Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS.

ABSTRACT:

Introduction

Tafamidis is approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). Many patients with ATTR-CM present with a mixed phenotype of both cardiac and neurologic symptoms, but real-world effectiveness studies of tafamidis in this population are lacking. This study assessed survival and other outcomes in a real-world, contemporary cohort of tafamidis-treated and untreated patients with mixed-phenotype ATTR-CM.

Methods

The Transthyretin Amyloidosis Outcomes Survey (THAOS) was a longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis and asymptomatic carriers of pathogenic transthyretin gene variants and was completed in June 2023. This analysis included a contemporary cohort of patients enrolled in THAOS in 2019-2023 who were ch

SUBMITTER: Wixner J 

PROVIDER: S-EPMC12680828 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature