Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy--International Kogenate-FS Study Group

Thromb Haemost. 2000 Jun;83(6):811-6.


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 (> or = 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.

Publication types

  • Clinical Trial
  • Comparative Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Antibodies / blood
  • Child
  • Cross-Over Studies
  • Drug Compounding
  • Drug Evaluation
  • Factor VIII / administration & dosage
  • Factor VIII / adverse effects
  • Factor VIII / pharmacokinetics
  • Hemophilia A / complications
  • Hemophilia A / drug therapy
  • Hemorrhage / drug therapy
  • Hemorrhage / prevention & control
  • Home Infusion Therapy
  • Humans
  • Male
  • Middle Aged
  • Partial Thromboplastin Time
  • Patient Satisfaction
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / pharmacokinetics
  • Sucrose*
  • Time Factors
  • Treatment Outcome


  • Antibodies
  • Recombinant Proteins
  • Sucrose
  • F8 protein, human
  • Factor VIII