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September 2007
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UCD part of landmark hemophilia study

The University of Colorado Denver was one of 15 institutions involved in a landmark hemophilia study that has shown significant reductions in the risk of developing joint damage in young children with hemophilia A.

The study, which was published in this month’s issue of The New England Journal of Medicine, indicated that infusions of clotting factor VIII used preventively can significantly reduce the risk of young children with hemophilia A developing joint damage associated with joint bleeding.

The boys participating in the study were treated either with prophylactic or regular, continuous infusions to prevent bleeding, or treated at the time of and in response to bleeding known as episodic on-demand treatment. The findings showed that 93 p.c. of boys who received prophylactic treatment had normal joints at age six compared to only 55 p.c. in the episodic treatment group – the strongest medical evidence to date comparing joint outcomes associated with the two forms of treatment.

“ Our results show for the first time that prophylaxis, initiated between six and 30 months of age, is effective at preventing joint bleeds and preserving joint function and structure in young boys with hemophilia A,” said Marilyn Manco-Johnson, MD, principal investigator of the study and director of the Mountain States Regional Hemophilia and Thrombosis Center at the UCD School of Medicine.

“ These results provide health care professionals – as well as parents of children with hemophilia A – with solid information to guide optimum treatment.”

The multi-center, five year study was in collaboration with the Centers for Disease Control and Prevention and the National Institutes of Health.

 

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