Efficacy and safety of thrombopoietin receptor agonists in children with chronic immune thrombocytopenia: a meta-analysis

Jian-Chun Guo, Yi Zheng, Hai-Tao Chen, Haixia Zhou, Xian-Hui Huang, Li-Ping Zhong, Huai-Bin Zhou, Yu Huang, Dan-Li Xie and Yong-Liang Lou _

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Oncotarget. 2018; 9:7112-7125. https://doi.org/10.18632/oncotarget.23487

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Jian-Chun Guo1,2,*, Yi Zheng1,2,*, Hai-Tao Chen4, Haixia Zhou3, Xian-Hui Huang1,2, Li-Ping Zhong1,2, Huai-Bin Zhou1,2, Yu Huang5, Dan-Li Xie1,2 and Yong-Liang Lou1,2

1Department of Microbiology and Immunology, School of Laboratory Medicine, Wenzhou Medical University, Wenzhou, Zhejiang 325035, China

2China Ministry of Education Key Lab of Laboratory Medicine, Wenzhou, Zhejiang 325035, China

3Department of Hematology, The Second Affiliated Hospital and Yuying Children’s Hospital of Wenzhou Medical University, Wenzhou, Zhejiang 325027, China

4Department of Orthopaedic Surgery, Zhongnan Hospital of Wuhan University, Wuhan, Hubei 430071, China

5Department of Infectious Diseases, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou Key Laboratory of Hepatology, Hepatology Institute of Wenzhou Medical University, Wenzhou, Zhejiang 325025, China

*These authors contributed equally to this work and should be considered co-first authors

Correspondence to:

Yong-Liang Lou, email: [email protected]

Dan-Li Xie, email: [email protected]

Keywords: thrombopoietin receptor agonists; immune thrombocytopenia; children; meta-analysis

Received: September 30, 2017     Accepted: November 15, 2017     Published: December 19, 2017


Background and Aim: Thrombopoietin receptor agonists (TPO-RAs) have been shown to be safe and effective for adults with chronic immune thrombocytopenia (ITP). The aim of this meta-analysis is to assess the efficacy and safety of thrombopoietin receptor agonists for children with chronic ITP.

Materials and Methods: Clinical randomized controlled trials (RCTs) evaluating the efficacy and safety of TPO-RAs in pediatric ITP patients published up to June 2017 were retrieved from PubMed, Cochrane Library, and Embase databases. Relevant data were extracted, and the Physiotherapy Evidence Database scale was used to assess the methodological quality. Stata/SE 12.0 was used to perform a meta-analysis.

Results: Seven RCTs were included, with 238 patients and 107 patients in the TPO-RA group and the control group, respectively. Assessing efficacy, better results were found in the TPO-RA group for the rate of overall platelet response, durable response, and rescue medication needed. Furthermore, the TPO-RA group yielded superior results in the incidence of clinically significant bleeding events but had a comparable result in the incidence of any bleeding events and severe bleeding events. No significant difference was found between the two groups in health-related quality of life and parental burden. Assessing safety, no significant difference was found between the two groups in the incidence of any adverse events and severe adverse events.

Conclusions: TPO-RAs are effective and safe agents for the treatment of chronic ITP in pediatric patients. Eltrombopag appears to be better than romiplostim in terms of the rate of rescue medication needed and clinically significant bleeding events.

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