Effects of tafamidis on exercise capacity, cardiac function and myocardial amyloid deposition in patients with transthyretin amyloid cardiomyopathy

Abstract

Abstract Background Transthyretin amyloid cardiomyopathy (ATTR-CA) is caused by deposition of amyloid fibrils in the myocardium. Tafamidis is a kinetic stabilizer of TTR that prevents tetramer dissociation and amyloidogenesis. Methods Thirty-eight patients with diagnosis of ATTR-CA were treated with tafamidis (20mg or 61mg) for a period of six months. In our explorative analysis we aimed to evaluate the effects of tafamdis by changes from baseline of the serum NT-proBNP concentration, 6MWD, as well as cardiac structure and function, compared to untreated amyloidosis patients. Results The analysis showed a significant reduction in the serum NT-proBNP concentration in tafamidis-treated patients compared to untreated patients (median difference, −1042.5 pg/mL, p=0.015). Tafamidis also improved the walking distance at month six, while reduction in untreated patients was observed (mean difference, +29.27 m, p=0.175). Echocardiographic findings revealed a decrease in LV size (mean, −1.56 mm) as well as improvements regarding the LA size (mean difference, −2.51 mm) and the GLS (mean difference, 0.80%) in tafamidis-treated patients. T1 mapping in cardiac MRI showed a decrease in ECV (mean, −1.79%) in patients receiving tafamidis, while an increase in ECV in untreated patients was observed (mean, +0.41%). Conclusion Treatment with tafamidis for a period of six months in patients with ATTR-CA results in a significant improvement in NT-proBNP levels and may have positive effects on exercise capacity, cardiac function and myocardial amyloid deposition compared to untreated amyloidosis patients. Picture 1. Change from baseline. Funding Acknowledgement Type of funding source: None