Clinical Outcomes and Events in a United States Generalized Myasthenia Gravis Population: Analysis of Real-world Data
Lesley-Ann Miller-Wilson1, Lincy Lal1, Joe Conyers2, Shiva Lauretta Birija2, Ciara Ringland2, Hannah Connolly2, Gregor Gibson2, Niall Hatchell2, Yuriy Edwards1
1Immunovant, Inc., 2Adelphi Real World
Objective:
To describe real-world clinical status and treatment patterns for a generalized myasthenia gravis (gMG) population in the United States (US).
Background:
gMG is a chronic autoimmune condition caused by pathologic autoantibodies that disrupt neuromuscular transmission, causing muscle weakness and fatigue. Treatments aim to improve clinical outcomes and reduce the frequency of exacerbations and life-threatening myasthenic crises.
Design/Methods:
Data were drawn from the Adelphi Myasthenia Gravis (MG) II Disease Specific Programme, with US data collected from February through August 2024.
Results:

Overall, 49 physicians reported on 312 patients with gMG (mean [SD] age, 55.5 [14.2] years; 53.2% male; 79.8% Caucasian/White). Patients had a mean (SD) time since diagnosis of 4.0 (5.3) years, 71.8% were anti-acetylcholine receptor antibody-positive, and 75.3% had comorbidities, most frequently hypertension (39.1%). Most patients were Myasthenia Gravis Foundation of America Class II or above (78.5%), and the mean (SD) MG-Activities of Daily Living (MG-ADL) score was 4.1 (3.1); mean MG-ADL scores in patients aged <50 and ≥50 years were 3.8 (2.9) and 4.3 (3.3), respectively. Patients had tried a mean (SD) of 1.6 (1.0) treatment regimens, and 81.7% were receiving maintenance treatment, with 46.3% receiving novel therapies (complement inhibitors, 16.5%; neonatal Fc receptor inhibitors, 15.7%; immunoglobulins, 12.5%). Exacerbation and myasthenic crisis since diagnosis were reported for 28.9% and 13.4% of patients, respectively; 45.6% of those with an exacerbation and 31.4% of those with a crisis experienced an event in the prior 12 months. Overall, 14.9% of patients had ≥1 MG-related hospitalization in the prior 12 months, and 17.3% had undergone thymectomy. 
Conclusions:
Despite the use of novel therapies, patients with gMG continue to experience clinical exacerbations and myasthenic crises, highlighting suboptimal disease control. Further longitudinal analyses are required to understand the real-world impact of new and emerging therapies on clinical outcomes, as well as the associated costs.
10.1212/WNL.0000000000209104
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