Corticosteroid Treatment-Resistance in Myasthenia Gravis

Abstract

Chronic, high-dose, oral prednisone has been the mainstay of myasthenia gravis treatment for decades and has proven to be highly beneficial in many, toxic in some way to all, and not effective in a significant minority. No patient characteristics or biomarkers are predictive of treatment response leading to many patients suffering adverse effects with no benefit. Presently, measurements of treatment response, whether taken from clinician or patient perspective, are appreciated to be limited by lack of good correlation, which then complicates correlation to biological measures. Treatment response may be limited because disease mechanisms are not influenced by corticosteroids, limits on dosage because of adverse effects, or individual differences in corticosteroids. This review evaluates potential mechanisms that underlie lack of response to glucocorticoids in patients with myasthenia gravis.

Patient perceptions of disease burden and treatment of myasthenia gravis based on sentiment analysis of digital conversations

“…However, in the present study, lack of efficacy was more prominent in digital conversations among steroid-treated patients (38%) than among IVIg-treated patients (28%), suggesting that patients with MG are concerned about steroid treatments and may be experiencing side effects. These findings are in line with previously published studies 18 , 33 , 34 . Furthermore, key quotes from digital conversations underscored the lack of efficacy as a treatment barrier, especially for steroid-treated patients.…”

The best and worst of times in therapy development for myasthenia gravis

“…7 Despite their usefulness, as many as 30% of patients may either have an inadequate response to steroids or develop intolerable side effects. 8 There was uniform agreement among attendees that there is an urgent and pressing need to develop an alternative to corticosteroids for treating MG. An orally administered agent that is fast-acting, associated with few side effects, better tolerated, and relatively inexpensive is considered a critically important goal for the field. As a complement to short-acting immunotherapeutics, it was noted that little progress has been made in developing symptomatic therapies.…”

“…However, their use is also associated with significant side effects, the burden of which is poorly documented 7 . Despite their usefulness, as many as 30% of patients may either have an inadequate response to steroids or develop intolerable side effects 8 . There was uniform agreement among attendees that there is an urgent and pressing need to develop an alternative to corticosteroids for treating MG. An orally administered agent that is fast‐acting, associated with few side effects, better tolerated, and relatively inexpensive is considered a critically important goal for the field.…”

Healthcare resource utilization, costs and treatment associated with myasthenia gravis exacerbations among patients with myasthenia gravis in the USA: a retrospective analysis of claims data

“…Myasthenia gravis (MG) is a rare, chronic, heterogeneous, autoimmune disease, in which patients present with fluctuating weakness and fatigue of certain muscular groups [ 1–3 ]. Approximately two-thirds of people who receive an MG diagnosis by 40 years of age are female, whereas people with late-onset MG are more likely to be male [ 4 ]. The estimated prevalence of MG in the US is 14 to 20 cases per 100,000 people, although it is likely that MG is underdiagnosed owing to the fluctuating symptoms that people with MG experience [ 5 ].…”