Myasthenia gravis (MG) is a heterogeneous disorder whose clinical presentation ranges from mild ocular deficits to severe widespread weakness. This variance poses a challenge when quantifying clinical deficits. Deficits and symptoms are quantified using standardized clinical scales and questionnaires which are often used as outcome measures. The past decades have seen the development of several validated outcome measures in MG, which are used in clinical trials to obtain regulatory approval. In recent years, emphasis has moved from objective assessments to patient-reported outcomes. Despite a growing body of literature on the validity of the MG-specific outcome measures, several unresolved factors remain. As several novel therapeutics are currently in clinical development, knowledge about capabilities and limitations of outcome measures is needed. In the present paper, we describe the most widely used clinical classifications and scales in MG. We highlight the choice of outcome measures in published and ongoing trials, and we denote whether trial efficacy was reached on these outcomes. We discuss advantages and limitations of the individual scales, and discuss some of the unresolved factors relating to outcome assessments in MG.

No abstract

No abstract

Introduction/Aims It is uncertain whether clinical outcomes differ between male and female patients with myasthenia gravis (MG) while receiving standard clinical care. Methods In a prospective cohort study of 107 patients with MG receiving standard of care from 2012 to 2019, the Quantitative MG (QMG), the MG Composite (MGC), the MG Activities of Daily Living (MG‐ADL), and the MG Quality of Life 15‐Items (QOL15) were determined. Clinical outcomes were analyzed in relation to gender. Results Mean follow‐up time was 4.8 (±0.4) y, and 70 patients completed all follow‐up assessments. Patients improved on all clinical scores: QMG ‐1.8 (P < .001), MGC ‐1.5 (P < .001), MG‐ADL ‐1.3 (P < .001), and QOL15 ‐3.0 (P < .001). Females improved less than males on the QMG (P = .01), MGC (P < .001), MG‐ADL (P = .006), and QOL15 (P < .001) independent of potential confounders. Males had moderate to strong correlations between changes in all clinical scales (r range 0.52 to 0.73), whereas females had weak correlations between changes in the QMG and MG‐ADL (r = 0.13), the QMG and QOL15 (r = 0.27), the MGC and MG‐ADL (r = 0.21), the MGC and QOL15 (r = 0.00), and the MG‐ADL and QOL15 (r = 0.31). Discussion Females improved less compared to males on objective and patient‐reported outcomes. Moreover, females improved more on objective measures than on patient‐reported outcomes. These gender differences should receive attention in clinical care and in the design of future trials.