Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

Inclusion Criteria:

- Able to provide written informed consent and any other authorizations required by
local law (e.g., Protected Health Information [PHI])

- Have severe or very severe aplastic anemia, or moderate aplastic anemia with platelet
counts that have dropped below 20,000/μl

- Have moderate, severe, or very severe aplastic anemia with moderate bleeding during or
after a surgical procedure, (including bone marrow biopsy, lumbar puncture,
thoracentesis, paracentesis, port placement, dermal biopsy) or minimal mucocutaneous
bleeding otherwise noted

- Subjects with current or previous exposure to approved medications for the treatment
of aplastic anemia will not be excluded; these include but may not be limited to,
anti-thymocyte globulin (ATG), cyclosporine, corticosteroids, and G-CSF.

Exclusion Criteria:

- Have diagnosis of Fanconi anemia

- Have infection not adequately responding to appropriate therapy

- Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater
than or equal to 50%

- Have known HIV positivity

- Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal

- Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper
limit of normal if the patient has been treated with ATG within three weeks of
screening.

- Have AST and/or ALT ≥ 3 times the upper limit of normal

- Have hypersensitivity to eltrombopag or its components

- Have chemotherapy given less than or equal to 14 days prior to initiating the study
medication. This does not include immunosuppressive agents and growth factor as
described above

- Are female and are nursing or pregnant or are unwilling to take oral contraceptives or
refrain from pregnancy if of childbearing potential

- Are unable to understand the investigational nature of the study or give informed
consent

- Have a history of arterial or venous thrombosis within the last 1 year (excluding
those due to indwelling lines)

- Have an ECOG performance status of 3 or greater

- Have had treatment with Campath within 6 months of entry into the study

- Have pre-existing cardiovascular disease (congestive heart failure with New York Heart
Association [NYHA] grade III/IV), arrhythmia known to increase the risk of
thromboembolic events (e.g. atrial fibrillation), unstable angina, or QTc > 450 msec
(QTc 480 msec for subjects with bundle branch block), or myocardial infarction within
the preceding 6 months) at study entry

- Have had other TPO-R agonists medication in the previous 4 weeks.