Half-life extended factor VIII for the treatment of hemophilia A

J Thromb Haemost. 2015 Jun:13 Suppl 1:S176-9. doi: 10.1111/jth.12929.

Abstract

Prophylactic infusion of factor VIII (FVIII) prevents joint bleeding and other hemorrhages in patients with hemophilia A. Conventional FVIII concentrates have a short half-life, with an average of about 12 h in adults, ranging in individual patients between 6 and 24 h, and even shorter in younger children. Therefore, effective prophylaxis requires frequent intravenous injection, usually three times per week or every other day. Several technologies are currently under investigation to extend the half-life of FVIII, including Fc fusion (Eloctate, Elocta, efmoroctocog alfa), addition of polyethylene glycol (turoctocog alfa pegol [N8-GP], BAY 94-9027, BAX 855), and a single-chain construct (CSL627). This review summarizes characteristics of products in clinical development and discusses their potential benefits.

Keywords: blood coagulation factors; hemophilia A; hemostasis; polyethylene glycol; recombinant proteins.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Discovery
  • Drugs, Investigational / adverse effects
  • Drugs, Investigational / pharmacokinetics
  • Drugs, Investigational / therapeutic use*
  • Factor VIII / adverse effects
  • Factor VIII / pharmacokinetics
  • Factor VIII / therapeutic use*
  • Half-Life
  • Hemophilia A / blood
  • Hemophilia A / diagnosis
  • Hemophilia A / drug therapy*
  • Hemorrhage / prevention & control*
  • Hemostasis / drug effects*
  • Hemostatics / adverse effects
  • Hemostatics / pharmacokinetics
  • Hemostatics / therapeutic use*
  • Humans
  • Recombinant Proteins / therapeutic use
  • Treatment Outcome

Substances

  • Drugs, Investigational
  • Hemostatics
  • Recombinant Proteins
  • F8 protein, human
  • Factor VIII