A recent study, published in Nature Medicine, looked at the effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) through a secondary analysis of the HELIOS-B clinical trial.
ATTR-CM is a rare amyloidosis caused by aging or genetic mutations. This results in the misfolding of TTR protein and accumulation of amyloid fibers in the cardiac myocardium. TTR fibrils build up in tissues, such as the peripheral nerves and heart, as well as the gastrointestinal tract, eyes, kidneys, central nervous system, thyroid, and bone marrow. This accumulation causes progressive dysfunction of these tissues. The disease eventually leads to heart failure.
Vutrisiran is an RNAi therapy that works upstream to decrease TTR production, addressing the cause of disease. It is administered in four subcutaneous doses per year. It is the first and only treatment approved by the U.S. Food and Drug Administration (FDA) for the treatment of ATTR-CM.
The HELIOS-B (NCT04153149) clinical trial is a phase 3 randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and efficacy of vutrisiran in patients with ATTR-CM. The objective of this secondary analysis was to evaluate vutrisiran’s effects on echocardiographic measures of cardiac structure and function in patients with ATTR-CM receiving vutrisiran or placebo (n=654).
At 30 months post-treatment, vutrisiran reduced increases in mean left ventricular (LV) wall thickness and LV mass index, compared to the placebo group. Treatment with vutrisiran also decreased declines in LV ejection fraction, absolute global longitudinal strain, and LV stroke volume. Decreases were also observed in the average ratio of early diastolic transmitral flow velocity to early diastolic mitral annular tissue velocity and the early to late diastolic transmitral flow velocities ratio, compared with placebo.
These echocardiographic findings indicate favorable effects of vutrisiran on cardiac structure and function in patients with ATTR-CM, alongside its clinical benefit.
To learn more ATTR-CM and other rare heart conditions, visit https://checkrare.com/diseases/heart-diseases/
