Treatment with the investigational drug tafamidis was associated with a reduction in all-cause mortality and cardiovascular hospitalizations in patients with transthyretin (TTR) amyloid cardiomyopathy, and improved functional capacity and quality of life, the phase III ATTR-ACT trial found. The mortality rate at 30 months was 29.5% in the tafamidis-treated group compared with 42.9% in the placebo group (HR 0.70, 95% CI 0.51-0.96), reported Claudio Rapezzi, MD, of the University of Bologna in Italy, and colleagues.
"In patients with this terrible, severe, previously drug-orphan disease, tafamidis can reduce all-cause mortality and cardiovascular-related hospitalizations," said Rapezzi in a late-breaking clinical trial session here at the European Society of Cardiology (ESC) meeting. The rate of cardiovascular-related hospitalizations during the trial was 0.48 per year with tafamidis vs 0.70 per year with placebo (relative risk ratio 0.68, 95% CI 0.56-0.81).