New Therapy Option for Congenital Thrombotic Thrombocytopenic Purpura

Congenital thrombotic thrombocytopenic purpura (TTP) is caused by inherited deficiency of ADAMTS13, which leads to accumulation of ultra-large von Willebrand multimers. Replenishing ADAMTS-13 with plasma products either as a prophylaxis or on-demand regimen remains the standard of care (SOC).

Researchers report a prespecified interim analysis of an industry-sponsored, multicenter, open-label, phase 3 trial of recombinant ADAMTS13 (rADAMTS13) prophylaxis in children and adults with genetically confirmed congenital TTP. In this randomized, controlled, crossover trial, patients received two 6-month treatments with either rADAMTS-13 infusion (40 IU/kg per week) or standard therapy, followed by 6 months of the alternate treatment, followed by 6 m…