Real-World Clinical Impact of Tafamidis on Transthyretin Amyloid Cardiomyopathy - A Decade of Experience From a Community-Based Hospital (2015-2024).

Abstract

Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is increasingly recognized as a major cause of heart failure in elderly patients with left ventricular hypertrophy. Although tafamidis was approved in 2019 following the ATTR-ACT study, its real-world survival impact in community settings remains unclear.

Methods and results: This retrospective study analyzed 117 patients diagnosed with ATTR-CM at a single center from 2015 to 2024, with 75 receiving tafamidis and 42 untreated. Among the 83 patients who underwent genetic testing, all had the wild-type genotype. ATTR-CM diagnoses increased significantly after the advent of ^{99 m}Tc-pyrophosphate scintigraphy and tafamidis. Kaplan-Meier analysis showed significantly longer survival in tafamidis-treated patients. Multivariate analysis identified New York Heart Association (NYHA) functional class, left ventricular wall thickness, N-terminal prohormone of brain natriuretic peptide (NT-proBNP) levels, and tafamidis treatment as independent survival predictors. Tafamidis treatment was associated with significantly improved survival in patients who were younger, had a less advanced NYHA functional class, and lower levels of NT-proBNP and troponin T. In contrast, its survival benefits were marginal in patients with older age, higher NYHA functional class, elevated NT-proBNP levels, and increased troponin T levels.

Conclusions: In this real-world cohort, tafamidis treatment was significantly associated with better survival in ATTR-CM patients, particularly when initiated in the early stage. Therefore, early detection and timely initiation of treatment are critical for optimizing clinical outcomes in this increasingly recognized condition.