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Pediatr Blood Cancer. 2013;60 Suppl 1:S30-3. doi: 10.1002/pbc.24338. Epub 2012 Oct 25.

Inhibitors in childhood hemophilia A: genetic and treatment-related risk factors for development and eradication.

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  • 1Division of Blood Diseases and Resources, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892-7950, USA.


The development of neutralizing antibodies remains a serious complication of hemophilia replacement therapy. Factor VIII inhibiting antibodies (inhibitors) occur commonly following replacement therapy in hemophilia A, creating a significant burden of clinical disease. This article will review our current understanding of risk factors and their known impact on inhibitor development in previously untreated or minimally treated children with severe and mild hemophilia A. It will also explore how the most recently elucidated immunology of inhibitor development might hold important clues to more effective inhibitor eradication and prevention in this heavily impacted patient population.

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