What is the primary treatment for thrombotic thrombocytopenic purpura (TTP)?

Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy characterized by the presence of microangiopathic hemolytic anemia, thrombocytopenia, neurological symptoms, renal dysfunction, and fever. The primary underlying issue in TTP is the deficiency of the von Willebrand factor-cleaving protease (ADAMTS13) due to either inherited mutations or acquired inhibitors.

Emergent large-volume plasmapheresis is the cornerstone of treatment in TTP. Plasmapheresis removes the patient's plasma, which contains the circulating inhibitors to ADAMTS13 as well as the abnormal von Willebrand factor multimers that contribute to the pathological thrombosis and thrombocytopenia. This therapy helps to restore normal levels of ADAMTS13 and alleviates the microvascular occlusions through the removal of the factor causing platelet aggregation.

While platelet transfusions, anticoagulation therapy, or bone marrow transplants may be relevant in certain contexts, they do not address the root cause of TTP. Platelet transfusions may worsen the condition by potentially exacerbating the thrombosis. Anticoagulant therapy is not a mainstay due to the appearance of excessive bleeding once the balance is disrupted.