The evolution of recombinant factor replacement for hemophilia

Transfus Apher Sci. 2019 Oct;58(5):596-600. doi: 10.1016/j.transci.2019.08.010. Epub 2019 Aug 9.

Abstract

Hemophilia A and Hemophilia B are the most common of the severe bleeding disorders and are caused by a deficiency in blood clotting factor VIII or factor IX respectively. Factor replacement therapy has been the cornerstone of treatment to treat life threatening bleeds and prevent joint disease. The treatment of hemophilia has evolved tremendously over the past five decades from fresh frozen plasma as the only available therapy to more specific plasma-derived and recombinant-derived factor replacement. Now due to innovations in bioengineering, there are even more efficacious factor replacement options available to patients. Here we review these recent advancements and their impact on the treatment and management of hemophilia.

Keywords: Emicizumab; Half-life; Hemophilia; Recombinant; Therapeutics.

Publication types

  • Review

MeSH terms

  • Factor IX / genetics
  • Factor IX / metabolism
  • Factor IX / therapeutic use*
  • Factor VIII / genetics
  • Factor VIII / metabolism
  • Factor VIII / therapeutic use*
  • Hemophilia A / blood
  • Hemophilia A / drug therapy*
  • Hemophilia A / genetics
  • Hemophilia B / blood
  • Hemophilia B / drug therapy*
  • Hemophilia B / genetics
  • Humans
  • Recombinant Proteins / therapeutic use

Substances

  • Recombinant Proteins
  • F8 protein, human
  • Factor VIII
  • Factor IX