title: The use of a single von Willebrand factor-containing, plasma-derived FVIII product in hemophilia A immune tolerance induction: the US experience.
abstract: BACKGROUND
  Inhibitors are a serious complication for patients with severe hemophilia A. Immune tolerance induction (ITI) is the primary method for eradicating these inhibitors. The role of type of concentrate and in particular the use of von Willebrand factor-containing, plasma-derived factor VIII (VWF/pd-FVIII) concentrate in primary or rescue ITI remains unclear.
  
  
  OBJECTIVE
  To report retrospective collection of data on the use of a single VWF/pd-FVIII concentrate in primary and rescue ITI.
  
  
  METHODS
  Retrospective chart review of hemophilia A inhibitor patients at 11 US institutions who received VWF/pd-FVIII concentrate in primary or rescue ITI.
  
  
  RESULTS
  Primary ITI was carried out in eight inhibitor patients with a 75% complete and partial success. Secondary ITI was carried out in 25 inhibitor patients, with 52% attaining complete or partial success.
  
  
  CONCLUSIONS
  This report represents the largest group of primarily pediatric, high-titer inhibitor patients treated with a single VWF/pd-FVIII concentrate. It adds retrospective data to the use of VWF-containing plasma-derived factor VIII concentrate in primary and rescue ITI, particularly in those patients with characteristics of poor response to ITI.
authors: Department of Pediatrics, Children's Hospitals and Clinics of Minnesota, Minneapolis, MN 55404, USA. Margaret.heisel@childrensmn.org
authors: M
authors: M
authors: Kurth
authors: J
authors: J
authors: Puetz
authors: P
authors: P
authors: Kouides
authors: J
authors: J
authors: Sanders
authors: C
authors: C
authors: Sexauer
authors: J
authors: J
authors: Bernstein
authors: R
authors: R
authors: Gruppo
authors: M
authors: M
authors: Manco-Johnson
authors: E J
authors: EJ
authors: Neufeld
authors: N
authors: N
authors: Rodriguez
authors: B
authors: B
authors: Wicklund
authors: D
authors: D
authors: Quon
authors: L
authors: L
authors: Aledort
mesh_terms: D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004338:Drug Combinations; D005169:Factor VIII / Q000276:immunology / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy* / Q000276:immunology; D006801:Humans; D007108:Immune Tolerance / Q000187:drug effects*; D007223:Infant; D012189:Retrospective Studies; D014481:United States; D014841:von Willebrand Factor / Q000276:immunology / Q000627:therapeutic use*
chemical_list: D004338:Drug Combinations; C509423:factor VIII, von Willebrand factor drug combination; D014841:von Willebrand Factor; D005169:Factor VIII