Current options for the treatment of idiopathic thrombocytopenic purpura

Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease characterized by low platelets and bleeding. Platelet autoantibodies result in accelerated platelet destruction by the reticuloendothelial cells in the spleen and liver, overwhelming the compensatory capability of the bone marrow to increase platelet production. The goal of treatment for patients with ITP is to raise the platelet count to high enough levels to prevent bleeding using the least toxic therapy, recognizing the generally benign nature of the illness. Corticosteroids, intravenous immune globulin, and splenectomy remain mainstays of treatment; however, newer therapies including rituximab and the thrombopoietin receptor agonists are remodeling conventional treatment algorithms. Immune suppressant medications and cytotoxic drugs continue to be used in patients with severe and chronic refractory ITP with some success; however, estimates of the effect of these and other treatments are limited by the lack of randomized trials using clinical end points. In this article, treatments for ITP are reviewed with a focus on their mechanism of action and the best available evidence from clinical studies. A move towards early aggressive therapy may alter the natural history of this self-perpetuating illness.