Retrospective comparisons of the Swedish see more and Dutch cohorts, where different strategies have been used, indicate that a costly, high-dose regimen improves outcome, but not dramatically. A prospective comparison is now underway. Treatment, clinical outcome, clotting factor consumption and socioeconomic

parameters will be compared between the two strategies. Results are expected to provide greater insight into the long-term consequences of the different prophylactic treatment strategies. The economic justification for prophylaxis has been addressed in several studies with varying results. While the majority (implicitly) suggest that prophylaxis is not cost effective at conventional willingness to pay for additional units in health thresholds, their results vary markedly. Closer

inspection suggests that the primary reasons results differ include different definitions of prophylaxis, clotting factor price, discount rates, choice of outcome measures and time horizon. Long-term replacement therapy prophylaxis, Selleck VX770 for haemophilia has a longstanding tradition in some countries. Cohort studies have shown prophylaxis not only to be superior to treatment on demand in terms of outcome, usually measured as haemophilic arthropathy, but also of quality of life and survival. Because of the rareness of the disease, extensive international collaboration and many years of follow-up are required to perform studies of high scientific merit. Thus, these have been completed only during the last several years. Together with larger and more long-term cohort studies, we now have firm evidence for the benefits of prophylaxis. However, several questions remain such as when to start prophylaxis, dose and dosing and when or if to stop. The focus for current research has increasingly become that of identifying the best strategy for providing a reasonable economic justification of prophylaxis so that countries with fewer economic 上海皓元医药股份有限公司 resources can also afford

it. In this article, the history of prophylaxis is reviewed and a comparison of the long-term outcomes of high-dose (Swedish) and intermediate-dose (Dutch) regimens are presented. Importantly, the economic justifications for prophylaxis are also examined. (Dr Berntorp) Studies conducted in Sweden by Ramgren and Ahlberg [1,2] during the 1960s showed that persons with haemophilia (PWH) with FVIII or IX levels above 1% of normal rarely developed severe disabling arthropathy. They hypothesized that it was logical to increase the level of factor activity in severe haemophilia to at least 1% by continuous prophylaxis. In The Netherlands, another pioneering country in this field, prophylaxis was introduced in 1968 [3]. Several attempts at prevention of bleeding with prophylaxis were documented during the late 1960s and the 1970s, both in Europe and North America.