Hemophilia Therapy

Hemophilia is a rare disorder in which a person’s blood does not clot properly because they do not produce enough of, or are missing, blood clotting proteins called factors. The body depends on these clotting factors to stop bleeding after injury and to promote healing.

The hemophilia community considers infusion (into a vein) therapy a viable option. For patients with hemophilia A, this is known as factor VIII replacement therapy. This therapy works as a substitute for the missing or nonfunctioning clotting factor VIII protein. When clotting factor is administered after an injury the body begins to use it immediately to form a clot. Replacement therapy works for about 24 hours. If bleeding continues, additional factor may need to be infused. This therapy is not a cure for hemophilia A - it is a temporary replacement therapy used to treat symptoms of hemophilia. A well-recognized treatment for hemophilia is an ultra-pure factor VIII concentrate that is made using recombinant gene technology.

 

The most significant advances in hemophilia treatment have been made in the last four decades. Baxter introduced the first commercially available plasma-derived factor concentrate in the mid 1960s. This was a major advancement over earlier treatments, which contained much lower concentrations of antihemophilic factor. In the early 1970s home treatment of hemophilia became widely available offering people with hemophilia greater independence and reduced hospital stays. Today recombinant DNA technology and the discovery of genes that control production of factor VIII have led to the development of recombinant factor concentrates that do not rely on plasma at all. In 2003, Baxter introduced the first Factor VIII therapy made without any added human or animal plasma proteins and albumin in the cell culture process, purification and final formulation.

 

Patients should discuss with their physician which factor VIII replacement therapy is best for them.

In some individuals with severe hemophilia, the factor VIII replacement therapy is identified as a foreign substance by their immune system.  If this happens their immune systems will make antibodies against factor VIII.  The higher the inhibitor level the more factor VIII replacement therapy it takes to overcome the inhibition and produce clotting.  Sometimes the physician may prescribe different types of therapies  to successfully treat  individuals who develop severe inhibitors.

 

The information provided is not intended to be a substitute for professional medical advice.  A licensed healthcare professional should be consulted for diagnosis and treatment of any and all medical conditions.