肥厚型心肌病:从药物治疗到先进的心力衰竭疗法。

IF 4.3 3区 材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC
ACS Applied Electronic Materials Pub Date : 2024-09-01 Epub Date: 2024-07-11 DOI:10.1007/s11886-024-02095-6
Matylda Mazur, Wojciech Braksator, Eric Popjes
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引用次数: 0

摘要

综述的目的:围绕肥厚型心肌病(HCM)患者的新型医疗疗法和心脏移植上市难题一直存在许多争论:最近的临床试验促使美国食品与药物管理局批准了马伐康坦(一种心脏肌球蛋白抑制剂),为一些肥厚型心肌病和左心室流出道梗阻(LVOTO)患者缓解了症状,并有可能推迟/避免了侵入性室间隔缩小疗法。对于有难治性症状和终末期心力衰竭的患者,心脏移植仍是金标准。然而,器官分配系统未能优先考虑这些患者的问题依然存在。HCM 是一种异质性遗传病,具有不同的渗透性和临床表现。尽管大部分患者仍无症状,但也有少数患者会出现药物治疗难治的衰弱症状。HT 后的短期和长期预后良好。然而,HT 候诊死亡率仍然很高。对于经过严格筛选的 HCM 患者,左心室辅助装置是一种可行的选择。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Hypertrophic Cardiomyopathy: From Medical Treatment to Advanced Heart Failure Therapies.

Hypertrophic Cardiomyopathy: From Medical Treatment to Advanced Heart Failure Therapies.

Purpose of review: There has been much debate surrounding novel medical therapies and heart transplantation listing challenges in patients with hypertrophic cardiomyopathy (HCM).

Recent findings: Recent clinical trials led to FDA approval of mavacamten (a cardiac myosin inhibitor), offering symptom relief and potentially delaying/avoiding invasive septal reduction therapies for some patients with HCM and left ventricular outflow obstruction (LVOTO). For those with refractory symptoms and end-stage heart failure, heart transplantation remains the gold standard. However, the concern for the organ allocation system failing to prioritize those individuals persists. HCM is a heterogeneous genetic condition with variable penetration and clinical presentation. Even though a large portion of patients remain asymptomatic, an important minority develops debilitating symptoms refractory to medical therapy. Post-HT short- and long-term outcomes are favorable. However, HT waitlist mortality remains high. For highly selected patients with HCM, a left ventricular assist device is a viable option.

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CiteScore
7.20
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4.30%
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