Healing History
History of Treatment
Each time internal bleeding occurs in a person with hemophilia, treatment with an infusion of a clotting factor may be required to replace the missing protein needed to form a blood clot. The infusion will stop the bleeding. However, because the infused clotting factor remains active for only a short time, serious bleeds may require repeated infusions to stop the bleeding. Many people with severe hemophilia use clotting factor on a regular basis to prevent or avoid bleeding episodes. This preventive use of factor is known as prophylaxis.
Until 1965, the only available treatment for hemophilia was whole blood or fresh-frozen plasma transfusions that could only be given in hospitals. These transfusions were only partly effective because the body cannot hold the large amounts of fluid needed to provide enough clotting factor to control bleeding fully. As a result, joint damage in persons with severe hemophilia was often serious enough to require the use of wheelchairs or crutches at an early age.
In 1965, Dr. Judith Graham Pool discovered cryoprecipitate, the factor-rich component of blood. Cryoprecipitate is the blood fraction containing concentrated factor VIII. Cryoprecipitate allowed for easier, more effective, and more efficient treatment because less fluid had to be transfused into the patient.
By the early 1970s, clotting factors VIII and IX became widely available in a new concentrated, freeze-dried form. This made it possible for people to self-infuse at home, work, and school, which meant fewer hospitalizations, more flexibility, and better opportunities for preventive treatment.
Since then, much progress has been made in the treatment of hemophilia. Examples of these improvements include the availability of new clotting factor products and drugs such as:
desmopressin acetate (DDAVP), used to treat mild-to-moderate hemophilia A and von Willebrand disease
new, synthetic (not derived from plasma) clotting products that take advantage of recombinant technologies
better screening methods to detect and remove viruses and other agents from factor concentrates and blood products
improved surgical options
advanced genetic testing methods
medically supervised home-infusion therapy
and prophylactic treatment.
Current Treatments
New Approaches to Treatment
Prophylaxis
An important improvement in bleeding disorders treatment involves the use of clotting factor on a regular basis to prevent bleeds before they occur. The prophylactic (preventive) use of clotting factor offers a number of benefits to persons with bleeding disorders. Such use might help to reduce or prevent joint disease, lower the number of hospitalizations, reduce time lost from work or school, increase independence, and improve quality of life. Talk with your healthcare provider to find out more about prophylaxis in order to decide if this is the best treatment choice for you or your child.
Genetically Engineered Factor Products
Recombinant factors are products created through an advanced method called recombinant DNA technology. "Recombinant" has been "recombined" in the laboratory by breaking up and splicing together DNA from several different types of organisms. When producing clotting factor, this process, known as genetic engineering, does not use blood. Thus the risk of human blood-borne viruses and contaminants is avoided. This is why recombinant clotting factors are the safest factor products available today. Recombinant factor VIII was first approved by the U.S. Food and Drug Administration (FDA) in 1993. In early 1997, the FDA licensed a recombinant factor IX product.
|
