How is hemophilia treated?
Treatment depends on the type and severity of the disorder. It consists of replacement therapy, in which human plasma concentrates or recombinant (produced from DNA) forms of the clotting factors VIII or IX are given to replace the blood clotting factors that are missing or deficient.
- Blood factor concentrates are made from donated human blood that has been treated and screened to reduce the risk of transmitting infectious diseases, such as hepatitis and HIV.
- Recombinant clotting factors, which are made in the laboratory and not from human blood, are commonly used today.
- Recombinant FVIII and FIX products, modified to survive longer in the circulation, are now available. They required less frequent dosing for prophylaxis or prevention of bleeding.
- Newer non-factor products are also being developed for prophylactic use in hemophilia. These have the advantage of much less frequent administration, and in some cases do not require intravenous administration.
During replacement therapy, the clotting factors are injected or infused (dripped) into a patient’s vein in the arm or a port in the chest. Usually, people with mild or moderate hemophilia do not need replacement therapy unless they are going to have surgery.
- In cases of severe hemophilia, treatment may be given to stop bleeding when it occurs. Patients who have frequent bleeding episodes may be candidates for prophylactic factor infusions. These are given two or three times per week to prevent bleeding from occurring.
- Some people with the mild or moderate form of hemophilia type A can be treated with desmopressin (DDAVP®), a synthetic (man-made) hormone that helps to stimulate the release of factor VIII and another blood factor that carries and binds to it. Sometimes desmopressin is given as a preventive measure before a person with hemophilia has dental work or some other minor surgical procedure. Desmopressin does not work for people with type B hemophilia or severe hemophilia A.
- Aminocaproic acid or tranexamic acid are agents that prevent clots from breaking down (“antifibrinolytics”). They may be used as an added therapy to treat nosebleeds or bleeding from having a tooth removed.
The only absolute cure for hemophilia is a liver transplant. However, much progress is being made in finding a genetic cure for persons with hemophilia.
Are there complications related to hemophilia treatment?
People with hemophilia who are treated with either plasma-purified or recombinant factor products may develop antibodies, called inhibitors, which will attack and neutralize their clotting factor. This is a severe complication that makes it very difficult to treat or prevent bleeding episodes. About one-third to one-fifth of people who have severe hemophilia A might develop an inhibitor. Inhibitors are much less common in hemophilia B. Hemophilia patients are tested frequently for a factor inhibitor, especially in childhood when they are first starting replacement therapy.
If you have an inhibitor, there are alternative medications that can bypass its negative effect and successfully treat a bleeding episode. For long-term control, however, a technique called immune tolerance induction (or ITI) is used to bring down the level of the inhibitor and enable factor replacement to work again. The protocol consists of daily doses of clotting factors given over a period of time, weeks or even years in some cases. Some people may also get immune-suppressing drugs. ITI is successful in about 70% of people with hemophilia A, and 30% for hemophilia B. New agents, specifically for patients with inhibitors have been developed and are quite effective. Your healthcare provider will discuss these options with you.
Are there home treatments for bleeds in people with hemophilia?
In most cases a child with severe hemophilia is managed at home by his parents, with the administration of factor on a prophylactic schedule. As the child gets older, he is taught how to self-administer his factor on schedule or when an acute bleed occurs. At the time of a joint bleed, you can treat the pain using the “RICE” (Rest, Ice, Compression, Elevation) protocol.
Pain medications are limited in hemophilia because common pain relievers such as aspirin, ibuprofen and naproxen can aggravate bleeding. If you or your child is suffering from pain related to an injury, you can use acetaminophen (sold under the brand name Tylenol®).