Heart failure & cardiomyopathiesSecondary analysis

Long-Term Efficacy of Tafamidis in Patients with Transthyretin Amyloid Cardiomyopathy by National Amyloidosis Centre stage

Eur J Heart FailPublished

  • Post-hoc analysis of the ATTR-ACT randomised trial and its long-term extension study
  • subgroup analysis by baseline National Amyloidosis Centre (NAC) stage

Authors' conclusion

Tafamidis treatment reduced the risk of mortality and hospitalization in patients with NAC stages I/II ATTR-CM, with favourable trends at stage III.

Study context

Population
Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in ATTR-ACT and its LTE, by baseline National Amyloidosis Centre (NAC) stage350 evaluable for NAC staging
Participants
353 patients
Comparison
Continuous tafamidis vs placebo to tafamidis
Primary endpoint
All-cause mortality by NAC stage
Follow-up
Up to 90 months

Primary result

As reported in the abstract

All-cause mortality by baseline NAC stage · end of study
NAC stage I: 36% vs. 61%
HR 0.43; P < 0.001
NAC stage II: 55% vs. 74%
HR 0.51; P = 0.003
NAC stage III: 69% vs. 88% (numerical trend)
HR 0.75; P = 0.298

Further findings

Cardiovascular (CV)-related mortality by NAC stage

Similar patterns as for all-cause mortality

CV-related hospitalizations and KCCQ-OS/CS scores by NAC stage · over follow-up

Stages I/II: lower CV-related hospitalization rates and frequently smaller declines in KCCQ-OS/CS with continuous tafamidis; favourable trends at stage III

Original publication

Long-Term Efficacy of Tafamidis in Patients with Transthyretin Amyloid Cardiomyopathy by National Amyloidosis Centre stage. Eur J Heart Fail. Published 9 June 2025. doi:10.1002/ejhf.3696

Summarised from the published abstract; every number is checked against it