Hemophilia is a genetic disorder which creates problems in the body’s blood clotting system. It is caused by a deficiency of factor VIII or factor IX. As a result, stable clots do not form and prolonged bleeding occurs. Hemophilia can be mild, moderate or severe, depending on factor levels in the blood. Contrary to popular myth, persons with bleeding disorders will not bleed to death from a cut or minor injury. They suffer mostly from internal bleeding into joints and muscles.
Hemophilia affects males most severely, but we now know females can have hemophilia too. Inhibitors in hemophilia present complicated treatment challenges. Hemophilia medicines, called factor concentrates, are among the most expensive medicines on the market. There are an estimated 1,000 persons with hemophilia in New England and 20,000 in the United States. For more information about Hemophilia and Bleeding Disorders, click here
Like hemophilia, von Willebrand disease is a genetic coagulation disorder. Symptoms may include easy bruising, frequent and prolonged nose bleeds, bleeding after dental procedures and surgeries, and for women, heavy menstrual periods. There are several different types of von Willebrand disease. Some types are due to a deficiency of von Willebrand factor in the blood, and some are due to a qualitative problem in the nature of the von Willebrand factor. VWD affects males and females in equal numbers. Diagnosis of VWD can be complicated and is best done at a specialized hemophilia treatment center.
Together with the National Hemophilia Foundation, NEHA is supporting "Victory for Women", a campaign to raise awareness among health care providers and the general public about women, girls and VWD.
Factor VII is the most prevalent of the rare deficiencies. The North American Rare Bleeding Disorders Registry (NARBDR) has documented that FVII deficient patients represent 46% of reported rare bleeding disorder patients (excluding FXI deficiency). 35% of FVII deficient patients have documented FVII levels of <20% activity level.
Although specific replacement products have been licensed for this disorder in much of the rest of the developed world, no replacement product is currently licensed to treat factor VII deficiency in the US. Recombinant factor VIIa is licensed to treat patients with iinhibitors to factor VIII and IX. Multiple reports exist in the literature regarding the use of rFVIIa for treatment of factor VII deficiency.
Click a link below for information about the specific rare factor deficiencies.
A common misperception exists that women do not have bleeding disorders. However, we now know this is far from true.
Von Willebrand disease, the most common bleeding disorder, affects both males and females in equal numbers. Carriers of hemophilia with low factor levels often suffer from problem bleeding. They have been called symptomatic carriers and may be said to have mild hemophilia. Women and girls may also have factor I, II, V, VII, X, XI, XIII deficiencies and platelet disorders.
Because of the myth that women do not have bleeding disorders, their condition often goes undiagnosed or misdiagnosed. This can cause serious consequences for women. Unfortunately it has led to some women suffering with debilitating menstrual bleeding every month without treatment. It has also resulted in unnecessary hysterectomies, life-threatening complicationsduring childbirth, surgery, and after injury and accidents. Because von Willebrand disease is so common, the American College of Obstetricians and Gynecologists has issued a recommendation that every adolescent girl with severe menorrhagia (heavy menstrual bleeding) be screened for von Willebrand disease. They have also recommended VWD screening for any adult woman with significant menorrhagia without another cause, and prior to any hysterectomy for excessive menstrual bleeding.
"Victory for Women", an initiative of the National Hemophilia Foundation, seeks to educate health care providers about women and bleeding disorders and build awareness among women and girls themselves. Informational pamphlets, reference lists, research results and other excellent materials are available through the NHF.
