Immune tolerance therapy for haemophilia

Abstract

The development of anti-factor VIII and anti-factor IX allo-antibodies in haemophilia A and B, respectively, remains a serious complication of treatment for these two X-linked haemostatic disorders, with major clinical and economic consequences. Treatment of this potentially fatal complication remains one of the greatest challenges facing haematologists at the beginning of the 21st century. Immune tolerance induction (ITI) therapy has been generally accepted as the best available treatment, extinguishing the inhibitor and permitting a resumption of standard dosing schedules. Although there have been several established protocols for ITI therapy developed over the last quarter century, the optimal scheme in terms of safety, clinical efficacy and pharmacoeconomic considerations has yet to be determined.