Rare Diseases

“Petechial rash often prompts further investigation into hematological, dermatological, or vasculitis causes. However, if the above investigations are negative and skin biopsy has not revealed a cause, there is a Renaissance-era diagnosis that is often overlooked but is easily investigated and treated,” wrote Andrew Dermawan, MD, and colleagues from Sir Charles Gairdner Hospital in Nedlands, Australia, in BMJ Case Reports. The diagnosis they highlight is scurvy, a disease that has faded from common medical concern but is reemerging, partly because of the rise in bariatric surgery.

Diagnosing Scurvy in the 2020s

In their article, Dermawan and colleagues present the case of a 50-year-old man with a bilateral petechial rash on his lower limbs, without any history of trauma. The patient, who exhibited no infectious symptoms, also had gross hematuria, microcytic anemia, mild neutropenia, and lymphopenia. Tests for autoimmune and hematological diseases were negative, as were abdominal and leg CT scans, ruling out abdominal hemorrhage and vasculitis. Additionally, a skin biopsy showed no causative findings.

The doctors noted that the patient had undergone sleeve gastrectomy, prompting them to inquire about his diet. They discovered that, because of financial difficulties, his diet primarily consisted of processed foods with little to no fruits or vegetables, and he had stopped taking supplements recommended by his gastroenterologist. Further tests revealed a vitamin D deficiency and a severe deficiency in vitamin C. With the diagnosis of scurvy confirmed, the doctors treated the patient with 1000 mg of ascorbic acid daily, along with cholecalciferol, folic acid, and a multivitamin complex, leading to a complete resolution of his symptoms.
 

Risk Factors Then and Now

Scurvy can present with a range of symptoms, including petechiae, perifollicular hemorrhage, ecchymosis, gingivitis, edema, anemia, delayed wound healing, malaise, weakness, joint swelling, arthralgia, anorexia, neuropathy, and vasomotor instability. It can cause mucosal and gastric hemorrhages, and if left untreated, it can lead to fatal bleeding.

Historically known as “sailors’ disease,” scurvy plagued men on long voyages who lacked access to fresh fruits or vegetables and thus did not get enough vitamin C. In 1747, James Lind, a British physician in the Royal Navy, demonstrated that the consumption of oranges and lemons could combat scurvy.

Today’s risk factors for scurvy include malnutrition, gastrointestinal disorders (eg, chronic inflammatory bowel diseases), alcohol and tobacco use, eating disorders, psychiatric illnesses, dialysis, and the use of medications that reduce the absorption of ascorbic acid (such as corticosteroids and proton pump inhibitors).

Scurvy remains more common among individuals with unfavorable socioeconomic conditions. The authors of the study emphasize how the rising cost of living — specifically in Australia but applicable elsewhere — is changing eating habits, leading to a high consumption of low-cost, nutritionally poor foods.

Poverty has always been a risk factor for scurvy, but today there may be an additional cause: bariatric surgery. Patients undergoing these procedures are at a risk for deficiencies in fat-soluble vitamins A, D, E, and K, and if their diet is inadequate, they may also experience a vitamin C deficiency. Awareness of this can facilitate the timely diagnosis of scurvy in these patients.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

“Petechial rash often prompts further investigation into hematological, dermatological, or vasculitis causes. However, if the above investigations are negative and skin biopsy has not revealed a cause, there is a Renaissance-era diagnosis that is often overlooked but is easily investigated and treated,” wrote Andrew Dermawan, MD, and colleagues from Sir Charles Gairdner Hospital in Nedlands, Australia, in BMJ Case Reports. The diagnosis they highlight is scurvy, a disease that has faded from common medical concern but is reemerging, partly because of the rise in bariatric surgery.

Diagnosing Scurvy in the 2020s

In their article, Dermawan and colleagues present the case of a 50-year-old man with a bilateral petechial rash on his lower limbs, without any history of trauma. The patient, who exhibited no infectious symptoms, also had gross hematuria, microcytic anemia, mild neutropenia, and lymphopenia. Tests for autoimmune and hematological diseases were negative, as were abdominal and leg CT scans, ruling out abdominal hemorrhage and vasculitis. Additionally, a skin biopsy showed no causative findings.

The doctors noted that the patient had undergone sleeve gastrectomy, prompting them to inquire about his diet. They discovered that, because of financial difficulties, his diet primarily consisted of processed foods with little to no fruits or vegetables, and he had stopped taking supplements recommended by his gastroenterologist. Further tests revealed a vitamin D deficiency and a severe deficiency in vitamin C. With the diagnosis of scurvy confirmed, the doctors treated the patient with 1000 mg of ascorbic acid daily, along with cholecalciferol, folic acid, and a multivitamin complex, leading to a complete resolution of his symptoms.
 

Risk Factors Then and Now

Scurvy can present with a range of symptoms, including petechiae, perifollicular hemorrhage, ecchymosis, gingivitis, edema, anemia, delayed wound healing, malaise, weakness, joint swelling, arthralgia, anorexia, neuropathy, and vasomotor instability. It can cause mucosal and gastric hemorrhages, and if left untreated, it can lead to fatal bleeding.

Historically known as “sailors’ disease,” scurvy plagued men on long voyages who lacked access to fresh fruits or vegetables and thus did not get enough vitamin C. In 1747, James Lind, a British physician in the Royal Navy, demonstrated that the consumption of oranges and lemons could combat scurvy.

Today’s risk factors for scurvy include malnutrition, gastrointestinal disorders (eg, chronic inflammatory bowel diseases), alcohol and tobacco use, eating disorders, psychiatric illnesses, dialysis, and the use of medications that reduce the absorption of ascorbic acid (such as corticosteroids and proton pump inhibitors).

Scurvy remains more common among individuals with unfavorable socioeconomic conditions. The authors of the study emphasize how the rising cost of living — specifically in Australia but applicable elsewhere — is changing eating habits, leading to a high consumption of low-cost, nutritionally poor foods.

Poverty has always been a risk factor for scurvy, but today there may be an additional cause: bariatric surgery. Patients undergoing these procedures are at a risk for deficiencies in fat-soluble vitamins A, D, E, and K, and if their diet is inadequate, they may also experience a vitamin C deficiency. Awareness of this can facilitate the timely diagnosis of scurvy in these patients.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

“Petechial rash often prompts further investigation into hematological, dermatological, or vasculitis causes. However, if the above investigations are negative and skin biopsy has not revealed a cause, there is a Renaissance-era diagnosis that is often overlooked but is easily investigated and treated,” wrote Andrew Dermawan, MD, and colleagues from Sir Charles Gairdner Hospital in Nedlands, Australia, in BMJ Case Reports. The diagnosis they highlight is scurvy, a disease that has faded from common medical concern but is reemerging, partly because of the rise in bariatric surgery.

Diagnosing Scurvy in the 2020s

In their article, Dermawan and colleagues present the case of a 50-year-old man with a bilateral petechial rash on his lower limbs, without any history of trauma. The patient, who exhibited no infectious symptoms, also had gross hematuria, microcytic anemia, mild neutropenia, and lymphopenia. Tests for autoimmune and hematological diseases were negative, as were abdominal and leg CT scans, ruling out abdominal hemorrhage and vasculitis. Additionally, a skin biopsy showed no causative findings.

