Eltrombopag As Initial Monotherapy for Transfusion Dependent Patients with Low to Intermediate Risk-1 Myelodysplastic Syndrome: Single Center Experience

Myelodysplastic syndrome (MDS) is a group of disorders involving hematopoietic dysfunction, with a leukemic transformation characteristic. The risk based therapeutic strategy was well applied in clinical. And for patients with IPSS low or intermediate risk-1, NCCN guideline recommended hematopoietic stimulation therapy, immune suppression therapy (IST), as well as demethylation therapy, along with best supportive care. Eltrombopag (EPAG) was firstly approved as the second line option for immune thrombocytopenia (ITP), and recently it was also investigated for treatment of aplastic anemia and MDS. In MDS, based on available reports, the EPAG was mostly used along with hypomethylating-agent, or as salvage treatment after treatment failure. However, there was study showed that the combination of EPAG to azacitidine (AZA) may worsen platelet recovery, with lower response rate and increased trend of leukemic transformation. The successful EPAG monotherapy for MDS case was report, with complete hematologic remission and blasts elimination.