Cardiac disease monitoring measures in patients with transthyretin amyloid cardiomyopathy treated with tafamidis

作者：Dimitrios Bampatsias, Abdirahman Wardhere, Lawrence Zeldin, Alfonsina Mirabal-Santos, Ariel Weinsaft, Juliana C Levy, Dia A. Smiley, Sergio Luis Teruya, Mathew S. Maurer · 发表于：Heart · 年份：2025 · DOI：10.1136/heartjnl-2024-324826 · 被引用次数：8 · 研究领域：Amyloidosis: Diagnosis, Treatment, Outcomes、Parathyroid Disorders and Treatments、Coagulation, Bradykinin, Polyphosphates, and Angioedema

BACKGROUND: Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) often experience worsening cardiac disease (WCD) despite disease-modifying treatment. However, a strict definition is lacking. Recent studies have shown that N-terminal pro-B-natriuretic peptide (NT-proBNP increase), or intensification of oral diuretics, is associated with increased mortality risk. AIM: To describe the pattern of WCD at 1 year in patients with ATTR-CM on tafamidis and explore the association with mortality and cardiovascular (CV) hospitalisation. METHODS: Patients diagnosed with ATTR-CM at Columbia University after 2018 who were treated with tafamidis were enrolled in the study. WCD was defined as: increase in NT-proBNP >700 pg/mL and >30% from baseline or oral diuretic intensification (ODI) or both. Survival and CV hospitalisation risk analysis was performed using Kaplan-Meier curves, Cox regression and competing risk regression adjusted for age, genotype and disease severity by the National Amyloidosis Centre (NAC) stage. RESULTS: A total of 238 patients were enrolled, 100 (42%) of whom showed WCD at 1 year. WCD was associated with increased mortality risk (log rank, p<0.0001, HR=1.91 (1.10-3.32), p=0.023, after adjustment for age, genotype and baseline NAC stage). In competing risk analysis, WCD was associated with increased risk for CV-related hospitalisation (HR=1.8 (1.17-2.99, p=0.009)) after adjustment for age, genotype and baseline NAC stage. CONCLUSIONS: NT-proBNP increase and O...