Tafamidis Reduced the Decline in Health-Related Quality of Life in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT)

Abstract

Introduction In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), tafamidis reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is associated with a significant burden of disease and tafamidis reduced the decline in health-related quality of life (as assessed by the Kansas City Cardiomyopathy Questionnaire-Overall Summary [KCCQ-OS] score). Hypothesis Analysis of the changes in KCCQ-OS domains in patients with ATTR-CM over 30 months, together with the impact of tafamidis on these changes, will provide new data on the progression of disease and the efficacy of tafamidis. Methods In ATTR-ACT, 441 adult patients with ATTR-CM (variant or wild-type) were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (80mg and 20mg) compared with placebo. Change from baseline in the four KCCQ-OS domain scores were pre-specified exploratory endpoints and analyzed using a mixed model, repeated measures ANCOVA. The KCCQ is a 23-item self-administered questionnaire in which scores range from 0 to 100, with higher scores reflecting better health status. Results Tafamidis significantly reduced the decline in the KCCQ-OS and all four of its domains (P Conclusions Treatment with tafamidis effectively reduced the decline in all components of the KCCQ-OS, providing further insight into its efficacy in health-related quality of life in patients with ATTR-CM.