The Efficacy and Safety of Tocilizumab in the Treatment of Refractory Ocular Myasthenia Gravis in Pediatric Patients.

IF 2.9 3区医学 Q2 CLINICAL NEUROLOGY

Muscle & Nerve Pub Date : 2026-10-12 DOI:10.1002/mus.70435

Qing-Qing Wang, Xiang-Qi Cao, Bi-Ying Zhang, Xiao-Xi Huang, Si-Jia Hao, Ting Chang, Zhe Ruan

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引用次数: 0

Abstract

Introduction/aims: Refractory pediatric ocular myasthenia gravis (OMG) remains difficult to manage. We evaluated tocilizumab's efficacy and safety in these patients.

Methods: In this single-center, retrospective analysis of prospectively collected registry data from Tangdu Hospital (China), pediatric refractory OMG patients received intravenous tocilizumab (8 mg/kg every 4 weeks) for 16 weeks as add-on therapy. Refractory disease required persistent symptoms despite adequate trials of ≥ 2 immunosuppressants, frequent relapses during glucocorticoid tapering, or intolerance of conventional oral immunosuppressants. The primary outcome was change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score from baseline to Weeks 4, 8, 12, and 16, analyzed using generalized estimating equations.

Results: All 10 patients (1 male, 9 females; median age 5.5 years, range 2-11) completed 16 weeks of follow-up. Seven (70%) were positive for antiacetylcholine receptor antibody (AChR-Ab). At Week 4, mean MG-ADL score decreased from 3.4 to 2.5 (incidence rate ratio[IRR], 0.74; 95% CI, 0.54-0.99; p = 0.048), with further reduction to 1.5 at week 16 (IRR, 0.44; 95% CI, 0.24-0.80; p = 0.007). Similarly, mean Quantitative MG (QMG) score dropped from 4.7 at baseline to 3.0 at Week 4 (IRR, 0.64; 95% CI, 0.41-0.99; p = 0.048) and further to 1.9 at Week 16 (IRR, 0.40; 95% CI, 0.24-0.69; p = 0.001). By Week 16, 50% of patients achieved minimal symptom expression, and 50% reduced prednisone requirements. Tocilizumab was well-tolerated with only one transient infusion-related dizziness.

Discussion: These preliminary findings suggest that tocilizumab may offer a safe and effective treatment option for children with refractory OMG.

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来源期刊

Muscle & Nerve 医学-临床神经学

CiteScore

6.40

自引率

5.90%

发文量

287

审稿时长

3-6 weeks

期刊介绍： Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.