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Thromb Haemost 2000; 83(06): 811-816
DOI: 10.1055/s-0037-1613925
Commentary
Schattauer GmbH

Sucrose Formulated Recombinant Human Antihemophilic Factor VIII Is Safe and Efficacious for Treatment of Hemophilia A in Home Therapy

Results of a Multicenter, International, Clinical Investigation
T. C. Abshire
1   From the Emory University School of Medicine, Atlanta, GA, USA
,
H.-H. Brackmann   *
2   University of Bonn, Bonn, Germany
,
I. Scharrer   *
3   University Hospital, Frankfurt, Germany
,
K. Hoots   *
4   University of Texas · Houston Medical School, Houston, TX, USA
,
C. Gazengel   *
5   Hôpital Necker, Paris, France
,
J. S. Powell   *
6   UC Davis Cancer Center, Sacramento, CA, USA
,
E. Gorina
7   QF Bayer SA, Barcelona, Spain
,
E. Kellermann
8   Bayer Vital, Leverkusen, Germany
,
E. Vosburgh
9   Bayer Corporation, West Haven, CT, USA
,
and the International Kogenate-FS Study Group › Author Affiliations
Further Information

Publication History

Received 08 November 1999

Accepted after revision 28 January 2000

Publication Date:
14 December 2017 (online)

Also available at
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Summary

To add an increased level of safety to antihemophilic factor replacement therapy, a full-length, recombinant Factor VIII (rFVIII) product has been developed without human-derived plasma proteins during purification and formulation and using an additional solvent/detergent viral inactivation step. This first clinical trial of a sucrose-formulated full-length rFVIII (rFVIII-FS) was conducted in previously treated patients (≥100 prior exposure days) with severe (<2% FVIII) hemophilia A in North America (NA) and Europe (EU). Pharmacokinetic profiles for rFVIII-FS were compared with those of currently licensed rFVIII product (Kogenate®) in 35 patients. Safety and efficacy during home therapy were evaluated in 71 patients. The new formulation displayed a pharmacokinetic profile similar to that of rFVIII. Patients on home therapy received a cumulative total of 11,867 exposure days, 12,546 infusions, and 22,443,694 IU of rFVIII-FS. Of 2585 bleeds, 93.5% were treated with 1-2 infusions and 80.5% of responses were rated as excellent or good. No evidence of de novo inhibitor formation was observed. Only 0.27% of infusions were associated with any drugrelated adverse event. Except for an episode of intermittent chest pain with palpitations which ceased after treatment with analgesics, associated adverse events were mild or moderate. Overall, rFVIII-FS provided excellent hemostatic control, was well-tolerated, and caused no significant adverse effects, thus demonstrating safety and efficacy for treatment of bleeds in patients with hemophilia A.

Key words

Hemophilia A - recombinant Factor VIII - full-length rFVIII

* Members of the International Kogenate-FS Study Group Writing Committee participated equally in development of this manuscript.


 

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