Tafamidis to treat patients with transthyretin cardiac amyloidosis (ATTR-CM): real-world data from a tertiary centre

Introduction Tafamidis 61 mg is the first therapy shown to improve survival and reduce cardiovascular hospitalizations in transthyretin cardiac amyloidosis (ATTR-CM). As real-world evidence continues to grow, further characterization of tafamidis use across diverse clinical settings remains important. Accordingly, we evaluated treatment patterns and outcomes in patients managed within a dedicated tertiary-care cardiomyopathy program. Methods We conducted a prospective, single-centre, all-comers study of consecutive patients with symptomatic Heart Failure (HF) due to ATTR-CM, up to November 2025. The diagnosis was performed based on the ESC multi-step algorithm. Management included HF therapy tailored to ATTR-CM (CHAD-STOP) and tafamidis 61 mg for patients who met the local protocol key criteria, including NYHA class I-II and no exclusion criteria [e.g., estimated glomerular filtration rate (GFR) <25 mL/min and/or clinical frailty scale >6]. The primary endpoint was a composite of cardiovascular hospitalization or all-cause death at 3 years. Subgroup analyses were performed according to the National Amyloidosis Centre (NAC) staging system. Results A total of 278 patients with ATTR-CM were included (median age 83 [78-87] years; 82% male; 8% hereditary ATTR-CM). Tafamidis 61mg was initiated in 173 patients (62.2%). Compared with patients on stand-alone HF treatment, those treated with tafamidis were significantly younger (median age: 82 [77-86] vs. 85 [81-89] years; p <0.001) with better functional status (NYHA I-II: 93% vs. 50%; p<0.001) and health status (e.g., Frailty Index >3: 14% vs. 68%; p<0.001), and having fewer comorbidities (e.g., median GFR 37 [31-50] vs. 31 [24-43] mL/min; p = 0.002). During a median follow-up of 1.9 [0.9–3.2] years, 109 patients (39.2%) met the primary endpoint, including 82 deaths. Kaplan–Meier analysis demonstrated an improved event-free survival favouring tafamidis (HR 0.17; 95% CI 0.11–0.25; p<0.001) (figure). In the multivariate model adjusted for age, sex, NYHA, left ventricular ejection fraction, NT-proBNP and creatinine, tafamidis remained an independent predictor of reduced risk for the primary endpoint (HR 0.28; 95% CI 0.18-0.43; p<0.001), driven by a reduction in death and cardiovascular hospitalization. These findings were consistent while assessing the cohort by NAC subgroups. Conclusion In this real-world cohort of patients with symptomatic HF due to ATTR-CM, tafamidis therapy was independently associated with a reduction in cardiovascular hospitalization or all-cause mortality. The magnitude of the benefit was comparable to that observed in the ATTR-ACT, supporting the effectiveness of applying trial-derived eligibility criteria to guide appropriate patient selection in real-world clinical practice.