INTRODUCTION: Nuwiq(  ) [ @entity1 cell line-derived recombinant factor VIII ( @entity1 -cl rhFVIII)] is a new generation rFVIII protein, without chemical modification or fusion to any other protein, produced in a @entity1 cell line. AIM/METHODS: This prospective, open-label, multinational phase III study assessed the efficacy and safety of @entity1 -cl rhFVIII in 32 adult previously treated @entity1 (PTPs) with severe @entity447 during standard prophylaxis for >= 6  months and >= 50 exposure days. Efficacy in treating @entity548 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 @entity1 for all @entity548 and in 100% of @entity1 for spontaneous @entity548 . Mean (SD) annualized bleeding rate was 2.28 (3.73) [median  =  0.9] for all @entity548 , 1.16 (2.57) [median  =  0] for spontaneous @entity548 and 1.00 (1.79) [median  =  0] for traumatic @entity548 . There were no @entity548 in 50% of @entity1 and there were no major, life-threatening @entity548 . Efficacy was 'excellent' or 'good' in treating 28 (100%) of 28 @entity548 . 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(-1) for all measurements and stable over 6  months. No @entity1 developed FVIII inhibitors and there were no treatment-related serious or severe adverse events. CONCLUSION: These results in adult PTPs indicate that @entity1 -cl rhFVIII is effective for the prevention and treatment of @entity548 in adults with severe @entity447 .

Novel, @entity1 cell line-derived recombinant factor VIII ( @entity1 -cl rhFVIII; Nuwiq(  ) ) in adults with severe XXXX : efficacy and safety.

@entity1 :: ('9606', 'Species') :: ['human', 'patients'] @entity548 :: ('MESH:D006470', 'Disease') :: ['bleeds'] @entity447 :: ('MESH:D006467', 'Disease') :: ['haemophilia A']
@entity447 :: (MESH:D006467,Disease) :: ['haemophilia A']
