Real-world outcomes of tafamidis in transthyretin amyloid cardiomyopathy

Javier M. Valcuende-Rosique, Cardiology Service, Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain
Pablo Revilla-Martí, Cardiology Service, Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain
Alejandro M. Valcuende-Rosique, Pharmacy Service, Hospital Pare Jofré, Valencia; Department of Pharmacology, Faculty of Medicine, Universidad Católica de Valencia San Vicente Mártir, Valencia. Spain
David Méndez-Portuburu, Cardiology Service, Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain
Luis R. Puglla-Sánchez, Cardiology Service, Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain
José R. Ruiz-Arroyo, Cardiology Service, Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain

Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive infiltrative cardiomyopathy associated with substantial morbidity and mortality. Although tafamidis has shown clinical benefit in randomized trials, real-world data are needed to contextualize outcomes in routine practice. Objective: To describe baseline characteristics, extracardiac diagnostic red flags, and longitudinal outcomes in patients with ATTR-CM treated with tafamidis. Method: This retrospective, longitudinal, single-center study included consecutive patients with ATTR-CM who started tafamidis between July 2019 and April 2025. The primary endpoint was 24-month overall survival estimated with Kaplan-Meier methods. Secondary endpoints included heart failure hospitalization and changes in New York Heart Association (NYHA) functional class. Results: Thirty-eight patients were included; the mean age was 82.1 ± 5.3 years, 92.1% were men, and 97.4% had wild-type disease. At baseline, 94.7% were in NYHA Class I-II, and 92.1% were in Gillmore Stage I-II. Extracardiac diagnostic red flags were frequent, particularly carpal tunnel syndrome (52.6%), which preceded cardiac diagnosis by a median of 7.0 years. During a median follow-up of 18.0 months, 3 patients (7.9%) died; estimated overall survival was 94.1% at 12 and 24 months. Only 2 patients (5.3%) had heart failure hospitalization, and 75.0% remained clinically stable or improved. Conclusion: In routine clinical practice, patients treated with tafamidis, mostly at an early disease stage, showed high short- and mid-term survival, low heart failure hospitalization, and predominant clinical stabilization. Extracardiac diagnostic red flags frequently preceded cardiac diagnosis, supporting earlier recognition.

Keywords: Transthyretin amyloidosis. Cardiac amyloidosis. Tafamidis. Heart failure. Cohort studies.