Survival in a Real-World Cohort of Patients With Transthyretin Amyloid Cardiomyopathy Treated With Tafamidis: An Analysis From the Transthyretin Amyloidosis Outcomes Survey (THAOS)
- PMID: 38909877
- 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)
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.
Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.
Conflict of interest statement
Disclosures PG-P served as a speaker in scientific meetings for Alnylam, BridgeBio, Ionis, Intellia, AstraZeneca, Novo Nordisk, and Pfizer, received funding from Alnylam and Pfizer for scientific meeting expenses, received consultancy fees from Alnylam, Attralus, BridgeBio, Neuroimmune, AstraZeneca, Novo Nordisk, Alexion, Intellia, and Pfizer, and his institution received research grants/educational support from Alnylam, BridgeBio, AstraZeneca, Novo Nordisk, Intellia, and Pfizer. AVK received research support from and attended advisory boards for Pfizer, Neurimmune, Alnylam, Intellia, Ionis, Akcea, Novo Nordisk, Astra Zeneca, and Alexion. BD received consultancy fees from Alnylam and Eidos. MC, LA and FSA are full-time employees of Pfizer and hold stock and/or stock options in Pfizer. MSM received grant support from NIH R01HL139671 and grants from Pfizer during the conduct of the study and grants and personal fees from Alnylam, Pfizer, BridgeBio, Prothena, and Ionis and personal fees from Astra Zeneca, Ionis, Intellia, and Novo Nordisk.
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