Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathic disorder (TMA) due to a severe deficiency of ADAMTS13 (A Disintegrin And Metalloprotease with Thrombo-Spondin type 1 repeats, member 13). The deficiency in ADAMTS13 can either be caused by mutations in ADAMTS13 (congenital TTP or Upshaw-Schulman syndrome, cTTP) or by anti-ADAMTS13 autoantibodies (immune-mediated TTP, iTTP). Diagnosis of TTP is challenging but crucial for the survival of the patient. TTP should be suspected when microangiopathic haemolytic anaemia and severe thrombocytopenia are observed. A severely decreased ADAMTS13 activity (activity <10%) should confirm the diagnosis of TTP. Standard treatment of TTP is plasma therapy (plasma exchange for iTTP, while plasma infusion for cTTP), but novel therapeutics like rituximab, caplacizumab and recombinant ADAMTS13 show promising results regarding the recovery and sustained remission of TTP patients. However, although major advances have been made in the management of TTP, TTP is a chronic disease and patients still relapse, careful and stringent patient follow-up is needed to improve the patients’ quality of life.
(BELG J HEMATOL 2020;11(6):253-60)