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Efficacy and safety of prophylaxis with once-weekly BAY 79–4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients

Journal: Thrombosis and Haemostasis
ISSN: 0340-6245
Topic:

Theme Issue
European Vascular Biology Meeting 2011 (Part 2)

DOI: http://dx.doi.org/10.1160/TH12-03-0188
Issue: 2012: 108/5 (Nov) pp. 801-1007
Pages: 913-922

Efficacy and safety of prophylaxis with once-weekly BAY 79–4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients

A randomised, active-controlled, double-blind study

Online Supplementary Material

J. Powell (1), U. Martinowitz (2), J. Windyga (3), G. Di Minno (4), A. Hellmann (5), I. Pabinger (6), M. Maas Enriquez (7), L. Schwartz (8), J. Ingerslev (9), the LipLong Study Investigators

(1) Division of Hematology/Oncology, School of Medicine, University of California at Davis, Davis, California, USA; (2) The National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; (3) Department of Disorders of Haemostasis and Internal Medicine, Institute of Haematology and Transfusion Medicine, Warsaw, Poland; (4) Regional Reference Centre for Coagulation Disorders, Department of Clinical and Experimental Medicine, Federico II University Hospital, Naples, Italy; (5) Department of Hematology, Medical University of Gdansk/Poland; (6) Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University Vienna, Austria; (7) Bayer Pharma AG, Wuppertal, Germany; (8) Bayer HealthCare Pharmaceuticals Inc., Montville, New Jersey, USA; (9) Center for Haemophilia and Thrombosis, Aarthus University Hospital, Skejby, Denmark

Keywords

Haemophilia, prophylaxis, pegylated liposome, rFVIII-FS

Summary

The benefits of prophylaxis of haemophilia A patients regarding joint health and quality-of-life are well established. However, adherence to an up to every-other-day infusion regimen is a barrier to widespread adoption of prophylaxis. BAY 79–4980 is an investigational drug consisting of rFVIII-FS (sucrose-formulated recombinant FVIII) reconstituted with liposome solvent. Previous clinical studies showed extended protection from bleeding after a single injection of BAY 79–4980 (13.3 ± 6.2 days) compared with rFVIII-FS (7.2 ± 1.7 days). The effect of once-a-week prophylaxis with BAY 79–4980 (35 IU/kg) compared with three times-per-week rFVIII-FS (25 IU/kg) in previously treated, severe haemophilia A patients was evaluated in a 52-week, double-blind, two-arm, randomised, controlled study. The primary and secondary endpoints were protection from total bleeds and joint bleeds, respectively. Short- and long-term safety and tolerability of BAY 79–4980 including effects on lipid levels were assessed. A total of 139 and 131 subjects were evaluable for safety and efficacy analyses, respectively. A large difference in efficacy between treatment groups was observed with 72.1% (49/68) in the rFVIII-FS control group demonstrating <9 bleeds/year compared with 38.1% (24/63) of BAY 79–4980-treated subjects. A similar difference was seen in annualised joint bleeds, with 43 subjects (63.2%) in the control group demonstrating <5 joint bleeds/year compared with 24 subjects (38.1%) treated with BAY 79–4980. The distribution of bleeds seven days post-prophylactic treatment with BAY 79–4980 showed that 61% of bleeds occurred after day 4 post dosing. There were no safety concerns identified. The investigational treatment arm was prematurely discontinued due to failure to achieve the primary endpoint.

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