Mechanisms of action of immune tolerance induction against factor VIII in patients with congenital haemophilia A and factor VIII inhibitors

Br J Haematol. 2007 Jan;136(1):12-25. doi: 10.1111/j.1365-2141.2006.06359.x.

Abstract

In its most severe form, haemophilia A is a life-threatening haemorrhagic bleeding disorder that is caused by mutations in the factor VIII (FVIII) gene. About 25% of patients who receive replacement therapy with intravenous FVIII products develop neutralising antibodies (FVIII inhibitors) that inhibit the function of substituted FVIII. Long-term application of high or low doses of FVIII has evolved as an effective strategy for eradicating antibodies and inducing long-lasting immune tolerance. Despite clinical experience with the therapy, little is known about the immunological mechanisms that cause the down modulation of FVIII-specific immune responses or the induction of long-lasting immune tolerance against FVIII. This review summarises current knowledge of the immunological mechanisms that might be involved in the induction of immune tolerance against FVIII in patients with haemophilia A who have FVIII inhibitors. In addition to data from patients with haemophilia A, data from patients who have had organ transplants or have immune-related disorders, such as autoimmune diseases, are considered as well as data from animal models.

Publication types

  • Review

MeSH terms

  • Animals
  • Antibody Formation
  • Autoantibodies / immunology
  • Autoimmune Diseases / immunology
  • Blood Coagulation Factor Inhibitors / immunology*
  • Coagulants / therapeutic use
  • Factor VIII / immunology*
  • Factor VIII / therapeutic use
  • Hemophilia A / immunology*
  • Hemophilia A / therapy
  • Humans
  • Immune Tolerance
  • Immunologic Memory
  • Models, Animal

Substances

  • Autoantibodies
  • Blood Coagulation Factor Inhibitors
  • Coagulants
  • Factor VIII