The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study

• Published in: Clinical and applied thrombosis/hemostasis Vol. 23; no. 7; pp. 851 - 858
• Main Authors: Yao, Wanru, Xiao, Juan, Cheng, Xiaoling, Feng, Guoshuang, Li, Changgang, Zhang, Xinsheng, Hu, Qun, Xu, Weiqun, Sun, Jing, Yang, Renchi, Li, Xiaojing, Zhou, Rongfu, Lian, Shimei, Gu, Jian, Wu, Junde, Zhao, Yongqiang, Wu, Runhui
• Format: Journal Article
• Language: English
• Published: Los Angeles, CA SAGE Publications 01.10.2017; SAGE PUBLICATIONS, INC
• Subjects: Adolescent; Asian Continental Ancestry Group; Child; Child, Preschool; Factor VIII - administration & dosage; Factor VIII - therapeutic use; Hemarthrosis - drug therapy; Hemarthrosis - prevention & control; Hemophilia; Hemophilia A - complications; Hemophilia A - drug therapy; Humans; Infant; Pediatrics; Premedication - methods; Recombinant Proteins - therapeutic use; Retrospective Studies; Treatment Outcome; rFVIII-FS; annual bleeding rate; annual joint bleeding rate; low-dose prophylaxis; severe hemophilia A; China
• ISSN: 1076-0296; 1938-2723
• DOI: 10.1177/1076029616679507

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.