Understanding ATTR Amyloidosis

ATTR amyloidosis is a systemic condition characterized by the misfolding and deposition of trans-thyretin proteins in various organs, predominantly affecting the heart in its wild-type form. The Apollo B trial investigated the efficacy of patisiran, an RNA interference drug, in improving patient outcomes by reducing these problematic proteins. The study included patients diagnosed with ATTR cardiac amyloidosis and measured outcomes such as functional capacity through the six-minute walk test and quality of life via the Kansas City Cardiomyopathy Questionnaire. While the drug showed a statistical reduction in the decline of functional capacity compared to placebo, the actual clinical relevance of this difference remains questionable, prompting concerns about its practical implications for patients.