The doctors noted that the patient had undergone sleeve gastrectomy, prompting them to inquire about his diet. They discovered that, because of financial difficulties, his diet primarily consisted of processed foods with little to no fruits or vegetables, and he had stopped taking supplements recommended by his gastroenterologist. Further tests revealed a vitamin D deficiency and a severe deficiency in vitamin C. With the diagnosis of scurvy confirmed, the doctors treated the patient with 1000 mg of ascorbic acid daily, along with cholecalciferol, folic acid, and a multivitamin complex, leading to a complete resolution of his symptoms.
 

Risk Factors Then and Now

Scurvy can present with a range of symptoms, including petechiae, perifollicular hemorrhage, ecchymosis, gingivitis, edema, anemia, delayed wound healing, malaise, weakness, joint swelling, arthralgia, anorexia, neuropathy, and vasomotor instability. It can cause mucosal and gastric hemorrhages, and if left untreated, it can lead to fatal bleeding.

Historically known as “sailors’ disease,” scurvy plagued men on long voyages who lacked access to fresh fruits or vegetables and thus did not get enough vitamin C. In 1747, James Lind, a British physician in the Royal Navy, demonstrated that the consumption of oranges and lemons could combat scurvy.

Today’s risk factors for scurvy include malnutrition, gastrointestinal disorders (eg, chronic inflammatory bowel diseases), alcohol and tobacco use, eating disorders, psychiatric illnesses, dialysis, and the use of medications that reduce the absorption of ascorbic acid (such as corticosteroids and proton pump inhibitors).

Scurvy remains more common among individuals with unfavorable socioeconomic conditions. The authors of the study emphasize how the rising cost of living — specifically in Australia but applicable elsewhere — is changing eating habits, leading to a high consumption of low-cost, nutritionally poor foods.

Poverty has always been a risk factor for scurvy, but today there may be an additional cause: bariatric surgery. Patients undergoing these procedures are at a risk for deficiencies in fat-soluble vitamins A, D, E, and K, and if their diet is inadequate, they may also experience a vitamin C deficiency. Awareness of this can facilitate the timely diagnosis of scurvy in these patients.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Should patients with myasthenia gravis continue to undergo the traditional therapy of immunosuppression with drugs like corticosteroids and nonsteroidal agents? Or is it time to embrace a new generation of medications?

In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.

On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.

But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
 

Traditional Therapy: Affordable, Tolerable, and Safe?

Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.

Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.

Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.

In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.

As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
 

Nonsteroidal Agents, Immune Globulin, Rituximab

He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.

As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.

Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.

What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
 

Steroids Are Ideal in MG? Not So Fast

In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.

For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”

In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”

Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”

As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
 

Kicking Traditional Therapy to the Curb

There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.

On top of all these risks, she said, 20%-30% of patients are resistant to steroids.

As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.

Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”

In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”

Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Should patients with myasthenia gravis continue to undergo the traditional therapy of immunosuppression with drugs like corticosteroids and nonsteroidal agents? Or is it time to embrace a new generation of medications?

In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.

On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.

But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
 

Traditional Therapy: Affordable, Tolerable, and Safe?

Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.

Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.

Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.

In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.

As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
 

Nonsteroidal Agents, Immune Globulin, Rituximab

He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.

As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.

Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.

What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
 

Steroids Are Ideal in MG? Not So Fast

In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.

For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”

In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”

Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”

As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
 

Kicking Traditional Therapy to the Curb

There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.

On top of all these risks, she said, 20%-30% of patients are resistant to steroids.

As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.

Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”

In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”

Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Should patients with myasthenia gravis continue to undergo the traditional therapy of immunosuppression with drugs like corticosteroids and nonsteroidal agents? Or is it time to embrace a new generation of medications?

In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.

On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.

But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
 

Traditional Therapy: Affordable, Tolerable, and Safe?

Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.

Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.

Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.

In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.

As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
 

Nonsteroidal Agents, Immune Globulin, Rituximab

He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.

As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.

Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.

What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
 

Steroids Are Ideal in MG? Not So Fast

In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.

For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”

In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”

Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”

As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
 

Kicking Traditional Therapy to the Curb

There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.

On top of all these risks, she said, 20%-30% of patients are resistant to steroids.

As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.

Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”

In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”

Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).

“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Now, the landscape of DMD therapy is transforming at a rapid pace. “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.

This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
 

Beware of Parents Who Reject Steroids

The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”

However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.

In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”

In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
 

Deflazacort or Vamorolone?

Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.

Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.

“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.

$700,000 a Year for Givinostat

Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.

The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.

Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”

As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”

Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).

“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Now, the landscape of DMD therapy is transforming at a rapid pace. “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.

This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
 

Beware of Parents Who Reject Steroids

The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”

However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.

In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”

In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
 

Deflazacort or Vamorolone?

Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.

Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.

“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.

$700,000 a Year for Givinostat

Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.

The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.

Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”

As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”

Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).

“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Now, the landscape of DMD therapy is transforming at a rapid pace. “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.

This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
 

Beware of Parents Who Reject Steroids

The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”

However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.

In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”

In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
 

Deflazacort or Vamorolone?

Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.

Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.

“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.

$700,000 a Year for Givinostat

Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.

The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.

Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”

As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”

Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
 

A version of this article appeared on Medscape.com.

TOPLINE:

The use of newer biologic or targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) for treating juvenile idiopathic arthritis (JIA) rose sharply from 2001 to 2022, while the use of conventional synthetic DMARDs (csDMARDs) plummeted, with adalimumab becoming the most commonly used b/tsDMARD.

METHODOLOGY:

• Researchers performed a serial cross-sectional study using Merative MarketScan Commercial Claims and Encounters data from 2000 to 2022 to describe recent trends in DMARD use for children with JIA in the United States.
• They identified 20,258 new episodes of DMARD use among 13,696 children with JIA (median age, 14 years; 67.5% girls) who newly initiated at least one DMARD.
• Participants were required to have ≥ 365 days of continuous healthcare and pharmacy eligibility prior to the index date, defined as the date of DMARD initiation.

TAKEAWAY:

• The use of csDMARDs declined from 89.5% to 43.2% between 2001 and 2022 (P < .001 for trend), whereas the use of bDMARDs increased from 10.5% to 50.0% over the same period (P < .001).
• Methotrexate was the most commonly used DMARD throughout the study period ; however, as with other csDMARDs, its use declined from 42.1% in 2001 to 21.5% in 2022 (P < .001 ).
• Use of the tumor necrosis factor inhibitor adalimumab doubled from 7% in 2007 to 14% in 2008 and increased further up to 20.5% by 2022; adalimumab also became the most predominantly used b/tsDMARD after csDMARD monotherapy, accounting for 77.8% of prescriptions following csDMARDs in 2022.
• Even though the use of individual TNF inhibitors increased, their overall popularity fell in recent years as the use of newer b/tsDMARDs, such as ustekinumab and secukinumab, increased.

IN PRACTICE:

“These real-world treatment patterns give us insight into how selection of therapies for JIA has evolved with increasing availability of effective agents and help prepare for future studies on comparative DMARD safety and effectiveness,” the authors wrote.

