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New Form of Recombinant Factor IX Requires Less Frequent Injections for Hemophilia B — Physician’s First Watch

Medical News |
December 5, 2013

New Form of Recombinant Factor IX Requires Less Frequent Injections for Hemophilia B

By Joe Elia

A modified version of recombinant factor IX allows patients with hemophilia B to undergo less frequent maintenance injections, according to a New England Journal of Medicine study. The modified factor IX (rFIXFc) incorporates an immunoglobulin fragment that prolongs its half-life in the blood.

The phase 3 study, conducted by the manufacturer, allocated some 120 patients with severe disease to various groups: the first group received weekly prophylaxis, the second received interval-based prophylaxis (starting with every 10 days), and the third got treatment as needed.

The half-life of the modified factor IX was more than double that of the standard. Prophylactic treatment reduced the frequency of bleeding episodes by over 80%, relative to on-demand treatment. Dosing frequencies in most participants went from at least two per week before the trial to one every 1 to 2 weeks.

In NEJM Journal Watch, hematologist David Green calculates the cost of prophylactic use at almost $200,000 a year.

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