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Using Drug for Prevention Might Help in Hard-to-Treat Hemophilia
Date:11/2/2011

By Amanda Gardner
HealthDay Reporter

WEDNESDAY, Nov. 2 (HealthDay News) -- A compound currently used to stop bleeding episodes in a subset of hard-to-treat hemophilia patients also seems to be effective in preventing bleeds before they start.

Although this study, published in the Nov. 3 edition of the New England Journal of Medicine, was a small one and didn't last long, the results do raise the possibility that, over time, this type of use will also prevent the much-feared joint damage that is a hallmark of the condition.

"This study shows that it is at least possible to keep these people relatively healthy, without bleeds, by using chronic prophylactic [preventive] agents," said Dr. Thomas Harrington, director of the Adult Hemophilia and Adult Sickle Cell programs at the University of Miami Miller School of Medicine. He was not involved with the study.

The research was funded by Baxter BioScience, which makes FEIBA (Factor VIII Inhibitor Bypassing Activity), the agent tested in this trial.

Most people with hemophilia have a genetic defect in a clotting factor known as factor VIII. This results in excessive bleeding and bruising either as the result of traumas -- even small ones -- or sometimes occurring spontaneously.

About half of the bleeds occur in the joints and can lead to painful and debilitating joint disease.

Clotting factor concentrates that contain factor VIII are extremely effective both to stop bleeds when they happen (called "on-demand" treatment) and to prevent bleeds when given three or so times a week.

The problem is that about one-third of hemophilia patients develop antibodies to factor VIII, meaning the compound just stops working.

So-called "bypassing agents" have been developed for patients who have factor VIII inhibitors, but these aren't nearly as effective and have only been used on an on-demand basis.

The authors designed this study t
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