Mixed Topics

Across all industries, studies by the U.S. Department of Labor have shown that women, on average, earn 83.7 percent of what their male peers earn. While a lot has been written about the struggles women face in medicine, there have been decidedly fewer analyses that focus on women who choose to become mothers while working in medicine.

Elina Maymind

I’ve been privileged to work with medical students and residents for the last 8 years as the director of graduate and medical student mental health at Rowan-Virtua School of Osteopathic Medicine in Mt. Laurel, N.J. Often, the women I see as patients speak about their struggles with the elusive goal of “having it all.” While both men and women in medicine have difficulty maintaining a work-life balance, I’ve learned, both personally and professionally, that many women face a unique set of challenges.

No matter what their professional status, our society often views a woman as the default parent. For example, the teacher often calls the mothers first. The camp nurse calls me first, not my husband, when our child scrapes a knee. After-school play dates are arranged by the mothers, not fathers.

But mothers also bring to medicine a wealth of unique experiences, ideas, and viewpoints. They learn firsthand how to foster affect regulation and frustration tolerance in their kids and become efficient at managing the constant, conflicting tug of war of demands.

Some may argue that, over time, women end up earning significantly less than their male counterparts because they leave the workforce while on maternity leave, ultimately delaying their upward career progression. It’s likely a much more complex problem. Many of my patients believe that, in our male-dominated society (and workforce), women are punished for being aggressive or stating bold opinions, while men are rewarded for the same actions. While a man may sound forceful and in charge, a women will likely be thought of as brusque and unappreciative.

Outside of work, many women may have more on their plate. A 2020 Gallup poll of more than 3,000 heterosexual couples found that women are responsible for the majority of household chores. Women continue to handle more of the emotional labor within their families, regardless of income, age, or professional status. This is sometimes called the “Mental Load’ or “Second Shift.” As our society continues to view women as the default parent for childcare, medical issues, and overarching social and emotional tasks vital to raising happy, healthy children, the struggle a female medical professional feels is palpable.

Despite the very real and difficult challenges in finding a perfect balance and having it all, both at home and at work, the role of mother and physician must be intimately intertwined. Raising kids requires a parent to consistently dole out control, predictability, and reassurance for a child to thrive. Good limit and boundary setting leads to healthy development from a young age.

Psychiatric patients (and perhaps all patients) also require control, predictability, and reassurance from their doctor. The lessons learned in being a good mother can be directly applied in patient care, and vice versa. The cross-pollination of this relationship continues to grow more powerful as a woman’s children grow and her career matures.

Pediatrician and psychoanalyst Donald Winnicott’s idea of a “good enough” mother cannot be a one-size-fits-all approach. Women who self-select into the world of medicine often hold themselves to a higher standard than “good enough.” Acknowledging that the demands from both home and work will fluctuate is key to achieving success both personally and professionally, and lessons from home can and should be utilized to become a more effective physician. The notion of having it all, and the definition of success, must evolve over time.

Dr. Maymind is director of medical and graduate student mental health at Rowan-Virtua School of Osteopathic Medicine in Mt. Laurel, N.J. She has no relevant disclosures.

Across all industries, studies by the U.S. Department of Labor have shown that women, on average, earn 83.7 percent of what their male peers earn. While a lot has been written about the struggles women face in medicine, there have been decidedly fewer analyses that focus on women who choose to become mothers while working in medicine.

Elina Maymind

I’ve been privileged to work with medical students and residents for the last 8 years as the director of graduate and medical student mental health at Rowan-Virtua School of Osteopathic Medicine in Mt. Laurel, N.J. Often, the women I see as patients speak about their struggles with the elusive goal of “having it all.” While both men and women in medicine have difficulty maintaining a work-life balance, I’ve learned, both personally and professionally, that many women face a unique set of challenges.

No matter what their professional status, our society often views a woman as the default parent. For example, the teacher often calls the mothers first. The camp nurse calls me first, not my husband, when our child scrapes a knee. After-school play dates are arranged by the mothers, not fathers.

But mothers also bring to medicine a wealth of unique experiences, ideas, and viewpoints. They learn firsthand how to foster affect regulation and frustration tolerance in their kids and become efficient at managing the constant, conflicting tug of war of demands.

Some may argue that, over time, women end up earning significantly less than their male counterparts because they leave the workforce while on maternity leave, ultimately delaying their upward career progression. It’s likely a much more complex problem. Many of my patients believe that, in our male-dominated society (and workforce), women are punished for being aggressive or stating bold opinions, while men are rewarded for the same actions. While a man may sound forceful and in charge, a women will likely be thought of as brusque and unappreciative.

Outside of work, many women may have more on their plate. A 2020 Gallup poll of more than 3,000 heterosexual couples found that women are responsible for the majority of household chores. Women continue to handle more of the emotional labor within their families, regardless of income, age, or professional status. This is sometimes called the “Mental Load’ or “Second Shift.” As our society continues to view women as the default parent for childcare, medical issues, and overarching social and emotional tasks vital to raising happy, healthy children, the struggle a female medical professional feels is palpable.

Despite the very real and difficult challenges in finding a perfect balance and having it all, both at home and at work, the role of mother and physician must be intimately intertwined. Raising kids requires a parent to consistently dole out control, predictability, and reassurance for a child to thrive. Good limit and boundary setting leads to healthy development from a young age.

Psychiatric patients (and perhaps all patients) also require control, predictability, and reassurance from their doctor. The lessons learned in being a good mother can be directly applied in patient care, and vice versa. The cross-pollination of this relationship continues to grow more powerful as a woman’s children grow and her career matures.

Pediatrician and psychoanalyst Donald Winnicott’s idea of a “good enough” mother cannot be a one-size-fits-all approach. Women who self-select into the world of medicine often hold themselves to a higher standard than “good enough.” Acknowledging that the demands from both home and work will fluctuate is key to achieving success both personally and professionally, and lessons from home can and should be utilized to become a more effective physician. The notion of having it all, and the definition of success, must evolve over time.

Dr. Maymind is director of medical and graduate student mental health at Rowan-Virtua School of Osteopathic Medicine in Mt. Laurel, N.J. She has no relevant disclosures.

Across all industries, studies by the U.S. Department of Labor have shown that women, on average, earn 83.7 percent of what their male peers earn. While a lot has been written about the struggles women face in medicine, there have been decidedly fewer analyses that focus on women who choose to become mothers while working in medicine.

Elina Maymind

I’ve been privileged to work with medical students and residents for the last 8 years as the director of graduate and medical student mental health at Rowan-Virtua School of Osteopathic Medicine in Mt. Laurel, N.J. Often, the women I see as patients speak about their struggles with the elusive goal of “having it all.” While both men and women in medicine have difficulty maintaining a work-life balance, I’ve learned, both personally and professionally, that many women face a unique set of challenges.

No matter what their professional status, our society often views a woman as the default parent. For example, the teacher often calls the mothers first. The camp nurse calls me first, not my husband, when our child scrapes a knee. After-school play dates are arranged by the mothers, not fathers.

But mothers also bring to medicine a wealth of unique experiences, ideas, and viewpoints. They learn firsthand how to foster affect regulation and frustration tolerance in their kids and become efficient at managing the constant, conflicting tug of war of demands.

Some may argue that, over time, women end up earning significantly less than their male counterparts because they leave the workforce while on maternity leave, ultimately delaying their upward career progression. It’s likely a much more complex problem. Many of my patients believe that, in our male-dominated society (and workforce), women are punished for being aggressive or stating bold opinions, while men are rewarded for the same actions. While a man may sound forceful and in charge, a women will likely be thought of as brusque and unappreciative.

Outside of work, many women may have more on their plate. A 2020 Gallup poll of more than 3,000 heterosexual couples found that women are responsible for the majority of household chores. Women continue to handle more of the emotional labor within their families, regardless of income, age, or professional status. This is sometimes called the “Mental Load’ or “Second Shift.” As our society continues to view women as the default parent for childcare, medical issues, and overarching social and emotional tasks vital to raising happy, healthy children, the struggle a female medical professional feels is palpable.

Despite the very real and difficult challenges in finding a perfect balance and having it all, both at home and at work, the role of mother and physician must be intimately intertwined. Raising kids requires a parent to consistently dole out control, predictability, and reassurance for a child to thrive. Good limit and boundary setting leads to healthy development from a young age.

Psychiatric patients (and perhaps all patients) also require control, predictability, and reassurance from their doctor. The lessons learned in being a good mother can be directly applied in patient care, and vice versa. The cross-pollination of this relationship continues to grow more powerful as a woman’s children grow and her career matures.

Pediatrician and psychoanalyst Donald Winnicott’s idea of a “good enough” mother cannot be a one-size-fits-all approach. Women who self-select into the world of medicine often hold themselves to a higher standard than “good enough.” Acknowledging that the demands from both home and work will fluctuate is key to achieving success both personally and professionally, and lessons from home can and should be utilized to become a more effective physician. The notion of having it all, and the definition of success, must evolve over time.

Dr. Maymind is director of medical and graduate student mental health at Rowan-Virtua School of Osteopathic Medicine in Mt. Laurel, N.J. She has no relevant disclosures.

Clinicians are using dupilumab off label to treat a wider range of allergic conditions in adults and children.

The drug, marketed as Dupixent, is currently approved in the United States to treat atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis, and prurigo nodularis in adults. Dupilumab is also approved to treat eosinophilic esophagitis in patients aged 12 years and older and atopic dermatitis and asthma in some patients as young as age 6 months.

As the roster of approved and off-label indications grows, skin specialists said, pediatricians and other primary care providers should become familiar with the drug – given the increasing likelihood that their patients may be taking the medication.

The U.S. Food and Drug Administration first approved dupilumab in 2017 for eczema and has continued to add new treatment indications, the most recent being for prurigo nodularis, in 2022. Sanofi, which markets the drug with Regeneron, announced in April 2022 that some 430,000 patients worldwide were taking the drug – a figure it hoped to raise by 1.5 million by 2025.
 

A well-tolerated – if expensive – drug

Dupilumab, an interleukin-4 (IL-4) receptor alpha-antagonist biologic, blocks both IL-4 and IL-13 signaling, Marlys Fassett, MD, PhD, associate professor of dermatology at the University of California, San Francisco, told this news organization.

Dr. Fassett said she prescribes the drug off label for chronic idiopathic urticaria, including in older patients, and finds that the side effects in older patients are similar to those in younger people. The medication costs $36,000 per year, although some patients can get it more cheaply.

“Dupixent is a super-safe drug because it doesn’t immunosuppress any other part of the immune system, so you still have good antibacterial, antiviral, and antifungal immunity,” she added. “That makes perfect sense as a biological mechanism, and it’s been found safe in clinical trials.”

Case reports of potential adverse reactions to dupilumab have included ocular surface disease, lichen planus, and rash on the face and neck.

“We’re still learning about complications and are watching patients carefully,” said Marissa J. Perman, MD, section chief of dermatology at Children’s Hospital of Philadelphia.

Many people with atopic dermatitis also have other allergic conditions, such as contact dermatitis, asthma, prurigo nodularis, allergic rhinitis, and seasonal allergies. Each of these conditions has a pathway that depends on IL-4 receptors, Dr. Fassett said.

“It’s amazing how many conditions Dupixent improves. Sometimes we prescribe on-label Dupixent for atopic dermatitis, and inadvertently, the drug also improves that patient’s other, off-label conditions,” Dr. Fassett said. “I think that’s the best evidence that Dupixent works in these off-label cases.”

Lindsay C. Strowd, MD, associate professor of dermatology at Wake Forest University, Winston-Salem, N.C., said she uses off-label dupilumab to treat bullous pemphigoid and intense pruritus of unknown etiology.

“And several times I have treated drug reaction with eosinophilia and systemic symptoms, a rare adverse drug reaction that causes a rash and eosinophilia,” Dr. Strowd added.

Tissa Hata, MD, professor of medicine and clinical service chief at the University of California, San Diego, mainly treats elderly patients. She uses dupilumab to treat bullous pemphigoid and chronic pruritus. “There have been reports of using Dupixent to treat adult alopecia areata, chronic urticaria, localized scleroderma, and even keloids,” she told this news organization.

As a pediatric dermatologist, Dr. Perman treats children with atopic dermatitis as young as 3 months of age. She also uses dupilumab for alopecia areata, graft vs. host disease, and pruritus not otherwise specified.

Conjunctivitis and facial redness are two side effects Dr. Fassett sometimes sees with dupilumab. They occur similarly with all conditions and in all age groups. “We don’t know why they occur, and we don’t always know how to alleviate them,” she said. “So a small number of patients stop using Dupixent because they can’t tolerate those two side effects.

“We’re not worried about infection risk,” Dr. Fassett said. “Your patients may have heard of dupilumab as an immunosuppressant, but its immunosuppression is very focused. You can reassure them that they’re not at increased risk for viral or bacterial infections when they’re on this drug.”

“I don’t think there are any different safety signals to watch for with on-label vs. off-label Dupixent use,” Dr. Strowd added. “In general, the medicine is very safe.”

Dr. Hata said she is impressed with dupilumab’s safety in her elderly patients. All her patients older than 85 years who have taken the drug for bullous pemphigoid have tolerated it well, she said.

“Dupixent seems to be a safe alternative for elderly patients with pruritus because they often cannot tolerate sedating antihistamines due to the risk of falling,” Dr. Hata said. “And UV therapy may be difficult for elderly patients due to problems with transport.”

Although some of Dr. Hata’s elderly patients with atopic dermatitis have discontinued use of the drug after developing conjunctivitis, none taking the drug off label have discontinued it because of side effects, she noted.

“Dupixent manages the condition, but it is not a cure,” Dr. Fassett noted. “Based on the current data, we think it’s safe and effective to take long term, potentially for life.”

Making injections less bothersome

Dupilumab is injected subcutaneously from a single-dose prefilled syringe or a prefilled pen (syringe hidden in an opaque sheath), typically in the thigh, arm, abdomen, or buttocks. According to Sanofi and Regeneron, patients receive dupilumab injections every 2 to 4 weeks in doses based on their age and weight.

“The medication is somewhat viscous, so taking the syringe or pen out of the refrigerator ahead of time to warm it up can make the experience less painful,” Dr. Strowd advised. “For pediatric patients, I sometimes prescribe topical lidocaine applied 30 minutes before injection.”

Dr. Hata suggested icing the skin prior to injecting or distracting the patient by tapping a different area of the skin.

For her pediatric patients, Dr. Perman said she uses “lots of distraction, EMLA cream, and having one person hold the child while a second person injects.”

Clinic and pharmacy staff may show patients how to inject properly, Dr. Fassett added; and the product website provides injection tutorials.
 

Off-label dupixent can be expensive, difficult to obtain

The list price per injection, regardless of dose, is around $1,800. But according to the company’s website, most patients have health insurance or qualify for other assistance, so “very few patients pay the list price.”

Even so, “due to cost and insurance coverage hurdles, obtaining Dupixent for off-label use can be difficult,” Dr. Strowd said.

“In academic medicine, we can obtain drugs for our patients that community doctors may not get approval for,” Dr. Fassett added. “Community doctors can use information in the medical literature and in news articles to press insurance companies to spend money to provide their patients with Dupixent.”

The experts who commented have disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

Clinicians are using dupilumab off label to treat a wider range of allergic conditions in adults and children.

The drug, marketed as Dupixent, is currently approved in the United States to treat atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis, and prurigo nodularis in adults. Dupilumab is also approved to treat eosinophilic esophagitis in patients aged 12 years and older and atopic dermatitis and asthma in some patients as young as age 6 months.

As the roster of approved and off-label indications grows, skin specialists said, pediatricians and other primary care providers should become familiar with the drug – given the increasing likelihood that their patients may be taking the medication.

The U.S. Food and Drug Administration first approved dupilumab in 2017 for eczema and has continued to add new treatment indications, the most recent being for prurigo nodularis, in 2022. Sanofi, which markets the drug with Regeneron, announced in April 2022 that some 430,000 patients worldwide were taking the drug – a figure it hoped to raise by 1.5 million by 2025.
 

A well-tolerated – if expensive – drug

Dupilumab, an interleukin-4 (IL-4) receptor alpha-antagonist biologic, blocks both IL-4 and IL-13 signaling, Marlys Fassett, MD, PhD, associate professor of dermatology at the University of California, San Francisco, told this news organization.

Dr. Fassett said she prescribes the drug off label for chronic idiopathic urticaria, including in older patients, and finds that the side effects in older patients are similar to those in younger people. The medication costs $36,000 per year, although some patients can get it more cheaply.

