Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis
5 affiliations
- 1Department of Cardiology, University Hospital Heidelberg
- 2Mayo Clinic
- 3Heart, Vascular and Thoracic Institute, Cleveland Clinic
- 4Pfizer Inc
- 5Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, and Centro Nacional Investigaciones Cardiovasculares (CNIC)
Abstract
Introduction Tafamidis is approved for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) and has shown a survival benefit in the phase 3 study ATTR-ACT and in real-world settings. This analysis compared survival in a contemporary, matched, real-world cohort of patients with ATTR-CM treated with tafamidis vs untreated. Methods Data were pooled from an early access cohort (NCT02791230) and from patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) in 2019–2023 with a mixed or predominantly cardiac phenotype. Propensity score matching (1:1) was performed to match treated and untreated patients in the pooled sample based on baseline age, New York Heart Association class, and phenotype. Treated patients received tafamidis at the approved dose for ATTR-CM. Results A total of 283 treated and 283 untreated patients were included. Of the 283 treated patients, 85 were from the early access cohort and 198 from THAOS. All untreated patients were from THAOS. In treated vs untreated patients, mean age at enrollment was 76.9 vs 77.4 years, 89.0% vs 83.4% were male, 11.0% vs 16.6% were female, and 80.9% vs 73.1% had wild-type ATTR-CM. Estimated adjusted survival probabilities for all-cause mortality for treated vs untreated patients were 90.2% vs 76.0% at 30 months and 85.3% vs 67.5% at 42 months. Treated patients had a 74% lower risk of all-cause mortality compared with untreated patients (hazard ratio 0.26; 95% confidence interval 0.13–0.51). No new safety signals were identified. Conclusions This matched-cohort analysis demonstrated improved survival in a contemporary, real-world cohort of patients with ATTR-CM receiving the approved dose of tafamidis compared with those untreated. These findings further extend the results of ATTR-ACT to contemporary populations of patients with ATTR-CM treated clinically with tafamidis and reinforce earlier real-world evidence supporting tafamidis use. Trial Registration Clinicaltrials.gov identifiers NCT00628745 and NCT02791230.
Related
- Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis
- Impact of Tafamidis on Transthyretin Concentration and Its Relation to All-Cause Mortality in Patients with Transthyretin Amyloid Cardiomyopathy: An Analysis from ATTR-ACT
- 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
- Phenotypic Considerations for Tafamidis Use in a Real-World Cohort of Older Patients with Transthyretin Amyloid Cardiomyopathy
- Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan
BibTeX
@article{siepen2026survival,
title = {{Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis}},
author = {Fabian aus dem Siepen and Angela Dispenzieri and Mazen Hanna and Martin Carlsson and Pablo Garcia-Pavia},
journal = {Cardiology and Therapy},
year = {2026},
month = aug,
doi = {10.1007/s40119-026-00464-6},
url = {https://doi.org/10.1007/s40119-026-00464-6},
note = {CC BY-NC},
}
RIS
TY - JOUR
TI - Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis
AU - Fabian aus dem Siepen
AU - Angela Dispenzieri
AU - Mazen Hanna
AU - Martin Carlsson
AU - Pablo Garcia-Pavia
PY - 2026
DA - 2026/08/01
JO - Cardiology and Therapy
DO - 10.1007/s40119-026-00464-6
UR - https://doi.org/10.1007/s40119-026-00464-6
AB - Introduction Tafamidis is approved for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) and has shown a survival benefit in the phase 3 study ATTR-ACT and in real-world settings. This analysis compared survival in a contemporary, matched, real-world cohort of patients with ATTR-CM treated with tafamidis vs untreated. Methods Data were pooled from an early access cohort (NCT02791230) and from patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) in 2019–2023 with a mixed or predominantly cardiac phenotype. Propensity score matching (1:1) was performed to match treated and untreated patients in the pooled sample based on baseline age, New York Heart Association class, and phenotype. Treated patients received tafamidis at the approved dose for ATTR-CM. Results A total of 283 treated and 283 untreated patients were included. Of the 283 treated patients, 85 were from the early access cohort and 198 from THAOS. All untreated patients were from THAOS. In treated vs untreated patients, mean age at enrollment was 76.9 vs 77.4 years, 89.0% vs 83.4% were male, 11.0% vs 16.6% were female, and 80.9% vs 73.1% had wild-type ATTR-CM. Estimated adjusted survival probabilities for all-cause mortality for treated vs untreated patients were 90.2% vs 76.0% at 30 months and 85.3% vs 67.5% at 42 months. Treated patients had a 74% lower risk of all-cause mortality compared with untreated patients (hazard ratio 0.26; 95% confidence interval 0.13–0.51). No new safety signals were identified. Conclusions This matched-cohort analysis demonstrated improved survival in a contemporary, real-world cohort of patients with ATTR-CM receiving the approved dose of tafamidis compared with those untreated. These findings further extend the results of ATTR-ACT to contemporary populations of patients with ATTR-CM treated clinically with tafamidis and reinforce earlier real-world evidence supporting tafamidis use. Trial Registration Clinicaltrials.gov identifiers NCT00628745 and NCT02791230.
SN - 2193-8261
SN - 2193-6544
ER -