Clotting Factor Replacement for Hemophilia

Treatment Overview

Hemophilia can be treated by replacing missing blood clotting factors. This is called clotting factor replacement therapy. Clotting factors are replaced by injecting (infusing) a clotting factor concentrate into a vein. Infusions of clotting factors help blood to clot normally.

Clotting factor replacement therapy can treat bleeding episodes or prevent bleeding. It can prevent severe blood loss and complications from bleeding such as damage to muscle, joints, and organs.

Clotting factor concentrate

• Plasma factor concentrate is made from human plasma. The plasma is processed to separate the clotting factors from the other parts of the plasma. This creates the clotting factor concentrate. The concentrate is processed to kill any viruses that might have been in the plasma.
• Recombinant clotting factors are made in a lab. They don't come from blood. They are made with recombinant DNA technology. They are concentrated into a powder form that is then mixed with sterile water and injected.

Clotting factor concentrate can be given in a hospital. You can also give it to yourself or to your child at home. It is easily stored. You can carry factor concentrate with you.

Adults and parents of children who have hemophilia can learn to inject replacement clotting factors. Children may also be taught to infuse themselves with clotting factors at about age 10. Younger children and those who have developed antibodies (inhibitors) may not be able to infuse themselves.

Deciding about regularly scheduled treatment

If you are considering regularly scheduled clotting factor treatments, talk with your doctor. Together you can discuss the medical information and your personal preferences. Then you can decide what is right for you.

To make your decision, think about these questions:

• What are the benefits and risks of regularly scheduled treatment (prophylaxis) to prevent bleeding? How do they compare to the benefits and risks of giving yourself injections only when you need them, in response to a bleed or before an activity that may cause bleeding?
• Do you want to give yourself the injections on a regular schedule? Or do you want to give them only when you need them?
• What types of activities do you do? Do they raise the risk of a bleeding episode?
• How much does the cost of therapy matter to you? Getting regularly scheduled treatment can be very expensive. Health insurers may not cover the cost.