Long-term aspects of hemophilia B treatment

Abstract

This paper reviews hemophilia prophylaxis in terms of patient selection for this approach, when to start treatment, and choosing therapy. Both primary and secondary prophylaxis have been shown to reduce the frequency of joint bleeds and improve physical and social functioning for children and adults with hemophilia, although only primary prophylaxis has been shown effective in preventing progression of arthropathy. Limited data are available on hemophilia B prophylaxis; target outcomes have been poorly defined and quality-of-life information is lacking in these patients, partly due to the need for standardized assessment tools.