Comparing outcomes of different treatment regimens for severe haemophilia

Abstract

Published retrospective reports from France, the Netherlands and Sweden were analysed for data relating to the long-term outcomes (primarily the development of arthropathy) of three regimens for the management of severe haemophilia: on-demand treatment, intermediate-dose prophylaxis and high-dose prophylaxis. The mean annual consumption of factor concentrate was also compared. These data indicate that both prophylaxis regimens resulted in significantly improved long-term outcomes, as assessed by pain, clinical and radiological assessment scores. At the same time, the most recently reported annual factor consumption levels of these young adult patients are comparable in the on-demand and intermediate-dose prophylaxis cohorts, suggesting that the improvement in long-term clinical outcomes and reduced risk of arthropathy may lead to reduced factor consumption in adult patients who received early prophylactic therapy.