Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients

Inclusion criteria

• Patient with a previous diagnosis of aplastic anemia and had no response or relapsed following at least one treatment course in a period time of ≥ 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
• Current diagnosis of aplastic anemia by bone marrow biopsy;
• did not receive HSCT nor were HSCT candidates;
• Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
• Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or <480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site.
• Subjects are able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.

Exclusion criteria

• Congenital aplastic anemia;
• Presence of chromosomal aberration;
• Evidence of a clonal hematologic bone marrow disorder on cytogenetics;
• Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years;
• AST or ALT ≥3 times the upper limit of normal;
• Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN;
• Cardiac disorder (NYHA) functional classification Grade II/III/IV;
• Past history of thromboembolic event (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
• Infection not adequately responding to appropriate therapy;
• Other known or suspected underlying primary immunodeficiency;
• Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin) receptor agonist;
• Pregnant or nursing (lactating) woman;