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Patients affected with hemophilia are currently at a crossroad in regards to treatment of their disease process. Science is broadening the collective manor that has been used to treat hemophilia. Historical treatment for persons with hemophilia has centered on intravenous (IV) replacement of the deficient factor protein to prevent and treat bleeds. Additionally immune reaction to factor replacement, known as inhibitor development, is a feared complication of hemophilia therapy that can make treatment with factor replacement ineffective and bleeds difficult to treat. The availability of prophylactic treatment for children has greatly improved quality of life and joint health in this population. Great advances have been made in gene therapy offering the potential for a cure, but this research continues to evolve slowly.
New therapies are providing the opportunity to confront some of these barriers. A vast number of extended half-life products have entered the market within the past few years and offer some relief to the challenges pertaining to the frequency of treatment. Extended half-life products are available to patients with hemophilia A (factor VIII deficiency) and B (factor IX deficiency). These products include Fc fusion proteins, pegylated proteins, and albumin fusion protein and are all slightly different in structure and formulation.
Novel agents are still currently in clinical trials, but preliminary data are showing exciting results that may significantly alter the treatment approach providers use for patients with hemophilia. These novel therapies utilize subcutaneous injections versus traditional peripheral IV prophylaxis, once weekly or once monthly injections versus thrice weekly infusions, and the agents can be used in patients with inhibitors.
Numerous changes have occurred over the past few years for the treatment of hemophilia. The treatment continues to evolve. This discussion will provide a comprehensive review of progressive treatments.
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