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

o see if bypassing agents might prevent bleeds in this subset of patients in the same way that factor VIII concentrates successfully prevent bleeds in people without inhibitors.

Thirty-four patients over the age of 2 were randomly assigned to receive either six months of preventive thrice-weekly FEIBA (also known as AICC or anti-inhibitor coagulant complex) or to receive on-demand therapy.

After a three-month "washout" period, the groups were switched.

Twenty-six participants completed the study. The bleeding rates were reduced by 62 percent in the prophylaxis group compared to the on-demand groups.

Even patients with the most severe disease saw benefits, with the average number of bleeding episodes decreasing from 3.8 to 1.1 in the seven patients who had the most frequent bleeding before the study. Two of these individuals had no bleeding at all during the study period.

There was one allergic reaction to FEIBA, while several participants had complications from the hemophilia: Two adults died from bleeding episodes and two experienced brain bleeds.

Cost remains a significant issue.

Using the bypassing agent prophylactically would cost more than double what it would for on-demand (nearly $500,000 versus nearly $210,000 per patient in this study), the authors said, although these calculations didn't subtract for hospitalizations avoided and prevention of long-term complications.

"The cost is so extreme but the benefits are pretty extreme, too," said Harrington. "Three times a week is very, very costly [both for factor VIII concentrates and bypassing agents] but it keeps the joints healthy and provides good quality of life so these people can live normal lives."

FEIBA is licensed in the United States for on-demand treatment of hemophilia only. According to study author Dr. Cindy Leissinger, Baxter BioScience has started a study that "I feel quite sure is being done with a labeling indication in mind."


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