Haemophilia B: current pharmacotherapy and future directions

Expert Opin Pharmacother. 2012 Oct;13(14):2053-63. doi: 10.1517/14656566.2012.721780. Epub 2012 Sep 4.

Abstract

Introduction: Hemophilia B is a rare hereditary hemorrhagic disorder characterized by deficiency of the clotting factor IX (FIX). Hemophilia B patients experience mild to severe bleeding complications according to the degree of FIX defect. Nowadays, the most challenging complication of individuals with hemophilia B is the development of alloantibodies, which render the standard replacement therapy with FIX concentrates ineffective, exposing them to a significantly increased morbidity and mortality.

Areas covered: This review summarizes the most important events leading to the development of the current FIX products available for the treatment of hemophilia B patients. In addition, it focuses on the more recent advances in the production of new FIX molecules aimed at improving the clinical management of such patients.

Expert opinion: Although the availability of plasma-derived FIX concentrates has greatly improved the clinical management of hemophilia B patients, the introduction of FIX products using recombinant DNA technology has represented the most significant therapeutic progress in hemophilia B therapy, ensuring an advanced level of safety. The development of rFIX products with extended half lives will further improve the therapeutic armamentarium for hemophilia B patients.

Publication types

  • Historical Article
  • Review

MeSH terms

  • Factor IX / therapeutic use*
  • Hemophilia B / drug therapy*
  • Hemophilia B / history
  • History, 20th Century
  • History, 21st Century
  • Humans
  • Recombinant Proteins / therapeutic use

Substances

  • Recombinant Proteins
  • Factor IX