Immune tolerance induction in hemophilia
نویسندگان
چکیده
2015 The development of inhibitors is the main complication of hemophilia therapy. Inhibitors occur in 25–30% and in 2–5% of patients with severe hemophilia A and B, respectively. They render treatment and prevention of bleeds difficult. The only known therapeutic strategy able to eliminate inhibitors is immune tolerance induction (ITI) that consists in regular high-dose FVIII/FIX infusions. ITI is a demanding treatment both for patients/parents and clinicians, it is costly, but provides 60–80% chance of success. Although used since late 1970, many aspects of ITI still needs to be optimized and further investigated. This review is aimed at reporting what is known from the published literature and what still need to be investigated in this field.
منابع مشابه
Low-Dose Immune Tolerance Induction in Hemophilia
In patients with hemophilia A and inhibitory alloantibodies against factor VIII, various dosage schedules are used to obtain immune tolerance. In this study, we have evaluated the results of 13 years of low-dose immune tolerance induction and factors that are predictive of a positive result. The effect of immune tolerance induction in relation to age at inhibitor development, number of exposure...
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Hemophilia B is a rare X-linked recessive disorder with plasma factor IX (FIX) deficiency. 1-3% of patients treated with exogenous FIX-containing products develop inhibitors (i.e. polyclonal high affinity immunoglobulins) that neutralize the procoagulant activity of a specific coagulation factor. Although the incidence of inhibitors in hemophilia B patients is low, most are "high titer" and fre...
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Although immune tolerance induction (ITI) has been used for 30 years to eliminate inhibitors and restore normal factor pharmacokinetics in patients with hemophilia, there is a paucity of scientific evidence to guide therapeutic decision-making. In an effort to provide direction for physicians and hemophilia treatment center staff members, an international panel of hemophilia opinion leaders met...
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Under certain instances, factor VIII (FVIII) stimulates an immune response, and the resulting neutralizing antibodies present a significant clinical challenge. Immunotherapies to re-establish or induce long-term tolerance would be beneficial, and an in-depth knowledge of mechanisms involved in tolerance induction is essential to develop immune-modulating strategies. We have developed a murine m...
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UNLABELLED INTRODUCTION The development of factor VIII inhibitors is a serious complication of replacement therapy in patients with congenital hemophilia A. Immune tolerance induction has been accepted as the only clinically proven treatment allowing antigen-specific tolerance to factor VIII. However, some of its issues, such as patient selection, timing, factor VIII dosing, use of immunosup...
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