Identifying and overcoming barriers to prophylaxis in the management of haemophilia

Abstract

Haemophilia is often characterized by acute haemarthrosis and synovitis arising from spontaneous bleeding episodes, particularly in the muscles and joints of the elbows, knees and ankles. Current treatment for patients with severe haemophilia involves coagulation factor concentrate (CFC) replacement therapy given on demand at the time of bleeding or through long-term prophylaxis aimed at preventing future bleeds and joint disease. Although prophylaxis has many advantages over on-demand therapy (particularly if started before age 2 and prior to any signs of joint disease), its practice varies widely even among developed countries because of several barriers. Such barriers include CFC costs and availability; patient perceptions, lifestyles and bleeding patterns; difficulties and complications arising from the use of intravenous access devices (IVADs); the development of inhibitors; and the lack of randomized clinical trials. These barriers can be overcome by tailoring treatment regimens according to individual patient bleeding patterns and CFC pharmacokinetic profiles, using IVADs selectively and judiciously, helping patients maintain normal weight and physical exercise and providing the families of patients with continuous support from healthcare providers.