Treatment of Hemophilia
Hemophilia is a complex disorder. Good quality medical care from doctors and nurses who know a lot about the disorder can help prevent some serious problems. Often the best choice is a comprehensive hemophilia treatment center (HTC). An HTC provides care to address all issues related to the disorder, as well as education. The team consists of physicians (hematologists or blood specialists), nurses, social workers, physical therapists and other health care providers, who are specialized in the care of people with bleeding disorders.
The best way to treat hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. This is typically done by injecting treatment products, called clotting factor concentrates, into a person’s vein or under the skin. Clinicians typically prescribe treatment products for episodic care or prophylactic care. Episodic care is used to stop a patients’ bleeding episodes. Prophylactic care is used to prevent bleeding episodes from occurring. Today, people with hemophilia and their families can learn how to give their own clotting factor treatment products at home. Giving factor treatment products at home means that bleeds can be treated quicker, resulting in less serious bleeding and fewer side effects.
Blood Roots Documentary
This documentary highlights the impetus and benefits of comprehensive hemophilia care in the United States. The video features the HTC clinician pioneers from the Western, Mountain and Pacific Northwest States who initiated the -then radical – concept of team based care. The documentary also features an HTC patient. These pioneers tell an important historic story vital to today’s health care reform efforts which seek to integrate care through the patient centered medical home model.
Blood Clotting Factor
The two main types of clotting factor concentrates available are:
Plasma-Derived Factor Concentrates
Plasma is the liquid part of blood. It is pale yellow or straw colored and contains proteins such as antibodies, albumin and clotting factors. Several factor concentrate treatment products are available that are made from human plasma proteins. All blood and parts of blood, such as plasma, are routinely tested for viruses. The plasma is collected from many people and then it goes through several processes to separate it into components, such as clotting factors. The clotting proteins are then made into a freeze-dried product. This product is tested and treated to kill any potential viruses before it is packaged for use.
Recombinant Factor Concentrates
Until 1992, all factor replacement products were made from human plasma. In 1992, the U.S. Food and Drug Administration (FDA) approved recombinant factor VIII (8) concentrate, which does not come from human plasma. The concentrate is genetically engineered using DNA technology. Commercially prepared factor concentrates are treated to remove or inactivate bloodborne viruses. In addition, recombinant factors VIII (8) and IX (9) are available that do not contain any plasma or albumin and, therefore, cannot transmit any bloodborne viruses.
Other Treatment Products:
DDAVP® (Desmopressin Acetate)
DDAVP® is a chemical that is similar to a hormone that occurs naturally in the body. It releases factor VIII (8) from where it is stored in the body tissues. For people with mild, and some cases of moderate hemophilia, this can work to increase their own factor VIII (8) levels so that they do not have to use clotting factor. This medicine can be given through a vein (DDAVP®) or through nasal spray (Stimate®).
Amicar® (Epsilon Amino Caproic Acid)
Amicar® is a chemical that can be given in a vein or by mouth (as a pill or a liquid). It prevents clots from breaking down, resulting in a firmer clot. It is often used for bleeding in the mouth or after a tooth has been removed because it blocks a substance found in the saliva (spit) that breaks down clots.
Cryoprecipitate is a substance that comes from thawing fresh frozen plasma. It is rich in factor VIII (8) and was commonly used to control serious bleeding in the past. However, because there is no method to kill viruses, such as HIV and hepatitis, in cryoprecipitate it is no longer used as the current standard of treatment in the U.S.