Treatment for Hemophilia
When a person with Hemophilia A or Hemophilia B has a small cut or scrape, the injured blood vessel gets smaller and the platelets make a plug. This is usually enough to stop the bleeding. You can help by pressing down on the cut with a bandage. More severe bleeding and bleeding inside the body must be treated as soon as possible. Bleeding inside the body can fill joints, muscles, and organs and cause damage.
The treatment for a person with Hemophilia focuses on reducing bleeding and preventing rebleeding and allowing healing. One of the strategies is to replace the clotting factor he/she is missing. This is called "factor replacement therapy." Most people with Hemophilia just call it "taking factor". The factor, also known as factor concentrate or clotting factor concentrate, which often comes as a white dry powder in a glass vial. When a person with Hemophilia needs to take factor, they add the liquid to the powder and mix until it dissolves. They then inject the liquid factor into a vein with a needle. The clotting factor goes right into the blood where it is needed. The clotting factor helps a fibrin clot to form and the bleeding stops. Additionally, there is “non-replacement therapy” which does not treat a bleeding episode but can prevent bleeding and reduce the need for factor replacement for bleeding episodes. Finally, your HTC may use a class of drugs called antifibrinolytics (aminocaproic acid and tranexamic acid) as adjunctive therapy. It is often used to improve overall healing and treat skin, menstrual and mucosal bleeding. It comes in an intravenous, oral, and nasal forms. The chapter called Treatment and Care Options talks more about taking factor, non-replacement therapies, and antifibrinolytics.
People with Hemophilia usually see a team of experts for their medical care. This is called comprehensive care. These experts are part of a Hemophilia Treatment Center (HTC), also called a comprehensive care center. The Treatment and Care Options chapter tells all about comprehensive care. This kind of care has been shown to reduce problems from Hemophilia, save money, and extend life.
Curing Hemophilia
At this time, there is no cure for Hemophilia. There is, however, great hope in a treatment known as gene therapy. Several types of gene therapy are being tested and 2 products are approved, one for Hemophilia A and the other for Hemophilia B. In general, gene therapy involves taking unaffected genes (the ones that tell the body to make clotting factor) or ones that produce large amounts of clotting factor and putting them into the body of a person with Hemophilia, typically in the liver. The new genes should cause the clotting factor level in the blood to rise, and this does not affect your own genes that carry the affected Hemophilia gene. People who had successful gene therapy may not need prophylactic factor replacement and only need it for bleeding episodes and procedures. They would be close to being cured of Hemophilia. It is possible that the genes would only provide a partial cure and last only 5-10 years. People with severe Hemophilia might be raised to the level of mild Hemophilia, needing to take factor less often.
In a small number of people with Hemophilia who have had liver transplants, their Hemophilia has been cured. This is because clotting factor is made in cells in the liver. The new livers they received made normal amounts of factor for them. Transplants are too risky to use as a cure for Hemophilia. Liver transplants are done only to save someone's life when the liver has stopped working because of infections related to chronic Hepatitis B or C.