Prophylaxis in bleeding disorders

Abstract

Primary prophylaxis with coagulation factor concentrates has become the standard of care for children with hemophilia to reduce the risk of bleeding and related morbidity. However, several important questions remain unanswered regarding the optimal use of prophylaxis in patients with bleeding disorders. Limited data are available on the use of primary prophylaxis in adults with hemophilia, although tailoring the dose and schedule of prophylaxis in adults based on the clinical course of the disease may improve convenience and reduce costs without compromising efficacy. Patients with severe forms of von Willebrand disease (VWD) are at risk of serious bleeding episodes and may therefore benefit from prophylaxis; results from ongoing trials, such as the VWD International Prophylaxis (VIP) trial, are expected to provide more insight into the efficacy and safety of prophylaxis in these patients. For patients with other rare bleeding disorders, prophylaxis may be considered, depending on the clinical course of the disease and the availability of factor replacement therapy products; definitive recommendations, however, are not possible given the lack of comprehensive studies evaluating prophylaxis in this setting. Ongoing studies will help further define the role of coagulation factor concentrate prophylaxis in patients with bleeding disorders.