What is a bleeding disorder? Bleeding disorders occur when there is poor clotting due to deficient or missing proteins called factor. These disorders can cause heavy and prolonged bleeding, either spontaneously or after an injury. Hemophilia and von Willebrand disease are 2 common bleeding disorders. Bleeding disorders can be mild, moderate, or severe in nature. Severe bleeding disorders are usually detected in infancy, while milder versions of the disease may not be diagnosed until adulthood. Many bleeding disorders, including hemophilia and von Willebrand disease, are inherited conditions. There are other causes as well.
What is an inhibitor? In about 10 – 15% of people with hemophilia, the immune system produces an antibody (called an inhibitor) that destroys the clotting factor before it stops the bleeding. It is unclear as to why this happens. People with inhibitors experience much higher treatment costs than those who don’t develop this complication.
What are symptoms of bleeding disorders? Bleeding can happen externally or internally, affecting joints, muscles, and other organs. External bleeds can be characterized by excessive bleeding, including menstrual bleeding, and bruising. Internal bleeds can cause severe pain, swelling, joint damage, and joint destruction.
Who is affected by bleeding disorders? About 20,000 people in the United States have hemophilia, while approximately 1.4 million Americans have von Willebrand disease. All ethnic and socioeconomic groups are affected by bleeding disorders.
How are bleeding disorders treated? There is no cure for hemophilia or von Willebrand disease. Clotting factor replacement therapy which involves the intravenous infusion (i.e. injection) of blood clotting products, enables those with bleeding disorders to lead normal and productive lives. Infusions must be given as soon as possible after the start of bleeding or taken regularly (as prophylaxis) to prevent uncontrolled bleeding. Mild von Willebrand disease may also be treated with an intranasal medicine. The average cost for treating hemophilia is about $60,000 annually, but some may need hundreds of thousands of dollars worth of treatment every year. There are no generic substitutes for these treatments.
Where do people with hemophilia receive medical care? According to the Center for Disease Control, 70% of people with hemophilia receive care from Hemophilia Treatment Centers staffed with doctors, nurses, and social workers. New England HTC’s are located at Brigham and Women’s Hospital (MA), Children’s Hospital (MA), Massachusetts General Hospital (MA), UMass Memorial Hospital (Worcester), Dartmouth-Hitchcock Hemophilia Center (NH), Maine Medical Center, Rhode Island Hospital, University of Connecticut Health Center, Yale University School of Medicine (CT), and Fletcher Allen Hospital (VT). Mortality rates and hospitalization rates for bleeding complications from hemophilia were 40% lower among people who received care in Hemophilia Treatment Centers than among those who did not receive this care. Others may receive care from their primary care physician or a local hematologist.
Where do people with hemophilia receive their “medication”? People with hemophilia either get their “medication,” i.e. factor, through a 340B pharmacy at their Hemophilia Treatment Center or through a Homecare Company. Homecare Companies are staffed with pharmacists and nurses who not only provide factor, but like HTC’s, ensure patients have the proper supplies, know how to self-infuse at home, and provide a variety of services and information they need to maintain good health. (http://www.newenglandhemophilia.org/homecare.html) There are almost a dozen pharmaceuticals that manufacture clotting factor. Representatives from these also provide information and support to patients with bleeding disorders. (http://www.newenglandhemophilia.org/Pharmaceuticals.html)
What is NEHA? The New England Hemophilia Association works to improve the quality of life for individuals with bleeding disorders and their families through education, advocacy, and support. (www.newenglandhemophilia.org)