Hematological response
The hematological response was evaluated at 3 months, 6 months and 12
months after IST and at the last follow-up in both groups. In the
eltrombopag group, the CR and OR rates were 30.3% and 63.6% at 3
months after IST, respectively, versus 8.2% and 49.0% in the control
group, respectively. There was a significant difference in CR between
the two groups at 3 months (P=0.004). There was no significant
difference in OR between the two groups at 3 months (P=0.074). At 6
months, the CR and OR rates were 50.0% and 71.2% in the eltrombopag
group, respectively. From 3 to 6 months, 3 NR cases achieved CR, 3 NR
cases were converted to PR, 10 PR cases were converted to CR, one PR
relapsed, and 18 cases maintained NR. In the control group, the CR and
OR rates were 10.2% and 57.1% at 6 months after IST. From 3 to 6
months, one NR case was converted to CR, 9 NR cases were converted to
PR, and 16 cases maintained NR. There was a significant difference in CR
between the two groups at 6 months (P<0.001). There was no significant
difference in OR between the two groups (P=0.117).
At 12 months after IST, the CR and OR rates were 51.5% and 71.2%,
respectively, in the eltrombopag group. From 6 to 12 months, 3 NR cases
achieved CR, one NR case was converted to PR, 3 PR cases were converted
to CR, one PR and one CR cases relapsed, and 18 cases retained NR. In
the control group at 12 months after IST, the CR and OR rates were
42.9% and 65.3%, respectively. From 6 to 12 months, 16 PR cases were
converted to CR, one PR case relapsed, and 17 cases retained NR. There
were no significant differences in CR (P=0.358) and OR (0.499) between
the two groups.
The clinical data were analyzed retrospectively to definite response
times in both groups. The median response time was 71 days
(interquartile range, 56 to 91 days) in the eltrombopag group. In the
control group, the median response time was 90 days (interquartile
range, 60 to 135 days). There was a significant difference in response
time between the eltrombopag and control groups (P=0.007). The median
times to CR in the eltrombopag and control groups were 98 days
(interquartile range, 65 to 158 days) and 360 days (interquartile range,
180 to 435 days), respectively, indicating a significant difference
between the two groups (p<0.001). We also compared the times to
factor-CSF, red blood cells and platelets between the two groups. There
were significant differences between the two groups in these variables
(Table 3).
Long term prognosis
We evaluated long-term prognosis by comparing OS, relapse, clone
evolution and EFS in both groups, as well as factors influencing
prognosis. It should be noted that the median follow-up time were 34
months in the eltrombopag group and 71 months in the control group.
Unfortunately, 10 patients were lost at the end of follow-up in the
control group.