Treatment of chronic immune thrombocytopenic purpura in adults

Abstract

Until recently, the management of refractory immune thrombocytopenic purpura (ITP) was a real challenge as shown by the large variety of treatments proposed in both American Society of Hematology and the British Committee for Standards in Haematology guidelines published 10 and 6 years ago, respectively. However, as in the past 5 years, new therapeutic approaches including eradication of Helicobacter pylori in infected patients and rituximab have been proposed and the thrombopoietinreceptor agonists have been developed and licensed for ITP. It is likely that the therapeutic strategy of ITP will be profoundly modified and revisited in the next future. The purpose of this article is to discuss the impact and the place of these new therapeutic options into the whole treatment strategy of chronic ITP and to draw the perspective of new experimental therapies.

Gender-related analysis of the clinical presentation, treatment response and outcome in patients with immune thrombocytopenia

“…Immune thrombocytopenia, also known as ITP, is an autoimmune disorder that results in acute or chronic thrombocytopenia and that may potentially lead to a life-threatening hemorrhagic event [1,2]. Despite major advances in our understanding of the pathophysiology of ITP (evidence of anti-platelet antibodies and the relative failure of bone marrow platelet production) [1][2][3], the diagnosis of ITP still is based on exclusion [1,2].…”

Romiplostim for the treatment of chronic immune thrombocytopenia in adult Japanese patients: a double-blind, randomized Phase III clinical trial

“…Treatment is not recommended for patients with platelet counts [50 9 10 9 /L in the absence of bleeding, trauma/surgery, or other risk factors [11]. Available treatment options include corticosteroids and other immunosuppressive agents, splenectomy, and immunoglobulins [6,11,23]. Approximately, 20-35% of ITP patients have disease refractory to treatment with steroids, immunoglobulins, or splenectomy [22,24], and only two-thirds of patients undergoing splenectomy experience sustained response without additional therapy [11].…”

Splenic proliferative lymphoid nodules distinct from germinal centers are sites of autoantigen stimulation in immune thrombocytopenia

“…14 These autoantibodies are directed to Ag on the platelet surface and lead to increased destruction of circulating platelets, resulting in thrombocytopenia and possibly hemorrhagic complications. Because subjects with ITP refractory to standard therapy may require splenectomy, 15 samples of spleen tissues are available for study. Importantly, it is thought that the autoimmune response to platelet antigens arises in the spleen, 11 making study of this tissue ideal to determine the nature of the immune response that underlies this autoimmune disease.…”