title: The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study.
abstract: 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).
  
  
  CONCLUSIONS
  Despite significant ABR/AJBR reduction in all 3 groups, the efficacy of the primary prophylaxis was better than the secondary and tertiary prophylaxes.
authors.affiliation: 1 Department of Haematology, Beijing Children's Hospital, Capital Medical University, Beijing, China.
authors.forename: Wanru
authors.initials: W
authors.lastname: Yao
authors.affiliation: 2 Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China.
authors.forename: Juan
authors.initials: J
authors.lastname: Xiao
authors.affiliation: 1 Department of Haematology, Beijing Children's Hospital, Capital Medical University, Beijing, China.
authors.forename: Xiaoling
authors.initials: X
authors.lastname: Cheng
authors.affiliation: 1 Department of Haematology, Beijing Children's Hospital, Capital Medical University, Beijing, China.
authors.forename: Guoshuang
authors.initials: G
authors.lastname: Feng
authors.affiliation: 3 Department of Hematology and Oncology, Shenzhen Children's Hospital, Shenzhen, China.
authors.forename: Changgang
authors.initials: C
authors.lastname: Li
authors.affiliation: 4 Shangdong Blood Center, Jinan, China.
authors.forename: Xinsheng
authors.initials: X
authors.lastname: Zhang
authors.affiliation: 5 Department of Hematology, TongJi Medical College of HUST, Wuhan, China.
authors.forename: Qun
authors.initials: Q
authors.lastname: Hu
authors.affiliation: 6 Department of Hematology, The Children's Hospital, Zhejiang University School of Medicine, Hangzhou, China.
authors.forename: Weiqun
authors.initials: W
authors.lastname: Xu
authors.affiliation: 7 Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
authors.forename: Jing
authors.initials: J
authors.lastname: Sun
authors.affiliation: 8 Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China.
authors.forename: Renchi
authors.initials: R
authors.lastname: Yang
authors.affiliation: 9 Department of Hematology, Chengdu Women and Children's Central Hospital, Chengdu, China.
authors.forename: Xiaojing
authors.initials: X
authors.lastname: Li
authors.affiliation: 10 Department of Hematology, Nanjing Drum Tower Hospital, Nanjing University Medical School, Nanjing, China.
authors.forename: Rongfu
authors.initials: R
authors.lastname: Zhou
authors.affiliation: 11 Department of Hematology, Dalian Municipal Central Hospital, Dalian Medical University, Dalian, China.
authors.forename: Shimei
authors.initials: S
authors.lastname: Lian
authors.affiliation: 12 Department of Hematology, Northern Jiangsu People's Hospital, Clinical Medical School of Yangzhou University, Yangzhou, China.
authors.forename: Jian
authors.initials: J
authors.lastname: Gu
authors.affiliation: 13 Department of Medical Affairs, Bayer Healthcare Co Ltd, Building Bayer Center, Beijing, China.
authors.forename: Junde
authors.initials: J
authors.lastname: Wu
authors.affiliation: 2 Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China.
authors.forename: Yongqiang
authors.initials: Y
authors.lastname: Zhao
authors.affiliation: 1 Department of Haematology, Beijing Children's Hospital, Capital Medical University, Beijing, China.
authors.forename: Runhui
authors.initials: R
authors.lastname: Wu
mesh_terms: D000293:Adolescent; D044466:Asian People; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000008:administration & dosage / Q000627:therapeutic use*; D006395:Hemarthrosis / Q000188:drug therapy / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D011292:Premedication / Q000379:methods; D011994:Recombinant Proteins / Q000627:therapeutic use; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D005169:Factor VIII
keywords: China; annual bleeding rate; annual joint bleeding rate; low-dose prophylaxis; rFVIII-FS; severe hemophilia A