Efficacy and safety of immunosuppressants and immunomodulators in juvenile myasthenia gravis: a systematic review and meta-analysis.

[OBJECTIVE] In the present meta-analysis, we aimed to explore the efficacy and safety of immunosuppressants and immunomodulators for the treatment of juvenile myasthenia gravis (JMG).

[METHODS] We conducted a systematic search for studies published between January 1st, 2000 and July 28th, 2025, in PubMed, Embase, Web of Science, and the Cochrane Library. Statistical analyses were performed using Stata (version 16.0). Cochran's Q test and the I statistic were used to assess the heterogeneity among the included studies. If significant heterogeneity existed (I ≥50% or P < 0.05), the random effects model was used; otherwise, the fixed effects model was used to calculate the pooled results.

[RESULTS] A total of 3029 articles were retrieved. This meta-analysis included 9 cohort and case-control studies, 11 case series, 3 single-arm studies, and 1 randomized controlled trial, focusing on tacrolimus, glucocorticoids, monoclonal antibodies, and intravenous immunoglobulin. Regarding tacrolimus, 9 studies involving 310 patients assessed the efficacy of tacrolimus for treating JMG. The results showed a significant reduction in both the Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Activities of Daily Living (MG-ADL) scores. Moreover, tacrolimus treatment allowed for a reduction in steroid dosage, with a response rate of 0.862 (95% CI: 0.716-0.967). For monoclonal antibodies, 6 studies with 67 patients analyzed the efficacy for JMG. The response rate of monoclonal antibodies was 0.993 (95% CI: 0.935-1.000). Descriptive analyses were conducted for glucocorticoids and IVIG. Besides, 5 studies with 348 patients assessed the efficacy of glucocorticoids for JMG. Included studies showed that the efficacy rate of glucocorticoid monotherapy for isolated ocular myasthenia gravis (OMG) was higher than that for patients with both OMG and generalized myasthenia gravis (GMG). Finally, regarding the use of IVIG, 4 studies reported efficacy for JMG. These investigations reported a response rate ranging from 47.06% to 94.3% for IVIG therapy.

[CONCLUSIONS] In summary, this was the first comprehensive meta-analysis of immunosuppressants and immunomodulators in JMG. However, most included studies were single-center retrospective observational studies. Future prospective multicenter studies are needed to further investigate the efficacy and safety of immunosuppressants and immunomodulators in JMG.