Abstract
Hemophilia A is an X-linked inherited bleeding disorder that affects approximately 1 in 5000 male live births. It is caused by a deficient plasma level of clotting factor VIII and can be treated by the intravenous administration of factor VIII concentrates. A severe complication of the treatment with factor VIII concentrates is the development of inhibiting antibodies against factor VIII, also called inhibitors. Inhibitors challenge the treatment of hemophilia A as they inactivate factor VIII that is administrated for treatment or prevention of bleeding episodes, resulting in a greater rate of complications, costs and disability. This thesis focuses on the etiology of inhibitor development in nonsevere hemophilia A and addresses multiple genetic- and environmental risk factors. The findings of the research improve our understanding of the pathophysiology of inhibitor development in nonsevere hemophilia A and will open up new possibilities for identification of high risk patients and high risk clinical situations. This knowledge indicate the areas of care that merit particular attention and may ultimately help to implement targeted preventive measures.
Original language | English |
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Qualification | Doctor of Philosophy |
Awarding Institution | |
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Award date | 17 Sept 2014 |
Print ISBNs | 9789461087386 |
Publication status | Published - 2014 |