Wanru Yao1, Juan Xiao2, Xiaoling Cheng1, Guoshuang Feng1, Changgang Li3, Xinsheng Zhang4, Qun Hu5, Weiqun Xu6, Jing Sun7, Renchi Yang8, Xiaojing Li9, Rongfu Zhou10, Shimei Lian11, Jian Gu12, Junde Wu13, Yongqiang Zhao2, Runhui Wu1. 1. 1 Department of Haematology, Beijing Children's Hospital, Capital Medical University, Beijing, China. 2. 2 Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China. 3. 3 Department of Hematology and Oncology, Shenzhen Children's Hospital, Shenzhen, China. 4. 4 Shangdong Blood Center, Jinan, China. 5. 5 Department of Hematology, TongJi Medical College of HUST, Wuhan, China. 6. 6 Department of Hematology, The Children's Hospital, Zhejiang University School of Medicine, Hangzhou, China. 7. 7 Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China. 8. 8 Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China. 9. 9 Department of Hematology, Chengdu Women and Children's Central Hospital, Chengdu, China. 10. 10 Department of Hematology, Nanjing Drum Tower Hospital, Nanjing University Medical School, Nanjing, China. 11. 11 Department of Hematology, Dalian Municipal Central Hospital, Dalian Medical University, Dalian, China. 12. 12 Department of Hematology, Northern Jiangsu People's Hospital, Clinical Medical School of Yangzhou University, Yangzhou, China. 13. 13 Department of Medical Affairs, Bayer Healthcare Co Ltd, Building Bayer Center, Beijing, China.
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). CONCLUSION: Despite significant ABR/AJBR reduction in all 3 groups, the efficacy of the primary prophylaxis was better than the secondary and tertiary prophylaxes.
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 APatients 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.
Entities:
Keywords:
China; annual bleeding rate; annual joint bleeding rate; low-dose prophylaxis; rFVIII-FS; severe hemophilia A