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
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
Abstract
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.
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.