Vutrisiran in Patients with TransthyretinAmyloidosis with Cardiomyopathy

BACKGROUND
Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, fatal
disease. Vutrisiran, a subcutaneously administered RNA interference therapeutic
agent, inhibits the production of hepatic transthyretin.
METHODS
In this double-blind, randomized trial, we assigned patients with ATTR-CM in a 1:1
ratio to receive vutrisiran (25 mg) or placebo every 12 weeks for up to 36 months.
The primary end point was a composite of death from any cause and recurrent cardiovascular events. Secondary end points included death from any cause, the change
from baseline in the distance covered on the 6-minute walk test, and the change
from baseline in the Kansas City Cardiomyopathy Questionnaire–Overall Summary
(KCCQ-OS) score. The efficacy end points were assessed in the overall population and
in the monotherapy population (the patients who were not receiving tafamidis at
baseline) and were tested hierarchically