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

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.