Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia

Inclusion Criteria: * Written study informed consent and (where applicable) assent from the subject, parent, or guardian must be obtained prior to participation in the study. * Subjects of East Asian ethnicity aged ≥ 6 years old at the time of written informed consent and assent form (if applicable). * SAA characterized by: * Bone marrow cellularity \< 25%, or 25-50% with \< 30% residual hematopoietic cells and pancytopenia, with at least two of the following parameters in peripheral blood: * Absolute neutrophil count \< 0.5×109/L * Platelet count \< 20×109/L * Absolute reticulocyte count \< 20×109/L * HSCT not suitable or available as a treatment option (determined as per local practices or national guidelines), or has been refused by subject. Exclusion Criteria: * Prior IST with any ATG/ALG , alemtuzumab, high dose cyclophosphamide (≥ 45 mg/kg/day), CsA within 6 months, or prior thrombopoietin receptor agonists. * Eastern Cooperative Oncology Group (ECOG) performance status (age ≥ 16 years) \>2, or Lansky performance status (age \< 16 years) \<50. * Prior and/or active medical history of: * Known underlying congenital/inherited bone marrow failure or aplastic anemia (e.g.,such as but not limited to Fanconi anemia, congenital dyskeratosis, congenital amegakaryocytic thrombocytopenia, or Shwachman-Diamond Syndrome) * Symptomatic paroxysmal nocturnal hemoglobinuria (PNH) and/or PNH clones \>50% of polymorphonuclear neutrophil (PMN) or RBC at time of enrollment * Myelodyspla…