Areas of Unmet Need Continue to Burden Patients With gMG

Impaired activities of daily living (ADL), fatigue, and reduced ability to work continue to present themselves as areas of unmet need among patients who have generalized myasthenia gravis (gMG), highlighting the significance of patient-reported outcomes measures in facilitating a greater understanding of the chronic autoimmuneneuromuscular disorder by this patient population and the clinicians who treat them.1

New real-world data from an October 2024 cross-sectional survey presented at the 2025 MDA Clinical & Scientific Conference reveal the persistent symptoms and worsening quality of life these patients must endure, despite being adherent to prescribed treatment regimens.

Rare Patient Voice facilitates connections between patients and caregivers of patients living with rare and nonrare diseases.2 Patients for the cross-sectional survey were recruited through this organization, with adult patients confirming their gMG diagnosis considered eligible for inclusion. Each patient provided information on demographics, symptoms, and baseline treatments, and their results for several scales: Myasthenia Gravis Activities of Daily Living (MG-ADL), Myasthenia Gravis Quality of Life 15-item Scale-Revised revised (MG-QoL-15r), Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue, 5-level EuroQoL-5 dimensions (EQ-5D-5L), and Work Productivity and Activity Impairment (WPAI).

Class III disease was most common (49.2%), per Myasthenia Gravis Foundation of America criteria, and class V disease was least common (2.5%).

Patient-reported outcomes measures in generalized myasthenia gravis are more important than ever to have a more nuanced understanding of experiences and difficulties. | Image Credit: © tippapatt-stock.adobe.com

Survey Findings

Of the 244 patients, 88.1% reported female biological sex; mean (SD) ages of 49.0 (14.45) years when they took the survey, 36.0 (16.7) years when their symptoms began, and 37.7 (16.3) years when they received their gMG diagnosis; and 0.3 (1.3) myasthenic crises and 5.1 (10.5) exacerbations in the past year. Their most common treatment overall was acetylcholinesterase inhibitors, and among those taking a biologic (41%), an FcRn inhibitor was the most common vs a complement inhibitor or rituximab or a biosimilar to rituximab (16.9% vs 13.9% vs 12.3%, respectively). Of the 29.9% receiving immunoglobulin therapy or plasmapheresis, the former was much more common (25.4% vs 4.9%).

Close to half of patients reported their overall quality of life to be poor or very poor (18.4%) and that this had worsened vs the same time of the previous year (30.3%), while mean overall and disease class scores on the MG-ADL, MG-QoL-15r, FACIT-Fatigue, and EQ-5D-5L bear out the negative impact of the disease:

• MG-ADL: 8.3 overall and rising continuously with each higher disease class to 13.0 for those with class V disease
• MG-QoL-15r: 16.3 and rising from class I (7.9) to class IV (22.4) disease before experiencing a slight drop among those with class V disease (21.6)
• FACIT-Fatigue: 20.6 and decreasing for class I (29.7) through class IV (12.8) disease before slightly improving for those with class V disease (17.0)
• EQ-5D-5L: 0.67 and worsening with each higher disease class to 0.40 for those with class V disease 

Higher scores on the MG-ADL and MG-QoL-15r indicate greater impairment and worsening quality of life, while lower scores on the FACIT-Fatigue and EQ-5D-5L indicate worsening fatigue and utilities.

There were universal reductions in work ability across the entire patient population of this survey, as indicated by activity and work impairment. The overall mean score for activity impairment was 55.2% (25.2%), and this progressively worsened for each disease class: class I, 35.7% (24.4%); class II, 42.4% (24.0%); class III, 60.0% (21.4%); class IV, 71.6% (22.2%); and class IV, 73.3% (34.5%).

The results for work impairment saw greater fluctuation and were only calculated among those who reported they were working at the time of the survey. Overall, the mean score was 42.0% (25.0%) and for class I, 47.7% (26.5%); class II, 34.2% (24.8%); class III, 47.6%, 23.7%; class IV, 41.4% (27.5%); and class V, 20.0% (0.0%).

Limitations

The authors highlight that because theirs was not a true random sampling of patients, inclusion criteria relied on patient self-recall, and participation hinged on each person’s willingness to complete the survey, generalizability of these findings is reduced.

Still, they believe their data underscore that persons living with gMG could be better served with more comprehensive and targeted resources that focus on fatigue and impaired activities of daily living and ability to work.

References

1. Miller-Wilson LA, Conyers J, Birija SL, et al. Patient-reported outcomes from a real-world United States generalized myasthenia gravis patient survey. Presented at: 2025 MDA Clinical & Scientific Conference; March 16-18, 2025; Dallas, TX. Poster 324.

2. About us. Rare Patient Voice. Accessed April 1, 2025. https://rarepatientvoice.com/about-us/