demanding for families. Toddlers who reacted to 500 mg or less of peanut protein in an entry food challenge were randomized in a 2:1 ratio to receive daily peanut flour or oat flour placebo. After initial dose escalation (from 0.1 mg to 6 mg) and biweekly buildup to a 2,000-mg target dose by week 30, participants continued with 20,00-mg daily maintenance dosing through week 134 – at which point they underwent a food challenge. They then went off treatment for 26 weeks and had another food challenge (week 160). In addition, participants came in for skin-prick and blood tests at baseline and at weeks 30, 82, 134, and 160.

In the placebo group, only 23 of 50 participants (46%) completed the study. “If you did 2½ years of this and then bombed the food challenge, you probably can guess that you were not on the real thing. And they were still asked to come back in 6 months and do it again. So, sure enough, a big chunk of those people chose not to continue, and you can’t blame them,” said Lancet co-author Edwin Kim, MD, in an interview. Dr. Kim directs the UNC Food Allergy Initiative at the University of North Carolina School of Medicine, Chapel Hill.

There was attrition in the treatment group as well. Among 96 children initially assigned to this arm, 68 (71%) passed the 5,000-mg peanut challenge at week 134 – but 11 withdrew in the study’s off-treatment phase. “It was a very tough decision. How much do you give toward science?” said Dr. Kim. “When push came to shove, some of the families couldn’t pull the trigger to potentially give up what they worked so hard for.”

In the intention-to-treat analysis, 20 of 96 treated participants (21%) could still tolerate 5,000 mg of peanut protein after going off therapy for 6 months. That translates to a 29% remission rate in the per-protocol subset (n = 70) who completed the study. Forty (57%) of these completers safely consumed at least 1,755 mg of peanut (cumulative dose). By comparison, the PALISADE trial of Palforzia used a 10,430-mg cumulative peanut dose to measure treatment efficacy.

On safety, 98% of treated participants – but also 80% of the placebo group – reported reactions, of which 35 were treated with epinephrine in 21 children receiving peanut OIT.

While some have noted that epinephrine use seemed high, Dr. Kim said, “we’re actually OK with that, because we’d much rather they overtreat and make sure that 1-year-old is safe than take any chances.” Overall, the safety profile looks similar to prior OIT studies of older children. “I think it suggests that, yeah, side effects will happen, [but] they’re all manageable, and people are not anaphylaxing left and right.”

On remission and immunologic parameters, benefits seemed stronger in the youngest subset (12 to 24 months), particularly those with lower peanut-specific IgE at baseline. These trends require further analyses, though, given the limited number of participants under 24 months.

Another noteworthy observation from longitudinal peanut-specific IgE trends in the placebo group: “Avoidance may not be benign,” Dr. Kim said. “If you look at their labs, they don’t stay flat. They actually go up.” The results jibe with the long-held idea of an early window of opportunity while a child’s immune system is maturing. “If you can grab this kid when his IgE is 10, versus next year when it might be 50, maybe you’ll get a different treatment effect,” Dr. Kim said. “We don’t know that for sure, but the placebo labs kind of point toward that.”

Beyond the science, there are practical advantages to starting OIT early. “Trying to convince a 9-year-old who’s been petrified of peanuts for their whole life to start doing this every day is not an easy task,” whereas with a 1- or 2-year-old, “you build it into their routine,” Dr. Kim said.

Plus, some say there’s no need for families to wait for regulatory approval of additional commercial products for very young children. Though some have advocated against the use of “grocery store” products, most peanut OIT research “has used the same 12% light roast defatted peanut flour used in IMPACT,” noted Marcus S. Shaker, MD, professor of pediatrics and of medicine at the Dartmouth Geisel School of Medicine and a physician at the Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire. The commercial product (Palforzia) and grocery-store products “come from the exact same source in the U.S.,” he said in an interview. “Both are an option for parents to consider, but a commercial product is not, nor has [it] ever been, a necessity.”

Dr. Bjelac reports no relevant financial relationships. Dr. Kim reports consultancy with Aimmune Therapeutics, Allako, AllerGenis, Belhaven Pharma, DBV Technologies, Duke Clinical Research Institute, and Nutricia; advisory board membership with ALK, DBV Technologies, Kenota Health, and Ukko; and grant support from the NIH’s National Institute of Allergy and Infectious Diseases, National Center for Complementary and Integrative Health and Immune Tolerance Network, Food Allergy Research and Education, and the Wallace Research Foundation. Dr. Shaker has participated in research funded by DBV, is cochair of the AAAAI/ACAAI Joint Task Force on Practice Parameters, is an associate editor at the Annals of Allergy, Asthma, and Immunology, and is an editorial board member of the Journal of Allergy and Clinical Immunology in Practice.

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

In a large, blinded study of peanut-allergic toddlers published in The Lancet, 71% of treated participants could safely consume 5,000 mg of peanut protein – equivalent to nearly 17 peanuts – after 2½ years on oral immunotherapy. Even after stopping maintenance dosing for the next 6 months, more than 1 in 5 maintained that level of protection, and nearly 3 in 5 still met the 600-mg benchmark (about 2 peanuts) set by the phase 3 PALISADE trial of the FDA-approved peanut-flour product, Palforzia.

About 2% of children in the United States are allergic to peanuts, and most will not outgrow this allergy. In addition, other research suggests that the immune system is more malleable during early childhood.