SOURCE:

The study was led by Priyanka Yalamanchili, PharmD, MS, Center for Pharmacoepidemiology and Treatment Science, Institute for Health, Rutgers University, New Brunswick, New Jersey, and was published online October 22, 2024, in Arthritis & Rheumatology.

LIMITATIONS: 

The dependence on commercial claims data may have limited the generalizability of the findings to other populations, such as those with public insurance or without insurance. The study did not have access to demographic data of the participants to investigate the presence of disparities in the use of DMARDs. Moreover, the lack of clinical details about the patients with JIA, including disease severity and specialty of prescribers, may have affected the interpretation of the results.

DISCLOSURES:

The study was supported by funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and several other institutes of the National Institutes of Health, as well as the Rheumatology Research Foundation and the Juvenile Diabetes Research Foundation. No conflicts of interest were reported by the authors.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

The use of newer biologic or targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) for treating juvenile idiopathic arthritis (JIA) rose sharply from 2001 to 2022, while the use of conventional synthetic DMARDs (csDMARDs) plummeted, with adalimumab becoming the most commonly used b/tsDMARD.

METHODOLOGY:

• Researchers performed a serial cross-sectional study using Merative MarketScan Commercial Claims and Encounters data from 2000 to 2022 to describe recent trends in DMARD use for children with JIA in the United States.
• They identified 20,258 new episodes of DMARD use among 13,696 children with JIA (median age, 14 years; 67.5% girls) who newly initiated at least one DMARD.
• Participants were required to have ≥ 365 days of continuous healthcare and pharmacy eligibility prior to the index date, defined as the date of DMARD initiation.

TAKEAWAY:

• The use of csDMARDs declined from 89.5% to 43.2% between 2001 and 2022 (P < .001 for trend), whereas the use of bDMARDs increased from 10.5% to 50.0% over the same period (P < .001).
• Methotrexate was the most commonly used DMARD throughout the study period ; however, as with other csDMARDs, its use declined from 42.1% in 2001 to 21.5% in 2022 (P < .001 ).
• Use of the tumor necrosis factor inhibitor adalimumab doubled from 7% in 2007 to 14% in 2008 and increased further up to 20.5% by 2022; adalimumab also became the most predominantly used b/tsDMARD after csDMARD monotherapy, accounting for 77.8% of prescriptions following csDMARDs in 2022.
• Even though the use of individual TNF inhibitors increased, their overall popularity fell in recent years as the use of newer b/tsDMARDs, such as ustekinumab and secukinumab, increased.

IN PRACTICE:

“These real-world treatment patterns give us insight into how selection of therapies for JIA has evolved with increasing availability of effective agents and help prepare for future studies on comparative DMARD safety and effectiveness,” the authors wrote.

SOURCE:

The study was led by Priyanka Yalamanchili, PharmD, MS, Center for Pharmacoepidemiology and Treatment Science, Institute for Health, Rutgers University, New Brunswick, New Jersey, and was published online October 22, 2024, in Arthritis & Rheumatology.

LIMITATIONS: 

The dependence on commercial claims data may have limited the generalizability of the findings to other populations, such as those with public insurance or without insurance. The study did not have access to demographic data of the participants to investigate the presence of disparities in the use of DMARDs. Moreover, the lack of clinical details about the patients with JIA, including disease severity and specialty of prescribers, may have affected the interpretation of the results.

DISCLOSURES:

The study was supported by funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and several other institutes of the National Institutes of Health, as well as the Rheumatology Research Foundation and the Juvenile Diabetes Research Foundation. No conflicts of interest were reported by the authors.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

The use of newer biologic or targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) for treating juvenile idiopathic arthritis (JIA) rose sharply from 2001 to 2022, while the use of conventional synthetic DMARDs (csDMARDs) plummeted, with adalimumab becoming the most commonly used b/tsDMARD.

METHODOLOGY:

• Researchers performed a serial cross-sectional study using Merative MarketScan Commercial Claims and Encounters data from 2000 to 2022 to describe recent trends in DMARD use for children with JIA in the United States.
• They identified 20,258 new episodes of DMARD use among 13,696 children with JIA (median age, 14 years; 67.5% girls) who newly initiated at least one DMARD.
• Participants were required to have ≥ 365 days of continuous healthcare and pharmacy eligibility prior to the index date, defined as the date of DMARD initiation.

TAKEAWAY:

• The use of csDMARDs declined from 89.5% to 43.2% between 2001 and 2022 (P < .001 for trend), whereas the use of bDMARDs increased from 10.5% to 50.0% over the same period (P < .001).
• Methotrexate was the most commonly used DMARD throughout the study period ; however, as with other csDMARDs, its use declined from 42.1% in 2001 to 21.5% in 2022 (P < .001 ).
• Use of the tumor necrosis factor inhibitor adalimumab doubled from 7% in 2007 to 14% in 2008 and increased further up to 20.5% by 2022; adalimumab also became the most predominantly used b/tsDMARD after csDMARD monotherapy, accounting for 77.8% of prescriptions following csDMARDs in 2022.
• Even though the use of individual TNF inhibitors increased, their overall popularity fell in recent years as the use of newer b/tsDMARDs, such as ustekinumab and secukinumab, increased.

IN PRACTICE:

“These real-world treatment patterns give us insight into how selection of therapies for JIA has evolved with increasing availability of effective agents and help prepare for future studies on comparative DMARD safety and effectiveness,” the authors wrote.

SOURCE:

The study was led by Priyanka Yalamanchili, PharmD, MS, Center for Pharmacoepidemiology and Treatment Science, Institute for Health, Rutgers University, New Brunswick, New Jersey, and was published online October 22, 2024, in Arthritis & Rheumatology.

LIMITATIONS: 

The dependence on commercial claims data may have limited the generalizability of the findings to other populations, such as those with public insurance or without insurance. The study did not have access to demographic data of the participants to investigate the presence of disparities in the use of DMARDs. Moreover, the lack of clinical details about the patients with JIA, including disease severity and specialty of prescribers, may have affected the interpretation of the results.

DISCLOSURES:

The study was supported by funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and several other institutes of the National Institutes of Health, as well as the Rheumatology Research Foundation and the Juvenile Diabetes Research Foundation. No conflicts of interest were reported by the authors.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE: 

In patients with giant cell arteritis (GCA), intravenous methylprednisolone compared with oral glucocorticoids alone does not improve visual acuity and increases the risk for diabetes within the first year. Survival rates do not differ with these two treatments.

METHODOLOGY:

• Researchers conducted a population-based retrospective study at three centers in Sweden to assess the clinical characteristics, treatment-related toxicity, and mortality in patients with GCA who were receiving high-dose intravenous methylprednisolone.
• A total of 419 patients with biopsy-confirmed GCA (mean age at diagnosis, 75 years; 69% women) diagnosed from 2004 to 2019 were included.
• Patients were treated with either intravenous methylprednisolone (n = 111) at a dose of 500-1000 mg per day for 3 consecutive days or oral glucocorticoids alone (n = 308).
• Ischemic visual complications considered to indicate visual involvement were confirmed by an ophthalmologist, and data on visual acuity were collected from ophthalmologic clinic records at initial consultations and follow-up at 3-18 months.

