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Introduction: Aplastic anemia is a clinical syndrome characterized by peripheral pancytopenia and deficiency of hematopoietic precursors in the bone marrow. Allogeneic hematopoietic stem cell transplantation (AHSCT) should be considered as first line treatment for young patients with an available donor. However,alternative therapy options are scant in patients who are not candidates for transplantation. Here, we report the efficacy of eltrombopag in a case of severe aplastic anemia.
Case Presentation: Twenty-two years old female patient was admitted to the Marmara University Hospital because of severe aplastic anemia. There was not an available HLA-matched sibling donor and immunosuppressive treatment with horse-derived ATG (40 mg per day for 5 days) and cyclosporine (5 mg/kg per day) was started. At the sixth month of therapy she was still in need of transfusion. Eltrombopag was prescribed at a dose of 50 mg and the dose was increased up to 150 mg per day in 2 months. Successful response was noted within 2 weeks of 150 mg dosage and this response was sustained at the 4th month following discontinuation of drug.
Conclusions: Eltrombopag provides good and permanent clinical response in refractory severe aplastic anemia.
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