SAVANNAH, Georgia — There is a lack of understanding among neuromuscular specialists on how to balance the risks for and benefits of corticosteroids (CSs) 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 CS dosing, duration, and toxicity underscores the need for more guidance, the investigators noted. Over 85% of respondents indicated that a tool for systematically monitoring CS toxicity would be valuable.
The results indicate “a lack of knowledge by this pool of neurologists about the guidelines and what they contain,” study investigator Gil Wolfe, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in Buffalo, New York, told Medscape Medical News.
Clearer guidance on how to administer CS 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 on October 16 at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Lack of Knowledge
Although guidelines for both CIDP and gMG recommend CSs 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 CS 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 CDIP were being treated with CS, 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) CS dose they considered safe in terms of minimizing adverse events, 43% of CIDP respondents and 51% of gMG respondents considered CS doses ≤ 10 mg/d (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 < 10 mg/d in < 6 months.
“Studies suggest safety is not seen until patients are on doses at 5 mg/d or less,” Wolfe told Medscape Medical News. “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 CS 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 CS 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 CSs, more than 80% of CIDP specialists reported personally monitoring for CS-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 CS 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 for Medscape Medical News, 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 CSs vs 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.