title: Efficacy, Safety, and Pharmacokinetics of Beroctocog Alfa in Patients Previously Treated for Hemophilia A.
abstract: OBJECTIVE
  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 ≥12 years previously treated for severe hemophilia A.
  
  
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Internal Medicine, Yonsei University Wonju College of Medicine, Wonju, Korea.
authors.forename: Shin Young
authors.initials: SY
authors.lastname: Hyun
authors.affiliation: Department of Hematology and Oncology, Seoul National University College of Medicine, Seoul, Korea.
authors.forename: Seon Yang
authors.initials: SY
authors.lastname: Park
authors.affiliation: Department of Pediatrics, Inje University College of Medicine, Busan Paik Hospital, Busan, Korea.
authors.forename: Soon Yong
authors.initials: SY
authors.lastname: Lee
authors.affiliation: Department of Pediatrics, Chonnam National University Hwasun Hospital, Hwasun, Korea.
authors.forename: Hoon
authors.initials: H
authors.lastname: Kook
authors.affiliation: Green Cross Research Center, Green Cross Corporation, Yongin, Korea.
authors.forename: Sang Hoon
authors.initials: SH
authors.lastname: Paik
authors.affiliation: Department of Clinical Pharmacology & Therapeutics, Seoul National University College of Medicine and Hospital, Seoul, Korea.
authors.forename: In-Jin
authors.initials: IJ
authors.lastname: Jang
authors.affiliation: Department of Pediatrics, Kyungpook National University School of Medicine, Daegu, Korea. kslee@knu.ac.kr.
authors.forename: Kun Soo
authors.initials: KS
authors.lastname: Lee
mesh_terms: D000328:Adult; D003257:Consumer Product Safety; D004417:Dyspnea; D005169:Factor VIII / Q000009:adverse effects / Q000493:pharmacokinetics* / Q000627:therapeutic use; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006487:Hemostasis; D006488:Hemostasis, Surgical / Q000379:methods; D006801:Humans; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000009:adverse effects / Q000493:pharmacokinetics* / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; C427184:recombinant factor VIII SQ; D005169:Factor VIII
keywords: B-domain-deleted factor VIII; Hemophilia A; factor VIII