Tafamidis: A game changer in transthyretin cardiomyopathy? A systematic review and meta-analysis of safety and efficacy

Abstract

Purpose: Tafamidis was FDA-approved for Transthyretin cardiomyopathy (ATTR-CM) due to its demonstrated reduction in mortality and hospitalizations.

Methods: 976 studies from PubMed and Embase were screened. Seven studies were included that compared tafamidis treatment with no tafamidis for ATTR-CM. The mantel-Haenszel method was used for binary outcomes, and Hedges' g was used for continuous outcomes.

Results: Tafamidis was associated with decreased odds of mortality (OR 0.55, 95 % CI 0.42-0.73, I²=41 %, p<0.0001) and reduced CHF exacerbations (OR 0.71, 95 % CI 0.51-0.99, I²= 0 %, p= 0.04). While, CHF related hospitalizations (OR 0.35, 95 % CI 0.07-1.67, I2= 87 %, p= 0.19), atrial arrhythmias (OR 0.98, 95 % CI 0.67-1.42, I²= 0 %, p= 0.9), change in left ventricular ejection fraction (SMD 0.87, 95 % CI -0.37-2.11, I²=98 %, p= 0.17), left ventricular end-diastolic diameter from baseline (SMD -0.12, 95 % CI -0.41-0.18, I²= 0 %, p= 0.4), interventricular septal thickness from baseline (SMD -0.7, 95 % CI -1.57-0.17, I²= 96 %, p= 0.11) were not statistically different for tafamidis compared to no tafamidis for ATTR-CM.

Conclusion: Tafamidis treatment in ATTR-CM is associated with reduced all-cause mortality and a lower incidence of CHF exacerbations. These observations are consistent with the ATTRACT trial, which supports the efficacy of tafamidis in treating ATTR-CM.

Keywords: Amyloidosis; Heart Failure Exacerbation; Mortality; Tafamidis; Transthyretin Cardiomyopathy.