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Why don’t doctors feel like heroes anymore?
In April 2020, as many Americans prepared to spend the Easter holiday in lockdown, pop star Mariah Carey released a video honoring the “sacrifices and courage” of frontline workers battling COVID-19 – her 1993 hit, “Hero.”
“The sorrow that you know will melt away,” Ms. Carey sang. “When you feel like hope is gone,” the song continued, strength and answers can be found within, and “a hero lies in you.”
For health care professionals, the reality of 2020 wasn’t quite so uplifting. PPE shortages and spillover ICUs had many feeling helpless, exhausted, and overwhelmed. Few if any medical professionals felt their sorrows “melt away.”
We can’t expect depth and nuance from pop songs, but we can find in them the imagery that runs through our culture. The “hero narrative” – the idea that doctors, nurses, and others in health care have superhuman endurance and selflessness – has long been an undercurrent in the medical field.
And yet, without a workforce willing to perform without adequate sleep, food, or time off, the health care system couldn’t function, says Brian Park, MD, MPH, a family medicine physician at Oregon Health & Science University, Portland. At many academic health centers, for example, residents are “the bedrock of the workforce,” he explains. If they didn’t work 80-100 hours per week, those systems wouldn’t exist.
So, how do we look at the health care system in a way that is both grateful and critical, Dr. Park wonders. “How do we honor extreme acts of heroism and also acknowledge that the system sometimes gets by on the acts of heroes to patch up some of the brokenness and fragmentation within it?”
Heroes are determined
Ala Stanford, MD, a pediatric surgeon in Philadelphia, has frequently been called a “health care hero.” Given the title by CNN in 2021, she has received numerous other awards and accolades, featured in Fortune Magazine’s “World’s 50 Greatest Leaders” in 2021 and USA Today’s “Women of the Year” in 2022.
In 2020, Dr. Stanford was sheltering in place and watching “way too much” cable news. “They would play solemn music and show photos of all the people who had died,” she recalls. “I thought, ‘All these people are Black or brown. What is going on?’”
The standard explanation was that people of color were more vulnerable because they were more likely to be essential workers or have chronic health conditions. But Dr. Stanford believed this was only part of the story. The reason she saw that local Black communities had higher positivity rates was because people couldn’t get a COVID test.
Dr. Stanford got call after call from Philadelphians who had been turned away from testing centers. When she questioned colleagues, “they gave me every reason under the sun,” Dr. Stanford says. “It was because someone took public transportation, and they were only testing people in cars, or because they weren’t over 65, or because they didn’t have other comorbid health conditions, or because they weren’t a health care worker, or because they hadn’t traveled to China ...” The list went on.
Dr. Stanford appealed to local, state, and federal health authorities. Finally, she took matters into her own hands. She found tests, packed a van with masks, gowns, and gloves, and drove across the city going door to door. Eventually, she organized testing in the parking lots of Black churches, sometimes seeing more than 400 people per day.
The services were funded entirely through her own bank account and donations until she was eventually awarded a CDC grant through the Coronavirus Aid, Relief, and Economic Security (CARES) Act of 2020 and began to receive contracts from the city.
Since then, Dr. Stanford’s mission has evolved. She and her team provided COVID vaccinations to thousands, and in 2021, opened the Dr. Ala Stanford Center for Health Equity. The center offers primary care for all ages in underserved communities.
Still, Dr. Stanford doesn’t think of herself as a hero, and she stresses that many other people contributed to her success. “I think the world was on fire, and we were all firefighters,” Dr. Stanford says. “Someone said to me, ‘Ala, you ran to the fire and everyone else was running away from it, and you didn’t have to.’ … I feel like I was able to galvanize people to realize the power that they actually had. Maybe independently, they couldn’t do a whole lot, but collectively, we were a force.”
Heroes are selfless
Nicole Jackson, RN, an emergency room manager and nurse at Advocate Trinity Hospital in Chicago, was recently honored as a Health Care Hero by the American Red Cross of Greater Chicago.
On June 23, 2022, Jackson’s emergency department was understaffed and struggling with an influx of patients when three gunshot victims arrived. Two needed to be transferred to a trauma center, and one – with multiple gunshot wounds – required a critical care nurse in the ambulance. But the ETA for that transport was 90 minutes, which meant the patient might not survive. Although Ms. Jackson was already working beyond her shift, she rode in the ambulance with the patient herself and probably saved his life.
While this incident stood out to a colleague who nominated her for the Red Cross award, Ms. Jackson finds herself working extra hours fairly often. “Since COVID, that’s pretty much been like any other hospital,” she says. “We’ve had staffing challenges that we work through every day. So, the nurses come, they show up, and they do the best that they can with what we have to keep our patients safe.”
A 2022 survey by McKinsey estimated that by 2025, there could be a gap of 200,000 to 450,000 nurses in the United States. A two-year impact assessment from the American Nurses Foundation found that among more than 12,500 nurses, 40% were considering leaving their positions before the pandemic. By 2022, that number had jumped to 52% with the top reasons being insufficient staffing and negative effects on health and well-being.
Can the “hero narrative” help that situation? Ms. Jackson says she doesn’t see herself as a hero, but the supportive environment and gestures of recognition by staff do make her feel appreciated. These include daily messages offering “kudos” and nominations for the DAISY Award, which she herself received in 2022.
“I have people who I have encouraged to become nurses,” Ms. Jackson says, “and when they saw [the award], they were really excited about becoming a nurse.”
Heroes are strong
Jasmine Marcelin, MD, an infectious disease physician with Nebraska Medicine in Omaha, understands the need for heroes as symbols and sources of inspiration. Dr. Marcelin is a fan of the superhero movie genre. There is value, she says, in feeling hope and excitement while watching Superman or Wonder Woman save the day. Who doesn’t want to believe (if only briefly) that the good guys will always win?
In reality, Dr. Marcelin says, “none of us are invincible.” And it’s dangerous to forget that “the people behind the symbols are also human.”
In 2021, Dr. Marcelin gave a TEDx talk entitled, “The Myth of the Health Care Hero.” In it she discussed the extreme physical and mental toll of the pandemic on health care workers and urged her audience to think less about extravagant praise and more about their personal responsibilities. “We don’t want or need to be called heroes,” Dr. Marcelin said. “Right now, our love language is action. We need your help, and we cannot save the world on our own.”
Dr. Marcelin also sees links between superhuman expectations and the high levels of burnout in the medical field.
“It’s a systemic issue,” she explains, “where it requires a revamping and revitalization of the entire psyche of health care to recognize that the people working within this profession are human. And the things that we think and feel and need are the same as anybody else.”
Heroes are self-sacrificing
Well-being, burnout, and disengagement in health care has become a focus for Oregon Health & Science’s Dr. Park, who is also director of RELATE Lab, an organization that aims to make health care more human-centered and equitable through leadership training, research, and community organizing.
For him, hearing neighbors banging pots and pans during the early pandemic was complicated. “The first phase for me was, ‘Thank you. I feel seen. I feel appreciated,’ ” he says. “Yes, I’m wearing a mask. I’m going in. I’m changing in the garage when I come home, so my kid and my partner don’t get sick.”
But after a while, the cheers started to feel like pressure. “Have I done anything heroic today?” Dr. Park asked himself. “Have I been as heroic as my friend who is in the hospital in the ICU? I don’t deserve this, so don’t bang those pots and pans for me.”
When your identity becomes about being a hero, Dr. Park says, when that becomes the standard by which you measure yourself, the result is often a sense of shame.
“I think a lot of people feel ashamed that they feel burnout,” he says, “because they’re supposed to be heroes, putting on their capes and masks. They’re waking up and saying, ‘I’m exhausted, and I can’t play that part today. But I know that’s the social expectation of me.’ “
Heroes are noble
There may not be a clear solution, but for many health care professionals, symbolic gestures alone are inadequate and, in certain cases, insulting.
On Doctor’s Day 2023, Alok Patel, MD, a pediatric hospitalist, tweeted a photo of an appreciation “gift” for staff from an unnamed hospital. The small items had metaphorical meanings – a rubber band “as a reminder to stay flexible,” a quarter “as a reminder to ‘call’ for help,” etc.
“Welcome to how you give thanks to ‘health care heroes,’ ” Dr. Patel tweeted.
For Dr. Patel, the issue is not lavish gifts but a need for an attitude shift. He recalls colleagues who felt ashamed asking for mental health services or time off, “because they were bombarded by the hero narrative, by the manufactured pressure that they needed to put their jobs above their own health – because that’s what ‘heroes’ do. I’m willing to bet most physicians would rather receive a sincere email with a transparent plan to better support health care workers than any Doctor’s Day gift,” he says.
In Dr. Marcelin’s TEDx talk, she quotes Spider-Man’s classic adage, “With great power, comes great responsibility.” She argues that this motto doesn’t just apply to those who can fly or deflect bullets; that’s not what heroism is. In fact, most people have their own definition of the word.
For Dr. Stanford, a hero is “someone who is selfless, putting the needs of others before their own.” Dr. Park believes there are no individual heroes. “It’s the work of the collective that’s truly heroic.”
By those standards, clearly anyone can step up, offer help, act with courage and kindness, and be heroic. “We humans, as ordinary as we are, can be extraordinary by using our power to do what’s right,” Dr. Marcelin says, “because there’s no such thing as health care heroes, just good people doing the right thing.”
A version of this article first appeared on Medscape.com.
In April 2020, as many Americans prepared to spend the Easter holiday in lockdown, pop star Mariah Carey released a video honoring the “sacrifices and courage” of frontline workers battling COVID-19 – her 1993 hit, “Hero.”
“The sorrow that you know will melt away,” Ms. Carey sang. “When you feel like hope is gone,” the song continued, strength and answers can be found within, and “a hero lies in you.”
For health care professionals, the reality of 2020 wasn’t quite so uplifting. PPE shortages and spillover ICUs had many feeling helpless, exhausted, and overwhelmed. Few if any medical professionals felt their sorrows “melt away.”
We can’t expect depth and nuance from pop songs, but we can find in them the imagery that runs through our culture. The “hero narrative” – the idea that doctors, nurses, and others in health care have superhuman endurance and selflessness – has long been an undercurrent in the medical field.
And yet, without a workforce willing to perform without adequate sleep, food, or time off, the health care system couldn’t function, says Brian Park, MD, MPH, a family medicine physician at Oregon Health & Science University, Portland. At many academic health centers, for example, residents are “the bedrock of the workforce,” he explains. If they didn’t work 80-100 hours per week, those systems wouldn’t exist.
So, how do we look at the health care system in a way that is both grateful and critical, Dr. Park wonders. “How do we honor extreme acts of heroism and also acknowledge that the system sometimes gets by on the acts of heroes to patch up some of the brokenness and fragmentation within it?”
Heroes are determined
Ala Stanford, MD, a pediatric surgeon in Philadelphia, has frequently been called a “health care hero.” Given the title by CNN in 2021, she has received numerous other awards and accolades, featured in Fortune Magazine’s “World’s 50 Greatest Leaders” in 2021 and USA Today’s “Women of the Year” in 2022.
In 2020, Dr. Stanford was sheltering in place and watching “way too much” cable news. “They would play solemn music and show photos of all the people who had died,” she recalls. “I thought, ‘All these people are Black or brown. What is going on?’”
The standard explanation was that people of color were more vulnerable because they were more likely to be essential workers or have chronic health conditions. But Dr. Stanford believed this was only part of the story. The reason she saw that local Black communities had higher positivity rates was because people couldn’t get a COVID test.
Dr. Stanford got call after call from Philadelphians who had been turned away from testing centers. When she questioned colleagues, “they gave me every reason under the sun,” Dr. Stanford says. “It was because someone took public transportation, and they were only testing people in cars, or because they weren’t over 65, or because they didn’t have other comorbid health conditions, or because they weren’t a health care worker, or because they hadn’t traveled to China ...” The list went on.
Dr. Stanford appealed to local, state, and federal health authorities. Finally, she took matters into her own hands. She found tests, packed a van with masks, gowns, and gloves, and drove across the city going door to door. Eventually, she organized testing in the parking lots of Black churches, sometimes seeing more than 400 people per day.
The services were funded entirely through her own bank account and donations until she was eventually awarded a CDC grant through the Coronavirus Aid, Relief, and Economic Security (CARES) Act of 2020 and began to receive contracts from the city.
Since then, Dr. Stanford’s mission has evolved. She and her team provided COVID vaccinations to thousands, and in 2021, opened the Dr. Ala Stanford Center for Health Equity. The center offers primary care for all ages in underserved communities.
Still, Dr. Stanford doesn’t think of herself as a hero, and she stresses that many other people contributed to her success. “I think the world was on fire, and we were all firefighters,” Dr. Stanford says. “Someone said to me, ‘Ala, you ran to the fire and everyone else was running away from it, and you didn’t have to.’ … I feel like I was able to galvanize people to realize the power that they actually had. Maybe independently, they couldn’t do a whole lot, but collectively, we were a force.”
Heroes are selfless
Nicole Jackson, RN, an emergency room manager and nurse at Advocate Trinity Hospital in Chicago, was recently honored as a Health Care Hero by the American Red Cross of Greater Chicago.
On June 23, 2022, Jackson’s emergency department was understaffed and struggling with an influx of patients when three gunshot victims arrived. Two needed to be transferred to a trauma center, and one – with multiple gunshot wounds – required a critical care nurse in the ambulance. But the ETA for that transport was 90 minutes, which meant the patient might not survive. Although Ms. Jackson was already working beyond her shift, she rode in the ambulance with the patient herself and probably saved his life.
While this incident stood out to a colleague who nominated her for the Red Cross award, Ms. Jackson finds herself working extra hours fairly often. “Since COVID, that’s pretty much been like any other hospital,” she says. “We’ve had staffing challenges that we work through every day. So, the nurses come, they show up, and they do the best that they can with what we have to keep our patients safe.”
A 2022 survey by McKinsey estimated that by 2025, there could be a gap of 200,000 to 450,000 nurses in the United States. A two-year impact assessment from the American Nurses Foundation found that among more than 12,500 nurses, 40% were considering leaving their positions before the pandemic. By 2022, that number had jumped to 52% with the top reasons being insufficient staffing and negative effects on health and well-being.
Can the “hero narrative” help that situation? Ms. Jackson says she doesn’t see herself as a hero, but the supportive environment and gestures of recognition by staff do make her feel appreciated. These include daily messages offering “kudos” and nominations for the DAISY Award, which she herself received in 2022.
“I have people who I have encouraged to become nurses,” Ms. Jackson says, “and when they saw [the award], they were really excited about becoming a nurse.”
Heroes are strong
Jasmine Marcelin, MD, an infectious disease physician with Nebraska Medicine in Omaha, understands the need for heroes as symbols and sources of inspiration. Dr. Marcelin is a fan of the superhero movie genre. There is value, she says, in feeling hope and excitement while watching Superman or Wonder Woman save the day. Who doesn’t want to believe (if only briefly) that the good guys will always win?
In reality, Dr. Marcelin says, “none of us are invincible.” And it’s dangerous to forget that “the people behind the symbols are also human.”
In 2021, Dr. Marcelin gave a TEDx talk entitled, “The Myth of the Health Care Hero.” In it she discussed the extreme physical and mental toll of the pandemic on health care workers and urged her audience to think less about extravagant praise and more about their personal responsibilities. “We don’t want or need to be called heroes,” Dr. Marcelin said. “Right now, our love language is action. We need your help, and we cannot save the world on our own.”
Dr. Marcelin also sees links between superhuman expectations and the high levels of burnout in the medical field.
“It’s a systemic issue,” she explains, “where it requires a revamping and revitalization of the entire psyche of health care to recognize that the people working within this profession are human. And the things that we think and feel and need are the same as anybody else.”
Heroes are self-sacrificing
Well-being, burnout, and disengagement in health care has become a focus for Oregon Health & Science’s Dr. Park, who is also director of RELATE Lab, an organization that aims to make health care more human-centered and equitable through leadership training, research, and community organizing.
