Modulating energy metabolism to treat non-obstructive hypertrophic cardiomyopathy? Insights from IMPROVE-HCM.

IF 4.5 2区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Alberto Beghini, Alberto Aimo, Andrew P Ambrosy, Daniela Tomasoni
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引用次数: 0

Abstract

Hypertrophic cardiomyopathy (HCM) is the most common genetic heart disease worldwide and may present with or without dynamic left ventricular outflow tract obstruction (LVOTO). Significant advances have been made in the management of obstructive HCM. On the other hand, despite their significant symptomatic burden, patients with non-obstructive HCM (nHCM) (i.e., without LVOTO) still do not have evidence-based therapeutical options. The recent IMPROVE-HCM study, a phase 2 randomized, double-blinded trial, aims to place a first step in filling this gap in knowledge. The study assessed the safety (primary endpoint) and efficacy (secondary endpoint) of ninerafaxstat, a novel cardiac mitotrope drug that increases adenosine triphosphate production. We highlighted the main findings of the trial, contextualizing these results within the larger landscape of completed and ongoing trials in nHCM.

调节能量代谢治疗非梗阻性肥厚型心肌病?来自 IMPROVE-HCM 的启示。
肥厚型心肌病(HCM)是全球最常见的遗传性心脏病,可伴有或不伴有动态左心室流出道梗阻(LVOTO)。阻塞性 HCM 的治疗已取得重大进展。另一方面,非阻塞性 HCM(nHCM)(即无左心室流出道梗阻)患者尽管症状严重,但仍然没有循证治疗方案。最近的 IMPROVE-HCM 研究是一项二期随机双盲试验,旨在为填补这一知识空白迈出第一步。该研究评估了尼那非司他的安全性(主要终点)和疗效(次要终点),尼那非司他是一种新型心脏有丝分裂药物,可增加三磷酸腺苷的产生。我们重点介绍了试验的主要发现,并将这些结果与已完成和正在进行的 nHCM 试验结合起来。
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来源期刊
Heart Failure Reviews
Heart Failure Reviews 医学-心血管系统
CiteScore
10.40
自引率
2.20%
发文量
90
审稿时长
6-12 weeks
期刊介绍: Heart Failure Reviews is an international journal which develops links between basic scientists and clinical investigators, creating a unique, interdisciplinary dialogue focused on heart failure, its pathogenesis and treatment. The journal accordingly publishes papers in both basic and clinical research fields. Topics covered include clinical and surgical approaches to therapy, basic pharmacology, biochemistry, molecular biology, pathology, and electrophysiology. The reviews are comprehensive, expanding the reader''s knowledge base and awareness of current research and new findings in this rapidly growing field of cardiovascular medicine. All reviews are thoroughly peer-reviewed before publication.
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