TAKEAWAY:

• Despite a tendency toward improvement, no significant difference in visual acuity was observed with intravenous methylprednisolone compared with oral glucocorticoids.
• Patients treated with intravenous methylprednisolone had a higher risk for newly diagnosed diabetes within a year of GCA diagnosis (odds ratio [OR], 2.59; P = .01).
• The risk for diabetes remained elevated even after adjustment for the cumulative oral glucocorticoid dose at 3 months (adjusted OR, 3.30; P = .01).
• Survival rates did not significantly differ between the treatment groups over a mean follow-up of 6.6 years.

IN PRACTICE:

“In this study on the use of intravenous methylprednisolone treatment in GCA, we found no evidence of a beneficial effect in improving visual acuity or enabling more rapid tapering of the oral glucocorticoid dose,” the authors wrote. “The use of IVMP [intravenous methylprednisolone] was associated with an increased risk of diabetes during the first year compared with oral GC [glucocorticoid], raising questions about the value of IVMP in GCA treatment.”

SOURCE:

The study, led by Hampus Henningson, Department of Clinical Sciences, Rheumatology, Lund University, Lund, Sweden, was published online in Rheumatology.

LIMITATIONS: 

The retrospective nature of the study may have resulted in missing data and difficulty in accurately quantifying the cumulative glucocorticoid doses. The study did not validate the diagnoses of comorbidities but relied solely on diagnostic codes.

DISCLOSURES:

This study was supported by the Swedish Research Council, Swedish Rheumatism Association, Swedish Medical Society, Alfred Österlund’s Foundation, and King Gustaf V’s 80-year foundation. The authors declared no conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE: 

In patients with giant cell arteritis (GCA), intravenous methylprednisolone compared with oral glucocorticoids alone does not improve visual acuity and increases the risk for diabetes within the first year. Survival rates do not differ with these two treatments.

METHODOLOGY:

• Researchers conducted a population-based retrospective study at three centers in Sweden to assess the clinical characteristics, treatment-related toxicity, and mortality in patients with GCA who were receiving high-dose intravenous methylprednisolone.
• A total of 419 patients with biopsy-confirmed GCA (mean age at diagnosis, 75 years; 69% women) diagnosed from 2004 to 2019 were included.
• Patients were treated with either intravenous methylprednisolone (n = 111) at a dose of 500-1000 mg per day for 3 consecutive days or oral glucocorticoids alone (n = 308).
• Ischemic visual complications considered to indicate visual involvement were confirmed by an ophthalmologist, and data on visual acuity were collected from ophthalmologic clinic records at initial consultations and follow-up at 3-18 months.

TAKEAWAY:

• Despite a tendency toward improvement, no significant difference in visual acuity was observed with intravenous methylprednisolone compared with oral glucocorticoids.
• Patients treated with intravenous methylprednisolone had a higher risk for newly diagnosed diabetes within a year of GCA diagnosis (odds ratio [OR], 2.59; P = .01).
• The risk for diabetes remained elevated even after adjustment for the cumulative oral glucocorticoid dose at 3 months (adjusted OR, 3.30; P = .01).
• Survival rates did not significantly differ between the treatment groups over a mean follow-up of 6.6 years.

IN PRACTICE:

“In this study on the use of intravenous methylprednisolone treatment in GCA, we found no evidence of a beneficial effect in improving visual acuity or enabling more rapid tapering of the oral glucocorticoid dose,” the authors wrote. “The use of IVMP [intravenous methylprednisolone] was associated with an increased risk of diabetes during the first year compared with oral GC [glucocorticoid], raising questions about the value of IVMP in GCA treatment.”

SOURCE:

The study, led by Hampus Henningson, Department of Clinical Sciences, Rheumatology, Lund University, Lund, Sweden, was published online in Rheumatology.

LIMITATIONS: 

The retrospective nature of the study may have resulted in missing data and difficulty in accurately quantifying the cumulative glucocorticoid doses. The study did not validate the diagnoses of comorbidities but relied solely on diagnostic codes.

DISCLOSURES:

This study was supported by the Swedish Research Council, Swedish Rheumatism Association, Swedish Medical Society, Alfred Österlund’s Foundation, and King Gustaf V’s 80-year foundation. The authors declared no conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE: 

In patients with giant cell arteritis (GCA), intravenous methylprednisolone compared with oral glucocorticoids alone does not improve visual acuity and increases the risk for diabetes within the first year. Survival rates do not differ with these two treatments.

METHODOLOGY:

• Researchers conducted a population-based retrospective study at three centers in Sweden to assess the clinical characteristics, treatment-related toxicity, and mortality in patients with GCA who were receiving high-dose intravenous methylprednisolone.
• A total of 419 patients with biopsy-confirmed GCA (mean age at diagnosis, 75 years; 69% women) diagnosed from 2004 to 2019 were included.
• Patients were treated with either intravenous methylprednisolone (n = 111) at a dose of 500-1000 mg per day for 3 consecutive days or oral glucocorticoids alone (n = 308).
• Ischemic visual complications considered to indicate visual involvement were confirmed by an ophthalmologist, and data on visual acuity were collected from ophthalmologic clinic records at initial consultations and follow-up at 3-18 months.

TAKEAWAY:

• Despite a tendency toward improvement, no significant difference in visual acuity was observed with intravenous methylprednisolone compared with oral glucocorticoids.
• Patients treated with intravenous methylprednisolone had a higher risk for newly diagnosed diabetes within a year of GCA diagnosis (odds ratio [OR], 2.59; P = .01).
• The risk for diabetes remained elevated even after adjustment for the cumulative oral glucocorticoid dose at 3 months (adjusted OR, 3.30; P = .01).
• Survival rates did not significantly differ between the treatment groups over a mean follow-up of 6.6 years.

IN PRACTICE:

“In this study on the use of intravenous methylprednisolone treatment in GCA, we found no evidence of a beneficial effect in improving visual acuity or enabling more rapid tapering of the oral glucocorticoid dose,” the authors wrote. “The use of IVMP [intravenous methylprednisolone] was associated with an increased risk of diabetes during the first year compared with oral GC [glucocorticoid], raising questions about the value of IVMP in GCA treatment.”

SOURCE:

The study, led by Hampus Henningson, Department of Clinical Sciences, Rheumatology, Lund University, Lund, Sweden, was published online in Rheumatology.

LIMITATIONS: 

The retrospective nature of the study may have resulted in missing data and difficulty in accurately quantifying the cumulative glucocorticoid doses. The study did not validate the diagnoses of comorbidities but relied solely on diagnostic codes.

DISCLOSURES:

This study was supported by the Swedish Research Council, Swedish Rheumatism Association, Swedish Medical Society, Alfred Österlund’s Foundation, and King Gustaf V’s 80-year foundation. The authors declared no conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

SAVANNAH, GEORGIA — There is a lack of understanding among neuromuscular specialists on how to balance the risks for and benefits of corticosteroids when treating patients with generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP), results of a US survey showed.