For him, hearing neighbors banging pots and pans during the early pandemic was complicated. “The first phase for me was, ‘Thank you. I feel seen. I feel appreciated,’ ” he says. “Yes, I’m wearing a mask. I’m going in. I’m changing in the garage when I come home, so my kid and my partner don’t get sick.”
But after a while, the cheers started to feel like pressure. “Have I done anything heroic today?” Dr. Park asked himself. “Have I been as heroic as my friend who is in the hospital in the ICU? I don’t deserve this, so don’t bang those pots and pans for me.”
When your identity becomes about being a hero, Dr. Park says, when that becomes the standard by which you measure yourself, the result is often a sense of shame.
“I think a lot of people feel ashamed that they feel burnout,” he says, “because they’re supposed to be heroes, putting on their capes and masks. They’re waking up and saying, ‘I’m exhausted, and I can’t play that part today. But I know that’s the social expectation of me.’ “
Heroes are noble
There may not be a clear solution, but for many health care professionals, symbolic gestures alone are inadequate and, in certain cases, insulting.
On Doctor’s Day 2023, Alok Patel, MD, a pediatric hospitalist, tweeted a photo of an appreciation “gift” for staff from an unnamed hospital. The small items had metaphorical meanings – a rubber band “as a reminder to stay flexible,” a quarter “as a reminder to ‘call’ for help,” etc.
“Welcome to how you give thanks to ‘health care heroes,’ ” Dr. Patel tweeted.
For Dr. Patel, the issue is not lavish gifts but a need for an attitude shift. He recalls colleagues who felt ashamed asking for mental health services or time off, “because they were bombarded by the hero narrative, by the manufactured pressure that they needed to put their jobs above their own health – because that’s what ‘heroes’ do. I’m willing to bet most physicians would rather receive a sincere email with a transparent plan to better support health care workers than any Doctor’s Day gift,” he says.
In Dr. Marcelin’s TEDx talk, she quotes Spider-Man’s classic adage, “With great power, comes great responsibility.” She argues that this motto doesn’t just apply to those who can fly or deflect bullets; that’s not what heroism is. In fact, most people have their own definition of the word.
For Dr. Stanford, a hero is “someone who is selfless, putting the needs of others before their own.” Dr. Park believes there are no individual heroes. “It’s the work of the collective that’s truly heroic.”
By those standards, clearly anyone can step up, offer help, act with courage and kindness, and be heroic. “We humans, as ordinary as we are, can be extraordinary by using our power to do what’s right,” Dr. Marcelin says, “because there’s no such thing as health care heroes, just good people doing the right thing.”
A version of this article first appeared on Medscape.com.
In April 2020, as many Americans prepared to spend the Easter holiday in lockdown, pop star Mariah Carey released a video honoring the “sacrifices and courage” of frontline workers battling COVID-19 – her 1993 hit, “Hero.”
“The sorrow that you know will melt away,” Ms. Carey sang. “When you feel like hope is gone,” the song continued, strength and answers can be found within, and “a hero lies in you.”
For health care professionals, the reality of 2020 wasn’t quite so uplifting. PPE shortages and spillover ICUs had many feeling helpless, exhausted, and overwhelmed. Few if any medical professionals felt their sorrows “melt away.”
We can’t expect depth and nuance from pop songs, but we can find in them the imagery that runs through our culture. The “hero narrative” – the idea that doctors, nurses, and others in health care have superhuman endurance and selflessness – has long been an undercurrent in the medical field.
And yet, without a workforce willing to perform without adequate sleep, food, or time off, the health care system couldn’t function, says Brian Park, MD, MPH, a family medicine physician at Oregon Health & Science University, Portland. At many academic health centers, for example, residents are “the bedrock of the workforce,” he explains. If they didn’t work 80-100 hours per week, those systems wouldn’t exist.
So, how do we look at the health care system in a way that is both grateful and critical, Dr. Park wonders. “How do we honor extreme acts of heroism and also acknowledge that the system sometimes gets by on the acts of heroes to patch up some of the brokenness and fragmentation within it?”
Heroes are determined
Ala Stanford, MD, a pediatric surgeon in Philadelphia, has frequently been called a “health care hero.” Given the title by CNN in 2021, she has received numerous other awards and accolades, featured in Fortune Magazine’s “World’s 50 Greatest Leaders” in 2021 and USA Today’s “Women of the Year” in 2022.
In 2020, Dr. Stanford was sheltering in place and watching “way too much” cable news. “They would play solemn music and show photos of all the people who had died,” she recalls. “I thought, ‘All these people are Black or brown. What is going on?’”
The standard explanation was that people of color were more vulnerable because they were more likely to be essential workers or have chronic health conditions. But Dr. Stanford believed this was only part of the story. The reason she saw that local Black communities had higher positivity rates was because people couldn’t get a COVID test.
Dr. Stanford got call after call from Philadelphians who had been turned away from testing centers. When she questioned colleagues, “they gave me every reason under the sun,” Dr. Stanford says. “It was because someone took public transportation, and they were only testing people in cars, or because they weren’t over 65, or because they didn’t have other comorbid health conditions, or because they weren’t a health care worker, or because they hadn’t traveled to China ...” The list went on.
Dr. Stanford appealed to local, state, and federal health authorities. Finally, she took matters into her own hands. She found tests, packed a van with masks, gowns, and gloves, and drove across the city going door to door. Eventually, she organized testing in the parking lots of Black churches, sometimes seeing more than 400 people per day.
The services were funded entirely through her own bank account and donations until she was eventually awarded a CDC grant through the Coronavirus Aid, Relief, and Economic Security (CARES) Act of 2020 and began to receive contracts from the city.
Since then, Dr. Stanford’s mission has evolved. She and her team provided COVID vaccinations to thousands, and in 2021, opened the Dr. Ala Stanford Center for Health Equity. The center offers primary care for all ages in underserved communities.
Still, Dr. Stanford doesn’t think of herself as a hero, and she stresses that many other people contributed to her success. “I think the world was on fire, and we were all firefighters,” Dr. Stanford says. “Someone said to me, ‘Ala, you ran to the fire and everyone else was running away from it, and you didn’t have to.’ … I feel like I was able to galvanize people to realize the power that they actually had. Maybe independently, they couldn’t do a whole lot, but collectively, we were a force.”
Heroes are selfless
Nicole Jackson, RN, an emergency room manager and nurse at Advocate Trinity Hospital in Chicago, was recently honored as a Health Care Hero by the American Red Cross of Greater Chicago.
On June 23, 2022, Jackson’s emergency department was understaffed and struggling with an influx of patients when three gunshot victims arrived. Two needed to be transferred to a trauma center, and one – with multiple gunshot wounds – required a critical care nurse in the ambulance. But the ETA for that transport was 90 minutes, which meant the patient might not survive. Although Ms. Jackson was already working beyond her shift, she rode in the ambulance with the patient herself and probably saved his life.
While this incident stood out to a colleague who nominated her for the Red Cross award, Ms. Jackson finds herself working extra hours fairly often. “Since COVID, that’s pretty much been like any other hospital,” she says. “We’ve had staffing challenges that we work through every day. So, the nurses come, they show up, and they do the best that they can with what we have to keep our patients safe.”
A 2022 survey by McKinsey estimated that by 2025, there could be a gap of 200,000 to 450,000 nurses in the United States. A two-year impact assessment from the American Nurses Foundation found that among more than 12,500 nurses, 40% were considering leaving their positions before the pandemic. By 2022, that number had jumped to 52% with the top reasons being insufficient staffing and negative effects on health and well-being.
Can the “hero narrative” help that situation? Ms. Jackson says she doesn’t see herself as a hero, but the supportive environment and gestures of recognition by staff do make her feel appreciated. These include daily messages offering “kudos” and nominations for the DAISY Award, which she herself received in 2022.
“I have people who I have encouraged to become nurses,” Ms. Jackson says, “and when they saw [the award], they were really excited about becoming a nurse.”
Heroes are strong
Jasmine Marcelin, MD, an infectious disease physician with Nebraska Medicine in Omaha, understands the need for heroes as symbols and sources of inspiration. Dr. Marcelin is a fan of the superhero movie genre. There is value, she says, in feeling hope and excitement while watching Superman or Wonder Woman save the day. Who doesn’t want to believe (if only briefly) that the good guys will always win?
In reality, Dr. Marcelin says, “none of us are invincible.” And it’s dangerous to forget that “the people behind the symbols are also human.”
In 2021, Dr. Marcelin gave a TEDx talk entitled, “The Myth of the Health Care Hero.” In it she discussed the extreme physical and mental toll of the pandemic on health care workers and urged her audience to think less about extravagant praise and more about their personal responsibilities. “We don’t want or need to be called heroes,” Dr. Marcelin said. “Right now, our love language is action. We need your help, and we cannot save the world on our own.”
Dr. Marcelin also sees links between superhuman expectations and the high levels of burnout in the medical field.
“It’s a systemic issue,” she explains, “where it requires a revamping and revitalization of the entire psyche of health care to recognize that the people working within this profession are human. And the things that we think and feel and need are the same as anybody else.”
Heroes are self-sacrificing
Well-being, burnout, and disengagement in health care has become a focus for Oregon Health & Science’s Dr. Park, who is also director of RELATE Lab, an organization that aims to make health care more human-centered and equitable through leadership training, research, and community organizing.
For him, hearing neighbors banging pots and pans during the early pandemic was complicated. “The first phase for me was, ‘Thank you. I feel seen. I feel appreciated,’ ” he says. “Yes, I’m wearing a mask. I’m going in. I’m changing in the garage when I come home, so my kid and my partner don’t get sick.”
But after a while, the cheers started to feel like pressure. “Have I done anything heroic today?” Dr. Park asked himself. “Have I been as heroic as my friend who is in the hospital in the ICU? I don’t deserve this, so don’t bang those pots and pans for me.”
When your identity becomes about being a hero, Dr. Park says, when that becomes the standard by which you measure yourself, the result is often a sense of shame.
“I think a lot of people feel ashamed that they feel burnout,” he says, “because they’re supposed to be heroes, putting on their capes and masks. They’re waking up and saying, ‘I’m exhausted, and I can’t play that part today. But I know that’s the social expectation of me.’ “
Heroes are noble
There may not be a clear solution, but for many health care professionals, symbolic gestures alone are inadequate and, in certain cases, insulting.
On Doctor’s Day 2023, Alok Patel, MD, a pediatric hospitalist, tweeted a photo of an appreciation “gift” for staff from an unnamed hospital. The small items had metaphorical meanings – a rubber band “as a reminder to stay flexible,” a quarter “as a reminder to ‘call’ for help,” etc.
“Welcome to how you give thanks to ‘health care heroes,’ ” Dr. Patel tweeted.
For Dr. Patel, the issue is not lavish gifts but a need for an attitude shift. He recalls colleagues who felt ashamed asking for mental health services or time off, “because they were bombarded by the hero narrative, by the manufactured pressure that they needed to put their jobs above their own health – because that’s what ‘heroes’ do. I’m willing to bet most physicians would rather receive a sincere email with a transparent plan to better support health care workers than any Doctor’s Day gift,” he says.
In Dr. Marcelin’s TEDx talk, she quotes Spider-Man’s classic adage, “With great power, comes great responsibility.” She argues that this motto doesn’t just apply to those who can fly or deflect bullets; that’s not what heroism is. In fact, most people have their own definition of the word.
For Dr. Stanford, a hero is “someone who is selfless, putting the needs of others before their own.” Dr. Park believes there are no individual heroes. “It’s the work of the collective that’s truly heroic.”
By those standards, clearly anyone can step up, offer help, act with courage and kindness, and be heroic. “We humans, as ordinary as we are, can be extraordinary by using our power to do what’s right,” Dr. Marcelin says, “because there’s no such thing as health care heroes, just good people doing the right thing.”
A version of this article first appeared on Medscape.com.
Intense exercise may lead to colds. A new study tells us why
Can too much of a healthy habit become bad?
Lots of evidence shows that regular exercise wards off respiratory infections such as colds, flu, and COVID-19. However, according to a new study.
The findings come as we enter another possible tripledemic this winter, with an increase in COVID, flu, and respiratory syncytial virus (RSV). Public health officials are on alert for a potentially severe flu season, following high flu activity this year in Australia (which can help predict how bad the U.S. flu season will be).
Studies show that the risk for acute respiratory infections is lower in people who exercise regularly. Physically active people are also less likely to suffer severe outcomes from COVID.
But while inactivity has emerged as a potential risk factor for respiratory infections, scientists have long proposed that too much activity, particularly of a prolonged and highly intense nature, may also increase susceptibility.
“The theory suggests that a short-term suppression of the immune system following intense exercise leads to an increase in susceptibility to infection, especially upper respiratory illness,” said Choukri Ben Mamoun, PhD, professor of medicine (infectious diseases) and microbial pathogenesis at the Yale Institute for Global Health, New Haven, Conn. Researchers have documented a greater incidence of upper respiratory illness “among both highly trained and healthy untrained individuals following increased activity during competition or heaving training blocks.”
That’s important if you treat athletes or patients with physically demanding jobs that push them to their physical limits, such as firefighters, police officers, or military personnel.
The new study was small but sheds light on a possible mechanism. Researchers tested blood, saliva, and urine samples from 11 firefighters before and 10 minutes after intense exercise designed to mimic wildfire fighting. The firefighters hiked over hilly terrain for 45 minutes in humid weather wearing up to 44 pounds of wildland gear.
After the workout, subjects had fewer proinflammatory cytokines and ceramides, and more antimicrobial peptides, changes that indicate a greater susceptibility to infection, researchers said. A systematic review adds weight to their findings, revealing a handful of studies in marathon runners, firefighters, soldiers, and soccer players that found an increase in respiratory symptoms after strenuous workouts.
“The relationship between exercise and the immune system is complex and varies from person to person,” said Dr. Mamoun, who was not part of the study. “Physicians can use this study’s findings to provide individualized exercise recommendations.”
An adaptive mechanism gone awry
During intense exercise, the body may reduce airway inflammation to help you breathe, say the authors. The boost in antimicrobial peptides found in the saliva samples could be the body’s way of compensating for the diminished immune function.
Antimicrobial peptides are part of the immune response but they’re “usually not very effective for viral infections,” said lead author Ernesto Nakayasu, PhD, senior research scientist at the Pacific Northwest National Laboratory, a U.S. Department of Energy lab in Richland, Washington. “That’s why we think it may make you more exposed to respiratory infections.”
The drop in proinflammatory molecules had an inverse relationship with opiorphin, a peripheral tissue vasodilator thought to increase blood flow and improve oxygen delivery to the muscles during exercise. This may be an adaptive mechanism to improve gas exchange in response to greater oxygen demand.
But as with many adaptive mechanisms, this one may have an unintended consequence. Fewer proinflammatory molecules on patrol may leave you more vulnerable to infection. Plus, during intense exercise, people tend to breathe through their mouths, bypassing the nasal barriers and allowing more microbes – including viruses – to penetrate and deposit in the distal airways of the lungs.
Advice for patients
More research is needed to know exactly how long and how strenuously one needs to exercise to trigger these immune changes, Dr. Nakayasu said.
As shown by their lactate accumulation (an indicator of anaerobic metabolism), the firefighters in the study outpaced the average person’s aerobic respiratory capacity, meaning the average person doing moderate exercise likely wouldn’t trigger these changes.
“Regular moderate exercise is generally associated with better health outcomes [and] improved immune function,” said Dr. Mamoun. For those who exercise to the extreme, proper rest and recovery are “essential for maintaining a robust immune system,” Dr. Mamoun said.
And of course, you can encourage patients to get vaccinated. Young, healthy patients may assume they don’t need COVID-19 or flu shots, as indicated by a recent survey that found one-third of Americans feel they don’t need these vaccinations if they’re not high risk.
A version of this article first appeared on Medscape.com.
Can too much of a healthy habit become bad?
Lots of evidence shows that regular exercise wards off respiratory infections such as colds, flu, and COVID-19. However, according to a new study.
The findings come as we enter another possible tripledemic this winter, with an increase in COVID, flu, and respiratory syncytial virus (RSV). Public health officials are on alert for a potentially severe flu season, following high flu activity this year in Australia (which can help predict how bad the U.S. flu season will be).
