Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project.
This analysis of the US 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 6196 males; mean age at first registry visit was 17.7 years; and median was 14 (range, 2 to 69). During this time, prophylaxis use increased from 31% to 59% overall, and by 2010, 75% of children and youths <20 years were on prophylaxis. On cross-sectional analysis, bleeding rates decreased dramatically for the entire population (P< .001) in parallel with increased prophylaxis usage, possibly because frequent bleeders adopted prophylaxis. Joint bleeding decreased proportionately with prophylaxis (22%) and nonprophylaxis (23%), and target joints decreased more with prophylaxis (80% vs 61%). Joint, total, and target joint bleeding on prophylaxis were 33%, 41%, and 27%, respectively, compared with nonprophylaxis. On longitudinal analysis of individuals over time, prophylaxis predicted decreased bleeding at any age (P< .001), but only prophylaxis initiation prior to age 4 years and nonobesity predicted preservation of joint motion (P< .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.
Hemophilia and Thrombosis Center, Department of Pediatrics, University of Colorado Denver, Aurora, CO.
Marilyn J
0000-0003-1272-1684
MJ
Manco-Johnson
National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA.
J Michael
JM
Soucie
Departments of Pediatrics and Medicine, Medical College of Wisconsin, Milwaukee, WI; and.|Comprehensive Center for Bleeding Disorders, BloodCenter of Wisconsin, Milwaukee, WI.
Joan Cox
JC
Gill
D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000368:Aged; D002648:Child; D002675:Child, Preschool; D003430:Cross-Sectional Studies; D005169:Factor VIII / Q000627:therapeutic use*; D006395:Hemarthrosis / Q000175:diagnosis / Q000503:physiopathology / Q000517:prevention & control*; D006467:Hemophilia A / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D006470:Hemorrhage / Q000175:diagnosis / Q000503:physiopathology / Q000517:prevention & control*; D006801:Humans; D007596:Joints / Q000098:blood supply / Q000187:drug effects / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D009819:Office Visits / Q000706:statistics & numerical data; D011322:Primary Prevention / Q000706:statistics & numerical data*; D011446:Prospective Studies; D016059:Range of Motion, Articular / Q000187:drug effects; D012042:Registries; D014481:United States
D005169:Factor VIII