For both MG and CIDP specialists, uncertainty around corticosteroid dosing, duration, and toxicity underscores the need for more guidance, the investigators noted. Over 85% of respondents indicated that a tool for systematically monitoring corticosteroid toxicity would be valuable.

The results indicate “a lack of knowledge by this pool of neurologists about the guidelines and what they contain,” said study investigator Gil Wolfe, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York.

Clearer guidance on how to administer corticosteroids and manage toxicities in patients with gMG and CIDP “would be welcomed by neurologists and have potential for benefit to patient care,” the team noted.

The findings were presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
 

Lack of Knowledge

Although guidelines for both CIDP and gMG recommend corticosteroids as first-line treatment and emphasize using the lowest effective dose to control symptoms, they do not include specific recommendations on dosing, duration, or toxicity monitoring, the researchers noted.

Despite this, a large proportion of survey respondents reported using guidelines to make clinical decisions on monitoring toxicity, with up to a third actually endorsing a guideline that doesn’t exist.

The cross-sectional, online survey was deployed in November and December 2023 and included 200 US neurologists. Of these, 99 answered questions on CIDP, and 101 answered similar questions on gMG.

To participate in the survey, respondents had to be board-certified neurologists, practicing for at least 2 years post-residency, and have treated or consulted on at least three patients with CIDP or 10 patients with gMG in the past year who were on a corticosteroid dose of at least 10 mg daily for 1 month or more.

CIDP respondents had been practicing a mean of 18.1 years since residency and were board certified in neuromuscular (20%), electrodiagnostic medicine/clinical neurophysiology (21%), and pediatric neurology (8%). Two thirds of them accepted referrals from other neurologists.

The gMG respondents had been practicing a mean of 20.5 years since residency and were board certified in neuromuscular (45%), electrodiagnostic medicine/clinical neurophysiology (35%), and pediatric neurology (17%). A total of 72% accepted referrals from other neurologists.

Respondents estimated that about 60% of their patients with gMG and 58% of patients with CIDP were being treated with corticosteroids, with gMG and CIDP respondents reporting a mean of 26.4 and 15.6 patients, respectively, meeting the study’s dosing criteria.
 

Appropriate Dosing

When asked what chronic, long-term (≥ 6 months) corticosteroid dose they considered safe in terms of minimizing adverse events, 43% of CIDP respondents and 51% of gMG respondents considered corticosteroid doses of 10 mg/d or less (prednisone equivalent) well tolerated; additionally, 32% and 31%, respectively, considered 20-40 mg/d well tolerated. Moreover, they said only about half of their patients would be able to taper to less than 10 mg/d in less than 6 months.

“Studies suggest safety is not seen until patients are on doses at 5 mg/d or less,” Wolfe said. “There is not enough appreciation that doses at levels we once considered safe really do pose significant risk,” he added.

“With the increasing number of treatment options in MG and to a lesser extent in CIDP, we need to do all we can to use corticosteroids as judiciously as possible and be aware of side effects our patients may not even report unless we make a pointed effort to ask about them.”

Familiarity with corticosteroid toxicities was more common among gMG respondents, of whom 77% reported being very/extremely familiar, than among 55% of CIDP respondents. Appetite/weight gain was reported among the most common adverse effects (AEs) associated with long-term CS use (reported by 68% of CIDP and 58% of gMG respondents). Other common AEs reported were insulin resistance (53% of CIDP and 50% of gMG respondents), decreased bone density (47% and 48%, respectively), immunosuppression (37% and 45%, respectively). Mood and behavioral change were noted by 56% of CIDP and 37% of gMG respondents, particularly mood swings, irritability, mania, and sleep disorders.

When asked how they balanced the risk for and benefit of corticosteroids, more than 80% of CIDP specialists reported personally monitoring for corticosteroid-related toxicity, and 42% reported they collaborated with the patient’s primary care provider. However, fewer than 10% reported ordering lab tests. Among neurologists treating gMG, 84% said they typically monitor corticosteroid toxicity independently, while 41% reported doing so in collaboration with primary care providers.

Two thirds of CIDP respondents and 53% of gMG respondents reported using guidelines to make clinical decisions on monitoring toxicity, and 34% of gMG respondents actually endorsed using the Guideline for Systematic Surveillance of Steroid Safety, which does not exist.
 

‘A Big Issue’ in Neurology

Commenting on the results, Said R. Beydoun, MD, professor and division chief, Neuromuscular Medicine, Department of Neurology at Keck Medicine of University of Southern California, Los Angeles, said steroid toxicity is “a big issue” in neurology.

These patients can be on chronic therapy, and they aren’t really monitored for osteoporosis or other complications, he said, adding that neurologists aren’t always taking the necessary precautions to prevent steroid toxicity.

Beydoun estimated that about half of neurologists are not adequately familiar with balancing the efficacy of corticosteroids versus in toxicity.

“Objective improvement, either on the functional scale or the muscle impairment scale — that’s really response treatment. Whereas adverse effects of a treatment are something separate. The patient may be improving but also maybe developing other complications from the treatment,” he said.

Also commenting, Ghazala Hayat, MD, professor of neurology and director of neuromuscular and clinical neurophysiology services at Saint Louis University in St. Louis, said there is a clear need for more education.

“I always say prednisone is our best friend initially, and then it becomes the worst enemy. If you don’t see lots of neuromuscular patients, you might not know even how to recognize toxicity or how to taper. Or the opposite to that, if you taper too quickly, patients relapse.”

The study was funded by argenx. Wolfe reported serving on advisory boards for Alexion, argenx, UCB, and Johnson & Johnson. Neelam Goyal, MD, is a consultant/advisor for Alexion, argenx, Amgen, Janssen, Lycia Therapeutics, and UCB and has received grant support from argenx. Beydoun reported receiving research support and consulting and speaking fees from Healey Center, Amylyx, AB Science, Sanofi, Janssen, Genentech, Regeneron, UCB, Abcuro argenx, Alnylam, AstraZeneca, Amylyx, CSL Behring, Grifols, Takeda, Octapharma, UCB, and Janssen. Hayat reported speaker and advisory roles with argenx, Alexion, and MTPA.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — There is a lack of understanding among neuromuscular specialists on how to balance the risks for and benefits of corticosteroids when treating patients with generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP), results of a US survey showed.

For both MG and CIDP specialists, uncertainty around corticosteroid dosing, duration, and toxicity underscores the need for more guidance, the investigators noted. Over 85% of respondents indicated that a tool for systematically monitoring corticosteroid toxicity would be valuable.

The results indicate “a lack of knowledge by this pool of neurologists about the guidelines and what they contain,” said study investigator Gil Wolfe, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York.

Clearer guidance on how to administer corticosteroids and manage toxicities in patients with gMG and CIDP “would be welcomed by neurologists and have potential for benefit to patient care,” the team noted.

The findings were presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
 

Lack of Knowledge

Although guidelines for both CIDP and gMG recommend corticosteroids as first-line treatment and emphasize using the lowest effective dose to control symptoms, they do not include specific recommendations on dosing, duration, or toxicity monitoring, the researchers noted.