Studies show that the risk for acute respiratory infections is lower in people who exercise regularly. Physically active people are also less likely to suffer severe outcomes from COVID.
But while inactivity has emerged as a potential risk factor for respiratory infections, scientists have long proposed that too much activity, particularly of a prolonged and highly intense nature, may also increase susceptibility.
“The theory suggests that a short-term suppression of the immune system following intense exercise leads to an increase in susceptibility to infection, especially upper respiratory illness,” said Choukri Ben Mamoun, PhD, professor of medicine (infectious diseases) and microbial pathogenesis at the Yale Institute for Global Health, New Haven, Conn. Researchers have documented a greater incidence of upper respiratory illness “among both highly trained and healthy untrained individuals following increased activity during competition or heaving training blocks.”
That’s important if you treat athletes or patients with physically demanding jobs that push them to their physical limits, such as firefighters, police officers, or military personnel.
The new study was small but sheds light on a possible mechanism. Researchers tested blood, saliva, and urine samples from 11 firefighters before and 10 minutes after intense exercise designed to mimic wildfire fighting. The firefighters hiked over hilly terrain for 45 minutes in humid weather wearing up to 44 pounds of wildland gear.
After the workout, subjects had fewer proinflammatory cytokines and ceramides, and more antimicrobial peptides, changes that indicate a greater susceptibility to infection, researchers said. A systematic review adds weight to their findings, revealing a handful of studies in marathon runners, firefighters, soldiers, and soccer players that found an increase in respiratory symptoms after strenuous workouts.
“The relationship between exercise and the immune system is complex and varies from person to person,” said Dr. Mamoun, who was not part of the study. “Physicians can use this study’s findings to provide individualized exercise recommendations.”
An adaptive mechanism gone awry
During intense exercise, the body may reduce airway inflammation to help you breathe, say the authors. The boost in antimicrobial peptides found in the saliva samples could be the body’s way of compensating for the diminished immune function.
Antimicrobial peptides are part of the immune response but they’re “usually not very effective for viral infections,” said lead author Ernesto Nakayasu, PhD, senior research scientist at the Pacific Northwest National Laboratory, a U.S. Department of Energy lab in Richland, Washington. “That’s why we think it may make you more exposed to respiratory infections.”
The drop in proinflammatory molecules had an inverse relationship with opiorphin, a peripheral tissue vasodilator thought to increase blood flow and improve oxygen delivery to the muscles during exercise. This may be an adaptive mechanism to improve gas exchange in response to greater oxygen demand.
But as with many adaptive mechanisms, this one may have an unintended consequence. Fewer proinflammatory molecules on patrol may leave you more vulnerable to infection. Plus, during intense exercise, people tend to breathe through their mouths, bypassing the nasal barriers and allowing more microbes – including viruses – to penetrate and deposit in the distal airways of the lungs.
Advice for patients
More research is needed to know exactly how long and how strenuously one needs to exercise to trigger these immune changes, Dr. Nakayasu said.
As shown by their lactate accumulation (an indicator of anaerobic metabolism), the firefighters in the study outpaced the average person’s aerobic respiratory capacity, meaning the average person doing moderate exercise likely wouldn’t trigger these changes.
“Regular moderate exercise is generally associated with better health outcomes [and] improved immune function,” said Dr. Mamoun. For those who exercise to the extreme, proper rest and recovery are “essential for maintaining a robust immune system,” Dr. Mamoun said.
And of course, you can encourage patients to get vaccinated. Young, healthy patients may assume they don’t need COVID-19 or flu shots, as indicated by a recent survey that found one-third of Americans feel they don’t need these vaccinations if they’re not high risk.
A version of this article first appeared on Medscape.com.
Can too much of a healthy habit become bad?
Lots of evidence shows that regular exercise wards off respiratory infections such as colds, flu, and COVID-19. However, according to a new study.
The findings come as we enter another possible tripledemic this winter, with an increase in COVID, flu, and respiratory syncytial virus (RSV). Public health officials are on alert for a potentially severe flu season, following high flu activity this year in Australia (which can help predict how bad the U.S. flu season will be).
Studies show that the risk for acute respiratory infections is lower in people who exercise regularly. Physically active people are also less likely to suffer severe outcomes from COVID.
But while inactivity has emerged as a potential risk factor for respiratory infections, scientists have long proposed that too much activity, particularly of a prolonged and highly intense nature, may also increase susceptibility.
“The theory suggests that a short-term suppression of the immune system following intense exercise leads to an increase in susceptibility to infection, especially upper respiratory illness,” said Choukri Ben Mamoun, PhD, professor of medicine (infectious diseases) and microbial pathogenesis at the Yale Institute for Global Health, New Haven, Conn. Researchers have documented a greater incidence of upper respiratory illness “among both highly trained and healthy untrained individuals following increased activity during competition or heaving training blocks.”
That’s important if you treat athletes or patients with physically demanding jobs that push them to their physical limits, such as firefighters, police officers, or military personnel.
The new study was small but sheds light on a possible mechanism. Researchers tested blood, saliva, and urine samples from 11 firefighters before and 10 minutes after intense exercise designed to mimic wildfire fighting. The firefighters hiked over hilly terrain for 45 minutes in humid weather wearing up to 44 pounds of wildland gear.
After the workout, subjects had fewer proinflammatory cytokines and ceramides, and more antimicrobial peptides, changes that indicate a greater susceptibility to infection, researchers said. A systematic review adds weight to their findings, revealing a handful of studies in marathon runners, firefighters, soldiers, and soccer players that found an increase in respiratory symptoms after strenuous workouts.
“The relationship between exercise and the immune system is complex and varies from person to person,” said Dr. Mamoun, who was not part of the study. “Physicians can use this study’s findings to provide individualized exercise recommendations.”
An adaptive mechanism gone awry
During intense exercise, the body may reduce airway inflammation to help you breathe, say the authors. The boost in antimicrobial peptides found in the saliva samples could be the body’s way of compensating for the diminished immune function.
Antimicrobial peptides are part of the immune response but they’re “usually not very effective for viral infections,” said lead author Ernesto Nakayasu, PhD, senior research scientist at the Pacific Northwest National Laboratory, a U.S. Department of Energy lab in Richland, Washington. “That’s why we think it may make you more exposed to respiratory infections.”
The drop in proinflammatory molecules had an inverse relationship with opiorphin, a peripheral tissue vasodilator thought to increase blood flow and improve oxygen delivery to the muscles during exercise. This may be an adaptive mechanism to improve gas exchange in response to greater oxygen demand.
But as with many adaptive mechanisms, this one may have an unintended consequence. Fewer proinflammatory molecules on patrol may leave you more vulnerable to infection. Plus, during intense exercise, people tend to breathe through their mouths, bypassing the nasal barriers and allowing more microbes – including viruses – to penetrate and deposit in the distal airways of the lungs.
Advice for patients
More research is needed to know exactly how long and how strenuously one needs to exercise to trigger these immune changes, Dr. Nakayasu said.
As shown by their lactate accumulation (an indicator of anaerobic metabolism), the firefighters in the study outpaced the average person’s aerobic respiratory capacity, meaning the average person doing moderate exercise likely wouldn’t trigger these changes.
“Regular moderate exercise is generally associated with better health outcomes [and] improved immune function,” said Dr. Mamoun. For those who exercise to the extreme, proper rest and recovery are “essential for maintaining a robust immune system,” Dr. Mamoun said.
And of course, you can encourage patients to get vaccinated. Young, healthy patients may assume they don’t need COVID-19 or flu shots, as indicated by a recent survey that found one-third of Americans feel they don’t need these vaccinations if they’re not high risk.
A version of this article first appeared on Medscape.com.
FROM MILITARY MEDICAL RESEARCH
Telitacicept shows efficacy, safety in methotrexate-resistant RA
SAN DIEGO – A new randomized, double-blind, phase 3 study of the first-in-class drug telitacicept showed efficacy and safety in patients aged 18-65 with rheumatoid arthritis who were resistant to methotrexate, researchers reported at the annual meeting of the American College of Rheumatology.
Telitacicept is a recombinant fusion protein that aims to target and neutralize B lymphocyte stimulator (also known as B-cell activating factor) and a proliferation-inducing ligand (APRIL), both of which are linked to autoimmunity. Per the industry-funded trial, patients who took 160 mg weekly of the drug versus placebo for 24 weeks met the primary endpoint of achieving at least a 20% improvement in ACR response criteria (60.0% vs. 26.9%, respectively, P < .001), researcher Qing Zuraw, MD, MPH, MBA, of drug developer RemeGen, said in her presentation.
Rheumatologist Jeffrey A. Sparks MD, MMSc, of Brigham and Women’s Hospital and Harvard Medical School, both in Boston, who did not take part in the study but is familiar with the findings, said in an interview that the results are promising. “The magnitude of effect is impressive, and it seems it was well tolerated with relatively few serious adverse events. I would want to see this replicated in other patient populations. Also, more data are needed to truly establish safety.”
He added that, “once more data emerges, there would be a debate on where to place this in the treatment lines.”
According to Dr. Sparks, nearly half of patients with RA either cannot tolerate methotrexate or have partial or no response. “There are currently several available treatment options. These patients typically use either [tumor necrosis factor] inhibitors, another targeted medication, or combination therapy.”
Telitacicept is approved in China to treat systemic lupus erythematosus, Dr. Zuraw noted.
The researchers randomly assigned patients with moderate to severe RA at a 3:1 ratio to the drug (n = 360) or placebo (n = 119). The participants were aged 18-65 with a mean age of 49-50 depending on group, and 81.1%-87.4% females depending on group. Ethnicity/race was not reported.
At week 24, patients in the telitacicept group were more likely to achieve an ACR 50 response versus placebo (21.4% vs. 5.9%, respectively; P < .001).
“Significantly more patients in the telitacicept 160 mg group showed no radiographic progression (change in modified Total Sharp Score [mTSS] ≤ 0) at week 24, compared with placebo (90.2% vs. 66.4%; P < .001),” the researchers reported. “Additionally, patients in the telitacicept 160-mg group showed significantly less progression of joint damage (as measured by mTSS, joint space narrowing score, erosion score) from baseline to week 24.”
Treatment-emergent adverse effect levels were similar between the two groups (79.7% in the drug group and 77.3% in the placebo group), as were serious adverse events (6.4% and 6.7%, respectively) and infections/infestations (41.1% and 42.0%, respectively).
Dr. Zuraw noted limitations: The trial lacked a comparator treatment, and the study took place in a Chinese population with limited racial diversity.
In a Q&A session following Dr. Zuraw’s presentation, an audience member offered some perspective instead of a question by noting that most biologic drugs reach an ACR 20 response rate of about 60%, and most fail to move patients to low disease activity. “That’s the case for your product,” he told Dr. Zuraw. “That’s true for basically everything.”
He added that failing to discuss clinical importance is “a flaw of many talks.”
Dr. Zuraw responded that the many drugs do indeed have a response rate in the range of 60%, and she said the company will consider providing information about clinical impact in future talks.
No information is available about the potential cost of the drug. “This targets two proteins, so it is possible that it could have additional costs than typical biologics,” Dr. Sparks said.
RemeGen funded the study. Dr. Zuraw and some other authors disclosed financial relationships with RemeGen. Dr. Sparks reported receiving support from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the R. Bruce and Joan M. Mickey Research Scholar Fund, and the Gordon and Llura Gund Foundation; research support from Bristol-Myers Squibb; and consulting for AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Gilead, Inova, Janssen, Optum, Pfizer, Recor, Sobi, and UCB.
SAN DIEGO – A new randomized, double-blind, phase 3 study of the first-in-class drug telitacicept showed efficacy and safety in patients aged 18-65 with rheumatoid arthritis who were resistant to methotrexate, researchers reported at the annual meeting of the American College of Rheumatology.
Telitacicept is a recombinant fusion protein that aims to target and neutralize B lymphocyte stimulator (also known as B-cell activating factor) and a proliferation-inducing ligand (APRIL), both of which are linked to autoimmunity. Per the industry-funded trial, patients who took 160 mg weekly of the drug versus placebo for 24 weeks met the primary endpoint of achieving at least a 20% improvement in ACR response criteria (60.0% vs. 26.9%, respectively, P < .001), researcher Qing Zuraw, MD, MPH, MBA, of drug developer RemeGen, said in her presentation.
Rheumatologist Jeffrey A. Sparks MD, MMSc, of Brigham and Women’s Hospital and Harvard Medical School, both in Boston, who did not take part in the study but is familiar with the findings, said in an interview that the results are promising. “The magnitude of effect is impressive, and it seems it was well tolerated with relatively few serious adverse events. I would want to see this replicated in other patient populations. Also, more data are needed to truly establish safety.”
He added that, “once more data emerges, there would be a debate on where to place this in the treatment lines.”
According to Dr. Sparks, nearly half of patients with RA either cannot tolerate methotrexate or have partial or no response. “There are currently several available treatment options. These patients typically use either [tumor necrosis factor] inhibitors, another targeted medication, or combination therapy.”
Telitacicept is approved in China to treat systemic lupus erythematosus, Dr. Zuraw noted.
The researchers randomly assigned patients with moderate to severe RA at a 3:1 ratio to the drug (n = 360) or placebo (n = 119). The participants were aged 18-65 with a mean age of 49-50 depending on group, and 81.1%-87.4% females depending on group. Ethnicity/race was not reported.
At week 24, patients in the telitacicept group were more likely to achieve an ACR 50 response versus placebo (21.4% vs. 5.9%, respectively; P < .001).
“Significantly more patients in the telitacicept 160 mg group showed no radiographic progression (change in modified Total Sharp Score [mTSS] ≤ 0) at week 24, compared with placebo (90.2% vs. 66.4%; P < .001),” the researchers reported. “Additionally, patients in the telitacicept 160-mg group showed significantly less progression of joint damage (as measured by mTSS, joint space narrowing score, erosion score) from baseline to week 24.”
Treatment-emergent adverse effect levels were similar between the two groups (79.7% in the drug group and 77.3% in the placebo group), as were serious adverse events (6.4% and 6.7%, respectively) and infections/infestations (41.1% and 42.0%, respectively).
Dr. Zuraw noted limitations: The trial lacked a comparator treatment, and the study took place in a Chinese population with limited racial diversity.
In a Q&A session following Dr. Zuraw’s presentation, an audience member offered some perspective instead of a question by noting that most biologic drugs reach an ACR 20 response rate of about 60%, and most fail to move patients to low disease activity. “That’s the case for your product,” he told Dr. Zuraw. “That’s true for basically everything.”
He added that failing to discuss clinical importance is “a flaw of many talks.”
Dr. Zuraw responded that the many drugs do indeed have a response rate in the range of 60%, and she said the company will consider providing information about clinical impact in future talks.
No information is available about the potential cost of the drug. “This targets two proteins, so it is possible that it could have additional costs than typical biologics,” Dr. Sparks said.
RemeGen funded the study. Dr. Zuraw and some other authors disclosed financial relationships with RemeGen. Dr. Sparks reported receiving support from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the R. Bruce and Joan M. Mickey Research Scholar Fund, and the Gordon and Llura Gund Foundation; research support from Bristol-Myers Squibb; and consulting for AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Gilead, Inova, Janssen, Optum, Pfizer, Recor, Sobi, and UCB.
SAN DIEGO – A new randomized, double-blind, phase 3 study of the first-in-class drug telitacicept showed efficacy and safety in patients aged 18-65 with rheumatoid arthritis who were resistant to methotrexate, researchers reported at the annual meeting of the American College of Rheumatology.
Telitacicept is a recombinant fusion protein that aims to target and neutralize B lymphocyte stimulator (also known as B-cell activating factor) and a proliferation-inducing ligand (APRIL), both of which are linked to autoimmunity. Per the industry-funded trial, patients who took 160 mg weekly of the drug versus placebo for 24 weeks met the primary endpoint of achieving at least a 20% improvement in ACR response criteria (60.0% vs. 26.9%, respectively, P < .001), researcher Qing Zuraw, MD, MPH, MBA, of drug developer RemeGen, said in her presentation.
