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Novel, human cell line‐derived recombinant factor VIII (human‐cl rhFVIII; Nuwiq ® ) in adults with severe haemophilia A: efficacy and safety
Author(s) -
Lissitchkov T.,
Hampton K.,
Depka M.,
Hay C.,
Rangarajan S.,
Tuddenham E.,
Holstein K.,
HuthKühne A.,
Pabinger I.,
Knaub S.,
Bichler J.,
Oldenburg J.
Publication year - 2016
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.12793
Subject(s) - medicine , haemophilia a , haemophilia , adverse effect , haemophilia b , surgery , clinical trial
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. Aim/methods This prospective, open‐label, multinational phase III study assessed the efficacy and safety of human‐cl rhFVIII in 32 adult previously treated patients ( PTP s) 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 −1 for all measurements and stable over 6 months. No patients developed FVIII inhibitors and there were no treatment‐related serious or severe adverse events. Conclusion These results in adult PTP s indicate that human‐cl rhFVIII is effective for the prevention and treatment of bleeds in adults with severe haemophilia A.