Hemophilia Treatment
While a cure for hemophilia is not currently available, the disorder can be managed effectively by doing the following:
- Get an annual comprehensive checkup at the hemophilia treatment center
- Get vaccinated – Hepatitis A and B are preventable
- Treat bleeds early and appropriately
- Exercise to protect joints
- Get tested regularly for blood-borne infections
Factor Replacement Therapy
Treating bleeds early is important and can be done by intravenous clotting factor replacement. It can even be done at home. Factor replacement prevents serious complications and allows individuals to live full and active lives. Factor replacement therapy's goal is to stop or prevent bleeding by increasing the level of the deficient factor. Both factors VIII and IX are available as natural or synthetic (recombinant) powders that can be diluted in sterile fluid and infused directly into a vein.
Treating Hemophilia A
Hemophilia A is caused by a deficiency of active clotting factor VIII. The current treatment for Hemophilia A is the infusion of factor VIII, which normalizes the clotting process and stops or prevents bleeding. Factor VIII is available in a powdered form called a concentrate. When clotting factor is needed, the powder is dissolved in a sterile solution and administered into a vein. Factor VIII can be prepared from human plasma, the liquid part of the blood. It is then treated or filtered in various ways to kill or remove blood-borne viruses. These products are highly purified forms of human factor VIII. The final product is heat-treated to reduce the risk of transmitting dangerous viruses such as hepatitis and HIV.
Synthetic, or recombinant factor VIII concentrate, is manufactured from cultured animal cells, sometimes with small amounts of human protein (albumin) added to stabilize the product. The recombinant factor VIII is identical to human factor VIII. The Medical and Science Advisory Committee (MASAC) of the National Hemophilia Foundation recommends recombinant factor concentrate as the preferred method of treatment because its primary source is not human blood.
An alternative type of treatment is available for mild Hemophilia A. A synthetic hormone called DDAVP causes the release of natural factor VIII in the blood. The hormone can be given by vein or as a nasal spray.
Treating Hemophilia B
Hemophilia B (Christmas disease) is caused by a lack of active clotting factor IX. It's less common than Hemophilia A. The current treatment for Hemophilia B is the infusion of factor IX directly into a vein to normalize the clotting process and stop or prevent bleeding. Factor IX is available in a powdered form called a concentrate. When factor is needed, the powder is dissolved in a sterile solution and administered into a vein. Factor IX can be prepared from human plasma, the liquid part of the blood. It is then treated or filtered in various ways to kill or remove blood-borne viruses.
Synthetic or recombinant factor IX concentrate is manufactured from cultured animal cells. The resulting product is identical to human factor IX. The Medical and Science Advisory Committee (MASAC) of the National Hemophilia Foundation recommends the recombinant form of factor IX concentrate as the preferred method of treatment.
Products
For Treating Hemophilia A
Plasma-derived, or Monoclonal, Factor VIII products:
- Method M
- Hemophil M
- Monoclate P
Synthetic, or recombinant factor VIII products:
- Recombinate
- Refacto
- Kogenate FS
- Helixate FS
- Advate
DDAVP Products:
- Intravenous preparations
- Nasal (Stimate) preparations
For Treating Hemophilia B
Plasma-derived Factor IX products:
Synthetic, or recombinant factor IX product:
Inhibitors
Some people with hemophilia form inhibitors, which are antibodies produced by the body's immune system to destroy or neutralize the infused clotting factor. This destruction makes the clotting factor ineffective in controlling bleeding.
Usually, inhibitors develop in individuals with severe hemophilia, but no relationship exists between the amount of clotting factor used and the likelihood of forming inhibitors. The cause of inhibitor formation remains unclear, although it appears that individuals with a family history of inhibitors are more likely to develop the complication. Normally, the inhibitors develop during early childhood. Screening for inhibitors is a routine part of the comprehensive examination at the hemophilia treatment center clinic.
Inhibitors can be categorized into two types: low and high responding inhibitors. Low responding inhibitors are ones in which the antibody level remains low despite repeated exposure to clotting factor. High responding inhibitors are ones for which the amount of antibody rises to very high levels upon repeated exposure to factor. The level of high responders may decrease if the person receives no further clotting factor; however, upon re-exposure, the inhibitor level will again rise.
In some persons, usually low responders, inhibitors can disappear without special treatment. If the inhibitor persists, immune tolerance therapy may be helpful. This treatment involves the frequent administration of large doses of clotting factor that reduces or eliminates the inhibitor. The treatment of bleeding in persons with inhibitors requires an individualized approach.
Written 4/06
