Burden of disease resulting from hemophilia in the U.S.

Background: Hemophilia is a hereditary bleeding disorder. Its complications can result in substantial morbidity, but few efforts have been made to quantify the disease burden.

Purpose: The objective of this analysis was to estimate the burden of disease due to hemophilia (A and B) in the U.S., using disability-adjusted life years (DALY).

Methods: The approach taken by the WHO in its Global Burden of Disease study was followed. Assumptions were drawn from published literature, and population estimates from the U.S. Census Bureau for the Year 2007 were used. Estimations of years of life lost resulting from mortality (YLL) and years of life lost resulting from morbidity (YLD) were done separately by gender, 5-year age intervals, and severity of disease (morbidity only) with their sum representing DALYs. Disability weights were derived from the quality-of-life tool EuroQol (EQ-5D). The stability of burden estimates was tested by performing sensitivity analyses, changing one assumption at a time.

Results: In the U.S. in 2007, hemophilia resulted in 110,095 DALYs, composed of 13,418 YLLs and 96,677 YLDs. Large differences between men/boys (107,346) and women/girls (2749) were observed, given that females are genetic carriers of the disorder and rarely present with disease. Sensitivity analyses revealed a relatively robust estimate with a maximum variation of 4.49%.

Conclusions: This first estimate of hemophilia-related DALYs in the U.S. indicates that control of hemophilia can potentially result in a gain of 1 healthy year of life for every 2700 people in the population.