Disease burden among patients with generalized myasthenia gravis in the United States receiving standard of care therapy: the patient perspective

Examination of disease burden experienced by patients with gMG treated with standard of care and not with advanced biologics can inform treatment decision-making. A cross-sectional, online survey was completed by adults with generalized myasthenia gravis (gMG; patients self-reported a confirmed diagnosis) in the United States. Patients had received treatment with standard of care (acetylcholinesterase inhibitors, non-steroidal immunosuppressants, corticosteroids, rituximab, immunoglobulins, plasmapheresis) but not advanced biologics (eculizumab, ravulizumab, zilucoplan, efgartigimod, rozanolixizumab, nipocalimab). Responses were compared between patients with mild or moderate/severe gMG (Myasthenia Gravis Activites of Daily Living [MG-ADL] score 0–4 or 5–24). Linear elastic-net regression analysis explored associations between MG-ADL score and selected variables. Overall, 157 eligible patients completed the survey (87.3% female, mean [SD] age 49.5 [14.4] years, mean [SD] time since diagnosis 11.0 [10.9] years). Mean (SD) MG-QoL-15R score was 16.2 (7.2), EQ5D-5L score was 0.6 (0.3), and FACIT-fatigue subscale score was 20.2 (11.3). Overall, 51.6% received additional support/care. Patients with moderate/severe gMG (136/157 [86.6%]) had greater symptom occurence, reduced treatment satisfaction, greater health-related quality of life impairment, and greater support/care needs, compared with patients with mild gMG. In a regression analysis, gMG severity was associated with severity of health-related quality of life impairment (as measured by MG-ADL score and MG-QOL-15R score; coefficient 0.103 [95% CI 0.043, 0.191]). Patients in the United States with gMG who are receiving only standard of care report a significant disease burden and impaired quality of life, with greater impairment among patients with severe gMG. Myasthenia gravis is a rare autoimmune disease that causes muscle weakness. At first, the muscles around your eyes can become weak (called “ocular myasthenia gravis”) but over time muscles in other parts of the body can also become weak (called “generalized myasthenia gravis”). It is important to understand the symptoms faced by people with myasthenia gravis and how these affect their quality of life, because this can help physicians to decide which treatments would help them the most. In this study, 157 people with generalized myasthenia gravis in the United States were asked to complete an online survey, answering questions about different aspects of their disease and their lived experience. All had received “standard of care” treatments but had not received newer, advanced biologics called complement inhibitors and neonatal Fc receptor blockers. The survey responses showed that many people had eye symptoms, muscle weakness, poor quality of life, and a need for support from a relative. This was more likely for people with moderate or severe myasthenia gravis and less likely for people with mild myasthenia gravis. This means that some people with myasthenia gravis in the United States who are receiving “standard of care” do not have good control of their symptoms despite receiving treatment.