Effect of Tafamidis on Clinical and Functional Parameters in Transthyretin Amyloid Cardiomyopathy

Transthyretin amyloid cardiomyopathy (ATTR-CM) has recently gained recognition as a rare and fatal disease. Tafamidis, a first-in-class transthyretin stabilizer, has emerged as a promising agent for attenuating disease progression. Nevertheless, how tafamidis alters clinical and imaging parameters remains unclear.

This systemic review and meta-analysis aimed to investigate how tafamidis remodels the myocardium and influences the disease trajectory of ATTR-CM.

PubMed, EMBASE, and the Cochrane Library were searched for literature from inception to February 2024 which reported either the effects of tafamidis treatment or natural course of ATTR-CM. Outcomes of interests were all clinical and imaging parameters available from at least 2 independent studies.

We identified 30 studies comprising 2,973 participants with ATTR-CM. Pooling all studies with outcomes of both tafamidis and placebo, tafamidis significantly reduced all-cause mortality (OR: 0.19; 95% CI: 0.07 to 0.56) and cardiovascular death (OR: 0.08; 95% CI: 0.02-0.30). Tafamidis also ameliorated the deterioration of 6-minute walk distance (standardized mean difference [SMD] 0.04 vs. -0.29, P = 0.002) and serum N-terminal pro-B-type natriuretic peptide level (SMD: -0.03 vs 0.41, P < 0.001). Regarding imaging parameters, better global longitudinal strain on echocardiography (SMD: 0.06 vs 0.50, P = 0.003), heart to contralateral ratio (SMD: -0.23 vs. -1.17, P = 0.037) on technetium-99m pyrophosphate scintigraphy, extracellular volume (P = 0.003), left (P < 0.001) and right (P = 0.001) ventricular ejection fraction, and right atrium area (P = 0.033) on cardiac magnetic resonance imaging were observed after tafamidis treatment.

Tafamidis improves clinical outcomes and limits the progression of cardiac remodeling in ATTR-CM.