How Hemophilia Is Treated
According to the U.S. Food and Drug Administration (FDA), although hemophilia remains incurable, treatment has improved tremendously in recent years. Treatment, which depends in part on the severity of the condition, may be given regularly to prevent bleeding or as needed.
Mild hemophilia (characterized by 6 to 49 percent of normal clotting factors in the blood) and moderate hemophilia (1 to 5 percent of normal clotting factors in the blood) usually do not require preventative (prophylactic) treatment. People with mild hemophilia generally only have bleeding problems following a serious injury or surgery, while those with moderate hemophilia may have bleeding problems after injury or surgery, or spontaneously.
The primary treatment for severe hemophilia (less than 1 percent of normal clotting factors in the blood) is called replacement therapy. Pain medication and physical therapy also may be used to reduce joint pain, swelling, and inflammation.
Replacement therapy involves injecting concentrated clotting factors (factor VIII for hemophilia A and factor IX for hemophilia B) into the patient's vein to replace the low or absent factors. Clotting factors from human blood (increasingly uncommon) or recombinant (artificial) DNA may be used.
More about Hemophilia Treatment
- Avoiding activities that carry a high risk of injury as well as medications (such as aspirin) that promote bleeding is recommended.
- A bleeding episode can be stopped by intravenous infusion of the missing coagulation factor. Factor VIII can often be administered by the patient himself; early treatment is best.
- In March 2014, the U.S. Food and Drug Administration (FDA) approved Alprolix, Coagulation Facter IX (Recombinant), Fc Fusion Protein to treat adults and children with hemophilia B. The goal of this treatment is to reduce the frequency of injections to prevent bleeding episodes.
- In June 2014, the FDA approved Eloctate, Antihemophilic Factor (Recombinant), Fc Fusion Protein to treat adults and children who have hemophilia A. This treatment is designed to last longer in the blood, requiring less frequent injections to reduce bleeding frequency.
- Episodes of uncontrollable bleeding require hospitalization and prolonged infusions of the missing clotting factor.
- Physical therapy may be advised to rehabilitate damaged joints. Activities such as swimming are encouraged; contact sports should be avoided.
- Desmopressin (DDAVP) injection or nasal spray may help stop bleeding by increasing levels of clotting factor.
- In 10 to 15 percent of patients, development of an inhibitor to Factor VIII renders treatment ineffective.
In October 2014, Antihemophilic Factor (Recombinant), Porcine Sequence (Obizur) was approved by the FDA to treat acquired hemophilia A. This acquired form of hemophilia is rare, but potentially life-threatening, and may be associated with pregnancy, cancer, certain medications, and other medical conditions. In about 50 percent of cases, acquired hemophilia A develops without a known cause.
When to Call a Doctor
See a doctor if you have a bout of uncontrolled bleeding.
Johns Hopkins Symptoms and Remedies: The Complete Home Medical Reference
Simeon Margolis, M.D., Ph.D., Medical Editor
Prepared by the Editors of The Johns Hopkins Medical Letter: Health After 50
U.S. Food and Drug Administration (FDA)
Updated by Remedy Health Media