Survival in a Real-World Cohort of Patients With Transthyretin Amyloid Cardiomyopathy Treated With Tafamidis: An Analysis From the Transthyretin Amyloidosis Outcomes Survey (THAOS)

Pablo Garcia-Pavia; Arnt V Kristen; Brian Drachman; Martin Carlsson; Leslie Amass; Franca Stedile Angeli; Mathew S Maurer; THAOS investigators

doi:10.1016/j.cardfail.2024.06.003

Survival in a Real-World Cohort of Patients With Transthyretin Amyloid Cardiomyopathy Treated With Tafamidis: An Analysis From the Transthyretin Amyloidosis Outcomes Survey (THAOS)

J Card Fail. 2024 Jun 21:S1071-9164(24)00222-7. doi: 10.1016/j.cardfail.2024.06.003. Online ahead of print.

Authors

Pablo Garcia-Pavia¹, Arnt V Kristen², Brian Drachman³, Martin Carlsson⁴, Leslie Amass⁴, Franca Stedile Angeli⁴, Mathew S Maurer⁵; THAOS investigators

Affiliations

¹ Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, Madrid, Spain; Universidad Francisco de Vitoria, Pozuelo de Alarcon, Spain; Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain. Electronic address: pablogpavia@yahoo.es.
² Department of Cardiology, Angiology, and Respiratory Medicine, Medical University of Heidelberg, Heidelberg, Germany.
³ University of Pennsylvania Health System, Philadelphia, PA, USA.
⁴ Pfizer Inc, New York, NY.
⁵ Columbia University College of Physicians and Surgeons, New York, NY, USA.

PMID: 38909877
DOI: 10.1016/j.cardfail.2024.06.003

Abstract

Background: In the pivotal Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), tafamidis significantly reduced mortality rates, leading to its approval in many countries for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Real-world evidence on survival in patients with ATTR-CM following tafamidis treatment has not been extensively reported.

Methods and results: The Transthyretin Amyloidosis Outcomes Survey (THAOS) was a longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis and asymptomatic participants carrying pathogenic transthyretin variants. Patients from THAOS with a predominantly cardiac phenotype at enrollment were included, and survival was analyzed according to tafamidis treatment status (treated or untreated). Results are based on the completed THAOS dataset. In tafamidis-treated (n = 587) and tafamidis-untreated (n = 854) patients, respectively, median age at enrollment was 77.7 and 76.4 years, 91.8% and 90.0% were male, and 91.8% and 83.8% had wild-type disease. Survival rates (95% CI) at 30 and 42 months, respectively, were 84.4% (80.5-87.7) and 76.8% (70.9-81.7) in tafamidis-treated patients, and 70.0% (66.4-73.2) and 59.3% (55.2-63.0) in tafamidis-untreated patients. Survival rates in genotype subgroups (wild-type and variant) were similar to those of the overall cohort. Survival rates were better in a contemporary cohort, as reflected by a sensitivity analysis performed in patients enrolled after vs before 2019. No new safety signals were identified.

Conclusions: In this real-world cohort of patients with ATTR-CM, survival rates were higher than in ATTR-ACT and consistent with more recent reports, suggesting early diagnosis and treatment with tafamidis has improved life expectancy in ATTR-CM. These results provide further evidence supporting tafamidis' safety and effectiveness.

Trial registration: ClinicalTrials.gov identifier: NCT00628745.

Keywords: Transthyretin amyloid cardiomyopathy; real-world; survival; tafamidis.

Associated data

ClinicalTrials.gov/NCT00628745