Tafamidis Therapy in Transthyretin Cardiac Amyloidosis: Insights from a Single Center

Authors : Selda Murat, Emre Yalvaç, Rabia Okumus, Yüksel Çavuşoğlu

Pages : 94-102

Doi:10.20515/otd.1764273

View : 44 | Download : 94

Publication Date : 2025-12-15

Article Type : Research Paper

Abstract :Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening disease characterized by extracellular deposition of amyloid fibrils. Tafamidis is currently the only approved pharmacologic therapy for ATTR-CM, with proven efficacy in clinical trials. However, real-world data, particularly from Türkiye, remain limited. This study aimed to assess the clinical characteristics, treatment responses, and outcomes of patients with ATTR-CM receiving tafamidis in real-world single-center Turkish patients. This was a retrospective, single center, observational study including 13 patients diagnosed with ATTR-CM. Demographic, clinical, echocardiographic, laboratory, and follow-up data were collected. Tafamidis usage, tolerance, and long-term outcomes including mortality, heart failure hospitalization, and biomarker trends were analyzed. Among 13 patients (mean age: 71.4 ±12.2 years; 53.8% male), 8 had ATTRwt and 5 had ATTRv. At baseline, 69.2% were NYHA Class III. The mean treatment duration was 21.6 ±10.4 months. At one year, 92.3% of patients remained on therapy; by year three, five patients had died (38.5%), mostly due to heart failure. Patients who died were older (79 ±6.4 vs. 66.6 ±12.0 years, p=0.035), had worse baseline NYHA class (p=0.033), higher sPAP (p=0.045), and reduced LA reservoir strain (p=0.046). NT-proBNP decreased in 23.1% of patients during the first year, predominantly in those with ATTRv. Tafamidis was generally well tolerated. This single-center real-world study from Türkiye offers valuable insights into tafamidis use in ATTR-CM, highlighting the prognostic relevance of early diagnosis and baseline functional status, and supporting its feasibility, tolerability, and potential clinical benefit in routine care despite the small sample size.
Keywords : Cardiac amyloidosis, real-world data, tafamidis, transthyretin, treatment

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