Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin Cardiomyopathy

Overview

Journal Clin Med Insights Cardiol

Publisher Sage Publications

Specialty Cardiology & Vascular Diseases

Date 2017 Sep 28

PMID 28951660

Citations 9

Authors

Marla B Sultan

Balarama Gundapaneni

Jennifer Schumacher

Jeffrey H Schwartz

Affiliations

Soon will be listed here.

Abstract

Background: Transthyretin cardiomyopathy (TTR-CM) is a progressive, fatal disease caused by the accumulation of misfolded transthyretin (TTR) amyloid fibrils in the heart. Tafamidis is a kinetic stabilizer of TTR that inhibits misfolding and amyloid formation.

Methods: In this post hoc analysis, data from an observational study (Transthyretin Amyloidosis Cardiac Study; n = 29) were compared with an open-label study of tafamidis in patients with TTR-CM (Fx1B-201; n = 35). To ensure comparable baseline disease severity, patients with New York Heart Association (NYHA) functional classification ≥III were excluded in this time-to-mortality analysis.

Results: Patients with either wild-type or Val122Ile genotypes treated with tafamidis have a significantly longer time to death compared with untreated patients ( = .0004). Similar results were obtained when limiting the analysis to wild-type patients only, without restricting NYHA functional classification ( = .0262).

Conclusions: These results support earlier conclusions suggesting that tafamidis slows disease progression compared with no treatment outside of standard of care and warrant further investigation.

Trial Registration: ClinicalTrials.gov, NCT00694161.

Citing Articles

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3-O-Methyltolcapone and Its Lipophilic Analogues Are Potent Inhibitors of Transthyretin Amyloidogenesis with High Permeability and Low Toxicity.

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Tafamidis treatment in patients with transthyretin amyloid cardiomyopathy: a systematic review and meta-analysis.

Wang J, Chen H, Tang Z, Zhang J, Xu Y, Wan K EClinicalMedicine. 2023; 63:102172.

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Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients: an analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and the open-label long-term extension studies.

Rozenbaum M, Garcia A, Grima D, Tran D, Bhambri R, Stewart M Eur Heart J Qual Care Clin Outcomes. 2021; 8(5):529-538.

PMID: 33895806 PMC: 9382662. DOI: 10.1093/ehjqcco/qcab031.

Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story.

Burton A, Castano A, Bruno M, Riley S, Schumacher J, Sultan M Drug Des Devel Ther. 2021; 15:1225-1243.

PMID: 33776421 PMC: 7987260. DOI: 10.2147/DDDT.S289772.

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