在阻塞性多变性心肌病中使用Aficamten或Metoprolol单疗法
Pablo Garcia-Pavia1,2, Martin S Maron3, Ahmad Masri4
1Hospital Universitario Puerta de Hierro de Majadahonda, Instituto Investigación Sanitaria Puerta de Hierro-Segovia de Arana, Centro de Investigación Biomédica en Red Enfermedades Cardiovaculares (CIBERCV), Madrid.
The New England journal of medicine
|September 1, 2025
概括
与甲醇相比,Aficamten单一治疗显著改善了阻塞性多动性肌肉病 (HCM) 患者的运动能力和症状. 这种心脏肌肉酶抑制剂为HCM提供了优越的治疗选择.
科学领域:
- 心脏病学
- 药理学
- 临床试验
背景情况:
- 阻塞性缩性心肌病 (HCM) 经常使用β抑制剂治疗,但其疗效的证据有限.
- 一种心脏肌肉酶抑制剂Aficamten在与常规药物一起使用时,有望改善HCM症状和运动能力.
研究的目的:
- 在有症状阻塞性HCM的患者中,比较aficamten单一治疗与metoprolol单一治疗的疗效.
- 评估aficamten对运动能力,功能状态和血液动力学参数的影响.
主要方法:
- 一项国际双盲双模拟试验随机选择了175名患有症状阻塞性HCM的成年人接受阿菲卡门特或美托普罗罗尔.
- 主要终点是24周时最大氧气摄入量的变化. 二级终点包括NYHA功能类,KCCQ-CSS和血液动力学的改善.
主要成果:
- 与美托普罗罗尔相比,阿菲卡门单一治疗显著增加了氧气吸收的峰值 (2. 3毫升/ 千克/ 分钟的差异,P< 0. 001).
- 接受aficamten治疗的患者在NYHA类,KCCQ-CSS,左心室外流通路梯度,NT-proBNP和左心室体积指数中表现出更大的改善.
- 在aficamten和metoprolol组之间,不良事件是相似的.
结论:
- 在症状阻塞性HCM中,Aficamten单一治疗优于甲醇单一治疗以改善运动能力和血液动力学.
- 在减少HCM患者的症状和改善生活质量方面,Aficamten显示出显著的临床益处.
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