Perspectives on the use of factor IX complex concentrates in the treatment of bleeding in persons with acquired factor VIII inhibition

Article Abstract:

Factor VIII is a blood protein that is required for normal blood clot formation. Acquired hemophilia is a rare and life-threatening bleeding disorder that occurs when the body makes autoantibodies that inhibit the activity of factor VIII (factor VIII inhibitors). This condition has been treated with drugs that suppress the immune system and with infusions of purified factor VIII. Another therapeutic approach involves giving infusions of blood clotting factor IX (FIX) to bypass the need for factor VIII. In an attempt to determine the effectiveness of FIX in controlling bleeding, the National Heart, Lung, and Blood Institute sponsored a study to compare two different brands of FIX with a placebo in the treatment patients with acquired hemophilia. In this study, either brand of FIX was effective in roughly 50 percent of the bleeding episodes. Similar results were reported in two subsequent studies. The disadvantages of using FIX include the need to use large doses, the risk of developing hepatitis, and the risk of heart attack due to the potential formation of blood clots inside blood vessels. Although FIX has been reported to be effective in controlling bleeding in some patients with acquired hemophilia, the risks associated with this form of treatment need to be considered. (Consumer Summary produced by Reliance Medical Information, Inc.)

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The use of porcine factor VIII in the treatment of patients with acquired hemophilia: the United Kingdom experience

Article Abstract:

Acquired hemophilia is a rare bleeding disorder that develops when the immune system makes antibodies that inhibit the activity of factor VIII (a blood protein required for blood clot formation). These antibodies are called factor VIII inhibitors. One approach to treating acquired hemophilia involves giving intravenous infusions of purified factor VIII. Factor VIII purified from pigs (porcine factor VIII) has been used for this purpose. This article describes the outcome of 12 patients with acquired hemophilia who were treated with porcine factor VIII. Ten of the patients were treated with drugs that suppress the immune system in addition to treatment with porcine factor VIII. The average length of treatment was 8.5 days and the average number of infusions during this period was 11. Eighty-two percent of the patients had a good or excellent clinical response, and only one patient experienced a severe reaction to the treatment. These results show that porcine factor VIII is a safe and effective treatment for preventing bleeding in patients with acquired hemophilia. (Consumer Summary produced by Reliance Medical Information, Inc.)

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Intravenous immunoglobulin in the treatment of spontaneously acquired factor VIII:C inhibitors

Article Abstract:

Intravenous infusions of immunoglobulin G (IgG) have been used to treat a variety of autoimmune diseases, which involve the production of autoantibodies that attack and destroy the body's own molecules. Acquired hemophilia is a rare autoimmune disease that can cause life-threatening episodes of bleeding. It results from the production of autoantibodies that inhibit the activity of factor VIII:C, a blood protein required for blood clot formation. Hence, these autoantibodies are called factor VIII:C inhibitors. Several studies have reported that intravenous infusions of gamma globulin (IV IgG) are effective in treating most patients with factor VIII:C inhibitors. However, this method of treatment is not effective in treating all patients with acquired hemophilia. IV IgG has been reported to decrease the amount of factor VIII:C inhibitor present in the blood within 24 to 48 hours of treatment. It is suggested that IV IgG works by binding directly to the factor VIII:C inhibitors present in blood. (Consumer Summary produced by Reliance Medical Information, Inc.)

Immunoglobulin G

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