Vutrisiran extension reports outcomes through 48 months in ATTR cardiomyopathy

An open-label extension of the HELIOS-B trial followed 466 participants with transthyretin amyloidosis and cardiomyopathy who had completed the randomized study. Through month 48, those treated continuously with vutrisiran had fewer cardiovascular events or deaths and better measures of health status than participants who switched from placebo to vutrisiran.

466 participants entered the open-label extension.
Continuous treatment was associated with lower event and mortality risks.
The extension compared earlier with delayed vutrisiran initiation.
No new safety signals were reported.

HELIOS-B previously found that vutrisiran reduced mortality and recurrent cardiovascular events versus placebo in patients with transthyretin (TTR) amyloidosis with cardiomyopathy. This report describes outcomes through month 48 in its ongoing open-label extension. Of 654 participants in the parent trial, 466 entered the extension: 243 continued vutrisiran 25 mg every three months, while 223 participants previously assigned placebo began vutrisiran at the same dose. Prespecified outcomes included mortality and cardiovascular events, health status, functional class, and cardiac biomarkers.

Compared with the placebo/vutrisiran group, the continuous-vutrisiran group had a 37% lower risk of a first cardiovascular event or all-cause death (hazard ratio [HR], 0.63; 95% CI, 0.51–0.79) and a 37% lower risk of all-cause death (HR, 0.63; 95% CI, 0.46–0.84). The groups also differed in Kansas City Cardiomyopathy Questionnaire overall score (least-squares mean difference, 9.0 points) and odds of improved or stable NYHA class (odds ratio, 1.6). NT-proBNP and troponin I measures favored continuous treatment. No new safety signals were reported.

The findings extend follow-up, but the open-label extension comparison is between earlier and later treatment rather than a continuing placebo-controlled comparison. The results support the potential importance of earlier treatment, but this report alone does not establish the effect of starting treatment at a particular time. The study was registered as NCT04153149.

In practice

For patients with TTR amyloidosis and cardiomyopathy who completed HELIOS-B, the open-label extension reports that those treated continuously with vutrisiran had better outcomes through month 48 than those who began vutrisiran after placebo, including lower risks of cardiovascular events or death. This supports the potential value of earlier treatment, but the extension is open-label and no longer provides a continuing placebo-controlled comparison. The findings do not establish a specific timing recommendation or independently determine treatment decisions.

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Source

Circulation: 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 ↗

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