Significant PK variability of plasma-derived FIX concentrates in chinese children with Haemophilia B: A fixed single-dose study of factor IX-CTBB
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.
Authors
- Runhui Wu (ORCID: https://orcid.org/0000-0003-4030-209X)
- Wanru Yao (ORCID: https://orcid.org/0000-0001-7080-3587)
- Di Ai (ORCID: https://orcid.org/0000-0002-9987-9099)
- Zhenping Chen (ORCID: https://orcid.org/0000-0002-5273-145X)
- Yingzi Zhen
- Gang Li (ORCID: https://orcid.org/0009-0002-1292-1723)
- Zekun Li (ORCID: https://orcid.org/0000-0002-1421-4590)
- Guoqing Liu (ORCID: https://orcid.org/0000-0002-5428-1720)
- Yaohan Zhou
- Xinyi Wu (ORCID: https://orcid.org/0000-0003-3288-7489)
- Yunyun Wei (ORCID: https://orcid.org/0000-0003-2547-2402)
Institutions
- Ministry of Education of the People's Republic of China (CN)
- Children's National (US)
- Capital Medical University (CN)
- Beijing Children’s Hospital (CN)
Publication Details
- Journal
- Global Medical Genetics
- Published
- 2025-02-06
- DOI
- https://doi.org/10.1016/j.gmg.2025.100040
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Beijing Municipal Administration of Hospitals