Evidence map›Paper›PMID 40166709›Full record

ArticleResearch and practice in thrombosis and haemostasis2025

Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China.

Lingling Fu, Xi Lin, Zhenping Chen, Zhifa Wang, Yan Liu, Lijuan Wang, Yu Hu, Jingyao Ma, Nan Wang, Xiaoling Cheng and 2 more

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Article in Research and practice in thrombosis and haemostasis, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Lingling FuHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Xi LinHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Zhenping ChenDepartment of Clinical Laboratory Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Zhifa WangHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Yan LiuHematology Department, Baoding Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Lijuan WangHematology Department, Henan Province Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Yu HuHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Jingyao MaHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Nan WangPharmacology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Xiaoling ChengPharmacology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Jie MaHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Runhui WuHematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: The first second-line international recommendation for children with severe persistent/chronic immune thrombocytopenia is thrombopoietin receptor agonist (TPO-RA)-based treatment; however, <30% can achieve sustained response off-treatment (SRoT), leading to a heavy medical burden. Objectives: This study aimed to confirm the efficacy of the stepwise response-guided treatment protocol compared with TPO-RA-based second-line therapy for children with severe P/CITP. Methods: The stepwise response-guided treatment protocol is an individualized stratified immune thrombocytopenia treatment starting with high-dose dexamethasone, then adding rituximab and TPO-RAs in sequential order according to treatment response. A prospective, multicenter clinical cohort study enrolled severe P/CITP children with a 1-year follow-up. We compared the treatment outcome response of platelet count, bleeding control, and treatment-related side effects and cost outcomes (escalation status, SRoT, and treatment costs) between the stepwise group and the TPO-RA-based second-line treatment group (TPO-RA group). Results: The study enrolled 143 cases of severe P/CITP children with a 12-month follow-up period. There were no differences in baseline characteristics between the stepwise and TPO-RA groups ( Conclusion: The stepwise response-guided treatment protocol effectively stratifies children with severe P/CITP based on treatment response, enabling individualized treatment strategies. This protocol achieves comparable efficacy and safety while reducing the treatment burden compared with TPO-RA-based second-line therapy, making it a preferable option for children with severe P/CITP.

Indexed as

childrenpersistent/chronic immune thrombocytopeniasecond-line therapystepwise treatmentthrombopoietin receptor agonists

Identifiers

PMID40166709
PMCPMC11957504

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.