Sustained Responses following Treatment with Romiplostim in Immune Thrombocytopenia

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease leading to a low peripheral blood platelet count. The underlying pathophysiology is complex and involves platelet destruction caused by antibodies directed at platelet glycoproteins and a relative platelet underproduction by the bone marrow. Until recently, most treatments have targeted only platelet destruction by inducing immune suppression. 

Recently a new class of therapy, the thrombopoietin receptor agonists (TRA), has been developed which enhances platelet production by the marrow. TRAs, which include the peptibody romiplostim, and the oral small molecule eltrombopag, are regarded as palliative therapies which elevate the platelet count in refractory patients, who continue to bleed, to cover invasive procedures or to delay or be a bridge to transplant. The expectation is that the platelet count will fall to baseline once the drug is stopped. Romiplostim is a thrombopoietin peptide mimetic agent recently approved for the treatment of adults with chronic immune thrombocytopenia (ITP). Read more>>>>>>>>>>>>