Gene therapy for immune tolerance induction in hemophilia with inhibitors

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Immune tolerance induction in hemophilia

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. IT...

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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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Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors.

Immune tolerance induction (ITI) eradicates inhibitors in patients with hemophilia A. This study was designed to investigate the success rate of ITI in high-responding inhibitor patients with severe hemophilia A using recombinant factor VIII (rFVIII). Twenty-six patients received different ITI regimens until a normal recovery (>66%) and half-life (>6 h) of infused FVIII was achieved. In order t...

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Immune tolerance induction in patients with severe hemophilia A with inhibitors

BACKGROUND Inhibitory antibodies to factor VIII (FVIII) are an important complication when managing patients with hemophilia A. Immune tolerance induction (ITI) has been regarded as a useful method for eradicating inhibitors. We report the results of a retrospective study in Korean patients with hemophilia A who underwent ITI. METHODS We reviewed the records of patients with hemophilia A with...

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Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper.

The development of inhibitory antibodies to factor VIII (FVIII) occurs in approximately 30% to 40% of patients with severe hemophilia A. Management options for patients with inhibitor include eradicating it via immune tolerance induction (ITI) or treating bleeding episodes with large quantities of hemostatic agents. ITI is costly, approaching $1 million for the average 5-year-old, but if succes...

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ژورنال

عنوان ژورنال: Journal of Thrombosis and Haemostasis

سال: 2016

ISSN: 1538-7933

DOI: 10.1111/jth.13331