Monitoring Disease Progression in Transthyretin Amyloid Cardiomyopathy.

Transthyretin amyloid cardiomyopathy is a progressive and fatal cardiomyopathy caused by the deposition of misfolded transthyretin in the form of amyloid fibrils in the myocardium. The advent of various highly efficacious transthyretin amyloid-specific disease-modifying therapies has sparked a growing interest in identifying the clinical indicators of disease progression that will be crucial in guiding treatment decisions. Markers of disease progression include changes in commonly measured biomarkers such as the N-terminal pro-B-type natriuretic peptide and the estimated glomerular filtration rate, a decline in the 6-m inute walk test distance, outpatient diuretic intensification, changes in heart failure symptom burden and also changes in various cardiac-imaging parameters. Considering the wide array of markers that can detect disease progression, it is likely that a comprehensive clinical assessment will involve monitoring multiple markers simultaneously. Integrating multiple markers of disease progression offers additional insights beyond individual markers, enabling a refined assessment of disease trajectory and mortality risk. Many of these markers are readily available, simple to measure and universally applicable, making them easy to implement in clinical practice for identifying patients with advancing disease and a heightened risk of mortality.