Did someone say Amyloidosis?
Posted on 09/03/2024 3:25:07 PM PDT by ConservativeMind
Vutrisiran significantly improved mortality, cardiovascular events and markers of disease progression in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), according to late-breaking research.
"ATTR is a progressive, fatal disease in which misfolded transthyretin protein accumulates as amyloid deposits in various parts of the body, often damaging the heart.
HELIOS-B was a randomized, double-blind trial in patients with ATTR-CM (hereditary or wild-type) who had evidence of cardiac amyloidosis by echocardiography and confirmed ATTR amyloid deposition. Patients were randomized in a 1:1 ratio to vutrisiran 25 mg or placebo administered subcutaneously once every three months for up to 36 months. If the patient was already receiving treatment with the disease stabilizer, tafamidis, this was continued.
The two primary endpoints were a composite of all-cause mortality and recurrent cardiovascular events when the last patient reached month 33.
The trial met the primary endpoints. Vutrisiran significantly reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% in the overall population (hazard ratio [HR] 0.72; 95% confidence interval [CI] 0.56–0.93; p=0.01) and by 33% in the monotherapy population (HR 0.67; 95% CI 0.49–0.93; p=0.016). In a prespecified subgroup analysis, the composite of all-cause mortality and recurrent cardiovascular events was reduced by more than 20% in patients on background tafamidis (HR 0.79; 95% CI 0.51–1.21).
Vutrisiran reduced all-cause mortality over 42 months by 36% in the overall population (HR 0.64; 95% CI 0.46–0.90; p=0.01) and by 35% in the monotherapy population (HR 0.65; 95% CI 0.44–0.97; p=0.045) vs. placebo. Other secondary endpoints related to functional capacity, health status and quality of life were significantly improved with vutrisiran vs. placebo.
The majority of adverse events were mild or moderate with vutrisiran.
"Our findings indicate that vutrisiran has the potential to become the new standard of care."
(Excerpt) Read more at medicalxpress.com ...
Did someone say Amyloidosis?
Bkmk
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