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Abstract Details

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
Neuromuscular and Clinical Neurophysiology (EMG)
P4 - Poster Session 4 (8:00 AM-9:00 AM)
9-005

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.

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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.

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.

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. 

Authors/Disclosures
Jenny Y. Park, PharmD, MS
PRESENTER
Dr. Park has received personal compensation for serving as an employee of Amgen Inc.. Dr. Park has or had stock in Amgen Inc..
Richard J. Nowak, MD (Yale University School of Medicine) Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Nowak has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio . Dr. Nowak has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amgen. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cour Pharma. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMD Serono. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janseen . The institution of Dr. Nowak has received research support from UCB. The institution of Dr. Nowak has received research support from Alexion . The institution of Dr. Nowak has received research support from Janseen. The institution of Dr. Nowak has received research support from Immunovant . The institution of Dr. Nowak has received research support from argenx. The institution of Dr. Nowak has received research support from Amgen. Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities.
Andrea Meyers, DrPH Dr. Meyers has received personal compensation for serving as an employee of Amgen. Dr. Meyers has stock in Amgen.
Blanca Canales, PharmD Dr. Canales has received personal compensation for serving as an employee of Amgen. Dr. Canales has stock in Amgen .
Michael Anderson, MD Dr. Anderson has nothing to disclose.
Minjee Park, MSc Ms. Park has received personal compensation for serving as an employee of Alira Health.
Amaia Zurinaga Miss Zurinaga has nothing to disclose.
Kristina R. Patterson, MD, PhD (Horizon Therapeutics) Dr. Patterson has received personal compensation for serving as an employee of Amgen. Dr. Patterson has stock in Amgen.
Ankur Bhambri, DO Dr. Bhambri has received personal compensation for serving as an employee of Amgen. Dr. Bhambri has stock in Amgen.
Ali A. Habib, MD (University of California, Irvine) Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon/ Amgen. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jansen/Johnson & Johnson. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMDSerono/Merck. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jansen/Joohnson & Johnson. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from Horizon/Amgen. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Regeneron. The institution of Dr. Habib has received research support from Arcellx. The institution of Dr. Habib has received research support from Novartis. The institution of Dr. Habib has received research support from EMDSerono/Merck. The institution of Dr. Habib has received research support from Cour Pharmaceuticals.