Treatments

Treatments for Hemophilia A

Back in the past of Queen Victoria , hemophilia was seen as a fatal disease that spells death for its victims. However the present holds a much brighter future for today's hemophiliacs. By using one of the following treatments, severe patients of Hemophilia A can lead a normal and carefree lifestyle.

 

Desmopressin :

An image of a typical bottle of desmopressin. Picture taken from http://www.petplace.com/drug-library/desmopressin-ddavp/page1.aspx

Desmopressin is a synthetic drug that is an analogue of antidiuretic hormone. This drug acts in the same way the antidiuretic hormone does. Desmopressin releases the von Willebrand Factor stored in the lining of our blood vessels. This is helpful to hemophiliacs, because one of von Willebrand Factor's duties is to transport Factor VIII to the injured site. Thus doctors believe that increasing the amount of von Willebrand Factor would increase the amount of Factor VIII in the blood stream.

However, desmopressin is only effective in mild and moderate cases of Hemophilia A. The drug is useless to severe cases of Hemophilia A and ineffective for Hemophilia B and C patients. Every individual is different, so consulting a doctor is the sure fire way to go in finding out whether desmopressin is best for you.

 

Factor Replacement Therapy:

Factor replacement therapy is the most widely used treatment for hemophilia. This treatment injects concentrates of Factor VIII into patients of Hemophilia A, giving them a temporarily normal clotting percentage. Factor replacement therapy gives the patients' “normal” blood, but the effects usually last for only a couple hours. This treatment is also available for patients of Hemophilia B.

Factor Concentrates are made into a powder that can be used and supplied at home. The concentrates are portable and relatively easy to apply. These concentrates must be infused or injected into the vein.

The frequency of using this therapy all depends on the case's severity.

There are currently two types of factor replacement therapy, each with their pros and cons.

>Plasma-Derived Concentrates

(From left to right): Hemofil-M, Alphanate SD, Novo Seven. Images taken from http://www.coagulife.com/3a_products.html

In the 1970s, plasma-derived concentrates were made widely available. The concentrates are compiled from donations of human plasma, and are often in consistent supply.

PRO: Cheaper than recombinant concentrates.

CON: Have been known to carry blood-borne illnesses

>Recombinant Concentrates

(From left to right): Recombinate, Advate, Helixate FS. Images taken from http://www.coagulife.com/3a_products.html

In 1992, recombinant concentrates, also known as genetically engineered concentrates, were made available to the public. The concentrates are created in a lab, and are absent of any animal or human blood. Although these factor concentrates are created artificially, they still have the same effects as plasma-derived concentrates. This type of replacement therapy is more commonly used than plasma replacement therapy.

PRO: Considered safer than plasma-derived concentrates, because it has no blood origin.

CON: Much more expensive!! Usually double to triple the price of plasma-derived concentrates.

 

Complications with Factor Replacement Therapy

>Blood-borne Illnesses

As I have previously mentioned, there have been problems with blood-borne illnesses in plasma-derived factor replacement therapy. There have been reports of Hepatitis A, B, and C in the pool of plasma donations. Between 1979 and 1985, twelve hundred individuals were infected with HIV-containing infusions.

After these instances, many precautions have been taken. The plasma concentrate now goes through viral inactivation measures, such as heating the concentrate with vapor and treating the concentrates with solvent detergents. Donors for the plasma are also thoroughly questioned. These methods have been said to be very effective, but most patients still use recombinant concentrates in fear of dangerous blood-borne illnesses.

>Inhibitors

When the factor concentrates are infused into the bloodstream, sometimes antibodies develop against the unknown substance. Antibodies, or inhibitors, fight against the factor concentrates, preventing the treatment from doing its job. The production of inhibitors most often transpires in children with severe Hemophilia A. Managing patients with inhibitors is very difficult for doctors. Doctors have found that using continuous injections of the concentrates would eventually stop the development of antibodies. However, this method is very costly and insufficient. Doctors and scientists are still researching a solution to the treatment's flaw.

 

Sources for this Page:

  • Bolton-Maggs, Paul H B, K John Pasi. "Haemophilias A and B." The Lancet Vol 361(2003): 1801-1809.
  • Mannuci, Pier M., Edward G.D. Tuddenham. "The Hemophilias - From Royal Genes to Gene Therapy." New England Journal of Medicine Vol. 344, No. 23(2001): 1773-1779.
  • "Bleeding Disorders, Factor Deficencies." Canadian Hemophilia Society. 2006. Canadian Hemophilia Society. 21 Nov 2006 < http://www.hemophilia.ca. >
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