Patient characteristics
A total of 115 patients with SAA were treated with rATG and CSA from March 2013 to July 2020. Sixty-six patients were administered eltrombopag combined with IST from July 2017 to July 2020, with a median follow-up of 34 months (range, 18-51 months), including 34 SAA and 32 VSAA cases. The median age was 5.59 years, and the male to female ratio was 1.36:1 in the eltrombopag group. The median disease course before IST was 55 days (range,17-609 days). PNH results were available at diagnosis for 66 patients in the eltrombopag group, and were negative in 40 (60.6%) patients. Totally 7(10.6%) patients had PNH clones between 5.01% and 10% and19 (28.8%) had PNH clones larger than 10% (range, 10.53%-63.59%) in the granulocyte lineage; no patient had clinically evident hemolysis or thrombosis at diagnosis. Forty-nine children with IST treated at our center from March 2013 to May 2017 were assessed as a historical control group; median follow-up was 71 months (range, 53-103 months). The patients included 22 SAA and 27 VSAA cases, with a median age of 6.20 years. The male to female ratio was 0.88:1 in the control group. There were no significant differences in age, sex and disease severity between the two groups (Table 1).