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.