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

Abstract

Background and AimThrombopoietin 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 MethodsClinical 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.ResultsSeven 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.ConclusionsTPO-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.

Immune thrombocytopenia: improving quality of life and patient outcomes

“…Studies of the TPO-RAs romiplostim and eltrombopag did not report HRQoL improvement in treated patients compared to placebo 49,54,55. A reduced parental burden compared to placebo was found in romiplostim studies,49,55 but the difference was not significant in a meta-analysis 56…”

<p>Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use</p>

“…However, in both studies individually, there was some evidence of improved parental impact scores in those treated with romiplostim versus controls, signifying possible decrease in parental burden. 69 Parental fear of bleeding significantly impacts HRQoL for caregivers with parental impact scores being lower than child-report score. 65,69 The reduction of parental burden in the setting of a high response rate to romiplostim supports the idea that parental burden and worry are likely dependent on platelet count and therapy response.…”

“…69 Parental fear of bleeding significantly impacts HRQoL for caregivers with parental impact scores being lower than child-report score. 65,69 The reduction of parental burden in the setting of a high response rate to romiplostim supports the idea that parental burden and worry are likely dependent on platelet count and therapy response. Conversely, the lack of improvement in self-report and parent-proxy scores suggests other factors such as activity limitations, medical visits, and anxiety with venipuncture likely drive decreased HRQoL scores among children.…”

“…However, in both studies individually, there was some evidence of improved parental impact scores in those treated with romiplostim versus controls, signifying possible decrease in parental burden. 69 …”

Efficacy and safety of eltrombopag in the treatment of Chinese children with chronic immune thrombocytopenia

“…These therapeutics primarily aim to reduce platelet destruction, and splenectomy removes the site of platelet clearance and autoantibody production [8,9]. Meanwhile, a unique ITP therapy, namely applying thrombopoietin receptor agonists (TPO-RAs), improves platelet production by activating the thrombopoietin pathway [10][11][12][13]. The small-molecule TPO-RAs interact with thrombopoietin receptor on megakaryocytes and hematopoietic stem cells, stimulating megakaryocytes [14].…”