Karan Shahi MSc , Robert J.H. Miller MD , Steven Dykstra PhD , Yuanchao Feng PhD , Jonathan G. Howlett MD , Victor Jimenez-Zepeda MD , Jan Veenhuyzen RN, BScN , James A. White MD , Nowell M. Fine MD, SM
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引用次数: 0
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.