Rheumatologist Jeffrey A. Sparks MD, MMSc, of Brigham and Women’s Hospital and Harvard Medical School, both in Boston, who did not take part in the study but is familiar with the findings, said in an interview that the results are promising. “The magnitude of effect is impressive, and it seems it was well tolerated with relatively few serious adverse events. I would want to see this replicated in other patient populations. Also, more data are needed to truly establish safety.”
He added that, “once more data emerges, there would be a debate on where to place this in the treatment lines.”
According to Dr. Sparks, nearly half of patients with RA either cannot tolerate methotrexate or have partial or no response. “There are currently several available treatment options. These patients typically use either [tumor necrosis factor] inhibitors, another targeted medication, or combination therapy.”
Telitacicept is approved in China to treat systemic lupus erythematosus, Dr. Zuraw noted.
The researchers randomly assigned patients with moderate to severe RA at a 3:1 ratio to the drug (n = 360) or placebo (n = 119). The participants were aged 18-65 with a mean age of 49-50 depending on group, and 81.1%-87.4% females depending on group. Ethnicity/race was not reported.
At week 24, patients in the telitacicept group were more likely to achieve an ACR 50 response versus placebo (21.4% vs. 5.9%, respectively; P < .001).
“Significantly more patients in the telitacicept 160 mg group showed no radiographic progression (change in modified Total Sharp Score [mTSS] ≤ 0) at week 24, compared with placebo (90.2% vs. 66.4%; P < .001),” the researchers reported. “Additionally, patients in the telitacicept 160-mg group showed significantly less progression of joint damage (as measured by mTSS, joint space narrowing score, erosion score) from baseline to week 24.”
Treatment-emergent adverse effect levels were similar between the two groups (79.7% in the drug group and 77.3% in the placebo group), as were serious adverse events (6.4% and 6.7%, respectively) and infections/infestations (41.1% and 42.0%, respectively).
Dr. Zuraw noted limitations: The trial lacked a comparator treatment, and the study took place in a Chinese population with limited racial diversity.
In a Q&A session following Dr. Zuraw’s presentation, an audience member offered some perspective instead of a question by noting that most biologic drugs reach an ACR 20 response rate of about 60%, and most fail to move patients to low disease activity. “That’s the case for your product,” he told Dr. Zuraw. “That’s true for basically everything.”
He added that failing to discuss clinical importance is “a flaw of many talks.”
Dr. Zuraw responded that the many drugs do indeed have a response rate in the range of 60%, and she said the company will consider providing information about clinical impact in future talks.
No information is available about the potential cost of the drug. “This targets two proteins, so it is possible that it could have additional costs than typical biologics,” Dr. Sparks said.
RemeGen funded the study. Dr. Zuraw and some other authors disclosed financial relationships with RemeGen. Dr. Sparks reported receiving support from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the R. Bruce and Joan M. Mickey Research Scholar Fund, and the Gordon and Llura Gund Foundation; research support from Bristol-Myers Squibb; and consulting for AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Gilead, Inova, Janssen, Optum, Pfizer, Recor, Sobi, and UCB.
AT ACR 2023
At-home blood pressure monitoring is cost effective over long term
Despite higher upfront costs, enhanced home blood pressure monitoring by hypertension patients could be cost-effective compared with standard clinical care over the longer term, a systematic review in JAMA Network Open found.
In an analysis of 16 studies, at-home blood pressure (HBPM) monitoring, particularly using automatic 24-hour continuous measurements alone or combined with additional support or team-based care, appeared to be economical over a minimum 10-year period compared with usual care – higher expenditures for equipment and training notwithstanding.
“Our findings suggest that clinicians, hospitals, health care systems, third-party payers, and other stakeholders should consider the long-term incremental benefits and improvements in patients’ blood pressure, quality of life, and reductions in adverse outcomes,” wrote Michelle A. Hayek, of the Population Informatics Lab, department of industrial and systems engineering, at Texas A&M University, College Station, Tex., and colleagues.
HBPM increased considerably during the COVID pandemic and is expected to increase further in the next decade, according to lead author Theodoros Giannouchos, PhD, MS, MPharm, assistant professor in the department of health policy and organization in the School of Public Health at the University of Alabama at Birmingham. “Because home blood pressure monitoring might add costs to insurers, patients, and the health care system – at least short term – we noticed a gap in the updated literature on whether this method is cost-effective relative to in-office monitoring. Hence, we conducted this review.”
Six of the 16 studies were conducted in the United States and six in the United Kingdom; 14 used a health care insurance system perspective to determine costs. In nearly half, quality-adjusted life-years gained and cost per 1–mm Hg reduction in blood pressure were used as outcomes.
Self-monitoring included self measurements transmitted to health care professionals and involved either periodic readings, such as twice each morning and evening during the first week of every month, 3 times per week, or 24-hour ambulatory readings with a portable device every 20 or 30 minutes. Among studies comparing HBPM alone versus 24-hour ambulatory BP monitoring (ABPM) or HBPM combined with additional support or team-based care, the latter two approaches were more cost effective. The benefits would appear to offset the costs of more resource-intensive at-home self-monitoring methods over office care and traditional at-home monitoring only.
In addition, the authors noted, ABPM in particular might detect elevated in-office, or white-coat hypertension, and masked hypertension, the latter referring to normal BP readings measured in the office but actual elevated pressures in the everyday home setting. An estimated 17.1 million adults in the United States have masked hypertension, and the authors say the new approach would allow early tailored interventions to mitigate the risk of masked hypertension or prevent unnecessary treatment because of white-coat hypertension. “Because of the growing market in blood pressure monitors, the technology and accuracy of monitors is expected to improve even more,” Dr. Giannouchos said. “If these technologies are properly used, they can improve patients’ quality of life and health outcomes at a justified level of cost.”
The findings align with previous research that synthesized costs and benefits of self-monitoring methods across various diseases and settings.
“Future work is needed to compare these alternatives directly from a cost-effectiveness standpoint and to provide clinicians, stakeholders, and patients with more evidence to prioritize specific home-based BP programs,” the authors wrote.
This research was supported by the Texas A&M President’s Office X-grant initiative, National Science Foundation PATHS-UP, and Population Informatics Lab. A study coauthor reported grants from National Science Foundation during the conduct of the study.
Despite higher upfront costs, enhanced home blood pressure monitoring by hypertension patients could be cost-effective compared with standard clinical care over the longer term, a systematic review in JAMA Network Open found.
In an analysis of 16 studies, at-home blood pressure (HBPM) monitoring, particularly using automatic 24-hour continuous measurements alone or combined with additional support or team-based care, appeared to be economical over a minimum 10-year period compared with usual care – higher expenditures for equipment and training notwithstanding.
“Our findings suggest that clinicians, hospitals, health care systems, third-party payers, and other stakeholders should consider the long-term incremental benefits and improvements in patients’ blood pressure, quality of life, and reductions in adverse outcomes,” wrote Michelle A. Hayek, of the Population Informatics Lab, department of industrial and systems engineering, at Texas A&M University, College Station, Tex., and colleagues.
HBPM increased considerably during the COVID pandemic and is expected to increase further in the next decade, according to lead author Theodoros Giannouchos, PhD, MS, MPharm, assistant professor in the department of health policy and organization in the School of Public Health at the University of Alabama at Birmingham. “Because home blood pressure monitoring might add costs to insurers, patients, and the health care system – at least short term – we noticed a gap in the updated literature on whether this method is cost-effective relative to in-office monitoring. Hence, we conducted this review.”
Six of the 16 studies were conducted in the United States and six in the United Kingdom; 14 used a health care insurance system perspective to determine costs. In nearly half, quality-adjusted life-years gained and cost per 1–mm Hg reduction in blood pressure were used as outcomes.
Self-monitoring included self measurements transmitted to health care professionals and involved either periodic readings, such as twice each morning and evening during the first week of every month, 3 times per week, or 24-hour ambulatory readings with a portable device every 20 or 30 minutes. Among studies comparing HBPM alone versus 24-hour ambulatory BP monitoring (ABPM) or HBPM combined with additional support or team-based care, the latter two approaches were more cost effective. The benefits would appear to offset the costs of more resource-intensive at-home self-monitoring methods over office care and traditional at-home monitoring only.
In addition, the authors noted, ABPM in particular might detect elevated in-office, or white-coat hypertension, and masked hypertension, the latter referring to normal BP readings measured in the office but actual elevated pressures in the everyday home setting. An estimated 17.1 million adults in the United States have masked hypertension, and the authors say the new approach would allow early tailored interventions to mitigate the risk of masked hypertension or prevent unnecessary treatment because of white-coat hypertension. “Because of the growing market in blood pressure monitors, the technology and accuracy of monitors is expected to improve even more,” Dr. Giannouchos said. “If these technologies are properly used, they can improve patients’ quality of life and health outcomes at a justified level of cost.”
The findings align with previous research that synthesized costs and benefits of self-monitoring methods across various diseases and settings.
“Future work is needed to compare these alternatives directly from a cost-effectiveness standpoint and to provide clinicians, stakeholders, and patients with more evidence to prioritize specific home-based BP programs,” the authors wrote.
This research was supported by the Texas A&M President’s Office X-grant initiative, National Science Foundation PATHS-UP, and Population Informatics Lab. A study coauthor reported grants from National Science Foundation during the conduct of the study.
Despite higher upfront costs, enhanced home blood pressure monitoring by hypertension patients could be cost-effective compared with standard clinical care over the longer term, a systematic review in JAMA Network Open found.
In an analysis of 16 studies, at-home blood pressure (HBPM) monitoring, particularly using automatic 24-hour continuous measurements alone or combined with additional support or team-based care, appeared to be economical over a minimum 10-year period compared with usual care – higher expenditures for equipment and training notwithstanding.
“Our findings suggest that clinicians, hospitals, health care systems, third-party payers, and other stakeholders should consider the long-term incremental benefits and improvements in patients’ blood pressure, quality of life, and reductions in adverse outcomes,” wrote Michelle A. Hayek, of the Population Informatics Lab, department of industrial and systems engineering, at Texas A&M University, College Station, Tex., and colleagues.
HBPM increased considerably during the COVID pandemic and is expected to increase further in the next decade, according to lead author Theodoros Giannouchos, PhD, MS, MPharm, assistant professor in the department of health policy and organization in the School of Public Health at the University of Alabama at Birmingham. “Because home blood pressure monitoring might add costs to insurers, patients, and the health care system – at least short term – we noticed a gap in the updated literature on whether this method is cost-effective relative to in-office monitoring. Hence, we conducted this review.”
Six of the 16 studies were conducted in the United States and six in the United Kingdom; 14 used a health care insurance system perspective to determine costs. In nearly half, quality-adjusted life-years gained and cost per 1–mm Hg reduction in blood pressure were used as outcomes.
Self-monitoring included self measurements transmitted to health care professionals and involved either periodic readings, such as twice each morning and evening during the first week of every month, 3 times per week, or 24-hour ambulatory readings with a portable device every 20 or 30 minutes. Among studies comparing HBPM alone versus 24-hour ambulatory BP monitoring (ABPM) or HBPM combined with additional support or team-based care, the latter two approaches were more cost effective. The benefits would appear to offset the costs of more resource-intensive at-home self-monitoring methods over office care and traditional at-home monitoring only.
In addition, the authors noted, ABPM in particular might detect elevated in-office, or white-coat hypertension, and masked hypertension, the latter referring to normal BP readings measured in the office but actual elevated pressures in the everyday home setting. An estimated 17.1 million adults in the United States have masked hypertension, and the authors say the new approach would allow early tailored interventions to mitigate the risk of masked hypertension or prevent unnecessary treatment because of white-coat hypertension. “Because of the growing market in blood pressure monitors, the technology and accuracy of monitors is expected to improve even more,” Dr. Giannouchos said. “If these technologies are properly used, they can improve patients’ quality of life and health outcomes at a justified level of cost.”
The findings align with previous research that synthesized costs and benefits of self-monitoring methods across various diseases and settings.
“Future work is needed to compare these alternatives directly from a cost-effectiveness standpoint and to provide clinicians, stakeholders, and patients with more evidence to prioritize specific home-based BP programs,” the authors wrote.
This research was supported by the Texas A&M President’s Office X-grant initiative, National Science Foundation PATHS-UP, and Population Informatics Lab. A study coauthor reported grants from National Science Foundation during the conduct of the study.
FROM JAMA NETWORK OPEN
Childhood immunization schedule includes new RSV, mpox, meningococcal, and pneumococcal vaccines
The immunization schedule for children and adolescents, summarized as an American Academy of Pediatrics policy statement in the journal Pediatrics, contains new entries for the monoclonal antibody immunization nirsevimab for respiratory syncytial virus in infants, the maternal RSV vaccine RSVpreF for pregnant people, the mpox vaccine for adolescents, the 2023-2024 COVID-19 vaccine, the 20-valent pneumococcal conjugate vaccine (PCV20), and the pentavalent meningococcal vaccine (MenACWY-TT/MenB-FHbp).
A number of immunizations have been deleted from the 2024 schedule, including the pentavalent meningococcal vaccine MenABCWY because of a discontinuation in its distribution in the United States, the bivalent mRNA COVID-19 vaccines, the diphtheria and tetanus toxoids adsorbed vaccine, the 13-valent pneumococcal conjugate vaccine (PCV13), and the pneumococcal polysaccharide vaccine (PPSV23).
The 2024 childhood and adolescent immunization schedule, also approved by the Centers for Disease Control and Prevention, American Academy of Family Physicians, American College of Obstetricians and Gynecologists, American College of Nurse-Midwives, American Academy of Physician Associates, and National Association of Pediatric Nurse Practitioners, is published each year based on current recommendations that have been approved for use by the Food and Drug Administration.
In a press release, the AAP said the CDC decided to publish the recommendations early to ensure health providers are able to administer immunizations and that they are covered by insurance. They also referenced CDC reports that found vaccination rates for kindergarteners have not bounced back since the beginning of the COVID-19 pandemic, and vaccine exemptions for the 2022-2023 school year were at an “all-time high.”
RSV
New to the schedule are the recently approved RSV monoclonal antibody nirsevimab for infants and the RSV vaccine RSVpreF for pregnant people. According to the CDC’s combined immunization schedule for 2024, the timing of the infant RSV immunization is heavily dependent upon when and whether a RSV vaccine was administered during pregnancy. The RSV vaccine should be routinely given between 32 weeks and 36 weeks of gestation between September and January in most of the United States with the caveat that either the maternal vaccine or the infant immunization is recommended.
Infants born between October and March in most of the United States are eligible for the RSV immunization within 14 days of birth if the pregnant parent did not receive an RSV vaccine during pregnancy, or if the parent received the vaccine in the 14 days prior to birth. For infants born between April and September RSV immunization is recommended prior to the start of RSV season.
The immunization is also recommended for infants who were hospitalized for conditions such as prematurity after birth between October and March, infants aged 8-19 months who are undergoing medical support related to prematurity, infants aged 8-19 months who are severely immunocompromised, and infants aged 9-19 months who are American Indian or Alaska Native, and infants undergoing cardiac surgery with cardiopulmonary bypass.
Mpox
Another new addition to the schedule is mpox, which is recommended for adolescents 18 years or older who are at risk for mpox infection, including gay, bisexual, nonbinary, transgender, or other individuals who have developed a sexually transmitted disease within the last 6 months, had more than one sexual partner, or engaged in sex in a commercial sex venue or public space with confirmed mpox transmission.
Currently, mpox vaccination during pregnancy is not recommended due to a lack of safety data on the vaccine during pregnancy; however, the CDC noted pregnant persons who have been exposed to any of the risk factors above may receive the vaccine.
