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Pharmacokinetic Studies of Factor VIII in Chinese Boys with Severe Hemophilia A: A Single-Center Study

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收录情况: ◇ SCIE ◇ 统计源期刊 ◇ CSCD-C ◇ 中华系列

机构: [1]Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing 100045, China [2]Department of Pediatrics and Laboratory Medicine, and Hemophilia Clinic, Children’s Hospital of Eastern Ontario and University of Ottawa, Ottawa, Ontario, K1H 8L1, Canada [3]Department of Pediatrics and Child Health Evaluative Sciences, Division of Hematology/Oncology, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, Ontario, M5G 1X8, Canada [4]Department of Medicine, Pediatrics and Oncology, and Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, University of Calgary, Foothills Hospital and Calgary Health Region, Calgary, Alberta, T2N2T9, Canada [5]Department of Laboratory Medicine, Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai 200025, China
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关键词: Boys Factor VIII Hemophilia A Pharmacokinetics

摘要:
Background: Although much attention has been paid to the pharmacokinetics (PKs) of different factor VIII (FVIII) concentrates in persons with hemophilia A (HA), limited information is available in young boys with severe HA. In this study, we aimed to assess the PK parameters of FVIII products in boys with severe HA in China. Methods: A total of 36 boys (plasma-derived [pd]-FVIII, n = 15; recombinant [r] FVIII, n = 21) were enrolled between January 2015 and May 2016 in Beijing Children's Hospital. PK characteristics of FVIII products were studied according to a reduced 4-sampling time point design (1 h, 9 h, 24 h, and 48 h postinfusion). Results: The mean FVIII half-life (t(1/2)) was 10.99 +/- 3.45 h (range 5.52-20.02 h), the mean in vivo recovery (IVR) was 2.01 +/- 0.42 IU/dl per rU/kg (range 1.24-3.02 IU/l per IU/kg) and mean clearance (CL) of FVIII is 4.34 +/- 1.58 ml'kg(-1) h(-1) (range 2.29-7.90 ml.kg(-1) h(-1)). We also analyzed the influence of several parameters that potentially modulate FVIII PK. The age was closely associated with FVIII half-life (R-2 = 0.32, P < 0.01). The t(1/2) of FVIII increased by 0.59 h per year. Besides age, von Willebrand factor antigen (VWF: Ag) also was associated with FVIII half-life (R-2 = 0.52, P < 0.01). Patients with blood Group O had a shorter FVIII half-life than patients with non-O blood group (9.40 +/- 0.68 h vs. 12.3 +/- 0.79 h, t = 2.70, P = 0.01). The FVIII IR correlated with age (R-2 = 0.21, P < 0.01) and VWF: Ag level (R-2 = 0.28, P < 0.01). CL rates were faster in young patients and in those with low-VWF: Ag levels. CL rates of FVIII are higher in blood Group O versus non-blood Group O persons (5.02 +/- 0.38 vs. 4.00 +/- 0.32 ml.kg(-1) h(-1), t = 2.53, P = 0.02). Conclusions: Chinese boys with severe HA have similar PK values to other ethnic groups and large differences in FVIII PK between individual patients. Age, blood group, and VWF: Ag levels are important determining factors for FVIII CL.

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出版当年[2017]版:
大类 | 4 区 医学
小类 | 4 区 医学:内科
最新[2023]版:
大类 | 3 区 医学
小类 | 3 区 医学:内科
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出版当年[2016]版:
Q3 MEDICINE, GENERAL & INTERNAL
最新[2023]版:
Q1 MEDICINE, GENERAL & INTERNAL

影响因子: 最新[2023版] 最新五年平均 出版当年[2016版] 出版当年五年平均 出版前一年[2015版] 出版后一年[2017版]

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第一作者机构: [1]Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing 100045, China
通讯作者:
通讯机构: [1]Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing 100045, China [*1]Beijing Children’s Hospital, Capital Medical University, 56 Nanlishi Road, Beijing 100045, China
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