Phase 3 prospective, randomized, controlled, open-label, multicenter, crossover study of recombinant ADAMTS13 in patients with congenital thrombotic thrombocytopenic purpura
Marie Scully, MBE, M.D.
University College London Hospitals
NHS Foundation Trust
Congenital thrombotic thrombocytopenic purpura (TTP) is caused by inherited ADAMTS13 deficiency and can be potentially life-threatening. Scully, M.D., presents results from phase 3 prospective, randomized, controlled, open-label, multicenter, crossover study evaluating the efficacy/safety of rADAMTS13 prophylaxis. rADAMTS13 prophylaxis lead to no acute TTP events, and non-acute TTP manifestations were lower in comparison to the standard of care, thus making rADAMTS13 an innovative therapeutic approach for cTTP.