背景:营养不良性和非营养不良性肌强直的罕见性质限制了美西律作为潜在治疗方法的有效性的现有证据。为了解决这个差距,我们进行了系统评价和荟萃分析,以评估美西律对营养不良和非营养不良性肌强直患者的有效性和安全性.
方法:直到2023年3月,在各种电子数据库上进行了搜索,以比较强直性患者的美西律与安慰剂的随机临床试验(RCT)。进行了偏差风险评估,相关数据被手动提取到在线表格中。采用RevMan软件(版本5.4)进行分析。
结果:共有5项研究,由186名患者组成,纳入荟萃分析。我们的研究结果表明,美西律在改善僵硬评分方面比安慰剂更有效(SMD=-1.19,95%CI[-1.53,-0.85]),以及减少手握力肌强直(MD=-1.36s,95%CI[-1.83,-0.89])。美西律还显着改善了仅患有非营养不良性肌强直的患者的SF-36身体和精神成分评分。关于安全,与安慰剂相比,美西律未显著改变心电图参数,但与更大的胃肠道症状(GIT)相关(RR3.7,95%CI[1.79,7.64]).其他不良事件无显著差异。
结论:结果支持美西律对肌强直性患者有效且安全;然而,它与GIT症状的高风险相关。由于已发表的随机对照试验的稀缺性和GIT症状的普遍性,我们建议进一步精心设计的RCT测试各种药物组合,以减轻GIT症状.
BACKGROUND: The rare nature of dystrophic and non-dystrophic myotonia has limited the available evidence on the efficacy of mexiletine as a potential treatment. To address this gap, we conducted a systematic
review and meta-analysis to evaluate the effectiveness and safety of mexiletine for both dystrophic and non-dystrophic myotonic patients.
METHODS: The search was conducted on various electronic databases up to March 2023, for randomized clinical trials (RCTs) comparing mexiletine versus placebo in myotonic patients. A risk of bias assessment was carried out, and relevant data was extracted manually into an online sheet. RevMan software (version 5.4) was employed for analysis.
RESULTS: A total of five studies, comprising 186 patients, were included in the meta-analysis. Our findings showed that mexiletine was significantly more effective than placebo in improving stiffness score (SMD = - 1.19, 95% CI [- 1.53, - 0.85]), as well as in reducing hand grip myotonia (MD = - 1.36 s, 95% CI [- 1.83, - 0.89]). Mexiletine also significantly improved SF-36 Physical and Mental Component Score in patients with non-dystrophic myotonia only. Regarding safety, mexiletine did not significantly alter ECG parameters but was associated with greater gastrointestinal symptoms (GIT) compared to placebo (RR 3.7, 95% CI [1.79, 7.64]). Other adverse events showed no significant differences.
CONCLUSIONS: The results support that mexiletine is effective and safe in myotonic patients; however, it is associated with a higher risk of GIT symptoms. Due to the scarcity of published RCTs and the prevalence of GIT symptoms, we recommend further well-designed RCTs testing various drug combinations to reduce GIT symptoms.