Chang Hsiu-Hao, Yang Yung-Li, Hung Mei-Hua, Tsay Woei, Shen Ming-Ching
Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
J Formos Med Assoc. 2007 Apr;106(4):281-7. doi: 10.1016/S0929-6646(09)60253-9.
BACKGROUND/PURPOSE: The pharmacokinetic (PK) study of recombinant human factor IX (rFIX) has been done in patients with hemophilia B in areas other than Taiwan. However, wide patient-related variability in recovery is noted and there is no PK study of rFIX in Taiwanese patients with hemophilia B. The purpose of this study is to evaluate the PKs of the rFIX in Taiwanese patients with hemophilia B.
A PK study of rFIX was performed in 10 previously treated Taiwanese patients with hemophilia B. Nine of them had severe hemophilia B and the other one had moderately severe hemophilia B. The mean age of our patients was 24.7 +/- 8.6 years (mean +/- SD) with a range of 15.5-47.0 years.
The infusion of 75 IU/kg of rFIX in our patients resulted in a mean FIX activity increase of 1.08 +/- 0.27 IU/dL per IU/kg with a range of 0.3-1.49 IU/dL per IU/kg, and a mean in vivo recovery of 44.0 +/- 9.5% with a range of 31.4-60.4%. The mean elimination half-life was 24.4 +/- 6.4 hours with a range of 14.1-35.3 hours. The mean values of area under the curve, total body clearance, volume of distribution at steady state were 1363 +/- 166 IU x hour/dL, 4.84 +/- 1.03 mL/hour/kg, and 144.3 +/- 41.8 mL/kg, respectively. In addition, rFIX was well tolerated in our patients. Only two patients experienced mild treatment-related adverse events, including fever and feeling sleepy, respectively. Both of them resolved spontaneously without any sequences, and there was no thrombosis formation or inhibitor development in the following 6 months after rFIX infusion.
The results of PK study of rFIX in our previously treated Taiwanese patients with hemophilia B are comparable to the reported results from other related studies conducted in different geographical areas.
背景/目的：重组人凝血因子IX（rFIX）的药代动力学（PK）研究已在台湾以外地区的B型血友病患者中开展。然而，已注意到患者间的回收率存在很大差异，且台湾B型血友病患者中尚无rFIX的PK研究。本研究的目的是评估台湾B型血友病患者中rFIX的药代动力学。
对10例既往接受过治疗的台湾B型血友病患者进行了rFIX的PK研究。其中9例为重度B型血友病，1例为中度重度B型血友病。我们患者的平均年龄为24.7±8.6岁（平均±标准差），范围为15.5 - 47.0岁。
在我们的患者中输注75 IU/kg的rFIX后，FIX活性平均每IU/kg增加1.08±0.27 IU/dL，范围为每IU/kg 0.3 - 1.49 IU/dL，体内平均回收率为44.0±9.5%，范围为31.4 - 60.4%。平均消除半衰期为24.4±6.4小时，范围为14.1 - 35.3小时。曲线下面积、总体清除率、稳态分布容积的平均值分别为1363±166 IU·小时/dL、4.84±1.03 mL/小时/kg和144.3±41.8 mL/kg。此外，我们的患者对rFIX耐受性良好。仅2例患者分别经历了与治疗相关的轻度不良事件，包括发热和嗜睡。两者均自行缓解，无任何后遗症，且在rFIX输注后的接下来6个月内未形成血栓或出现抑制剂。
我们既往接受过治疗的台湾B型血友病患者中rFIX的PK研究结果与在不同地理区域进行的其他相关研究报告的结果相当。
