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Thromb Haemost 2005; 93(03): 457-467
DOI: 10.1160/TH03-10-0643
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A

Results of an international clinical investigation
Wolfhart Kreuz
7   The Center of Pediatrics III, Department of Hematology, Oncology and Hemostaseology, Johann Wolfgang Goethe-University Hospital, Frankfurt am Main, Germany
,
Joan C. Gill
1   Blood Center of Southeastern Wisconsin, Medical College of Wisconsin, Milwaukee, Wisconsin, USA
,
Chantal Rothschild
2   Hôpital Necker, Centre de l'Hemophilie F. Josso, Paris, France
,
Marilyn J. Manco-Johnson
3   Department of Pediatrics, University of Colorado Health Sciences Center, Denver, Colorado, USA
,
Jeanne M. Lusher
4   Children's Hospital of Michigan, Detroit, Michigan, USA
,
Elke Kellermann
5   Bayer Vital GmbH Biological Products, Leverkusen, Germany
,
Eduard Gorina
6   Bayer Health Care, Biological Products Division, Research Triangle Park, North Carolina, USA
,
Peter J. Larson
6   Bayer Health Care, Biological Products Division, Research Triangle Park, North Carolina, USA
,
and the International Kogenate-FS Study Group › Author AffiliationsGrants/Financial Support: This work was funded by Bayer Health Care.
Further Information

Publication History

Received 22 October 2003

Accepted after resubmission 28 January 2004

Publication Date:
14 December 2017 (online)

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Summary

The safety and efficacy of a full-length sucrose-formulated recombinant factor VIII product (rFVIII-FS; KogenateFS; Kogenate Bayer) was evaluated in previously untreated (PUPs) and minimally treated (MTP) patients with severe haemophilia A (FVIII < 2%). Patients (37 PUPs; 24 MTPs) aged 0.1–25.7 months were treated with rFVIII-FS for a cumulative of 9,141 exposure days (EDs), median 114 EDs (range 4–478), on prophylactic or on-demand therapy. Eighty-nine percent of all treated bleeding episodes were successfully treated with 1 (74%) or 2 (15%) infusions. Clinical response to first infusion for each bleeding episode was rated as ‘excellent’ in 58%, or ‘good’ in 33%, of all cases. Recombinant FVIII-FS was used in 27 surgical procedures, mainly catheter implantations, which were all conducted without bleeding complications. FVIII recovery mean values (~2%/kg/ IU) were as expected for any licensed FVIII concentrate. FVIII neutralizing antibody formation was 15% (9/60). Aside from inhibitor formation, three adverse events were rated as ‘at least possibly drug-related’ for a total drug-related adverse event rate of 0.14%. No viral seroconversions were observed. Overall, excellent safety and efficacy were demonstrated with rFVIII-FS for therapy of young children with severe haemophilia A.

Keywords

Haemophilia A - FVIII inhibitor - pediatric - PUPs
 

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