Use of Eltrombopag in Patients with Platelet Engraftment Failure Following Second Allogeneic Peripheral Stem Cell Transplantation

Abstract

Thrombocytopenia after peripheral stem cell transplantation (PSCT) is associated with morbidity and mortality. Eltrombopag, athrombopoietin receptor agonist, is successfully used primarily in the treatment of chronic idiopathic thrombocytopenic purpuraand other thrombocytopenias associated with aplastic anemia and myelodysplastic syndrome. Recently, the use of eltrombopagin the treatment of thrombocytopenia after allogeneic PSCT has shown promising results. The use of eltrombopag in three patientswith hematologic malignancy who experienced graft failure after the first PSCT and developed platelet engraftment failurefollowing the second bone marrow (BM) transplantation was presented retrospectively. The patients included two males and onefemale, with the mean age of 52 (45–57) years. The diagnoses were acute myeloid leukemia, non-hodgkin lymphoma (NHL), acutelymphocytic leukemia. All patients underwent allogeneic PSCT with the myeloablative regimen. Platelet engraftment failure wasdetected during the follow-up of the patients. Acute grade 3 skin graft versus host disease developed in the patient with NHL. Mycophenolate-mofetil,cyclosporin-A, steroid-based immunosuppression therapy was given. Graft versus host disease completelyresponded to this treatment in the first week of treatment. However, thrombocytopenia persisted. None of the patients had anyviral infection or relapse. BM biopsies of patients were hypocellular, and the number of megakaryocytes were found to be decreased.Eltrombopag was initiated in three patients after 110 (60–144) days of transplantation. Responses were obtained in all ofthe patients; the platelet value was ≥30 × 103/µL (30.000–247.000). The mean duration of response was 27 (20–35) days. Althoughengraftment failure is not a routine indication of the eltrombopag, it can be used safely and effectively in patients with plateletengraftment failure after PSCT.