title: Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients.
abstract: OBJECTIVE
  A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers.
  
  
  RESULTS
  Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII < 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies.
  
  
  CONCLUSIONS
  This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.
authors.affiliation: Hemophilia Treatment Center, Caen University Hospital, Caen, France.
authors.forename: A
authors.initials: A
authors.lastname: Borel-Derlon
authors.forename: A B
authors.initials: AB
authors.lastname: Federici
authors.forename: V
authors.initials: V
authors.lastname: Roussel-Robert
authors.forename: J
authors.initials: J
authors.lastname: Goudemand
authors.forename: C A
authors.initials: CA
authors.lastname: Lee
authors.forename: I
authors.initials: I
authors.lastname: Scharrer
authors.forename: C
authors.initials: C
authors.lastname: Rothschild
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
authors.forename: C
authors.initials: C
authors.lastname: Henriet
authors.forename: Z
authors.initials: Z
authors.lastname: Tellier
authors.forename: F
authors.initials: F
authors.lastname: Bridey
authors.forename: P M
authors.initials: PM
authors.lastname: Mannucci
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy / Q000517:prevention & control; D006487:Hemostasis / Q000187:drug effects; D006801:Humans; D008297:Male; D008875:Middle Aged; D019106:Postoperative Hemorrhage / Q000188:drug therapy / Q000517:prevention & control; D011446:Prospective Studies; D012449:Safety; D014842:von Willebrand Diseases / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000008:administration & dosage / Q000009:adverse effects / Q000302:isolation & purification / Q000627:therapeutic use*
chemical_list: D014841:von Willebrand Factor; D005169:Factor VIII