title: Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety.
abstract: BACKGROUND
  Nuwiq®[human cell line-derived recombinant factor VIII (human-cl rhFVIII)] is a new generation rFVIII protein, without chemical modification or fusion to any other protein, produced in a human cell line.
  
  
  OBJECTIVE
  This prospective, open-label, multinational phase III study assessed the efficacy and safety of human-cl rhFVIII in 32 adult previously treated patients (PTPs) with severe haemophilia A during standard prophylaxis for ≥6 months and ≥50 exposure days. Efficacy in treating bleeds and during surgical prophylaxis was also assessed.
  
  
  RESULTS
  Prophylactic efficacy, based on mean monthly bleeding rate, was rated as 'excellent' or 'good' in 97% of patients for all bleeds and in 100% of patients for spontaneous bleeds. Mean (SD) annualized bleeding rate was 2.28 (3.73) [median = 0.9] for all bleeds, 1.16 (2.57) [median = 0] for spontaneous bleeds and 1.00 (1.79) [median = 0] for traumatic bleeds. There were no bleeds in 50% of patients and there were no major, life-threatening bleeds. Efficacy was 'excellent' or 'good' in treating 28 (100%) of 28 bleeds. Overall efficacy was rated as 'excellent' during four surgical procedures (three major, one minor) and 'moderate' during one major surgery. Incremental in vivo recovery (IVR) data were comparable with the one-stage and chromogenic assays. IVR was >2.0% per IU kg-1for all measurements and stable over 6 months. No patients developed FVIII inhibitors and there were no treatment-related serious or severe adverse events.
  
  
  CONCLUSIONS
  These results in adult PTPs indicate that human-cl rhFVIII is effective for the prevention and treatment of bleeds in adults with severe haemophilia A.
authors: Specialised Hospital for Active Treatment "Joan Pavel", Sofia, Bulgaria.
authors: T
authors: T
authors: Lissitchkov
authors: Royal Hallamshire Hospital, Sheffield, UK.
authors: K
authors: K
authors: Hampton
authors: Werlhof-Institut für Hämostaseologie GmbH, Hannover, Germany.
authors: M
authors: M
authors: von Depka
authors: Manchester Royal Infirmary, Manchester, UK.
authors: C
authors: C
authors: Hay
authors: Basingstoke and North Hampshire Hospital, Basingstoke, UK.
authors: S
authors: S
authors: Rangarajan
authors: Royal Free Hospital, London, UK.
authors: E
authors: E
authors: Tuddenham
authors: University Hospital Hamburg-Eppendorf, Hamburg, Germany.
authors: K
authors: K
authors: Holstein
authors: SRH Kurpfalzkrankenhaus and Hemophilia Center, Heidelberg, Germany.
authors: A
authors: A
authors: Huth-Kühne
authors: Medical University of Vienna, Vienna, Austria.
authors: I
authors: I
authors: Pabinger
authors: Octapharma AG, Lachen, Switzerland.
authors: S
authors: S
authors: Knaub
authors: Octapharma AG, Lachen, Switzerland.
authors: J
authors: J
authors: Bichler
authors: Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany.
authors: J
authors: J
authors: Oldenburg
keywords: Nuwiq; factor VIII inhibitors; haemophilia A; human cell line factor VIII; prophylaxis; recombinant factor VIII