AI Article Synopsis
- The HELIOS-B trial is the first randomized study testing a gene silencing treatment, vutrisiran, for transthyretin cardiac amyloidosis (ATTR-CM) against a placebo.
- Vutrisiran showed a 28% reduction in all-cause mortality and recurrent cardiovascular events, along with significant improvements in patient-reported outcomes like the 6-minute walk test and quality of life metrics.
- Both treatment and control groups had similar rates of discontinuation and adverse events, indicating vutrisiran is well tolerated among patients.
Article Abstract
This focused review will highlight the results of HELIOS-B, the first randomized outcomes trial evaluating a gene silencing treatment for transthyretin cardiac amyloidosis (ATTR-CM). In HELIOS-B, vutrisiran was tested against placebo and demonstrated a 28% reduction in the composite of all-cause mortality and recurrent cardiovascular events. Additionally, there were clinically significant benefits on the 6-min walk test, Kansas City Cardiomyopathy Questionnaire, and NYHA class. Discontinuation rates and adverse events were similar between treatment and control arms, suggesting that vutrisiran is well tolerated. In this review, these promising results are explored and compared with other treatment trials in ATTR-CM.
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| http://dx.doi.org/10.1007/s10741-024-10444-4 | DOI Listing |
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