COVID, influenza, pneumococcal vaccines
The COVID-19 vaccine recommendations were updated to reflect the 2023-2023 formulation of the vaccine. Unvaccinated children between 6 months and 4 years of age will now receive the 2023-2024 formula mRNA vaccines, which includes the two-dose Moderna vaccine and three-dose Pfizer vaccine for use in that age group. Children with a previous history of COVID-19 vaccination are eligible to receive an age-appropriate COVID-19 vaccine from the 2023-2024 formulation, and children between 5-11 years old and 12-18 years old can receive a single dose of an mRNA vaccine regardless of vaccine history; unvaccinated children 12-18 years old are also eligible to receive the two-dose Novavax vaccine.
For influenza, the schedule refers to the Advisory Committee on Immunization Practices recommendations released in August, with a note indicating that individuals with an egg allergy can receive another vaccine recommended for their age group without concerns for safety.
The pneumococcal vaccine recommendations have removed PCV13 completely, with updates on the PCV15, PCV20, and PPSV23 in sections on routine vaccination, catch-up vaccination, and special situations. The poliovirus section has also seen its catch-up section revised with a recommendation to complete a vaccination series in adolescents 18 years old known or suspected to have an incomplete series, and to count trivalent oral poliovirus vaccines and OPV administered before April 2016 toward U.S. vaccination requirements.
‘Timely and necessary’ changes
Michael Pichichero, MD, director of the Rochester (N.Y.) General Hospital Research Institute, said in an interview that the committee that developed the immunization schedule was thorough in its recommendations for children and adolescents.
“The additions are timely and necessary as the landscape of vaccines for children changes,” he said.
Bonnie M. Word, MD, director of the Houston Travel Medicine Clinic, said that the immunization schedule “sets the standard and provides clarification and uniformity for administration of all recommended vaccines for U.S. children.”
The U.S. immunization program “is one of the best success stories in medicine,” Dr. Wood said. She noted it is important for providers to become familiar with these vaccines and their indications “to provide advice and be able to respond to questions of parents and/or patients.
“Often patients spend more time with office staff than the physician. It is helpful to make sure everyone in the office understands the importance of and the rationale for immunizing, so families hear consistent messaging,” she said.
Dr. Pichichero and Dr. Word reported no relevant conflicts of interest.
The immunization schedule for children and adolescents, summarized as an American Academy of Pediatrics policy statement in the journal Pediatrics, contains new entries for the monoclonal antibody immunization nirsevimab for respiratory syncytial virus in infants, the maternal RSV vaccine RSVpreF for pregnant people, the mpox vaccine for adolescents, the 2023-2024 COVID-19 vaccine, the 20-valent pneumococcal conjugate vaccine (PCV20), and the pentavalent meningococcal vaccine (MenACWY-TT/MenB-FHbp).
A number of immunizations have been deleted from the 2024 schedule, including the pentavalent meningococcal vaccine MenABCWY because of a discontinuation in its distribution in the United States, the bivalent mRNA COVID-19 vaccines, the diphtheria and tetanus toxoids adsorbed vaccine, the 13-valent pneumococcal conjugate vaccine (PCV13), and the pneumococcal polysaccharide vaccine (PPSV23).
The 2024 childhood and adolescent immunization schedule, also approved by the Centers for Disease Control and Prevention, American Academy of Family Physicians, American College of Obstetricians and Gynecologists, American College of Nurse-Midwives, American Academy of Physician Associates, and National Association of Pediatric Nurse Practitioners, is published each year based on current recommendations that have been approved for use by the Food and Drug Administration.
In a press release, the AAP said the CDC decided to publish the recommendations early to ensure health providers are able to administer immunizations and that they are covered by insurance. They also referenced CDC reports that found vaccination rates for kindergarteners have not bounced back since the beginning of the COVID-19 pandemic, and vaccine exemptions for the 2022-2023 school year were at an “all-time high.”
RSV
New to the schedule are the recently approved RSV monoclonal antibody nirsevimab for infants and the RSV vaccine RSVpreF for pregnant people. According to the CDC’s combined immunization schedule for 2024, the timing of the infant RSV immunization is heavily dependent upon when and whether a RSV vaccine was administered during pregnancy. The RSV vaccine should be routinely given between 32 weeks and 36 weeks of gestation between September and January in most of the United States with the caveat that either the maternal vaccine or the infant immunization is recommended.
Infants born between October and March in most of the United States are eligible for the RSV immunization within 14 days of birth if the pregnant parent did not receive an RSV vaccine during pregnancy, or if the parent received the vaccine in the 14 days prior to birth. For infants born between April and September RSV immunization is recommended prior to the start of RSV season.
The immunization is also recommended for infants who were hospitalized for conditions such as prematurity after birth between October and March, infants aged 8-19 months who are undergoing medical support related to prematurity, infants aged 8-19 months who are severely immunocompromised, and infants aged 9-19 months who are American Indian or Alaska Native, and infants undergoing cardiac surgery with cardiopulmonary bypass.
Mpox
Another new addition to the schedule is mpox, which is recommended for adolescents 18 years or older who are at risk for mpox infection, including gay, bisexual, nonbinary, transgender, or other individuals who have developed a sexually transmitted disease within the last 6 months, had more than one sexual partner, or engaged in sex in a commercial sex venue or public space with confirmed mpox transmission.
Currently, mpox vaccination during pregnancy is not recommended due to a lack of safety data on the vaccine during pregnancy; however, the CDC noted pregnant persons who have been exposed to any of the risk factors above may receive the vaccine.
COVID, influenza, pneumococcal vaccines
The COVID-19 vaccine recommendations were updated to reflect the 2023-2023 formulation of the vaccine. Unvaccinated children between 6 months and 4 years of age will now receive the 2023-2024 formula mRNA vaccines, which includes the two-dose Moderna vaccine and three-dose Pfizer vaccine for use in that age group. Children with a previous history of COVID-19 vaccination are eligible to receive an age-appropriate COVID-19 vaccine from the 2023-2024 formulation, and children between 5-11 years old and 12-18 years old can receive a single dose of an mRNA vaccine regardless of vaccine history; unvaccinated children 12-18 years old are also eligible to receive the two-dose Novavax vaccine.
For influenza, the schedule refers to the Advisory Committee on Immunization Practices recommendations released in August, with a note indicating that individuals with an egg allergy can receive another vaccine recommended for their age group without concerns for safety.
The pneumococcal vaccine recommendations have removed PCV13 completely, with updates on the PCV15, PCV20, and PPSV23 in sections on routine vaccination, catch-up vaccination, and special situations. The poliovirus section has also seen its catch-up section revised with a recommendation to complete a vaccination series in adolescents 18 years old known or suspected to have an incomplete series, and to count trivalent oral poliovirus vaccines and OPV administered before April 2016 toward U.S. vaccination requirements.
‘Timely and necessary’ changes
Michael Pichichero, MD, director of the Rochester (N.Y.) General Hospital Research Institute, said in an interview that the committee that developed the immunization schedule was thorough in its recommendations for children and adolescents.
“The additions are timely and necessary as the landscape of vaccines for children changes,” he said.
Bonnie M. Word, MD, director of the Houston Travel Medicine Clinic, said that the immunization schedule “sets the standard and provides clarification and uniformity for administration of all recommended vaccines for U.S. children.”
The U.S. immunization program “is one of the best success stories in medicine,” Dr. Wood said. She noted it is important for providers to become familiar with these vaccines and their indications “to provide advice and be able to respond to questions of parents and/or patients.
“Often patients spend more time with office staff than the physician. It is helpful to make sure everyone in the office understands the importance of and the rationale for immunizing, so families hear consistent messaging,” she said.
Dr. Pichichero and Dr. Word reported no relevant conflicts of interest.
The immunization schedule for children and adolescents, summarized as an American Academy of Pediatrics policy statement in the journal Pediatrics, contains new entries for the monoclonal antibody immunization nirsevimab for respiratory syncytial virus in infants, the maternal RSV vaccine RSVpreF for pregnant people, the mpox vaccine for adolescents, the 2023-2024 COVID-19 vaccine, the 20-valent pneumococcal conjugate vaccine (PCV20), and the pentavalent meningococcal vaccine (MenACWY-TT/MenB-FHbp).
A number of immunizations have been deleted from the 2024 schedule, including the pentavalent meningococcal vaccine MenABCWY because of a discontinuation in its distribution in the United States, the bivalent mRNA COVID-19 vaccines, the diphtheria and tetanus toxoids adsorbed vaccine, the 13-valent pneumococcal conjugate vaccine (PCV13), and the pneumococcal polysaccharide vaccine (PPSV23).
The 2024 childhood and adolescent immunization schedule, also approved by the Centers for Disease Control and Prevention, American Academy of Family Physicians, American College of Obstetricians and Gynecologists, American College of Nurse-Midwives, American Academy of Physician Associates, and National Association of Pediatric Nurse Practitioners, is published each year based on current recommendations that have been approved for use by the Food and Drug Administration.
In a press release, the AAP said the CDC decided to publish the recommendations early to ensure health providers are able to administer immunizations and that they are covered by insurance. They also referenced CDC reports that found vaccination rates for kindergarteners have not bounced back since the beginning of the COVID-19 pandemic, and vaccine exemptions for the 2022-2023 school year were at an “all-time high.”
RSV
New to the schedule are the recently approved RSV monoclonal antibody nirsevimab for infants and the RSV vaccine RSVpreF for pregnant people. According to the CDC’s combined immunization schedule for 2024, the timing of the infant RSV immunization is heavily dependent upon when and whether a RSV vaccine was administered during pregnancy. The RSV vaccine should be routinely given between 32 weeks and 36 weeks of gestation between September and January in most of the United States with the caveat that either the maternal vaccine or the infant immunization is recommended.
Infants born between October and March in most of the United States are eligible for the RSV immunization within 14 days of birth if the pregnant parent did not receive an RSV vaccine during pregnancy, or if the parent received the vaccine in the 14 days prior to birth. For infants born between April and September RSV immunization is recommended prior to the start of RSV season.
The immunization is also recommended for infants who were hospitalized for conditions such as prematurity after birth between October and March, infants aged 8-19 months who are undergoing medical support related to prematurity, infants aged 8-19 months who are severely immunocompromised, and infants aged 9-19 months who are American Indian or Alaska Native, and infants undergoing cardiac surgery with cardiopulmonary bypass.
Mpox
Another new addition to the schedule is mpox, which is recommended for adolescents 18 years or older who are at risk for mpox infection, including gay, bisexual, nonbinary, transgender, or other individuals who have developed a sexually transmitted disease within the last 6 months, had more than one sexual partner, or engaged in sex in a commercial sex venue or public space with confirmed mpox transmission.
Currently, mpox vaccination during pregnancy is not recommended due to a lack of safety data on the vaccine during pregnancy; however, the CDC noted pregnant persons who have been exposed to any of the risk factors above may receive the vaccine.
COVID, influenza, pneumococcal vaccines
The COVID-19 vaccine recommendations were updated to reflect the 2023-2023 formulation of the vaccine. Unvaccinated children between 6 months and 4 years of age will now receive the 2023-2024 formula mRNA vaccines, which includes the two-dose Moderna vaccine and three-dose Pfizer vaccine for use in that age group. Children with a previous history of COVID-19 vaccination are eligible to receive an age-appropriate COVID-19 vaccine from the 2023-2024 formulation, and children between 5-11 years old and 12-18 years old can receive a single dose of an mRNA vaccine regardless of vaccine history; unvaccinated children 12-18 years old are also eligible to receive the two-dose Novavax vaccine.
For influenza, the schedule refers to the Advisory Committee on Immunization Practices recommendations released in August, with a note indicating that individuals with an egg allergy can receive another vaccine recommended for their age group without concerns for safety.
The pneumococcal vaccine recommendations have removed PCV13 completely, with updates on the PCV15, PCV20, and PPSV23 in sections on routine vaccination, catch-up vaccination, and special situations. The poliovirus section has also seen its catch-up section revised with a recommendation to complete a vaccination series in adolescents 18 years old known or suspected to have an incomplete series, and to count trivalent oral poliovirus vaccines and OPV administered before April 2016 toward U.S. vaccination requirements.
‘Timely and necessary’ changes
Michael Pichichero, MD, director of the Rochester (N.Y.) General Hospital Research Institute, said in an interview that the committee that developed the immunization schedule was thorough in its recommendations for children and adolescents.
“The additions are timely and necessary as the landscape of vaccines for children changes,” he said.
Bonnie M. Word, MD, director of the Houston Travel Medicine Clinic, said that the immunization schedule “sets the standard and provides clarification and uniformity for administration of all recommended vaccines for U.S. children.”
The U.S. immunization program “is one of the best success stories in medicine,” Dr. Wood said. She noted it is important for providers to become familiar with these vaccines and their indications “to provide advice and be able to respond to questions of parents and/or patients.
“Often patients spend more time with office staff than the physician. It is helpful to make sure everyone in the office understands the importance of and the rationale for immunizing, so families hear consistent messaging,” she said.
Dr. Pichichero and Dr. Word reported no relevant conflicts of interest.
FROM PEDIATRICS
Fatal and nonfatal injuries
I suspect that, like me, you were saddened, but maybe not shocked, to learn that firearm-related fatalities have recently surpassed motor vehicle–related fatalities as the leading cause of death among children. For those of us living in Maine, this revelation came at a particularly difficult time. The body of the presumed shooter in the Lewiston massacre was found less than 10 miles from where I am writing you this letter. There is a good chance he may have been a former patient of mine, but I no longer have access to my records to confirm that.
This reshuffling at the top of the list of mortality causes is just one example of the shifting trends that have occurred in pediatric fatality statistics. In a recent analysis of the Centers for Disease Control and Prevention statistics published in Pediatrics investigators discovered that while, in general, fatal injuries have increased over the study period (2011-2021) nonfatal injuries have decreased.
We should no longer be surprised to learn that firearm-related deaths increased more than 87%. Fatal drug poisoning was up 133% and suffocation-related deaths increased 12.5% over that 10-year period. Given this profile of fatalities, it shouldn’t surprise us that nonfatal injuries due to firearms, poisoning, and self-harm also increased.
However, nonfatal injuries in other broad categories decreased: falls were down 52.8%, overexertion 63%, struck by [something or someone] 47.3%, motor vehicle occupant 36.7%, and cut pierce 36.7%. Nonfatal drownings were unchanged.
Diverging trends
What are we to make of these diverging trends? I suspect that when it comes to both firearms and drug poisonings, both fatal and nonfatal, children are now living in an environment in which the sheer volume of guns and drugs have grown the point, and will continue to grow, that contact and its consequences will continue to increase until we reach a saturation point at some unpredictable point in the future. There still may be some opportunities to curb the flow of drugs. But, I am afraid when it comes to firearms, that ship has sailed. We may have a chance to curb assault weapons, but hand guns have become ubiquitous to the point that they will continue to be a threat to children.
The increase in self-harm injuries is clearly a reflection of the increase in pediatric and adolescent mental health disturbances, which in turn is a reflection of the gloom hanging over the population in general.
But, what’s going on with the decrease in nonfatal injuries caused by falls, overexertion, struck by, and cut pierce? Is this a bit of sunshine in an otherwise cloudy picture? The authors of the paper see it as a reflection of our “public health interventions targeting pediatric safety partnered with technological advancement and legislative requirements.” Maybe when we are talking about booster seats and other automotive safety advancements. But I’m not so sure we should be too vigorous as we pat ourselves on the back.
On the other hand, aren’t these decreases in injuries related to activity just more evidence of our increasingly sedentary pediatric population? Falling off the couch seldom creates an injury that generates an ED statistic. Myopia and obesity related to excess screen time doesn’t trigger data points in this study. Overexertion injuries are down. We already know the consequences of underexertion are up.
I’m not sure we need to cut back on our efforts at injury prevention but I worry that we may run the risk of discouraging healthy activity if we aren’t careful with our voices of caution.
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].
I suspect that, like me, you were saddened, but maybe not shocked, to learn that firearm-related fatalities have recently surpassed motor vehicle–related fatalities as the leading cause of death among children. For those of us living in Maine, this revelation came at a particularly difficult time. The body of the presumed shooter in the Lewiston massacre was found less than 10 miles from where I am writing you this letter. There is a good chance he may have been a former patient of mine, but I no longer have access to my records to confirm that.
