Hemophilia
Treatment

The present goal of therapy is to raise factor levels, decrease the frequency and severity of bleeding episodes and prevent the complications of bleeding. This is done by injecting the missing clotting factor into your child's vein soon after he has injury or shows signs of bleeding.

Clotting factor concentrate, also called "factor," is a dried powder form of the clotting factor. It is mixed with water to form a liquid before it is given. Some clotting factor products, called plasma-derived factor, are made from donated human blood plasma. Others, called recombinant factor, are made in a laboratory and do not use human blood proteins. Because recombinant products do not contain human blood, they are much safer since they avoid potential transmission of a virus from donated blood.

When clotting factor is administered, it immediately circulates in the blood so the body can use it to form a blood clot. Once the blood clot is established and the bleeding has stopped, the body begins to reabsorb the blood that has leaked into the tissues and joints.

If your child does not receive prompt treatment, extra blood can pool in the joint or soft tissue and cause pain and swelling that takes longer to go away. Over time, repeated bleeding into a joint can lead to severe joint damage and arthritis. Early treatment will minimize the risk of joint damage.

At first, your child may only need to be treated episodically for bleeding disorders, that is, each time he or she experiences a bleeding episode. However, as the child gets older and becomes more active, the frequency of bleeding episodes may increase. Doctors may recommend giving factor replacement treatments every other day, a therapy regimen called prophylaxis, to prevent most bleeding. Prophylaxis reduces the number of bleeds, but does not prevent all bleeding. The goal of prophylaxis is to make a person with severe hemophilia reach factor VIII or factor IX levels similar to patients with moderate hemophilia, about 1 percent to 5 percent.

Treatments include home therapy, over-the-counter medications and gene therapy.

Home Therapy

All factor treatments are infused or injected intravenously into a child's vein. At first, a child will be treated at a hemophilia treatment center, his doctor's office or an emergency room. Later, parents may be taught how to give the factor at home. Devices, called ports, can be surgically inserted under the skin in the chest area to make it easier to administer clotting factor products.

At first, it is helpful to have your child evaluated and treated for each bleed by your doctor. As your child grows, especially if he has severe hemophilia and bleeds frequently, you may want to learn how to give the factor-replacement treatments at home. Most families find home therapy a fast, easy way to treat a child with frequent bleeds. Moreover, most children who receive treatment at home eventually learn how to do the infusions for themselves. If you have questions or would like to try home therapy, talk to your doctor.

Whether or not your child is on home treatment, you should always have factor concentrate at home to take to the emergency room when your child needs a treatment. If the decision is made to infuse factor to treat your child's bleeding episode, the most important thing you can do is to give it as soon as possible. If there is a delay, however, you can apply ice to help shrink the size of the leaking blood vessels, limit the amount of bleeding into joints or tissues and prevent a small bleed from becoming a larger one. To avoid ice burn, place a cloth, such as a washcloth or a clean diaper, between your child's skin and the ice.

After your child receives factor treatment for a joint bleed, "rest, ice, compression and elevation" or RICE is required. Your child may also benefit from support devices, such as crutches, following a bleed into the knee or ankle or a sling following a bleed into a muscle or joint in the arm. Depending on the site of the bleed, your child may have to limit his activities for a few days after a bleed. Our health care team can help you decide what is right for your child.

Over-the-Counter Medications

Acetaminophen, sold under the brand names Tempra and Tylenol, is recommended as a safe pain reliever for children with hemophilia. Follow the directions carefully and be sure to give your child only the recommended amount of the medicine.

However, never give your child any product with aspirin, or acetylsalicylic acid, in it. Aspirin can interfere with clotting. Many common household remedies, such as Alka-Seltzer, contain aspirin, so read labels very carefully before you give your child any medication. Ibuprofen, such as Advil, Aleve and Motrin, also may interfere with clotting and should not be used by your child. If you have any questions about what is or is not safe for your child to take, talk to your doctor or hemophilia medical staff.

In addition, if your child has a head injury or symptoms of a head injury, do not give him any pain medicine unless your doctor instructs you to do so. Pain medicine can mask symptoms and make it difficult for the doctor to make a diagnosis about the seriousness of the injury.

Gene Therapy

With modern treatment, children born with hemophilia can expect to live a long, full life. Until the 1990s, this was not necessarily the case. But with safe recombinant clotting factors and with the prospect of gene therapy on the horizon, children born today can expect to live into their 70s or 80s.

Unfortunately, there is not yet a cure for hemophilia, though new developments may make a cure possible in the next five to 10 years. Technically, hemophilia can be cured through a liver transplant, but the risks involved in the surgery and the requirement for lifelong medications to prevent rejection of the new liver may outweigh the benefits.

Researchers are currently working on a way to insert the factor VIII or factor IX gene into the cells of patients with hemophilia to produce some clotting factor. People treated with this gene therapy should have fewer bleeding episodes. The present goal of gene therapy is to raise factor levels enough to decrease the frequency and severity of bleeding episodes and to prevent the complications of bleeding. Gene therapy does not replace the altered factor VIII or IX gene on the male's X chromosome. So, the daughters of a man with hemophilia still will be carriers, even if he is treated with gene therapy.

Reviewed by health care specialists at UCSF Benioff Children's Hospital.
Last updated July 14, 2010