Academic Journal
Significant PK variability of plasma-derived FIX concentrates in chinese children with Haemophilia B: A fixed single-dose study of factor IX-CTBB
| Title: | Significant PK variability of plasma-derived FIX concentrates in chinese children with Haemophilia B: A fixed single-dose study of factor IX-CTBB |
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| Authors: | Guoqing Liu, Di Ai, Gang Li, Yingzi Zhen, Xinyi Wu, Wanru Yao, Yunyun Wei, Zekun Li, Yaohan Zhou, Runhui Wu, Zhenping Chen |
| Source: | Global Medical Genetics, Vol 12, Iss 2, Pp 100040- (2025) |
| Publisher Information: | KeAi Communications Co., Ltd., 2025. |
| Publication Year: | 2025 |
| Collection: | LCC:Genetics LCC:Neoplasms. Tumors. Oncology. Including cancer and carcinogens |
| Subject Terms: | Haemophilia B, Pharmacokinetics, Children, Plasma-derived FIX, Genetics, QH426-470, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282 |
| More Details: | Background: The pharmacokinetics (PK) characters of plasma-derived Factor IX (pdFIX) concentrate in Chinese children with Haemophilia B(HB) have not yet been reported. Aim: To assess the PK parameters of pdFIX in children with severe HB and identify factors that influence FIX PK. Methods: This non-randomized, open-label PK study enrolled children with severe HB (FIX≤2 %). Patients received 50 IU± 5 IU/kg pdFIX (Human coagulation Factor IX-CTBB) after at least 96 h wash-out period. Blood samples for PK assessments were collected before infusion (pre-dose) and at 15 min, 30 min, 1 h, 3 h, 6 h, 9 h, 24 h, 48 h, 72 h and 96 h post-infusion. FIX activity was measured by a one-stage assay. Results: Twenty patients were enrolled with a median age of 8.3 (range 1.8–15.4) years. The peak plasma levels of FIX: C in all patients were observed within 15 min. Their median terminal half-life (t1/2) was 32.6 (range 23.3–52.0) hours. The median values of in vivo recovery (IVR) at 15 min, clearance (CL), volume of distribution at state (Vss) and area under the curve (AUC) were 1.0 (0.9, 1.2) IU/dL per IU/kg, 5.2 (IQR 4.8, 6.4) mL/h/kg, 207.9 (IQR 183.5, 301.4) mL/kg, 9.77 (IQR 7.76, 11.23) U*h/mL respectively. The t1/2, Vss and mean residence time after intravenous injection (MRT) decreased with increasing age and body weight. Changes in CL with body weight were similar to t1/2, but no significant correlation exists with age. Conclusions: There is a significant inter-individual variability in PK profiles among Chinese children with severe HB, which is related to age and body weight changes, indicating the necessity of individualized prophylaxis driven by PK. |
| Document Type: | article |
| File Description: | electronic resource |
| Language: | English |
| ISSN: | 2699-9404 |
| Relation: | http://www.sciencedirect.com/science/article/pii/S2699940425000414; https://doaj.org/toc/2699-9404 |
| DOI: | 10.1016/j.gmg.2025.100040 |
| Access URL: | https://doaj.org/article/c81654c9c01f44058ede5ec714e23572 |
| Accession Number: | edsdoj.81654c9c01f44058ede5ec714e23572 |
| Database: | Directory of Open Access Journals |
| ISSN: | 26999404 |
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| DOI: | 10.1016/j.gmg.2025.100040 |
| Published in: | Global Medical Genetics |
| Language: | English |