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.

What To Expect

If you infuse soon after a bleed starts, you can stop the bleed before it gets bad. You might need more medical care. So work with your doctor to make a plan for what to do if you or your child has a bleed.

Why It Is Done

Control bleeding. Clotting factors can treat bleeding episodes. They are given as soon as possible after a bleed begins.

Prevent bleeding. Clotting factors can be given just before a procedure or surgery to prevent bleeding. You can also give yourself clotting factors just before you do an activity that might cause a bleed. Some people give themselves clotting factors on a regular schedule, such as 3 times a week, to prevent bleeding. This is called prophylactic therapy.

How Well It Works

If you have a bleed, the infusion of clotting factors stops bleeding within hours, although the exact amount of time varies. Heavy bleeding takes longer to control than light bleeding. If the proper amount of clotting factors is given, bleeding will stop normally.

Clotting factors can prevent bleeding when they are given before a procedure, surgery, or an activity that has a high risk of causing a bleeding problem.

If you get clotting factors on a regular schedule to prevent bleeding, you likely will have fewer bleeding episodes. You might also have a lower risk of problems linked to bleeding. These problems include long-term damage from repeated bleeding into your muscles or joints.

Risks

Inhibitors.

Some people develop antibodies to the injected clotting factors. These antibodies are called inhibitors. If you develop inhibitors, the usual forms of clotting factors may not effectively prevent or stop bleeding. Children are more likely to develop inhibitors than adults.

Viral infection.

The risk of getting a viral infection from plasma factor concentrates is very low. These products are processed to kill viruses. The products are also tested to make sure they do not contain viruses. There is almost no risk of an infection from a recombinant clotting factor.