Understanding Hemophilia -- Diagnosis and Treatment
How Do I Know if I Have Hemophilia?
Normally, when bleeding begins, a complex series of chemical events produces a "plug" to stop the bleeding; this plug is called a fibrin clot. The fibrin clot is the end-product of many different "clotting factors" reacting in the blood. Hemophilia is an inherited condition in which one of these clotting factors (mainly factor VIII or IX) is absent from the blood, so that it does not clot normally. If your health care provider suspects that you have hemophilia, you may be given blood tests to examine how well your blood creates this clot. A lab mixes your blood with specific chemicals in a test tube, to produce a fibrin clot. If such tests are abnormal, other blood tests are done to determine the amounts of factors VIII and IX in the blood. These tests help doctors diagnose the type of hemophilia and its severity.
What Are the Treatments for Hemophilia?
Treatments for hemophilia include:
- Receiving clotting factors replacement therapy
- Treatment for joint bleeding and other problems associated with hemophilia
The treatments you need will depend on the type of hemophilia and the severity of hemophilia. For instance, if you have mild hemophilia, you may need treatment only when you've been injured or in preparation for surgery. However, if you have severe hemophilia and bleed frequently, you may need regular treatment to help prevent bleeding and help protect your joints from deformity and disability.
Clotting Factors for Hemophilia
People with hemophilia receive the appropriate clotting factor (factor VIII or factor IX). The clotting factor is given intravenously (through a needle in your vein) to stop or prevent bleeding. Factor preparations come from two sources:
- Donated blood plasma
- Synthetic (DNA-derived)
In 2015, the FDA approved the drug Adynovate to help control bleeding in adults and children ages 12 and older with hemophilia A. Adynovate can be used both preventatively and as-needed to create clotting, and the drug reduces the frequency of Factor VIII infusions needed.
A form of clotting factor VIII derived from pigs (called Obizur) is approved to stop bleeding episodes in people with acquired hemophilia A. This rare, dangerous form of the disease is not inherited. Acquired hemophilia A can be related to pregnancy, cancer, or the use of certain medications. However, no cause can be found in about half of cases.