Among patients with hemophilia A who receive primary prophylaxis, quality of life (QoL) may be lower compared with the general population, according to research published in Haemophilia.
Among patients with severe hemophilia, prophylaxis with clotting factor replacement therapy is the standard treatment, though patients vary in when they initiate therapy. In the optimal scenario, patients will receive prophylaxis prior to the development of clinically evident joint disease, because prophylaxis cannot reverse joint damage.
Primary prophylaxis is defined as treatment initiated prior to the second clinically evident large joint bleed, and where the patient is aged 3 years. Secondary and tertiary prophylaxis refer to treatment initiated after 2 or more bleeds and after the onset of joint disease, respectively; prophylaxis may also be used intermittently. Each of these forms of treatment is distinct from on-demand treatment, where each bleeding episode is treated separately.
Previous research suggests that on-demand treatment leads to worse morbidity and QoL. However, there is limited evidence as to whether use of primary prophylaxis leads to improved morbidity and QoL compared with other forms of treatment, or with the general population. For this study, researchers evaluated QoL and functional variables among patients with severe hemophilia A treated with primary prophylaxis and who had not received inhibitors with several groups: a) patients receiving on-demand treatment or secondary prophylaxis, b) the general population, and c) a population of patients with osteoarthritis.