“Dupixent is a super-safe drug because it doesn’t immunosuppress any other part of the immune system, so you still have good antibacterial, antiviral, and antifungal immunity,” she added. “That makes perfect sense as a biological mechanism, and it’s been found safe in clinical trials.”

Case reports of potential adverse reactions to dupilumab have included ocular surface disease, lichen planus, and rash on the face and neck.

“We’re still learning about complications and are watching patients carefully,” said Marissa J. Perman, MD, section chief of dermatology at Children’s Hospital of Philadelphia.

Many people with atopic dermatitis also have other allergic conditions, such as contact dermatitis, asthma, prurigo nodularis, allergic rhinitis, and seasonal allergies. Each of these conditions has a pathway that depends on IL-4 receptors, Dr. Fassett said.

“It’s amazing how many conditions Dupixent improves. Sometimes we prescribe on-label Dupixent for atopic dermatitis, and inadvertently, the drug also improves that patient’s other, off-label conditions,” Dr. Fassett said. “I think that’s the best evidence that Dupixent works in these off-label cases.”

Lindsay C. Strowd, MD, associate professor of dermatology at Wake Forest University, Winston-Salem, N.C., said she uses off-label dupilumab to treat bullous pemphigoid and intense pruritus of unknown etiology.

“And several times I have treated drug reaction with eosinophilia and systemic symptoms, a rare adverse drug reaction that causes a rash and eosinophilia,” Dr. Strowd added.

Tissa Hata, MD, professor of medicine and clinical service chief at the University of California, San Diego, mainly treats elderly patients. She uses dupilumab to treat bullous pemphigoid and chronic pruritus. “There have been reports of using Dupixent to treat adult alopecia areata, chronic urticaria, localized scleroderma, and even keloids,” she told this news organization.

As a pediatric dermatologist, Dr. Perman treats children with atopic dermatitis as young as 3 months of age. She also uses dupilumab for alopecia areata, graft vs. host disease, and pruritus not otherwise specified.

Conjunctivitis and facial redness are two side effects Dr. Fassett sometimes sees with dupilumab. They occur similarly with all conditions and in all age groups. “We don’t know why they occur, and we don’t always know how to alleviate them,” she said. “So a small number of patients stop using Dupixent because they can’t tolerate those two side effects.

“We’re not worried about infection risk,” Dr. Fassett said. “Your patients may have heard of dupilumab as an immunosuppressant, but its immunosuppression is very focused. You can reassure them that they’re not at increased risk for viral or bacterial infections when they’re on this drug.”

“I don’t think there are any different safety signals to watch for with on-label vs. off-label Dupixent use,” Dr. Strowd added. “In general, the medicine is very safe.”

Dr. Hata said she is impressed with dupilumab’s safety in her elderly patients. All her patients older than 85 years who have taken the drug for bullous pemphigoid have tolerated it well, she said.

“Dupixent seems to be a safe alternative for elderly patients with pruritus because they often cannot tolerate sedating antihistamines due to the risk of falling,” Dr. Hata said. “And UV therapy may be difficult for elderly patients due to problems with transport.”

Although some of Dr. Hata’s elderly patients with atopic dermatitis have discontinued use of the drug after developing conjunctivitis, none taking the drug off label have discontinued it because of side effects, she noted.

“Dupixent manages the condition, but it is not a cure,” Dr. Fassett noted. “Based on the current data, we think it’s safe and effective to take long term, potentially for life.”

Making injections less bothersome

Dupilumab is injected subcutaneously from a single-dose prefilled syringe or a prefilled pen (syringe hidden in an opaque sheath), typically in the thigh, arm, abdomen, or buttocks. According to Sanofi and Regeneron, patients receive dupilumab injections every 2 to 4 weeks in doses based on their age and weight.

“The medication is somewhat viscous, so taking the syringe or pen out of the refrigerator ahead of time to warm it up can make the experience less painful,” Dr. Strowd advised. “For pediatric patients, I sometimes prescribe topical lidocaine applied 30 minutes before injection.”

Dr. Hata suggested icing the skin prior to injecting or distracting the patient by tapping a different area of the skin.

For her pediatric patients, Dr. Perman said she uses “lots of distraction, EMLA cream, and having one person hold the child while a second person injects.”

Clinic and pharmacy staff may show patients how to inject properly, Dr. Fassett added; and the product website provides injection tutorials.
 

Off-label dupixent can be expensive, difficult to obtain

The list price per injection, regardless of dose, is around $1,800. But according to the company’s website, most patients have health insurance or qualify for other assistance, so “very few patients pay the list price.”

Even so, “due to cost and insurance coverage hurdles, obtaining Dupixent for off-label use can be difficult,” Dr. Strowd said.

“In academic medicine, we can obtain drugs for our patients that community doctors may not get approval for,” Dr. Fassett added. “Community doctors can use information in the medical literature and in news articles to press insurance companies to spend money to provide their patients with Dupixent.”

The experts who commented have disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

Clinicians are using dupilumab off label to treat a wider range of allergic conditions in adults and children.

The drug, marketed as Dupixent, is currently approved in the United States to treat atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis, and prurigo nodularis in adults. Dupilumab is also approved to treat eosinophilic esophagitis in patients aged 12 years and older and atopic dermatitis and asthma in some patients as young as age 6 months.

As the roster of approved and off-label indications grows, skin specialists said, pediatricians and other primary care providers should become familiar with the drug – given the increasing likelihood that their patients may be taking the medication.

The U.S. Food and Drug Administration first approved dupilumab in 2017 for eczema and has continued to add new treatment indications, the most recent being for prurigo nodularis, in 2022. Sanofi, which markets the drug with Regeneron, announced in April 2022 that some 430,000 patients worldwide were taking the drug – a figure it hoped to raise by 1.5 million by 2025.
 

A well-tolerated – if expensive – drug

Dupilumab, an interleukin-4 (IL-4) receptor alpha-antagonist biologic, blocks both IL-4 and IL-13 signaling, Marlys Fassett, MD, PhD, associate professor of dermatology at the University of California, San Francisco, told this news organization.

Dr. Fassett said she prescribes the drug off label for chronic idiopathic urticaria, including in older patients, and finds that the side effects in older patients are similar to those in younger people. The medication costs $36,000 per year, although some patients can get it more cheaply.

“Dupixent is a super-safe drug because it doesn’t immunosuppress any other part of the immune system, so you still have good antibacterial, antiviral, and antifungal immunity,” she added. “That makes perfect sense as a biological mechanism, and it’s been found safe in clinical trials.”

Case reports of potential adverse reactions to dupilumab have included ocular surface disease, lichen planus, and rash on the face and neck.

“We’re still learning about complications and are watching patients carefully,” said Marissa J. Perman, MD, section chief of dermatology at Children’s Hospital of Philadelphia.

Many people with atopic dermatitis also have other allergic conditions, such as contact dermatitis, asthma, prurigo nodularis, allergic rhinitis, and seasonal allergies. Each of these conditions has a pathway that depends on IL-4 receptors, Dr. Fassett said.

“It’s amazing how many conditions Dupixent improves. Sometimes we prescribe on-label Dupixent for atopic dermatitis, and inadvertently, the drug also improves that patient’s other, off-label conditions,” Dr. Fassett said. “I think that’s the best evidence that Dupixent works in these off-label cases.”

Lindsay C. Strowd, MD, associate professor of dermatology at Wake Forest University, Winston-Salem, N.C., said she uses off-label dupilumab to treat bullous pemphigoid and intense pruritus of unknown etiology.

“And several times I have treated drug reaction with eosinophilia and systemic symptoms, a rare adverse drug reaction that causes a rash and eosinophilia,” Dr. Strowd added.

Tissa Hata, MD, professor of medicine and clinical service chief at the University of California, San Diego, mainly treats elderly patients. She uses dupilumab to treat bullous pemphigoid and chronic pruritus. “There have been reports of using Dupixent to treat adult alopecia areata, chronic urticaria, localized scleroderma, and even keloids,” she told this news organization.

As a pediatric dermatologist, Dr. Perman treats children with atopic dermatitis as young as 3 months of age. She also uses dupilumab for alopecia areata, graft vs. host disease, and pruritus not otherwise specified.

Conjunctivitis and facial redness are two side effects Dr. Fassett sometimes sees with dupilumab. They occur similarly with all conditions and in all age groups. “We don’t know why they occur, and we don’t always know how to alleviate them,” she said. “So a small number of patients stop using Dupixent because they can’t tolerate those two side effects.

“We’re not worried about infection risk,” Dr. Fassett said. “Your patients may have heard of dupilumab as an immunosuppressant, but its immunosuppression is very focused. You can reassure them that they’re not at increased risk for viral or bacterial infections when they’re on this drug.”

“I don’t think there are any different safety signals to watch for with on-label vs. off-label Dupixent use,” Dr. Strowd added. “In general, the medicine is very safe.”

Dr. Hata said she is impressed with dupilumab’s safety in her elderly patients. All her patients older than 85 years who have taken the drug for bullous pemphigoid have tolerated it well, she said.

“Dupixent seems to be a safe alternative for elderly patients with pruritus because they often cannot tolerate sedating antihistamines due to the risk of falling,” Dr. Hata said. “And UV therapy may be difficult for elderly patients due to problems with transport.”

Although some of Dr. Hata’s elderly patients with atopic dermatitis have discontinued use of the drug after developing conjunctivitis, none taking the drug off label have discontinued it because of side effects, she noted.

“Dupixent manages the condition, but it is not a cure,” Dr. Fassett noted. “Based on the current data, we think it’s safe and effective to take long term, potentially for life.”

Making injections less bothersome

Dupilumab is injected subcutaneously from a single-dose prefilled syringe or a prefilled pen (syringe hidden in an opaque sheath), typically in the thigh, arm, abdomen, or buttocks. According to Sanofi and Regeneron, patients receive dupilumab injections every 2 to 4 weeks in doses based on their age and weight.

“The medication is somewhat viscous, so taking the syringe or pen out of the refrigerator ahead of time to warm it up can make the experience less painful,” Dr. Strowd advised. “For pediatric patients, I sometimes prescribe topical lidocaine applied 30 minutes before injection.”

Dr. Hata suggested icing the skin prior to injecting or distracting the patient by tapping a different area of the skin.

For her pediatric patients, Dr. Perman said she uses “lots of distraction, EMLA cream, and having one person hold the child while a second person injects.”

Clinic and pharmacy staff may show patients how to inject properly, Dr. Fassett added; and the product website provides injection tutorials.
 

Off-label dupixent can be expensive, difficult to obtain

The list price per injection, regardless of dose, is around $1,800. But according to the company’s website, most patients have health insurance or qualify for other assistance, so “very few patients pay the list price.”

Even so, “due to cost and insurance coverage hurdles, obtaining Dupixent for off-label use can be difficult,” Dr. Strowd said.

“In academic medicine, we can obtain drugs for our patients that community doctors may not get approval for,” Dr. Fassett added. “Community doctors can use information in the medical literature and in news articles to press insurance companies to spend money to provide their patients with Dupixent.”

The experts who commented have disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

The Cutis Editorial Board is now accepting applications for the 2024 Resident Corner column. The Editorial Board will select 2 to 3 residents to serve as the Resident Corner columnists for 1 year. Articles are posted online only at www.mdedge.com/dermatology but will be referenced in Index Medicus. All applicants must be current residents and will be in residency throughout 2024.

For consideration, send your curriculum vitae along with a brief (not to exceed 500 words) statement of why you enjoy Cutis and what you can offer your fellow residents in contributing a monthly column.

A signed letter of recommendation from the Director of the dermatology residency program also should be supplied.

All materials should be submitted via email to Melissa Sears (msears@mdedge.com) by November 1. The residents who are selected to write the column for the upcoming year will be notified by November 15.

We look forward to continuing to educate dermatology residents on topics that are most important to them!

The Cutis Editorial Board is now accepting applications for the 2024 Resident Corner column. The Editorial Board will select 2 to 3 residents to serve as the Resident Corner columnists for 1 year. Articles are posted online only at www.mdedge.com/dermatology but will be referenced in Index Medicus. All applicants must be current residents and will be in residency throughout 2024.

For consideration, send your curriculum vitae along with a brief (not to exceed 500 words) statement of why you enjoy Cutis and what you can offer your fellow residents in contributing a monthly column.

A signed letter of recommendation from the Director of the dermatology residency program also should be supplied.

All materials should be submitted via email to Melissa Sears (msears@mdedge.com) by November 1. The residents who are selected to write the column for the upcoming year will be notified by November 15.

We look forward to continuing to educate dermatology residents on topics that are most important to them!

The Cutis Editorial Board is now accepting applications for the 2024 Resident Corner column. The Editorial Board will select 2 to 3 residents to serve as the Resident Corner columnists for 1 year. Articles are posted online only at www.mdedge.com/dermatology but will be referenced in Index Medicus. All applicants must be current residents and will be in residency throughout 2024.

For consideration, send your curriculum vitae along with a brief (not to exceed 500 words) statement of why you enjoy Cutis and what you can offer your fellow residents in contributing a monthly column.

A signed letter of recommendation from the Director of the dermatology residency program also should be supplied.

All materials should be submitted via email to Melissa Sears (msears@mdedge.com) by November 1. The residents who are selected to write the column for the upcoming year will be notified by November 15.

We look forward to continuing to educate dermatology residents on topics that are most important to them!

This transcript has been edited for clarity.

I’d like to present you today with a case that raised a large amount of discussion and debate. I got involved as an ethics consultant on the case. I think you’ll find it very interesting and I also think there are going to be some differences of opinion about how to manage the case. I’ll be looking forward to getting comments and feedback on this.

The case involved a 14-year-old boy who had been brought into the hospital by his parents, suffering from severe bouts of anxiety that were just almost overwhelming to him. When he was brought in, he was assigned a health care provider who had a West African last name. Prior to meeting the patient, I have to say that the father of this kid told the intake department nurse that he requested someone else. He saw the name – he hadn’t even met the provider – and he said he wanted someone who might be Catholic.

The parents are both from the Dominican Republic. They identified as White, but they appeared to be non-White Latinx to the nurse who was doing some of the initial intake. They got reassigned to a different provider in the department who identified as African American.

The first month of treatment for the young boy went very well, and he seemed to be getting along extremely well with his provider. He was reporting relief to both parents of some of his anxiety, and the provider felt very connected to the child. A good doctor-patient alliance had been formed.

Nevertheless, at the end of the first month, the father connected back to one of the administrators at the hospital and complained, saying he still wanted a different provider. When asked why, he said, “Well, I don’t really want to answer that,” but getting pressed, he basically said he wasn’t comfortable with having an African American doctor take care of his child. He eventually went back to the argument that what he wanted was someone with a Catholic background, although I don’t know that he knew whether this particular provider was religious – Catholic or anything else.

The issue became what to do in the face of these continued demands by the dad for a change. Some people felt that, as the father in charge of the child’s care, if we could accommodate what he wanted in terms of the parents being comfortable, then that’s something we should do. I absolutely did not agree.

My view is that in a situation where a strong provider-patient relationship has been established, where trust is going both ways, where there are no issues coming up between this 14-year-old and the provider, and when a serious mental health issue is being adequately addressed, the patient’s interest must come first.

Once that therapeutic alliance had been established and both the patient and the provider felt satisfied, I don’t think the father’s wishes made any sense. He may have been acting more out of bigotry or just discomfort about difference in terms of who the provider was. I don’t think that’s something that any health system should have to accommodate unless it is getting in the way of patient care.

I hope that we treat all physicians as properly trained to deal with all kinds of patients, regardless of their religion, ethnicity, or skin color. They should have the skills to manage and do well with any patient. There may be situations where it just doesn’t work or where people don’t get along. Yes, I think we then should try, perhaps, to shift the doctor, get a different nurse, or have a different person do an exam. That’s because of the inability to get the patient’s health interests addressed.

Listening to this dad about what he preferred in terms of religion or ethnicity seemed to me to be interfering with medical success. Could I stop him from moving this patient out entirely from the care setting? Probably not, but I think the way to manage this is to try to talk to him – and, by the way, to talk to the mother.

When we did bring the mom into the situation, she was very happy with the health care provider. She didn’t agree with the dad and wanted to have a meeting with the social worker, the dad, and her to get him to get over the worries, concerns, and maybe even biases he was bringing in about the kind of provider he wanted. That’s exactly what we did.

