A comparison between prophylaxis and on demand treatment for severe haemophilia

Abstract

Patients with severe haemophilia can be treated for bleeding either prophylactically or on demand. Each treatment modality has advantages and disadvantages from both a medical and economic point of view. This study aims to find which modality requires more units of clotting factors per body weight per year and to compare the number of bleeds between the two. The study sample consisted of 133 patients with severe haemophilia A and B treated in the Katharine Dormandy Haemophilia Centre at the Royal Free Hampstead NHS Trust in London. The average number of clotting factors used per body weight per year was 2181.7 units for prophylaxis vs. 711 units for on demand treatment (P = 0.000). Although more units used means more money spent, and although prophylaxis has additional complications, namely venipunctures and increased risk of viral contamination, other criteria must be considered including the total number of bleeds and health-related quality of life. The total number of bleeds per year was significantly (P = 0.021) less for prophylactically (7.4) vs. on-demand treated patients (11.4). This suggests that prophylaxis reduces the risk of arthropathies, the number of future hospital visits and orthopaedic surgeries, and is thus the optimal modality of treatment for patients with severe haemophilia. © 2005 Blackwell Publishing Ltd.