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A second-line escalating treatment strategy for children with severe chronic immune thrombocytopenia: A retrospective data from a single-center
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  • lingling FU,
  • Jie Ma,
  • Hao Gu,
  • Jingyao Ma,
  • Yunyun Wei,
  • Zhenping Chen,
  • Runhui Wu
lingling FU
Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health
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Jie Ma
Beijing Children's Hospital
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Hao Gu
Beijing Children's Hospital Capital Medical University
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Jingyao Ma
Beijing Children's Hospital Capital Medical University National Center of Children's Health, China
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Yunyun Wei
Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health
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Zhenping Chen
Beijing Children’s Hospital Capital Medical University
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Runhui Wu
Beijing Children's Hospital
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Abstract

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.

Peer review status:IN REVISION

30 Nov 2020Submitted to Pediatric Blood & Cancer
30 Nov 2020Submission Checks Completed
30 Nov 2020Assigned to Editor
30 Nov 2020Reviewer(s) Assigned
11 Dec 2020Review(s) Completed, Editorial Evaluation Pending
11 Dec 2020Editorial Decision: Revise Major
18 Jan 20211st Revision Received
18 Jan 2021Submission Checks Completed
18 Jan 2021Assigned to Editor
19 Jan 2021Reviewer(s) Assigned
25 Jan 2021Review(s) Completed, Editorial Evaluation Pending
25 Jan 2021Editorial Decision: Revise Minor