I know that there are many instances where patients may say, “I don’t want a particular doctor or a particular type.” My view is that we shouldn’t accommodate that. We should say that our doctors are trained to help and care for all manner of people. Unless we can think of some reason that there might be a gap or a problem in the actual delivery of the quality of care, we are not going to accommodate racism, bigotry, or bias.

We certainly shouldn’t be accommodating that once a successful therapeutic relationship is established. Even when it’s a child, I would argue that the patient’s best interest has to trump parental desires, parental worries, and parental concerns about the background, ethnicity, and religion of the provider.

Dr. Caplan is director of the division of medical ethics at NYU Langone Medical Center, New York. He disclosed a conflict of interest with Johnson & Johnson.

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

This transcript has been edited for clarity.

I’d like to present you today with a case that raised a large amount of discussion and debate. I got involved as an ethics consultant on the case. I think you’ll find it very interesting and I also think there are going to be some differences of opinion about how to manage the case. I’ll be looking forward to getting comments and feedback on this.

The case involved a 14-year-old boy who had been brought into the hospital by his parents, suffering from severe bouts of anxiety that were just almost overwhelming to him. When he was brought in, he was assigned a health care provider who had a West African last name. Prior to meeting the patient, I have to say that the father of this kid told the intake department nurse that he requested someone else. He saw the name – he hadn’t even met the provider – and he said he wanted someone who might be Catholic.

The parents are both from the Dominican Republic. They identified as White, but they appeared to be non-White Latinx to the nurse who was doing some of the initial intake. They got reassigned to a different provider in the department who identified as African American.

The first month of treatment for the young boy went very well, and he seemed to be getting along extremely well with his provider. He was reporting relief to both parents of some of his anxiety, and the provider felt very connected to the child. A good doctor-patient alliance had been formed.

Nevertheless, at the end of the first month, the father connected back to one of the administrators at the hospital and complained, saying he still wanted a different provider. When asked why, he said, “Well, I don’t really want to answer that,” but getting pressed, he basically said he wasn’t comfortable with having an African American doctor take care of his child. He eventually went back to the argument that what he wanted was someone with a Catholic background, although I don’t know that he knew whether this particular provider was religious – Catholic or anything else.

The issue became what to do in the face of these continued demands by the dad for a change. Some people felt that, as the father in charge of the child’s care, if we could accommodate what he wanted in terms of the parents being comfortable, then that’s something we should do. I absolutely did not agree.

My view is that in a situation where a strong provider-patient relationship has been established, where trust is going both ways, where there are no issues coming up between this 14-year-old and the provider, and when a serious mental health issue is being adequately addressed, the patient’s interest must come first.

Once that therapeutic alliance had been established and both the patient and the provider felt satisfied, I don’t think the father’s wishes made any sense. He may have been acting more out of bigotry or just discomfort about difference in terms of who the provider was. I don’t think that’s something that any health system should have to accommodate unless it is getting in the way of patient care.

I hope that we treat all physicians as properly trained to deal with all kinds of patients, regardless of their religion, ethnicity, or skin color. They should have the skills to manage and do well with any patient. There may be situations where it just doesn’t work or where people don’t get along. Yes, I think we then should try, perhaps, to shift the doctor, get a different nurse, or have a different person do an exam. That’s because of the inability to get the patient’s health interests addressed.

Listening to this dad about what he preferred in terms of religion or ethnicity seemed to me to be interfering with medical success. Could I stop him from moving this patient out entirely from the care setting? Probably not, but I think the way to manage this is to try to talk to him – and, by the way, to talk to the mother.

When we did bring the mom into the situation, she was very happy with the health care provider. She didn’t agree with the dad and wanted to have a meeting with the social worker, the dad, and her to get him to get over the worries, concerns, and maybe even biases he was bringing in about the kind of provider he wanted. That’s exactly what we did.

I know that there are many instances where patients may say, “I don’t want a particular doctor or a particular type.” My view is that we shouldn’t accommodate that. We should say that our doctors are trained to help and care for all manner of people. Unless we can think of some reason that there might be a gap or a problem in the actual delivery of the quality of care, we are not going to accommodate racism, bigotry, or bias.

We certainly shouldn’t be accommodating that once a successful therapeutic relationship is established. Even when it’s a child, I would argue that the patient’s best interest has to trump parental desires, parental worries, and parental concerns about the background, ethnicity, and religion of the provider.

Dr. Caplan is director of the division of medical ethics at NYU Langone Medical Center, New York. He disclosed a conflict of interest with Johnson & Johnson.

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

This transcript has been edited for clarity.

I’d like to present you today with a case that raised a large amount of discussion and debate. I got involved as an ethics consultant on the case. I think you’ll find it very interesting and I also think there are going to be some differences of opinion about how to manage the case. I’ll be looking forward to getting comments and feedback on this.

The case involved a 14-year-old boy who had been brought into the hospital by his parents, suffering from severe bouts of anxiety that were just almost overwhelming to him. When he was brought in, he was assigned a health care provider who had a West African last name. Prior to meeting the patient, I have to say that the father of this kid told the intake department nurse that he requested someone else. He saw the name – he hadn’t even met the provider – and he said he wanted someone who might be Catholic.

The parents are both from the Dominican Republic. They identified as White, but they appeared to be non-White Latinx to the nurse who was doing some of the initial intake. They got reassigned to a different provider in the department who identified as African American.

The first month of treatment for the young boy went very well, and he seemed to be getting along extremely well with his provider. He was reporting relief to both parents of some of his anxiety, and the provider felt very connected to the child. A good doctor-patient alliance had been formed.

Nevertheless, at the end of the first month, the father connected back to one of the administrators at the hospital and complained, saying he still wanted a different provider. When asked why, he said, “Well, I don’t really want to answer that,” but getting pressed, he basically said he wasn’t comfortable with having an African American doctor take care of his child. He eventually went back to the argument that what he wanted was someone with a Catholic background, although I don’t know that he knew whether this particular provider was religious – Catholic or anything else.

The issue became what to do in the face of these continued demands by the dad for a change. Some people felt that, as the father in charge of the child’s care, if we could accommodate what he wanted in terms of the parents being comfortable, then that’s something we should do. I absolutely did not agree.

My view is that in a situation where a strong provider-patient relationship has been established, where trust is going both ways, where there are no issues coming up between this 14-year-old and the provider, and when a serious mental health issue is being adequately addressed, the patient’s interest must come first.

Once that therapeutic alliance had been established and both the patient and the provider felt satisfied, I don’t think the father’s wishes made any sense. He may have been acting more out of bigotry or just discomfort about difference in terms of who the provider was. I don’t think that’s something that any health system should have to accommodate unless it is getting in the way of patient care.

I hope that we treat all physicians as properly trained to deal with all kinds of patients, regardless of their religion, ethnicity, or skin color. They should have the skills to manage and do well with any patient. There may be situations where it just doesn’t work or where people don’t get along. Yes, I think we then should try, perhaps, to shift the doctor, get a different nurse, or have a different person do an exam. That’s because of the inability to get the patient’s health interests addressed.

Listening to this dad about what he preferred in terms of religion or ethnicity seemed to me to be interfering with medical success. Could I stop him from moving this patient out entirely from the care setting? Probably not, but I think the way to manage this is to try to talk to him – and, by the way, to talk to the mother.

When we did bring the mom into the situation, she was very happy with the health care provider. She didn’t agree with the dad and wanted to have a meeting with the social worker, the dad, and her to get him to get over the worries, concerns, and maybe even biases he was bringing in about the kind of provider he wanted. That’s exactly what we did.

I know that there are many instances where patients may say, “I don’t want a particular doctor or a particular type.” My view is that we shouldn’t accommodate that. We should say that our doctors are trained to help and care for all manner of people. Unless we can think of some reason that there might be a gap or a problem in the actual delivery of the quality of care, we are not going to accommodate racism, bigotry, or bias.

We certainly shouldn’t be accommodating that once a successful therapeutic relationship is established. Even when it’s a child, I would argue that the patient’s best interest has to trump parental desires, parental worries, and parental concerns about the background, ethnicity, and religion of the provider.

Dr. Caplan is director of the division of medical ethics at NYU Langone Medical Center, New York. He disclosed a conflict of interest with Johnson & Johnson.

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

Back in 1997, a young Stanford (Calif.) University student named Christopher Flowers made his debut in the medical literature with a Nature Medicine report that called out the pharmaceutical industry for overlooking the crucial role of clinical investigators in drug development.

“My research uncovered a series of physicians who served as ‘clinical champions’ and dramatically sped the process of drug development,” Dr. Flowers recalled in an interview. “This early career research inspired me to become the type of clinical champion that I uncovered.”

MD Anderson Cancer Center

Over his career, hematologist-oncologist Dr. Flowers has developed lifesaving therapies for lymphoma, which has transformed into a highly treatable and even curable disease. He’s listed as a coauthor of hundreds of peer-reviewed cancer studies, reports, and medical society guidelines. And he’s revealed stark disparities in blood cancer care: His research shows that non-White patients suffer from worse outcomes, regardless of factors like income and insurance coverage.

The University of Texas MD Anderson Cancer Center, Houston, recently named physician-scientist Dr. Flowers as division head of cancer medicine, a position he’s held on an interim basis. As of Sept. 1, he will permanently oversee 300 faculty and more than 2,000 staff members.
 

A running start in Seattle

For Dr. Flowers, track and field is a sport that runs in the family. His grandfather was a top runner in both high school and college, and both Dr. Flowers and his brother ran competitively in Seattle, where they grew up. But Dr. Flowers chose a career in oncology, earning a medical degree at Stanford and master’s degrees at both Stanford and the University of Washington, Seattle.

The late Kenneth Melmon, MD, a groundbreaking pharmacologist, was a major influence. “He was one of the first people that I met when I began as an undergraduate at Stanford. We grew to be long-standing friends, and he demonstrated what outstanding mentorship looks like. In our research collaboration, we investigated the work of Dr. Gertrude Elion and Dr. George Hitchings involving the translation of pharmacological data from cellular and animal models to clinically useful drugs including 6-mercaptopurine, allopurinol, azathioprine, acyclovir, and zidovudine.”

The late Oliver Press, MD, a blood cancer specialist, inspired Dr. Flower’s interest in lymphoma. “I began work with him during an internship at the University of Washington. Ollie was a great inspiration and a key leader in the development of innovative therapies for lymphoma. He embodied the role of a clinical champion translating work in radioimmunotherapy to new therapeutics for patients with lymphomas. Working with him ultimately led me to pursue a career in hematology and oncology with a focus on the care for patients with lymphomas.”
 

Career blooms as lymphoma care advances

Dr. Flowers went on to Emory University, Atlanta, where he served as scientific director of the Research Informatics Shared Resource and a faculty member in the department of biomedical informatics. “I applied my training in informatics and my clinical expertise to support active grants from the Burroughs Wellcome Fund for Innovation in Regulatory Science and from the National Cancer Institute to develop informatics tools for pathology image analysis and prognostic modeling.”

For 13 years, he also served the Winship Cancer Institute as director of the Emory Healthcare lymphoma program (where his patients included Kansas City Chiefs football star Eric Berry), and for 4 years as scientific director of research informatics. Meanwhile, Dr. Flowers helped develop national practice guidelines for the American Society of Clinical Oncology, the American Cancer Society, and the American College of Radiology. He also chaired the ASCO guideline on management of febrile neutropenia.

In 2019, MD Anderson hired Dr. Flowers as chair of the department of lymphoma/myeloma. A year later, he was appointed division head ad interim for cancer medicine.

“Chris is a unique leader who expertly combines mentorship, sponsorship, and bidirectional open, honest communication,” said Sairah Ahmed, MD, associate professor of lymphoma at MD Anderson. “He doesn’t just empower his team to reach their goals. He also inspires those around him to turn vision into reality.”

As Dr. Flowers noted, many patients with lymphoma are now able to recover and live normal lives. He himself played a direct role himself in boosting lifespans.

“I have been fortunate to play a role in the development of several treatments that have led to advances in first-line therapy for patients with aggressive lymphomas. I partnered with others at MD Anderson, including Dr. Sattva Neelapu and Dr. Jason Westin, who have developed novel therapies like chimeric antigen receptor T-cell therapy for patients with relapse lymphomas,” he said. “Leaders in the field at MD Anderson like Dr. Michael Wang have developed new oral treatments for patients with rare lymphoma subtypes like mantle cell lymphoma. Other colleagues such as Dr. Nathan Fowler and Dr. Loretta Nastoupil have focused on the care for patients with indolent lymphomas and developed less-toxic therapies that are now in common use.”
 

Exposing the disparities in blood cancer care

Dr. Flowers, who’s African American, has also been a leader in health disparity research. In 2016, for example, he was coauthor of a study into non-Hodgkin’s lymphoma that revealed that Blacks in the United States have dramatically lower survival rates than Whites. The 10-year survival rate for Black women with chronic lymphocytic leukemia was just 47%, for example, compared with 66% for White females. “Although incidence rates of lymphoid neoplasms are generally higher among Whites, Black men tend to have poorer survival,” Dr. Flowers and colleagues wrote.

In a 2021 report for the ASCO Educational Book, Dr. Flowers and hematologist-oncologist Demetria Smith-Graziani, MD, now with Emory University, explored disparities across blood cancers and barriers to minority enrollment in clinical trials. “Some approaches that clinicians can apply to address these disparities include increasing systems-level awareness, improving access to care, and reducing biases in clinical setting,” the authors wrote.

Luis Malpica Castillo, MD, assistant professor of lymphoma at MD Anderson Cancer Center, lauded the work of Dr. Flowers in expanding opportunities for minority patients with the disease.

“During the past years, Dr. Flowers’ work has not only had a positive impact on the Texan community, but minority populations living with cancer in the United States and abroad,” he said. “Currently, we are implementing cancer care networks aimed to increase diversity in clinical trials by enrolling a larger number of Hispanic and African American patients, who otherwise may not have benefited from novel therapies. The ultimate goal is to provide high-quality care to all patients living with cancer.”

In addition to his research work, Dr. Flowers is an advocate for diversity within the hematology community. He’s a founding member and former chair of the American Society of Hematology’s Committee on Diversity, Equity and Inclusion (formerly the Committee on Promoting Diversity), and he helped develop the society’s Minority Recruitment Initiative.

What’s next for Dr. Flowers? For one, he plans to continue working as a mentor; he received the ASH Mentor Award in honor of his service in 2022. “I am strongly committed to increasing the number of tenure-track investigators trained in clinical and translational cancer research and to promote their career development.”

And he looks forward to helping develop MD Anderson’s recently announced $2.5 billion hospital in Austin. “This will extend the exceptional care that we provide as the No. 1 cancer center in the United States,” he said. “It will also create new opportunities for research and collaboration with experts at UT Austin.”

When he’s not in clinic, Dr. Flowers embraces his lifelong love of speeding through life on his own two feet. He’s even inspired his children to share his passion. “I run most days of the week,” he said. “Running provides a great opportunity to think and process new research ideas, work through leadership challenges, and sometimes just to relax and let go of the day.”

Back in 1997, a young Stanford (Calif.) University student named Christopher Flowers made his debut in the medical literature with a Nature Medicine report that called out the pharmaceutical industry for overlooking the crucial role of clinical investigators in drug development.

“My research uncovered a series of physicians who served as ‘clinical champions’ and dramatically sped the process of drug development,” Dr. Flowers recalled in an interview. “This early career research inspired me to become the type of clinical champion that I uncovered.”

MD Anderson Cancer Center

Over his career, hematologist-oncologist Dr. Flowers has developed lifesaving therapies for lymphoma, which has transformed into a highly treatable and even curable disease. He’s listed as a coauthor of hundreds of peer-reviewed cancer studies, reports, and medical society guidelines. And he’s revealed stark disparities in blood cancer care: His research shows that non-White patients suffer from worse outcomes, regardless of factors like income and insurance coverage.

The University of Texas MD Anderson Cancer Center, Houston, recently named physician-scientist Dr. Flowers as division head of cancer medicine, a position he’s held on an interim basis. As of Sept. 1, he will permanently oversee 300 faculty and more than 2,000 staff members.
 

A running start in Seattle

For Dr. Flowers, track and field is a sport that runs in the family. His grandfather was a top runner in both high school and college, and both Dr. Flowers and his brother ran competitively in Seattle, where they grew up. But Dr. Flowers chose a career in oncology, earning a medical degree at Stanford and master’s degrees at both Stanford and the University of Washington, Seattle.

