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. Newer recombinant products are now available which survive longer in the circulation, which means fewer doses are needed for treatment.

During replacement therapy, the clotting factors are injected or infused (dripped) into a patient’s vein. 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 DDAVP is given as a preventive measure before a person with hemophilia has dental work or some other minor surgical procedure.

DDAVP is not effective for people with type B hemophilia or severe hemophilia type A. Aminocaproic acid or tranexamic acid are agents which prevent breakdown of clots (“antifibrinolytic”), and are often used as adjunctive therapy to treat nosebleeds or bleeding from the mouth (such as after a tooth extraction).