vtrisiran对转甲状腺蛋白淀粉样变合并心肌病患者功能能力、健康状况和生活质量的维持或改善：来自HELIOS-B研究的数据

IF 2.3 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS

Archives of Cardiovascular Diseases Pub Date : 2025-05-30 DOI:10.1016/j.acvd.2025.04.024

F.H. Sheikh , G. Habib , W.H.W. Tang , J. Gillmore , U. Egolum , S. Longhi , G. Jia , E. Aldinc , M. Fontana

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引用次数: 0

摘要

甲状腺素转蛋白淀粉样变合并心肌病（atr - cm）会逐渐损害患者的功能、健康状况和生活质量（QOL）。在HELIOS-B试验中，与安慰剂（pcb）相比，vutrisiran降低了atr - cm患者的全因死亡率和心血管事件，并在疾病进展的多项临床指标上显示出显著的益处。目的：研究旨在确定乌曲西兰在当代atr - cm患者中的有效性和安全性。方法在HELIOS-B中，6分钟步行测试（6MWT）和堪萨斯城心肌病问卷-总体总结（KCCQ-OS）评分是评估功能能力和健康状况/生活质量的次要终点。我们进一步详细介绍了vutrisiran对患者功能能力、健康状况和生活质量的影响。结果在第30个月的总体人群中，ls -平均变化与基线的差异（vutrisiran-pcb）为26.46 m (95% CI 13.38,39.55；P & lt;0.001)和5.80点(95% CI 2.40,9.20；P & lt;0.001)的KCCQ-OS。30个月后，在总体人群中，分别有64%和47%的患者接受了乌曲西兰和多氯联苯治疗(P <；0.05)[单药人群；65%和38% (P <；0.05)]，增加了<；KCCQ-OS下降5个点。在预先指定的亚组、子域和不同的临界值/临床阈值中，观察到vutrisiran对6MWT和KCCQ-OS的一致益处。同样的趋势也出现在基线他法底亚组。结论与多氯联苯相比，氟曲西兰治疗的患者维持或改善的功能能力和健康状况/生活质量明显高于氟曲西兰治疗，进一步证明了氟曲西兰治疗的疾病改善作用。HELIOS-B研究的次要终点分析表明，与pcb相比，vutrisiran可保持atr - cm患者的功能容量（6MWT）和健康状态（KCCQ-OS）。在预先指定的亚组、子域和不同的临界值/临床阈值中，vutrisiran观察到的益处是一致的。

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Maintenance or improvement of functional capacity, health status, and quality of life with vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy: Data from the HELIOS-B study

Background

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) progressively impairs patients’ (pts) functional capacity, health status, and quality of life (QOL). In HELIOS-B, vutrisiran reduced all-cause mortality and cardiovascular events compared with placebo (pcb) in pts with ATTR-CM and demonstrated significant benefit on multiple clinical measures of disease progression.

Objectives

Study aimed to establish the efficacy and safety of vutrisiran in a contemporary population of pts with ATTR-CM.

Methods

In HELIOS-B, 6-minute walk test (6MWT) and Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS) score were secondary endpoints assessing functional capacity and health status/QOL. We present further details on the efficacy of vutrisiran on pts’ functional capacity, health status, and QOL.

Results

In the overall population at Month 30, the difference of LS-mean change from baseline (vutrisiran–pcb) was 26.46 m (95% CI 13.38,39.55; P < 0.001) for the 6MWT and 5.80 points (95% CI 2.40,9.20; P < 0.001) for the KCCQ-OS. Over 30 months, in the overall population, 64% and 47% of pts treated with vutrisiran and pcb, respectively (P < 0.05) [monotherapy population; 65% and 38% (P < 0.05)], had an increase or < 5-point decrease in KCCQ-OS. Consistent benefits with vutrisiran were observed for both 6MWT and KCCQ-OS in prespecified subgroups, subdomains and different cut-off values/clinical thresholds. Similar trends were observed in the baseline tafamidis subgroup.

Conclusion

Significantly more pts treated with vutrisiran maintained or improved functional capacity and health status/QOL compared with pcb, providing further evidence of the disease-modifying effect of vutrisiran treatment. Secondary endpoint analyses of the HELIOS-B study demonstrated that vutrisiran preserved functional capacity (6MWT) and health status (KCCQ-OS) compared with pcb in pts with ATTR-CM. Benefits observed with vutrisiran were consistent in pre-specified subgroups, subdomains and different cut-off values/clinical thresholds.

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来源期刊

Archives of Cardiovascular Diseases 医学-心血管系统

CiteScore

4.40

自引率

6.70%

发文量

审稿时长

34 days

期刊介绍： The Journal publishes original peer-reviewed clinical and research articles, epidemiological studies, new methodological clinical approaches, review articles and editorials. Topics covered include coronary artery and valve diseases, interventional and pediatric cardiology, cardiovascular surgery, cardiomyopathy and heart failure, arrhythmias and stimulation, cardiovascular imaging, vascular medicine and hypertension, epidemiology and risk factors, and large multicenter studies. Archives of Cardiovascular Diseases also publishes abstracts of papers presented at the annual sessions of the Journées Européennes de la Société Française de Cardiologie and the guidelines edited by the French Society of Cardiology.