在治疗转甲状腺素心脏淀粉样变性病的临床试验的大背景下理解 HELIOS-B 的结果。

IF 4.5 2区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

Heart Failure Reviews Pub Date : 2024-10-01 DOI:10.1007/s10741-024-10444-4

Andrew A Girard, Brett W Sperry

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引用次数: 0

摘要

HELIOS-B 是首个评估转甲状腺素心脏淀粉样变性（ATTR-CM）基因沉默疗法的随机结果试验。在 HELIOS-B 试验中，vutrisiran 与安慰剂进行了对比试验，结果显示，全因死亡率和复发性心血管事件的综合死亡率降低了 28%。此外，在6分钟步行测试、堪萨斯城心肌病问卷调查和NYHA分级方面也有显著的临床疗效。治疗组和对照组的停药率和不良反应相似，表明武曲西兰的耐受性良好。本综述探讨了这些令人鼓舞的结果，并与其他 ATTR-CM 治疗试验进行了比较。

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Contextualizing the results of HELIOS-B in the broader landscape of clinical trials for the treatment of transthyretin cardiac amyloidosis.

This focused review will highlight the results of HELIOS-B, the first randomized outcomes trial evaluating a gene silencing treatment for transthyretin cardiac amyloidosis (ATTR-CM). In HELIOS-B, vutrisiran was tested against placebo and demonstrated a 28% reduction in the composite of all-cause mortality and recurrent cardiovascular events. Additionally, there were clinically significant benefits on the 6-min walk test, Kansas City Cardiomyopathy Questionnaire, and NYHA class. Discontinuation rates and adverse events were similar between treatment and control arms, suggesting that vutrisiran is well tolerated. In this review, these promising results are explored and compared with other treatment trials in ATTR-CM.

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来源期刊

Heart Failure Reviews 医学-心血管系统

CiteScore

10.40

自引率

2.20%

发文量

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.