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

Thomas C. Abshire, H. H. Brackmann, I. Scharrer, K. Hoots, C. Gazengel, Jerry S Powell, E. Gorina, E. Kellermann, E. Vosburgh

Research output: Contribution to journalArticle

112 Scopus citations

Abstract

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 drug-related 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.

Original languageEnglish (US)
Pages (from-to)811-816
Number of pages6
JournalThrombosis and Haemostasis
Volume83
Issue number6
StatePublished - 2000

Keywords

  • Full-length rFVIII
  • Hemophilia A
  • Recombinant Factor VIII

ASJC Scopus subject areas

  • Hematology

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