Long-term efficacy of tafamidis in patients with transthyretin amyloid cardiomyopathy by National Amyloidosis Centre stage.

ABSTRACT:

Aims

Tafamidis is an approved treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM) based on the 30-month Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT). This post-hoc analysis evaluated outcomes in ATTR-ACT and its long-term extension study (LTE) by baseline National Amyloidosis Centre (NAC) stage.

Methods and results

Patients received either the approved dose of tafamidis 80 mg or placebo in ATTR-ACT and tafamidis in the LTE. All-cause and cardiovascular (CV)-related mortality, CV-related hospitalizations, and Kansas City Cardiomyopathy Questionnaire overall summary and clinical summary (KCCQ-OS/CS) scores were assessed up to 90 months of follow-up. Of 353 patients, 350 were evaluable for NAC staging. At baseline, 42%, 38%, and 20%

SUBMITTER: Damy T 

PROVIDER: S-EPMC12803551 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature