Impact of Vutrisiran on Cardiac Biomarkers in Patients With Transthyretin Amyloidosis With Cardiomyopathy From HELIOS-B

作者：Matthew J. Maurer, John L. Berk, Thibaud Damy, Farooq H. Sheikh, José González‐Costello, Caroline Morbach, Diego Delgado, Antoine Bondue, Olga Azevedo, Steen Hvitfeldt Poulsen, Ewa A. Jankowska, Lili Yang, Shaun Bender, Satish A. Eraly, Patrick Y. Jay, John Vest, Marianna Fontana · 发表于：Journal of the American College of Cardiology · 年份：2025 · DOI：10.1016/j.jacc.2025.04.055 · 被引用次数：10 · 研究领域：Amyloidosis: Diagnosis, Treatment, Outcomes、Dermatological and Skeletal Disorders、Parathyroid Disorders and Treatments

BACKGROUND: Before the development of disease-modifying therapies for transthyretin amyloidosis cardiomyopathy (ATTR-CM), N-terminal prohormone of B-type natriuretic peptide (NT-proBNP) and troponin I/T were recognized as independent prognostic biomarkers of mortality. This study evaluated the prognostic value of these biomarkers in a contemporary patient population and the impact of vutrisiran, an RNA interference therapeutic that rapidly knocks down circulating transthyretin, on biomarker levels. OBJECTIVES: This study sought to evaluate the association between risk of cardiovascular events and all-cause mortality with baseline NT-proBNP and troponin I levels and changes from baseline at month 6 in patients from HELIOS-B and explore how vutrisiran impacts biomarkers over time. METHODS: In HELIOS-B, a double-blind, placebo-controlled study, 655 patients with ATTR-CM were randomized 1:1 to receive vutrisiran or placebo for up to 36 months. The primary endpoint was a composite outcome of all-cause mortality and recurrent cardiovascular events. All-cause mortality through 42 months was a secondary endpoint. NT-proBNP and troponin I were assessed as prespecified exploratory endpoints. RESULTS: Baseline NT-proBNP and troponin I levels were independently associated with risks of the composite outcome and all-cause mortality (P < 0.0001 for both biomarkers and endpoints). At month 6, increases in NT-proBNP from baseline were associated with higher risk of the composite outcome and ...