Girard Andrew A, Sperry Brett W
Saint Luke's Mid America Heart Institute, 4401 Wornall Road MO, Kansas City, 64111, USA.
University of Missouri-Kansas City, Kansas City, MO, USA.
Heart Fail Rev. 2025 Jan;30(1):69-73. doi: 10.1007/s10741-024-10444-4. Epub 2024 Oct 1.
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.
本聚焦综述将重点介绍HELIOS-B的结果，这是第一项评估用于转甲状腺素蛋白心脏淀粉样变性（ATTR-CM）的基因沉默治疗的随机结局试验。在HELIOS-B中，对vutrisiran与安慰剂进行了对比测试，结果显示全因死亡率和复发性心血管事件的综合发生率降低了28%。此外，在6分钟步行试验、堪萨斯城心肌病问卷和纽约心脏协会（NYHA）心功能分级方面也有临床显著益处。治疗组和对照组的停药率及不良事件相似，这表明vutrisiran耐受性良好。在本综述中，将探讨这些有前景的结果，并与ATTR-CM的其他治疗试验进行比较。
