[Pubmed] Clinical outcomes and quality of life associated with eculizumab in patients with late-onset myasthenia gravis
Posté : 29 juin 2026 12:00
Front Immunol. 2026 Jun 12;17:1834680. doi: 10.3389/fimmu.2026.1834680. eCollection 2026.
ABSTRACT
INTRODUCTION: Late-onset myasthenia gravis (LOMG) features prominent immunosenescence, high myasthenic crisis risk, and refractoriness due to comorbidities limiting conventional immunosuppressants, leading to poor prognosis. Eculizumab, which blocks complement C5 activation, may provide rapid symptom improvement and has shown an acceptable safety profile in previous studies, holding potential for LOMG management. Although the REGAIN trial and some real-world studies have demonstrated the efficacy of eculizumab, evidence for its use in patients with refractory LOMG is scarce. This study aimed to evaluate the clinical outcomes, safety profile, and impact on quality of life of eculizumab treatment in patients with refractory acetylcholine receptor antibody-positive [AChR-Ab(+)] generalized LOMG.
METHODS: This study retrospectively enrolled 31 patients with refractory AChR-Ab(+) generalized LOMG treated with eculizumab for ≥2 months. Primary outcome was change in myasthenia gravis activities of daily living (MG-ADL) score analyzed using mixed models for repeated measures. Secondary outcomes included 15-item Revised Myasthenia Gravis Quality of Life Questionnaire (MG-QOL 15r) score, Minimal Symptom Expression (MSE) response rate, clinically meaningful improvement (a decrease of ≥3 points in MG-ADL, CMI) in ADL, and concomitant immunosuppressant dosage reduction. Safety was assessed via drug-related adverse events.
RESULTS: The mean age of onset in the 31 patients with refractory LOMG was 62.87 ± 5.95 years, with a mean follow-up duration of 10 ± 4.29 months after eculizumab treatment. Compared with baseline, the MG-ADL score was reduced by 2.976 points at Month 1 and 5.430 points at Month 12 (both P
Source: https://pubmed.ncbi.nlm.nih.gov/4236779 ... 2&v=2.20.0