Efficacy and Safety of Efgartigimod for Patients With Myasthenia Gravis in a Real-World Cohort of 77 Patients.

ABSTRACT:

Aims

Efgartigimod, a first-in-class neonatal Fc receptor antagonist, is approved for generalized myasthenia gravis (gMG). Its safety and efficacy across MG subtypes remain unclear.

Methods

This single-center real-world study (September 2023-July 2024) analyzed patients from an MG registry study in China. The primary efficacy outcome is the mean MG-ADL score changes from baseline at weeks 4, 8, and 12, analyzed via generalized estimating equations. Safety was assessed by adverse events.

Results

Among 77 patients (mean age 56.1 ± 15.2 years; 59.7% male), 76 completed at least one treatment cycle (20 completed 2 cycles; 1 completed 3 cycles). After efgartigimod treatment, MG-ADL scores decreased significantly by week 4 (mean difference -6.4, 95% CI -7.2 to -5.6, p < 0.00

SUBMITTER: Hao S 

PROVIDER: S-EPMC12001068 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature