The basic treatment to stop or prevent bleeding in people with hemophilia A and B is factor replacement therapy. This is the infusion (injection into the bloodstream) of factor VIII and IX concentrates to prevent or control bleeding.
These concentrates come from two sources:
The clotting factors available in Canada today have many advantages over products available in the 1950s, 1960s, 1970s and 1980s. (See The history of hemophilia.) These are the advantages.
Factor VIII and IX concentrates can be divided into two categories:
Genetically engineered (recombinant) factor VIII is the clotting factor concentrate used by most Canadians with hemophilia A. It has been the product of choice since being introduced in 1993. The brand names used in Canada are Kogenate®FS, Advate®, Helixate®FS and Xyntha®.
Plasma-derived factor VIII is sometimes used in particular situations for the treatment of hemophilia A: 1) for previously untreated patients who may be at higher risk for the development of inhibitors and 2) for immune tolerance induction for patients who have already developed antibodies to factor VIII. Brand names used in Canada are Humate P® and Wilate®.
Recombinant factor IX, which goes under the brand name of Benefix® is the blood product used by most Canadians with hemophilia B. It was introduced in Canada on a regular basis in 1998.
Plasma-derived factor IX is also used by Canadians with hemophilia B. The brand name used in Canada is ImmunineVH®.
Depending on the person, factor concentrates are given:
In prophylaxis therapy, hemophiliacs receive factor concentrates one or more times a week to prevent bleeding. The goal is to keep the levels of factor VIII or IX in the blood high enough that bleeding does not happen. This therapy is common with children with severe hemophilia.
On-demand therapy is the infusion of factor concentrates immediately after the beginning of a bleed. The goal is to stop the bleeding quickly, before any damage is done to the joint or muscle.
Research has shown that prophylaxis therapy gives children the best chance to reach adulthood without damage to their joints.
Yes, for most hemophiliacs, factor concentrates are very effective in stopping bleeding. Hemophiliacs can even have major operations without bleeding more than a person whose blood is normal.
However, some hemophiliacs' bodies reject the infusions of factor concentrates. They develop inhibitors. Inhibitors are the body's way of fighting off what it sees as a foreign agent. This means that as soon as the coagulation factor is injected, it is eliminated. This often happens before the factor concentrate is able to do its work and stop the bleeding.
Fortunately, there are ways to help most hemophiliacs who have inhibitors. (See The complications of hemophilia.)
No. Minor bleeding episodes associated with hemophilia sometimes do not require medical treatment. For example:
Bleeding into a joint or a muscle (especially around the hip, calf or forearm) is never minor. Treatment with factor concentrates is essential.
Doctors say, 'When in doubt, infuse. Ask questions later.'