This reshuffling at the top of the list of mortality causes is just one example of the shifting trends that have occurred in pediatric fatality statistics. In a recent analysis of the Centers for Disease Control and Prevention statistics published in Pediatrics investigators discovered that while, in general, fatal injuries have increased over the study period (2011-2021) nonfatal injuries have decreased.
We should no longer be surprised to learn that firearm-related deaths increased more than 87%. Fatal drug poisoning was up 133% and suffocation-related deaths increased 12.5% over that 10-year period. Given this profile of fatalities, it shouldn’t surprise us that nonfatal injuries due to firearms, poisoning, and self-harm also increased.
However, nonfatal injuries in other broad categories decreased: falls were down 52.8%, overexertion 63%, struck by [something or someone] 47.3%, motor vehicle occupant 36.7%, and cut pierce 36.7%. Nonfatal drownings were unchanged.
Diverging trends
What are we to make of these diverging trends? I suspect that when it comes to both firearms and drug poisonings, both fatal and nonfatal, children are now living in an environment in which the sheer volume of guns and drugs have grown the point, and will continue to grow, that contact and its consequences will continue to increase until we reach a saturation point at some unpredictable point in the future. There still may be some opportunities to curb the flow of drugs. But, I am afraid when it comes to firearms, that ship has sailed. We may have a chance to curb assault weapons, but hand guns have become ubiquitous to the point that they will continue to be a threat to children.
The increase in self-harm injuries is clearly a reflection of the increase in pediatric and adolescent mental health disturbances, which in turn is a reflection of the gloom hanging over the population in general.
But, what’s going on with the decrease in nonfatal injuries caused by falls, overexertion, struck by, and cut pierce? Is this a bit of sunshine in an otherwise cloudy picture? The authors of the paper see it as a reflection of our “public health interventions targeting pediatric safety partnered with technological advancement and legislative requirements.” Maybe when we are talking about booster seats and other automotive safety advancements. But I’m not so sure we should be too vigorous as we pat ourselves on the back.
On the other hand, aren’t these decreases in injuries related to activity just more evidence of our increasingly sedentary pediatric population? Falling off the couch seldom creates an injury that generates an ED statistic. Myopia and obesity related to excess screen time doesn’t trigger data points in this study. Overexertion injuries are down. We already know the consequences of underexertion are up.
I’m not sure we need to cut back on our efforts at injury prevention but I worry that we may run the risk of discouraging healthy activity if we aren’t careful with our voices of caution.
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].
I suspect that, like me, you were saddened, but maybe not shocked, to learn that firearm-related fatalities have recently surpassed motor vehicle–related fatalities as the leading cause of death among children. For those of us living in Maine, this revelation came at a particularly difficult time. The body of the presumed shooter in the Lewiston massacre was found less than 10 miles from where I am writing you this letter. There is a good chance he may have been a former patient of mine, but I no longer have access to my records to confirm that.
This reshuffling at the top of the list of mortality causes is just one example of the shifting trends that have occurred in pediatric fatality statistics. In a recent analysis of the Centers for Disease Control and Prevention statistics published in Pediatrics investigators discovered that while, in general, fatal injuries have increased over the study period (2011-2021) nonfatal injuries have decreased.
We should no longer be surprised to learn that firearm-related deaths increased more than 87%. Fatal drug poisoning was up 133% and suffocation-related deaths increased 12.5% over that 10-year period. Given this profile of fatalities, it shouldn’t surprise us that nonfatal injuries due to firearms, poisoning, and self-harm also increased.
However, nonfatal injuries in other broad categories decreased: falls were down 52.8%, overexertion 63%, struck by [something or someone] 47.3%, motor vehicle occupant 36.7%, and cut pierce 36.7%. Nonfatal drownings were unchanged.
Diverging trends
What are we to make of these diverging trends? I suspect that when it comes to both firearms and drug poisonings, both fatal and nonfatal, children are now living in an environment in which the sheer volume of guns and drugs have grown the point, and will continue to grow, that contact and its consequences will continue to increase until we reach a saturation point at some unpredictable point in the future. There still may be some opportunities to curb the flow of drugs. But, I am afraid when it comes to firearms, that ship has sailed. We may have a chance to curb assault weapons, but hand guns have become ubiquitous to the point that they will continue to be a threat to children.
The increase in self-harm injuries is clearly a reflection of the increase in pediatric and adolescent mental health disturbances, which in turn is a reflection of the gloom hanging over the population in general.
But, what’s going on with the decrease in nonfatal injuries caused by falls, overexertion, struck by, and cut pierce? Is this a bit of sunshine in an otherwise cloudy picture? The authors of the paper see it as a reflection of our “public health interventions targeting pediatric safety partnered with technological advancement and legislative requirements.” Maybe when we are talking about booster seats and other automotive safety advancements. But I’m not so sure we should be too vigorous as we pat ourselves on the back.
On the other hand, aren’t these decreases in injuries related to activity just more evidence of our increasingly sedentary pediatric population? Falling off the couch seldom creates an injury that generates an ED statistic. Myopia and obesity related to excess screen time doesn’t trigger data points in this study. Overexertion injuries are down. We already know the consequences of underexertion are up.
I’m not sure we need to cut back on our efforts at injury prevention but I worry that we may run the risk of discouraging healthy activity if we aren’t careful with our voices of caution.
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].
Staying in my lane
In one of my favorite fall rituals, I fired up my hot tub. In Phoenix the months for relaxing in one are limited, so I try to enjoy it when I can.
It’s a routine I know well. Remove the coverings it’s been hiding under for the 8-month Phoenix summer. Clean out the dust, dead bugs, leaves. Connect the pipes and hoses, tighten the clamps, and begin filling it with water. A pinch of water softener, a dash of chlorine, plug in the motor, and stir.
After a few minutes of running, however, I noticed water starting to come out from under the motor unit. That ain’t good. I checked the fittings to make sure everything was in place and tight. They were. So the problem was somewhere inside the motor.
I unplugged the hot tub and went to my computer. Using the ever-popular Google, I looked at diagrams of motor plumbing and wiring, then watched several DIY videos on YouTube.
After about an hour of this I got some screwdrivers, went back out to the hot tub, stared at it for a few minutes ... then I realized I was out of my league and called a repair service.
I’m a neurologist. I hope I’m a decent one. But I’m not a hot tub mechanic anymore than I am a neurosurgeon. Or, as Bones McCoy would have said, “Dammit, Jim, I’m a doctor, not a spa repairman.” Either way, I was out of my league and I knew it. My taking the motor apart – while certainly feasible – was more likely to make things worse.
Unfortunately, not everyone feels the same way. Although I spent quite a few years learning to be a neurologist, it’s amazing how many people feel that, after a similar time on Google, they’re qualified to diagnose and treat neurological (or any medical) conditions. They know what they have, what tests need to be done, and which medications should be prescribed. They only need me to rubber stamp their plan, since their insurance won’t take orders from them. (They often won’t take orders from me, either, but that’s another matter).
While occasionally they’re correct, most of the time I have to politely explain why I disagree with them, or why other possibilities have to be considered and treated. No one is forcing them to follow my plan, but if they aren’t happy with it, they’ll have to find another neurologist. I have to go with my education and experience over theirs. I mean, this is my job. I’ve been doing it for more than 25 years.
I’m sure if the hot tub mechanic showed up and I told him where and how to fix the problem he’d wonder what my qualifications were. I may be right, but I may be crazy (or at least wrong).
Unlike medicine, there’s nothing to keep me from trying to fix the hot tub myself, except my own awareness that I have no practical idea how to do so.
I try my best not to practice outside of my field. Google isn’t going to make me a cardiologist any more than it will let me fix hot tubs.
I’m all for knowledge, but it also has its limits. Why some patients think the Internet is a good substitute for 9 years of education and 25 years of ongoing experience, I have no idea.
Dr. Block has a solo neurology practice in Scottsdale, Ariz.
In one of my favorite fall rituals, I fired up my hot tub. In Phoenix the months for relaxing in one are limited, so I try to enjoy it when I can.
It’s a routine I know well. Remove the coverings it’s been hiding under for the 8-month Phoenix summer. Clean out the dust, dead bugs, leaves. Connect the pipes and hoses, tighten the clamps, and begin filling it with water. A pinch of water softener, a dash of chlorine, plug in the motor, and stir.
After a few minutes of running, however, I noticed water starting to come out from under the motor unit. That ain’t good. I checked the fittings to make sure everything was in place and tight. They were. So the problem was somewhere inside the motor.
I unplugged the hot tub and went to my computer. Using the ever-popular Google, I looked at diagrams of motor plumbing and wiring, then watched several DIY videos on YouTube.
After about an hour of this I got some screwdrivers, went back out to the hot tub, stared at it for a few minutes ... then I realized I was out of my league and called a repair service.
I’m a neurologist. I hope I’m a decent one. But I’m not a hot tub mechanic anymore than I am a neurosurgeon. Or, as Bones McCoy would have said, “Dammit, Jim, I’m a doctor, not a spa repairman.” Either way, I was out of my league and I knew it. My taking the motor apart – while certainly feasible – was more likely to make things worse.
Unfortunately, not everyone feels the same way. Although I spent quite a few years learning to be a neurologist, it’s amazing how many people feel that, after a similar time on Google, they’re qualified to diagnose and treat neurological (or any medical) conditions. They know what they have, what tests need to be done, and which medications should be prescribed. They only need me to rubber stamp their plan, since their insurance won’t take orders from them. (They often won’t take orders from me, either, but that’s another matter).
While occasionally they’re correct, most of the time I have to politely explain why I disagree with them, or why other possibilities have to be considered and treated. No one is forcing them to follow my plan, but if they aren’t happy with it, they’ll have to find another neurologist. I have to go with my education and experience over theirs. I mean, this is my job. I’ve been doing it for more than 25 years.
I’m sure if the hot tub mechanic showed up and I told him where and how to fix the problem he’d wonder what my qualifications were. I may be right, but I may be crazy (or at least wrong).
Unlike medicine, there’s nothing to keep me from trying to fix the hot tub myself, except my own awareness that I have no practical idea how to do so.
I try my best not to practice outside of my field. Google isn’t going to make me a cardiologist any more than it will let me fix hot tubs.
I’m all for knowledge, but it also has its limits. Why some patients think the Internet is a good substitute for 9 years of education and 25 years of ongoing experience, I have no idea.
Dr. Block has a solo neurology practice in Scottsdale, Ariz.
In one of my favorite fall rituals, I fired up my hot tub. In Phoenix the months for relaxing in one are limited, so I try to enjoy it when I can.
It’s a routine I know well. Remove the coverings it’s been hiding under for the 8-month Phoenix summer. Clean out the dust, dead bugs, leaves. Connect the pipes and hoses, tighten the clamps, and begin filling it with water. A pinch of water softener, a dash of chlorine, plug in the motor, and stir.
After a few minutes of running, however, I noticed water starting to come out from under the motor unit. That ain’t good. I checked the fittings to make sure everything was in place and tight. They were. So the problem was somewhere inside the motor.
I unplugged the hot tub and went to my computer. Using the ever-popular Google, I looked at diagrams of motor plumbing and wiring, then watched several DIY videos on YouTube.
After about an hour of this I got some screwdrivers, went back out to the hot tub, stared at it for a few minutes ... then I realized I was out of my league and called a repair service.
I’m a neurologist. I hope I’m a decent one. But I’m not a hot tub mechanic anymore than I am a neurosurgeon. Or, as Bones McCoy would have said, “Dammit, Jim, I’m a doctor, not a spa repairman.” Either way, I was out of my league and I knew it. My taking the motor apart – while certainly feasible – was more likely to make things worse.
Unfortunately, not everyone feels the same way. Although I spent quite a few years learning to be a neurologist, it’s amazing how many people feel that, after a similar time on Google, they’re qualified to diagnose and treat neurological (or any medical) conditions. They know what they have, what tests need to be done, and which medications should be prescribed. They only need me to rubber stamp their plan, since their insurance won’t take orders from them. (They often won’t take orders from me, either, but that’s another matter).
While occasionally they’re correct, most of the time I have to politely explain why I disagree with them, or why other possibilities have to be considered and treated. No one is forcing them to follow my plan, but if they aren’t happy with it, they’ll have to find another neurologist. I have to go with my education and experience over theirs. I mean, this is my job. I’ve been doing it for more than 25 years.
I’m sure if the hot tub mechanic showed up and I told him where and how to fix the problem he’d wonder what my qualifications were. I may be right, but I may be crazy (or at least wrong).
Unlike medicine, there’s nothing to keep me from trying to fix the hot tub myself, except my own awareness that I have no practical idea how to do so.
I try my best not to practice outside of my field. Google isn’t going to make me a cardiologist any more than it will let me fix hot tubs.
I’m all for knowledge, but it also has its limits. Why some patients think the Internet is a good substitute for 9 years of education and 25 years of ongoing experience, I have no idea.
Dr. Block has a solo neurology practice in Scottsdale, Ariz.
Parent concerns a factor when treating eczema in children with darker skin types
NEW YORK –
Skin diseases pose a greater risk of both hyper- and hypopigmentation in patients with darker skin types, but the fear and concern that this raises for permanent disfigurement is not limited to Blacks, Dr. Heath, assistant professor of pediatric dermatology at Temple University, Philadelphia, said at the Skin of Color Update 2023.
“Culturally, pigmentation changes can be huge. For people of Indian descent, for example, pigmentary changes like light spots on the skin might be an obstacle to marriage, so it can really be life changing,” she added.
In patients with darker skin tones presenting with an inflammatory skin disease, such as AD or psoriasis, Dr. Heath advised asking specifically about change in skin tone even if it is not readily apparent. In pediatric patients, it is also appropriate to include parents in this conversation.
Consider the parent’s perspective
“When you are taking care of a child or adolescent, the patient is likely to be concerned about changes in pigmentation, but it is important to remember that the adult in the room might have had their own journey with brown skin and has dealt with the burden of pigment changes,” Dr. Heath said.
For the parent, the pigmentation changes, rather than the inflammation, might be the governing issue and the reason that he or she brought the child to the clinician. Dr. Heath suggested that it is important for caregivers to explicitly recognize their concern, explain that addressing the pigmentary changes is part of the treatment plan, and to create realistic expectations about how long pigmentary changes will take to resolve.
As an example, Dr. Heath recounted a difficult case of a Black infant with disseminated hyperpigmentation and features that did not preclude pathology other than AD. Dr. Heath created a multifaceted treatment plan to address the inflammation in distinct areas of the body that included low-strength topical steroids for the face, stronger steroids for the body, and advice on scalp and skin care.
“I thought this was a great treatment plan out of the gate – I was covering all of the things on my differential list – I thought that the mom would be thinking, this doctor is amazing,” Dr. Heath said.
Pigmentary changes are a priority
However, that was not what the patient’s mother was thinking. Having failed to explicitly recognize her concern about the pigmentation changes and how the treatment would address this issue, the mother was disappointed.
“She had one question: Will my baby ever be one color? That was her main concern,” said Dr. Heath, indicating that other clinicians seeing inflammatory diseases in children with darker skin types can learn from her experience.
“Really, you have to acknowledge that the condition you are treating is causing the pigmentation change, and we do see that and that we have a treatment plan in place,” she said.
Because of differences in how inflammatory skin diseases present in darker skin types, there is plenty of room for a delayed diagnosis for clinicians who do not see many of these patients, according to Dr. Heath. Follicular eczema, which is common in skin of color, often presents with pruritus but differences in the appearance of the underlying disease can threaten a delay in diagnosis.
In cases of follicular eczema with itch in darker skin, the bumps look and feel like goose bumps, which “means that the eczema is really active and inflamed,” Dr. Heath said. When the skin becomes smooth and the itch dissipates, “you know that they are under great control.”
Psoriasis is often missed in children with darker skin types based on the misperception that it is rare. Although it is true that it is less common in Blacks than Whites, it is not rare, according to Dr. Heath. In inspecting the telltale erythematous plaque–like lesions, clinicians might start to consider alternative diagnoses when they do not detect the same erythematous appearance, but the reddish tone is often concealed in darker skin.
