Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study.
The first recombinant factor VIII (rFVIII) product was launched in China in 2007. However, until now, no study has been conducted to describe the efficacy and safety of prophylaxis with rFVIII in Chinese pediatric patients with hemophilia A (HA).



To summarize the efficacy and safety data on prophylaxis with rFVIII in Chinese pediatric patients with HA.



ReCARE (Retrospective study in Chinese pediatric hemophilia A patients with rFVIII contained regular prophylaxis) was a retrospective study conducted in 12 hemophilia treatment centers (HTCs) across China. The primary endpoints included reduction in annualized bleeding rate (ABR); the secondary endpoints included evaluation of joint function (number and sites of target joints) using Gilbert score and Hemophilia Joint Health Score (HJHS), quality of life (QoL) and factors affecting treatment choices. Safety assessment of rFVIII was also conducted.



We analyzed a total of 183 male pediatric patients (mean age, 7.1 ± 4.23 years) who received prophylaxis between 1 November 2007 and 31 May 2013. Compared with baseline, prophylaxis with rFVIII significantly reduced overall annualized joint bleed rate (AJBR) (p < .001) and ABR (p < .001). Inhibitor formation was reported in 5 (2.7%) patients and hemarthrosis was reported in 1 patient. The mean number of target joints was positively related to age (p < .001) and weight (p = .003) at baseline. Responses from survey questionnaires reported that effective bleeding control, joint protection, improvement in quality of life, favorable medical insurance policies, and economic capability were reasons for choosing prophylaxis.



Prophylaxis with rFVIII reduced bleeding and number of target joints, even with a low-dose regimen, in Chinese pediatric patients with HA. Other than the efficacy and safety, factors such as poor disease control, improved economic stability and stable financial support made prophylaxis as an attractive treatment option. ClinicalTrials.gov ID: NCT02263066.
a Department of Hematology and Oncology , Shenzhen Children's Hospital , Shenzhen , China.
Changgang
C
Li
b Hemophilia Treatment Centre, Shandong Blood Centre , China.
Xinsheng
X
Zhang
c Department of Hematology , Peking Union Medical College Hospital , Beijing , China.
Yongqiang
Y
Zhao
d Hematology/Oncology Centre, Beijing Children's Hospital, Capital Medical University , Beijing , China.
Runhui
R
Wu
e Department of Hematology , Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology (HUST) , Hankou, Wuhan , China.
Qun
Q
Hu
f Department of Hematology , The Children's Hospital, Zhengjiang University School of Medicine , Hangzhou , China.
Weiqun
W
Xu
g Department of Hematology , Nan fang Hospital, Southern Medical University , Guangzhou , China.
Jing
J
Sun
h Thrombosis and Hemostasis Centre , Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College , Tianjin , China.
Renchi
R
Yang
i Department of Hematology , Chengdu Women & Children's Central Hospital , Chengdu , China.
Xiaojing
X
Li
j Department of Hematology , Nanjing Drum Tower Hospital, Nanjing University Medical School , Nanjing , China.
Rongfu
R
Zhou
k Department of Hematology , Dalian Municipal Central Hospital, Dalian Medical University , Dalian , China.
Shinmei
S
Lian
l Department of Hematology , Northern Jiangsu People's Hospital, Clinical Medical School of Yangzhou University , Yangzhou.
Jian
J
Gu
m Bayer HealthCare Pharmaceuticals Inc., Bayer Centre , Beijing , China.
Junde
J
Wu
m Bayer HealthCare Pharmaceuticals Inc., Bayer Centre , Beijing , China.
Qingsong
Q
Hou
D044466:Asian People; D002648:Child; D002675:Child, Preschool; D002681:China; D004334:Drug Administration Schedule; D005169:Factor VIII / Q000008:administration & dosage*; D006395:Hemarthrosis / Q000453:epidemiology; D006467:Hemophilia A / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D008297:Male; D011788:Quality of Life; D012189:Retrospective Studies
D005169:Factor VIII
Hemophilia A; Kogenate; Pediatric; Prophylaxis; ReCARE; annualized bleeding rate; rFVIII