Myasthenia Gravis Activities of Daily Living Deep Phenotyping and Longitudinal

Item-level MG-Activities of Daily Living profiles in myasthenia gravis identify five distinct symptom phenotypes, and longitudinal transitions can occur despite only one-point changes in total scores.

• Why it matters: Aggregate MG-ADL total scores may miss clinically meaningful domain-specific changes and obscure how symptom patterns evolve over time in clinical trials and routine care.
• What they did: MG-ADL evaluations from seven clinical trials and observational studies (5,259 observations) were analyzed with principal component analysis and K-medoids clustering (Cityblock distance), then compared with total-score changes in three longitudinal cohorts.
• The result: Five phenotypes were found—mild ocular (23.7%), severe ocular-dominant (23.5%), bulbar-respiratory dominant (22.6%), severe generalized (16.0%), and proximal limb-axial weakness (14.3%)—with phenotype instability in 38–49% (Explore), 47–49% (Promise), and 20–25% (MGTX) and phenotype shifts often linked to only one-point total-score changes.