title: Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A.
abstract: BACKGROUND
  Hemophilia A is an X-linked bleeding disorder that results from insufficient levels of factor VIII (FVIII) coagulant activity.
  
  
  OBJECTIVE
  To evaluate the efficacy and safety of ADVATE rAHF-PFM (Baxter Healthcare Corporation), a recombinant FVIII concentrate manufactured without human or bovine blood-derived additives, and to assess the effect of compliance with prophylactic use in preventing bleeding episodes (BEs).
  
  
  METHODS
  Clinical data were integrated from six prospective studies. Two hundred thirty-four hemophilia A subjects (FVIII levels < or = 2%) (median age 14.7 (range: 0.02 - 72.7) years) were included.
  
  
  RESULTS
  BEs were managed with one or two infusions and nearly all (1953/1956) responded to treatment. Compliance with a prophylactic treatment regimen significantly reduced the incidence of BEs (p = 0.0061) and prevented non-traumatic joint BEs (median annualized BE rate was 0). One previously treated subject developed an inhibitor; no other safety concerns were observed.
  
  
  CONCLUSIONS
  These results reinforce the efficacy and safety of rAHF-PFM and suggest that compliance is an essential contributor to the effectiveness of prophylaxis in the treatment of hemophilia A.
authors.affiliation: Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Rd, Suite 420, Indianapolis, IN 46260, USA. ashapiro@ihtc.org
authors.forename: Amy
authors.initials: A
authors.lastname: Shapiro
authors.forename: Ralph
authors.initials: R
authors.lastname: Gruppo
authors.forename: Ingrid
authors.initials: I
authors.lastname: Pabinger
authors.forename: Peter W
authors.initials: PW
authors.lastname: Collins
authors.forename: Charles R M
authors.initials: CR
authors.lastname: Hay
authors.forename: Phillip
authors.initials: P
authors.lastname: Schroth
authors.forename: Kathleen
authors.initials: K
authors.lastname: Casey
authors.forename: Lisa
authors.initials: L
authors.lastname: Patrone
authors.forename: Hartmut
authors.initials: H
authors.lastname: Ehrlich
authors.forename: Bruce M
authors.initials: BM
authors.lastname: Ewenstein
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000627:therapeutic use*; D006467:Hemophilia A / Q000276:immunology / Q000628:therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D007223:Infant; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D010349:Patient Compliance; D011446:Prospective Studies; D012449:Safety; D012709:Serum Albumin / Q000172:deficiency; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D012709:Serum Albumin; C078147:F8 protein, human; D005169:Factor VIII