TAFAMIDIS, a promising agent reduces mortality and slows cardiac deterioration in transthyretin amyloid cardiomyopathy (ATTR-CM), enhancing clinical and imaging outcomes.
ATTR-CM, a rare and often fatal condition, has gained increasing attention due to advances in understanding and treatment. Tafamidis, a transthyretin stabiliser, is a novel therapy that shows promise in mitigating disease progression. This review synthesises data to evaluate its effects on clinical and imaging parameters, aiming to clarify how tafamidis influences the disease trajectory of ATTR-CM.
A systematic review and meta-analysis of 30 studies, encompassing 2,973 participants, assessed outcomes in tafamidis-treated versus untreated (placebo or natural progression) patients. Tafamidis significantly reduced all-cause mortality (odds ratio [OR]: 0.19; 95% CI: 0.07–0.56) and cardiovascular death (OR: 0.08; 95% CI: 0.02–0.30). Functional parameters, including the six-minute walk distance, showed improved stability (standardised mean difference [SMD]: 0.04 versus −0.29, P=0.002). Biomarker trends favoured tafamidis, with serum NT-proBNP levels declining (standardised mean difference: −0.03 versus 0.41, P<0.001). Imaging revealed improved global longitudinal strain (SMD: 0.06 versus 0.50, P=0.003), heart-to-contralateral ratios on scintigraphy (SMD: −0.23 versus −1.17, P=0.037), and ventricular ejection fractions (P<0.001).
Tafamidis not only improves survival but also mitigates cardiac structural changes and functional declines, underscoring its value in clinical practice. As ATTR-CM awareness grows, this study highlights tafamidis as a cornerstone treatment for altering the disease trajectory.
Katheeja Imani, EMJ
Reference
Kao TW et al. Effect of tafamidis on clinical and functional parameters in transthyretin amyloid cardiomyopathy. JACC: Advances. 2025;4(2):101511.
