Lim Jaehyun, Cho Jae Yeong, Kwak Soongu, Park Chan-Soon, Park Jiesuck, Choi Hong-Mi, Cho Goo-Yeong, Choi Ga Hui, Kim Jihoon, Na Jin-Oh, Lee Sun Hwa, Jung Mi-Hyang, Kim Kye Hun, Jung Hae Ok, Lee Sang-Chol, Kim Hyung-Kwan
Division of Cardiology, Department of Internal Medicine, Seoul National University Hospital and Seoul National University College of Medicine, Seoul, Korea.
Department of Cardiovascular Medicine, Chonnam National University Hospital, Chonnam National University Medical School, Gwangju, Korea.
Korean Circ J. 2025 Apr;55(4):339-354. doi: 10.4070/kcj.2024.0443. Epub 2025 Mar 19.
Mavacamten has shown promise in obstructive hypertrophic cardiomyopathy (oHCM); however, real-world evidence is limited in Asians. We aimed to provide the first multicenter, real-world analysis of mavacamten use in Korea.
This prospective observational study included symptomatic oHCM patients treated at 7 tertiary hospitals in Korea. Changes in key parameters, including left ventricular outflow tract (LVOT) gradients, N-terminal pro-B-type natriuretic peptide (NT-proBNP), and New York Heart Association functional class (NYHA Fc), were serially assessed. Adverse events, including death, arrhythmia, and heart failure hospitalizations were documented.
Forty-six patients were followed for a median of 147 days (interquartile range 56-205). There was one sudden cardiac death and one non-cardiac death. Resting and Valsalva LVOT gradients significantly decreased from 58.4±46.4 mmHg and 92.6±46.8 mmHg to 17.5±21.3 mmHg and 25.7±27.1 mmHg, respectively (both p<0.001). NT-proBNP levels also decreased significantly, strongly correlating with LVOT gradient reductions. Among the 44 survivors, 25 (58.1%) had at least one NYHA Fc improvement. Moreover, left atrial volume and maximal left ventricular (LV) wall thickness significantly decreased. However, mean reduction in LV ejection fraction (LVEF) was -3.4% with one patient experiencing LVEF <50%. We observed 2 newly-detected atrial fibrillations and 2 ischemic strokes.
This first real-world experience of mavacamten in Korean patients with oHCM demonstrates its efficacy and safety, aligning with previous pivotal trials. With the implementation of national insurance coverage, mavacamten will be more accessible to broader oHCM population, offering a non-invasive and effective therapeutic option aside from invasive septal reduction therapies.
马伐卡坦在梗阻性肥厚型心肌病（oHCM）治疗中展现出前景；然而，亚洲人群的真实世界证据有限。我们旨在提供韩国首例关于马伐卡坦使用情况的多中心真实世界分析。
这项前瞻性观察性研究纳入了在韩国7家三级医院接受治疗的有症状oHCM患者。对包括左心室流出道（LVOT）压差、N末端B型利钠肽原（NT-proBNP）和纽约心脏协会心功能分级（NYHA Fc）在内的关键参数变化进行连续评估。记录不良事件，包括死亡、心律失常和因心力衰竭住院情况。
46例患者的中位随访时间为147天（四分位间距56 - 205天）。发生1例心源性猝死和1例非心源性死亡。静息和瓦尔萨尔瓦动作时的LVOT压差分别从58.4±46.4 mmHg和92.6±46.8 mmHg显著降至17.5±21.3 mmHg和25.7±27.1 mmHg（均p<0.001）。NT-proBNP水平也显著降低，与LVOT压差降低密切相关。在44名幸存者中，25名（58.1%）至少有一次NYHA Fc改善。此外，左心房容积和最大左心室（LV）壁厚度显著减小。然而，LV射血分数（LVEF）的平均降低幅度为-3.至50%。我们观察到2例新发心房颤动和2例缺血性卒中。
韩国oHCM患者使用马伐卡坦的首例真实世界经验证明了其有效性和安全性，与先前的关键试验结果一致。随着国家医保覆盖的实施，马伐卡坦将更广泛地惠及oHCM患者群体，为侵入性室间隔减容治疗之外提供一种非侵入性的有效治疗选择。
