Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura

This phase 3, open label, crossover trial evaluated the efficacy and safety of recombinant ADAMTS13 (rADAMTS13) versus standard plasma based therapies in patients with congenital TTP. Among 48 randomized patients, no acute TTP events occurred during rADAMTS13 prophylaxis, while one event occurred with standard therapy. rADAMTS13 achieved near-normal ADAMTS13 activity levels (~101%), reduced TTP manifestations (especially thrombocytopenia), and showed a favorable safety profile with fewer treatment-related adverse events and no development of neutralizing antibodies. Pharmacokinetic data confirmed higher and more sustained ADAMTS13 activity with rADAMTS13 compared to plasma derived prADAMTS13 as a promising prophylactic treatment for congenital TTP.  

This phase 3, open label, crossover trial evaluated the efficacy and safety of recombinant ADAMTS13 (rADAMTS13) versus standard plasma based therapies in patients with congenital TTP. Among 48 randomized patients, no acute TTP events occurred during rADAMTS13 prophylaxis, while one event occurred with standard therapy. rADAMTS13 achieved near-normal ADAMTS13 activity levels (~101%), reduced TTP manifestations (especially thrombocytopenia), and showed a favorable safety profile with fewer treatment-related adverse events and no development of neutralizing antibodies. Pharmacokinetic data confirmed higher and more sustained ADAMTS13 activity with rADAMTS13 compared to plasma derived prADAMTS13 as a promising prophylactic treatment for congenital TTP.