J Card Fail. 2026 May;32(5):853-863. doi: 10.1016/j.cardfail.2026.02.049.
ABSTRACT
BACKGROUND: In HELIOS-B (HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy), vutrisiran reduced the risk of all-cause mortality and cardiovascular events associated with ATTR-CM. We assessed the effects of vutrisiran on renal function and on outcomes by renal function.
METHODS: Patients were randomized to double-blind treatment with vutrisiran 25 mg or placebo every 3 months for ≤36 months. We evaluated the proportion of patients with a ≥40% decline in the estimated glomerular filtration rate (eGFR) from baseline and the primary composite end point (all-cause mortality or recurrent cardiovascular events) in subgroups based on baseline eGFR levels, and development of chronic kidney disease (CKD) stage ≥4 (eGFR <30 mL/min/1.73 m2) during treatment.
RESULTS: In HELIOS-B, 654 patients were randomized and treated with vutrisiran (n = 326) or placebo (n = 328). A ≥40% decline in the eGFR was seen in significantly fewer patients receiving vutrisiran vs placebo in the overall (12.7% vs 21.2%, P = .0041) and monotherapy (12.0% vs 21.9%, P = .0102) populations. The effect of vutrisiran on the primary composite end point in subgroups based on renal function was directionally consistent with the effect in the overall population. Among patients receiving vutrisiran vs placebo, 9.5% vs 9.8%, respectively, developed CKD stage ≥4. The risk of a primary composite end point event was significantly lower with vutrisiran vs placebo in patients who developed CKD stage ≥4 (hazard ratio 0.467, 95% confidence interval 0.258-0.845). No new safety concerns were identified.
CONCLUSIONS: Vutrisiran may slow eGFR decline and is effective and well-tolerated in patients with ATTR-CM with declining renal function, including those developing CKD stage ≥4.
PMID:42128579 | DOI:10.1016/j.cardfail.2026.02.049
