Multicenter study of patients with transthyretin cardiac amyloidosis in Poland: Clinical characteristics, genotyping, electrocardiographic features, outcome predictors, and tafamidis treatment outcomes.

BACKGROUND

We present the first multicenter, non-interventional, longitudinal observational study of patients diagnosed with cardiac transthyretin amyloidosis (ATTR-CA) in Poland.

AIMS: The aim of this study was to describe the clinical characteristics, survival rates, and treatment outcomes of patients with ATTR-CA in Poland, and to identify potential markers linked to an increased risk of disease progression.

METHODS

Between 2017 and 2025, consecutive patients with confirmed ATTR-CA were evaluated at 4 cardiology centers. Assessments included blood tests, standard 12-lead electrocardiography, and transthoracic echocardiography. The primary endpoint was defined as death or heart transplantation.

RESULTS

ATTR-CA was confirmed in 115 patients, including 21 (18.2%) with hereditary disease. The median follow-up was 24 (12-41) months for the entire cohort and 25 (14.2-46.7) months for surviving patients (n = 78). The primary endpoint occurred in 37 (32.2%) patients. In multivariable analysis, the ratio of the transmitral early peak velocity (E) estimated by pulsed wave Doppler over the early mitral annulus velocity (e'), body mass index, and levels of high-sensitivity cardiac troponin T and N-terminal pro-B-type natriuretic peptide were independent predictors of the primary endpoint. Tafamidis was received by 81 (70.4%) patients and was associated with significantly lower risk of the primary endpoint (P = 0.005), stroke, or transient ischemic attack (P = 0.047), and a markedly longer median survival (84 months [95% confidence interval, 68-116] vs. 14 months [95% confidence interval, 7-36]; log-rank P <0.0001). In tafamidis-treated patients, N-terminal pro-B-type natriuretic peptide and high-sensitivity cardiac troponin T levels remained stable at 6 and 12 months of follow-up, in contrast to untreated patients, who showed an increase in these biomarkers.

CONCLUSIONS

This study provides the first multicenter data on ATTR-CA in Poland. Our findings enhance understanding of the disease course and treatment effects, underline the importance of disease-modifying therapy in improving outcomes, provide valuable real-world data on tafamidis treatment, and indicate directions for further research.