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

IF 3.2 3区医学 Q2 CLINICAL NEUROLOGY

Pediatric neurology Pub Date : 2025-02-20 DOI:10.1016/j.pediatrneurol.2025.02.007

Aidou Jiang MS, Qiaozhi Hu PhD, Zhidan Wang BS, Fengbo Wu PhD

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引用次数: 0

Abstract

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, I² = 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.

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他克莫司治疗青少年重症肌无力的疗效和安全性：单臂荟萃分析

背景：许多指南推荐在说明书外使用他克莫司（TAC）治疗成人重症肌无力（MG）。本研究旨在评估TAC是否对儿童幼年MG （JMG）患者有益。方法采用关键词“重症肌无力”、“TAC”、“青少年”及其同义词进行系统的文献检索。检索了PubMed、Embase、Web of Science、Cochrane Library和中文数据库，检索了2024年4月1日前发表的文章。两名审稿人独立确定并提取相关的回顾性/前瞻性比较研究或随机对照试验，并评估每项研究的偏倚风险。符合条件的研究随后被纳入荟萃分析，使用固定效应和随机效应模型评估TAC治疗JMG的临床结果。在最初确定的203篇文章中，有9篇被纳入meta分析。这些研究包括313名被诊断为JMG的儿童，年龄从0岁到13.5岁不等。在纳入的研究中，两项是比较试验，而其余七项采用单组前测后测设计。两项研究被认为是高质量的，七项是中等质量的。经计算，JMG患者对TAC治疗的总有效率为3.92(95%可信区间：2.06 ~ 7.45,I2 = 71%, P <；0.001)。本荟萃分析发现，TAC可改善JMG患者的症状和mg相关评分，且不良反应最小。这些发现强调了TAC治疗JMG的重要性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pediatric neurology 医学-临床神经学

CiteScore

4.80

自引率

2.60%

发文量

176

审稿时长

78 days

期刊介绍： Pediatric Neurology publishes timely peer-reviewed clinical and research articles covering all aspects of the developing nervous system. Pediatric Neurology features up-to-the-minute publication of the latest advances in the diagnosis, management, and treatment of pediatric neurologic disorders. The journal''s editor, E. Steve Roach, in conjunction with the team of Associate Editors, heads an internationally recognized editorial board, ensuring the most authoritative and extensive coverage of the field. Among the topics covered are: epilepsy, mitochondrial diseases, congenital malformations, chromosomopathies, peripheral neuropathies, perinatal and childhood stroke, cerebral palsy, as well as other diseases affecting the developing nervous system.