Heart failure & cardiomyopathiesSecondary analysis
Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy
- Secondary analysis of the HELIOS-B randomised trial
- exploratory subgroup analysis
Authors' conclusion
Vutrisiran demonstrated evidence of benefit across the range of baseline disease severities in HELIOS-B, with the greatest benefit in earlier, less severe disease.
Study context
- Population
- Patients with ATTR-CM, NYHA class I–III (class IV or class III with NAC stage 3 excluded)
- Participants
- 654 patients
- Comparison
- Vutrisiran vs placebo by baseline HF severity
- Primary endpoint
- Composite of:
- All-cause mortality
- Recurrent cardiovascular (CV) events
Primary result
As reported in the abstract
All-cause mortality and recurrent CV events, by baseline severity subgroup
Vutrisiran vs PlaceboHazard ratio (95% CI)
NYHA class I
0.54(0.27–1.10)
NYHA class II
0.77(0.57–1.03)
NYHA class III
0.68(0.33–1.41)
NT-proBNP <1,368 ng/L
0.52(0.30–0.88)
NT-proBNP ≥1,368 and <2,691 ng/L
0.61(0.37–1.00)
NT-proBNP ≥2,691 ng/L
0.93(0.64–1.35)
NAC stage 1
0.49(0.34–0.72)
NAC stage 2/3
1.08(0.74–1.56)
Columbia early stage
0.69(0.45–1.07)
Columbia intermediate/late stage
0.74(0.53–1.02)
Further findings
Monotherapy population (not on tafamidis) and additional functional and biomarker endpoints
- Similar effects in the monotherapy population and across additional endpoints
Original publication
Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy. J Am Coll Cardiol. Published 17 March 2025. doi:10.1016/j.jacc.2025.03.477
Summarised from the published abstract; every number is checked against it