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

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

SUBMITTER: Sultan MB 

PROVIDER: S-EPMC5606341 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature