New drug combo shows promise for rare blood disorder

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

-INCLUSION CRITERIA: 1. Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes) AND At least two of the following: * Absolute neutrophil count less than 500/microL * Platelet count less than 20,000/microL Absolute reticulocyte count less than 60,000/microL 2. Age greater than or equal to 2 years old 3. Weight greater than 12 kg EXCLUSION CRITERIA: 1. Known diagnosis of Fanconi anemia 2. Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry. Patients with super severe neutropenia (ANC less than 200 /microL) will not be excluded initially if cytogenetics are not available or pending. If evidence of a clonal disorder consistent with myelodysplasia is later identified, the patient will go off study. 3. Prior immunosuppressive therapy with any ATG, alemtuzumab, or high dose cyclophosphamide 4. SGOT or SGPT \>5 times the upper limit of normal 5. Subjects with known liver cirrhosis in severity that would preclude tolerability of cyclosporine and eltrombopag as evidenced by albumin \< 35g/L 6. Hypersensitivity to eltrombopag or its components 7. Infection not adequately responding to appropriate therapy 8. 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 9. Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible 10. Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of this study 11. Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent.