Nativi-Nicolau J, Judge DP, Hoffman JE, et al. - The study demonstrates that individuals with transthyretin amyloid cardiomyopathy (ATTR-CM) experience severe, progressive disease. In ATTR-ACT, in comparison with wild-type (ATTRwt), placebo-treated patients with hereditary (ATTRv) had the more severe disease at baseline, and their disease progressed more rapidly as shown by mortality, hospitalizations, and quality of life over time.

• Researchers evaluated changes in clinical endpoints (mortality, cardiovascular [CV]-related hospitalizations, 6-min walk test [6MWT] distance, and Kansas City Cardiomyopathy Questionnaire Overall Summary [KCCQ-OS] score) from baseline to Month 30 in the 177 patients (134 ATTRwt, 43 ATTRv) who received placebo in ATTR-ACT.

• The findings indicated that ATTRwt patients tended to have the less severe disease at baseline.

• There were 76 (42.9%) all-cause deaths, and 107 (60.5%) patients had a CV-related hospitalization over the duration of ATTR-ACT.

• A lower proportion of all-cause deaths was observed in ATTRwt (49, 36.6%) than ATTRv (27, 62.8%).

• The data showed a similar, steady decline in mean (SD) 6MWT distance from the baseline to Month 30 in ATTRwt (93.9 [93.7] m) and ATTRv (89.1 [107.2] m) patients.

• The findings revealed that a decline in mean (SD) KCCQ-OS score was less severe in ATTRwt (13.8 [20.7]) than ATTRv (21.0 [26.4]) patients.