Prophylactic treatment for severe haemophilia: comparison of an intermediate‐dose to a high‐dose regimen

Abstract

Summary. A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970–90. 42 Swedish patients (high‐dose prophylaxis), were compared with 86 Dutch patients (intermediate‐dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high‐dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg⁻¹ year⁻¹. Patients treated with high‐dose prophylaxis had fewer joint bleeds (median 0.3 year⁻¹ vs. 3.3 year⁻¹) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age‐adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate‐dose prophylaxis, high‐dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow‐up.