The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study
机构：[1]Department of Haematology, Beijing Children’s Hospital, Capital Medical University, Beijing, China医技科室血液中心首都医科大学附属北京儿童医院[2]Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China[3]Department of Hematology and Oncology, Shenzhen Children’s Hospital, Shenzhen, China[4]Shangdong Blood Center, Jinan, China[5]Department of Hematology, TongJi Medical College of HUST, Wuhan, China[6]Department of Hematology, The Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China[7]Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China[8]Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China[9]Department of Hematology, Chengdu Women and Children’s Central Hospital, Chengdu, China[10]Department of Hematology, Nanjing Drum Tower Hospital, Nanjing University Medical School, Nanjing, China[11]Department of Hematology, Dalian Municipal Central Hospital, Dalian Medical University, Dalian, China[12]Department of Hematology, Northern Jiangsu People’s Hospital, Clinical Medical School of Yangzhou University, Yangzhou, China内科系统血液内科江苏省人民医院[13]Department of Medical Affairs, Bayer Healthcare Co Ltd, Building Bayer Center, Beijing, China
Objective: This study explores the efficacy of recombinant factor VIII (rFVIII) low-dose prophylaxis in Chinese pediatric patients with severe hemophilia A from the Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFVIII Contained Regular Prophylaxis (ReCARE) population. Methods: This is additional analysis of the multicenter, retrospective ReCARE study, in which the annual bleeding rate (ABR), annual joint bleeding rate (AJBR), and safety of > 12-week, low dose (10-30 IU/kg/wk) rFVIII prophylaxis divided into primary, secondary, and tertiary groups based on the joint status and joint bleeding history were analyzed. Results: A total of 57 patients (median age: 8.2 [0.4-17.3] years) from the ReCARE study receiving primary (n = 3), secondary (n = 21), and tertiary (n = 33) prophylaxes were included. Low-dose prophylaxis had significant bleeding reduction in all 3 groups compared to the baseline (S = 408.5, P < .001), with median ABR rates of -4.0 (-8.0 to -3.1), -4.0 (-24.0 to 8.0), and -13.9 (-110.6 to 20.6) in the primary, secondary, and tertiary groups, respectively, with a significant difference between the secondary and tertiary groups (P = .008). Median AJBR reduction rates were -2.3 (-6.3 to 8.4) and -14.9 (-61.5 to 19.1) in the secondary and tertiary groups, respectively. But there was no significant difference in AJBRs between the secondary and tertiary groups (P = .061), which was related to damaged joint status. Hence, longer prophylaxis was associated with better prevention of joint bleeding (P = .024). Conclusion: Despite significant ABR/AJBR reduction in all 3 groups, the efficacy of the primary prophylaxis was better than the secondary and tertiary prophylaxes.
基金：
1Department of Haematology, Beijing Children’s Hospital, Capital Medical University, Beijing, China 2Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China 3Department of Hematology and Oncology, Shenzhen Children’s Hospital, Shenzhen, China 4Shangdong Blood Center, Jinan, China 5Department of Hematology, TongJi Medical College of HUST, Wuhan, China 6Department of Hematology, The Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China 7Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China 8Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China 9Department of Hematology, Chengdu Women and Children’s Central Hospital, Chengdu, China 10Department of Hematology, Nanjing Drum Tower Hospital, Nanjing University Medical School, Nanjing, China 11Department of Hematology, Dalian Municipal Central Hospital, Dalian Medical University, Dalian, China 12Department of Hematology, Northern Jiangsu People’s Hospital, Clinical Medical School of Yangzhou University, Yangzhou, China 13Department of Medical Affairs, Bayer Healthcare Co Ltd, Building Bayer Center, Beijing, China
第一作者机构：[1]Department of Haematology, Beijing Children’s Hospital, Capital Medical University, Beijing, China
通讯作者：
通讯机构：[1]Department of Haematology, Beijing Children’s Hospital, Capital Medical University, Beijing, China[*1]Department of Haematology, Beijing Children’s Hospital, Capital Medical University, No. 56 Nanlishi Road, Beijing 100045, China
推荐引用方式(GB/T 7714)：
Wanru Yao,Juan Xiao,Xiaoling Cheng,et al.The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study[J].CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS.2017,23(7):851-858.doi:10.1177/1076029616679507.
APA：
Wanru Yao,Juan Xiao,Xiaoling Cheng,Guoshuang Feng,Changgang Li...&Runhui Wu.(2017).The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study.CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS,23,(7)
MLA：
Wanru Yao,et al."The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study".CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS 23..7(2017):851-858
