A Systematic Review and Meta-analysis of Efficacy and Safety of Mavacamten for the Treatment of Hypertrophic Cardiomyopathy.

IF 16.4 1区化学 Q1 CHEMISTRY, MULTIDISCIPLINARY

Accounts of Chemical Research Pub Date : 2024-10-23 eCollection Date: 2024-10-01 DOI:10.31083/j.rcm2510375

Li Zheng, Xiaotong Gu, Yumiao Chen, Deping Liu

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引用次数: 0

Abstract

Background: Hypertrophic cardiomyopathy (HCM) is a common hereditary cardiomyopathy. Mavacamten, a first-in-class cardiac myosin inhibitor, is considered to be a specific drug for the treatment of HCM. This meta-analysis aimed to assess the efficacy and safety of mavacamten in patients with HCM.

Methods: PubMed, Cochrane Library, Embase and Clinical Trials.gov databases were searched from inception to February 6, 2024 for randomized controlled trials (RCTs) which compared the efficacy and safety between mavacamten and placebo in treating HCM.

Results: Six RCTs involving 732 patients were included in this meta-analysis. This meta-analysis showed that mavacamten improved the New York Heart Association (NYHA) function class [risk ratios (RR): 2.21, 95% confidence interval (CI): 1.48 to 3.30, p = 0.00001], Clinical Summary Score of the Kansas City Cardiomyopathy Questionnaire (KCCQ-CSS) scores [mean difference (MD): 9.33, 95% CI: 7.09 to 11.57, p < 0.00001] and composite functional end point (RR: 1.86, 95% CI: 1.25 to 2.78, p = 0.002). Meanwhile, mavacamten decreased N-terminal pro-B-type natriuretic peptide (NT-proBNP) (MD: -492.28, 95% CI: -611.55 to -373.02, p < 0.00001), cardiac troponin I (cTnI) (MD: -14.58, 95% CI: -26.98 to -2.17, p = 0.02) and Valsalva left ventricular outflow tract (LVOT) gradient (MD: -57.96, 95% CI: -82.15 to -33.78, p < 0.00001). The results for the incidence of ≥1 total emergent adverse event (TEAE) and ≥1 serious adverse event (SAE) showed that there was no significant difference between both groups (RR: 1.9, 95% CI: 0.97 to 1.24, p = 0.16) (RR: 1.06, 95% CI: 0.46 to 2.44, p = 0.90).

Conclusions: Mavacamten has great efficacy for the treatment of HCM. Meanwhile, mavacamten did not increase the incidence of adverse events or serious adverse events.

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马伐康汀治疗肥厚型心肌病的疗效和安全性的系统回顾和荟萃分析。

背景：肥厚型心肌病（HCM）是一种常见的遗传性心肌病：肥厚型心肌病（HCM）是一种常见的遗传性心肌病。马伐康坦是第一类心脏肌球蛋白抑制剂，被认为是治疗 HCM 的特效药。这项荟萃分析旨在评估马伐康坦对 HCM 患者的疗效和安全性：方法：在PubMed、Cochrane Library、Embase和Clinical Trials.gov数据库中检索了从开始到2024年2月6日比较马伐康坦和安慰剂治疗HCM疗效和安全性的随机对照试验（RCT）：本荟萃分析共纳入了六项随机对照试验，涉及 732 名患者。荟萃分析表明，马伐康坦可改善纽约心脏协会（NYHA）功能分级[风险比（RR）：2.21，95% 置信区间（CI）：1.48 至 3.30，P = 0.00001]、堪萨斯城心肌病问卷临床总结评分（KCCQ-CSS）得分[平均差（MD）：9.33，95% CI：7.09 至 11.57，p < 0.00001]和复合功能终点（RR：1.86，95% CI：1.25 至 2.78，p = 0.002）。同时，马伐康坦可降低 N 端前 B 型钠尿肽（NT-proBNP）（MD：-492.28，95% CI：-611.55 至 -373.02，P <0.00001）、心肌肌钙蛋白 I（cTnI）（MD：-14.58，95% CI：-26.98 至 -2.17，p = 0.02）和 Valsalva 左心室流出道（LVOT）梯度（MD：-57.96，95% CI：-82.15 至 -33.78，p < 0.00001）。≥1次总紧急不良事件（TEAE）和≥1次严重不良事件（SAE）的发生率结果显示，两组间无显著差异（RR：1.9，95% CI：0.97至1.24，P=0.16）（RR：1.06，95% CI：0.46至2.44，P=0.90）：结论：马伐康定对治疗 HCM 有显著疗效。结论：马伐康定对治疗 HCM 有很好的疗效，同时马伐康定不会增加不良事件或严重不良事件的发生率。

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

Accounts of Chemical Research 化学-化学综合

CiteScore

31.40

自引率

1.10%

发文量

312

审稿时长

2 months

期刊介绍： Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.