Recombinant ADAMTS13 in congenital thrombotic thrombocytopenic purpura: final analysis from a randomized phase 3 trial

The authors aimed to evaluate the efficacy and safety of recombinant ADAMTS13 (rADAMTS13) compared to plasma-based therapy (PBT) in preventing acute and subacute events in patients with congenital thrombotic thrombocytopenic purpura (TTP). The final analysis of a phase 3 trial demonstrated that rADAMTS13 prophylaxis significantly reduced the occurrence of acute TTP events and treatment-related adverse events, while also enhancing patient satisfaction and maintaining higher ADAMTS13 activity levels compared to PBT. These findings support the clinical benefit of rADAMTS13 as a prophylactic treatment for congenital TTP.