3,4-二氨基吡啶治疗成人兰伯特-伊顿肌无力综合征：随机对照试验的荟萃分析

3,4-diaminopyridine treatment for Lambert-Eaton myasthenic syndrome in adults: a meta-analysis of randomized controlled trials.

作者信息

Zhang Na, Hong Daojun, Ouyang Taohui, Meng Wei, Huang Jingwei, Li Meihua, Hong Tao

机构信息

Department of Neurology, the First Affiliated Hospital of Nanchang University, Jiangxi, China.

Department of Neurosurgery, the First Affiliated Hospital of Nanchang University, No.17, Yongwai Street, Jiangxi Province, 330006, Nanchang, China.

出版信息

BMC Neurol. 2021 Sep 25;21(1):371. doi: 10.1186/s12883-021-02405-3.

DOI:10.1186/s12883-021-02405-3

PMID:34563155

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8464094/

Abstract

BACKGROUND

Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder of neuromuscular transmission. The objective was to examine the efficacy and safety of 3,4-diaminopyridine (3,4-DAP) in patients with LEMS.

METHODS

We searched several databases to identify relevant studies, including PubMed, EMBASE, Web of Science, MEDLINE, Cochrane Neuromuscular Disease Group Specialized Register and the Cochrane Central Register of Controlled Trials(CENTRAL). The primary outcome, quantitative myasthenia gravis (QMG) score and the secondary outcome, compound muscle action potentials (CMAP) amplitude were pooled by meta-analysis.

RESULTS

Six randomised controlled trials (RCTs) involving 115 patients with LEMS were included. QMG score showed a significant decrease (improvement) of 2.76 points (95 % CI, -4.08 to -1.45, p < 0.001) after treatment with 3, 4-DAP. Moreover, the overall mean CMAP amplitude improved significantly in LEMS patients with 3, 4-DAP treatment, compared with placebo treatment (mean difference 1.34 mV, 95 % CI, 0.98 to 1.70, p < 0.001). The overall assessment of all included trials showed a low risk of bias and low heterogeneity.

CONCLUSIONS

The pooled results of RCTs demonsrated with moderate to high evidence that 3,4-DAP has a significant effect on LEMS treatment, with improvements in muscle strength score and CMAP amplitude.

摘要

背景

兰伯特-伊顿肌无力综合征（LEMS）是一种罕见的神经肌肉传递自身免疫性疾病。目的是研究3,4-二氨基吡啶（3,4-DAP）治疗LEMS患者的疗效和安全性。

方法

我们检索了多个数据库以识别相关研究，包括PubMed、EMBASE、科学网、MEDLINE、Cochrane神经肌肉疾病组专业注册库和Cochrane对照试验中央注册库（CENTRAL）。主要结局指标重症肌无力定量（QMG）评分和次要结局指标复合肌肉动作电位（CMAP）波幅通过荟萃分析进行汇总。

结果

纳入了6项涉及115例LEMS患者的随机对照试验（RCT）。3,4-DAP治疗后QMG评分显著降低（改善）2.76分（95%CI，-4.08至-1.45，p<0.001）。此外，与安慰剂治疗相比，3,4-DAP治疗的LEMS患者总体平均CMAP波幅显著改善（平均差值1.34mV，95%CI，0.98至1.70，p<0.001）。所有纳入试验的总体评估显示偏倚风险低且异质性低。