hemophilia

Treatment With Replacement Therapy





The main treatment for hemophilia is called replacement therapy. Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are slowly dripped or injected into a vein. These infusions help replace the clotting factor that's missing or low.

Clotting factor concentrates can be made from human blood. The blood is treated to prevent the spread of diseases, such as hepatitis. With the current methods of screening and treating donated blood, the risk of getting an infectious disease from human clotting factors is very small.

To further reduce the risk, you or your child can take clotting factor concentrates that aren't made from human blood. These are called recombinant clotting factors. Clotting factors are easy to store, mix, and use at home—it only takes about 15 minutes to receive the factor.

Home Treatment With Replacement Therapy

You can do both preventive (ongoing) and demand (as-needed) replacement therapy 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 quicker treatment when bleeding happens. Early treatment lowers the risk of complications.
  • 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.

Desmopressin

Desmopressin (DDAVP) is a man-made hormone used to treat people who have mild hemophilia A. DDAVP isn't used to treat hemophilia B or severe hemophilia A.

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

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

Gene Therapy

Researchers are trying to find ways to correct the faulty genes that cause hemophilia. Gene therapy hasn't yet developed to the point that it's an accepted treatment for hemophilia. However, researchers continue to test gene therapy in clinical trials.

Treatment of a Specific Bleeding Site

Pain medicines, steroids, and physical therapy may be used to reduce pain and swelling in an affected joint. Talk with your doctor or pharmacist about which medicines are safe for you to take.

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, the activities you'll be doing, and the dental or medical procedures you'll be having.

Mild hemophilia

Replacement therapy usually isn't needed for mild hemophilia. Sometimes, though, DDAVP is given to raise the body's level of factor VIII.

Moderate hemophilia

You may need replacement therapy only when bleeding occurs or to prevent bleeding that could occur when doing certain activities. Your doctor also may recommend DDAVP 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 damage your joints, muscles, or other parts of your body. Typically, replacement therapy is given at home two or three times a week. This preventive therapy usually is started in patients at a young age and may need to continue for life.

For both types of hemophilia, getting quick treatment for bleeding is important. Quick treatment can limit damage to your body. If you or your child has hemophilia, learn to recognize signs of bleeding.



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