Adherence to prophylactic infusions of factor VIII or factor IX for haemophilia

Medication non-adherence is a true public health problem. Despite advancements in the molecular understanding of disease and improvements in therapy, patient health outcomes will not improve unless patients take prescribed medications. Decreasing the gap between efficacious and effective therapy for patients with haemophilia is an essential research agenda. Adherence is particularly important for patients on prophylaxis. Prophylactic infusion of factor VIII or IX has proven efficacy in preventing chronic joint disease and is considered the standard of care for children with severe haemophilia A and B [1,2]. However, the effectiveness of the prophylaxis depends on the recommendation of prophylaxis by the physician and adherence to the recommended regimen. Less than 40% of patients with severe haemophilia currently receive prophylaxis [3,4]. In a survey of haemophilia treatment centres, 37% of physicians reported that patient compliance was a barrier in implementing prophylactic therapy [5]. Haemophilia is a chronic disease, which requires long-term prophylactic therapy to prevent joint disease. Patients on primary prophylaxis are asymptomatic and may not see the benefits of prophylaxis. The treatment regimen is complex, requiring factor preparation and intravenous infusion. The treatment is also homebased so that it is not directly supervised by a physician or nurse. Previous adherence research in