How Is Hemophilia Treated?
The purpose of treatment is to prevent bleeding before it starts, stop bleeding after it begins, and lower a child’s risk of bleeding complications. Children with hemophilia should see their comprehensive care team every six to 12 months for follow-up care. Patients with special issues, such as frequent joint bleeds, may need to come to the clinic more often.
Treatment for hemophilia can include:
- Factor replacement therapy (for hemophilia A and B)
- Antifibrinolytics (for hemophilia A and B)
- Bispecific antibody therapy (hemophilia A only)
- Desmopressin (DDAVP) (hemophilia A only)
Talk to the care team about which over-the-counter drugs your child can have. If you are treating pain symptoms or a fever, it’s important to avoid non-steroidal anti-inflammatory drugs (such as Advil, Motrin, ibuprofen, naproxen, or aspirin). These drugs can increase bleeding problems in children with hemophilia.
Factor Replacement Therapy
Factor replacement therapy is one of the main treatments for hemophilia. It involves replacing a person’s missing clotting factor so that the blood can clot properly. Factor replacement therapy can stop bleeding episodes that are already happening. It can prevent most bleeding episodes when given regularly.
Your child can receive factor replacement therapy through a vein in the arm or a port in their chest. Children who need regular treatment can learn how to give themselves factor replacement therapy at home
Replacement factor can be made from:
- Recombinant factor. This type of factor is made in a laboratory. It is not made from human blood. Unlike factor made from human plasma, it carries no risk of infection.
- Plasma from healthy blood donors. Factor made from plasma is tested for safety. It could cause infection. Using factor made from plasma could lessen a child’s risk of forming inhibitors. Inhibitors are proteins that prevent factor from working correctly.
Antifibrinolytics
Antifibrinolytics (like tranexamic acid and Amicar®) are drugs that stop clots from breaking down too fast. They do not help the body form a new clot. Antifibrinolytics are available in liquid or pill form. They can be given through an intravenous (IV) line. They treat nosebleeds and bleeding in the mouth. This includes bleeding that can happen after a dental procedure.
Bispecific Antibody Therapy
Bispecific antibody therapy helps the blood to clot. The name of this therapy is emicizumab or Hemlibra®. This medication is injected under the skin into fatty tissue on a regular schedule. It is usually given every week, every two weeks or every four weeks.
Monoclonal antibody therapy acts like factor VIII, but is not factor. It doesn’t treat active bleeds. It can help prevent bleeds in children who have moderate or severe hemophilia A. This medicine can be used in children who have inhibitors and those who don’t.
Desmopressin (DDAVP)
Desmopressin, also known as DDAVP, is a medication for some children with mild hemophilia A. It is used to stop bleeding. It can be given into the vein or inhaled through the nose in a spray form. Not all children with mild hemophilia A will respond to this medication.
“Prophylactic” vs. “On-Demand” Treatment for Hemophilia
Children with hemophilia may need “prophylactic” treatment to prevent bleeding, or “on-demand” treatment for active bleeds.
Prophylactic Treatment
Children with severe or moderately severe hemophilia need prophylactic (preventative) treatment to help prevent spontaneous bleeding. Those on prophylactic treatment can treat with factor replacement therapy or the bispecific antibody therapy. The care team will teach you and your child how to give this treatment at home. Prophylactic treatment can help reduce hospital time. It can decrease future joint damage.
On-Demand Treatment
Children who are having an active bleeding episode need “on-demand treatment” with factor. This type of therapy is for children who have mild or moderate hemophilia. It often happens in the hospital or clinic setting.
If you think that your child has a bleed, they should get treatment as soon as possible.
Having treatment quickly will:
- Lessen pain
- Prevent long-term damage
- Decrease the number of treatments needed for the bleed or injury
Inhibitor
About one in five people with hemophilia A and three in 100 people develop an antibody that stops the replacement factor from working. This antibody is called an inhibitor. It makes treating bleeds more difficult. Standard treatments don’t work. So the care team has to use different strategies to treat bleeds.
