Use of eltrombopag in treatment programs for patients with aplastic anemia

Introduction . The use of thrombopoietin receptor agonists, especially eltrombopag, in the treatment aplastic anemia (AA) patients who did not respond to previous immunosuppressive therapy (IST), is accompanied by development a hematological response 40–60 % patients. Aim — study effi cacy using eltrombopag programs for AA refractory IST. Methods included 20 were treated at National Research Center Hematology from 2015 2020. These conducted IST (ATG + CsA). Eltrombopag was administered dose 150 mg/day. results assessed 3 and 6 months: achievement improvement, partial complete remission, as well identifi cation possible clonal evolution determined. Results Eleven out (55 %) responded treatment: 2 developed remission. All 11 within 12 months start but further positive dynamics parameters are possible. median duration with (1–48) months. Most combination CsA. course depended on received (stable detection evolution) or its absence need ATG BMT. An aberrant karyotype found eltrombopag: one patient monosomy chromosome 7 detected 1 month after treatment, another patient, 37 later, clone derivative 16 t(1;16) subclone complex disorders without signs myelodysplasia bone marrow. Conclusion inclusion TPO agonist program allows stable remission have effectiveness determined adherence algorithm, optimal course, drug used. There still long-term observation control morphological cytogenetic studies.