Factor XIII deficiency can be treated by factor XIII concentrates. Factor XIII levels only need to be elevated slightly to prevent or stop the bleeding symptoms associated with the disorder.
Factor XIII concentrate, which is a blood product that contains a concentrated form of factor XIII, is used to treat individuals with factor XIII deficiency. Such products are created from the plasma of thousands of different blood donors. These products undergo a viral inactivation process that kills any viruses or similar pathogens that can potentially be present in the blood.
In the past, individuals with factor XIII deficiency were treated with fresh frozen plasma or cryoprecipitates. Fresh frozen plasma may be used if factor XIII concentrates are unavailable. Cryoprecipitates are no longer recommended because of the risk (albeit small) of infection from a virus or similar pathogen. There is also a risk of an allergic reaction with fresh frozen plasma or cryoprecipitates.
It is recommended that individuals with factor XIII deficiency undergo preventive (prophylactic) therapy with FXIII concentrate every 3-4 weeks in an attempt to prevent or minimize the symptoms of the disorder. Prophylactic therapy has been used to prevent mostly bleeding into the brain. The decision to undergo prophylactic therapy in factor XIII deficiency is made after careful consultation with a patient’s medical team.
In 2011, the U.S. Food and Drug and Administration (FDA) approved Corifact (FXIII Concentrate) for the routine prophylactic treatment of congenital factor XIII deficiency. Corifact is administered intravenously. In some cases, Corifact has been associated with adverse side effects such as blood clots (thromboses), and the benefits versus the risks of such therapy must be assessed on an individual basis. For more information, contact:
CSL Behring Website: http://www.corifact.com/
In extremely rare cases, inhibitors have developed in individuals with factor XIII deficiency. Inhibitors are autoantibodies. Antibodies are specialized proteins produced by the body’s immune system that destroy foreign substances directly or coats them with a substance that marks them for destruction by white blood cells. When antibodies target healthy tissue they may be referred to as autoantibodies. In factor XIII deficiency they are also called inhibitors because they mistakenly attack replacement factor XIII, inhibiting the effectiveness of the treatment. When inhibitors develop in individuals with factor XIII deficiency, additional therapy is required, specifically drugs that reduce the activity of the immune system (immunosuppressive agents).
In 2014, Tretten, a recombinant factor XIII replacement product, was approved for the prevention of bleeding in adults and children who have the rare clotting disorder congenital factor XIII A-subunit deficiency. Tretten is distributed by Novo Nordisk, Inc. USA. For more information, contact:
Novo Nordisk, Inc. Website: http://www.tretten-us.com/
Additional treatment for individuals with factor XIII deficiency is symptomatic and supportive. For example, excessive menstrual bleeding in women may be treated by hormonal contraceptives such as birth control pills or drugs known as antifibrinolytics, which prevent the breakdown of clots in the blood. Genetic counseling may be of benefit for affected individuals and their families.