What is this summary about?This summary explains the results of the HELIOS-B study that assessedvutrisiran treatment in transthyretin amyloidosis with cardiomyopathy(ATTR-CM). In ATTR-CM, a protein called transthyretin accumulates in clumpscalled amyloid in different parts of the body, such as the heart and nerves,causing damage. In the heart, this decreases functioning and leads to heartfailure and therefore more frequent hospital visits and a greater risk of dying.Vutrisiran lowers the amount of transthyretin in the blood.
In the HELIOS-B study, 654 participants received the drug vutrisiran orplacebo (liquid containing no drug), injected under the skin every 12 weeksfor up to 3 years. The study assessed the effect of vutrisiran on deaths, cardiachospitalizations, and urgent doctor visits for heart failure. The study alsolooked at changes in physical function (ability to walk), quality of life andheart failure symptoms, and levels of proteins linked to heart failure.What were the results?
