适用于症状性阻塞性多变性心肌病
Martin S Maron1, Ahmad Masri1, Michael E Nassif1
1From Lahey Hospital and Medical Center, Burlington (M.S.M.), and the Cardiovascular Division, Brigham and Women's Hospital, Harvard Medical School (B.C., S.D.S.), the Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School (J.L.J., G.D.L.), and the Baim Institute for Clinical Research (J.L.J.), Boston - all in Massachusetts; Oregon Health and Science University, Portland (A.M.); the University of Missouri Kansas City Healthcare Institute for Innovations in Quality and Saint Luke's Mid America Heart Institute, Kansas City (M.E.N., J.A.S.); Complejo Hospitalario Universitario de A Coruña, Instituto de Investigación Biomédica de A Coruña, Centro de Investigación Biomédica en Red de Enfermedades Cardiovaculares (CIBERCV)-Instituto de Salud Carlos III, A Coruña (R.B.-V.), and Hospital Universitario Puerta de Hierro de Majadahonda, Instituto de Investigación Sanitaria Puerta de Hierro-Segovia de Arana, CIBERCV, and Centro Nacional de Investigaciones Cardiovasculares, Madrid (P.G.-P.) - both in Spain; Chaim Sheba Medical Center, Ramat Gan and Tel Aviv University, Tel Aviv, Israel (M.A.); Hospital Companhia União Fabril Descobertas, Lisbon, Portugal (N.C.); Northwestern University Feinberg School of Medicine, Chicago (L.C.); the School of Cardiovascular and Metabolic Health, University of Glasgow, Glasgow (C.J.C., M.M.Y.L.), and Radcliffe Department of Medicine, University of Oxford, Oxford (H.W.) - both in the United Kingdom; Charité Campus Virchow-Klinikum, Berlin (H.-D.D.); Département de Cardiologie, Hôpital Européen Georges-Pompidou, Assistance Publique-Hôpitaux de Paris, Paris (A.A.H.); Beijing Anzhen Hospital, Capital Medical University, Beijing (C.-S.M.); the Department of Cardiology, Thoraxcenter, Erasmus Medical Center, Rotterdam (M.M.) and Zwolle (M.S.) - both in the Netherlands; Meyer Children's Hospital, Istituto di Ricovero e Cura a Carattere Scientifico, Florence, Italy (I.O.); National Institute of Cardiology, Warsaw, Poland (A.O.); University of Pennsylvania Perelman School of Medicine, Philadelphia (A.T.O.); the Section of Forensic Genetics, Department of Forensic Medicine, Faculty of Health and Medical Sciences, University of Copenhagen, and the Department of Cardiology, Copenhagen University Hospital Rigshospitalet, Copenhagen (J.T.-H.); the Department of Cardiology, Motol University Hospital, Prague, Czech Republic (J.V.); Cytokinetics, South San Francisco (D.L.J., S.B.H., S.K., F.I.M., L.M., A.W.), and the University of California, San Francisco, San Francisco (T.P.A.) - both in California.
在患有阻塞性多变性心肌病 (HCM) 的患者中,Aficamten显著改善了峰值氧气吸收. 这种心脏肌抑制剂在所有次要终点上表现出比安慰剂更高的疗效,提高了生活质量.
科学领域:
- 心脏病学 心脏病学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 阻塞性缩性心肌病 (HCM) 导致运动不耐受,原因是左心室外流通道阻塞导致心内压力升高.
- 选择性心脏肌抑制剂Aficamten针对超收缩性,以减少左心室外流通道梯度.
研究的目的:
- 评估aficamten在症状阻塞性HCM患者的疗效和安全性.
- 评估药物对运动能力和功能状态的影响.
主要方法:
- 一个第三阶段的双盲试验随机将282名患有症状阻塞性HCM的成年人分为aficamten或安慰剂,持续24周.
- 主要终点是通过心肺运动测试评估的峰值氧气吸收的变化.
- 二级终点包括生活质量的变化 (KCCQ-CSS),NYHA功能类和左心室外流通道梯度.
主要成果:
- 与安慰剂组的0.0毫升/千克/分钟相比,Aficamten显著增加了1.8毫升/千克/分钟的峰值氧气吸收 (P<0.001).
- 所有10个预规定的二级终点都显示了aficamten与安慰剂的显著改善.
- 在aficamten和安慰剂组之间,不良事件发生率相似.
结论:
- 与安慰剂相比,在症状阻塞性HCM患者中,Aficamten治疗导致峰值氧气吸收的显著改善.
- 在阻塞性HCM中,Aficamten是一种有效的治疗选择,可以改善运动能力和功能状态.
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