Longitudinal Changes in Multiple Cardiac Biomarkers in Transthyretin Amyloidosis Cardiomyopathy Patients Treated Vs Untreated with Tafamidis.

Overview

abstract

BACKGROUND: Tafamidis is an oral transthyretin stabilizer that improves survival in transthyretin amyloidosis cardiomyopathy (ATTR-CM), but only limited real-world data describe serial cardiac biomarker changes following treatment initiation. The primary objective of this study was to characterize longitudinal changes across multiple cardiac biomarker domains in tafamidis-treated ATTR-CM patients, to describe how these parameters evolve over time in routine clinical practice. We also report the same outcomes in untreated patients to reflect the natural disease history in a modern real-world cohort. METHODS: Clinical, biochemical, and cardiac imaging parameters were serially assessed at baseline and 1-year follow-up for 145 ATTR-CM patients, both those treated and those untreated with tafamidis. RESULTS: The median age was 80 years (range: 73-86), and 80 patients (55%) received tafamidis. At baseline, the treated group was younger and exhibited less advanced disease, relative to the untreated group. Treatment with tafamadis was associated with stabilization in N-terminal pro-B-type natriuretic peptide (NTproBNP) level, troponin-T level, and New York Heart Association functional class at 1-year follow-up, whereas the untreated group demonstrated worsening (all comparisons P < 0.05). Tafamidis treatment status was not significantly associated with National Amyloidosis Center or Mayo Clinic disease stage. CONCLUSIONS: NTproBNP level, troponin-T level, and New York Heart Association functional class remain stable over 1 year in a real-world cohort of tafamidis-treated ATTR-CM patients. These results may help inform therapeutic monitoring strategies in clinical practice.