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梗阻性肥厚型心肌病的治疗策略进展。

Evolving Strategies for the Management of Obstructive Hypertrophic Cardiomyopathy.

机构信息

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.

出版信息

J Card Fail. 2024 Sep;30(9):1136-1153. doi: 10.1016/j.cardfail.2024.04.024. Epub 2024 May 20.

DOI:10.1016/j.cardfail.2024.04.024
PMID:38777216
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11415289/
Abstract

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.

摘要

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。

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引用本文的文献

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Biomedicines. 2025 May 21;13(5):1256. doi: 10.3390/biomedicines13051256.

本文引用的文献

1
Effect of Mavacamten on Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy: The EXPLORER-CN Randomized Clinical Trial.马卡丹特治疗中国有症状梗阻性肥厚型心肌病患者的疗效:EXPLORER-CN 随机临床试验。
JAMA Cardiol. 2023 Oct 1;8(10):957-965. doi: 10.1001/jamacardio.2023.3030.
2
Aficamten for Drug-Refractory Severe Obstructive Hypertrophic Cardiomyopathy in Patients Receiving Disopyramide: REDWOOD-HCM Cohort 3.接受丙吡胺治疗的药物难治性重度梗阻性肥厚型心肌病患者使用阿菲卡坦:红木-HCM队列3
J Card Fail. 2023 Nov;29(11):1576-1582. doi: 10.1016/j.cardfail.2023.07.003. Epub 2023 Jul 18.
3
Phase 2 Study of Aficamten in Patients With Obstructive Hypertrophic Cardiomyopathy.
阿菲卡明治疗梗阻性肥厚型心肌病患者的2期研究。
J Am Coll Cardiol. 2023 Jan 3;81(1):34-45. doi: 10.1016/j.jacc.2022.10.020.
4
Myosin Inhibition in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy.梗阻性肥厚型心肌病患者行室间隔减少术的肌球蛋白抑制治疗。
J Am Coll Cardiol. 2022 Jul 12;80(2):95-108. doi: 10.1016/j.jacc.2022.04.048.
5
Transcatheter Myotomy to Treat Hypertrophic Cardiomyopathy and Enable Transcatheter Mitral Valve Replacement: First-in-Human Report of Septal Scoring Along the Midline Endocardium.经导管心肌切开术治疗肥厚型心肌病并实现经导管二尖瓣置换:沿中线心内膜进行间隔评分的首例人体报告。
Circ Cardiovasc Interv. 2022 Jun;15(6):e012106. doi: 10.1161/CIRCINTERVENTIONS.122.012106. Epub 2022 May 23.
6
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J Am Heart Assoc. 2022 May 3;11(9):e024656. doi: 10.1161/JAHA.121.024656.
7
Survival Following Alcohol Septal Ablation or Septal Myectomy for Patients With Obstructive Hypertrophic Cardiomyopathy.酒精间隔消融术或间隔心肌切除术治疗梗阻性肥厚型心肌病患者后的生存率
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J Am Coll Cardiol. 2021 Dec 21;78(25):2518-2532. doi: 10.1016/j.jacc.2021.09.1381.