Despite this, a large proportion of survey respondents reported using guidelines to make clinical decisions on monitoring toxicity, with up to a third actually endorsing a guideline that doesn’t exist.

The cross-sectional, online survey was deployed in November and December 2023 and included 200 US neurologists. Of these, 99 answered questions on CIDP, and 101 answered similar questions on gMG.

To participate in the survey, respondents had to be board-certified neurologists, practicing for at least 2 years post-residency, and have treated or consulted on at least three patients with CIDP or 10 patients with gMG in the past year who were on a corticosteroid dose of at least 10 mg daily for 1 month or more.

CIDP respondents had been practicing a mean of 18.1 years since residency and were board certified in neuromuscular (20%), electrodiagnostic medicine/clinical neurophysiology (21%), and pediatric neurology (8%). Two thirds of them accepted referrals from other neurologists.

The gMG respondents had been practicing a mean of 20.5 years since residency and were board certified in neuromuscular (45%), electrodiagnostic medicine/clinical neurophysiology (35%), and pediatric neurology (17%). A total of 72% accepted referrals from other neurologists.

Respondents estimated that about 60% of their patients with gMG and 58% of patients with CIDP were being treated with corticosteroids, with gMG and CIDP respondents reporting a mean of 26.4 and 15.6 patients, respectively, meeting the study’s dosing criteria.
 

Appropriate Dosing

When asked what chronic, long-term (≥ 6 months) corticosteroid dose they considered safe in terms of minimizing adverse events, 43% of CIDP respondents and 51% of gMG respondents considered corticosteroid doses of 10 mg/d or less (prednisone equivalent) well tolerated; additionally, 32% and 31%, respectively, considered 20-40 mg/d well tolerated. Moreover, they said only about half of their patients would be able to taper to less than 10 mg/d in less than 6 months.

“Studies suggest safety is not seen until patients are on doses at 5 mg/d or less,” Wolfe said. “There is not enough appreciation that doses at levels we once considered safe really do pose significant risk,” he added.

“With the increasing number of treatment options in MG and to a lesser extent in CIDP, we need to do all we can to use corticosteroids as judiciously as possible and be aware of side effects our patients may not even report unless we make a pointed effort to ask about them.”

Familiarity with corticosteroid toxicities was more common among gMG respondents, of whom 77% reported being very/extremely familiar, than among 55% of CIDP respondents. Appetite/weight gain was reported among the most common adverse effects (AEs) associated with long-term CS use (reported by 68% of CIDP and 58% of gMG respondents). Other common AEs reported were insulin resistance (53% of CIDP and 50% of gMG respondents), decreased bone density (47% and 48%, respectively), immunosuppression (37% and 45%, respectively). Mood and behavioral change were noted by 56% of CIDP and 37% of gMG respondents, particularly mood swings, irritability, mania, and sleep disorders.

When asked how they balanced the risk for and benefit of corticosteroids, more than 80% of CIDP specialists reported personally monitoring for corticosteroid-related toxicity, and 42% reported they collaborated with the patient’s primary care provider. However, fewer than 10% reported ordering lab tests. Among neurologists treating gMG, 84% said they typically monitor corticosteroid toxicity independently, while 41% reported doing so in collaboration with primary care providers.

Two thirds of CIDP respondents and 53% of gMG respondents reported using guidelines to make clinical decisions on monitoring toxicity, and 34% of gMG respondents actually endorsed using the Guideline for Systematic Surveillance of Steroid Safety, which does not exist.
 

‘A Big Issue’ in Neurology

Commenting on the results, Said R. Beydoun, MD, professor and division chief, Neuromuscular Medicine, Department of Neurology at Keck Medicine of University of Southern California, Los Angeles, said steroid toxicity is “a big issue” in neurology.

These patients can be on chronic therapy, and they aren’t really monitored for osteoporosis or other complications, he said, adding that neurologists aren’t always taking the necessary precautions to prevent steroid toxicity.

Beydoun estimated that about half of neurologists are not adequately familiar with balancing the efficacy of corticosteroids versus in toxicity.

“Objective improvement, either on the functional scale or the muscle impairment scale — that’s really response treatment. Whereas adverse effects of a treatment are something separate. The patient may be improving but also maybe developing other complications from the treatment,” he said.

Also commenting, Ghazala Hayat, MD, professor of neurology and director of neuromuscular and clinical neurophysiology services at Saint Louis University in St. Louis, said there is a clear need for more education.

“I always say prednisone is our best friend initially, and then it becomes the worst enemy. If you don’t see lots of neuromuscular patients, you might not know even how to recognize toxicity or how to taper. Or the opposite to that, if you taper too quickly, patients relapse.”

The study was funded by argenx. Wolfe reported serving on advisory boards for Alexion, argenx, UCB, and Johnson & Johnson. Neelam Goyal, MD, is a consultant/advisor for Alexion, argenx, Amgen, Janssen, Lycia Therapeutics, and UCB and has received grant support from argenx. Beydoun reported receiving research support and consulting and speaking fees from Healey Center, Amylyx, AB Science, Sanofi, Janssen, Genentech, Regeneron, UCB, Abcuro argenx, Alnylam, AstraZeneca, Amylyx, CSL Behring, Grifols, Takeda, Octapharma, UCB, and Janssen. Hayat reported speaker and advisory roles with argenx, Alexion, and MTPA.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — There is a lack of understanding among neuromuscular specialists on how to balance the risks for and benefits of corticosteroids when treating patients with generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP), results of a US survey showed.

For both MG and CIDP specialists, uncertainty around corticosteroid dosing, duration, and toxicity underscores the need for more guidance, the investigators noted. Over 85% of respondents indicated that a tool for systematically monitoring corticosteroid toxicity would be valuable.

The results indicate “a lack of knowledge by this pool of neurologists about the guidelines and what they contain,” said study investigator Gil Wolfe, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York.

Clearer guidance on how to administer corticosteroids and manage toxicities in patients with gMG and CIDP “would be welcomed by neurologists and have potential for benefit to patient care,” the team noted.

The findings were presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
 

Lack of Knowledge

Although guidelines for both CIDP and gMG recommend corticosteroids as first-line treatment and emphasize using the lowest effective dose to control symptoms, they do not include specific recommendations on dosing, duration, or toxicity monitoring, the researchers noted.

Despite this, a large proportion of survey respondents reported using guidelines to make clinical decisions on monitoring toxicity, with up to a third actually endorsing a guideline that doesn’t exist.

The cross-sectional, online survey was deployed in November and December 2023 and included 200 US neurologists. Of these, 99 answered questions on CIDP, and 101 answered similar questions on gMG.

To participate in the survey, respondents had to be board-certified neurologists, practicing for at least 2 years post-residency, and have treated or consulted on at least three patients with CIDP or 10 patients with gMG in the past year who were on a corticosteroid dose of at least 10 mg daily for 1 month or more.

CIDP respondents had been practicing a mean of 18.1 years since residency and were board certified in neuromuscular (20%), electrodiagnostic medicine/clinical neurophysiology (21%), and pediatric neurology (8%). Two thirds of them accepted referrals from other neurologists.

