Long-Term Outcomes of Tafamidis Treatment for Transthyretin Amyloid Cardiomyopathy in Two Patients

Abstract

Tafamidis is the only currently available disease-modifying agent for transthyretin amyloid cardiomyopathy (ATTR-CM); however, reports on its long-term efficacy, safety, and longitudinal cardiac parameter outcomes are lacking. Herein, we present 2 cases of wild-type ATTR-CM receiving tafamidis for 9 years, in which serial long-term follow-up data were obtained. In both cases, tafamidis treatment was continued without any adverse effects, and no hospitalization due to heart failure occurred. Notably, longitudinal observation revealed sustained inhibition of deterioration in cardiac biomarkers, left ventricular function, and hypertrophy during approximately a decade of tafamidis treatment. This case series suggests that tafamidis has the potential to sustainably delay progression of ATTR-CM.