How Is Hemophilia Treated?

Treatment With Replacement Therapy

The main treatment for hemophilia is called replacement therapy—giving or replacing the clotting factor that’s too low or missing. Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are slowly dripped in or injected 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. 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—it takes only about 15 minutes to receive the factor.

You may have replacement therapy on a regular basis to prevent bleeding. This is called preventive or prophylactic (PRO-fih-lac-tik) therapy. Or, you may only need replacement therapy to stop bleeding when it occurs. This use of the treatment, on an as-needed basis, is called demand therapy. Therapy that’s given as needed is less intensive and less expensive than preventive therapy. However, there is a risk that bleeding will cause damage before the as-needed treatment is given.

Complications of Replacement Therapy

Complications of replacement therapy include:

• Developing antibodies, which are proteins that act against the clotting factors
• Developing viral infections from human clotting factors
• Damage to joints, muscles, or other parts of the body resulting from delays in treatment

Antibodies to the clotting factor. Antibodies destroy the clotting factor before it has a chance to work. This is a very serious problem, because it makes the main treatment for hemophilia—replacing clotting factors—no longer effective.

Antibodies to clotting factor develop in about 20 percent of people with severe hemophilia A and 1 percent of people with hemophilia B.

When antibodies develop, doctors may use larger doses of clotting factors or try different sources of the clotting factor. Sometimes, the antibodies go away. Researchers are studying ways to deal with antibodies to clotting factors.

Viruses from human blood factors. The viruses that cause AIDS (HIV) and hepatitis can be carried in clotting factors. However, there has been no documented case of these viruses being transmitted during replacement therapy for about a decade. Transmission of viruses has been prevented by:

• Careful screening of blood donors
• Testing of donated blood products
• Treating donated blood products with a detergent and heat to destroy viruses
• Vaccinating people with hemophilia for hepatitis A and B

Researchers continue to find ways to make blood products safer.

Home Treatment With Replacement Therapy

Both preventive and as-needed replacement therapy can be done at home. Many people learn to do the infusions at home for their child or for themselves. Home treatment has several advantages:

• You or your child can get treatment quicker when bleeding happens. Early treatment means that fewer complications are likely to occur.
• Fewer visits to the doctor or emergency room are needed.
• Home treatment costs less than treatment in a medical care setting.
• Home treatment helps children accept treatment and take responsibility for their own health.

Discuss options for home treatment with your doctor or your child’s doctor. A doctor or other health care provider can teach you the steps and safety procedures for home treatment. Another valuable resource for learning about home treatment is hemophilia treatment centers (discussed under “Living With Hemophilia”).

Vein access devices can be surgically implanted to make it easier to get into a vein for treatment with replacement therapy. These devices can be helpful when such treatment occurs often. However, infections can be a problem with these devices. Your doctor can help you decide whether this type of device is right for you or your child.

Other Types of Treatment

Desmopressin

Desmopressin (DDAVP) is a man-made hormone used to treat people with mild to moderate hemophilia A. DDAVP can’t be used to treat hemophilia B or severe hemophilia A.

DDAVP stimulates the release of stored factor VIII and von Willebrand factor and increases the level of these proteins in your blood. Von Willebrand factor carries and binds factor VIII, which then can stay in the bloodstream longer.

DDAVP usually is given by injection or in a nasal spray. Because the effect of this medicine wears off when used often, it’s given only in certain situations. For example, your doctor may have you take this medicine prior to dental work or before playing certain sports to prevent or reduce bleeding.

Antifibrinolytic Medicines

Antifibrinolytic medicines (including tranexamic acid and aminocaproic acid) may be used with replacement therapy. They're usually given as a pill, and they help keep clots from breaking down. They’re most often used:

• Before dental work
• For treating bleeding from the mouth or nose
• For mild intestinal bleeding

Gene Therapy

Researchers are trying to develop ways to correct the defective genes that cause hemophilia to cure the disorder. Such gene therapy hasn’t yet developed to the point that it’s an accepted treatment. But researchers continue to test gene therapies for hemophilia in clinical trials.

Which Treatment Is Best for You?

The type of treatment you or your child receives depends on several things, including how severe the hemophilia is, what activities you will be doing, and what dental or medical procedures you will be having.

• Mild hemophilia—Replacement therapy isn’t usually needed for mild hemophilia. But DDAVP is sometimes given to raise the body’s levels of factor VIII.
• Moderate hemophilia—You may need replacement therapy only when bleeding occurs or to prevent bleeding that could occur when participating in some activity. DDAVP is another treatment option on occasion, prior to having a procedure or doing an activity that increases the risk of bleeding.
• Severe hemophilia—You usually need replacement therapy to prevent bleeding that could cause permanent damage to your joints, muscles, or other parts of the body. Typically, replacement therapy is given at home two or three times a week. It may be needed on a long-term basis or just for short periods when you expect to do an activity that might increase your risk of bleeding. However, some people with severe hemophilia receive treatment only when bleeding occurs.

For all types of hemophilia, getting treatment quickly for bleeding to limit damage is important. Learn to recognize signs of bleeding. Family members also should learn to watch for signs of bleeding in a child with hemophilia. Children sometimes ignore signs of bleeding because they want to avoid the discomfort of treatment.