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    Treatment investigated to prevent blood-clotting disorders

    A new subcutaneous therapy could offer a promising solution to a bleeding disorder in both children and adults.

    Hemophilia A is an inherited blood-clotting disorder that affects about 20,000 persons in the United States—with about half of those having a severe form of the disorder. The disorder causes uncontrolled, spontaneous bleeding because of a lack of the clotting protein called factor VIII, which normally brings together clotting factors IXa and X in healthy individuals to stop bleeding. For individuals with hemophilia A, this clotting does not occur and bleeding can be life threatening. Replacement of the clotting factors isn’t easy, either, because some patients can develop antibodies that can block replacement of factor VIII.

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    Now a once-weekly treatment that could offer “dramatic” results for children with hemophilia A is under priority review by the US Food and Drug Administration (FDA).

    The treatment being investigated is a bispecific monoclonal antibody called emicizumab, according to data from the biotechnology company Genentech (South San Francisco, California). It was designed to utilize IXa and X proteins, which are required to activate the coagulation cascade needed for blood clotting. Two phase III studies are under way, dubbed the Haven 1 study examining the efficacy of the treatment in adolescents and adults aged older than 12 years with and without factor VIII inhibitors, and the smaller Haven 2 study examining the effect of the treatment on children aged younger than 12 years with factor VIII inhibitors. In both studies, the treatment is delivered subcutaneously once a week through a ready-to-use solution. Ongoing studies are also investigating whether less frequent dosing also may be as effective, according to Genentech.

    Guy Young, MD, director of the Hemostasis and Thrombosis Program at Children’s Hospital Los Angeles, California, professor of Pediatrics at the Keck School of Medicine of the University of Southern California, Los Angeles, and a Haven investigator, says the treatment could substantially improve quality of life and outcomes for patients with hemophilia A.

    “It offers the potential for improving the lives of patients with hemophilia by improving their outcomes while simultaneously reducing the treatment burden by offering a subcutaneous, once-weekly therapy instead of a several-times-a-week intravenous therapy,” Young says. “The trial data are dramatic. These are patients who are bleeding 20 to 30 times per year, and to see that number drop basically to zero is really something. It’s a very, very exciting innovation in an area where we are really desperate for some better medication.”

    NEXT: Promising study results

    Rachael Zimlich, RN
    Rachael Zimlich is a freelance writer in Cleveland, Ohio. She writes regularly for Contemporary Pediatrics, Managed Healthcare ...


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