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Deprecated: Implicit conversion from float 211.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Clin+Med+Insights+Cardiol 2017 ; 11 (ä): ä Nephropedia Template TP
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Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin Cardiomyopathy #MMPMID28951660
Sultan MB; Gundapaneni B; Schumacher J; Schwartz JH
Clin Med Insights Cardiol 2017[]; 11 (ä): ä PMID28951660show ga
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.