Hemophilia

Bleeding problems caused by hemophilia can be prevented and treated with clotting factor replacement therapy. Clotting factors come from blood donors (plasma-derived clotting factors) or are produced in a laboratory (recombinant or DNA-engineered). The amount of clotting factor needed is determined by how severe the hemophilia is and/or the location of the bleeding. More clotting factor is needed for surgery or bleeding in the brain than for less serious situations, such as routine dental procedures.

Depending upon how severe your disease is, you may choose either to:

• Receive regularly scheduled infusions of clotting factor to prevent bleeding.
• Receive clotting factor on demand, that is, only after bleeding starts or before you participate in an activity that will likely cause bleeding.

Should I have regularly scheduled or on-demand clotting factor replacement therapy for hemophilia?

Sometimes a person's body develops antibodies—called inhibitors—to the injected clotting factor. People who have severe hemophilia or who receive clotting factor replacement for the first time are more likely to develop inhibitors, as are children. When this occurs, specially engineered replacement clotting factors may be required. Other treatment for clotting factor inhibitors includes therapy to suppress the immune system (immunosuppressive therapy).

Other Treatment Choices

Clotting factor replacement

Treatment for people who have inhibitors

What To Think About

Gene therapy may eventually be able to boost the body's ability to make clotting factor. Researchers have developed genes that can cause a person to make clotting factors (for example, factor VIII). These genes have been transplanted into people with hemophilia in research studies. But the people could only produce a very low level of factor VIII. And the clotting factor lasted for less than a year. But no serious side effects were seen. Gene therapy is still a promising treatment for hemophilia, and research is ongoing.²