PB0604 - Plasma concentration-adjusted dose of eltrombopag for the individualised treatment of immune thrombocytopenia in children：apilot study from Beijing Children’s Hospital

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

SD

Shuyue Dong

Resident physician
Department II of Hematology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)

Presenting Author(s)

Zhifa Wang, PhD

Resident physician
Department II of Hematology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)

Co-Author(s)

NW

Nan Wang, Master student

Department of Pharmacy
Beijing Children’s Hospital, Capital Medical University,
Beijing, Beijing, China (People's Republic)
Jingyao Ma, MD

attending physician
Hematology Department, National Center for Children’s Health, Beijing Children’s Hospital, Capital Medical University, Beijing 101300, China.
Beijing, Beijing, China (People's Republic)
JM

Jinxi Meng

Resident physician
Department II of Hematology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)
XC

Xiaoling Cheng

Department of Pharmacy
Beijing Children’s Hospital, Capital Medical University,
Beijing, Beijing, China (People's Republic)
Zhenping Chen

Associate professor
Hematologic Diseases Laboratory, Hematology Center, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, ChinaChildren’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)
Runhui Wu, PhD, MD

Professor
Department of Hematology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)

Background: Eltrombopag is recommended for pediatric primary immune thrombocytopenia (ITP). However, there are variations in individual eltrombopag concentrations that may impact its efficacy and adverse drug reactions (ADRs).

Aims: We tailored eltrombopag dosage based on concentration combined with bleeding symptoms and platelet counts, then followed up to assess the value of concentration in pediatric ITP.

Methods: This is a single-center, prospective, longitudinal cohort study. Patients who received eltrombopag and underwent concentration detection were enrolled. Based on previous concentration detection data, patients with bleeding or platelet count below 50×109/L were prescribed an increased dose if the concentration was below 4.33 mg/L. Conversely, patients with ADRs discontinued eltrombopag when the concentration was higher than 4.33 mg/L. Efficacy and ADRs were recorded and analyzed.

Results: A total of 39 patients were enrolled, of which 13 increased their dose and 3 discontinued. 3 patients suffered an elevated platelet count exceeding 400×109/L and discontinued eltrombopag. Their concentrations were markedly higher than those of patients without dose adjustment (t=3.606, p=0.001). 13 patients experienced bleeding or platelet count below 50×109/L and increased their dose. Compared to patients without dose adjustment, their concentration and platelet count was significantly lower (t=2.461, p=0.019; t=4.633, p< 0.001). Two months after increasing their dose, the number of patients reaching CR and R respectively increased by 2 and 3, while the platelet count was significantly raised (Z=-2.411, p=0.016).

Conclusion(s): Plasma concentration serves as a benchmark for managing pediatric ITP with eltrombopag. Patients could tailor eltrombopag dose based on concentration to improve efficacy and minimize ADRs.