The late Kenneth Melmon, MD, a groundbreaking pharmacologist, was a major influence. “He was one of the first people that I met when I began as an undergraduate at Stanford. We grew to be long-standing friends, and he demonstrated what outstanding mentorship looks like. In our research collaboration, we investigated the work of Dr. Gertrude Elion and Dr. George Hitchings involving the translation of pharmacological data from cellular and animal models to clinically useful drugs including 6-mercaptopurine, allopurinol, azathioprine, acyclovir, and zidovudine.”

The late Oliver Press, MD, a blood cancer specialist, inspired Dr. Flower’s interest in lymphoma. “I began work with him during an internship at the University of Washington. Ollie was a great inspiration and a key leader in the development of innovative therapies for lymphoma. He embodied the role of a clinical champion translating work in radioimmunotherapy to new therapeutics for patients with lymphomas. Working with him ultimately led me to pursue a career in hematology and oncology with a focus on the care for patients with lymphomas.”
 

Career blooms as lymphoma care advances

Dr. Flowers went on to Emory University, Atlanta, where he served as scientific director of the Research Informatics Shared Resource and a faculty member in the department of biomedical informatics. “I applied my training in informatics and my clinical expertise to support active grants from the Burroughs Wellcome Fund for Innovation in Regulatory Science and from the National Cancer Institute to develop informatics tools for pathology image analysis and prognostic modeling.”

For 13 years, he also served the Winship Cancer Institute as director of the Emory Healthcare lymphoma program (where his patients included Kansas City Chiefs football star Eric Berry), and for 4 years as scientific director of research informatics. Meanwhile, Dr. Flowers helped develop national practice guidelines for the American Society of Clinical Oncology, the American Cancer Society, and the American College of Radiology. He also chaired the ASCO guideline on management of febrile neutropenia.

In 2019, MD Anderson hired Dr. Flowers as chair of the department of lymphoma/myeloma. A year later, he was appointed division head ad interim for cancer medicine.

“Chris is a unique leader who expertly combines mentorship, sponsorship, and bidirectional open, honest communication,” said Sairah Ahmed, MD, associate professor of lymphoma at MD Anderson. “He doesn’t just empower his team to reach their goals. He also inspires those around him to turn vision into reality.”

As Dr. Flowers noted, many patients with lymphoma are now able to recover and live normal lives. He himself played a direct role himself in boosting lifespans.

“I have been fortunate to play a role in the development of several treatments that have led to advances in first-line therapy for patients with aggressive lymphomas. I partnered with others at MD Anderson, including Dr. Sattva Neelapu and Dr. Jason Westin, who have developed novel therapies like chimeric antigen receptor T-cell therapy for patients with relapse lymphomas,” he said. “Leaders in the field at MD Anderson like Dr. Michael Wang have developed new oral treatments for patients with rare lymphoma subtypes like mantle cell lymphoma. Other colleagues such as Dr. Nathan Fowler and Dr. Loretta Nastoupil have focused on the care for patients with indolent lymphomas and developed less-toxic therapies that are now in common use.”
 

Exposing the disparities in blood cancer care

Dr. Flowers, who’s African American, has also been a leader in health disparity research. In 2016, for example, he was coauthor of a study into non-Hodgkin’s lymphoma that revealed that Blacks in the United States have dramatically lower survival rates than Whites. The 10-year survival rate for Black women with chronic lymphocytic leukemia was just 47%, for example, compared with 66% for White females. “Although incidence rates of lymphoid neoplasms are generally higher among Whites, Black men tend to have poorer survival,” Dr. Flowers and colleagues wrote.

In a 2021 report for the ASCO Educational Book, Dr. Flowers and hematologist-oncologist Demetria Smith-Graziani, MD, now with Emory University, explored disparities across blood cancers and barriers to minority enrollment in clinical trials. “Some approaches that clinicians can apply to address these disparities include increasing systems-level awareness, improving access to care, and reducing biases in clinical setting,” the authors wrote.

Luis Malpica Castillo, MD, assistant professor of lymphoma at MD Anderson Cancer Center, lauded the work of Dr. Flowers in expanding opportunities for minority patients with the disease.

“During the past years, Dr. Flowers’ work has not only had a positive impact on the Texan community, but minority populations living with cancer in the United States and abroad,” he said. “Currently, we are implementing cancer care networks aimed to increase diversity in clinical trials by enrolling a larger number of Hispanic and African American patients, who otherwise may not have benefited from novel therapies. The ultimate goal is to provide high-quality care to all patients living with cancer.”

In addition to his research work, Dr. Flowers is an advocate for diversity within the hematology community. He’s a founding member and former chair of the American Society of Hematology’s Committee on Diversity, Equity and Inclusion (formerly the Committee on Promoting Diversity), and he helped develop the society’s Minority Recruitment Initiative.

What’s next for Dr. Flowers? For one, he plans to continue working as a mentor; he received the ASH Mentor Award in honor of his service in 2022. “I am strongly committed to increasing the number of tenure-track investigators trained in clinical and translational cancer research and to promote their career development.”

And he looks forward to helping develop MD Anderson’s recently announced $2.5 billion hospital in Austin. “This will extend the exceptional care that we provide as the No. 1 cancer center in the United States,” he said. “It will also create new opportunities for research and collaboration with experts at UT Austin.”

When he’s not in clinic, Dr. Flowers embraces his lifelong love of speeding through life on his own two feet. He’s even inspired his children to share his passion. “I run most days of the week,” he said. “Running provides a great opportunity to think and process new research ideas, work through leadership challenges, and sometimes just to relax and let go of the day.”

Back in 1997, a young Stanford (Calif.) University student named Christopher Flowers made his debut in the medical literature with a Nature Medicine report that called out the pharmaceutical industry for overlooking the crucial role of clinical investigators in drug development.

“My research uncovered a series of physicians who served as ‘clinical champions’ and dramatically sped the process of drug development,” Dr. Flowers recalled in an interview. “This early career research inspired me to become the type of clinical champion that I uncovered.”

MD Anderson Cancer Center

Over his career, hematologist-oncologist Dr. Flowers has developed lifesaving therapies for lymphoma, which has transformed into a highly treatable and even curable disease. He’s listed as a coauthor of hundreds of peer-reviewed cancer studies, reports, and medical society guidelines. And he’s revealed stark disparities in blood cancer care: His research shows that non-White patients suffer from worse outcomes, regardless of factors like income and insurance coverage.

The University of Texas MD Anderson Cancer Center, Houston, recently named physician-scientist Dr. Flowers as division head of cancer medicine, a position he’s held on an interim basis. As of Sept. 1, he will permanently oversee 300 faculty and more than 2,000 staff members.
 

A running start in Seattle

For Dr. Flowers, track and field is a sport that runs in the family. His grandfather was a top runner in both high school and college, and both Dr. Flowers and his brother ran competitively in Seattle, where they grew up. But Dr. Flowers chose a career in oncology, earning a medical degree at Stanford and master’s degrees at both Stanford and the University of Washington, Seattle.

The late Kenneth Melmon, MD, a groundbreaking pharmacologist, was a major influence. “He was one of the first people that I met when I began as an undergraduate at Stanford. We grew to be long-standing friends, and he demonstrated what outstanding mentorship looks like. In our research collaboration, we investigated the work of Dr. Gertrude Elion and Dr. George Hitchings involving the translation of pharmacological data from cellular and animal models to clinically useful drugs including 6-mercaptopurine, allopurinol, azathioprine, acyclovir, and zidovudine.”

The late Oliver Press, MD, a blood cancer specialist, inspired Dr. Flower’s interest in lymphoma. “I began work with him during an internship at the University of Washington. Ollie was a great inspiration and a key leader in the development of innovative therapies for lymphoma. He embodied the role of a clinical champion translating work in radioimmunotherapy to new therapeutics for patients with lymphomas. Working with him ultimately led me to pursue a career in hematology and oncology with a focus on the care for patients with lymphomas.”
 

Career blooms as lymphoma care advances

Dr. Flowers went on to Emory University, Atlanta, where he served as scientific director of the Research Informatics Shared Resource and a faculty member in the department of biomedical informatics. “I applied my training in informatics and my clinical expertise to support active grants from the Burroughs Wellcome Fund for Innovation in Regulatory Science and from the National Cancer Institute to develop informatics tools for pathology image analysis and prognostic modeling.”

For 13 years, he also served the Winship Cancer Institute as director of the Emory Healthcare lymphoma program (where his patients included Kansas City Chiefs football star Eric Berry), and for 4 years as scientific director of research informatics. Meanwhile, Dr. Flowers helped develop national practice guidelines for the American Society of Clinical Oncology, the American Cancer Society, and the American College of Radiology. He also chaired the ASCO guideline on management of febrile neutropenia.

In 2019, MD Anderson hired Dr. Flowers as chair of the department of lymphoma/myeloma. A year later, he was appointed division head ad interim for cancer medicine.

“Chris is a unique leader who expertly combines mentorship, sponsorship, and bidirectional open, honest communication,” said Sairah Ahmed, MD, associate professor of lymphoma at MD Anderson. “He doesn’t just empower his team to reach their goals. He also inspires those around him to turn vision into reality.”

As Dr. Flowers noted, many patients with lymphoma are now able to recover and live normal lives. He himself played a direct role himself in boosting lifespans.

“I have been fortunate to play a role in the development of several treatments that have led to advances in first-line therapy for patients with aggressive lymphomas. I partnered with others at MD Anderson, including Dr. Sattva Neelapu and Dr. Jason Westin, who have developed novel therapies like chimeric antigen receptor T-cell therapy for patients with relapse lymphomas,” he said. “Leaders in the field at MD Anderson like Dr. Michael Wang have developed new oral treatments for patients with rare lymphoma subtypes like mantle cell lymphoma. Other colleagues such as Dr. Nathan Fowler and Dr. Loretta Nastoupil have focused on the care for patients with indolent lymphomas and developed less-toxic therapies that are now in common use.”
 

Exposing the disparities in blood cancer care

Dr. Flowers, who’s African American, has also been a leader in health disparity research. In 2016, for example, he was coauthor of a study into non-Hodgkin’s lymphoma that revealed that Blacks in the United States have dramatically lower survival rates than Whites. The 10-year survival rate for Black women with chronic lymphocytic leukemia was just 47%, for example, compared with 66% for White females. “Although incidence rates of lymphoid neoplasms are generally higher among Whites, Black men tend to have poorer survival,” Dr. Flowers and colleagues wrote.

In a 2021 report for the ASCO Educational Book, Dr. Flowers and hematologist-oncologist Demetria Smith-Graziani, MD, now with Emory University, explored disparities across blood cancers and barriers to minority enrollment in clinical trials. “Some approaches that clinicians can apply to address these disparities include increasing systems-level awareness, improving access to care, and reducing biases in clinical setting,” the authors wrote.

Luis Malpica Castillo, MD, assistant professor of lymphoma at MD Anderson Cancer Center, lauded the work of Dr. Flowers in expanding opportunities for minority patients with the disease.

“During the past years, Dr. Flowers’ work has not only had a positive impact on the Texan community, but minority populations living with cancer in the United States and abroad,” he said. “Currently, we are implementing cancer care networks aimed to increase diversity in clinical trials by enrolling a larger number of Hispanic and African American patients, who otherwise may not have benefited from novel therapies. The ultimate goal is to provide high-quality care to all patients living with cancer.”

In addition to his research work, Dr. Flowers is an advocate for diversity within the hematology community. He’s a founding member and former chair of the American Society of Hematology’s Committee on Diversity, Equity and Inclusion (formerly the Committee on Promoting Diversity), and he helped develop the society’s Minority Recruitment Initiative.

What’s next for Dr. Flowers? For one, he plans to continue working as a mentor; he received the ASH Mentor Award in honor of his service in 2022. “I am strongly committed to increasing the number of tenure-track investigators trained in clinical and translational cancer research and to promote their career development.”

And he looks forward to helping develop MD Anderson’s recently announced $2.5 billion hospital in Austin. “This will extend the exceptional care that we provide as the No. 1 cancer center in the United States,” he said. “It will also create new opportunities for research and collaboration with experts at UT Austin.”

When he’s not in clinic, Dr. Flowers embraces his lifelong love of speeding through life on his own two feet. He’s even inspired his children to share his passion. “I run most days of the week,” he said. “Running provides a great opportunity to think and process new research ideas, work through leadership challenges, and sometimes just to relax and let go of the day.”

TOPLINE:

More than 3.6 million women of reproductive age lack access to fertility clinics offering standard fertility preservation services before cancer treatment, a study shows.

METHODOLOGY:

• In this cross-sectional analysis, researchers identified 370 fertility centers in the United States (361 in the continental U.S.) that provide oncofertility services and that met criteria for an oncofertility center.
• Clinics were considered oncofertility centers if they offered oocyte cryopreservation, had performed at least one fertility preservation cycle in 2018, reported serving people without partners, and had an embryology laboratory that was accredited per Centers for Disease Control and Prevention guidelines.
• Researchers then quantified the number of young women potentially eligible for oncofertility services who lived farther than 2 hours from an oncofertility center.
• In a secondary analysis, the team assessed the association between geographic access and state fertility preservation insurance mandates.

TAKEAWAY:

• Overall, 3.63 million (5.7%) young women of reproductive age in the United States (aged 15-4) live more than 2 hours from an oncofertility center.
• The greatest gaps in access are in the Mountain West, West North Central, and Southwest regions; for instance, in Montana, North Dakota, and Wyoming fewer than half of the at-risk population has geographic access to an oncofertility center.
• Among the 11 states with fertility preservation insurance mandates, 98.5% of at-risk women have geographic access to an oncofertility center; in the 17 states without fertility preservation legislation, 79.6% of at-risk women have access to an oncofertility center.

IN PRACTICE:

Just over 3.6 million “reproductive-age female individuals lack geographic access to oncofertility services, especially in the Mountain West and West North Central regions,” the authors concluded. “Significant geographic disparities in access to fertility preservation in the U.S. require strategic expansion of care, especially given the growing demand for oncofertility services.”

SOURCE:

The study, led by Benjamin Peipert, MD, with Duke University, Durham, N.C., was published online in JAMA Oncology.

LIMITATIONS:

The authors relied on clinic data reported to the CDC, made assumptions about reasonable travel time, and may have underestimated access in some areas.

DISCLOSURES:

The study had no commercial funding. The authors have disclosed no relevant financial relationships.

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

TOPLINE:

More than 3.6 million women of reproductive age lack access to fertility clinics offering standard fertility preservation services before cancer treatment, a study shows.

METHODOLOGY:

• In this cross-sectional analysis, researchers identified 370 fertility centers in the United States (361 in the continental U.S.) that provide oncofertility services and that met criteria for an oncofertility center.
• Clinics were considered oncofertility centers if they offered oocyte cryopreservation, had performed at least one fertility preservation cycle in 2018, reported serving people without partners, and had an embryology laboratory that was accredited per Centers for Disease Control and Prevention guidelines.
• Researchers then quantified the number of young women potentially eligible for oncofertility services who lived farther than 2 hours from an oncofertility center.
• In a secondary analysis, the team assessed the association between geographic access and state fertility preservation insurance mandates.

TAKEAWAY:

• Overall, 3.63 million (5.7%) young women of reproductive age in the United States (aged 15-4) live more than 2 hours from an oncofertility center.
• The greatest gaps in access are in the Mountain West, West North Central, and Southwest regions; for instance, in Montana, North Dakota, and Wyoming fewer than half of the at-risk population has geographic access to an oncofertility center.
• Among the 11 states with fertility preservation insurance mandates, 98.5% of at-risk women have geographic access to an oncofertility center; in the 17 states without fertility preservation legislation, 79.6% of at-risk women have access to an oncofertility center.

IN PRACTICE:

Just over 3.6 million “reproductive-age female individuals lack geographic access to oncofertility services, especially in the Mountain West and West North Central regions,” the authors concluded. “Significant geographic disparities in access to fertility preservation in the U.S. require strategic expansion of care, especially given the growing demand for oncofertility services.”

SOURCE:

The study, led by Benjamin Peipert, MD, with Duke University, Durham, N.C., was published online in JAMA Oncology.

LIMITATIONS:

The authors relied on clinic data reported to the CDC, made assumptions about reasonable travel time, and may have underestimated access in some areas.

DISCLOSURES:

The study had no commercial funding. The authors have disclosed no relevant financial relationships.

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

TOPLINE:

More than 3.6 million women of reproductive age lack access to fertility clinics offering standard fertility preservation services before cancer treatment, a study shows.

