Severe aplastic anemia (SAA) is caused by immune-mediated destruction. Standard immunosuppressive therapy (IST) is effective, but needs to be improved. A total of 115 patients (60 males; median age of 5.77 years and median follow-up time of 45 months) were enrolled in this historical control study. The complete response (CR) rates in the eltrombopag group were 30.3% at 3 months, 50.0% at 6 months, conpared to 8.2% at 3 months, 10.2% at 6 months in the control group. There were significant differences between the two groups at 3 months and 6 months after IST. The overall response rates in the two groups showed no significant differences during the study. There were significant differences in the times separated from granulocyte colony stimulating factor (G-CSF) G-CSF, red blood cell transfusion and Platelet transfusion between the two groups. Overall survival rates were 97.0% in the eltrombopag group and 96.0% in the control group (P=0.998). In the eltrombopag group 10.2% cases relapsed compared to 4.1% in the control group (P=0.703). No case progressed to myelodysplastic syndrome or myeloid leukemia in the eltrombopag group; one patient (2.0%) progressed to myelodysplastic syndrome in the control group. Totally 11 patients (16.7%) showed myeloid gene mutations in the eltrombopag group. Event-free survival (EFS) was 66.6% in the eltrombopag group and 57.1% in the control group. There were no significant differences in EFS between the two groups (P=0.967). In the eltrombopag group the common adverse reactions were transient and reversible hyperbilirubinemia, elevated liver enzymesand hyperuricemia.

Background: Eltrombopag (E-PAG) is being investigated for the treatment of aplastic anemia (AA) by stimulating hematopoietic stem cell (HSC) proliferation. Objective: To evaluate the effectiveness and safety of E-PAG in first-line therapy for pediatric AA. Methods: The present retrospective study reviewed the pediatric patients with newly diagnosed AA in immunosuppressive therapy (IST) therapy with E-PAG at the single center from March to September in 2017. All patients were followed up for >2 years. Results: A total of 14 patients (8 males), aged 86 months, were enrolled in this study. E-PAG was administered with a median time to initiation of 19.5 days after IST, and the median course of treatment was 253 days. The rate of complete response and overall response at 6 months were 64.3% (9/14 case) and 78.6% (11/14 cases) respectively. The survival rate was 100%, and no relapses occurred in responders. E-PAG was well-tolerated; however, the most common adverse events included indirect bilirubin elevation, jaundice, and transient liver-enzyme elevation. By the end of follow-up, bone marrow chromosomes were normal, and no abnormal myelodysplastic syndromes (MDS) -related clones appeared. Conclusions: The addition of E-PAG to IST was associated with the markedly increased complete response with respect to hematology in pediatric patients with SAA than in a historical cohort without the unacceptable side effects.