{"id":"93253baad7ca","type":"article","url":"https://hartvaat.nl/2025/05/27/vutrisiran-bij-attr-cm-impact-van-hartfalenernst-op-effectiviteit/","title":"Vutrisiran bij ATTR-CM: impact van hartfalenernst op effectiviteit","title_en":"Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy.","category":"hartfalen","category_label":"Hartfalen","professions":["cardioloog"],"tags":[],"journal":"Journal of the American College of Cardiology","doi":"10.1016/j.jacc.2025.03.477","source_url":"https://doi.org/10.1016/j.jacc.2025.03.477","authors":["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"],"significance":6,"published":"2025-05-27","source_date":"2025-05-27","image":"","kennis":["https://hartvaat.nl/kennis/hartfalen/hartfalen-stadiumindeling-nyha/","https://hartvaat.nl/kennis/hartfalen/digoxine-bij-hartfalen/"],"congress":"","summary_en":"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.","created":"2026-07-03T10:31:38Z","updated":"2026-07-03T13:30:38Z","licence":"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.","body_markdown":"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.","abstract_original":"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)."}