Immune tolerance and the treatment of hemophiliacs with an inhibitor

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A major complication preventing adequate treatment of hemophilia patients is the development of inhibitory antibodies against factor VIII. In this case, one of the best therapeutic approaches currently available is the induction of immune tolerance, a method first described in 1976. Immune tolerance can be restored in most hemophiliacs who have developed an inhibitor through the administration of high doses of factor VIII, making it possible to treat bleeding episodes with normal quantities of factor VIII, to increase the patient’s survival chances and to reduce the cost of further treatment. Presenting the proceedings of an international workshop, this publication reviews the epidemiology and pathophysiology of FVIII inhibitors, and the mechanisms leading to immune tolerance. Covering the various treatment protocols applied worldwide, clinical issues as well as economic and organizational aspects, it provides essential information for doctors treating hemophiliacs and all those working in the fields of hematology, immunology, pharmacology, pediatrics and blood transfusion.