Cardiology MCQ – Treatment of transthyretin amyloid cardiomyopathy – Answer
Recently developed pharmacotherapy which has been shown to halt and reverse the progression of transthyretin amyloid cardiomyopathy:
Correct answer: 2. Tafamidis
Transthyretin amyloid cardiomyopathy is due to transthyretin amyloid fibril deposition in the myocardium. Tafamidis acts by binding to transthyretin and prevents tetramer dissociation, thus preventing amyloid formation.
Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016.