Heart failure & cardiomyopathies
Impact of Vutrisiran on Functional Capacity and Quality of Life in Transthyretin Amyloidosis With Cardiomyopathy
Authors' conclusion
Vutrisiran maintained or improved functional capacity, health status, and QOL in more patients with ATTR-CM vs placebo over 30 months.
Study context
- Population
- Patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)
- Comparison
- Vutrisiran vs placebo
- Primary endpoint
- Maintained or improved functional capacity (6-MWT) and health status/quality of life (QOL) (KCCQ-OS) by worsening cutoffs (>7, >15, >35 m; >5, >10 points)
- Follow-up
- Month 30
Methodological details1:1 randomisation, placebo-controlled, prespecified subgroups
- 1:1 randomisation
- Placebo-controlled
- Prespecified subgroups
Primary result
As reported in the abstract
6-MWT, >7 m cutoff
6-MWT, >15 m cutoff
6-MWT, >35 m cutoff
KCCQ-OS, >5 points cutoff
P < 0.001
KCCQ-OS, >10 points cutoff
P < 0.01
- Vutrisiran vs Placebo: P < 0.001 — 6-MWT, all cutoffs
Further findings
LS mean difference in change from baseline in KCCQ subdomains, and in 6-MWT and Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS) across prespecified subgroups
- Favored vutrisiran vs placebo
Monotherapy population
- Benefits with vutrisiran similar in the monotherapy population
Original publication
Impact of Vutrisiran on Functional Capacity and Quality of Life in Transthyretin Amyloidosis With Cardiomyopathy. J Am Coll Cardiol. Published 17 March 2025. doi:10. 1016/ j. jacc. 2025. 03. 454
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