Eculizumab shows promise in treating juvenile generalized myasthenia gravis

Eculizumab is effective and well tolerated in reducing the disease burden for adolescents with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis, showing significant improvements in disease severity and quality of life over a 26-week period, according to a study.

This phase 3, open-label, multicenter study involved 11 adolescents aged 12 to 17 years, who received an initial induction dose of eculizumab followed by maintenance doses every 2 weeks. Researchers evaluated changes in disease severity, with the primary endpoint being the change in the Quantitative Myasthenia Gravis (QMG) total score from baseline to week 26.

Results indicated a significant improvement, with a change in the QMG total score of -5.8 (SE 1.2; P = 0.0004) and in the Myasthenia Gravis-Activities of Daily Living total score of -2.3 (SE 0.6; P = 0.0017). All secondary efficacy endpoints also showed statistically significant improvements, which were sustained throughout the study duration.

Complete terminal complement inhibition was maintained in all patients through the 26-week period. The treatment was well tolerated, with all adverse events being mild to moderate and mostly unrelated to eculizumab.

Reference
Brandsema JF, Ginsberg M, Hoshino H, et al; MG 303 Study Group. Eculizumab in Adolescent Patients With Refractory Generalized Myasthenia Gravis: A Phase 3, Open-Label, Multicenter Study. Pediatr Neurol. 2024;156:198-207. doi: 10.1016/j.pediatrneurol.2024.04.020. Epub 2024 Apr 26. PMID: 38810600.