Long-Term Impact of Vutrisiran in Patients With TTR Amyloidosis With Cardiomyopathy: 1-Year Efficacy and Safety Outcomes From the HELIOS-B Open-Label Extension.

Continuous vutrisiran in HELIOS-B participants with transthyretin (TTR) amyloidosis with cardiomyopathy reduced risk of first cardiovascular event or all-cause mortality by 37% through month 48.

• Why it matters: The study addresses whether vutrisiran’s earlier benefits persist long term in real-world follow-up, including mortality, cardiovascular events, biomarkers, and patient-reported function.
• What they did: Patients completing HELIOS-B entered an open-label extension and received vutrisiran 25 mg every 3 months, either continuously (n=243) or after switching from placebo (n=223); outcomes were assessed through month 48.
• The result: Through month 48, continuous treatment preserved Kansas City Cardiomyopathy Questionnaire-Overall Summary (difference 9.0; P<0.0001), improved or stabilized NYHA class (odds ratio 1.6; P=0.0084), and maintained NT-proBNP and troponin I (fold-change ratios 0.66 and 0.71; both P<0.0001), with no new safety signals.

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