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肥厚型心肌病的新兴药理和结构治疗方法。

Emerging pharmacologic and structural therapies for hypertrophic cardiomyopathy.

机构信息

Department of Medicine, UCLA, 200 UCLA Medical Plaza Suite 420, Los Angeles, CA, 90095, USA.

Ahmanson-UCLA Cardiomyopathy Center, Division of Cardiology, Department of Medicine, UCLA, Los Angeles, CA, USA.

出版信息

Heart Fail Rev. 2017 Nov;22(6):879-888. doi: 10.1007/s10741-017-9648-x.

Abstract

Hypertrophic cardiomyopathy is the most common inherited heart disease. Although it was first described over 50 years ago, there has been little in the way of novel disease-specific therapeutic development for these patients. Current treatment practice largely aims at symptomatic control using old drugs made for other diseases and does little to modify the disease course. Septal reduction by surgical myectomy or percutaneous alcohol septal ablation are well-established treatments for pharmacologic-refractory left ventricular outflow tract obstruction in hypertrophic cardiomyopathy patients. In recent years, there has been a relative surge in the development of innovative therapeutics, which aim to target the complex molecular pathophysiology and resulting hemodynamics that underlie hypertrophic cardiomyopathy. Herein, we review the new and emerging therapeutics for hypertrophic cardiomyopathy, which include pharmacologic attenuation of sarcomeric calcium sensitivity, allosteric inhibition of cardiac myosin, myocardial metabolic modulation, and renin-angiotensin-aldosterone system inhibition, as well as structural intervention by percutaneous mitral valve plication and endocardial radiofrequency ablation of septal hypertrophy. In conclusion, while further development of these therapeutic strategies is ongoing, they each mark a significant and promising advancement in treatment for hypertrophic cardiomyopathy patients.

摘要

肥厚型心肌病是最常见的遗传性心脏病。尽管它在 50 多年前就被首次描述,但针对这些患者的新型疾病特异性治疗方法却很少。目前的治疗实践主要旨在通过使用针对其他疾病的旧药物来控制症状,而很少能改变疾病进程。对于肥厚型心肌病患者药物难治性左心室流出道梗阻,外科心肌切除术或经皮酒精室间隔消融术是成熟的治疗方法。近年来,针对肥厚型心肌病基础上复杂的分子病理生理学和血流动力学的创新治疗方法的发展相对迅速。本文综述了肥厚型心肌病的新型和新兴治疗方法,包括肌球蛋白钙敏感性的药物衰减、心肌球蛋白变构抑制、心肌代谢调节和肾素-血管紧张素-醛固酮系统抑制,以及经皮二尖瓣折叠术和心内膜射频消融术的结构性干预肥厚的室间隔。总之,虽然这些治疗策略的进一步发展仍在进行中,但它们都标志着肥厚型心肌病患者治疗的重大而有前途的进展。

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