METHODOLOGY:

• In this cross-sectional analysis, researchers identified 370 fertility centers in the United States (361 in the continental U.S.) that provide oncofertility services and that met criteria for an oncofertility center.
• Clinics were considered oncofertility centers if they offered oocyte cryopreservation, had performed at least one fertility preservation cycle in 2018, reported serving people without partners, and had an embryology laboratory that was accredited per Centers for Disease Control and Prevention guidelines.
• Researchers then quantified the number of young women potentially eligible for oncofertility services who lived farther than 2 hours from an oncofertility center.
• In a secondary analysis, the team assessed the association between geographic access and state fertility preservation insurance mandates.

TAKEAWAY:

• Overall, 3.63 million (5.7%) young women of reproductive age in the United States (aged 15-4) live more than 2 hours from an oncofertility center.
• The greatest gaps in access are in the Mountain West, West North Central, and Southwest regions; for instance, in Montana, North Dakota, and Wyoming fewer than half of the at-risk population has geographic access to an oncofertility center.
• Among the 11 states with fertility preservation insurance mandates, 98.5% of at-risk women have geographic access to an oncofertility center; in the 17 states without fertility preservation legislation, 79.6% of at-risk women have access to an oncofertility center.

IN PRACTICE:

Just over 3.6 million “reproductive-age female individuals lack geographic access to oncofertility services, especially in the Mountain West and West North Central regions,” the authors concluded. “Significant geographic disparities in access to fertility preservation in the U.S. require strategic expansion of care, especially given the growing demand for oncofertility services.”

SOURCE:

The study, led by Benjamin Peipert, MD, with Duke University, Durham, N.C., was published online in JAMA Oncology.

LIMITATIONS:

The authors relied on clinic data reported to the CDC, made assumptions about reasonable travel time, and may have underestimated access in some areas.

DISCLOSURES:

The study had no commercial funding. The authors have disclosed no relevant financial relationships.

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

Among patients with female pattern hair loss taking low-dose oral minoxidil (LDOM) for at least 4 months, minimal changes from baseline were observed in systolic blood pressure, diastolic blood pressure, and heart rate, results from a small retrospective analysis showed.

“Additionally, few patients experienced hair loss progression while slightly over a third experienced hair regrowth,” the study’s first author, Reese Imhof, MD, a third-year resident in the department of dermatology at Mayo Clinic, Rochester, Minn., said in an interview. The results were published online in JAAD International.

At low doses, oral minoxidil, approved as an antihypertensive over 40 years ago, has become an increasingly popular treatment for hair loss, particularly since an article about its use for hair loss was published in the New York Times in August 2022. (Oral minoxidil is not approved for treating alopecia, and is used off label for this purpose.)

To evaluate the effects of LDOM in female patients with female pattern hair loss, Dr. Imhof, along with colleagues Beija Villalpando, MD, of the department of medicine and Rochelle R. Torgerson, MD, PhD, of the department of dermatology at the Mayo Clinic, reviewed the records of 25 adult women who were evaluated for female pattern hair loss at the Mayo Clinic over a 5-year period that ended on Nov. 27, 2022. Previous studies have looked at the cardiovascular effects of treatment with oral minoxidil and impact on BP in men, but “few studies have reported on female patients receiving LDOM as monotherapy for female pattern hair loss,” the authors noted.

The mean age of the women in their study was 61 years, and they took LDOM for a mean of 6.2 months. Slightly more than half (52%) took a dose of 1.25 mg daily, while 40% took 2.5 mg daily and 8% took 0.625 mg daily.

Of the 25 patients, 10 (40%) had previously tried topical minoxidil but had discontinued it because of local side effects or challenges with adherence. Also, three patients (12%) had previously tried finasteride and spironolactone but discontinued those medications because of adverse side effects.

The researchers noted disease improvement and hair regrowth was observed in nine patients who were treated with LDOM (36%), while three patients (12%) had “unaltered disease progression.” Adverse side effects observed in the cohort included four patients with facial hypertrichosis (16%) and one patient with fluid retention/lower limb edema (4%).

The patients who developed hypertrichosis did not discontinue LDOM, but the patient who developed edema did stop treatment.

At baseline, systolic BP (SBP) ranged from 107-161 mm Hg, diastolic BP (DBP) ranged from 58-88 mm Hg, and heart rate ranged from 54-114 beats per minute. Post treatment, SBP ranged from 102-152 mm Hg, DBP ranged from 63-90 mm Hg, and heart rate ranged from 56 to 105 bpm. “It was surprising how little ambulatory blood pressure and heart rate changed after an average of 6 months of treatment,” Dr. Imhof said in an interview. “On average, SBP decreased by 2.8 mm HG while DBP decreased by 1.4 mm Hg. Heart rate increased an average of 4.4 beats per minute.”

He acknowledged certain limitations of the study, including its small sample size and lack of inclusion of patients who were being treated for hypertension with concomitant antihypertensive medications. “Some unique aspects of our study are that we focused on women, and we had a slightly older cohort than prior studies (61 years old on average) as well as exposure to higher doses of LDOM, with most patients on either 1.25 mg daily or 2.5 mg daily,” Dr. Imhof said.

The researchers reported having no relevant disclosures, and there was no funding source for the study.

Among patients with female pattern hair loss taking low-dose oral minoxidil (LDOM) for at least 4 months, minimal changes from baseline were observed in systolic blood pressure, diastolic blood pressure, and heart rate, results from a small retrospective analysis showed.

“Additionally, few patients experienced hair loss progression while slightly over a third experienced hair regrowth,” the study’s first author, Reese Imhof, MD, a third-year resident in the department of dermatology at Mayo Clinic, Rochester, Minn., said in an interview. The results were published online in JAAD International.

At low doses, oral minoxidil, approved as an antihypertensive over 40 years ago, has become an increasingly popular treatment for hair loss, particularly since an article about its use for hair loss was published in the New York Times in August 2022. (Oral minoxidil is not approved for treating alopecia, and is used off label for this purpose.)

To evaluate the effects of LDOM in female patients with female pattern hair loss, Dr. Imhof, along with colleagues Beija Villalpando, MD, of the department of medicine and Rochelle R. Torgerson, MD, PhD, of the department of dermatology at the Mayo Clinic, reviewed the records of 25 adult women who were evaluated for female pattern hair loss at the Mayo Clinic over a 5-year period that ended on Nov. 27, 2022. Previous studies have looked at the cardiovascular effects of treatment with oral minoxidil and impact on BP in men, but “few studies have reported on female patients receiving LDOM as monotherapy for female pattern hair loss,” the authors noted.

The mean age of the women in their study was 61 years, and they took LDOM for a mean of 6.2 months. Slightly more than half (52%) took a dose of 1.25 mg daily, while 40% took 2.5 mg daily and 8% took 0.625 mg daily.

Of the 25 patients, 10 (40%) had previously tried topical minoxidil but had discontinued it because of local side effects or challenges with adherence. Also, three patients (12%) had previously tried finasteride and spironolactone but discontinued those medications because of adverse side effects.

The researchers noted disease improvement and hair regrowth was observed in nine patients who were treated with LDOM (36%), while three patients (12%) had “unaltered disease progression.” Adverse side effects observed in the cohort included four patients with facial hypertrichosis (16%) and one patient with fluid retention/lower limb edema (4%).

The patients who developed hypertrichosis did not discontinue LDOM, but the patient who developed edema did stop treatment.

At baseline, systolic BP (SBP) ranged from 107-161 mm Hg, diastolic BP (DBP) ranged from 58-88 mm Hg, and heart rate ranged from 54-114 beats per minute. Post treatment, SBP ranged from 102-152 mm Hg, DBP ranged from 63-90 mm Hg, and heart rate ranged from 56 to 105 bpm. “It was surprising how little ambulatory blood pressure and heart rate changed after an average of 6 months of treatment,” Dr. Imhof said in an interview. “On average, SBP decreased by 2.8 mm HG while DBP decreased by 1.4 mm Hg. Heart rate increased an average of 4.4 beats per minute.”

He acknowledged certain limitations of the study, including its small sample size and lack of inclusion of patients who were being treated for hypertension with concomitant antihypertensive medications. “Some unique aspects of our study are that we focused on women, and we had a slightly older cohort than prior studies (61 years old on average) as well as exposure to higher doses of LDOM, with most patients on either 1.25 mg daily or 2.5 mg daily,” Dr. Imhof said.

The researchers reported having no relevant disclosures, and there was no funding source for the study.

Among patients with female pattern hair loss taking low-dose oral minoxidil (LDOM) for at least 4 months, minimal changes from baseline were observed in systolic blood pressure, diastolic blood pressure, and heart rate, results from a small retrospective analysis showed.

“Additionally, few patients experienced hair loss progression while slightly over a third experienced hair regrowth,” the study’s first author, Reese Imhof, MD, a third-year resident in the department of dermatology at Mayo Clinic, Rochester, Minn., said in an interview. The results were published online in JAAD International.

At low doses, oral minoxidil, approved as an antihypertensive over 40 years ago, has become an increasingly popular treatment for hair loss, particularly since an article about its use for hair loss was published in the New York Times in August 2022. (Oral minoxidil is not approved for treating alopecia, and is used off label for this purpose.)

To evaluate the effects of LDOM in female patients with female pattern hair loss, Dr. Imhof, along with colleagues Beija Villalpando, MD, of the department of medicine and Rochelle R. Torgerson, MD, PhD, of the department of dermatology at the Mayo Clinic, reviewed the records of 25 adult women who were evaluated for female pattern hair loss at the Mayo Clinic over a 5-year period that ended on Nov. 27, 2022. Previous studies have looked at the cardiovascular effects of treatment with oral minoxidil and impact on BP in men, but “few studies have reported on female patients receiving LDOM as monotherapy for female pattern hair loss,” the authors noted.

The mean age of the women in their study was 61 years, and they took LDOM for a mean of 6.2 months. Slightly more than half (52%) took a dose of 1.25 mg daily, while 40% took 2.5 mg daily and 8% took 0.625 mg daily.

Of the 25 patients, 10 (40%) had previously tried topical minoxidil but had discontinued it because of local side effects or challenges with adherence. Also, three patients (12%) had previously tried finasteride and spironolactone but discontinued those medications because of adverse side effects.

The researchers noted disease improvement and hair regrowth was observed in nine patients who were treated with LDOM (36%), while three patients (12%) had “unaltered disease progression.” Adverse side effects observed in the cohort included four patients with facial hypertrichosis (16%) and one patient with fluid retention/lower limb edema (4%).

The patients who developed hypertrichosis did not discontinue LDOM, but the patient who developed edema did stop treatment.

At baseline, systolic BP (SBP) ranged from 107-161 mm Hg, diastolic BP (DBP) ranged from 58-88 mm Hg, and heart rate ranged from 54-114 beats per minute. Post treatment, SBP ranged from 102-152 mm Hg, DBP ranged from 63-90 mm Hg, and heart rate ranged from 56 to 105 bpm. “It was surprising how little ambulatory blood pressure and heart rate changed after an average of 6 months of treatment,” Dr. Imhof said in an interview. “On average, SBP decreased by 2.8 mm HG while DBP decreased by 1.4 mm Hg. Heart rate increased an average of 4.4 beats per minute.”

He acknowledged certain limitations of the study, including its small sample size and lack of inclusion of patients who were being treated for hypertension with concomitant antihypertensive medications. “Some unique aspects of our study are that we focused on women, and we had a slightly older cohort than prior studies (61 years old on average) as well as exposure to higher doses of LDOM, with most patients on either 1.25 mg daily or 2.5 mg daily,” Dr. Imhof said.

The researchers reported having no relevant disclosures, and there was no funding source for the study.

Pediatric oncologist Lena Winestone, MD, recalls treating a 2-year-old leukemia patient who underwent a bone marrow transplant as her only chance for a cure.

The girl’s family, who spoke only Spanish and struggled with literacy, could not pay their rent or afford the girl’s weekly transportation to the hospital for after-transplant care. The family had three other children and lived more than 2 hours from the transplant center, remembers Dr. Winestone, an assistant professor of pediatrics in the division of malignancies and bone & marrow transplant at the University of California, San Francisco.

The hospital’s social worker was able to secure grant support for the family’s housing and worked with the patient’s insurance to arrange for transportation. However, the departure times were rigid, Dr. Winestone said, and the family sometimes had to leave the hospital before the child’s graft vs. host disease (GvHD) treatment was complete for the day. 

“If we had not finished her treatment, we had to disconnect her from the machine early,” Dr. Winestone said. “Her mother also had to load her oxygen tanks [three of them], her BiPAP machine, and her tube feeds into the transportation every week in order to make sure she could be safely transported. She was treated for GvHD for almost 2 years, but unfortunately, her GvHD started to affect her lungs and ultimately, she passed away.”

Dr. Winestone says it’s difficult to know whether the girl’s death was directly related to her socioeconomic status, but that it certainly made all aspects of the child’s care more complicated and forced health care providers to adapt her cancer care to accommodate the family’s circumstances.

This story is one of countless cases where socioeconomic status impacted a young patient’s cancer care and likely contributed to a worse outcome. A plethora of data has demonstrated that children with cancer who are Black, Hispanic, or of lower socioeconomic status are more likely to relapse and die even when treated uniformly on clinical trials.

A 2022 study for example, found that children from marginalized racial/ethnic groups and those living in poverty were more likely to have inferior 5-year overall survival, compared with other children, even when assigned to receive the same initial treatment. Of 696 children with high-risk neuroblastoma, 47% of Hispanic children had a 5-year overall survival (OS), compared with 50% for other non-Hispanic children, and 61% for white non-Hispanic patients. Children on public health insurance (a proxy for household poverty) had a 53% 5-year OS, compared with 63% for children unexposed to household poverty. Pediatric patients exposed to neighborhood poverty had a 54% 5-year OS, compared with 62% for unexposed children.

In another study, children with acute lymphoblastic leukemia who lived in high-poverty areas were more likely to experience early relapse than other patients, despite having the same treatment. Of the 575 children studied, 92% of children from high-poverty areas who relapsed, experienced early relapse, defined as less than 36 months after remission. By comparison, only 48% of other children who relapsed experienced early relapse.

Reasons behind the relapse and survival disparities are multifold, which has led to challenges in addressing the gaps and improving cancer outcomes for poverty-stricken children. A research infrastructure that is largely based on biological, rather than social determinants of health, acts as another barrier, oncologists say.

Historically, interventions to address disparities in pediatric oncology have never been evaluated, said Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. This is in large part because the body of literature illustrating the disparities is relatively new, said Dr. Bona, whose research focuses on poverty-associated outcome disparities in childhood cancer.

However, new efforts aim to change this landscape by using the growing data to develop and analyze possible interventions. A set of three novel interventions led by Dr. Bona and her research team are in the works, some of which have shown promise in early studies.

“Now is the time to begin to actively intervene on disparities in childhood cancer,” Dr. Bona said. “We’re really good at studying genetic mutations in cancer cells that might lead to a risk of relapse, and when we identify those mutations, what we do is intervene. We try new chemotherapy agents, new ways of delivering therapy. We are now at the point where we have identified that social determinants of health may be equally ‘risky’ but we haven’t taken the next step to begin intervening in the same way.”
 

What is causing disparities in pediatric cancer outcomes?

Lack of access to the health care system is a top contributor to the disparities, although there is no single root cause, said Sharon Castellino, MD, director of the Leukemia and Lymphoma Program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta, and a professor in the department of pediatrics at Emory University, Atlanta.

Even before cancer diagnosis, Dr. Castellino notes that many children of color and/or of lower socioeconomic status are not receiving regular health care, leading to sicker children and more advanced-stage cancer by the time they are diagnosed.

Lack of insurance is a primary barrier to this access, adds Xu Ji, PhD, MSPH, an assistant professor in the department of pediatrics at Emory University and a member of the Cancer Prevention and Control Research Program at the university’s Winship Cancer Institute.

Studies  have long shown that uninsured children are more likely to go without needed care, compared with those with private insurance. Patients of color are at much higher risk of being uninsured than White patients, with the uninsured rates for Hispanic, American Indian, and Alaska Native patients being more than 2.5 times higher than that of White patients.

“We all know that insurance is a strong predictor of health outcomes,” said Dr. Ji, whose research focuses on insurance disparities and gains among cancer patients. “Lack of insurance coverage and therefore lack of access to care along the pediatric cancer continuum from early detection to early diagnosis to timely initiation of treatment to receipt of high-quality treatment to access to recommended survivorship care and even access to palliative and end-of-life care are all very important constructs in the pathway from poverty to ultimate cancer outcomes for children.”

Unstable housing, employment difficulties, and lack of family support can also come into play. Dr. Castellino remembers the case of a 12-year-old cancer patient who entered treatment with advanced-stage Hodgkin Lymphoma. The girl came from a low-income, single-parent household without stable housing. Dr. Castellino said when the child was granted a wish from the Make-a-Wish Foundation, she asked for her own bed.

