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Haemophilia. 2016 Mar;22(2):225-231. doi: 10.1111/hae.12793. Epub 2015 Aug 28.

Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq® ) in adults with severe haemophilia A: efficacy and safety.

Author information

1
Specialised Hospital for Active Treatment "Joan Pavel", Sofia, Bulgaria.
2
Royal Hallamshire Hospital, Sheffield, UK.
3
Werlhof-Institut für Hämostaseologie GmbH, Hannover, Germany.
4
Manchester Royal Infirmary, Manchester, UK.
5
Basingstoke and North Hampshire Hospital, Basingstoke, UK.
6
Royal Free Hospital, London, UK.
7
University Hospital Hamburg-Eppendorf, Hamburg, Germany.
8
SRH Kurpfalzkrankenhaus and Hemophilia Center, Heidelberg, Germany.
9
Medical University of Vienna, Vienna, Austria.
10
Octapharma AG, Lachen, Switzerland.
11
Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany.

Abstract

INTRODUCTION:

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 (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-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 PTPs indicate that human-cl rhFVIII is effective for the prevention and treatment of bleeds in adults with severe haemophilia A.

KEYWORDS:

Nuwiq; factor VIII inhibitors; haemophilia A; human cell line factor VIII; prophylaxis; recombinant factor VIII

PMID:
26315974
DOI:
10.1111/hae.12793

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