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Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR‐ACT) and long‐term extension study

European Journal of Heart Failure Oct 23, 2020

Damy T, Garcia‐Pavia P, Hanna M, et al. - Since tafamidis represented an effective therapeutic choice for transthyretin amyloid cardiomyopathy (ATTR‐CM) in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR‐ACT), therefore, researchers carried out a dose‐specific evaluation by performing further analysis from ATTR‐ACT. A long‐term extension study (LTE) was also conducted to ascertain the optimal dose. Patients were randomly assigned (2:1:2) to tafamidis 80mg, 20mg, or placebo for 30 months in ATTR‐ACT. In the LTE (with placebo‐treated patients randomized to tafamidis 80mg or 20mg; 2:1), patients finishing ATTR‐ACT could enroll and all were switched to high‐dose tafamidis. Findings showed the effectiveness of tafamidis, both 80mg and 20mg, in decreasing mortality as well as cardiovascular‐related hospitalizations in patients suffering from ATTR‐CM. Tafamidis 80mg was supported as the optimal dose, based on longer‐term survival data and the lack of dose‐related safety concerns.

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