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

IF 3.1 3区 医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Current Cardiology Reports 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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Abstract

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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肥厚型心肌病:从药物治疗到先进的心力衰竭疗法。
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。
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来源期刊
Current Cardiology Reports
Current Cardiology Reports CARDIAC & CARDIOVASCULAR SYSTEMS-
CiteScore
6.20
自引率
2.70%
发文量
209
期刊介绍: The aim of this journal is to provide timely perspectives from experts on current advances in cardiovascular medicine. We also seek to provide reviews that highlight the most important recently published papers selected from the wealth of available cardiovascular literature. We accomplish this aim by appointing key authorities in major subject areas across the discipline. Section editors select topics to be reviewed by leading experts who emphasize recent developments and highlight important papers published over the past year. An Editorial Board of internationally diverse members suggests topics of special interest to their country/region and ensures that topics are current and include emerging research. We also provide commentaries from well-known figures in the field.
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