Unmet Needs Amongst Patients With Generalized Myasthenia Gravis in the U.S.: A Descriptive Comparison Across Two Time Periods Using the MGFA Global MG Patient Registry
Jenny Park1, Richard Nowak2, Andrea Meyers1, Blanca Canales1, Seth Anderson1, Minjee Park3, Amaia Zurinaga3, Kristina Patterson1, Ankur Bhambri1, Ali Habib4
1Amgen Inc., 2Yale University School of Medicine, 3Alira Health, 4University of California, Irvine
Objective:

To descriptively compare clinical burden, quality of life, comorbidities, and healthcare resource utilization (HRU) among U.S. myasthenia gravis (MG) patients enrolled in the MGFA Global MG Patient Registry (MGFAPR) across two time periods: before and after biologic availability.

Background:
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Design/Methods:
This retrospective, cross-sectional study analyzed enrollment surveys from adults with MG in the MGFAPR. Cohort A comprised patients enrolled in 2013–2017 and Cohort B in 2020–2024. Between-cohort comparisons used descriptive statistics with two-sided tests at α=0.05; an exploratory matched analysis, based on age, gender, and disease duration, was also conducted.
Results:

The analysis included 2,926 adults from MGFAPR, comprising 2,203 in Cohort A and 723 in Cohort B. Mean age at enrollment was 58.0 (SD 15.2) in Cohort A and 59.3 (SD 15.3) in Cohort B, and women comprised 62% and 63% of each cohort, respectively. Biologic use remained modest (Cohort A: 1.6%, Cohort B: 11.0%) and steroid use remained high around 40% in both cohorts; intravenous immunoglobulin (IVIg) (Cohort A: 17%, Cohort B: 27%) and plasma exchange (PLEX) (Cohort A: 5%, Cohort B: 9%) significantly increased.

Clinical burden remained elevated as measured by significantly higher MG-ADL scores (Cohort A: 5.45, Cohort B: 6.12). Quality of life as measured by MG-QoL15r showed no statistical difference. Psychosocial, autoimmune, and cardiovascular comorbid burden significantly increased. HRU was significantly higher in Cohort B (p<0.001) as observed by ER visits (Cohort A: 12%, Cohort B: 19%) and hospitalizations (Cohort A: 8%, Cohort B: 14%). Differences across cohorts persisted, even after matched analyses.

Conclusions:

In this large U.S. registry, patient-reported burden remained substantial in 2020–2024, despite the availability of new targeted therapies, as compared to 2013–2017. While the uptake of new therapies was limited, these findings highlight growing unmet needs and opportunities to further optimize and improve disease management. 

10.1212/WNL.0000000000217208
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