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In hemophilia A and B and von Willebrand disease, coagulation factors are absent or deficient. This impairs the capacity of the blood to coagulate and increases the risk of bleeding. The diseases are hereditary. If inadequately treated, hemophilia causes painful bleeding in joints and leads to disability. Bleeds can also occur in internal organs, e.g., the brain.
Hemophilia can be treated by replacing missing coagulation factors. The availability of coagulation factors has drastically reduced morbidity, and since the 1950s survival has increased from 15 years to nearly normal life expectancy.
In the past, patients were exposed to high risk of HIV infection and Hepatitis C transmitted via blood and blood products. Since the mid 1980s, concentrated coagulation factors have been produced by methods that have practically eliminated the infection risks. Nevertheless, several questions remain concerning optimum treatment, e.g., selecting the most appropriate dose and dosing strategy. Another question concerns the treatment of bleeding episodes (bleeds) in patients that have developed antibodies (inhibitors) that counteract the effects of factor concentrates.
How to cite this report: SBU. Treatment of Hemophilia A and B and von Willebrand disease. Stockholm: Swedish Council on Health Technology Assessment (SBU); 2012. SBU report no 208E.
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