马伐康汀治疗有症状的非梗阻性肥厚型心肌病

IF 10.3 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

JACC. Heart failure Pub Date : 2025-02-01 DOI:10.1016/j.jchf.2024.11.013

Milind Y. Desai MD, MBA , Steve E. Nissen MD , Theodore Abraham MD , Iacopo Olivotto MD , Pablo Garcia-Pavia MD, PhD , Renato D. Lopes MD, PhD , Nicolas Verheyen MD , Omar Wever-Pinzon MD , Kathy Wolski MPH , Wael Jaber MD , Lisa Mitchell RN , Deborah Davey RN , Jonathan Myers PhD , Thomas Rano PhD , Vandana Bhatia PhD , Yue Zhong PhD , Suzanne Carter-Bonanza RN , Victoria Florea MD , Ron Aronson MD , Anjali T. Owens MD

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引用次数: 0

摘要

目前尚无针对症状性非梗阻性肥厚型心肌病（nHCM）患者的获批疗法。作者描述了 ODYSSEY-HCM（Mavacamten 治疗非梗阻性肥厚型心肌病的研究）的基线特征，这是一项在全球 201 个地点进行的 3 期随机、双盲、安慰剂对照试验，评估了 mavacamten 在有症状的成年 nHCM 患者中的应用。2 个主要终点是以下指标从基线到第 48 周的变化：1）堪萨斯城心肌病问卷 23 项临床总分；2）心肺运动测试的峰值耗氧量 (pVO2)。剂量滴定根据盲法核心实验室评估结果进行。在 1088 名接受筛查的患者中，580 人被随机选中（平均年龄 56 ± 15 岁，46% 为女性，43% 有家族病史）。所有患者均为非梗阻性无症状患者（70%为NYHA功能分级II级，30%为III级），堪萨斯城心肌病问卷23项临床总分平均为58±20分，77%的患者正在服用β-受体阻滞剂。平均左室射血分数和 pVO2 分别为 66% ± 4% 和 18 ± 6 mL/kg/min。ODYSSEY-HCM 将报告马伐康坦是否能改善无症状 nHCM 患者的患者报告健康状况和运动能力。(马伐康坦治疗非梗阻性肥厚型心肌病的研究（ODYSSEY-HCM）；NCT05582395）

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Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy

There are no approved therapies for patients with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). The authors describe the baseline characteristics of ODYSSEY-HCM (A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy), a phase 3, randomized, double-blind, placebo-controlled trial conducted worldwide at 201 sites evaluating mavacamten in symptomatic adult patients with nHCM. The 2 primary endpoints are the changes from baseline to week 48 in: 1) Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score; and 2) peak oxygen consumption (pVO₂) on cardiopulmonary exercise testing. Dose titrations are made on blinded core laboratory assessments. Of 1,088 patients screened, 580 are randomized (mean age 56 ± 15 years, 46% women, 43% with family histories). All patients are nonobstructive and symptomatic (70% in NYHA functional class II and 30% class III), with a mean Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score of 58 ± 20, and 77% are on beta-blockers. The mean left ventricular ejection fraction and pVO₂ are 66% ± 4% and 18 ± 6 mL/kg/min, respectively. ODYSSEY-HCM will report if mavacamten improves patient-reported health status and exercise capacity in patients with symptomatic nHCM. (A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy (ODYSSEY-HCM); NCT05582395)

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来源期刊

JACC. Heart failure CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

21.20

自引率

2.30%

发文量

164

期刊介绍： JACC: Heart Failure publishes crucial findings on the pathophysiology, diagnosis, treatment, and care of heart failure patients. The goal is to enhance understanding through timely scientific communication on disease, clinical trials, outcomes, and therapeutic advances. The Journal fosters interdisciplinary connections with neuroscience, pulmonary medicine, nephrology, electrophysiology, and surgery related to heart failure. It also covers articles on pharmacogenetics, biomarkers, and metabolomics.