Abstract：
BACKGROUND: 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.
RESULTS: 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/m2.
CONCLUSIONS: 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.
RESULTS: 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/m2.
CONCLUSIONS: 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.
摘要：
背景：梗阻性肥厚型心肌病患者的症状负担增加。根据2项随机对照试验，Mavacamten最近被批准用于治疗阻塞性肥厚型心肌病。然而,它在现实条件下和不同人群中的使用研究不足。
结果：这是一项前瞻性观察性队列研究，对象是在2022年7月7日至2024年1月6日期间在约翰霍普金斯HCM中心看到的患者，并为阻塞性肥厚型心肌病开了mavacamten处方。对患者进行纵向随访，根据风险评估和缓解策略计划的要求，进行连续超声心动图和临床评估。66例患者接受了mavacampen（平均年龄59岁，47%男性,29%非白色[黑色，西班牙裔/拉丁裔,亚洲人,夏威夷原住民或太平洋岛民]，47%肥胖)。治疗前,所有患者均有纽约心脏协会II级(51.5%)或III级(48.5%)心力衰竭症状.最初最大峰值左心室流出道梯度为107±46mmHg。中位治疗时间为9个月。对于≥6个月后服用mavacampen的患者（n=43），72％的患者的症状改善了≥1个纽约心脏协会班级，左心室流出道梯度峰值下降80±46mmHg，在79.1%的患者中消除血流动力学显著的左心室流出道梗阻。3例患者因左心室射血分数下降<50%而暂时停药。没有药物相关的不良事件。白人和非白人患者的有效性和安全性相似，但体重指数≥35kg/m2的患者症状缓解减弱.
结论：Mavacamten在真实世界条件下用于有症状的梗阻性肥厚型心肌病患者的种族多样性人群中是有效和安全的。在使用mavacamten时，合并肥胖的患者不太可能出现症状改善。
结果：这是一项前瞻性观察性队列研究，对象是在2022年7月7日至2024年1月6日期间在约翰霍普金斯HCM中心看到的患者，并为阻塞性肥厚型心肌病开了mavacamten处方。对患者进行纵向随访，根据风险评估和缓解策略计划的要求，进行连续超声心动图和临床评估。66例患者接受了mavacampen（平均年龄59岁，47%男性,29%非白色[黑色，西班牙裔/拉丁裔,亚洲人,夏威夷原住民或太平洋岛民]，47%肥胖)。治疗前,所有患者均有纽约心脏协会II级(51.5%)或III级(48.5%)心力衰竭症状.最初最大峰值左心室流出道梯度为107±46mmHg。中位治疗时间为9个月。对于≥6个月后服用mavacampen的患者（n=43），72％的患者的症状改善了≥1个纽约心脏协会班级，左心室流出道梯度峰值下降80±46mmHg，在79.1%的患者中消除血流动力学显著的左心室流出道梗阻。3例患者因左心室射血分数下降<50%而暂时停药。没有药物相关的不良事件。白人和非白人患者的有效性和安全性相似，但体重指数≥35kg/m2的患者症状缓解减弱.
结论：Mavacamten在真实世界条件下用于有症状的梗阻性肥厚型心肌病患者的种族多样性人群中是有效和安全的。在使用mavacamten时，合并肥胖的患者不太可能出现症状改善。
