Extended Dosing With Eltrombopag for Severe Aplastic Anemia
Condition: Severe Aplastic Anemia (SAA)
MeSH terms: Anemia; Anemia, Aplastic
Intervention: Eltrombopag
Intervention description: Oral administration of eltrombopag 150mg/day (75 mg/day for East Asian ancestry) for 6 months
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 40.0
Sex: All
Minimum age: 2 Years
Maximum age: 100 Years
Healthy volunteers: No
Keywords: Cytokine; Hematopoiesis; Autoimmunity; Thrombocytopenia; Neutropenia

Summary:

      Background:

      - Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve
      blood counts in these patients. However, researchers do not know how long the drug can and
      should be taken for this type of anemia.

      Objectives:

      - To look at whether 6 months of treatment with eltrombopag can improve patient s blood
      counts.

      Eligibility:

      - Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia.

      Design:

        -  Participants will take eltrombopag by mouth once a day for 6 months.

        -  Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow
           samples will be collected at 3 and 6 months. These samples will look at the effects of
           the study drug on the marrow.

        -  Participants will continue to take the study drug for as long as it is effective and if
           the side effects are not severe.
    

Eligibility criteria:

        -  INCLUSION CRITERIA:

          -  Previous diagnosis of refractory severe aplastic anemia and following at least one
             treatment course of immunosuppression with a regimen containing antithymocyte
             globulin, alemtuzumab or cyclophosphamide.

          -  One or more of the following three clinically-significant cytopenias: platelet count
             less than or equal to 30,000/micro L or platelet-transfusion-dependence (requiring at
             least 4 platelet transfusions in the 8 weeks prior to study entry); neutrophil count
             less than 500/micro L; hemoglobin less than 9.0 g/dL or red cell
             transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior
             to study entry)

          -  Age greater than or equal to 2 years old

          -  Weight > 12 kg

        EXCLUSION CRITERIA:

          -  Infection not adequately responding to appropriate therapy

          -  Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study
             entry.

          -  Creatinine > 2.5 mg/dL

          -  Direct Bilirubin > 2.0 mg/dL

          -  SGOT or SGPT >5 times the upper limit of normal

          -  Hypersensitivity to eltrombopag or its components<TAB>

          -  Female subjects who are nursing or pregnant or are unwilling to take oral
             contraceptives or refrain from pregnancy if of childbearing potential

          -  Unable to understand the investigational nature of the study or give 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 ATG, cyclophophamide or alemtuzamab within 6 months of study entry.