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Patients with acquired aplastic anemia who are refractory to immunosuppressive agents and ineligible for bone marrow transplantation have few treatment options. Most patients become transfusion-dependent and succumb to infections. Cytokines such as erythropoietin and granulocyte colony-stimulating factor are usually ineffective, but drugs that specifically engage stem-cell receptors might be beneficial. One such agent, eltrombopag, is a synthetic small-molecule thrombopoietin agonist that is FDA approved for the treatment of chronic immune thrombocytopenia.
To evaluate the usefulness of eltrombopag in patients with aplastic anemia, investigators conducted a phase II study of 25 patients with disease that was refractory to immunosuppression a…