Eltrombopag in the treatment of aplastic anemia in Croatia – a CROHEM study

Aim: The aim of this study was to present the first Croatian experience with eltrombopag (a peroral thrombopoietin receptor agonist) as novel treatment option for aplastic anemia (AA). ----- Patients and Methods: This a retrospective analysis on use adult patients acquired AA among hematology centers. Results: Between 2015 2019, thirteen (nine female (69%), median age 59 (26-79) years) were treated eltrombopag: six them had severe AA, very one paroxysmal nocturnal hemoglobinuria fenotype. All received eltrombopag after at least previous line treatment, either monotherapy or in combination cyclosporine +/- low dose methylprednisolone. Hematological response achieved seven (54%) patients: complete remission four, partial two, minimal patient, while (46%) refractory. Median duration administration four (1,5-40) months. 150 mg per day adverse events consistent known safety data drug. Sixty-nine percent still alive follow up 16 (2-48) One patient who relapsed eight months developed new cytogenetic change (trisomy 8). Four died, (1-12) discontinuation therapy, due comorbidities infections. Conclusion: Presented from centers real-world setting confirm that is an interesting modality treat especially those are refractory prior immunosuppressive therapy and/or unsuitable allogeneic hematopoietic stem cell transplantation.