RESEARCH AGENDA ON COMPLICATIONS OF HEMOPHILIA
AND OTHER BLEEDING DISORDERS

EXECUTIVE SUMMARY

The House Committee on Appropriations in its Report 105-635 urged the National Heart, Lung, and Blood Institute (NHLBI) to develop a research action plan, working with the National Institute of Allergy and Infectious Diseases (NIAID) and the hemophilia scientific and medical community, to address complications of hemophilia and other bleeding disorders. In developing the plan, the Committee requested that the NHLBI work collaboratively with the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) on how to improve hepatitis treatment options for persons with hemophilia. The Senate Committee on Appropriations in its Report 105-300 urged the NHLBI to develop a collaborative research plan for development of promising hemophilia gene therapy technologies with the NIDDK, the National Human Genome Research Institute (NHGRI), and the hemophilia scientific and medical community. Both the House and Senate requested a report on the steps taken by March 31, 1999.

The major complications of hemophilia include hemorrhage; inability of the blood to form clots; development of arthritis in joints that incur hemorrhages; development of pseudotumors that can damage adjacent muscle, nerve, and bone; development of inhibitor antibodies that preclude effective treatment with replacement factors; and infection with human immunodeficiency virus (HIV), hepatitis viruses, and other blood-borne agents. Among other inherited bleeding disorders, the most common is von Willebrand disease (vWD). Severe vWD has complications similar to those of hemophilia.

The National Institutes of Health (NIH) supports a broad program of research relevant to hemophilia and other bleeding disorders. Overarching research goals are to (1) develop effective replacement products to address complications of hemophilia and other bleeding disorders, (2) assure the safety of the blood supply against transfusion-transmitted agents, (3) develop strategies and therapies to treat patients infected by transfusion-transmitted agents, and (4) find the means to cure bleeding disorders. Areas of research include studies of the mechanisms of bleeding and clotting; strategies to avoid formation of antibodies to blood coagulation factors; improvement of approaches to gene therapy; and therapeutic interventions for HIV/AIDS, hepatitis viruses/liver disease, and other transfusion-transmitted infections. Blood safety issues are of the highest priority and include continuous monitoring of the blood supply for infectious blood-borne agents and efforts to improve methods for detecting and inactivating them.

Research on all aspects of hemophilia and other bleeding disorders will remain a high NIH priority in the years ahead. For example, expanded efforts are being planned that will lead to better understanding of factors that affect the progression of HCV infection in hemophilia patients and other frequently transfused populations, development of safer and more effective vectors for gene transfer, and induction of immune tolerance to factor VIII.

The federal government recognizes blood safety, an issue of extreme importance to patients with bleeding disorders, as one of its most important health priorities. Federal blood safety activities are coordinated at several levels throughout the government. Key committees include the DHHS Advisory Committee on Blood Safety and Availability, the Public Health Service Blood Safety Committee, the Trans-Agency Hepatitis Working Group, and the Trans-NIH Hepatitis C Virus Working Group.

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