“We had been working with her every week for 6 months when that request came up,” she recalled. “We said, ‘You don’t have to wait for your make-a-wish, we can get you a bed now.’ We don’t even know the extent of what happens at home for many of these children.”

The impact of toxic stress on child cancer patients is an emerging area of research, said Dr. Winestone, whose research explores racial, ethnic, and socioeconomic disparities in access to care and outcomes of leukemia and lymphoma treatment. For example, Dr. Winestone’s research includes understanding how exposure to poverty or adverse experiences in childhood may influence a patient’s biological response to chemotherapy.

Other contributors to disparities include transportation issues, lack of childcare for other children, literacy, and language barriers. A 2016 study  suggests that language barriers negatively impact the quality of informed decision-making and the care experience for Spanish-speaking parents of pediatric cancer patients with limited English proficiency.

Such access issues are also compounded by systemic factors, including a shortage of physicians of color who may be able to forge better trust relationships with families of similar race and ethnicity, Dr. Castellino adds. Lower enrollment of pediatric cancer patients with higher social vulnerabilities in clinical trials is another problem.

“In childhood cancer, I believe our improvements have been built on the backs of prior generations of families and children who have enrolled in trials. We learn things, and the next generation of therapy improves,” Dr. Castellino said. “If you have a whole group of the population not represented in trials, you don’t know what’s driving the fact they may or may not improve.”
 

Working toward solutions  

With such a diverse set of factors fueling outcome gaps, a similarly diverse approach is needed to help bridge the divide, say disparity researchers.

To this end, Dr. Bona and her research team are currently building the first portfolio of health equity interventions, each designed to address a different adverse social determinant of health differently.

The Pediatric Cancer Resource Equity (PediCARE) intervention is a centrally delivered, household material hardship (HMH)–targeted intervention that provides transportation and groceries to low-income pediatric oncology families. The intervention was recently studied in a pilot, randomized, controlled trial at Dana-Farber Cancer Institute and the University of Alabama between May 2019 and August 2021.  

Families were first screened for HMH and randomized into receiving either the intervention or usual care for 6 months. The intervention group received groceries via Instacart and transportation to and from the hospital coordinated through the Ride Health platform using Uber or Lyft. For families with their own cars, gas cards were provided. Of the families offered the chance to participate, 100% agreed to participate in the program, and there was 0% attrition in either arm of the program during the 6 months, according to the study findings, which were presented at the 2023 American Society of Clinical Oncology annual meeting in June.   

Among families who received the PediCARE intervention, 100% successfully received grocery and transportation resources, 100% reported that it was “easier to buy food for my family,” 85% reported it was easier to get to and from the hospital, and 95% reported they would be “very likely to recommend the intervention to other families,” according to the results.

“The key takeaway is that we had excellent feasibility outcomes,” said Haley Newman, MD, lead author of the study and an attending physician in the division of oncology at The Children’s Hospital of Philadelphia. “From this study, we learned that PediCARE is accessible and feasible in very diverse settings. From this, what we really took away is that PediCARE could be successfully rolled out in a phase 3 randomized trial, which would be the best way to examine efficacy.”

Another initiative in its early stages is Pediatric RISE, a guaranteed income intervention being developed with support from the Children’s Cancer Research Fund, the American Cancer Society, and other donors. The intervention will provide unrestricted cash transfers to low-income families during the early months of chemotherapy, Dr. Bona said. Families are currently being enrolled in a pilot study with a goal of refining the intervention before it’s tested for feasibility and efficacy.

“The goal here is ultimately to evaluate the question: If we are able to successfully provide income support to low-income families going through childhood cancer treatment, might we be able to ameliorate some of the disparities associated with living in poverty that we have already described in childhood cancer,” Dr. Bona said.

Pediatric Assist, a developing intervention centering on benefits, is a third initiative that will soon be evaluated. The intervention will provide newly diagnosed families with systemic access to a centralized benefits counselor who can help them determine which existing government benefits they might be eligible for and assist them in navigating the application process.

“The idea here is that we know many lower-income families in the U.S. are eligible for existing supports, but may not be accessing them because of how incredibly difficult the system is to navigate,” Dr. Bona said. “For example, we know that low-income families may be eligible for SNAP benefits, but figuring out if you are eligible and then applying for SNAP involves multiple, complicated steps that are often infeasible for a parent when their child is admitted to the hospital with a newly diagnosed, life-threatening illness.”

Pilot refinement of the intervention is expected in the fall of 2023.
 

Overcoming barriers, addressing challenges

Investigators are also making headway in proving that collecting social determinants of health (SDoH) data during existing clinical trials is easily achievable.

Past Children’s Oncology Group trials have collected only race, ethnicity, insurance, and zip code data as proxies for exposure to adverse SDoH. Dr. Winestone and her colleagues recently investigated the feasibility and acceptability of the first COG trial to prospectively embed SDoH data collection.  

Of eligible participants, 360 of 413 opted-in to the embedded SDOH aim across 101 COG sites (87.2% consent rate). Among participants, 316 surveys (87.8%) were completed a median of 11 days post enrollment, according to the findings, which were presented at the ASCO annual meeting.

“We’ve come to realize the importance of the social determinants of health [as it pertains] to outcomes, but it has been a process to learn how to effectively collect that data in a large collaborative environment,” said Dr. Winestone. “This abstract demonstrates that patients are very willing to provide this data, and they’re able to do it in an efficient way. People think of these questions as very sensitive and that families may not want to share the answers, but this study demonstrates those presumptions are false.”

The authors hope the findings fuel incorporation of SDoH data collection in future National Clinical Trials Network trials to inform impactful health equity research.

While such research and intervention efforts are gaining momentum, challenges to do the work remain. A lack of research funding and support are among the obstacles, Dr. Winestone said.

To date, much of pediatric cancer work has focused on developing new therapeutic approaches to reach a cure for more patients, she explained.

“While that’s incredibly essential, if we’re creating these approaches that only work for a subset of patients that have resources, we’re contributing to the inequities in the system,” Dr. Winestone said. “Really, [we need] dedicated support to studying how to make sure the interventions we know are effective are reaching all populations, and that the patients are poised to benefit from those interventions by setting them up for success.”

A strong research infrastructure exists to evaluate and support clinical drug trials in pediatric oncology, but the same does not exist for health equity interventions, Dr. Bona adds. A significant question that needs to be addressed is how best to integrate health equity evaluation into existing infrastructure or whether to build a parallel infrastructure.

Despite the challenges, Dr. Bona believes now is exactly the right time to investigate and intervene in poverty as a risk factor for childhood cancer relapse and outcomes. What has led to success in childhood cancer is how pediatric oncology has collaborated across the country to operate clinical drug trials at various centers, all in the same way, to identify which treatments work best, she said.

“We have an opportunity now in pediatrics to take advantage of this highly successful clinical trials research infrastructure to integrate interventions to address disparities in a way that has not been done previously,” she said. “The opportunity to significantly improve survival in childhood cancer by reducing disparities exists if we take this head on from a research and funding perspective and approach social risk factors just as we already know how to approach tumor genomic risk factors.”

Pediatric oncologist Lena Winestone, MD, recalls treating a 2-year-old leukemia patient who underwent a bone marrow transplant as her only chance for a cure.

The girl’s family, who spoke only Spanish and struggled with literacy, could not pay their rent or afford the girl’s weekly transportation to the hospital for after-transplant care. The family had three other children and lived more than 2 hours from the transplant center, remembers Dr. Winestone, an assistant professor of pediatrics in the division of malignancies and bone & marrow transplant at the University of California, San Francisco.

The hospital’s social worker was able to secure grant support for the family’s housing and worked with the patient’s insurance to arrange for transportation. However, the departure times were rigid, Dr. Winestone said, and the family sometimes had to leave the hospital before the child’s graft vs. host disease (GvHD) treatment was complete for the day. 

“If we had not finished her treatment, we had to disconnect her from the machine early,” Dr. Winestone said. “Her mother also had to load her oxygen tanks [three of them], her BiPAP machine, and her tube feeds into the transportation every week in order to make sure she could be safely transported. She was treated for GvHD for almost 2 years, but unfortunately, her GvHD started to affect her lungs and ultimately, she passed away.”

Dr. Winestone says it’s difficult to know whether the girl’s death was directly related to her socioeconomic status, but that it certainly made all aspects of the child’s care more complicated and forced health care providers to adapt her cancer care to accommodate the family’s circumstances.

This story is one of countless cases where socioeconomic status impacted a young patient’s cancer care and likely contributed to a worse outcome. A plethora of data has demonstrated that children with cancer who are Black, Hispanic, or of lower socioeconomic status are more likely to relapse and die even when treated uniformly on clinical trials.

A 2022 study for example, found that children from marginalized racial/ethnic groups and those living in poverty were more likely to have inferior 5-year overall survival, compared with other children, even when assigned to receive the same initial treatment. Of 696 children with high-risk neuroblastoma, 47% of Hispanic children had a 5-year overall survival (OS), compared with 50% for other non-Hispanic children, and 61% for white non-Hispanic patients. Children on public health insurance (a proxy for household poverty) had a 53% 5-year OS, compared with 63% for children unexposed to household poverty. Pediatric patients exposed to neighborhood poverty had a 54% 5-year OS, compared with 62% for unexposed children.

In another study, children with acute lymphoblastic leukemia who lived in high-poverty areas were more likely to experience early relapse than other patients, despite having the same treatment. Of the 575 children studied, 92% of children from high-poverty areas who relapsed, experienced early relapse, defined as less than 36 months after remission. By comparison, only 48% of other children who relapsed experienced early relapse.

Reasons behind the relapse and survival disparities are multifold, which has led to challenges in addressing the gaps and improving cancer outcomes for poverty-stricken children. A research infrastructure that is largely based on biological, rather than social determinants of health, acts as another barrier, oncologists say.

Historically, interventions to address disparities in pediatric oncology have never been evaluated, said Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. This is in large part because the body of literature illustrating the disparities is relatively new, said Dr. Bona, whose research focuses on poverty-associated outcome disparities in childhood cancer.

However, new efforts aim to change this landscape by using the growing data to develop and analyze possible interventions. A set of three novel interventions led by Dr. Bona and her research team are in the works, some of which have shown promise in early studies.

“Now is the time to begin to actively intervene on disparities in childhood cancer,” Dr. Bona said. “We’re really good at studying genetic mutations in cancer cells that might lead to a risk of relapse, and when we identify those mutations, what we do is intervene. We try new chemotherapy agents, new ways of delivering therapy. We are now at the point where we have identified that social determinants of health may be equally ‘risky’ but we haven’t taken the next step to begin intervening in the same way.”
 

What is causing disparities in pediatric cancer outcomes?

Lack of access to the health care system is a top contributor to the disparities, although there is no single root cause, said Sharon Castellino, MD, director of the Leukemia and Lymphoma Program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta, and a professor in the department of pediatrics at Emory University, Atlanta.

Even before cancer diagnosis, Dr. Castellino notes that many children of color and/or of lower socioeconomic status are not receiving regular health care, leading to sicker children and more advanced-stage cancer by the time they are diagnosed.

Lack of insurance is a primary barrier to this access, adds Xu Ji, PhD, MSPH, an assistant professor in the department of pediatrics at Emory University and a member of the Cancer Prevention and Control Research Program at the university’s Winship Cancer Institute.

Studies  have long shown that uninsured children are more likely to go without needed care, compared with those with private insurance. Patients of color are at much higher risk of being uninsured than White patients, with the uninsured rates for Hispanic, American Indian, and Alaska Native patients being more than 2.5 times higher than that of White patients.

“We all know that insurance is a strong predictor of health outcomes,” said Dr. Ji, whose research focuses on insurance disparities and gains among cancer patients. “Lack of insurance coverage and therefore lack of access to care along the pediatric cancer continuum from early detection to early diagnosis to timely initiation of treatment to receipt of high-quality treatment to access to recommended survivorship care and even access to palliative and end-of-life care are all very important constructs in the pathway from poverty to ultimate cancer outcomes for children.”

Unstable housing, employment difficulties, and lack of family support can also come into play. Dr. Castellino remembers the case of a 12-year-old cancer patient who entered treatment with advanced-stage Hodgkin Lymphoma. The girl came from a low-income, single-parent household without stable housing. Dr. Castellino said when the child was granted a wish from the Make-a-Wish Foundation, she asked for her own bed.

“We had been working with her every week for 6 months when that request came up,” she recalled. “We said, ‘You don’t have to wait for your make-a-wish, we can get you a bed now.’ We don’t even know the extent of what happens at home for many of these children.”

The impact of toxic stress on child cancer patients is an emerging area of research, said Dr. Winestone, whose research explores racial, ethnic, and socioeconomic disparities in access to care and outcomes of leukemia and lymphoma treatment. For example, Dr. Winestone’s research includes understanding how exposure to poverty or adverse experiences in childhood may influence a patient’s biological response to chemotherapy.

Other contributors to disparities include transportation issues, lack of childcare for other children, literacy, and language barriers. A 2016 study  suggests that language barriers negatively impact the quality of informed decision-making and the care experience for Spanish-speaking parents of pediatric cancer patients with limited English proficiency.

Such access issues are also compounded by systemic factors, including a shortage of physicians of color who may be able to forge better trust relationships with families of similar race and ethnicity, Dr. Castellino adds. Lower enrollment of pediatric cancer patients with higher social vulnerabilities in clinical trials is another problem.

“In childhood cancer, I believe our improvements have been built on the backs of prior generations of families and children who have enrolled in trials. We learn things, and the next generation of therapy improves,” Dr. Castellino said. “If you have a whole group of the population not represented in trials, you don’t know what’s driving the fact they may or may not improve.”
 

Working toward solutions  

With such a diverse set of factors fueling outcome gaps, a similarly diverse approach is needed to help bridge the divide, say disparity researchers.

To this end, Dr. Bona and her research team are currently building the first portfolio of health equity interventions, each designed to address a different adverse social determinant of health differently.

The Pediatric Cancer Resource Equity (PediCARE) intervention is a centrally delivered, household material hardship (HMH)–targeted intervention that provides transportation and groceries to low-income pediatric oncology families. The intervention was recently studied in a pilot, randomized, controlled trial at Dana-Farber Cancer Institute and the University of Alabama between May 2019 and August 2021.  

Families were first screened for HMH and randomized into receiving either the intervention or usual care for 6 months. The intervention group received groceries via Instacart and transportation to and from the hospital coordinated through the Ride Health platform using Uber or Lyft. For families with their own cars, gas cards were provided. Of the families offered the chance to participate, 100% agreed to participate in the program, and there was 0% attrition in either arm of the program during the 6 months, according to the study findings, which were presented at the 2023 American Society of Clinical Oncology annual meeting in June.   

Among families who received the PediCARE intervention, 100% successfully received grocery and transportation resources, 100% reported that it was “easier to buy food for my family,” 85% reported it was easier to get to and from the hospital, and 95% reported they would be “very likely to recommend the intervention to other families,” according to the results.

“The key takeaway is that we had excellent feasibility outcomes,” said Haley Newman, MD, lead author of the study and an attending physician in the division of oncology at The Children’s Hospital of Philadelphia. “From this study, we learned that PediCARE is accessible and feasible in very diverse settings. From this, what we really took away is that PediCARE could be successfully rolled out in a phase 3 randomized trial, which would be the best way to examine efficacy.”

Another initiative in its early stages is Pediatric RISE, a guaranteed income intervention being developed with support from the Children’s Cancer Research Fund, the American Cancer Society, and other donors. The intervention will provide unrestricted cash transfers to low-income families during the early months of chemotherapy, Dr. Bona said. Families are currently being enrolled in a pilot study with a goal of refining the intervention before it’s tested for feasibility and efficacy.

“The goal here is ultimately to evaluate the question: If we are able to successfully provide income support to low-income families going through childhood cancer treatment, might we be able to ameliorate some of the disparities associated with living in poverty that we have already described in childhood cancer,” Dr. Bona said.

Pediatric Assist, a developing intervention centering on benefits, is a third initiative that will soon be evaluated. The intervention will provide newly diagnosed families with systemic access to a centralized benefits counselor who can help them determine which existing government benefits they might be eligible for and assist them in navigating the application process.

