Treatment of Variant or Wild-Type Transthyretin Cardiac Amyloidosis

Transthyretin amyloidosis, also known as ATTR amyloidosis, can cause progressive infiltrative cardiomyopathy that commonly leads to death, with a median survival of 2 to 6 years. Patisiran is an RNA interference therapeutic agent that targets the TTR messenger RNA in the liver and reduces circulating transthyretin protein; the FDA approved it for treating variant (hereditary) ATTR in patients with polyneuropathy. Now, investigators report findings of a phase 3, randomized, placebo-controlled trial of intravenous patisiran for patients with variant or wild-type ATTR cardiac amyloidosis.

The study enrolled 360 participants in 21 countries; mean age was 76 years, 10% were women, and about 80% were white people. The primary endpoint was the chan…