Hemophilia B

Alternate Names : Factor IX Deficiency, Christmas Disease

What are the treatments for the disease?

An individual who develops bleeding problems needs to be treated regardless of the severity of the disease. Although fresh frozen plasma was used in the past, factor IX concentrate has been the standard of care for many years. This is purified, treated, concentrated human plasma that is high in factor IX activity. An individual's dose of factor IX and the duration of treatment are based on several factors. This includes the baseline factor IX activity level, total body weight, severity of bleeding, and site of bleeding. Someone with internal bleeding or central nervous system bleeding is treated more aggressively.

Factor IX is given by slow, intravenous infusion. A person can do this at home. A person who requires frequent infusions often has a central line surgically implanted. This is a catheter that goes into one of the large veins, and provides a painless way to give the factor IX. Serious hemorrhage may require treatment in the hospital.

Some persons have immune system problems that make hemophilia B worse. These persons may require treatment with medications to suppress the immune system.

Chronic joint deformity due to hemophilia is becoming less common. However, some individuals require operations for joint problems. The most common sites for surgery include the weight bearing bones of the hips and knees.

What are the side effects of the treatments?

Too much infusion of factor IX can cause blood clots to form in the veins. Medication to suppress the immune system can make the person more susceptible to infections.

Before current screening procedures, a factor IX transfusion carried a risk of HIV or hepatitis. Currently, with present screening techniques for factor IX products, the risk of acquiring hepatitis and HIV is very low. Individuals who have only been receiving blood products in the past 10 years have a very low risk of acquiring hepatitis and HIV from factor IX products.

What happens after treatment for the disease?

A person with hemophilia B is often tested after treatment to measure how effective the factor IX infusion has been. The site of bleeding may complicate this evaluation.

How is the disease monitored?

Periodic testing of factor IX levels may be used to help assess the response to treatment. It also can determine when another treatment is necessary. Monitoring may also require special x-ray tests, surgery, or other blood tests.