The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia
BackgroundDifferences in the clinical efficacy and adverse drug reactions (ADRs) 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 E...
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Frontiers Media S.A.
2025-08-01
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2025.1597641/full |
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| author | Nan Wang Nan Wang Shuyue Dong Yixin Sun Yixin Sun Jingjing Liu Jingjing Liu Zhifa Wang Jingyao Ma Xiaoling Wang Runhui Wu Xiaoling Cheng |
| author_facet | Nan Wang Nan Wang Shuyue Dong Yixin Sun Yixin Sun Jingjing Liu Jingjing Liu Zhifa Wang Jingyao Ma Xiaoling Wang Runhui Wu Xiaoling Cheng |
| author_sort | Nan Wang |
| collection | DOAJ |
| description | BackgroundDifferences in the clinical efficacy and adverse drug reactions (ADRs) 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.MethodsA 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% (42/49) of them were male.ResultsSubgroup analyses revealed significant differences in ELT concentration related to the age, efficacy, and ADR occurrence. The expected concentration reference range was determined using the dose-related concentration (DRC) factor combined with the 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.ConclusionSerum trough concentration monitoring based on the established reference ranges could enhance the precision of individualized ELT therapy in pediatric ITP patients. |
| format | Article |
| id | doaj-art-b87b263f373245708ef3f2e1ff4b454c |
| institution | Kabale University |
| issn | 1663-9812 |
| language | English |
| publishDate | 2025-08-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj-art-b87b263f373245708ef3f2e1ff4b454c2025-08-21T05:27:29ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122025-08-011610.3389/fphar.2025.15976411597641The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopeniaNan Wang0Nan Wang1Shuyue Dong2Yixin Sun3Yixin Sun4Jingjing Liu5Jingjing Liu6Zhifa Wang7Jingyao Ma8Xiaoling Wang9Runhui Wu10Xiaoling Cheng11Department of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, ChinaSchool of Basic Medicine and Clinical Pharmacy, China Pharmaceutical University, Nanjing, Jiangsu, ChinaDepartment of Hematology, Beijing Children’s Hospital, Hemophilia Comprehensive Care Center, National Center for Children’s Health, Capital Medical University, Beijing, ChinaDepartment of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, ChinaBeijing Children’s Hospital, Clinical Research Center, National Center for Children’s Health, Capital Medical University, Beijing, ChinaDepartment of Hematology, Beijing Children’s Hospital, Hemophilia Comprehensive Care Center, National Center for Children’s Health, Capital Medical University, Beijing, ChinaDepartment of Pediatric, Beijing Shijitan Hospital of Capital Medical University, Beijing, ChinaDepartment of Hematology, Beijing Children’s Hospital, Hemophilia Comprehensive Care Center, National Center for Children’s Health, Capital Medical University, Beijing, ChinaDepartment of Hematology, Beijing Children’s Hospital, Hemophilia Comprehensive Care Center, National Center for Children’s Health, Capital Medical University, Beijing, ChinaDepartment of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, ChinaDepartment of Hematology, Beijing Children’s Hospital, Hemophilia Comprehensive Care Center, National Center for Children’s Health, Capital Medical University, Beijing, ChinaDepartment of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, ChinaBackgroundDifferences in the clinical efficacy and adverse drug reactions (ADRs) 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.MethodsA 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% (42/49) of them were male.ResultsSubgroup analyses revealed significant differences in ELT concentration related to the age, efficacy, and ADR occurrence. The expected concentration reference range was determined using the dose-related concentration (DRC) factor combined with the 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.ConclusionSerum trough concentration monitoring based on the established reference ranges could enhance the precision of individualized ELT therapy in pediatric ITP patients.https://www.frontiersin.org/articles/10.3389/fphar.2025.1597641/fulleltrombopagimmune thrombocytopeniachildrenindividualized treatmenttherapeutic drug monitoring |
| spellingShingle | Nan Wang Nan Wang Shuyue Dong Yixin Sun Yixin Sun Jingjing Liu Jingjing Liu Zhifa Wang Jingyao Ma Xiaoling Wang Runhui Wu Xiaoling Cheng The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia Frontiers in Pharmacology eltrombopag immune thrombocytopenia children individualized treatment therapeutic drug monitoring |
| title | The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia |
| title_full | The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia |
| title_fullStr | The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia |
| title_full_unstemmed | The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia |
| title_short | The establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia |
| title_sort | establishment of an expected concentration reference range for eltrombopag in the individualized treatment of pediatric immune thrombocytopenia |
| topic | eltrombopag immune thrombocytopenia children individualized treatment therapeutic drug monitoring |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2025.1597641/full |
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