She said that predominant involvement in the head and neck and diaper area is often more common in children of color and that nail or scalp involvement, when present, is often a clue that psoriasis is the diagnosis.
Again, because many clinicians do not think immediately of psoriasis in darker skin children with lesions in the scalp, Dr. Heath advised this is another reason to include psoriasis in the differential diagnosis.
“If you have a child that has failed multiple courses of treatment for tinea capitis and they have well-demarcated plaques, it’s time to really start to think about pediatric psoriasis,” she said.
Restoring skin tone can be the priority
Asked to comment on Dr. Heath’s advice about the importance of acknowledging pigmentary changes associated with inflammatory skin diseases in patients of color, Jenna Lester, MD, the founding director of the Skin of Color Clinic at the University of California, San Francisco, called it an “often unspoken concern of patients.”
“Pigmentary changes that occur secondary to an inflammatory condition should be addressed and treated alongside the inciting condition,” she agreed.
Even if changes in skin color or skin tone are not a specific complaint of the patients, Dr. Lester also urged clinicians to raise the topic. If change in skin pigmentation is part of the clinical picture, this should be targeted in the treatment plan.
“In acne, for example, often times I find that patients are as worried about postinflammatory hyperpigmentation as they are about their acne,” she said, reiterating the advice provided by Dr. Heath.
Dr. Heath has financial relationships with Arcutis, Janssen, Johnson & Johnson, Lilly, and Regeneron. Dr. Lester reported no potential conflicts of interest.
NEW YORK –
Skin diseases pose a greater risk of both hyper- and hypopigmentation in patients with darker skin types, but the fear and concern that this raises for permanent disfigurement is not limited to Blacks, Dr. Heath, assistant professor of pediatric dermatology at Temple University, Philadelphia, said at the Skin of Color Update 2023.
“Culturally, pigmentation changes can be huge. For people of Indian descent, for example, pigmentary changes like light spots on the skin might be an obstacle to marriage, so it can really be life changing,” she added.
In patients with darker skin tones presenting with an inflammatory skin disease, such as AD or psoriasis, Dr. Heath advised asking specifically about change in skin tone even if it is not readily apparent. In pediatric patients, it is also appropriate to include parents in this conversation.
Consider the parent’s perspective
“When you are taking care of a child or adolescent, the patient is likely to be concerned about changes in pigmentation, but it is important to remember that the adult in the room might have had their own journey with brown skin and has dealt with the burden of pigment changes,” Dr. Heath said.
For the parent, the pigmentation changes, rather than the inflammation, might be the governing issue and the reason that he or she brought the child to the clinician. Dr. Heath suggested that it is important for caregivers to explicitly recognize their concern, explain that addressing the pigmentary changes is part of the treatment plan, and to create realistic expectations about how long pigmentary changes will take to resolve.
As an example, Dr. Heath recounted a difficult case of a Black infant with disseminated hyperpigmentation and features that did not preclude pathology other than AD. Dr. Heath created a multifaceted treatment plan to address the inflammation in distinct areas of the body that included low-strength topical steroids for the face, stronger steroids for the body, and advice on scalp and skin care.
“I thought this was a great treatment plan out of the gate – I was covering all of the things on my differential list – I thought that the mom would be thinking, this doctor is amazing,” Dr. Heath said.
Pigmentary changes are a priority
However, that was not what the patient’s mother was thinking. Having failed to explicitly recognize her concern about the pigmentation changes and how the treatment would address this issue, the mother was disappointed.
“She had one question: Will my baby ever be one color? That was her main concern,” said Dr. Heath, indicating that other clinicians seeing inflammatory diseases in children with darker skin types can learn from her experience.
“Really, you have to acknowledge that the condition you are treating is causing the pigmentation change, and we do see that and that we have a treatment plan in place,” she said.
Because of differences in how inflammatory skin diseases present in darker skin types, there is plenty of room for a delayed diagnosis for clinicians who do not see many of these patients, according to Dr. Heath. Follicular eczema, which is common in skin of color, often presents with pruritus but differences in the appearance of the underlying disease can threaten a delay in diagnosis.
In cases of follicular eczema with itch in darker skin, the bumps look and feel like goose bumps, which “means that the eczema is really active and inflamed,” Dr. Heath said. When the skin becomes smooth and the itch dissipates, “you know that they are under great control.”
Psoriasis is often missed in children with darker skin types based on the misperception that it is rare. Although it is true that it is less common in Blacks than Whites, it is not rare, according to Dr. Heath. In inspecting the telltale erythematous plaque–like lesions, clinicians might start to consider alternative diagnoses when they do not detect the same erythematous appearance, but the reddish tone is often concealed in darker skin.
She said that predominant involvement in the head and neck and diaper area is often more common in children of color and that nail or scalp involvement, when present, is often a clue that psoriasis is the diagnosis.
Again, because many clinicians do not think immediately of psoriasis in darker skin children with lesions in the scalp, Dr. Heath advised this is another reason to include psoriasis in the differential diagnosis.
“If you have a child that has failed multiple courses of treatment for tinea capitis and they have well-demarcated plaques, it’s time to really start to think about pediatric psoriasis,” she said.
Restoring skin tone can be the priority
Asked to comment on Dr. Heath’s advice about the importance of acknowledging pigmentary changes associated with inflammatory skin diseases in patients of color, Jenna Lester, MD, the founding director of the Skin of Color Clinic at the University of California, San Francisco, called it an “often unspoken concern of patients.”
“Pigmentary changes that occur secondary to an inflammatory condition should be addressed and treated alongside the inciting condition,” she agreed.
Even if changes in skin color or skin tone are not a specific complaint of the patients, Dr. Lester also urged clinicians to raise the topic. If change in skin pigmentation is part of the clinical picture, this should be targeted in the treatment plan.
“In acne, for example, often times I find that patients are as worried about postinflammatory hyperpigmentation as they are about their acne,” she said, reiterating the advice provided by Dr. Heath.
Dr. Heath has financial relationships with Arcutis, Janssen, Johnson & Johnson, Lilly, and Regeneron. Dr. Lester reported no potential conflicts of interest.
NEW YORK –
Skin diseases pose a greater risk of both hyper- and hypopigmentation in patients with darker skin types, but the fear and concern that this raises for permanent disfigurement is not limited to Blacks, Dr. Heath, assistant professor of pediatric dermatology at Temple University, Philadelphia, said at the Skin of Color Update 2023.
“Culturally, pigmentation changes can be huge. For people of Indian descent, for example, pigmentary changes like light spots on the skin might be an obstacle to marriage, so it can really be life changing,” she added.
In patients with darker skin tones presenting with an inflammatory skin disease, such as AD or psoriasis, Dr. Heath advised asking specifically about change in skin tone even if it is not readily apparent. In pediatric patients, it is also appropriate to include parents in this conversation.
Consider the parent’s perspective
“When you are taking care of a child or adolescent, the patient is likely to be concerned about changes in pigmentation, but it is important to remember that the adult in the room might have had their own journey with brown skin and has dealt with the burden of pigment changes,” Dr. Heath said.
For the parent, the pigmentation changes, rather than the inflammation, might be the governing issue and the reason that he or she brought the child to the clinician. Dr. Heath suggested that it is important for caregivers to explicitly recognize their concern, explain that addressing the pigmentary changes is part of the treatment plan, and to create realistic expectations about how long pigmentary changes will take to resolve.
As an example, Dr. Heath recounted a difficult case of a Black infant with disseminated hyperpigmentation and features that did not preclude pathology other than AD. Dr. Heath created a multifaceted treatment plan to address the inflammation in distinct areas of the body that included low-strength topical steroids for the face, stronger steroids for the body, and advice on scalp and skin care.
“I thought this was a great treatment plan out of the gate – I was covering all of the things on my differential list – I thought that the mom would be thinking, this doctor is amazing,” Dr. Heath said.
Pigmentary changes are a priority
However, that was not what the patient’s mother was thinking. Having failed to explicitly recognize her concern about the pigmentation changes and how the treatment would address this issue, the mother was disappointed.
“She had one question: Will my baby ever be one color? That was her main concern,” said Dr. Heath, indicating that other clinicians seeing inflammatory diseases in children with darker skin types can learn from her experience.
“Really, you have to acknowledge that the condition you are treating is causing the pigmentation change, and we do see that and that we have a treatment plan in place,” she said.
Because of differences in how inflammatory skin diseases present in darker skin types, there is plenty of room for a delayed diagnosis for clinicians who do not see many of these patients, according to Dr. Heath. Follicular eczema, which is common in skin of color, often presents with pruritus but differences in the appearance of the underlying disease can threaten a delay in diagnosis.
In cases of follicular eczema with itch in darker skin, the bumps look and feel like goose bumps, which “means that the eczema is really active and inflamed,” Dr. Heath said. When the skin becomes smooth and the itch dissipates, “you know that they are under great control.”
Psoriasis is often missed in children with darker skin types based on the misperception that it is rare. Although it is true that it is less common in Blacks than Whites, it is not rare, according to Dr. Heath. In inspecting the telltale erythematous plaque–like lesions, clinicians might start to consider alternative diagnoses when they do not detect the same erythematous appearance, but the reddish tone is often concealed in darker skin.
She said that predominant involvement in the head and neck and diaper area is often more common in children of color and that nail or scalp involvement, when present, is often a clue that psoriasis is the diagnosis.
Again, because many clinicians do not think immediately of psoriasis in darker skin children with lesions in the scalp, Dr. Heath advised this is another reason to include psoriasis in the differential diagnosis.
“If you have a child that has failed multiple courses of treatment for tinea capitis and they have well-demarcated plaques, it’s time to really start to think about pediatric psoriasis,” she said.
Restoring skin tone can be the priority
Asked to comment on Dr. Heath’s advice about the importance of acknowledging pigmentary changes associated with inflammatory skin diseases in patients of color, Jenna Lester, MD, the founding director of the Skin of Color Clinic at the University of California, San Francisco, called it an “often unspoken concern of patients.”
“Pigmentary changes that occur secondary to an inflammatory condition should be addressed and treated alongside the inciting condition,” she agreed.
Even if changes in skin color or skin tone are not a specific complaint of the patients, Dr. Lester also urged clinicians to raise the topic. If change in skin pigmentation is part of the clinical picture, this should be targeted in the treatment plan.
“In acne, for example, often times I find that patients are as worried about postinflammatory hyperpigmentation as they are about their acne,” she said, reiterating the advice provided by Dr. Heath.
Dr. Heath has financial relationships with Arcutis, Janssen, Johnson & Johnson, Lilly, and Regeneron. Dr. Lester reported no potential conflicts of interest.
AT SOC 2023
Redispensing unused cancer meds cuts waste, saves money
TOPLINE:
to save money and reduce waste, a Dutch study has found.
METHODOLOGY:
- Ongoing drug shortages and growing drug prices contribute to access issues in oncology.
- Researchers compared the reduction in drug waste and cost savings from redispensing oral anticancer drugs versus the standard practice of disposing of them.
- Outpatient pharmacies at four Dutch hospitals participated. A total of 1,071 patients with cancer receiving oral anticancer drugs for at-home use were given special packaging for returning unused medication to the pharmacy.
- The pharmacy ensured the quality of returned drugs based on authenticity, appearance, remaining shelf-life, and adequate storage temperature.
TAKEAWAY:
- A total of 13,069 oral anticancer drug packages, containing an average of 27 daily doses per package, were dispensed during the study period.
- Overall, 16% of patients (n = 171) returned 335 (2.6%) unused oral anticancer drug packages, of which 68% were redispensed after passing quality control.
- Redispensing unused oral anticancer drugs reduced waste by 68%, compared with disposing of them, and provided a mean net annual cost savings of €576 (U.S. $682) per patient per year.
- When just those patients who took targeted oral anticancer drugs for up to 24 months were looked at, the mean net annual cost savings associated with the quality check protocol increased to €934 (U.S. $1,019) per patient or of only the visual quality check was €1,348 (U.S. $1,474) per patient.
IN PRACTICE:
“New strategies targeting waste are required to improve financial and ecologic sustainability of expensive therapies, such as oral anticancer drugs, that frequently remain unused by patients,” the authors write. “These findings provide a waste-minimizing strategy to contribute to sustainable and affordable access to drugs.”
SOURCE:
The study, by Elisabeth M. Smale, PharmD, of Radboud University Medical Center, the Netherlands, and colleagues, was published online in JAMA Oncology.
LIMITATIONS:
Novel drugs are substantially more expensive in the United States, and the Dutch findings might underestimate potential cost savings generated through redispensing programs in the United States. Participants were prompted to return unused oral anticancer drugs through reminders at the pharmacy, but all such drugs may not have been returned.
DISCLOSURES:
The study was funded by ZonMw, the Dutch national organization for health research and development. The authors have disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
TOPLINE:
to save money and reduce waste, a Dutch study has found.
METHODOLOGY:
- Ongoing drug shortages and growing drug prices contribute to access issues in oncology.
- Researchers compared the reduction in drug waste and cost savings from redispensing oral anticancer drugs versus the standard practice of disposing of them.
- Outpatient pharmacies at four Dutch hospitals participated. A total of 1,071 patients with cancer receiving oral anticancer drugs for at-home use were given special packaging for returning unused medication to the pharmacy.
- The pharmacy ensured the quality of returned drugs based on authenticity, appearance, remaining shelf-life, and adequate storage temperature.
TAKEAWAY:
- A total of 13,069 oral anticancer drug packages, containing an average of 27 daily doses per package, were dispensed during the study period.
- Overall, 16% of patients (n = 171) returned 335 (2.6%) unused oral anticancer drug packages, of which 68% were redispensed after passing quality control.
- Redispensing unused oral anticancer drugs reduced waste by 68%, compared with disposing of them, and provided a mean net annual cost savings of €576 (U.S. $682) per patient per year.
- When just those patients who took targeted oral anticancer drugs for up to 24 months were looked at, the mean net annual cost savings associated with the quality check protocol increased to €934 (U.S. $1,019) per patient or of only the visual quality check was €1,348 (U.S. $1,474) per patient.
IN PRACTICE:
“New strategies targeting waste are required to improve financial and ecologic sustainability of expensive therapies, such as oral anticancer drugs, that frequently remain unused by patients,” the authors write. “These findings provide a waste-minimizing strategy to contribute to sustainable and affordable access to drugs.”
SOURCE:
The study, by Elisabeth M. Smale, PharmD, of Radboud University Medical Center, the Netherlands, and colleagues, was published online in JAMA Oncology.
LIMITATIONS:
Novel drugs are substantially more expensive in the United States, and the Dutch findings might underestimate potential cost savings generated through redispensing programs in the United States. Participants were prompted to return unused oral anticancer drugs through reminders at the pharmacy, but all such drugs may not have been returned.
DISCLOSURES:
The study was funded by ZonMw, the Dutch national organization for health research and development. The authors have disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
TOPLINE:
to save money and reduce waste, a Dutch study has found.
METHODOLOGY:
- Ongoing drug shortages and growing drug prices contribute to access issues in oncology.
- Researchers compared the reduction in drug waste and cost savings from redispensing oral anticancer drugs versus the standard practice of disposing of them.
- Outpatient pharmacies at four Dutch hospitals participated. A total of 1,071 patients with cancer receiving oral anticancer drugs for at-home use were given special packaging for returning unused medication to the pharmacy.
- The pharmacy ensured the quality of returned drugs based on authenticity, appearance, remaining shelf-life, and adequate storage temperature.
TAKEAWAY:
- A total of 13,069 oral anticancer drug packages, containing an average of 27 daily doses per package, were dispensed during the study period.
- Overall, 16% of patients (n = 171) returned 335 (2.6%) unused oral anticancer drug packages, of which 68% were redispensed after passing quality control.
- Redispensing unused oral anticancer drugs reduced waste by 68%, compared with disposing of them, and provided a mean net annual cost savings of €576 (U.S. $682) per patient per year.
