Efficacy and Safety of Tacrolimus Therapy in Patients With Juvenile Myasthenia Gravis: A Single-Arm Meta-Analysis.

BACKGROUND

Numerous guidelines recommend off-label use of tacrolimus (TAC) to treat myasthenia gravis (MG) in adults. This study aimed to evaluate whether TAC is beneficial in pediatric patients with juvenile MG (JMG).

METHODS

We conducted a systematic literature search using the keywords "Myasthenia Gravis," "TAC," "juveniles," and their synonyms. PubMed, Embase, Web of Science, Cochrane Library, and Chinese databases were searched for articles published until April 1, 2024. Two reviewers independently identified and extracted relevant retrospective/prospective comparison studies or randomized controlled trials and assessed the risk of bias for each study. Eligible studies were subsequently included in a meta-analysis that evaluated the clinical outcomes of TAC treatment for JMG using fixed- and random-effects models.

RESULTS

Of the 203 articles initially identified, nine were included in the meta-analysis. These studies included 313 children diagnosed with JMG, with ages ranging from 0 to 13.5 years. Among the included studies, two were comparison trials, whereas the remaining seven employed a single-group pretest-post-test design. Two studies were deemed to be of high quality, and seven were of moderate quality. The pooled overall response rate of the definite responder rate to TAC treatment in JMG was calculated as 3.92 (95% confidence interval: 2.06 to 7.45, I = 71%, P < 0.001).

CONCLUSIONS

This meta-analysis found that TAC improved symptoms and MG-related scores in patients with JMG with minimal adverse effects. These findings underscore the importance of TAC therapy for the treatment of JMG.