“The idea here is that we know many lower-income families in the U.S. are eligible for existing supports, but may not be accessing them because of how incredibly difficult the system is to navigate,” Dr. Bona said. “For example, we know that low-income families may be eligible for SNAP benefits, but figuring out if you are eligible and then applying for SNAP involves multiple, complicated steps that are often infeasible for a parent when their child is admitted to the hospital with a newly diagnosed, life-threatening illness.”

Pilot refinement of the intervention is expected in the fall of 2023.
 

Overcoming barriers, addressing challenges

Investigators are also making headway in proving that collecting social determinants of health (SDoH) data during existing clinical trials is easily achievable.

Past Children’s Oncology Group trials have collected only race, ethnicity, insurance, and zip code data as proxies for exposure to adverse SDoH. Dr. Winestone and her colleagues recently investigated the feasibility and acceptability of the first COG trial to prospectively embed SDoH data collection.  

Of eligible participants, 360 of 413 opted-in to the embedded SDOH aim across 101 COG sites (87.2% consent rate). Among participants, 316 surveys (87.8%) were completed a median of 11 days post enrollment, according to the findings, which were presented at the ASCO annual meeting.

“We’ve come to realize the importance of the social determinants of health [as it pertains] to outcomes, but it has been a process to learn how to effectively collect that data in a large collaborative environment,” said Dr. Winestone. “This abstract demonstrates that patients are very willing to provide this data, and they’re able to do it in an efficient way. People think of these questions as very sensitive and that families may not want to share the answers, but this study demonstrates those presumptions are false.”

The authors hope the findings fuel incorporation of SDoH data collection in future National Clinical Trials Network trials to inform impactful health equity research.

While such research and intervention efforts are gaining momentum, challenges to do the work remain. A lack of research funding and support are among the obstacles, Dr. Winestone said.

To date, much of pediatric cancer work has focused on developing new therapeutic approaches to reach a cure for more patients, she explained.

“While that’s incredibly essential, if we’re creating these approaches that only work for a subset of patients that have resources, we’re contributing to the inequities in the system,” Dr. Winestone said. “Really, [we need] dedicated support to studying how to make sure the interventions we know are effective are reaching all populations, and that the patients are poised to benefit from those interventions by setting them up for success.”

A strong research infrastructure exists to evaluate and support clinical drug trials in pediatric oncology, but the same does not exist for health equity interventions, Dr. Bona adds. A significant question that needs to be addressed is how best to integrate health equity evaluation into existing infrastructure or whether to build a parallel infrastructure.

Despite the challenges, Dr. Bona believes now is exactly the right time to investigate and intervene in poverty as a risk factor for childhood cancer relapse and outcomes. What has led to success in childhood cancer is how pediatric oncology has collaborated across the country to operate clinical drug trials at various centers, all in the same way, to identify which treatments work best, she said.

“We have an opportunity now in pediatrics to take advantage of this highly successful clinical trials research infrastructure to integrate interventions to address disparities in a way that has not been done previously,” she said. “The opportunity to significantly improve survival in childhood cancer by reducing disparities exists if we take this head on from a research and funding perspective and approach social risk factors just as we already know how to approach tumor genomic risk factors.”

Pediatric oncologist Lena Winestone, MD, recalls treating a 2-year-old leukemia patient who underwent a bone marrow transplant as her only chance for a cure.

The girl’s family, who spoke only Spanish and struggled with literacy, could not pay their rent or afford the girl’s weekly transportation to the hospital for after-transplant care. The family had three other children and lived more than 2 hours from the transplant center, remembers Dr. Winestone, an assistant professor of pediatrics in the division of malignancies and bone & marrow transplant at the University of California, San Francisco.

The hospital’s social worker was able to secure grant support for the family’s housing and worked with the patient’s insurance to arrange for transportation. However, the departure times were rigid, Dr. Winestone said, and the family sometimes had to leave the hospital before the child’s graft vs. host disease (GvHD) treatment was complete for the day. 

“If we had not finished her treatment, we had to disconnect her from the machine early,” Dr. Winestone said. “Her mother also had to load her oxygen tanks [three of them], her BiPAP machine, and her tube feeds into the transportation every week in order to make sure she could be safely transported. She was treated for GvHD for almost 2 years, but unfortunately, her GvHD started to affect her lungs and ultimately, she passed away.”

Dr. Winestone says it’s difficult to know whether the girl’s death was directly related to her socioeconomic status, but that it certainly made all aspects of the child’s care more complicated and forced health care providers to adapt her cancer care to accommodate the family’s circumstances.

This story is one of countless cases where socioeconomic status impacted a young patient’s cancer care and likely contributed to a worse outcome. A plethora of data has demonstrated that children with cancer who are Black, Hispanic, or of lower socioeconomic status are more likely to relapse and die even when treated uniformly on clinical trials.

A 2022 study for example, found that children from marginalized racial/ethnic groups and those living in poverty were more likely to have inferior 5-year overall survival, compared with other children, even when assigned to receive the same initial treatment. Of 696 children with high-risk neuroblastoma, 47% of Hispanic children had a 5-year overall survival (OS), compared with 50% for other non-Hispanic children, and 61% for white non-Hispanic patients. Children on public health insurance (a proxy for household poverty) had a 53% 5-year OS, compared with 63% for children unexposed to household poverty. Pediatric patients exposed to neighborhood poverty had a 54% 5-year OS, compared with 62% for unexposed children.

In another study, children with acute lymphoblastic leukemia who lived in high-poverty areas were more likely to experience early relapse than other patients, despite having the same treatment. Of the 575 children studied, 92% of children from high-poverty areas who relapsed, experienced early relapse, defined as less than 36 months after remission. By comparison, only 48% of other children who relapsed experienced early relapse.

Reasons behind the relapse and survival disparities are multifold, which has led to challenges in addressing the gaps and improving cancer outcomes for poverty-stricken children. A research infrastructure that is largely based on biological, rather than social determinants of health, acts as another barrier, oncologists say.

Historically, interventions to address disparities in pediatric oncology have never been evaluated, said Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. This is in large part because the body of literature illustrating the disparities is relatively new, said Dr. Bona, whose research focuses on poverty-associated outcome disparities in childhood cancer.

However, new efforts aim to change this landscape by using the growing data to develop and analyze possible interventions. A set of three novel interventions led by Dr. Bona and her research team are in the works, some of which have shown promise in early studies.

“Now is the time to begin to actively intervene on disparities in childhood cancer,” Dr. Bona said. “We’re really good at studying genetic mutations in cancer cells that might lead to a risk of relapse, and when we identify those mutations, what we do is intervene. We try new chemotherapy agents, new ways of delivering therapy. We are now at the point where we have identified that social determinants of health may be equally ‘risky’ but we haven’t taken the next step to begin intervening in the same way.”
 

What is causing disparities in pediatric cancer outcomes?

Lack of access to the health care system is a top contributor to the disparities, although there is no single root cause, said Sharon Castellino, MD, director of the Leukemia and Lymphoma Program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta, and a professor in the department of pediatrics at Emory University, Atlanta.

Even before cancer diagnosis, Dr. Castellino notes that many children of color and/or of lower socioeconomic status are not receiving regular health care, leading to sicker children and more advanced-stage cancer by the time they are diagnosed.

Lack of insurance is a primary barrier to this access, adds Xu Ji, PhD, MSPH, an assistant professor in the department of pediatrics at Emory University and a member of the Cancer Prevention and Control Research Program at the university’s Winship Cancer Institute.

Studies  have long shown that uninsured children are more likely to go without needed care, compared with those with private insurance. Patients of color are at much higher risk of being uninsured than White patients, with the uninsured rates for Hispanic, American Indian, and Alaska Native patients being more than 2.5 times higher than that of White patients.

“We all know that insurance is a strong predictor of health outcomes,” said Dr. Ji, whose research focuses on insurance disparities and gains among cancer patients. “Lack of insurance coverage and therefore lack of access to care along the pediatric cancer continuum from early detection to early diagnosis to timely initiation of treatment to receipt of high-quality treatment to access to recommended survivorship care and even access to palliative and end-of-life care are all very important constructs in the pathway from poverty to ultimate cancer outcomes for children.”

Unstable housing, employment difficulties, and lack of family support can also come into play. Dr. Castellino remembers the case of a 12-year-old cancer patient who entered treatment with advanced-stage Hodgkin Lymphoma. The girl came from a low-income, single-parent household without stable housing. Dr. Castellino said when the child was granted a wish from the Make-a-Wish Foundation, she asked for her own bed.

“We had been working with her every week for 6 months when that request came up,” she recalled. “We said, ‘You don’t have to wait for your make-a-wish, we can get you a bed now.’ We don’t even know the extent of what happens at home for many of these children.”

The impact of toxic stress on child cancer patients is an emerging area of research, said Dr. Winestone, whose research explores racial, ethnic, and socioeconomic disparities in access to care and outcomes of leukemia and lymphoma treatment. For example, Dr. Winestone’s research includes understanding how exposure to poverty or adverse experiences in childhood may influence a patient’s biological response to chemotherapy.

Other contributors to disparities include transportation issues, lack of childcare for other children, literacy, and language barriers. A 2016 study  suggests that language barriers negatively impact the quality of informed decision-making and the care experience for Spanish-speaking parents of pediatric cancer patients with limited English proficiency.

Such access issues are also compounded by systemic factors, including a shortage of physicians of color who may be able to forge better trust relationships with families of similar race and ethnicity, Dr. Castellino adds. Lower enrollment of pediatric cancer patients with higher social vulnerabilities in clinical trials is another problem.

“In childhood cancer, I believe our improvements have been built on the backs of prior generations of families and children who have enrolled in trials. We learn things, and the next generation of therapy improves,” Dr. Castellino said. “If you have a whole group of the population not represented in trials, you don’t know what’s driving the fact they may or may not improve.”
 

Working toward solutions  

With such a diverse set of factors fueling outcome gaps, a similarly diverse approach is needed to help bridge the divide, say disparity researchers.

To this end, Dr. Bona and her research team are currently building the first portfolio of health equity interventions, each designed to address a different adverse social determinant of health differently.

The Pediatric Cancer Resource Equity (PediCARE) intervention is a centrally delivered, household material hardship (HMH)–targeted intervention that provides transportation and groceries to low-income pediatric oncology families. The intervention was recently studied in a pilot, randomized, controlled trial at Dana-Farber Cancer Institute and the University of Alabama between May 2019 and August 2021.  

Families were first screened for HMH and randomized into receiving either the intervention or usual care for 6 months. The intervention group received groceries via Instacart and transportation to and from the hospital coordinated through the Ride Health platform using Uber or Lyft. For families with their own cars, gas cards were provided. Of the families offered the chance to participate, 100% agreed to participate in the program, and there was 0% attrition in either arm of the program during the 6 months, according to the study findings, which were presented at the 2023 American Society of Clinical Oncology annual meeting in June.   

Among families who received the PediCARE intervention, 100% successfully received grocery and transportation resources, 100% reported that it was “easier to buy food for my family,” 85% reported it was easier to get to and from the hospital, and 95% reported they would be “very likely to recommend the intervention to other families,” according to the results.

“The key takeaway is that we had excellent feasibility outcomes,” said Haley Newman, MD, lead author of the study and an attending physician in the division of oncology at The Children’s Hospital of Philadelphia. “From this study, we learned that PediCARE is accessible and feasible in very diverse settings. From this, what we really took away is that PediCARE could be successfully rolled out in a phase 3 randomized trial, which would be the best way to examine efficacy.”

Another initiative in its early stages is Pediatric RISE, a guaranteed income intervention being developed with support from the Children’s Cancer Research Fund, the American Cancer Society, and other donors. The intervention will provide unrestricted cash transfers to low-income families during the early months of chemotherapy, Dr. Bona said. Families are currently being enrolled in a pilot study with a goal of refining the intervention before it’s tested for feasibility and efficacy.

“The goal here is ultimately to evaluate the question: If we are able to successfully provide income support to low-income families going through childhood cancer treatment, might we be able to ameliorate some of the disparities associated with living in poverty that we have already described in childhood cancer,” Dr. Bona said.

Pediatric Assist, a developing intervention centering on benefits, is a third initiative that will soon be evaluated. The intervention will provide newly diagnosed families with systemic access to a centralized benefits counselor who can help them determine which existing government benefits they might be eligible for and assist them in navigating the application process.

“The idea here is that we know many lower-income families in the U.S. are eligible for existing supports, but may not be accessing them because of how incredibly difficult the system is to navigate,” Dr. Bona said. “For example, we know that low-income families may be eligible for SNAP benefits, but figuring out if you are eligible and then applying for SNAP involves multiple, complicated steps that are often infeasible for a parent when their child is admitted to the hospital with a newly diagnosed, life-threatening illness.”

Pilot refinement of the intervention is expected in the fall of 2023.
 

Overcoming barriers, addressing challenges

Investigators are also making headway in proving that collecting social determinants of health (SDoH) data during existing clinical trials is easily achievable.

Past Children’s Oncology Group trials have collected only race, ethnicity, insurance, and zip code data as proxies for exposure to adverse SDoH. Dr. Winestone and her colleagues recently investigated the feasibility and acceptability of the first COG trial to prospectively embed SDoH data collection.  

Of eligible participants, 360 of 413 opted-in to the embedded SDOH aim across 101 COG sites (87.2% consent rate). Among participants, 316 surveys (87.8%) were completed a median of 11 days post enrollment, according to the findings, which were presented at the ASCO annual meeting.

“We’ve come to realize the importance of the social determinants of health [as it pertains] to outcomes, but it has been a process to learn how to effectively collect that data in a large collaborative environment,” said Dr. Winestone. “This abstract demonstrates that patients are very willing to provide this data, and they’re able to do it in an efficient way. People think of these questions as very sensitive and that families may not want to share the answers, but this study demonstrates those presumptions are false.”

The authors hope the findings fuel incorporation of SDoH data collection in future National Clinical Trials Network trials to inform impactful health equity research.

While such research and intervention efforts are gaining momentum, challenges to do the work remain. A lack of research funding and support are among the obstacles, Dr. Winestone said.

To date, much of pediatric cancer work has focused on developing new therapeutic approaches to reach a cure for more patients, she explained.

“While that’s incredibly essential, if we’re creating these approaches that only work for a subset of patients that have resources, we’re contributing to the inequities in the system,” Dr. Winestone said. “Really, [we need] dedicated support to studying how to make sure the interventions we know are effective are reaching all populations, and that the patients are poised to benefit from those interventions by setting them up for success.”

A strong research infrastructure exists to evaluate and support clinical drug trials in pediatric oncology, but the same does not exist for health equity interventions, Dr. Bona adds. A significant question that needs to be addressed is how best to integrate health equity evaluation into existing infrastructure or whether to build a parallel infrastructure.

Despite the challenges, Dr. Bona believes now is exactly the right time to investigate and intervene in poverty as a risk factor for childhood cancer relapse and outcomes. What has led to success in childhood cancer is how pediatric oncology has collaborated across the country to operate clinical drug trials at various centers, all in the same way, to identify which treatments work best, she said.

“We have an opportunity now in pediatrics to take advantage of this highly successful clinical trials research infrastructure to integrate interventions to address disparities in a way that has not been done previously,” she said. “The opportunity to significantly improve survival in childhood cancer by reducing disparities exists if we take this head on from a research and funding perspective and approach social risk factors just as we already know how to approach tumor genomic risk factors.”

French researchers have been working on an innovative vaccine that targets tick microbiota to indirectly reduce the bacterial load within the vector. The results of their study were published in the journal Microbiome.

Ticks are vectors of many harmful pathogens that can cause life-threatening illnesses. Ixodes ricinus (in Europe) and Ixodes scapularis (in Canada and the United States) carry Borrelia, the bacteria that cause Lyme disease. At the moment, there is no vaccine for this disease. But that could all change, thanks to the findings of scientists at the National Research Institute for Agriculture, Food, and Environment (INRAE), in collaboration with the Agency for Food, Environmental, and Occupational Health and Safety and the National Veterinary School of Alfort, France.

“Ticks can transmit a broad variety of pathogens of medical importance, including Borrelia afzelii, the causative agent of Lyme borreliosis in Europe. Tick microbiota is an important factor modulating not only vector physiology, but also the vector competence,” the team reported. They focused their efforts on developing a vaccine that would disturb the tick microbiota and thus reduce Borrelia colonization.

To explore this indirect approach, they injected a harmless strain of Escherichia coli bacteria into mice, which then produced antibodies. Their reasoning was that when a tick bites one of these mice, the antibodies would pass into the arachnid’s microbiota and disturb it, thereby making the tick less harmful. And indeed, the researchers’ work showed that in the ticks that fed on vaccinated mice, levels of Borrelia levels were much lower than in than ticks that fed on unvaccinated mice (see video for an explanation). So, when given to a mouse, this vaccine “protects” the tick against colonization by Borrelia but does not protect the mouse against the disease.

