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).