Hemophilia
What Is Hemophilia?
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.
Diagnoses can be mild, moderate or severe, depending on factor levels in the individual’s 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.
It is estimated that roughly 33,000 people in the United States live with hemophilia. While this affects males most severely, females can have hemophilia, too. Inhibitors in hemophilia present complicated treatment challenges.
Treatment
There is no cure for hemophilia, yet.
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). These facilities are typically staffed with doctors, nurses, social workers, physical therapists, dentists, and many other specialists who help oversee the comprehensive care of people living with a bleeding disorder. Others may receive care from their primary care physician or a local hematologist.
Inhibitors
Roughly 15 – 20% of people living hemophilia have an inhibitor. This is when the immune system produces an antibody (called an inhibitor) that destroys the clotting factor treatment, 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.