Treatment & therapy
Hemophilia treatment is usually done by replacing or administering the clotting factor that is abnormal or missing. This is a protein we can infuse in the blood through a vein, and there are commercial concentrates we can use for this purpose. After some time, patients with hemophilia learn to use these commercial concentrates by themselves whenever they have a bleeding episode.
Besides using the clotting factor concentrates to stop bleeding, we can also use them as a prophylaxis to prevent new bleeding episodes. By doing this, it is possible to avoid severe bleeding episodes, especially before specific medical procedures.
Treatment is more difficult and expensive when hemophilia is caused by an inhibitor that prevents clotting factors from functioning correctly. These patients need more clotting factors than usual or another type of clotting factor that is even more expensive.
Treatment is usually administered at special medical facilities known as Hemophilia Treatment Centers. In these facilities, professionals will address medical treatment and provide the education and instruction hemophilia patients need to improve their quality of life.
Treatment medications typically include:
- Plasma-derived factor concentrates: It’s a concentrate of plasma, which is the yellow-colored liquid of the blood. It contains several proteins, including albumin and all of the clotting factors. Plasma is tested to make sure they do not carry any virus, and then it is separated into concentrates of its components. In this case, we use a concentrate of clotting factors, which are freeze-dried to make sure they do not contain pathogens.
- Recombinant factor concentrates: It is another alternative that does not come from human plasma. Instead, recombinant factor concentrates are genetically engineered and prepared for commercial use. They do not have any plasma particle, albumin, or anything else that may potentially carry a virus. The downside of using a recombinant factor concentrate is that it brings a higher chance of developing inhibitors.
Other therapeutic options available for patients with hemophilia include:
- Emicizumab: The commercial name is Hemlibra, and it is also known as ACE 910. It replaces the function of FVIII and can be used to prevent bleeding episodes in hemophilia A. It is injected under the skin, but similar to recombinant factor concentrates, this therapy carries a higher chance of developing inhibitors.
- Desmopressin Acetate: The commercial name is Stimate or DDAVP. This drug works similarly to a hormone in the body that releases more FVIII. It is appropriate in cases of mild hemophilia and applied intravenously or through a nasal spray.
- Epsilon Amino Caproic Acid: The commercial name is Amicar and has the ability to prevent the breakdown of blood clots. Thus, patients will have a firmer and more resistant clot that promotes hemostasis. It is commonly used in order to avoid gum or mouth bleeding, which is inhibited by substances in the saliva.