Safety and Efficacy of Romiplostim in Patients with Severe, Chronic Idiopathic Thrombocytopenic Purpura

Immune Thrombocytopenia (ITP) is an autoimmune disorder characterized by autoantibody-mediated destruction of platelets. In general, the goal of therapy is the prevention of bleeding complications. Since the risk of life-threatening bleeding complications (eg, intracranial hemorrhage) is extremely rare, the most optimal treatment must remain individualized on the basis of all clinical conditions related to the affected patient. Based on this finding and on our own experiences the majority of affected patients may not require treatment. If indicated, the accepted standard therapies are corticosteroid-base treatments, intravenous immunoglobulins (IVIG) or anti-D, and in severely affected and unresponsive cases, splenectomy. Romiplostim is a 60 kDa molecule that is composed of four TPO-mimetic peptides that are attached via glycine bridges to an IgG heavy-chain Fc molecule. It effectively competes with eTPO for binding to TPO-receptor, leading to an increase in platelet counts within five to ten days after weekly subcutaneous injection. Romiplostim has been approved for the treatment of ITP in the U.S. and Europe. The aim of this review is to summarize results the current data from controlled clinical trials on the safety an efficacy of romiplostim in the treatment of ITP.