An approach to target joint bleeding in hemophilia: Prophylaxis for all or individualized treatment?

Ten years ago, the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation recommended prophylaxis as optimal therapy for severe hemophilia (MASAC Medical Bulletin #193). Oddly, this important but expensive treatment modality was fully embraced without the debate and scrutiny usually reserved for costly diseases. And why not? Hemophilia, the “royal disease,” has usually commanded a larger medical focus than the approximately 18,000 current US hemophilia patients would seem to dictate. Hemophilia can indeed “hold court” because of its association with the lineage of Queen Victoria and, more important, the cost of treatment. In this issue of The Journal, the role of prophylaxis in preventing joint disease has resurfaced for a novel fiscal analysis. Kern and colleagues thoughtfully weigh the financial impact of joint disease on hemophilia treatment.¹ There are no studies that focus on the cost implication surrounding the development of a target joint in this population.

MASAC, Medical and Scientific Advisory Council