Abstract
Background/Objectives: Cardiac amyloidosis (CA) is a rare and severe multisystem disorder, associated with an average survival of approximately five years. Recently, Tafamidis has emerged as a promising treatment for transthyretin-related CA. This retrospective study aimed to assess disease progression through echocardiographic findings in patients with transthyretin-related CA, with a specific focus on evaluating the impact of Tafamidis in a cohort managed at our Cardiomyopathy Clinic. Methods: A total of 39 patients were included, of whom 28 received Tafamidis treatment, while 11 did not. Clinical, electrocardiographic, echocardiographic, biological, and other imaging data were collected at diagnosis. Comprehensive echocardiographic data were collected every six months over a two-year period (2021-2023). Results: At 1-year follow-up, the Tafamidis-treated cohort demonstrated stable global systolic and diastolic function. Left ventricular (LV) global longitudinal strain (GLS) and global work index (GWI) showed minimal change (GLS -12.9% (-15.6; -10.7) vs. -13.0% (-14.0; -10.7), p = 0.054; GWI 1113 mmHg/% (963; 1301) vs. 1208 mmHg/% (850; 1420), p = 0.054), and there was no significant increase in indexed LV mass (135.0 g/m(2) (118.0; 167.0) vs. 148.0 (128.0; 173.0), p = 0.25). Similarly, valvular heart disease severity remained unchanged. Longitudinal analysis using generalized linear mixed models further confirmed the stability of echocardiographic parameters over the 2-year follow-up period. Systolic function metrics, including LV ejection fraction (slope: -0.0098 ± 0.011, p = 0.38) and GLS (slope: 0.0036 ± 0.0041, p = 0.39) showed no significant decline. Diastolic function assessed through E/A ratio (slope: -0.0007 ± 0.0013, p = 0.59) and E/e' (slope: -0.0042 ± 0.0073, p = 0.57) also remained stable. Indexed LV mass exhibited no significant progression (slope: 0.050 ± 0.061, p = 0.41). These findings were consistent across the various subgroups. Conclusions: Tafamidis appears to effectively stabilize transthyretin-related CA, limiting disease progression over the follow-up period.