Desai Milind Y, Nissen Steve E, Abraham Theodore, Olivotto Iacopo, Garcia-Pavia Pablo, Lopes Renato D, Verheyen Nicolas, Wever-Pinzon Omar, Wolski Kathy, Jaber Wael, Mitchell Lisa, Davey Deborah, Myers Jonathan, Rano Thomas, Bhatia Vandana, Zhong Yue, Carter-Bonanza Suzanne, Florea Victoria, Aronson Ron, Owens Anjali T
Hypertrophic Cardiomyopathy Center, Cleveland, Ohio, USA; Cleveland Clinic Coordinating Center for Clinical Research, Heart, Vascular and Thoracic Institute, Cleveland Clinic, Cleveland, Ohio, USA.
Department of Cardiology, University of San Francisco, San Francisco, California, USA.
JACC Heart Fail. 2025 Feb;13(2):358-370. doi: 10.1016/j.jchf.2024.11.013.
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 (pVO) 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 pVO 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).
对于有症状的非梗阻性肥厚型心肌病（nHCM）患者，目前尚无获批的治疗方法。作者描述了ODYSSEY-HCM（玛伐卡坦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，评估玛伐卡坦对有症状的成年nHCM患者的疗效。两个主要终点是从基线到第48周的变化：1）堪萨斯城心肌病问卷23项临床总结评分；2）心肺运动试验中的峰值耗氧量（pVO）。剂量滴定基于盲态核心实验室评估进行。在1088名筛查的患者中，580名被随机分组（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且有症状（70%为纽约心脏协会功能分级II级，30%为III级），堪萨斯城心肌病问卷23项临床总结评分平均为58±20，77%的患者正在服用β受体阻滞剂。平均左心室射血分数和pVO分别为66%±4%和18±6 mL/kg/min。ODYSSEY-HCM将报告玛伐卡坦是否能改善有症状nHCM患者的患者报告健康状况和运动能力。（玛伐卡坦治疗非梗阻性肥厚型心肌病的研究（ODYSSEY-HCM）；NCT05582395）
