Clinical Research Directory

* INCLUSION CRITERIA: Current diagnosis of moderate aplastic anemia or unilineage bone marrow failure disorders. * Moderate aplastic anemia is defined as aplastic anemia (hypocellular bone marrow for age) with no evidence for other disease processes causing marrow failure, and depression of at least two out of three blood counts below the normal values: * ANC less than or equal to 1200/mm(3) * platelet count less than or equal to 70,000/mm(3) * anemia with hemoglobin less than or equal to 8.5 g/dL and absolute reticulocyte count less than or equal to 60,000/mm(3) in transfusion-dependent patients but not fulfilling the criteria for severe disease defined by depression of two of the three peripheral counts: * ANC less than or equal to 500/mm(3) * platelet count less than or equal to 20,000/mm(3) * reticulocyte count less than or equal to 60,000/mm(3) * Unilineage bone marrow failure disorders are defined: * Hemoglobin less than 8.5 g/dL and reticulocyte count less than 60,000 or red cell transfusion dependent and hypocellular to normocellular bone marrow for age with significantly reduced erythroid precursors. * OR thrombocytopenia less than or equal to 30,000/uL or platelet transfusion dependent and hypocellular to normocellular bone marrow for age with reduced megakaryocytes. * No evidence of viral or drug suppression of the marrow, dysplasia, or underproduction anemias secondary to B12, folate, iron or other reversible causes. Platelet transfusion dependent is defined as the need for platelet transfusion due to platelet counts of \< 10,000/microL with no bleeding (prophylactic transfusion) or \< 20,000/microL with bleeding (therapeutic transfusion). Red cell transfusion dependent is defined as transfusion of greater than 4 units of blood in the 8 weeks prior to study entry. Age greater than or equal to 2 years old Weight greater than 12 kg EXCLUSION CRITERIA: Known diagnosis of Fanconi anemia Counts that meet criteria for severe aplastic anemia Infection not adequately responding to appropriate therapy HIV positivity Creatinine \> 2.5 mg/dL Bilirubin \> 2.0 mg/dL, including congenital abnormalities in the bilirubin count SGOT or SGPT \>5 times the upper limit of normal Hypersensitivity to eltrombopag or its components Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential Evidence of an active malignant hematological or clonal disorder, or abnormal cytogenetic studies of the bone marrow performed within 12 weeks of study entry. Unable to understand the investigational nature of the study or give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely. Treatment with horse or rabbit ATG or Campath within 6 months of study entry. Treatment with cytokines such as G-CSF or Erythropoietin. Subjects with known cirrhosis in severity that would preclude tolerability of eltrombopag as evidenced by albumin less than 35g/L. Life expectancy of less than 3 months Patients with an active diagnosis of cancer who have received chemotherapeutic treatment or other specific antineoplastic drugs or radiation therapy within 6 months of study entry. Unable to take investigational drug