Eltrombopag in Patients With Severe Aplastic Anemia or Recurrent Aplastic Anemia


This is a phase II, open-label, multi-center, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.


  • Ages 1 to 17 years old
  • 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.

For additional eligibility criteria please contact the study team.


  • Drug Treatment
  • Follow up
  • Physical exam
  • Medical Record review

Additional study details and procedures will be explained by the study team.


Additional eligibility criteria and study procedure details can be explained by the study team.  Call or email:

  • Phone:  (352) 273-8675
  • Email:  trials@cancer.ufl.edu


18 and under




Bone Marrow disease, Eltrombopag, Anemia, Aplastic Anemia, Pharmacokinetics

Principal Investigator

Paul Castillo Caro, M.D.



Contact Information


(352) 273-8675

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