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

// Jian-Chun Guo 1, 2, * , Yi Zheng 1, 2, * , Hai-Tao Chen 4 , Haixia Zhou 3 , Xian-Hui Huang 1, 2 , Li-Ping Zhong 1, 2 , Huai-Bin Zhou 1, 2 , Yu Huang 5 , Dan-Li Xie 1, 2 and Yong-Liang Lou 1, 2 1 Department of Microbiology and Immunology, School of Laboratory Medicine, Wenzhou Medical University, Wenzhou, Zhejiang 325035, China 2 China Ministry of Education Key Lab of Laboratory Medicine, Wenzhou, Zhejiang 325035, China 3 Department of Hematology, The Second Affiliated Hospital and Yuying Children’s Hospital of Wenzhou Medical University, Wenzhou, Zhejiang 325027, China 4 Department of Orthopaedic Surgery, Zhongnan Hospital of Wuhan University, Wuhan, Hubei 430071, China 5 Department 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: lyl@wmu.edu.cn Dan-Li Xie, email: xiedl001@wmu.edu.cn Keywords: thrombopoietin receptor agonists; immune thrombocytopenia; children; meta-analysis Received: September 30, 2017      Accepted: November 15, 2017      Published: December 19, 2017 ABSTRACT 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.