The gMG respondents had been practicing a mean of 20.5 years since residency and were board certified in neuromuscular (45%), electrodiagnostic medicine/clinical neurophysiology (35%), and pediatric neurology (17%). A total of 72% accepted referrals from other neurologists.

Respondents estimated that about 60% of their patients with gMG and 58% of patients with CIDP were being treated with corticosteroids, with gMG and CIDP respondents reporting a mean of 26.4 and 15.6 patients, respectively, meeting the study’s dosing criteria.
 

Appropriate Dosing

When asked what chronic, long-term (≥ 6 months) corticosteroid dose they considered safe in terms of minimizing adverse events, 43% of CIDP respondents and 51% of gMG respondents considered corticosteroid doses of 10 mg/d or less (prednisone equivalent) well tolerated; additionally, 32% and 31%, respectively, considered 20-40 mg/d well tolerated. Moreover, they said only about half of their patients would be able to taper to less than 10 mg/d in less than 6 months.

“Studies suggest safety is not seen until patients are on doses at 5 mg/d or less,” Wolfe said. “There is not enough appreciation that doses at levels we once considered safe really do pose significant risk,” he added.

“With the increasing number of treatment options in MG and to a lesser extent in CIDP, we need to do all we can to use corticosteroids as judiciously as possible and be aware of side effects our patients may not even report unless we make a pointed effort to ask about them.”

Familiarity with corticosteroid toxicities was more common among gMG respondents, of whom 77% reported being very/extremely familiar, than among 55% of CIDP respondents. Appetite/weight gain was reported among the most common adverse effects (AEs) associated with long-term CS use (reported by 68% of CIDP and 58% of gMG respondents). Other common AEs reported were insulin resistance (53% of CIDP and 50% of gMG respondents), decreased bone density (47% and 48%, respectively), immunosuppression (37% and 45%, respectively). Mood and behavioral change were noted by 56% of CIDP and 37% of gMG respondents, particularly mood swings, irritability, mania, and sleep disorders.

When asked how they balanced the risk for and benefit of corticosteroids, more than 80% of CIDP specialists reported personally monitoring for corticosteroid-related toxicity, and 42% reported they collaborated with the patient’s primary care provider. However, fewer than 10% reported ordering lab tests. Among neurologists treating gMG, 84% said they typically monitor corticosteroid toxicity independently, while 41% reported doing so in collaboration with primary care providers.

Two thirds of CIDP respondents and 53% of gMG respondents reported using guidelines to make clinical decisions on monitoring toxicity, and 34% of gMG respondents actually endorsed using the Guideline for Systematic Surveillance of Steroid Safety, which does not exist.
 

‘A Big Issue’ in Neurology

Commenting on the results, Said R. Beydoun, MD, professor and division chief, Neuromuscular Medicine, Department of Neurology at Keck Medicine of University of Southern California, Los Angeles, said steroid toxicity is “a big issue” in neurology.

These patients can be on chronic therapy, and they aren’t really monitored for osteoporosis or other complications, he said, adding that neurologists aren’t always taking the necessary precautions to prevent steroid toxicity.

Beydoun estimated that about half of neurologists are not adequately familiar with balancing the efficacy of corticosteroids versus in toxicity.

“Objective improvement, either on the functional scale or the muscle impairment scale — that’s really response treatment. Whereas adverse effects of a treatment are something separate. The patient may be improving but also maybe developing other complications from the treatment,” he said.

Also commenting, Ghazala Hayat, MD, professor of neurology and director of neuromuscular and clinical neurophysiology services at Saint Louis University in St. Louis, said there is a clear need for more education.

“I always say prednisone is our best friend initially, and then it becomes the worst enemy. If you don’t see lots of neuromuscular patients, you might not know even how to recognize toxicity or how to taper. Or the opposite to that, if you taper too quickly, patients relapse.”

The study was funded by argenx. Wolfe reported serving on advisory boards for Alexion, argenx, UCB, and Johnson & Johnson. Neelam Goyal, MD, is a consultant/advisor for Alexion, argenx, Amgen, Janssen, Lycia Therapeutics, and UCB and has received grant support from argenx. Beydoun reported receiving research support and consulting and speaking fees from Healey Center, Amylyx, AB Science, Sanofi, Janssen, Genentech, Regeneron, UCB, Abcuro argenx, Alnylam, AstraZeneca, Amylyx, CSL Behring, Grifols, Takeda, Octapharma, UCB, and Janssen. Hayat reported speaker and advisory roles with argenx, Alexion, and MTPA.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Teamwork and transdisciplinary collaboration create an effective system of care for amyotrophic lateral sclerosis (ALS), ensuring improved health both for patients and clinicians alike, said one expert.

In a plenary address at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, Ileana Howard, MD, medical co-director of the ALS Center of Excellence at VA Puget Sound in Seattle, said the recently released National Academies report “Living with ALS” cited the Veterans Administration as “a bright spot in the landscape of ALS care due to its interdisciplinary, holistic, and proactive approach to care.”

Since the early 2000s and the publication of several studies linking active military service with ALS, the US Department of Veterans Affairs (VA) has opened an ALS registry, a tissue and brain biobank, and in 2008, granted 100% presumptive service connection to any individual who served more than 90 days of active duty and was later diagnosed with ALS, she said.

“We now serve approximately 4000 veterans with ALS across the system, and we count 47 full interdisciplinary clinics within VA across the nation, with ALS coordinators designated for all 170 VA facilities, regardless of whether they had an ALS clinic or not, to serve as a navigator for patients and their families, to identify the closest ALS clinic that could meet their needs.” 
 

Multidisciplinary vs Interdisciplinary

Howard emphasized that transdisciplinary collaboration is essential for maintaining an effective system. She pointed out that the term “multidisciplinary” is outdated, referring to teams that work independently but in parallel on the same issue.

In contrast, interdisciplinary teams integrate their assessments into a cohesive plan of care, whereas transdisciplinary teams take it further by combining both their assessments and care plans, allowing for greater intentional overlap.

The VA’s ALS handbook lists approximately 20 essential clinicians for a VA ALS clinic, including recreation therapists, assistive technology specialists, and veteran benefit service officers to assist with disability benefits application, among others, she said.

Essential to this collaboration is “role release,” which deliberately blurs the boundaries between disciplines. “The future of our specialty hinges on effective and selfless collaboration,” she said.

Howard encouraged ALS healthcare providers to move away from outdated terminology rooted in hierarchical team models and to break down silos that no longer benefit either the patients or the care teams.

She noted that while teamwork can enhance patient outcomes and overall health, it has also been associated with better health among healthcare providers. It’s well-known, she said, that neurologists and physiatrists are among the specialties with the highest burnout rates, and ALS teams, in particular, experience significant stress and burnout.
 

Better Together

A recent Canadian study on resiliency and burnout in ALS clinics surveyed a wide range of practitioners within ALS centers and found respondents drew resiliency through relationships with patients and colleagues, and that there was a strongly expressed desire for increased resources, team building/debriefing, and formal training in emotional exhaustion and burnout.

