Regular clotting factor replacement therapy to prevent joint disease in people with severe hemophilia A or B

Hemophilia A and B are X-linked inherited bleeding disorders, in which the major clinical problem is repeated bleeding into joints. As this disorder progresses, joints become deformed and movement limited. Current therapy for treating and preventing bleeding includes plasma-derived or recombinant clotting factor concentrates. This review includes six randomised controlled trials. Two compare the regular use of clotting factor concentrates to prevent joint bleeds with their use 'on demand'. Four compare different regimens of regular use in children and adults with hemophilia. It was clearly evident that preventative therapy, as intravenous infusion of factor concentrate repeated more times a week and started early in childhood was able to reduce joint deterioration as compared to treatment administered after bleeding occurred. This favourable effect is due to a consistent reduction in total bleeds and hemarthrosis (bleeding into joints) and leads to a significant improvement in quality of life. Preventative therapy is linked to an increased factor usage and cost of treatment. We found weaker evidence (due to lack of data) to show preventative therapy reduced joint deterioration when treatment is started after joint damage has been established. Further studies are needed to establish the best preventative regimen, i.e. for example starting time, dosage frequency, minimally effective dose.