“Clinicians use a lot of prednisone because it works and is cheap - but we miss a big part of the cost equation if we don’t factor in the burden of steroid-toxicity - and the GTI provides a rigorous way to identify and quantify that burden.”
Neelam Goyal, MD is a Clinical Associate Professor in Neurology and Neurological Sciences at Stanford and specializes in the diagnosis, management and electrophysiological testing of neuromuscular disorders, with expertise in immune-mediated disorders such as myositis, myasthenia gravis, chronic inflammatory demyelinating polyneuropathy (CIDP), and vasculitis.
Steroids are often part of the first-line treatment response for these immune-mediated disorders where the immune system erroneously attacks the nerves or muscles. Many of these diseases are rare diseases - for example, there are fewer than 100,000 myasthenia gravis patients in the US. Years ago, more than a third of myasthenia gravis patients suffered an early death; however, today most patients have a normal life span.
During Dr Goyal’s residency at Stanford University Medical Hospital, she was speaking with a neuro-oncologist who told her, “you can either be a disease-hating or patient-loving clinician.”
“This conversation opened my eyes. For me as a clinician, I have been careful to balance out these two approaches and focus on asking the difficult questions about what we are doing to our patients as physicians.”
“As I started becoming aware of the impact of steroid-toxicity on my patients, I also became aware of the lack of literature and understanding about how steroids and steroid-toxicity impact our patients. This is perhaps surprising as a majority of patients with myasthenia gravis will receive glucocorticoids as a first line of treatment.”
To assess the magnitude of the problem for myasthenia gravis patients, Dr Goyal is in the process of enrolling patients in a prospective cohort clinical study. The study leaders aim to enroll 50 myasthenia gravis patients who have been on at least 10 mg of prednisone over three points in the past year.
Data for the GTI will be collected alongside myasthenia gravis outcome measures to answer several important questions about the use of the GTI in this patient population, and to see if early signs of toxicity can be identified.
“My ultimate goal is to highlight the toxicity that we are exposing our patients to by prescribing them steroids, and to better understand the total cost to the patient - especially when we take into account the burden of steroid-toxicity and the impact on the patient as well as the care system.”
The study will not look to alter the clinical care patients receive and will focus on assessing steroid usage and collecting steroid-toxicity data. The team also intends to show that measuring and monitoring steroid-toxicity is important and relatively easy to do, and to make the case for it to become part of standard practice.
“Unlike many other neuromuscular diseases such as ALS, we have some really good therapies for myasthenia gravis patients - and as we look to develop new guidelines, we now have an instrument in the GTI that can help us quantify the benefits in terms of steroid-toxicity reduction.”
Dr Goyal aims to complete patient recruitment in early 2024 and to report the results in 2025. The results will play an important role in informing the development of new guidelines about the usage of steroids for myasthenia gravis patients.
“We have taken a lot of our steroid-toxicity literature and steroid-usage guidelines from rheumatology, and I do wonder if our patients are somehow different and how much of the rheumatology guidelines for steroids are appropriate for our patients.”
Citing literature that shows even low doses for a short period such as 3 months can impact bone health, Dr Goyal recommends that all her patients who are prescribed steroids supplement with calcium and vitamin D.
She also spends considerable time educating patients about the side effects, as there are so many.
“Having a validated instrument like the GTI reminds us to stay mindful at every follow-up visit to check for toxicities. It can also help us create larger datasets and make more informed decisions.”
“At the moment, we have very mixed data, and that makes guidelines fairly vague with everyone interpreting them differently. Studies like ours using the GTI aim to provide more specific insight and highlight even simple things that should be changed. I feel like there are low-hanging fruits in the use of steroids in myasthenia gravis where we could develop best practices that can really help our patients.”
Over the past few years, there has been increased research into new myasthenia gravis treatments - with several new monoclonal antibodies, antibody fragments and new peptide therapeutics having been approved in the past five or six years.
“In myasthenia gravis, we are gaining increased access to targeted therapies that have a much lower toxicity profile than steroids, but these are not cheap medications like prednisone. "
"Clinicians use a lot of prednisone because it works and is cheap - but we miss a big part of the cost equation if we don’t factor in the burden of steroid-toxicity - and the GTI provides a rigorous way to identify and quantify that burden.”
Dr Goyal is a Clinical Associate Professor in Neurology and Neurological Sciences at Stanford and specializes in the diagnosis, management and electrophysiological testing of neuromuscular disorders (including SFEMG), with expertise in immune-mediated disorders (myositis, myasthenia gravis, CIDP, and vasculitis). Her research interests include the monitoring and management of short and long-term toxicity of immunosuppressive agents.
Dr Goyal completed her medical school education at SUNY Downstate in Brooklyn, NY. She then finished her neurology residency including her chief year followed by a fellowship year in neurophysiology with a focus on neuromuscular disorders and EMG nerve conduction studies at Stanford University Medical Hospital.
After graduation, she joined the faculty of Stanford University School of Medicine in 2012 as a Clinical Assistant Professor of Neurology and Neurological Sciences in the division of Neuromuscular Medicine. She took on the position of co-Director of the Muscular Dystrophy Association/ALS Clinic in 2016. In 2020, she was promoted to Clinical Associate Professor. She serves on multiple committees within Stanford, including the Clinical Assistant Professor Appointment and Promotions Committee and Health Information Management Committee, as well as multiple committees within the American Association of Neuromuscular and Neurodiagnostic Medicine (AANEM).