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 (VWD) are two 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 VWD, are inherited conditions. There are other causes as well.

In about 15 – 20% 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.

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.

Approximately 33,000 people in the United States have hemophilia, while roughly 3.2 million Americans have Von Willebrand Disease. All ethnic and socioeconomic groups are affected by bleeding disorders.

There is no cure for hemophilia or Von Willebrand Disease.

Clotting factor replacement therapy, which involves an intravenous infusion of blood clotting products or a subcutaneous injection of a non-factor replacement therapy, enables those with bleeding disorders to lead normal and productive lives. Treatments must be given as soon as possible after the start of bleeding or taken regularly (as prophylaxis) to prevent uncontrolled bleeding. Mild VWD may also be treated with an intranasal medicine.

The average cost for treating hemophilia is about $300,000 annually, but some may require much more treatment every year. There are no generic substitutes for these treatments.

According to the Center for Disease Control, 70% of people with hemophilia receive care from Hemophilia Treatment Centers (HTCs) staffed with doctors, nurses, and social workers. A list of HTCs in New England can be found by clicking here.

Mortality rates and hospitalization rates for bleeding complications from hemophilia were 40% lower among people who received care in an HTC than among those who did not receive this care. Others may receive care from their primary care physician or a local hematologist.

People with hemophilia either get their “medication,” i.e. factor, through a 340B pharmacy at their Hemophilia Treatment Center (HTC) or through a Homecare Company. Homecare Companies are staffed with pharmacists and nurses who not only provide factor, but like HTCs, they also 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. There are almost a dozen pharmaceuticals that manufacture clotting factor. Representatives from these also provide information and support to patients with bleeding disorders.