Division of Cardiology, Department of Medicine, Columbia University Irving Medical Center, New York, NY.
Division of Cardiology, Department of Medicine, Columbia University Irving Medical Center, New York, NY.
J Card Fail. 2024 Sep;30(9):1136-1153. doi: 10.1016/j.cardfail.2024.04.024. Epub 2024 May 20.
For many years, treatment of hypertrophic cardiomyopathy (HCM) has focused on non-disease-specific therapies. Cardiac myosin modulators (ie, mavacamten and aficamten) reduce the pathologic actin-myosin interactions that are characteristic of HCM, leading to improved cardiac energetics and reduction in hypercontractility. Several recently published randomized clinical trials have demonstrated that mavacamten improves exercise capacity, left ventricular outflow tract obstruction and symptoms in patients with obstructive HCM and may delay the need for septal-reduction therapy. Long-term data in real-world populations will be needed to fully assess the safety and efficacy of mavacamten. Importantly, HCM is a complex and heterogeneous disease, and not all patients will respond to mavacamten; therefore, careful patient selection and shared decision making will be necessary in guiding the use of mavacamten in obstructive HCM.
多年来,肥厚型心肌病(HCM)的治疗一直集中在非疾病特异性治疗上。心肌球蛋白调节剂(即 mavacamten 和 aficamten)可减少 HCM 特有的病理性肌球蛋白-肌动蛋白相互作用,从而改善心脏能量代谢并减少过度收缩。最近发表的几项随机临床试验表明,mavacamten 可改善梗阻性 HCM 患者的运动能力、左心室流出道梗阻和症状,并可能延迟需要间隔减少治疗。需要来自真实世界人群的长期数据来全面评估 mavacamten 的安全性和疗效。重要的是,HCM 是一种复杂且异质性的疾病,并非所有患者对 mavacamten 都有反应;因此,在指导梗阻性 HCM 中使用 mavacamten 时,需要仔细选择患者并进行共同决策。