Park Young-Shil, Yoo Ki-Young, Park Sang Kyu, Hwang Taiju, Jung Aeran, Choi Eun Jin
Department of Pediatrics, Kyung Hee University Hospital, Gangdong, Seoul, Republic of Korea.
Korea Hemophilia Foundation Clinic, Seoul, Republic of Korea.
Blood Res. 2024 Aug 28;59(1):29. doi: 10.1007/s44313-024-00023-9.
This study aimed to investigate the pharmacokinetics (PK) of factor VIII (FVIII) in Korean patients, as limited information is available on the PK of FVIII in this population.
We collected the FVIII PK results from patients with moderate-to-severe hemophilia A using myPKFiT. PK variations were assessed according to age, blood type, inhibitor history, von Willebrand factor antigen (vWF:Ag) level, and body mass index. Additionally, the correlation between the PK profile and prophylaxis regimen was specifically analyzed for each product in severe cases.
The PK data of 48 and 81 patients treated with octocog alfa and rurioctocog alfa pegol, respectively, were obtained. The median half-lives of octocog alfa and rurioctocog alfa pegol were 9.9 (range: 6.3-15.2) h and 15.3 (range: 10.4-23.9) h, respectively. The PK profiles for each product did not differ according to age group; however, blood type-O patients had shorter half-lives and time to 1% compared to non-blood type-O patients. In regression analysis, the PK of octocog alfa showed a statistically significant difference according to age, whereas the PK of rurioctocog alfa pegol correlated with vWF:Ag. Only the frequency of rurioctocog alfa pegol use showed a statistically significant difference in relation to time to 1%, although the coefficient of determination was small.
This study confirmed significant interpatient variation in the PK of FVIII among Korean patients with hemophilia A. To achieve optimized prophylaxis, personalizing the regimen based on the PK profile of each individual patient is essential.
本研究旨在调查韩国患者中凝血因子 VIII（FVIII）的药代动力学（PK），因为该人群中关于 FVIII PK 的信息有限。
我们使用 myPKFiT 收集了中重度甲型血友病患者的 FVIII PK 结果。根据年龄、血型、抑制剂史、血管性血友病因子抗原（vWF:Ag）水平和体重指数评估 PK 差异。此外，针对严重病例中的每种产品，专门分析了 PK 曲线与预防方案之间的相关性。
分别获得了 48 例和 81 例接受重组人凝血因子 VIII 和聚乙二醇化重组人凝血因子 VIII 治疗患者的 PK 数据。重组人凝血因子 VIII 和聚乙二醇化重组人凝血因子 VIII 的中位半衰期分别为 9.9（范围：6.3 - 15.2）小时和 15.3（范围：10.4 - 23.9）小时。每种产品的 PK 曲线在不同年龄组之间没有差异；然而，与非 O 型血患者相比，O 型血患者的半衰期和达到 1%水平的时间更短。在回归分析中，重组人凝血因子 VIII 的 PK 根据年龄显示出统计学上的显著差异，而聚乙二醇化重组人凝血因子 VIII 的 PK 与 vWF:Ag 相关。尽管决定系数较小，但只有聚乙二醇化重组人凝血因子 VIII 的使用频率在达到 1%水平的时间方面显示出统计学上的显著差异。
本研究证实了韩国甲型血友病患者中 FVIII 的 PK 存在显著的个体间差异。为实现优化预防，根据每个患者的 PK 曲线个性化制定治疗方案至关重要。
