ArticleFrontiers in immunology2026
Clinical outcomes and quality of life associated with eculizumab in patients with late-onset myasthenia gravis.
Article in Frontiers in immunology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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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<0.001); MG-QOL 15r scores reduced by 7.724 and 15.343 points respectively (both P<0.001). Alluvial plots showed that MG-ADL and MG-QOL 15r scores continuously shifted to lower ranges with treatment. MSE cumulative rate was 22.58% at Month 1 and 72.24% at Month 12, with CMI rate rose from 51.61% to 96.41%. Mean prednisone daily dose decreased by 7.574 mg/day (P<0.001). Sensitivity analyses using multiple imputation and conservative baseline observation carried forward (BOCF) yielded consistent results, supporting the robustness of the findings. One patient reported headache (CTCAE 1 grade), no serious adverse events occurred. Conclusion: Eculizumab was associated with clinical improvement in patients with refractory AChR-Ab(+)-generalized LOMG. In this cohort, patients demonstrated rapid symptom reduction and improvements in long-term quality of life.
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