Efficacy of standard prophylaxis versus on-demand treatment with bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A

• Published in: Pediatric hematology and oncology Vol. 34; no. 3; pp. 146 - 156
• Main Authors: Zhao, Yongqiang, Xiao, Juan, Yang, Renchi, Wu, Runhui, Hu, Yu, Beckmann, Horst, Wu, Junde, Hou, Qingsong, Sun, Jing
• Format: Journal Article
• Language: English
• Published: England Taylor & Francis 03.04.2017
• Subjects: Adolescent; Asian Continental Ancestry Group; Child; Child, Preschool; China; Factor VIII - administration & dosage; Factor VIII - adverse effects; Female; Follow-Up Studies; hemophilia A; Hemophilia A - complications; Hemophilia A - drug therapy; Hemorrhage - etiology; Hemorrhage - prevention & control; Humans; Male; NCT01810666 (ClinicalTrials.gov); prophylaxis; rFVIII-FS; Sucrose - administration & dosage; Sucrose - adverse effects; Time Factors; rFVIII-FS; hemophilia A; NCT01810666 (ClinicalTrials.gov); Child; prophylaxis
• ISSN: 0888-0018; 1521-0669
• DOI: 10.1080/08880018.2017.1313921

In China, care of patients with severe hemophilia primarily involves insufficient dosing of on-demand treatment and secondary low-dose prophylaxis (10 IU/kg 2× /wk). We sought to evaluate 3× /wk, standard-dose prophylaxis with sucrose-formulated recombinant factor VIII (rFVIII-FS; Bayer) compared with on-demand treatment in Chinese children with severe hemophilia A. Children and adolescents aged 2-16 years with severe hemophilia A, no inhibitors, and no prophylaxis for >6 consecutive months before study entry were eligible for this 24-week, interventional, sequential-treatment study. Patients received rFVIII-FS on demand for 12 weeks followed by a 12-week prophylaxis period (25 IU/kg 3× /wk). The primary efficacy endpoint was comparison of the annualized bleeding rate (ABR) of all bleeds in the prophylaxis versus on-demand phase. Additional variables included ABR of joint bleeds, school attendance/activity, daily activity, and hemophilia Joint Health Score (HJHS). Thirty patients (median age, 12 years) were treated and analyzed. Compared with on-demand treatment, prophylaxis reduced median (quartile [Q1; Q3]) ABR of all bleeds (57.5 [44.5; 73.9] vs 0 [0; 4.0]) and joint bleeds (34.5 [26.1; 56.5] vs 0 [0; 4.0]). Median (range) total HJHS improved after both the prophylaxis and on-demand phases (8.0 [0-48.0] and 11.0 [0-55.0], respectively) compared with baseline (16.0 [0-56.0]). School attendance/activity and daily activity improved with prophylaxis versus on demand. No inhibitors or treatment-related adverse events were reported. In this first prospective, standard-dose, secondary prophylaxis study in China, rFVIII-FS prophylaxis reduced bleeding and improved health outcomes versus on-demand treatment in children with severe hemophilia A.