“A consistent theme was the lack of adequate allied health support (nursing, social work, occupational therapy) to address the complex needs of patients,” said the report’s senior author Kerri Lynn Schellenberg, MD, medical director of the ALS/Motor Neuron Diseases clinic and associate professor at the University of Saskatchewan College of Medicine in Saskatoon, Saskatchewan, Canada.

“The majority of participants felt they would benefit from more consistent team building exercises and debriefing,” noted the authors.

Schellenberg agreed, emphasizing that care teams perform best when there is mutual appreciation and support among members. By learning from one another and reaching consensus together, the care plan benefits from the collective expertise of the team. “We are stronger together,” she said.

Howard and Schellenberg reported no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Teamwork and transdisciplinary collaboration create an effective system of care for amyotrophic lateral sclerosis (ALS), ensuring improved health both for patients and clinicians alike, said one expert.

In a plenary address at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, Ileana Howard, MD, medical co-director of the ALS Center of Excellence at VA Puget Sound in Seattle, said the recently released National Academies report “Living with ALS” cited the Veterans Administration as “a bright spot in the landscape of ALS care due to its interdisciplinary, holistic, and proactive approach to care.”

Since the early 2000s and the publication of several studies linking active military service with ALS, the US Department of Veterans Affairs (VA) has opened an ALS registry, a tissue and brain biobank, and in 2008, granted 100% presumptive service connection to any individual who served more than 90 days of active duty and was later diagnosed with ALS, she said.

“We now serve approximately 4000 veterans with ALS across the system, and we count 47 full interdisciplinary clinics within VA across the nation, with ALS coordinators designated for all 170 VA facilities, regardless of whether they had an ALS clinic or not, to serve as a navigator for patients and their families, to identify the closest ALS clinic that could meet their needs.” 
 

Multidisciplinary vs Interdisciplinary

Howard emphasized that transdisciplinary collaboration is essential for maintaining an effective system. She pointed out that the term “multidisciplinary” is outdated, referring to teams that work independently but in parallel on the same issue.

In contrast, interdisciplinary teams integrate their assessments into a cohesive plan of care, whereas transdisciplinary teams take it further by combining both their assessments and care plans, allowing for greater intentional overlap.

The VA’s ALS handbook lists approximately 20 essential clinicians for a VA ALS clinic, including recreation therapists, assistive technology specialists, and veteran benefit service officers to assist with disability benefits application, among others, she said.

Essential to this collaboration is “role release,” which deliberately blurs the boundaries between disciplines. “The future of our specialty hinges on effective and selfless collaboration,” she said.

Howard encouraged ALS healthcare providers to move away from outdated terminology rooted in hierarchical team models and to break down silos that no longer benefit either the patients or the care teams.

She noted that while teamwork can enhance patient outcomes and overall health, it has also been associated with better health among healthcare providers. It’s well-known, she said, that neurologists and physiatrists are among the specialties with the highest burnout rates, and ALS teams, in particular, experience significant stress and burnout.
 

Better Together

A recent Canadian study on resiliency and burnout in ALS clinics surveyed a wide range of practitioners within ALS centers and found respondents drew resiliency through relationships with patients and colleagues, and that there was a strongly expressed desire for increased resources, team building/debriefing, and formal training in emotional exhaustion and burnout.

“A consistent theme was the lack of adequate allied health support (nursing, social work, occupational therapy) to address the complex needs of patients,” said the report’s senior author Kerri Lynn Schellenberg, MD, medical director of the ALS/Motor Neuron Diseases clinic and associate professor at the University of Saskatchewan College of Medicine in Saskatoon, Saskatchewan, Canada.

“The majority of participants felt they would benefit from more consistent team building exercises and debriefing,” noted the authors.

Schellenberg agreed, emphasizing that care teams perform best when there is mutual appreciation and support among members. By learning from one another and reaching consensus together, the care plan benefits from the collective expertise of the team. “We are stronger together,” she said.

Howard and Schellenberg reported no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Teamwork and transdisciplinary collaboration create an effective system of care for amyotrophic lateral sclerosis (ALS), ensuring improved health both for patients and clinicians alike, said one expert.

In a plenary address at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, Ileana Howard, MD, medical co-director of the ALS Center of Excellence at VA Puget Sound in Seattle, said the recently released National Academies report “Living with ALS” cited the Veterans Administration as “a bright spot in the landscape of ALS care due to its interdisciplinary, holistic, and proactive approach to care.”

Since the early 2000s and the publication of several studies linking active military service with ALS, the US Department of Veterans Affairs (VA) has opened an ALS registry, a tissue and brain biobank, and in 2008, granted 100% presumptive service connection to any individual who served more than 90 days of active duty and was later diagnosed with ALS, she said.

“We now serve approximately 4000 veterans with ALS across the system, and we count 47 full interdisciplinary clinics within VA across the nation, with ALS coordinators designated for all 170 VA facilities, regardless of whether they had an ALS clinic or not, to serve as a navigator for patients and their families, to identify the closest ALS clinic that could meet their needs.” 
 

Multidisciplinary vs Interdisciplinary

Howard emphasized that transdisciplinary collaboration is essential for maintaining an effective system. She pointed out that the term “multidisciplinary” is outdated, referring to teams that work independently but in parallel on the same issue.

In contrast, interdisciplinary teams integrate their assessments into a cohesive plan of care, whereas transdisciplinary teams take it further by combining both their assessments and care plans, allowing for greater intentional overlap.

The VA’s ALS handbook lists approximately 20 essential clinicians for a VA ALS clinic, including recreation therapists, assistive technology specialists, and veteran benefit service officers to assist with disability benefits application, among others, she said.

Essential to this collaboration is “role release,” which deliberately blurs the boundaries between disciplines. “The future of our specialty hinges on effective and selfless collaboration,” she said.

Howard encouraged ALS healthcare providers to move away from outdated terminology rooted in hierarchical team models and to break down silos that no longer benefit either the patients or the care teams.

She noted that while teamwork can enhance patient outcomes and overall health, it has also been associated with better health among healthcare providers. It’s well-known, she said, that neurologists and physiatrists are among the specialties with the highest burnout rates, and ALS teams, in particular, experience significant stress and burnout.
 

Better Together

A recent Canadian study on resiliency and burnout in ALS clinics surveyed a wide range of practitioners within ALS centers and found respondents drew resiliency through relationships with patients and colleagues, and that there was a strongly expressed desire for increased resources, team building/debriefing, and formal training in emotional exhaustion and burnout.

“A consistent theme was the lack of adequate allied health support (nursing, social work, occupational therapy) to address the complex needs of patients,” said the report’s senior author Kerri Lynn Schellenberg, MD, medical director of the ALS/Motor Neuron Diseases clinic and associate professor at the University of Saskatchewan College of Medicine in Saskatoon, Saskatchewan, Canada.

“The majority of participants felt they would benefit from more consistent team building exercises and debriefing,” noted the authors.

Schellenberg agreed, emphasizing that care teams perform best when there is mutual appreciation and support among members. By learning from one another and reaching consensus together, the care plan benefits from the collective expertise of the team. “We are stronger together,” she said.

Howard and Schellenberg reported no disclosures.
 

A version of this article appeared on Medscape.com.