Marie Scully, Ana Antun, Spero R Cataland, Paul Coppo, Claire Dossier, Nathalie Biebuyck, Wolf-Achim Hassenpflug, Karim Kentouche, Paul Knöbl, Johanna A Kremer Hovinga, M Fernanda López-Fernández, Masanori Matsumoto, Thomas L Ortel, Jerzy Windyga, Indranil Bhattacharya, Michael Cronin, Hong Li, Björn Mellgård, Munjal Patel, Parth Patwari, Shan Xiao, Pinghai Zhang, Linda T Wang
BACKGROUND: Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived products) administered as routine prophylaxis or on-demand treatment in patients with congenital TTP is not known. METHODS: In this phase 3, open-label, crossover trial, we randomly assigned patients in a 1:1 ratio to two 6-month periods of prophylaxis with recombinant ADAMTS13 (40 IU per kilogram of body weight, administered intravenously) or standard therapy, followed by the alternate treatment; thereafter, all the patients received recombinant ADAMTS13 for an additional 6 months...
May 2, 2024: New England Journal of Medicine