Treatments for Hemophilia

Treatment list for Hemophilia: The list of treatments mentioned in various sources for Hemophilia includes the following list. Always seek professional medical advice about any treatment or change in treatment plans.

• Transfusions
• Home intravenous transfusions
• Transfusion treatment strategies
  • On-demand infusion - the traditional use of infusion only when there are bleeding symptoms.
  • Periodic infusion (prophylaxis) - a preventive ongoing method that reduces complications but is costly and more involved.
• Clotting factor therapy - the use of replacements for the missing blood products by transfusion of concentrated clotting factors from blood donor.s
  • Factor VIII concentrates
  • Factor IX concentrates
• Special treatment required prior to surgery
• Special treatment required prior to dental procedures
• Special treatment required for any injury

Treatment of Hemophilia: medical news summaries: The following medical news items are relevant to treatment of Hemophilia:

• Federal Government promises inquiry into contaminated polio vaccine

Treatments of Hemophilia discussion: The introduction of factor VIII concentrate products in the 1970s allowed treatment at home and largely replaced the need for transfusion of whole blood or plasma in the hospital. An unfortunate result of increased freedom from bleeding as a cause of disability and death was infection with hepatitis from products made from pooled plasma. This culminated in the early 1980s with the unexpected and devastating infection of about 70 % of severe hemophiliac patients with HIV, the cause of AIDS. Since then, the development and improvement of methods for inactivating viruses with lipid envelopes in plasma, including factor VIII and IX concentrates, has improved the safety of transfused products to the point where the threat of infection with HIV, hepatitis B(HBV) or hepatitis C (HCV) viruses has been essentially eliminated. ¹

Although most individuals with hemophilia can use replacement products repeatedly without problems, about 20% develop neutralizing antibodies that make the product less effective. Antibody inhibitors are more likely to occur in individuals with severe hemophilia. At this time, it is not possible to predict who will develop the antibody inhibitors, but there is some evidence for genetic predisposition for an immune response. The treatment for people with inhibitors can be complex and expensive. Often more than one approach is tried before the bleeding is arrested. The decreased ability to control bleeding in the joints can lead to earlier development of arthritis. In some cases, immune tolerance can be induced which allows standard treatment to again be effective. Studies are being conducted to avoid or modify the immune response and to prepare recombinant factor VIII proteins with reduced antigenicity. ¹

Approximately 15 percent of severe hemophilia A patients and 2.5 percent of hemophilia B patients develop such antibodies after exposure to transfused factors. When inhibitors are present in large amounts, the patient may require very high and expensive quantities of transfused clotting factors to stem bleeding, and, in some instances, even that may not be effective. The factor VIII products produced through biotechnology have been found to cause inhibitors in only about 5 percent of patients and are, thus, safer in this respect. Nevertheless, these inhibiting antibodies will remain a concern for hemophilia patients unless our ability to understand and control the immune system is improved. A number of NHLBI-supported scientists are directing research at this problem. ²

Several European countries are treating hemophiliacs by periodic infusion (prophylaxis) regardless of bleeding status. This approach maintains the factor level high enough that bleeding, joint destruction, and life-threatening hemorrhage are almost entirely avoided. There are, nevertheless, serious disadvantages such as the need for frequent infusions, the requirement for almost continuous access to veins by catheters, and the considerable cost of factor. In the United States, it is estimated that most patients on prophylaxis which is begun in the first few years of life will easily exceed the common life-time insurance cap of $1,000,000 by the second decade of life. The treatment decisions are not easy ones. ²

Footnotes:
1. excerpt from Hemophilia Update 1997: NHLBI
2. excerpt from Hemophilia: NHLBI

Last revision: May 28, 2003