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- Title
Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin Cardiomyopathy.
- Authors
Sultan, Marla B.; Gundapaneni, Balarama; Schumacher, Jennifer; Schwartz, Jeffrey H.
- 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 (P = .0004). Similar results were obtained when limiting the analysis to wild-type patients only, without restricting NYHA functional classification (P = .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.
- Subjects
COMPARATIVE studies; MEDICAL quality control; MORTALITY; CARDIOMYOPATHIES; SCIENTIFIC observation; STATISTICS; DATA analysis; DISEASE progression; CARDIAC amyloidosis; MEMBRANE transport proteins; GENOTYPES
- Publication
Clinical Medicine Insights: Cardiology, 2017, Vol 11, p1
- ISSN
1179-5468
- Publication type
Article
- DOI
10.1177/1179546817730322