# Vutrisiran bij ATTR-CM: impact van hartfalenernst op effectiviteit

*geplaatst 2025-05-27 · Hartfalen · Journal of the American College of Cardiology · doi 10.1016/j.jacc.2025.03.477 · https://hartvaat.nl/2025/05/27/vutrisiran-bij-attr-cm-impact-van-hartfalenernst-op-effectiviteit/*

Analyse onderzocht het effect van hartfalenernst op de effectiviteit van vutrisiran bij ATTR-cardiomyopathie. Het voordeel was consistent over NYHA-klassen, wat breed gebruik ondersteunt.

## English: Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy.

This analysis showed that vutrisiran's cardiovascular benefit in ATTR cardiomyopathy is consistent regardless of heart failure severity, supporting RNA interference-based TTR silencing across the disease spectrum.

## Abstract (original, from the publication)

BACKGROUND: Vutrisiran reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular (CV) events in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in HELIOS-B (A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy; NCT04153149). OBJECTIVES: This study sought to assess the effect of vutrisiran in HELIOS-B patients with different heart failure severities. METHODS: HELIOS-B randomized patients with ATTR-CM with NYHA functional class I-III (functional class IV or functional class III with National Amyloidosis Centre [NAC] stage 3 were excluded) 1:1 to vutrisiran 25 mg or placebo every 3 months for up to 36 months. This exploratory subgroup analysis assessed the primary composite endpoint of ACM and recurrent CV events, ACM, and additional functional and biomarker endpoints. RESULTS: Of 654 patients, 84 (13%), 508 (78%), and 62 (9%) were in NYHA functional class I, II, and III, respectively. Median baseline N-terminal pro-B-type natriuretic peptide (NT-proBNP) level was 1,920 ng/L. Lower risk of ACM and recurrent CV events was observed with vutrisiran vs placebo across baseline severity subgroups: respective HRs were 0.54 (95% CI: 0.27-1.10), 0.77 (95% CI: 0.57-1.03), and 0.68 (95% CI: 0.33-1.41) in NYHA functional classes I, II, and III, respectively; 0.52 (95% CI: 0.30-0.88), 0.61 (95% CI: 0.37-1.00), and 0.93 (95% CI: 0.64-1.35) in NT-proBNP tertiles <1,368 ng/L, ≥1,368 and <2,691 ng/L, and ≥2,691 ng/L; 0.49 (95% CI: 0.34-0.72) and 1.08 (95% CI: 0.74-1.56) in NAC stages 1 and 2/3, respectively; and 0.69 (95% CI: 0.45-1.07) and 0.74 (95% CI: 0.53-1.02) in Columbia early and intermediate/late stages, respectively. Similar effects were observed in the monotherapy population (patients not on tafamidis at baseline) and across the additional endpoints evaluated. CONCLUSIONS: Vutrisiran demonstrated evidence of benefit across the range of baseline disease severities in HELIOS-B, with the greatest benefit in earlier, less severe disease. (A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy [HELIOS-B]; NCT04153149).

Auteurs: Mathew S Maurer, Ronald M Witteles, Pablo Garcia-Pavia, Farooq H Sheikh, Caroline Morbach, Daniel Rodriguez Duque, Emre Aldinc, Satish A Eraly, Julian D Gillmore

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Bron: Journal of the American College of Cardiology, https://doi.org/10.1016/j.jacc.2025.03.477. Bijgewerkt 2026-07-03T13:30:38Z. Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.