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, 9 (6), 7112-7125

Efficacy and Safety of Thrombopoietin Receptor Agonists in Children With Chronic Immune Thrombocytopenia: A Meta-Analysis


Efficacy and Safety of Thrombopoietin Receptor Agonists in Children With Chronic Immune Thrombocytopenia: A Meta-Analysis

Jian-Chun Guo et al. Oncotarget.


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.

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

Conflict of interest statement

CONFLICTS OF INTEREST All authors declare no conflicts of interest.


Figure 1
Figure 1. Flowchart of article selection process
Figure 2
Figure 2. Forest plot of primary outcomes
(A) overall platelet response. (B) Durable response. (C) Forest plot of rescue medication. RR: relative risk, CI: confidence interval, 1: romiplostim, and 2: eltrombopag.
Figure 3
Figure 3. Forest plot of bleeding events
(A) Any bleeding events. (B) Severe bleeding events. (C) Clinically significant bleeding events.
Figure 4
Figure 4. Forest plot of second outcomes
(A) Health-related quality of life. (B) Parental burden.
Figure 5
Figure 5. Forest plot of safety profiles
(A) Any adverse events. (B) Severe adverse events.
Figure 6
Figure 6. Funnel plot of publication bias for overall platelet response

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