Objective: To analyze the effect of a novel second-line escalating
treatment strategy (high-dose dexamethasone (HDD), low-dose rituximab to
eltrombopag) for children with severe chronic immune thrombocytopenia
(SCITP). Materials and Methods: This study was a single-center,
retrospective cohort study. The second-line escalating strategy included
3 steps: Step I (6 courses high-dose dexamethasone: HDD), Step II (HDD
combined with low-dose rituximab), and Step III (eltrombopag). Results:
A total of 30 cases (18 males and 12 females) were included; the median
age was 8.83 (1.42-13.9) year-old, the duration time of ITP was 20.5
(12.0-96.0) months, and the platelet counts were 15 (3-29) ×109/L. After
the median 14 (12-37) months’ treatment, the remission rate was 36.7%
(11/30), and the sustained response (SR) rate was 68.2% (15/22). In
eltrombopag (step III) cases, 47.5% (8/17) maintained platelet
≥50×109/L, 37.5% (3/8) dose tapering, and 25% (2/8) were successfully
discontinued from medication. The number of patients at 12th, 24th, and
36th months was 30, 7, and 2, with the total response (TR) and remission
rates of 80% (36.7%), 57.1% (28.6%), and 50% (50%), respectively.
The total relapse rate was 26.7% (8/30)，three cases(75%, 3/4)from
Step II and 5 cases (41.7% ,5/12)from Step III, none in Step I.
Conclusion: The new second-line escalating strategy for children SCITP
has an effective improving rate with 36.7% remission and 68.2% SR;
30% could benefit and retain stable response from HDD treatment.
Combined treatment with eltrombopag can reduce the relapse rate of
low-dose rituximab.
Efficacy and Safety of Avatrombopag in Patients with Chronic Immune Thrombocytopenia: A Systematic Literature Review and Network Meta-Analysis
