New-generation drugs that stimulate platelet production in chronic immune thrombocytopenic purpura

SummaryIdiopathic thrombocytopenic purpura is an acquired disease characterised by a low platelet count. Development of autoantibodies is a main cause of the disease. Although many patients have few symptoms, life-threatening bleeding can arise and hence, when platelet counts fall to unacceptable values treatment should be initiated. However, conventional immunosuppressive approaches can fail, perhaps because of the heterogeneous nature of the disease. Newly developed agents that increase platelet production by stimulating megakaryocytes—such as drugs that bind to the thrombopoietin receptor c-MPL—offer an alternative treatment strategy. Although initial thrombopoietin analogues caused adverse immune reactions, second-generation thrombopoietin-receptor agonists that are in late-stage clinical development seem promising. In particular, eltrombopag and romiplostim safely increase and maintain platelet production in patients with refractory disease. However, long-term side-effects are being assessed and the exact role of these agents in the overall treatment strategy of chronic idiopathic thrombocytopenic purpura remains to be established.