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New Findings Offer Hope for Those With Severe Hemophilia

One study pinpoints when standard therapy works best; another uncovers the promise of a new drug

THURSDAY, May 26, 2016 (HealthDay News) — Two new studies could pave the way to major changes in how severe cases of hemophilia are treated. Both studies were published in the May 26 issue of the New England Journal of Medicine.

In one study, an international team of researchers randomly assigned 264 young children newly diagnosed with severe hemophilia to start replacement therapy with either blood-derived or recombinant factor VIII.

Overall, 37 percent of children on the recombinant therapy developed antibodies. That compared with 23 percent of children on blood-based therapy. Lead researcher Flora Peyvandi, M.D., of the University of Milan, said the findings suggest blood-derived factor VIII is the “better choice” for children beginning therapy. The findings do not apply to patients who’ve been on therapy for a while, Peyvandi told HealthDay.

The second study tested emicizumab in 18 patients with severe hemophilia. Over three months, 72 percent had no bleeding episodes, and it was just as effective in patients who’d developed antibodies to factor VIII as those who were antibody-free.

The second study was funded by Chugai Pharmaceutical, one of the companies developing emicizumab.

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