ORIGINAL RESEARCH article
Front. Pharmacol.
Sec. Obstetric and Pediatric Pharmacology
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1597641
This article is part of the Research TopicExploring Adverse Drug Reactions, Medication Adherence, and Forensic Markers in Pediatrics and ObstetricsView all 11 articles
The Establishment of an Expected Concentration Reference Range for Eltrombopag in the Individualized Treatment of Pediatric Immune Thrombocytopenia
Provisionally accepted
Nan Wang1*
Shuyue Dong2
Yixin Sun2
Jingjing Liu2
Zhifa Wang2
Jingyao Ma2
Xiaoling Wang2
Runhui WU2
Xiaoling Cheng2*- 1China Pharmaceutical University, Nanjing, China
- 2Beijing Children’s Hospital, Capital Medical University, Beijing, Beijing Municipality, China
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Background: Differences in the clinical efficacy and adverse drug reaction (ADR) of eltrombopag (ELT) in children with immune thrombocytopenia (ITP) may be positively correlated with the serum trough concentration of ELT. Individual pharmacokinetic variations primarily contribute to differences in ELT concentration among individuals. This study is the first to establish an expected concentration reference range for ELT in treating pediatric persistent/chronic ITP (P/CITP) across different age groups. Methods: A total of 94 patients with 111 serum trough concentrations were analyzed to validate this range. The median age of patients was 7.68 (5.35, 10.21) years, and 44.7% (44/94) were male. Results: Subgroup analyses revealed significant differences in ELT concentration related to age, efficacy, and ADR occurrence. The expected concentration reference range was determined using the dose-related concentration (DRC) factor combined with ELT dosage. The DRC factor ranges were as follows: 0.083-0.216 (mg/L)/mg in children aged 1-6 years, 0.058-0.125 (mg/L)/mg in children aged 7-12 years, and 0.043-0.097 (mg/L)/mg in children aged 13-18 years.Among patients with measured trough concentrations within the expected reference range, 84.3% (59/70) achieved response/complete response (R/CR), and 88.6% (62/70) did not experience ADR.: Serum trough concentration monitoring based on the established reference ranges could enhance the precision of individualized ELT therapy in pediatric ITP patients.
Keywords: eltrombopag, immune thrombocytopenia, Children, Individualized treatment, Therapeutic drug monitoring
Received: 21 Mar 2025; Accepted: 16 Jul 2025.
Copyright: © 2025 Wang, Dong, Sun, Liu, Wang, Ma, Wang, WU and Cheng. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Nan Wang, China Pharmaceutical University, Nanjing, China
Xiaoling Cheng, Beijing Children’s Hospital, Capital Medical University, Beijing, 100045, Beijing Municipality, China
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