玛伐卡坦与常规药物治疗肥厚型心肌病的疗效比较
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四川省科技厅自然科学基金项目(2022NSFSC0711)


Efficacy comparison of mavacamten and conventional drugs in the treatment of hypertrophic cardiomyopathy
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    摘要:

    目的 探讨新型药物玛伐卡坦(mavacamten)与常规药物治疗肥厚型心肌病(HCM)的疗效比较。方法 在PubMed、Cochrane Library、Embase和Web of science数据库以“Hypertrophic cardiomyopathy”、“Mavacamten”、“β-blockers”、“Calcium channel blockers”、“Angiotensin II receptor blocker”等检索词搜索mavacamten或常规药物治疗HCM 患者的随机对照试验(RCT)。主要结局指标为静息状态下左心室流出道(LVOT)梯度的变化。次要结局指标是左心室射血分数(LVEF)、峰值耗氧量(pVO2)和N末端B型钠尿肽前体(NT-proBNP)的变化。采用合并加权均数差(WMD)及其95%置信区间(CI)进行传统meta分析和间接比较meta分析。结果 共纳入11项随机对照试验,共933例患者,其中3项研究评估mavacamten,4项研究评估血管紧张素Ⅱ受体拮抗剂(ARB) ,3项研究评估抗心绞痛药物,1项研究评估螺内酯。传统meta分析结果显示,与安慰剂相比,mavacamten显著降低静息LVOT梯度(WMD=-33.34,95%CI:-39.13~-27.55,P<0.001),LVEF(WMD=-3.37,95%CI:-4.83~-1.90,P<0.001)和NT-proBNP(WMD=-408.99,95%CI:-817.56~-0.42,P=0.05)。间接比较meta分析结果显示,与ARB 相比,mavacamten显著降低静息LVOT(WMD=-34.64,95%CI:-41.90~-27.38)和LVEF(WMD=〖CD*3/5〗5.12,95%CI:-8.16~-2.07)。此外,在降低静息LVOT梯度方面,mavacamten优于抗心绞痛药物(WMD=-35.54,95%CI:-44.00~-27.08)。结论 在HCM患者治疗中,与安慰剂和常规药物相比,mavacamten在降低静息LVOT梯度、NT-proBNP和改善异常升高的LVEF方面显示出优效性

    Abstract:

    Objective To compare the efficacy of a new drug mavacamten with conventional drugs for the treatment of hypertrophic cardiomyopathy(HCM). Methods We searched the PubMed, Cochrane Library, Embase, and Web of science databases for randomized controlled trials (RCT) on the mavacamten or conventional treatment of HCM patients using the search terms “Hypertrophic cardiomyopathy”, “Mavacamten”, “β-blockers”, “Calcium channel blockers”, “Angiotensin II receptor blocker”. The primary outcome was changes in resting left ventricular outflow tract(LVOT) gradient. Secondary outcomes were changes in left ventricular ejection fraction (LVEF), peak oxygen consumption (pVO2) and N-terminal pro-B-type natriuretic peptide (NT-proBNP). The pairwise and indirect-comparisons(network) meta-analysis used pooled weighted mean difference (WMD) and 95% confidence interval(CI). Results Eleven RCTs with 933 patients were enrolled in the pairwise meta-analysis, of which three trials were designed to evaluate the efficacy of mavacamten in patients with HCM, four trials to investigate the efficacy of ARB, three trials to evaluate the efficacy of antianginal drugs(ranolazine, trimetazidine, perhexiline), and one trial to investigate the efficacy of spironolactone. Traditional meta analysis results showed that compared with placebo, mavacamten significantly reduced the resting LVOT gradient(WMD=-33.34,95%CI:-39.13~-27.55,P<0.001), LVEF (WMD=-3.37,95%CI:-4.83~ -1.90, P<0.001) and NT-proBNP (WMD=-408.99, 95%CI:817.56~-0.42,P=0.05).In the indirect-comparisons meta-analysis, mavacamten significantly decrease LVOT (WMD=-34.64,95%CI:-41.90~ -27.38) and LVEF (WMD=-5.12,95%CI:-8.16~ -2.07), compared with ARB. In addition, mavacamten was superior to antianginal drugs in reducing resting LVOT gradient (WMD=-35.54,95%CI:-44.00~-27.08).Conclusion Compared with placebo or conventional drugs, mavacamten showed superiority in the reduction of resting LVOT gradient, NT-proBNP and abnormally elevated LVEF among patients with HCM

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  • 在线发布日期: 2025-07-21
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