Efficacy, Safety, and Pharmacokinetics of Beroctocog Alfa in Patients Previously Treated for Hemophilia A.
10.3349/ymj.2015.56.4.935
- Author:
Shin Young HYUN
1
;
Seon Yang PARK
;
Soon Yong LEE
;
Hoon KOOK
;
Sang Hoon PAIK
;
In Jin JANG
;
Kun Soo LEE
Author Information
1. Department of Internal Medicine, Yonsei University Wonju College of Medicine, Wonju, Korea.
- Publication Type:Original Article ; Research Support, Non-U.S. Gov't
- Keywords:
Hemophilia A;
factor VIII;
B-domain-deleted factor VIII
- MeSH:
Adult;
Consumer Product Safety;
Dyspnea;
Factor VIII/adverse effects/*pharmacokinetics/therapeutic use;
Female;
Hemophilia A/*drug therapy;
Hemorrhage/prevention & control;
Hemostasis;
Hemostasis, Surgical/methods;
Humans;
Male;
Middle Aged;
Prospective Studies;
Recombinant Proteins/adverse effects/*pharmacokinetics/*therapeutic use;
Treatment Outcome
- From:Yonsei Medical Journal
2015;56(4):935-943
- CountryRepublic of Korea
- Language:English
-
Abstract:
PURPOSE: Beroctocog alfa is a second generation recombinant factor VIII manufactured by removing the B-domain from factor VIII. This prospective clinical trial was conducted to evaluate the efficacy, safety, and pharmacokinetics of beroctocog alfa in patients of ages > or =12 years previously treated for severe hemophilia A. MATERIALS AND METHODS: Seventy subjects received beroctocog alfa as an on-demand treatment for acute hemorrhage. RESULTS: The final hemostatic effect was excellent in 35 subjects (50%) and good in 26 subjects (37.1%). The drug showed an overall efficacy rate of 87.1%. The majority of acute hemorrhages was treated by administering the study drug once (86.2%) or twice (10.0%), and the mean dose administered per single infusion was 28.55+/-6.53 IU/kg. Ten subjects underwent 12 surgical procedures, and hemostatic efficacy was excellent in seven cases (58.3%) and good in five cases (41.7%), showing a 100% efficacy rate. A total of 52 of 88 subjects (59.0%) experienced 168 adverse events. There were 18 serious adverse events (10.7%) in 11 subjects, and two (mild dyspnea and facial edema) in one subject were related to the study drug. Only one subject formed a de novo factor VIII inhibitor, for an occurrence rate of 1.4% (one-sided 95% upper confidence limit: 3.85%). The final elimination half-life was 13.3 h and 12.6 h at baseline and 6 months after administration, respectively. CONCLUSION: Our results suggest that beroctocog alfa is safe and efficacious as either an on-demand treatment for acute hemorrhage or a surgical prophylaxis in patients with hemophilia A.
