Division of Cardiology, Department of Medicine Johns Hopkins University Baltimore MD.
J Am Heart Assoc. 2024 Aug 6;13(15):e034069. doi: 10.1161/JAHA.123.034069. Epub 2024 Jul 31.
Patients with obstructive hypertrophic cardiomyopathy have increased symptomatic burden. Mavacamten was recently approved for treatment of obstructive hypertrophic cardiomyopathy based on 2 randomized controlled trials. However, its use under real-world conditions and in diverse populations is under-studied.
This was a prospective observational cohort study of patients seen at the Johns Hopkins HCM center and prescribed mavacamten for obstructive hypertrophic cardiomyopathy between July 7, 2022 and January 6, 2024. Patients were followed longitudinally, with serial echocardiography and clinical evaluation as mandated by the risk evaluation and mitigation strategy program. Sixty-six patients received mavacamten (mean age 59 years, 47% male, 29% non-White [Black, Hispanic/Latino, Asian, Native Hawaiian or Pacific Islander], 47% obese). Before treatment, all patients had New York Heart Association class II (51.5%) or III (48.5%) heart failure symptoms. Initial maximum peak left ventricular outflow tract gradient was 107±46 mm Hg. Median treatment duration was 9 months. For patients on mavacamten after ≥6 months (n=43), symptoms improved by ≥1 New York Heart Association class in 72% of patients, and peak left ventricular outflow tract gradient decreased by 80±46 mm Hg, eliminating hemodynamically significant left ventricular outflow tract obstruction in 79.1% of patients. Mavacamten was temporarily discontinued in 3 patients due to left ventricular ejection fraction decrease <50%. There were no medication-related adverse events. Effectiveness and safety were similar between White and non-White patients, but symptomatic relief was attenuated in patients with body-mass index ≥35 kg/m.
Mavacamten was effective and safe when used under real-world conditions in a racially diverse population of symptomatic patients with obstructive hypertrophic cardiomyopathy. Patients with comorbid obesity were less likely to experience symptomatic improvement while on mavacamten.
梗阻性肥厚型心肌病患者的症状负担增加。Mavacamten 最近基于两项随机对照试验被批准用于治疗梗阻性肥厚型心肌病。然而，其在真实世界环境中和不同人群中的使用情况仍研究不足。
这是一项前瞻性观察性队列研究，纳入了 2022 年 7 月 7 日至 2024 年 1 月 6 日期间在约翰霍普金斯 HCM 中心就诊并接受 Mavacamten 治疗梗阻性肥厚型心肌病的患者。患者接受了纵向随访，根据风险评估和缓解策略计划的要求进行了连续的超声心动图和临床评估。66 名患者接受了 Mavacamten 治疗（平均年龄 59 岁，47%为男性，29%为非白人[黑种人、西班牙裔/拉丁裔、亚洲人、夏威夷原住民或太平洋岛民]，47%为肥胖）。治疗前，所有患者均有纽约心脏协会心功能 II 级（51.5%）或 III 级（48.5%）心力衰竭症状。初始最大峰值左心室流出道梯度为 107±46mmHg。中位治疗持续时间为 9 个月。在至少接受 6 个月 Mavacamten 治疗的患者（n=43）中，72%的患者症状至少改善了 1 个纽约心脏协会心功能分级，峰值左心室流出道梯度降低了 80±46mmHg，79.1%的患者消除了血流动力学显著的左心室流出道梗阻。由于左心室射血分数降低至<50%，有 3 名患者暂时停用了 Mavacamten。无药物相关不良事件。白人患者和非白人患者的疗效和安全性相似，但体重指数≥35kg/m 的患者的症状缓解程度减弱。
Mavacamten 在种族多样化的梗阻性肥厚型心肌病有症状患者的真实世界环境中使用时是有效且安全的。合并肥胖症的患者在接受 Mavacamten 治疗时不太可能出现症状改善。
