For Cohort A:
Prior history of diagnosis of SAA
Diagnosis of relapsed/refractory SAA or recurrent AA following IST for SAA at the time of enrollment. Patients with recurrent AA (e.g., losing their response) are exempt from meeting the diagnostic criteria for relapsed SAA at the time of enrollment, but must have been previously diagnosed with SAA.
Agree to concurrent eltrombopag treatment with appropriate, investigator-selected IST with either hATG + CsA or CsA.
For Cohort B:
Diagnosis of SAA at the time of enrollment
Patients must not have been previously treated for SAA
Patients must agree to treatment with hATG + CsA concurrent with eltrombopag.
For all patients, regardless of cohort:
Age 1 to <18 years
Where appropriate, assessments to rule out congenital/inherited bone marrow failure syndromes and other causes of immune-mediated pancytopenia, which may be treated with transplant, must be completed prior to enrollment.
Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient. (Candidacy for HSCT will be determined as per local practice.)
Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first dose of eltrombopag
Normal karyotype with FISH for chromosomes 7 and 8
Performance status score: Karnofsky ≥50 or Lansky ≥50 (depending on age)
Serum creatinine ≤2.5 × ULN
Total bilirubin ≤1.5 × ULN
Written informed consent signed by a parent or legal guardian prior to initiation of any study specific procedure.
Prior and/or active medical history of:
Fanconi anemia (via chromosomal breakage test or growth arrest by flow cytometry)
Other known underlying congenital/inherited marrow failure syndromes
Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and/or PNH clones >50% of PMN or RBC at time of enrollment
Any cytogenetic abnormalities, including but not limited to chromosome 7 or myelodysplasia, in bone marrow within 4 weeks of study enrollment
Myelodysplastic syndrome (MDS)
Other known or suspected underlying primary immunodeficiency
Active infection not responding to appropriate therapy
Prior eltrombopag or other thrombopoietin receptor (TPO-R) agonist treatment for at least 2 months and a lack of response.
Any out of range lab values Creatinine >2.5 × upper limit of normal (ULN), Total bilirubin >1.5 × ULN Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5 × ULN