Recombinant, B-domain deleted factor VIII (r-VIII SQ): pharmacokinetics and initial safety aspects in hemophilia A patients

Thromb Haemost. 1997 Feb;77(2):298-302.

Abstract

The pharmacokinetics of a second-generation recombinant B-domain deleted factor VIII (FVIII) preparation (r-VIII SQ) were studied in 36 patients with severe hemophilia A. In contrast to full-length recombinant FVIII, no albumin needs to be added to stabilize the final formulation of this B-domain deleted FVIII preparation. The in vivo recovery and half-life of r-VIII SQ were similar to those of plasma-derived (pd) FVIII (mean half-life of r-VIII SQ, 11.7 h). The volume of distribution and clearance were slightly, but significantly, higher for r-VIII SQ than for pdFVIII (p < 0.05). Peak plasma levels of FVIII were consistently related to the administered dose of r-VIII SQ (r = 0.94, p < 0.0001). The pharmacokinetic profile of r-VIII SQ remained essentially unchanged in a dose range of 25-100 IU/kg body weight and could be reproduced after repeated doses. r-VIII SQ was well tolerated. In conclusion, deletion of the B-domain of FVIII does not influence its in vivo pharmacokinetics.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Cross-Over Studies
  • Factor VIII / adverse effects
  • Factor VIII / pharmacokinetics*
  • Factor VIII / therapeutic use
  • Half-Life
  • Hemophilia A / blood
  • Hemophilia A / drug therapy*
  • Humans
  • Male
  • Middle Aged
  • Peptide Fragments / adverse effects
  • Peptide Fragments / pharmacokinetics*
  • Peptide Fragments / therapeutic use
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / pharmacokinetics
  • Recombinant Fusion Proteins / therapeutic use
  • Safety
  • Sequence Deletion
  • Single-Blind Method

Substances

  • Peptide Fragments
  • Recombinant Fusion Proteins
  • Factor VIII