Hemophilia: Treatment

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Factor Replacement Therapy

Depending on the severity of the disorder – mild, moderate or severe – a person with hemophilia will usually treat his condition with an intravenous infusion of replacement clotting factor medication. Clotting factor (or just “factor”) medication is engineered to act as a replacement for the deficient clotting protein. Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are infused into a vein.

Clotting factor concentrates can be made from human blood that has been treated to prevent the spread of diseases, such as hepatitis or HIV. With the new methods of screening and treating donated blood, the risk of developing an infectious disease from clotting factors taken from human blood is now very small.

To further reduce that risk, you or your child can take clotting factor concentrates that don’t use human blood. These are called recombinant clotting factors. Clotting factors are easy to store, mix, and use at home, and typically take only about 15 minutes to infuse.

For some people with moderate to severe hemophilia – primarily children and teenagers – a preventive form of factor infusion therapy, called “prophylaxis”, is recommended to prevent bleeding episodes before they start. This may include a regular schedule of infusions 2 to 4 times per week. This form of therapy has been shown to improve outcomes by preventing bleeds and reducing long-term degradation of joints.

Other people with hemophilia may only need replacement therapy to stop bleeding when it occurs. This use of the treatment, on an as-needed basis, is called on-demand therapy. Therapy that’s given as needed is less intensive; however, there is a risk that bleeding will cause damage before the as-needed treatment is given. To manage this form of therapy, a patient should be able to detect an internal bleed early and promptly self-infuse before additional damage can occur.

Still others may use a mixture of treatment schedules that might include “planned preventive” infusions (or “secondary prophylaxis”). This form of treatment calls for infusions prior to sports, exercise or other activities that are likely to increase the risk of bleeding.