Treatment Burden According to Patients with Generalized Myasthenia Gravis
Katherine Ruzhansky1, Neelam Goyal2, Suraj Muley3, Tuan Vu4
1Medical University of South Carolina, 2Stanford University, 3Gregory W. Fulton ALS and Neuromuscular Center, 4University of South Florida
Objective:
To understand experiences of patients with generalized myasthenia gravis (gMG) and identify challenges and unmet needs.
Background:
Advances in gMG treatments have led to marked reduction in mortality associated with the disease. However, many patients living with gMG continue to experience high disease and treatment burden related to persistent symptoms and toxicity of currently available treatments. 
Design/Methods:
In May 2021, we conducted a cross-sectional study of U.S. adults with a self-reported gMG diagnosis. Respondents were recruited from online panels to complete a survey about their condition and treatment. The study received IRB exemption.
Results:

152 patients with gMG completed the online survey. Mean disease duration since diagnosis was 12.5 years. The majority (72%) of patients agreed their current treatment was effective in controlling their gMG symptoms, but most (75%) reported not being in remission. Two-thirds said it took a long time to get a treatment plan in place that works for them. Patients were taking an average of 2.4 treatments for gMG, primarily acetylcholinesterase inhibitors (77%), non-steroidal immunosuppressants (NSID, 47%), and corticosteroids (CS, 41%). Side effects were common, and nearly all those on CS and NSIDs expressed concerns regarding short and long-term side effects. Some patients (22%) did not want to risk gMG exacerbation by switching or adding treatments as it took a while to find a stable therapeutic regimen. Many patients (63%) reported a high willingness to take any measure possible even if that means experiencing treatment and/or side effect burden. Most (89%) agreed that treatments specifically targeting the cause of gMG would advance the management of this disease.

Conclusions:
Although many patients believe their current treatment regimen is effective, there is room for improvement in disease control, treatment toxicity, and amount of time to arrive at an effective treatment. There is preference for targeted gMG therapy with low side effect profile.