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Updated: May 27, 2026

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Physical functioning in boys with hemophilia in the U.S
Paul E Monahan1, Judith R Baker, Brenda Riske
1Department of Pediatrics, Hematology/Oncology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599-7016, USA. Paul_Monahan@med.unc.edu
Children with hemophilia (an inherited bleeding disorder) face physical limitations due to recurrent hemorrhages. Key risk factors include age, joint bleeds, inhibitors, obesity, and Medicaid insurance, highlighting needs for targeted interventions and equitable care.
Area of Science:
- Pediatric Hematology
- Orthopedics
- Public Health
Background:
- Hemophilia is a common inherited severe bleeding disorder.
- Recurrent hemorrhages lead to joint disease and variable functional impairment.
Purpose of the Study:
- To identify characteristics associated with physical functioning limitations in boys with hemophilia.
- To analyze a national database for risk factors of functional decline due to bleeding.
Main Methods:
- Utilized data from the Universal Data Collection (UDC) program (1998-2008).
- Examined 15 potential predictors in boys aged ≤18 years.
- Performed bivariate and multivariate analyses on activity limitations, absenteeism, and mobility aid reliance.
Main Results:
- Increasing age, joint bleeding, and inhibitor antibodies independently predicted poor physical function.
- African-American race and Asian/Pacific Island ethnicity were associated with activity restrictions and absenteeism, respectively.
- Obesity and Medicaid insurance were linked to multiple poor functional outcomes.
Conclusions:
- Interventions should target inhibitor elimination, support for African-American children, and weight management.
- Ensuring adequate insurance coverage is crucial to overcome economic barriers to optimal outcomes.
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