ArticleJACC. Advances2025
Early Access to Tafamidis for Patients With Transthyretin Amyloid Cardiomyopathy.
Article in JACC. Advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02791230 (A PHASE 3 MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE), which is not on this map. Cited by 3 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A phase 3 multicenter, open-label study to evaluate the safety of daily oral dosing of tafamidis meglumine (pf-06291826-83) 20 mg or 80 mg [or tafamidis (pf-06291826-00) 61 mg] in subjects diagnosed with transthyretin cardiomyopathy (attr-cm)
Who cites it
3 citing papers in PubMed.
- Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis.Cardiology and therapy · 2026Article
- Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis.Cardiology and therapy · 2026Article
- Cardiovascular-Related Mortality and Hospitalizations in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: A Post Hoc Analysis of the Phase 3 ATTR-ACT and Long-Term Extension.Cardiology and therapy · 2026Article
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTafamidis is a standard of care treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM). While evidence was being assessed by regulatory authorities, a new, independent, and inclusive cohort of the phase 3 long-term extension study offered early access to tafamidis.
objectivesThe purpose of this study was to present safety, mortality, and hospitalization findings for patients who received early access tafamidis.
methodsPatients with ATTR-CM and who had not taken part in the phase 3 study were able to receive tafamidis free acid 61 mg (the dose later approved) for up to 60 months or until commercial availability in their region. Enrollment criteria were minimal.
resultsAmong the 1,476 patients initiating tafamidis in the study between 2018 and 2023, mean (SD) age at enrollment was 76.5 (7.8) years, 88.8% were male, 85.6% had wild-type ATTR-CM, and 52.9% had NYHA functional class II symptoms (I: 14.9%, III: 30.8%, IV: 1.3%). Median exposure and follow-up were 12 (range: 0-55) and 19 (95% CI: 18.4-20.7) months, respectively. Overall, 7.6% of patients reported treatment-related adverse events, with 0.6% considered serious and 0.6% leading to study discontinuation. No new safety signals were identified. In Kaplan-Meier analyses, all-cause and cardiovascular (CV)-related mortality occurred in 23.4% and 13.8% of patients over the study period. Furthermore, 43.3% and 26.5% of patients had all-cause and CV-related hospitalizations. The total annual CV-related hospitalization rate was 0.26.
conclusionsIn an inclusive patient cohort receiving early access to tafamidis, safety findings were consistent with those reported from other trials and real-world studies. (Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy; NCT02791230).
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