- When just those patients who took targeted oral anticancer drugs for up to 24 months were looked at, the mean net annual cost savings associated with the quality check protocol increased to €934 (U.S. $1,019) per patient or of only the visual quality check was €1,348 (U.S. $1,474) per patient.
IN PRACTICE:
“New strategies targeting waste are required to improve financial and ecologic sustainability of expensive therapies, such as oral anticancer drugs, that frequently remain unused by patients,” the authors write. “These findings provide a waste-minimizing strategy to contribute to sustainable and affordable access to drugs.”
SOURCE:
The study, by Elisabeth M. Smale, PharmD, of Radboud University Medical Center, the Netherlands, and colleagues, was published online in JAMA Oncology.
LIMITATIONS:
Novel drugs are substantially more expensive in the United States, and the Dutch findings might underestimate potential cost savings generated through redispensing programs in the United States. Participants were prompted to return unused oral anticancer drugs through reminders at the pharmacy, but all such drugs may not have been returned.
DISCLOSURES:
The study was funded by ZonMw, the Dutch national organization for health research and development. The authors have disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Are we ready for systematic newborn genome sequencing?
PARIS – Routine sampling and analysis of newborn DNA would allow us to screen for many hundreds of childhood genetic diseases. This is the claim made by David Geneviève, MD, PhD, chair of the French Association of Clinical Geneticists and lecturer at the University of Montpellier (France), at the 9th annual conference of the French Society of Predictive and Personalized Medicine.
To date, newborn screening has consisted of taking a drop of blood from a newborn’s heel. In the future, DNA samples could be taken from babies for whole genome sequencing to look for diseases that are likely to crop up later in life.
The challenge
“In France, nearly all of the 720,000 babies born each year undergo newborn screening (only 300 refuse),” said Dr. Geneviève. For 60 years, newborn screening has tested for phenylketonuria, congenital hypothyroidism, congenital adrenal hyperplasia, sickle cell disease, cystic fibrosis, and medium-chain acyl-coenzyme A dehydrogenase deficiency.
On Jan. 1, 2023, France’s national newborn screening program added seven new diseases, bringing the number of rare diseases screened for to 13. The new diseases are homocystinuria, maple syrup urine disease, tyrosinemia type 1, isovaleric acidemia, glutaric aciduria type I, long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, and carnitine deficiency.
“There aren’t just 13 childhood diseases,” continued Dr. Geneviève. “There are several hundred rare diseases, and genome sequencing tools allow us to broaden our screening capabilities. It’s inevitable that the ability to sequence your child’s genome at birth will become a possibility. It’s highly likely that within 10-15 years, all newborns will have their genome determined at birth for screening purposes.”
Current international trials
Genome sequencing has already been studied for several years in multiple countries. New York’s Guardian study requires all newborns taking part to undergo genome sequencing. “Our English-speaking colleagues use the genome to screen for childhood diseases that would benefit from treatment (235 can be treated) but also as a preventive measure and a way of providing early therapeutic education,” said Dr. Geneviève.
In 2016, American researchers launched the BabySeq Project, which was conducted at several sites (Boston, New York, Birmingham, Detroit, and Philadelphia). One of its aims is to assess the medical, psychological, and financial impact of screening via genome sequencing at birth, compared with conventional screening.
In North Carolina, 25,000 newborns took part in the Early Check study, a neonatal genetic screening project focusing on childhood spinal muscular atrophy, fragile X syndrome, and Duchenne muscular dystrophy.
In the United Kingdom, Genomics England seeks to assess the feasibility, benefits, and risks of whole genome sequencing as part of the Newborn Genomes Programme, an analysis of 100,000 newborn genomes. Projects are also underway in Belgium, Italy, and France (PeriGEN MED in Dijon).
Dijon’s specialist team
The conditions for considering neonatal screening of a disease are determined by the health care authorities in each country and vary greatly from one state to the next.
To date, in France, the only genetic screening authorized is for childhood spinal muscular atrophy via identification of an anomaly on SMN1. It has not yet been implemented, but a pilot study of its use is underway.
“If we are able to identify the 40 newborns affected by spinal muscular atrophy from birth, we can offer these patients gene therapy and stop them from dying at 1 or 2 years of age,” said Dr. Geneviève.
In the future, France should draw up a list of diseases for which genetic screening is useful, he added.
Although France’s initiative for genomic medicine, France Génomique 2025, does not envisage a neonatal genome sequencing screening program, a team in Dijon is studying several dozen genomes to determine the medical and financial benefits of such a program, explained Dr. Geneviève.
Ethical issues
Of course, this technological achievement raises ethical issues. “What do we do with the genetic data obtained at birth that won’t become apparent until adulthood, if we find a BRCA1 or BRCA2 variant in a newborn’s genome?” asked Dr. Genevieve.
Will the information obtained be stored somewhere? “This is a real issue,” he said. “The English have a national system. In their newborn screening program, when an infant grows into adulthood, he or she can have access to the genetic data.”
There is also a big risk that women will be pressured to undergo genetic testing during pregnancy. “No genome-related antenatal tests are carried out unless there are concerning ultrasound findings and only to look for particularly severe incurable diseases,” said Dr. Geneviève.
Not like Gattaca*
Financial obstacles should be quickly pushed aside. The cost of genome sequencing has decreased in the past few years. The first sequencing in 2003 cost close to $3 billion. Nowadays, it can be done for less than 1,000 € (just over $1,000).
Although neonatal genetic screening would enable us to limit the development of serious diseases, the decision to use such testing routinely must be made by society as a whole, Dr. Geneviève concluded.
“We often oppose preventive and personalized treatment strategies. Now the two have joined forces,” said Pascal Pujol, MD, PhD, chair of SFMPP.
For Dr. Pujol, broadening the application of genome sequencing is a no-brainer. “It won’t be like in Gattaca,” he reassures us. “It wouldn’t be done to determine a person’s character but [rather] to prevent those rare diseases that affect 4 to 5% of the population.”
*A reference to Andrew Niccol’s 1997 science fiction movie Gattaca. The film is set in a futuristic world in which parents can choose the genotype of their children to conceive test-tube babies with the fewest defects and the most advantages possible for society.
This article was translated from the Medscape French edition and a version appeared on Medscape.com.
PARIS – Routine sampling and analysis of newborn DNA would allow us to screen for many hundreds of childhood genetic diseases. This is the claim made by David Geneviève, MD, PhD, chair of the French Association of Clinical Geneticists and lecturer at the University of Montpellier (France), at the 9th annual conference of the French Society of Predictive and Personalized Medicine.
To date, newborn screening has consisted of taking a drop of blood from a newborn’s heel. In the future, DNA samples could be taken from babies for whole genome sequencing to look for diseases that are likely to crop up later in life.
The challenge
“In France, nearly all of the 720,000 babies born each year undergo newborn screening (only 300 refuse),” said Dr. Geneviève. For 60 years, newborn screening has tested for phenylketonuria, congenital hypothyroidism, congenital adrenal hyperplasia, sickle cell disease, cystic fibrosis, and medium-chain acyl-coenzyme A dehydrogenase deficiency.
On Jan. 1, 2023, France’s national newborn screening program added seven new diseases, bringing the number of rare diseases screened for to 13. The new diseases are homocystinuria, maple syrup urine disease, tyrosinemia type 1, isovaleric acidemia, glutaric aciduria type I, long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, and carnitine deficiency.
“There aren’t just 13 childhood diseases,” continued Dr. Geneviève. “There are several hundred rare diseases, and genome sequencing tools allow us to broaden our screening capabilities. It’s inevitable that the ability to sequence your child’s genome at birth will become a possibility. It’s highly likely that within 10-15 years, all newborns will have their genome determined at birth for screening purposes.”
Current international trials
Genome sequencing has already been studied for several years in multiple countries. New York’s Guardian study requires all newborns taking part to undergo genome sequencing. “Our English-speaking colleagues use the genome to screen for childhood diseases that would benefit from treatment (235 can be treated) but also as a preventive measure and a way of providing early therapeutic education,” said Dr. Geneviève.
In 2016, American researchers launched the BabySeq Project, which was conducted at several sites (Boston, New York, Birmingham, Detroit, and Philadelphia). One of its aims is to assess the medical, psychological, and financial impact of screening via genome sequencing at birth, compared with conventional screening.
In North Carolina, 25,000 newborns took part in the Early Check study, a neonatal genetic screening project focusing on childhood spinal muscular atrophy, fragile X syndrome, and Duchenne muscular dystrophy.
In the United Kingdom, Genomics England seeks to assess the feasibility, benefits, and risks of whole genome sequencing as part of the Newborn Genomes Programme, an analysis of 100,000 newborn genomes. Projects are also underway in Belgium, Italy, and France (PeriGEN MED in Dijon).
Dijon’s specialist team
The conditions for considering neonatal screening of a disease are determined by the health care authorities in each country and vary greatly from one state to the next.
To date, in France, the only genetic screening authorized is for childhood spinal muscular atrophy via identification of an anomaly on SMN1. It has not yet been implemented, but a pilot study of its use is underway.
“If we are able to identify the 40 newborns affected by spinal muscular atrophy from birth, we can offer these patients gene therapy and stop them from dying at 1 or 2 years of age,” said Dr. Geneviève.
In the future, France should draw up a list of diseases for which genetic screening is useful, he added.
Although France’s initiative for genomic medicine, France Génomique 2025, does not envisage a neonatal genome sequencing screening program, a team in Dijon is studying several dozen genomes to determine the medical and financial benefits of such a program, explained Dr. Geneviève.
Ethical issues
Of course, this technological achievement raises ethical issues. “What do we do with the genetic data obtained at birth that won’t become apparent until adulthood, if we find a BRCA1 or BRCA2 variant in a newborn’s genome?” asked Dr. Genevieve.
Will the information obtained be stored somewhere? “This is a real issue,” he said. “The English have a national system. In their newborn screening program, when an infant grows into adulthood, he or she can have access to the genetic data.”
There is also a big risk that women will be pressured to undergo genetic testing during pregnancy. “No genome-related antenatal tests are carried out unless there are concerning ultrasound findings and only to look for particularly severe incurable diseases,” said Dr. Geneviève.
Not like Gattaca*
Financial obstacles should be quickly pushed aside. The cost of genome sequencing has decreased in the past few years. The first sequencing in 2003 cost close to $3 billion. Nowadays, it can be done for less than 1,000 € (just over $1,000).
Although neonatal genetic screening would enable us to limit the development of serious diseases, the decision to use such testing routinely must be made by society as a whole, Dr. Geneviève concluded.
“We often oppose preventive and personalized treatment strategies. Now the two have joined forces,” said Pascal Pujol, MD, PhD, chair of SFMPP.
For Dr. Pujol, broadening the application of genome sequencing is a no-brainer. “It won’t be like in Gattaca,” he reassures us. “It wouldn’t be done to determine a person’s character but [rather] to prevent those rare diseases that affect 4 to 5% of the population.”
*A reference to Andrew Niccol’s 1997 science fiction movie Gattaca. The film is set in a futuristic world in which parents can choose the genotype of their children to conceive test-tube babies with the fewest defects and the most advantages possible for society.
This article was translated from the Medscape French edition and a version appeared on Medscape.com.
PARIS – Routine sampling and analysis of newborn DNA would allow us to screen for many hundreds of childhood genetic diseases. This is the claim made by David Geneviève, MD, PhD, chair of the French Association of Clinical Geneticists and lecturer at the University of Montpellier (France), at the 9th annual conference of the French Society of Predictive and Personalized Medicine.
To date, newborn screening has consisted of taking a drop of blood from a newborn’s heel. In the future, DNA samples could be taken from babies for whole genome sequencing to look for diseases that are likely to crop up later in life.
The challenge
“In France, nearly all of the 720,000 babies born each year undergo newborn screening (only 300 refuse),” said Dr. Geneviève. For 60 years, newborn screening has tested for phenylketonuria, congenital hypothyroidism, congenital adrenal hyperplasia, sickle cell disease, cystic fibrosis, and medium-chain acyl-coenzyme A dehydrogenase deficiency.
On Jan. 1, 2023, France’s national newborn screening program added seven new diseases, bringing the number of rare diseases screened for to 13. The new diseases are homocystinuria, maple syrup urine disease, tyrosinemia type 1, isovaleric acidemia, glutaric aciduria type I, long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, and carnitine deficiency.
“There aren’t just 13 childhood diseases,” continued Dr. Geneviève. “There are several hundred rare diseases, and genome sequencing tools allow us to broaden our screening capabilities. It’s inevitable that the ability to sequence your child’s genome at birth will become a possibility. It’s highly likely that within 10-15 years, all newborns will have their genome determined at birth for screening purposes.”
Current international trials
Genome sequencing has already been studied for several years in multiple countries. New York’s Guardian study requires all newborns taking part to undergo genome sequencing. “Our English-speaking colleagues use the genome to screen for childhood diseases that would benefit from treatment (235 can be treated) but also as a preventive measure and a way of providing early therapeutic education,” said Dr. Geneviève.
In 2016, American researchers launched the BabySeq Project, which was conducted at several sites (Boston, New York, Birmingham, Detroit, and Philadelphia). One of its aims is to assess the medical, psychological, and financial impact of screening via genome sequencing at birth, compared with conventional screening.
In North Carolina, 25,000 newborns took part in the Early Check study, a neonatal genetic screening project focusing on childhood spinal muscular atrophy, fragile X syndrome, and Duchenne muscular dystrophy.
In the United Kingdom, Genomics England seeks to assess the feasibility, benefits, and risks of whole genome sequencing as part of the Newborn Genomes Programme, an analysis of 100,000 newborn genomes. Projects are also underway in Belgium, Italy, and France (PeriGEN MED in Dijon).
Dijon’s specialist team
The conditions for considering neonatal screening of a disease are determined by the health care authorities in each country and vary greatly from one state to the next.
To date, in France, the only genetic screening authorized is for childhood spinal muscular atrophy via identification of an anomaly on SMN1. It has not yet been implemented, but a pilot study of its use is underway.
“If we are able to identify the 40 newborns affected by spinal muscular atrophy from birth, we can offer these patients gene therapy and stop them from dying at 1 or 2 years of age,” said Dr. Geneviève.
In the future, France should draw up a list of diseases for which genetic screening is useful, he added.
Although France’s initiative for genomic medicine, France Génomique 2025, does not envisage a neonatal genome sequencing screening program, a team in Dijon is studying several dozen genomes to determine the medical and financial benefits of such a program, explained Dr. Geneviève.
Ethical issues
Of course, this technological achievement raises ethical issues. “What do we do with the genetic data obtained at birth that won’t become apparent until adulthood, if we find a BRCA1 or BRCA2 variant in a newborn’s genome?” asked Dr. Genevieve.
Will the information obtained be stored somewhere? “This is a real issue,” he said. “The English have a national system. In their newborn screening program, when an infant grows into adulthood, he or she can have access to the genetic data.”
There is also a big risk that women will be pressured to undergo genetic testing during pregnancy. “No genome-related antenatal tests are carried out unless there are concerning ultrasound findings and only to look for particularly severe incurable diseases,” said Dr. Geneviève.
Not like Gattaca*
Financial obstacles should be quickly pushed aside. The cost of genome sequencing has decreased in the past few years. The first sequencing in 2003 cost close to $3 billion. Nowadays, it can be done for less than 1,000 € (just over $1,000).
Although neonatal genetic screening would enable us to limit the development of serious diseases, the decision to use such testing routinely must be made by society as a whole, Dr. Geneviève concluded.
“We often oppose preventive and personalized treatment strategies. Now the two have joined forces,” said Pascal Pujol, MD, PhD, chair of SFMPP.
For Dr. Pujol, broadening the application of genome sequencing is a no-brainer. “It won’t be like in Gattaca,” he reassures us. “It wouldn’t be done to determine a person’s character but [rather] to prevent those rare diseases that affect 4 to 5% of the population.”
*A reference to Andrew Niccol’s 1997 science fiction movie Gattaca. The film is set in a futuristic world in which parents can choose the genotype of their children to conceive test-tube babies with the fewest defects and the most advantages possible for society.
This article was translated from the Medscape French edition and a version appeared on Medscape.com.