2026 Volume 43 Issue 3 Pages 387-395
Background :
Complement inhibitor administration has recently been introduced for myasthenia gravis (MG) cases that show insufficient response to conventional immunotherapies. To date, real–world use data are limited. We evaluated the clinical characteristics and treatment responses of patients with generalized MG treated with complement inhibitors at a single center.
Methods :
We retrospectively analyzed 21 patients with acetylcholine receptor antibody–positive generalized MG who initiated complement inhibitor therapy between April 2019 and May 2025. Thirteen patients received ravulizumab (ravulizumab group) and eight received zilucoplan (zilucoplan group). Clinical background, MG severity scale scores, and hospitalization frequency due to MG exacerbation were compared between the two groups. Clinical outcomes were evaluated for up to 26 weeks after treatment initiation.
Results :
Patients in the zilucoplan group tended to exhibit more severe symptoms at baseline than those in the ravulizumab group. Despite this difference, improvement in disease severity was observed in both groups during the 26–week observation period. Four ravulizumab group patients and three zilucoplan group patients achieved minimal manifestation status at 26 weeks. The number of hospitalizations related to MG exacerbation significantly decreased in both groups after treatment initiation. Treatment was discontinued in several patients.
Conclusions :
While complement inhibitors are associated with clinical improvement and reduced hospitalization frequency in patients with refractory generalized myasthenia gravis, treatment responses vary among individuals, highlighting a need for personalized treatment strategies when selecting complement inhibitors.
