Real-World Observations in Patients With Obstructive Hypertrophic Cardiomyopathy Treated With Mavacamten: Evidence of Favorable Disease Modification.

IF 5.3 1区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Journal of the American Heart Association Pub Date : 2025-10-07 Epub Date: 2025-10-08 DOI:10.1161/JAHA.125.044537
Milind Y Desai, Andrew Gaballa, Yuichiro Okushi, Mohammed Abusafia, Shada Jadam, Katy Rutkowski, Susan Ospina, Sanaa Sultana, Maran Thamilarasan, Craig Asher, Zoran B Popovic, Bo Xu
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引用次数: 0

Abstract

Background: Mavacamten is commercially approved for use in symptomatic patients with obstructive hypertrophic cardiomyopathy. This study evaluated its real-world impact on symptoms, echocardiographic changes, and the need for septal reduction therapy in patients with obstructive hypertrophic cardiomyopathy treated at a multihospital referral center.

Methods: We included 244 patients with obstructive hypertrophic cardiomyopathy (mean age, 64 years; 57% women, 21% with atrial fibrillation, 51% New York Heart Association class III, 82% on β-blockers) treated with mavacamten for ≥3 months (171 for ≥12 months). Serial clinical and echocardiographic assessments were performed.

Results: New York Heart Association class distribution improved over time: at 3 months 21% class I, 61% class II, and 18% class III; at 12 months 36% class I, 57% class II, and 8% class III. After a mean of 1.02 years, significant improvements were observed in resting and post-Valsalva left ventricular outflow tract gradients (-31±33 and -57±46 mm Hg), interventricular septal thickness (-1.7±4 mm), left ventricular mass index (-9.5±32 g/m2), left atrial volume index (-12±13 mL/m2), E/e' (-5.5±6.4), left ventricular global longitudinal strain (1.04%±3.1%), and various left atrial and right ventricular strain measures (all P<0.05). Temporary interruption occurred in 8 patients (3.2%), and 3 (1.2%) discontinued mavacamten permanently. There were 2 noncardiac deaths, 7 heart failure admissions, 14 cases of new-onset atrial fibrillation, and 1 patient required septal reduction therapy. At the last visit, most patients were on 5 mg (49%) or 10 mg (26%) mavacamten.

Conclusions: In this real-world cohort, mavacamten treatment was associated with sustained symptomatic and echocardiographic improvements, suggesting a favorable disease-modifying impact with minimal need for septal reduction therapy.

使用马伐卡坦治疗的阻塞性肥厚性心肌病患者的真实世界观察:有利疾病改善的证据。
背景:马伐卡坦被商业批准用于症状性梗阻性肥厚性心肌病(oHCM)患者。本研究评估了其对在多家医院转诊中心治疗的oHCM患者的症状、超声心动图变化和室间隔缩小治疗(SRT)需求的实际影响。方法:我们纳入了244例oHCM患者(平均年龄64岁,57%为女性,21%为房颤,51%为纽约心脏协会[NYHA] III类,82%为β受体阻滞剂),使用马伐卡坦治疗≥3个月(171例≥12个月)。进行了一系列临床和超声心动图评估。结果:NYHA分类分布随着时间的推移而改善:3个月时,I类21%,II类61%,III类18%;在12个月时,36%为一类,57%为二类,8%为三类。平均1.02年后,观察到静息和valsalva后左室流出道(LVOT)梯度(-31±33和-57±46 mm Hg)、室间隔厚度(-1.7±4 mm)、左室质量指数(-9.5±32 g/m2)、左房容积指数(-12±13 ml/m2)、E/ E′(-5.5±6.4)、左室整体纵向应变(1.04±3.1%)以及左房和右室各项应变测量均有显著改善(均p)。在这个现实世界的队列中,马伐卡坦治疗与持续的症状和超声心动图改善相关,表明在最小的SRT需求下具有有利的疾病改善作用。
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来源期刊
Journal of the American Heart Association
Journal of the American Heart Association CARDIAC & CARDIOVASCULAR SYSTEMS-
CiteScore
9.40
自引率
1.90%
发文量
1749
审稿时长
12 weeks
期刊介绍: As an Open Access journal, JAHA - Journal of the American Heart Association is rapidly and freely available, accelerating the translation of strong science into effective practice. JAHA is an authoritative, peer-reviewed Open Access journal focusing on cardiovascular and cerebrovascular disease. JAHA provides a global forum for basic and clinical research and timely reviews on cardiovascular disease and stroke. As an Open Access journal, its content is free on publication to read, download, and share, accelerating the translation of strong science into effective practice.
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