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