Recombinant ADAMT S-13 as a new alternative for the patients with congenital thrombocytopenic purpura

Congenital thrombocytopenic purpura (cTTP), also known as Upshaw–Schulman syndrome is a rare disease, caused by ADAMTS13 deficiency. This condition may result in various complications, including haemolytic anaemia, renal dysfunction or thrombotic events. To this very day, the most common treatment of this condition is transfusion of fresh frozen plasma. Recently, recombinant ADAMTS13 (rADAMTS13) has been introduced, representing a revolutionary advancement in the treatment of cTTP, and giving patients new, more comfortable alternative. We present here a case report of a patient with congenital thrombocytopenic purpura, who after 30 years of being treated with FFP transfusions and developing complications from both the disease and its treatment, was successfully transitioned to rADAMTS13 therapy.