Consistent with this idea, prior research showed that toddlers can succeed with peanut oral immunotherapy (OIT) – a regimen that builds tolerance through small amounts of the allergen consumed daily for months. However, that trial (DEVIL) was small, was conducted at a single site, and had no placebo group.

In contrast, the Peanut Oral Immunotherapy in Children Trial (IMPACT) enrolled 146 children aged 1-3 years at five academic medical centers in the United States – the first placebo-controlled study of OIT in this younger age group.

“This is a well done study,” Jaclyn Bjelac, MD, associate director of the Food Allergy Center of Excellence at the Cleveland Clinic, told this news organization. “We have seen improved outcomes in OIT, both in our own experience and other published studies, so while this is no surprise, the outcomes and large number of participants contribute to this being a really exciting publication.”

The trial was long and demanding for families. Toddlers who reacted to 500 mg or less of peanut protein in an entry food challenge were randomized in a 2:1 ratio to receive daily peanut flour or oat flour placebo. After initial dose escalation (from 0.1 mg to 6 mg) and biweekly buildup to a 2,000-mg target dose by week 30, participants continued with 20,00-mg daily maintenance dosing through week 134 – at which point they underwent a food challenge. They then went off treatment for 26 weeks and had another food challenge (week 160). In addition, participants came in for skin-prick and blood tests at baseline and at weeks 30, 82, 134, and 160.

In the placebo group, only 23 of 50 participants (46%) completed the study. “If you did 2½ years of this and then bombed the food challenge, you probably can guess that you were not on the real thing. And they were still asked to come back in 6 months and do it again. So, sure enough, a big chunk of those people chose not to continue, and you can’t blame them,” said Lancet co-author Edwin Kim, MD, in an interview. Dr. Kim directs the UNC Food Allergy Initiative at the University of North Carolina School of Medicine, Chapel Hill.

There was attrition in the treatment group as well. Among 96 children initially assigned to this arm, 68 (71%) passed the 5,000-mg peanut challenge at week 134 – but 11 withdrew in the study’s off-treatment phase. “It was a very tough decision. How much do you give toward science?” said Dr. Kim. “When push came to shove, some of the families couldn’t pull the trigger to potentially give up what they worked so hard for.”

In the intention-to-treat analysis, 20 of 96 treated participants (21%) could still tolerate 5,000 mg of peanut protein after going off therapy for 6 months. That translates to a 29% remission rate in the per-protocol subset (n = 70) who completed the study. Forty (57%) of these completers safely consumed at least 1,755 mg of peanut (cumulative dose). By comparison, the PALISADE trial of Palforzia used a 10,430-mg cumulative peanut dose to measure treatment efficacy.

On safety, 98% of treated participants – but also 80% of the placebo group – reported reactions, of which 35 were treated with epinephrine in 21 children receiving peanut OIT.

While some have noted that epinephrine use seemed high, Dr. Kim said, “we’re actually OK with that, because we’d much rather they overtreat and make sure that 1-year-old is safe than take any chances.” Overall, the safety profile looks similar to prior OIT studies of older children. “I think it suggests that, yeah, side effects will happen, [but] they’re all manageable, and people are not anaphylaxing left and right.”

On remission and immunologic parameters, benefits seemed stronger in the youngest subset (12 to 24 months), particularly those with lower peanut-specific IgE at baseline. These trends require further analyses, though, given the limited number of participants under 24 months.

Another noteworthy observation from longitudinal peanut-specific IgE trends in the placebo group: “Avoidance may not be benign,” Dr. Kim said. “If you look at their labs, they don’t stay flat. They actually go up.” The results jibe with the long-held idea of an early window of opportunity while a child’s immune system is maturing. “If you can grab this kid when his IgE is 10, versus next year when it might be 50, maybe you’ll get a different treatment effect,” Dr. Kim said. “We don’t know that for sure, but the placebo labs kind of point toward that.”

Beyond the science, there are practical advantages to starting OIT early. “Trying to convince a 9-year-old who’s been petrified of peanuts for their whole life to start doing this every day is not an easy task,” whereas with a 1- or 2-year-old, “you build it into their routine,” Dr. Kim said.

Plus, some say there’s no need for families to wait for regulatory approval of additional commercial products for very young children. Though some have advocated against the use of “grocery store” products, most peanut OIT research “has used the same 12% light roast defatted peanut flour used in IMPACT,” noted Marcus S. Shaker, MD, professor of pediatrics and of medicine at the Dartmouth Geisel School of Medicine and a physician at the Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire. The commercial product (Palforzia) and grocery-store products “come from the exact same source in the U.S.,” he said in an interview. “Both are an option for parents to consider, but a commercial product is not, nor has [it] ever been, a necessity.”

Dr. Bjelac reports no relevant financial relationships. Dr. Kim reports consultancy with Aimmune Therapeutics, Allako, AllerGenis, Belhaven Pharma, DBV Technologies, Duke Clinical Research Institute, and Nutricia; advisory board membership with ALK, DBV Technologies, Kenota Health, and Ukko; and grant support from the NIH’s National Institute of Allergy and Infectious Diseases, National Center for Complementary and Integrative Health and Immune Tolerance Network, Food Allergy Research and Education, and the Wallace Research Foundation. Dr. Shaker has participated in research funded by DBV, is cochair of the AAAAI/ACAAI Joint Task Force on Practice Parameters, is an associate editor at the Annals of Allergy, Asthma, and Immunology, and is an editorial board member of the Journal of Allergy and Clinical Immunology in Practice.

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