Successful treatment of amegakaryocytic thrombocytopenia with eltrombopag in a patient with systemic lupus erythematosus (SLE).

Systemic lupus erythematosus (SLE) is an inflammatory connective tissue disease mediated by a deregulated immune system and aberrant production of proinflammatory cytokines leading to multiorgan and tissue damage. Thrombocytopenia is a common complication in patients with SLE, with a prevalence that ranges from 7–30%.1-5 The 2 main mechanisms of thrombocytopenia in SLE are increased platelet destruction mediated by antiplatelet autoantibodies, similar to the mechanism seen in immune thrombocytopenic purpura (ITP), and impaired thrombopoiesis, due to the absence of or a decreased number of megakaryocytes in the bone marrow. The latter disorder is also known as amegakaryocytic thrombocytopenia (AMT). T-cell–mediated and antibody-mediated suppression of megakaryocyte colony formation have both been reported as possible mechanisms of AMT.1 Similar to ITP, treatment modalities include immunosuppressive agents such as prednisone, immunomodulators such as intravenous immunoglobulin (IVIG), and splenectomy. We report a case of SLE-associated AMT successfully treated with eltrombopag (Promacta, GlaxoSmithKline), a thrombopoietin (TPO) nonpeptide mimetic that binds to and activates the TPO receptor leading to increased production of megakaryocytes and platelets. To our knowledge, this is the first reported case of eltrombopag use in SLE-associated AMT.