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

Wolfhart Kreuz; Joan C. Gill; Chantal Rothschild; Marilyn J. Manco-Johnson; Jeanne M. Lusher; Elke Kellermann; Eduard Gorina; Peter J. Larson; and the International Kogenate-FS Study Group

doi:10.1160/TH03-10-0643

Thrombosis and Haemostasis, Table of Contents

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

⁷The Center of Pediatrics III, Department of Hematology, Oncology and Hemostaseology, Johann Wolfgang Goethe-University Hospital, Frankfurt am Main, Germany

,

Joan C. Gill

¹Blood Center of Southeastern Wisconsin, Medical College of Wisconsin, Milwaukee, Wisconsin, USA

,

Chantal Rothschild

²Hôpital Necker, Centre de l'Hemophilie F. Josso, Paris, France

,

Marilyn J. Manco-Johnson

³Department of Pediatrics, University of Colorado Health Sciences Center, Denver, Colorado, USA

,

Jeanne M. Lusher

⁴Children's Hospital of Michigan, Detroit, Michigan, USA

,

Elke Kellermann

⁵Bayer Vital GmbH Biological Products, Leverkusen, Germany

,

Eduard Gorina

⁶Bayer Health Care, Biological Products Division, Research Triangle Park, North Carolina, USA

,

Peter J. Larson

⁶Bayer Health Care, Biological Products Division, Research Triangle Park, North Carolina, USA

,

and the International Kogenate-FS Study Group

› Author Affiliations

Abstract

Summary

The safety and efficacy of a full-length sucrose-formulated recombinant factor VIII product (rFVIII-FS; Kogenate^FS; 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

Full Text

References

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