Recombinant ADAMTS13 in congenital thrombotic thrombocytopenic purpura: final analysis from a randomized phase 3 trial.
Recombinant ADAMTS13 (rADAMTS13) was approved for prophylactic or on-demand ADAMTS13 replacement therapy for congenital thrombotic thrombocytopenic purpura (TTP) based on data from a preplanned interim analysis of a phase 3, open-label, crossover trial (NCT03393975). Here, we report results from the final analysis with...