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. 2024 Jan;12(1):150-160.
doi: 10.1016/j.jchf.2023.08.032. Epub 2023 Nov 8.

Tafamidis Efficacy Among Octogenarian Patients in the Phase 3 ATTR-ACT and Ongoing Long-Term Extension Study

Pablo Garcia-Pavia  1 , Marla B Sultan  2 , Balarama Gundapaneni  3 , Yoshiki Sekijima  4 , Federico Perfetto  5 , Mazen Hanna  6 , Ronald Witteles  7
Affiliations
Free article

Tafamidis Efficacy Among Octogenarian Patients in the Phase 3 ATTR-ACT and Ongoing Long-Term Extension Study

Pablo Garcia-Pavia et al. JACC Heart Fail. 2024 Jan.
Free article

Erratum in

  • Correction.
    [No authors listed] [No authors listed] JACC Heart Fail. 2024 Jul;12(7):1316. doi: 10.1016/j.jchf.2024.05.015. JACC Heart Fail. 2024. PMID: 38960528 No abstract available.

Abstract

Background: Tafamidis was approved to treat patients with transthyretin amyloid cardiomyopathy (ATTR-CM) on the basis of findings from the phase 3 Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT).

Objectives: This study was a post hoc analysis exploring tafamidis efficacy in octogenarian patients.

Methods: Analysis of patients aged <80 and ≥80 years in ATTR-ACT and its ongoing open-label long-term extension (LTE) study, where all patients receive tafamidis.

Results: After 30 months in ATTR-ACT, least squares (LS) mean change from baseline in 6-minute walk test (6MWT) distance, N-terminal pro-B-type natriuretic peptide (NT-proBNP) concentration, and Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score were smaller (all P < 0.05) in patients aged ≥80 years treated with tafamidis (n = 51) vs placebo (n = 37). At the LTE study interim analysis, patients aged ≥80 years treated continuously with tafamidis had a smaller decline in KCCQ-OS score (P < 0.05) and trended toward longer median survival (45 vs 27 months; all-cause mortality HR: 0.6828 [95% CI: 0.4048-1.1517]; P = 0.1526) than those initially treated with placebo in ATTR-ACT. Similar efficacy was observed in patients aged <80 years in ATTR-ACT, including smaller LS mean change from baseline in 6MWT distance, NT-proBNP concentration, and KCCQ-OS score, and lower rate of cardiovascular-related hospitalizations with tafamidis (n = 125) vs placebo (n = 140). In the LTE study, patients aged <80 years treated continuously with tafamidis had a longer median survival (80 vs 41 months; HR = 0.4513 [95% CI: 0.3176-0.6413]; P < 0.0001) and a smaller decline in KCCQ-OS score than those initially treated with placebo.

Conclusions: The findings demonstrate tafamidis efficacy for patients with ATTR-CM both in those aged <80 and those aged ≥80 years. (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial [ATTR-ACT]; NCT01994889/Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy; NCT02791230).

Keywords: age; amyloidosis; elderly; heart failure with preserved ejection fraction; survival.

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Conflict of interest statement

Funding Support and Author Disclosures This study was sponsored by Pfizer. Pfizer contributed to the design and conduct of the study and management and collection of data. In their role as authors, employees of Pfizer were involved in the analysis and interpretation of data, preparation, review, and approval of the manuscript and the decision to submit for publication, along with their co-authors. The study sponsor approved the manuscript from an intellectual property perspective but had no right to veto the publication. Dr Garcia-Pavia has served as a speaker in scientific meetings for Alexion, Alnylam, BridgeBio, Ionis, AstraZeneca, Novo Nordisk, and Pfizer; has received funding from Alnylam and Pfizer for scientific meeting expenses; has received consultancy fees from Alnylam, Attralus, BridgeBio, Neuroimmune, AstraZeneca, Novo Nordisk, Alexion, Intellia, and Pfizer; and his institution has received research grants/educational support from Alnylam, AstraZeneca, BridgeBio, Intellia, and Pfizer. Dr Sultan and Mr Gundapaneni are full-time employees of Pfizer and hold stock/stock options. Dr Sekijima has a patent concerning tafamidis; has received honoraria for lectures and advisory board participation from Pfizer and Alnylam; and his institution has received research grants from Pfizer and Alnylam. Dr Perfetto has received honoraria for advisory board participation from Pfizer, Alnylam, and Akcea. Dr Hanna has received honoraria for advisory board participation from Pfizer, Alnylam, Akcea, Alexion, and Eidos; and has served as a speaker for a scientific meeting session funded by Alnylam. Dr Witteles has received honoraria for advisory board participation from Pfizer, Alnylam, Ionis, AstraZeneca, Janssen, Intellia, BridgeBio, Novo Nordisk, and Alexion.

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