Heart failure & cardiomyopathies
Long-Term Efficacy of Tafamidis in Patients with Transthyretin Amyloid Cardiomyopathy by National Amyloidosis Centre 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
Methodological detailssubgroup analysis by baseline NAC stage
- Subgroup analysis by baseline NAC stage
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.
Summarised from the published abstract; every number is checked against it