MINIMAL SYMPTOM EXPRESSION IN GENERALIZED MYASTHENIA GRAVIS TREATED WITH TRADITIONAL THERAPIES: A REAL-WORLD BENCHMARK FROM MYREALWORLD-MG
Author(s)
Cynthia Qi, BA, MBA1, Perseverence Savieri, PhD2, James F. Howard, Jr., MD, PhD3, Andreas Meisel, MD, PhD4, Hiroyuki Murai, MD, PhD5, Renato Mantegazza, MD, PhD6, Kristin Heerlein, MD, PhD, MBA1, Glenn A. Phillips, PhD1, Febe Brackx, MSc2, Sarah Dewilde, PhD2.
1argenx, Ghent, Belgium, 2SHE BV, Brussels, Belgium, 3Department of Neurology, The University of North Carolina at Chapel Hill, CHAPEL HILL, NC, USA, 4Department of Neurology and Experimental Neurology, Charité – Universitätsmedizin Berlin, Berlin, Germany, 5Department of Neurology, International University of Health and Welfare, Narita, Japan, 6Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
1argenx, Ghent, Belgium, 2SHE BV, Brussels, Belgium, 3Department of Neurology, The University of North Carolina at Chapel Hill, CHAPEL HILL, NC, USA, 4Department of Neurology and Experimental Neurology, Charité – Universitätsmedizin Berlin, Berlin, Germany, 5Department of Neurology, International University of Health and Welfare, Narita, Japan, 6Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
OBJECTIVES: Minimal symptom expression (MSE; MG-ADL 0-1) is an emerging treatment target in generalized myasthenia gravis (gMG). Real-world evidence on MSE achievement under traditional therapies, and on whether MSE corresponds to meaningful gains in broader humanistic, work, and caregiver outcomes, remains limited.
The objective is to assess MSE prevalence and examine associations with humanistic burden, work impact, and caregiver support in adults with gMG.
METHODS: This cross-sectional analysis evaluated the enrollment survey of 1,029 adults with gMG (MGFA class II-V) from MyRealWorld-MG, a nine-country observational study of MG (2019-2024). Outcomes included MG-ADL, EQ-5D-5L, MG-QoL15r, HADS, work impairment, and caregiver support. General-population benchmarks were obtained from POPUP, a multi-country digital observational study (n=9,000). Multivariable logistic regression identified factors associated with MSE.
RESULTS: Overall MSE prevalence was 12.2% (125/1,029). Among non-MSE patients, mean (SD) MG-ADL score was 7.3 (3.6), with 74.1% having moderate-severe burden (MG-ADL≥5). Most (84.7%) received conventional oral therapies; 18.4% used immunoglobulin or plasma exchange; 4.9% received rituximab and 4.1% received eculizumab. Patients in MSE reported better humanistic outcomes than non-MSE, with outcomes approaching general-population norms: EQ-5D utility (0.909 vs 0.679; POPUP 0.841), MG-QoL15r (6.0 vs 14.7; POPUP 4.9), HADS anxiety (6.43 vs 8.82; POPUP 6.43), and HADS depression (4.68 vs 7.61; POPUP 5.01). Work and caregiver outcomes showed similar findings: stopped working (2.6% vs 12.9%; POPUP 3.4%), sick leave (21.7% vs 39.8%; POPUP 10.9%), and caregiver need (7.4% vs 45.4%; POPUP 8.3%). Higher MGFA class, greater comorbidity burden, and shorter time since diagnosis were independently associated with lower odds of having MSE (p<0.001, p<0.001, and p=0.025).
CONCLUSIONS: In a real-world cohort treated predominantly with traditional therapies, overall MSE prevalence was 12%, providing a benchmark before broad uptake of targeted biologics. Patients in MSE reported humanistic, work, and caregiver outcomes approaching general-population levels, supporting MSE as a clinically meaningful, patient-centric treatment target.
The objective is to assess MSE prevalence and examine associations with humanistic burden, work impact, and caregiver support in adults with gMG.
METHODS: This cross-sectional analysis evaluated the enrollment survey of 1,029 adults with gMG (MGFA class II-V) from MyRealWorld-MG, a nine-country observational study of MG (2019-2024). Outcomes included MG-ADL, EQ-5D-5L, MG-QoL15r, HADS, work impairment, and caregiver support. General-population benchmarks were obtained from POPUP, a multi-country digital observational study (n=9,000). Multivariable logistic regression identified factors associated with MSE.
RESULTS: Overall MSE prevalence was 12.2% (125/1,029). Among non-MSE patients, mean (SD) MG-ADL score was 7.3 (3.6), with 74.1% having moderate-severe burden (MG-ADL≥5). Most (84.7%) received conventional oral therapies; 18.4% used immunoglobulin or plasma exchange; 4.9% received rituximab and 4.1% received eculizumab. Patients in MSE reported better humanistic outcomes than non-MSE, with outcomes approaching general-population norms: EQ-5D utility (0.909 vs 0.679; POPUP 0.841), MG-QoL15r (6.0 vs 14.7; POPUP 4.9), HADS anxiety (6.43 vs 8.82; POPUP 6.43), and HADS depression (4.68 vs 7.61; POPUP 5.01). Work and caregiver outcomes showed similar findings: stopped working (2.6% vs 12.9%; POPUP 3.4%), sick leave (21.7% vs 39.8%; POPUP 10.9%), and caregiver need (7.4% vs 45.4%; POPUP 8.3%). Higher MGFA class, greater comorbidity burden, and shorter time since diagnosis were independently associated with lower odds of having MSE (p<0.001, p<0.001, and p=0.025).
CONCLUSIONS: In a real-world cohort treated predominantly with traditional therapies, overall MSE prevalence was 12%, providing a benchmark before broad uptake of targeted biologics. Patients in MSE reported humanistic, work, and caregiver outcomes approaching general-population levels, supporting MSE as a clinically meaningful, patient-centric treatment target.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR144
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)