Bleeding in US hemophilia patients has decreased dramatically in parallel with increased use of prophylaxis
Only prophylaxis started before age 4 years preserves normal joint function
This analysis of the United States Hemophilia Treatment Center Network and the Centers for Disease Control and Prevention surveillance registry assessed trends in prophylaxis use and its impact on key indicators of arthropathy across the life span among participants with severe hemophilia A. Data on demographics, clinical characteristics and outcomes were collected prospectively between 1999 and 2010 at annual clinical visits to 134 hemophilia treatment centers. Trends in treatment and outcomes were evaluated using cross-sectional and longitudinal analyses. Data analyzed included 26,614 visits for 6,196 males, mean age at first registry visit 17.7 years, median 14, range 2 to 69. During this time, prophylaxis use increased from 31% to 59% overall, with 75% of children and youths less than 20 years on prophylaxis by 2010. On cross-sectional analysis bleeding rates decreased dramatically for the entire population (p< 0.001) in parallel with increased prophylaxis usage, possibly as frequent bleeders adopted prophylaxis. Joint bleeding decreased proportionately with prophylaxis (22%) and non-prophylaxis (23%); target joints decreased more with prophylaxis (80% vs. 61%). Joint, total and target joint bleeding on prophylaxis were 33%, 41% and 27%, respectively, compared to non-prophylaxis. On longitudinal analysis of individuals over time, prophylaxis predicted decreased bleeding at any age (p < 0.001), but only prophylaxis initiation prior to age 4 years and non-obesity predicted preservation of joint motion (p<0.001 for each). Using a national registry care providers in a specialized health care network for a rare disorder were able to detect and track trends in outcomes over time.
- Submitted February 9, 2016.
- Accepted January 7, 2017.
- Copyright © 2017 American Society of Hematology
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