The study has advanced this area of research in two significant ways: It provides new information on the importance of the microbiota when it comes to ticks that are infected with Borrelia, and it suggests an innovative vaccination strategy. Indeed, the results confirm that tick microbiota is essential for the development of Borrelia in the arachnid. As noted in an INRAE press release, “This is a key piece of data that opens the door to one day having an innovative vaccination strategy aimed at perturbing the microbiota of the vector of the Lyme disease agent.”

Dengue, Zika virus, and malaria are also transmitted by a vector – the mosquito. Innovative antimicrobiota vaccines may be able to control these diseases as well.

This article was translated from the Medscape French Edition. A version of this article appeared on Medscape.com.

French researchers have been working on an innovative vaccine that targets tick microbiota to indirectly reduce the bacterial load within the vector. The results of their study were published in the journal Microbiome.

Ticks are vectors of many harmful pathogens that can cause life-threatening illnesses. Ixodes ricinus (in Europe) and Ixodes scapularis (in Canada and the United States) carry Borrelia, the bacteria that cause Lyme disease. At the moment, there is no vaccine for this disease. But that could all change, thanks to the findings of scientists at the National Research Institute for Agriculture, Food, and Environment (INRAE), in collaboration with the Agency for Food, Environmental, and Occupational Health and Safety and the National Veterinary School of Alfort, France.

“Ticks can transmit a broad variety of pathogens of medical importance, including Borrelia afzelii, the causative agent of Lyme borreliosis in Europe. Tick microbiota is an important factor modulating not only vector physiology, but also the vector competence,” the team reported. They focused their efforts on developing a vaccine that would disturb the tick microbiota and thus reduce Borrelia colonization.

To explore this indirect approach, they injected a harmless strain of Escherichia coli bacteria into mice, which then produced antibodies. Their reasoning was that when a tick bites one of these mice, the antibodies would pass into the arachnid’s microbiota and disturb it, thereby making the tick less harmful. And indeed, the researchers’ work showed that in the ticks that fed on vaccinated mice, levels of Borrelia levels were much lower than in than ticks that fed on unvaccinated mice (see video for an explanation). So, when given to a mouse, this vaccine “protects” the tick against colonization by Borrelia but does not protect the mouse against the disease.

The study has advanced this area of research in two significant ways: It provides new information on the importance of the microbiota when it comes to ticks that are infected with Borrelia, and it suggests an innovative vaccination strategy. Indeed, the results confirm that tick microbiota is essential for the development of Borrelia in the arachnid. As noted in an INRAE press release, “This is a key piece of data that opens the door to one day having an innovative vaccination strategy aimed at perturbing the microbiota of the vector of the Lyme disease agent.”

Dengue, Zika virus, and malaria are also transmitted by a vector – the mosquito. Innovative antimicrobiota vaccines may be able to control these diseases as well.

This article was translated from the Medscape French Edition. A version of this article appeared on Medscape.com.

French researchers have been working on an innovative vaccine that targets tick microbiota to indirectly reduce the bacterial load within the vector. The results of their study were published in the journal Microbiome.

Ticks are vectors of many harmful pathogens that can cause life-threatening illnesses. Ixodes ricinus (in Europe) and Ixodes scapularis (in Canada and the United States) carry Borrelia, the bacteria that cause Lyme disease. At the moment, there is no vaccine for this disease. But that could all change, thanks to the findings of scientists at the National Research Institute for Agriculture, Food, and Environment (INRAE), in collaboration with the Agency for Food, Environmental, and Occupational Health and Safety and the National Veterinary School of Alfort, France.

“Ticks can transmit a broad variety of pathogens of medical importance, including Borrelia afzelii, the causative agent of Lyme borreliosis in Europe. Tick microbiota is an important factor modulating not only vector physiology, but also the vector competence,” the team reported. They focused their efforts on developing a vaccine that would disturb the tick microbiota and thus reduce Borrelia colonization.

To explore this indirect approach, they injected a harmless strain of Escherichia coli bacteria into mice, which then produced antibodies. Their reasoning was that when a tick bites one of these mice, the antibodies would pass into the arachnid’s microbiota and disturb it, thereby making the tick less harmful. And indeed, the researchers’ work showed that in the ticks that fed on vaccinated mice, levels of Borrelia levels were much lower than in than ticks that fed on unvaccinated mice (see video for an explanation). So, when given to a mouse, this vaccine “protects” the tick against colonization by Borrelia but does not protect the mouse against the disease.

The study has advanced this area of research in two significant ways: It provides new information on the importance of the microbiota when it comes to ticks that are infected with Borrelia, and it suggests an innovative vaccination strategy. Indeed, the results confirm that tick microbiota is essential for the development of Borrelia in the arachnid. As noted in an INRAE press release, “This is a key piece of data that opens the door to one day having an innovative vaccination strategy aimed at perturbing the microbiota of the vector of the Lyme disease agent.”

Dengue, Zika virus, and malaria are also transmitted by a vector – the mosquito. Innovative antimicrobiota vaccines may be able to control these diseases as well.

This article was translated from the Medscape French Edition. A version of this article appeared on Medscape.com.

A large brain imaging study of adults with six different psychiatric illnesses shows that heterogeneity in regional gray matter volume deviations is a general feature of psychiatric illness, but that these regionally heterogeneous areas are often embedded within common functional circuits and networks.

The findings suggest that “targeting brain circuits, rather than specific brain regions, may be a more effective way of developing new treatments,” study investigator Ashlea Segal said in an email.

The findings also suggest that it’s “unlikely that a single cause or mechanism of a given disorder exists, and that a ‘one-size-fits-all’ approach to treatment is likely only appropriate for a small subset of individuals. In fact, one size doesn’t fit all. It probably doesn’t even fit most,” said Ms. Segal, a PhD candidate with the Turner Institute for Brain and Mental Health’s Neural Systems and Behaviour Lab at Monash University in Melbourne.

“Focusing on brain alterations at an individual level allows us to develop more personally tailored treatments,” Ms. Segal added.

Regional heterogeneity, the authors write, “thus offers a plausible explanation for the well-described clinical heterogeneity observed in psychiatric disorders, while circuit- and network-level aggregation of deviations is a putative neural substrate for phenotypic similarities between patients assigned the same diagnosis.”

The study was published online in Nature Neuroscience
 

Beyond group averages

For decades, researchers have mapped brain areas showing reduced gray matter volume (GMV) in people diagnosed with a variety of mental illnesses, but these maps have only been generated at the level of group averages, Ms. Segal explained.

“This means that we understand how the brains of people with, say, schizophrenia, differ from those without schizophrenia on average, but we can’t really say much about individual people,” Ms. Segal said.

For their study, the researchers used new statistical techniques developed by Andre Marquand, PhD, who co-led the project, to characterize the heterogeneity of GMV differences in 1,294 individuals diagnosed with one of six psychiatric conditions and 1,465 matched controls. Dr. Marquand is affiliated with the Donders Institute for Brain, Cognition, and Behavior in Nijmegen, the Netherlands.

These techniques “allow us to benchmark the size of over 1,000 different brain regions in any given person relative to what we should expect to see in the general population. In this way, we can identify, for any person, brain regions showing unusually small or large volumes, given that person’s age and sex,” Ms. Segal told this news organization.

The clinical sample included 202 individuals with autism spectrum disorder, 153 with attention-deficit/hyperactivity disorder (ADHD), 228 with bipolar disorder, 161 with major depressive disorder, 167 with obsessive-compulsive disorder, and 383 individuals with schizophrenia.

Confirming earlier findings, those with psychiatric illness showed more GMV deviations than healthy controls, the researchers found.

However, at the individual level, deviations from population expectations for regional gray matter volumes were “highly heterogeneous,” affecting the same area in less than 7% of people with the same diagnosis, they note. “This result means that it is difficult to pinpoint treatment targets or causal mechanisms by focusing on group averages alone,” Alex Fornito, PhD, of Monash University, who led the research team, said in a statement.

“It may also explain why people with the same diagnosis show wide variability in their symptom profiles and treatment outcomes,” Dr. Fornito added.

Yet, despite considerable heterogeneity at the regional level across different diagnoses, these deviations were embedded within common functional circuits and networks in up to 56% of cases. 

The salience-ventral attention network, for example, which plays a central role in cognitive control, interoceptive awareness, and switching between internally and externally focused attention, was implicated across diagnoses, with other neural networks selectively involved in depression, bipolar disorder, schizophrenia, and ADHD.

The researchers say the approach they developed opens new opportunities for mapping brain changes in mental illness.

“The framework we have developed allows us to understand the diversity of brain changes in people with mental illness at different levels, from individual regions through to more widespread brain circuits and networks, offering a deeper insight into how the brain is affected in individual people,” Dr. Fornito said in a statement.

The study had no commercial funding. Ms. Segal, Dr. Fornito, and Dr. Marquand report no relevant financial relationships.

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

A large brain imaging study of adults with six different psychiatric illnesses shows that heterogeneity in regional gray matter volume deviations is a general feature of psychiatric illness, but that these regionally heterogeneous areas are often embedded within common functional circuits and networks.

The findings suggest that “targeting brain circuits, rather than specific brain regions, may be a more effective way of developing new treatments,” study investigator Ashlea Segal said in an email.

The findings also suggest that it’s “unlikely that a single cause or mechanism of a given disorder exists, and that a ‘one-size-fits-all’ approach to treatment is likely only appropriate for a small subset of individuals. In fact, one size doesn’t fit all. It probably doesn’t even fit most,” said Ms. Segal, a PhD candidate with the Turner Institute for Brain and Mental Health’s Neural Systems and Behaviour Lab at Monash University in Melbourne.

“Focusing on brain alterations at an individual level allows us to develop more personally tailored treatments,” Ms. Segal added.

Regional heterogeneity, the authors write, “thus offers a plausible explanation for the well-described clinical heterogeneity observed in psychiatric disorders, while circuit- and network-level aggregation of deviations is a putative neural substrate for phenotypic similarities between patients assigned the same diagnosis.”

The study was published online in Nature Neuroscience
 

Beyond group averages

For decades, researchers have mapped brain areas showing reduced gray matter volume (GMV) in people diagnosed with a variety of mental illnesses, but these maps have only been generated at the level of group averages, Ms. Segal explained.

“This means that we understand how the brains of people with, say, schizophrenia, differ from those without schizophrenia on average, but we can’t really say much about individual people,” Ms. Segal said.

For their study, the researchers used new statistical techniques developed by Andre Marquand, PhD, who co-led the project, to characterize the heterogeneity of GMV differences in 1,294 individuals diagnosed with one of six psychiatric conditions and 1,465 matched controls. Dr. Marquand is affiliated with the Donders Institute for Brain, Cognition, and Behavior in Nijmegen, the Netherlands.

These techniques “allow us to benchmark the size of over 1,000 different brain regions in any given person relative to what we should expect to see in the general population. In this way, we can identify, for any person, brain regions showing unusually small or large volumes, given that person’s age and sex,” Ms. Segal told this news organization.

The clinical sample included 202 individuals with autism spectrum disorder, 153 with attention-deficit/hyperactivity disorder (ADHD), 228 with bipolar disorder, 161 with major depressive disorder, 167 with obsessive-compulsive disorder, and 383 individuals with schizophrenia.

Confirming earlier findings, those with psychiatric illness showed more GMV deviations than healthy controls, the researchers found.

However, at the individual level, deviations from population expectations for regional gray matter volumes were “highly heterogeneous,” affecting the same area in less than 7% of people with the same diagnosis, they note. “This result means that it is difficult to pinpoint treatment targets or causal mechanisms by focusing on group averages alone,” Alex Fornito, PhD, of Monash University, who led the research team, said in a statement.

“It may also explain why people with the same diagnosis show wide variability in their symptom profiles and treatment outcomes,” Dr. Fornito added.

Yet, despite considerable heterogeneity at the regional level across different diagnoses, these deviations were embedded within common functional circuits and networks in up to 56% of cases. 

The salience-ventral attention network, for example, which plays a central role in cognitive control, interoceptive awareness, and switching between internally and externally focused attention, was implicated across diagnoses, with other neural networks selectively involved in depression, bipolar disorder, schizophrenia, and ADHD.

The researchers say the approach they developed opens new opportunities for mapping brain changes in mental illness.

“The framework we have developed allows us to understand the diversity of brain changes in people with mental illness at different levels, from individual regions through to more widespread brain circuits and networks, offering a deeper insight into how the brain is affected in individual people,” Dr. Fornito said in a statement.

The study had no commercial funding. Ms. Segal, Dr. Fornito, and Dr. Marquand report no relevant financial relationships.

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

A large brain imaging study of adults with six different psychiatric illnesses shows that heterogeneity in regional gray matter volume deviations is a general feature of psychiatric illness, but that these regionally heterogeneous areas are often embedded within common functional circuits and networks.

The findings suggest that “targeting brain circuits, rather than specific brain regions, may be a more effective way of developing new treatments,” study investigator Ashlea Segal said in an email.

The findings also suggest that it’s “unlikely that a single cause or mechanism of a given disorder exists, and that a ‘one-size-fits-all’ approach to treatment is likely only appropriate for a small subset of individuals. In fact, one size doesn’t fit all. It probably doesn’t even fit most,” said Ms. Segal, a PhD candidate with the Turner Institute for Brain and Mental Health’s Neural Systems and Behaviour Lab at Monash University in Melbourne.

“Focusing on brain alterations at an individual level allows us to develop more personally tailored treatments,” Ms. Segal added.

Regional heterogeneity, the authors write, “thus offers a plausible explanation for the well-described clinical heterogeneity observed in psychiatric disorders, while circuit- and network-level aggregation of deviations is a putative neural substrate for phenotypic similarities between patients assigned the same diagnosis.”

The study was published online in Nature Neuroscience
 

Beyond group averages

For decades, researchers have mapped brain areas showing reduced gray matter volume (GMV) in people diagnosed with a variety of mental illnesses, but these maps have only been generated at the level of group averages, Ms. Segal explained.

“This means that we understand how the brains of people with, say, schizophrenia, differ from those without schizophrenia on average, but we can’t really say much about individual people,” Ms. Segal said.

For their study, the researchers used new statistical techniques developed by Andre Marquand, PhD, who co-led the project, to characterize the heterogeneity of GMV differences in 1,294 individuals diagnosed with one of six psychiatric conditions and 1,465 matched controls. Dr. Marquand is affiliated with the Donders Institute for Brain, Cognition, and Behavior in Nijmegen, the Netherlands.

These techniques “allow us to benchmark the size of over 1,000 different brain regions in any given person relative to what we should expect to see in the general population. In this way, we can identify, for any person, brain regions showing unusually small or large volumes, given that person’s age and sex,” Ms. Segal told this news organization.

The clinical sample included 202 individuals with autism spectrum disorder, 153 with attention-deficit/hyperactivity disorder (ADHD), 228 with bipolar disorder, 161 with major depressive disorder, 167 with obsessive-compulsive disorder, and 383 individuals with schizophrenia.

Confirming earlier findings, those with psychiatric illness showed more GMV deviations than healthy controls, the researchers found.

However, at the individual level, deviations from population expectations for regional gray matter volumes were “highly heterogeneous,” affecting the same area in less than 7% of people with the same diagnosis, they note. “This result means that it is difficult to pinpoint treatment targets or causal mechanisms by focusing on group averages alone,” Alex Fornito, PhD, of Monash University, who led the research team, said in a statement.

“It may also explain why people with the same diagnosis show wide variability in their symptom profiles and treatment outcomes,” Dr. Fornito added.

Yet, despite considerable heterogeneity at the regional level across different diagnoses, these deviations were embedded within common functional circuits and networks in up to 56% of cases. 

The salience-ventral attention network, for example, which plays a central role in cognitive control, interoceptive awareness, and switching between internally and externally focused attention, was implicated across diagnoses, with other neural networks selectively involved in depression, bipolar disorder, schizophrenia, and ADHD.

The researchers say the approach they developed opens new opportunities for mapping brain changes in mental illness.

“The framework we have developed allows us to understand the diversity of brain changes in people with mental illness at different levels, from individual regions through to more widespread brain circuits and networks, offering a deeper insight into how the brain is affected in individual people,” Dr. Fornito said in a statement.

The study had no commercial funding. Ms. Segal, Dr. Fornito, and Dr. Marquand report no relevant financial relationships.

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