Minimal Symptom Expression in Generalized Myasthenia Gravis: A Valuable Patient-Centric Treatment Goal - PubMed
3 hours ago
- #treatment goals
- #myasthenia gravis
- #patient-centric care
- Generalized myasthenia gravis (gMG) is a chronic autoimmune neuromuscular disorder causing muscle weakness and fatigue.
- Minimal Symptom Expression (MSE), defined as a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1, is a patient-centric treatment goal.
- MSE aligns with regulatory priorities for patient-reported outcomes and reflects minimal functional burden.
- Phase 3 trial data show MSE is achievable with biologic therapies like complement and neonatal Fc receptor inhibitors.
- Achieving MSE correlates with improvements in physician-assessed outcomes and quality of life metrics.
- Inconsistencies in defining and reporting sustained MSE limit comparability across trials.
- MSE can be used alongside clinician-assessed and safety measures for an integrated treatment goal in gMG.
- Future research should further define the clinical utility of MSE and incorporate stakeholder input for validation.