{"title":"心肌肌球蛋白抑制剂马卡松和阿非卡坦治疗肥厚性心肌病的疗效：随机对照试验的系统评价和荟萃分析。","authors":"Ayesha Aman, Arfa Akram, Bisma Akram, Momina Maham, Masooma Zainab Bokhari, Aleena Akram, Sania Akram, Furqan Yaqub","doi":"10.1136/openhrt-2025-003215","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Unlike other suggested therapies, myosin inhibitors have been shown to change the course of hypertrophic cardiomyopathy by altering the contractile mechanics of cardiomyocytes. This meta-analysis sought to determine the efficacy of mavacamten and aficamten in hypertrophic cardiomyopathy.</p><p><strong>Methods: </strong>The online databases were searched from inception to July 2024, including the Cochrane Central Register of Controlled Trials (CENTRAL), PubMed, ClinicalTrials.gov. The meta-analytical data were pooled using risk ratios (RRs) with 95% CI, standard mean difference (SMD) and SE.</p><p><strong>Results: </strong>A total of 6 randomised controlled trials with 826 hypertrophic cardiomyopathy patients (mean age±SD up to 59.8±14.2 years in intervention vs 60.9±10.5 years in placebo) were included in our study. Of these, 443 received a cardiac myosin inhibitor and 383 received a placebo. The resting left ventricular outflow tract (LVOT) gradient between the two groups was considerably improved by cardiac myosin inhibitors (MD -57.27; 95% CI -63.05 to -51.49). Significant differences were also observed in the post-Valsalva LVOT gradient between the two groups (MD -55.86; 95% CI -65.55 to -46.18). Significantly decreased left ventricle ejection fraction (LVEF) was also seen (MD -4.74; 95% CI -7.22 to -2.26). The New York Health Association (NYHA) class improvement between the two groups also changed significantly (RR 2.21; 95% CI 1.75 to 2.80). Cardiac myosin inhibitors also caused significant improvement in the Kansas City Cardiomyopathy Questionnaire in a Clinical Summary Score between the two groups (MD 7.71; 95% CI 5.37 to 10.05) and significant reduction in the N-terminal pro-B-type natriuretic peptide (SMD -13.27; 95% CI -17.51 to -9.03) and the cardiac troponin I (SMD -11.90; 95% CI -15.07 to -8.72).</p><p><strong>Conclusion: </strong>According to our meta-analysis, cardiac myosin inhibitors significantly improve the resting and post-Valsalva LVOT gradient, reduce the LVEF and improve the NYHA class and cardiac biomarkers when compared with the placebo.</p><p><strong>Prospero registration number: </strong>CRD52024586161.</p>","PeriodicalId":19505,"journal":{"name":"Open Heart","volume":"12 1","pages":""},"PeriodicalIF":2.8000,"publicationDate":"2025-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11848667/pdf/","citationCount":"0","resultStr":"{\"title\":\"Efficacy of cardiac myosin inhibitors mavacamten and aficamten in hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomised controlled trials.\",\"authors\":\"Ayesha Aman, Arfa Akram, Bisma Akram, Momina Maham, Masooma Zainab Bokhari, Aleena Akram, Sania Akram, Furqan Yaqub\",\"doi\":\"10.1136/openhrt-2025-003215\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Unlike other suggested therapies, myosin inhibitors have been shown to change the course of hypertrophic cardiomyopathy by altering the contractile mechanics of cardiomyocytes. 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Significant differences were also observed in the post-Valsalva LVOT gradient between the two groups (MD -55.86; 95% CI -65.55 to -46.18). Significantly decreased left ventricle ejection fraction (LVEF) was also seen (MD -4.74; 95% CI -7.22 to -2.26). The New York Health Association (NYHA) class improvement between the two groups also changed significantly (RR 2.21; 95% CI 1.75 to 2.80). 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引用次数: 0
摘要
