Issue: 2011 > November/December > review

New therapeutic options for immune thrombocytopenia

M. Schipperus, R. Fijnheer


Understanding of the mechanisms and aetiology of immune thrombocytopenia (ITP) has progressed significantly in recent years. It is now recognised to be an autoimmune condition, involving not only platelet destruction, but also deficits in platelet production. This has led to widespread research exploring potential mechanisms for therapy, the result of which has been the development of romiplostim and eltrombopag. These new treatments target the thrombopoietin receptor (TPO-R), promoting formation of megakaryocytes and survival of platelets. Furthermore, the advances in the understanding of ITP have led to the production of guidelines to assist healthcare professionals in the diagnosis and treatment of ITP. This review examines the recommendations made in these guidelines, particularly the American Society of Haematology
(ASH) 2011 evidence-based practice guidelines. In addition, searches were carried out to retrieve information on clinical trials of new molecules and off-label treatments for ITP. Corticosteroids, anti-Rho(D) immunoglobulins (anti-D), intravenous immunoglobulins (IVIg) and splenectomy are well-established treatments and continue to be recommended in the guidelines. The recently available romiplostim and eltrombopag, which are specific for treatment of IT P, are also included in the recommendations. The only off-label therapy to be recommended in the guidelines is the chimeric monoclonal antibody rituximab. However, investigations are ongoing into products approved for other indications, which may be beneficial to patients suffering from refractory ITP.