Patient-Reported Outcomes from a Survey of Real-World Generalized Myasthenia Gravis Patients in the United States

Topic:

Other

Poster Number: P324

Author(s):

Lesley-Ann Miller-Wilson, PhD, Immunovant, Joe Conyers, MSc, Adelphi Real World, Shiva Lauretta Birija, BSc, Adelphi Real World, Ciara Ringland, MSc, Adelphi Real World, Bollington, UK, Hannah Connolly, Adelphi Real World, Gregor Gibson, BSc, Adelphi Real World, Lincy Lal, PharmD, PhD, Immunovant, Inc., Yuriy Edwards, MD, PhD, Immunovant, Inc.

Background: Generalized myasthenia gravis (gMG) is a chronic neuromuscular condition characterized by skeletal muscle weakness and fatigue. Symptoms impair patients’ ability to perform activities of daily living (ADL) and negatively impact employment and quality of life (QoL).
Objective: To describe patient-reported outcome measures (PROM) in patients with gMG.
Methods: Data were drawn from a cross-sectional survey of patients with gMG in the US in October 2024. Patients reported demographic characteristics, current symptoms, prescribed treatments, and PROM including the Myasthenia Gravis (MG)-ADL, MG-QoL-15r, Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue, EuroQol 5-dimension 5-level (EQ-5D-5L) (US tariff), EuroQol Visual Analogue Scale (EQ-VAS), and Work Productivity and Activity Impairment (WPAI) questionnaires.
Results: Patients (N=244) were primarily female (88.1%), with a mean (SD) age of 49.0 (14.5) years at time of survey and 36.0 (16.7) years at first onset of symptoms. The most frequent current symptoms were physical fatigue (88.9%), weakness in the legs (69.3%) or arms (68.4%), blurred or double vision (66.0%) and mental fatigue (65.6%). At the time of survey completion, acetylcholinesterase inhibitors were the most commonly prescribed medications (74.6%), followed by non-steroidal immunosuppressants (43.0%), corticosteroids (40.6%), immunoglobulins/plasmapheresis (29.9%), neonatal fragment crystallizable receptor inhibitors (16.0%) and complement inhibitors (13.9%). Mean (SD) scores on the MG-ADL, MG-QOL-15r, FACIT-Fatigue, EQ-VAS and EQ-5D-5L were 8.3 (3.7), 16.3 (7.4), 20.6 (11.3), 58.6 (18.5), and 0.67 (0.21), respectively. Overall QoL was subjectively reported as “poor” or “very poor” by 18.4% of patients, and 30.3% reported having worse QoL than 12 months ago. Mean (SD) WPAI scores for overall productivity loss and activity impairment were 42.0% (25.0%) and 55.2% (25.2%), respectively.
Conclusion: This gMG patient population reported ongoing symptoms and worsening QoL >10 years after initial onset, despite prescribed treatments. PROM scores highlight specific areas of unmet need, including impaired ADL, fatigue, and reduced ability to work.