背景：与其他建议的治疗方法不同，肌球蛋白抑制剂已被证明通过改变心肌细胞的收缩机制来改变肥厚性心肌病的病程。本荟萃分析旨在确定马伐卡坦和阿非卡坦治疗肥厚性心肌病的疗效。方法：检索Cochrane Central Register of Controlled Trials （Central）、PubMed、ClinicalTrials.gov等自成立至2024年7月的在线数据库。meta分析数据采用95% CI的风险比（RRs）、标准平均差（SMD）和SE进行汇总。结果：我们的研究共纳入6项随机对照试验，共纳入826例肥厚性心肌病患者（干预组平均年龄±SD为59.8±14.2岁，安慰剂组平均年龄为60.9±10.5岁）。其中443人接受了心肌肌球蛋白抑制剂治疗，383人接受了安慰剂治疗。两组间静息左室流出道（LVOT）梯度显著改善心肌肌球蛋白抑制剂(MD -57.27；95% CI为-63.05 ~ -51.49)。两组valsalva后LVOT梯度也有显著差异(MD -55.86；95% CI -65.55 ~ -46.18)。左心室射血分数（LVEF）也显著降低(MD -4.74；95% CI -7.22至-2.26)。两组间纽约健康协会（NYHA）分级改善也有显著变化(RR 2.21；95% CI 1.75 ~ 2.80)。在堪萨斯城心肌病问卷的临床总结评分中，心肌肌球蛋白抑制剂也引起了两组之间的显著改善(MD 7.71；95% CI 5.37 ~ 10.05)和n端前b型利钠肽显著减少(SMD -13.27；95% CI -17.51 ~ -9.03)和心肌肌钙蛋白I (SMD -11.90；95% CI -15.07至-8.72)。结论：根据我们的荟萃分析，与安慰剂相比，心肌肌球蛋白抑制剂可显著改善静息和valsalva后LVOT梯度，降低LVEF，改善NYHA分类和心脏生物标志物。普洛斯彼罗注册号：CRD52024586161。
Efficacy of cardiac myosin inhibitors mavacamten and aficamten in hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomised controlled trials.
Background: Unlike other suggested therapies, myosin inhibitors have been shown to change the course of hypertrophic cardiomyopathy by altering the contractile mechanics of cardiomyocytes. This meta-analysis sought to determine the efficacy of mavacamten and aficamten in hypertrophic cardiomyopathy.
Methods: The online databases were searched from inception to July 2024, including the Cochrane Central Register of Controlled Trials (CENTRAL), PubMed, ClinicalTrials.gov. The meta-analytical data were pooled using risk ratios (RRs) with 95% CI, standard mean difference (SMD) and SE.
Results: A total of 6 randomised controlled trials with 826 hypertrophic cardiomyopathy patients (mean age±SD up to 59.8±14.2 years in intervention vs 60.9±10.5 years in placebo) were included in our study. Of these, 443 received a cardiac myosin inhibitor and 383 received a placebo. The resting left ventricular outflow tract (LVOT) gradient between the two groups was considerably improved by cardiac myosin inhibitors (MD -57.27; 95% CI -63.05 to -51.49). Significant differences were also observed in the post-Valsalva LVOT gradient between the two groups (MD -55.86; 95% CI -65.55 to -46.18). Significantly decreased left ventricle ejection fraction (LVEF) was also seen (MD -4.74; 95% CI -7.22 to -2.26). The New York Health Association (NYHA) class improvement between the two groups also changed significantly (RR 2.21; 95% CI 1.75 to 2.80). Cardiac myosin inhibitors also caused significant improvement in the Kansas City Cardiomyopathy Questionnaire in a Clinical Summary Score between the two groups (MD 7.71; 95% CI 5.37 to 10.05) and significant reduction in the N-terminal pro-B-type natriuretic peptide (SMD -13.27; 95% CI -17.51 to -9.03) and the cardiac troponin I (SMD -11.90; 95% CI -15.07 to -8.72).
Conclusion: According to our meta-analysis, cardiac myosin inhibitors significantly improve the resting and post-Valsalva LVOT gradient, reduce the LVEF and improve the NYHA class and cardiac biomarkers when compared with the placebo.
期刊介绍：
Open Heart is an online-only, open access cardiology journal that aims to be “open” in many ways: open access (free access for all readers), open peer review (unblinded peer review) and open data (data sharing is encouraged). The goal is to ensure maximum transparency and maximum impact on research progress and patient care. The journal is dedicated to publishing high quality, peer reviewed medical research in all disciplines and therapeutic areas of cardiovascular medicine. Research is published across all study phases and designs, from study protocols to phase I trials to meta-analyses, including small or specialist studies. Opinionated discussions on controversial topics are welcomed. Open Heart aims to operate a fast submission and review process with continuous publication online, to ensure timely, up-to-date research is available worldwide. The journal adheres to a rigorous and transparent peer review process, and all articles go through a statistical assessment to ensure robustness of the analyses. Open Heart is an official journal of the British Cardiovascular Society.
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