Event-free survival
At the final follow-up in the eltrombopag group, two patients had died, five relapsed, and three (4.5%) received secondary treatment, including oral stanozolol, eltrombopag and traditional Chinese medicine. Totally 12 patients received HSCT, of whom 11 achieved CR and one died of transplantation complications. No patient progressed to myelodysplastic syndrome or myeloid leukemia. Therefore, the EFS rate was 66.6% in the eltrombopag group. In the control group, two patients had died, two relapsed, and 8 (16.32%) showed decreased blood counts and received oral stanozolol and traditional Chinese medicine. Totally 9 patients received HSCT and achieved CR; the remaining patient progressed to myelodysplastic syndrome at 13 months after IST treatment. Therefore, the EFS rate was 57.1% in the control group. There was no significant difference in EFS between the two groups (P=0.967) (Figure 2).
In the eltrombopag group, factors influencing hematological response in the 66 patients were evaluated by logistic regression analysis. It was found that age (P=0.623), gender (P=0.636), absolute neutrophil count (P=0.556) and lymphocyte count (P=0.121) at diagnosis, and PNH clones (P=0.540) and the time from diagnosis to IST treatment (P=0.405) had no effects on prognosis.