Tafamidis for the treatment of wild-type transthyretin cardiac amyloidosis: insights from a regional hospital experience
A Martins, A Vazao, J Pereira, M Amado, C Esteves, C Ruivo, C Domingues, D DuraoAbstract
Introduction
Wild-type transthyretin cardiac amyloidosis (wtATTR-CM) is an increasingly recognized cause of heart failure (HF). Tafamidis is the first approved disease-modifying therapy and has been shown to reduce cardiovascular hospitalizations and improve survival in selected populations.
Objectives
To evaluate the impact of tafamidis on clinical outcomes in patients with wtATTR-CM followed at a Cardiomyopathy Clinic in a regional hospital in Portugal.
Methods
Retrospective single-center study of patients diagnosed with wtATTR-CM according to the ESC algorithm between 2018 and 2024. Data on tafamidis use, as well as clinical, laboratory, echocardiographic, and electrocardiographic parameters, were collected at the time of diagnosis (Table 1). Management strategies comprised HF therapy tailored to ATTR-CM, along with disease-modifying therapy with tafamidis 61 mg. Tafamidis was considered in patients meeting local protocol criteria, including NYHA functional class I–II and absence of exclusion criteria (e.g., estimated glomerular filtration rate [eGFR] <25 mL/min or NT-proBNP <135 pg/mL). The primary endpoint, a composite of cardiovascular hospitalization or all-cause mortality, was assessed 18 months after diagnosis. Patients who achieved the primary endpoint (Group 1) were compared with those who did not (Group 2).
Results
A total of 70 patients were included; 94% were male, with a median age of 81 (IQR 7) years. Baseline left ventricular ejection fraction (LVEF) was 55% (IQR 12). Tafamidis was prescribed in 15 patients (21%), and 34 patients (49%) achieved the primary endpoint. Baseline characteristics, including gender, hypertension, dyslipidemia, diabetes, conduction disturbances, and echocardiographic parameters, were similar between groups. Likewise, no differences were observed for guideline-directed therapies, including beta-blockers, renin–angiotensin system inhibitors (RASI), and sodium-glucose co-transporter 2 inhibitors. Patients who achieved the primary endpoint were less likely to receive tafamidis (9 vs 33%, p=0.019), and more likely to have atrial fibrillation (AF, 82 vs 47%, p=0.002) and chronic kidney disease (CKD, 79 vs 28%, p<0.001). They also had higher NT-proBNP levels (4705.0 [IQR 5225.0] vs 2060.0 pg/mL [IQR 3250.0], p<0.001) and lower eGFR (46 [IQR 24] vs 67 [IQR 31], p=0.002). In multivariate regression analysis, tafamidis use was independently associated with a reduced risk of the primary endpoint, whereas AF and CKD were independent predictors of adverse outcomes. Kaplan–Meier survival curves confirmed a higher event rate among patients not receiving tafamidis (log-rank p = 0.017).
Conclusion
In this real-world cohort of wtATTR-CM patients, tafamidis was associated with a reduced risk of the combined endpoint of HF hospitalization and all-cause mortality. Additionally, AF and CKD were independent predictors of adverse outcomes.Table/Figure 1For image description, please refer to the figure legend and surrounding text.