Humanistic burden and medical care patterns of real-world patients with myasthenia gravis in Japan

Introduction Myasthenia gravis (MG) is a rare autoimmune disease characterized by skeletal muscle weakness. As limited real-world data are available in Japan, we aimed to describe the humanistic burden of disease (primary aim), mainly with regards to health-related quality of life (HRQoL), and treatment patterns (secondary aim) in patients with generalized MG (gMG). Methods Data were drawn from the Adelphi Real World MG Disease Specific Programme™, a cross-sectional survey of neurologists and their patients in Japan from August 2023 and January 2024. Analyses were descriptive. Results Overall, 40 neurologists reported data for 128 patients, where 29 patients had self-reported data. Mean (standard deviation) patient age was 57.9 (16.0) years and 53.9% were female. At data collection, 98.4% of patients were receiving maintenance therapy (including novel treatments). Nonsteroidal immunosuppressant therapies were used at first-line of therapy in 54.5% of cases ( n = 67/123). Oral systemic steroids were most used [78.0% of patients at first-line ( n = 96/123), 77.9% at second-line ( n = 53/68), and 75.0% at third-line ( n = 15/20)]. The median (IQR) duration from symptom onset to diagnosis was 2.0 (0.9–4.3) months. Of 28 patients with EQ-5D-5L data, 46.4% reported difficulties with usual activities, 42.9% with mobility, 21.4% with self-care, 53.6% with pain/discomfort, and 39.3% with anxiety/depression. Conclusion Most patients in this Japanese cohort with gMG received maintenance therapy and the time from symptom onset to diagnosis was relatively short. However, impaired HRQoL remained.