ATTR-ACT

Tafamidis in Transthyretin Amyloid Cardiomyopathy

Patient / Population Intervention / Exposure Comparison Outcome

In 441 patients with transthyretin amyloid cardiomyopathy, tafamidis reduced all-cause mortality and cardiovascular hospitalisation compared with placebo.

441

Design

Double-blind RCT

Endpoint

All-cause mortality + CV hospitalisation

Relevance

ResultMortality HR 0.70; fewer CV hospitalisations.

Maurer MS, et al. N Engl J Med. 2018;379(11):1007-1016. 10.1056/NEJMoa1805689

Discussion & critique

First disease-modifying therapy for ATTR cardiomyopathy. Survival curves separate only after ~ 18 months; cost and the need for early diagnosis limit reach.