Hemophilia Care In The New Millennium (advances In Experimental Medicine And Biology)

There should be, and in the best of cases there is, a synergy between basic research and patient care. However, this synergy is hard to develop because the techniques required to be a successful researcher are so different from the skills required to be an outstanding physician. Harold R. Roberts, M.D., of the University of North Carolina at Chapel Hill, is an example of a physician-researcher who has benefited from having his feet in both the world of patient care and the world of the laboratory: he has let clinical problems direct his basic research effort and conversely has adopted research advances in his care of patients. Dr. Roberts's long and continuing career has included many research and clinical advances. He was part of the first group to determine the amino acid sequence of the important thrombin inhibitor hirudin and part of the group that prepared the first cryoprecipitates which were the first alternative to plasma as therapy in hemophilia A. Dr. Roberts has made significant advances in understanding the protein chemistry behind hemophilia B; he was among the first researchers to identify some patients as not being completely deficient but instead as having measurable levels of protein and subsequently demonstrated that this protein was dysfunctional. This important advance led him to a classification scheme for patients into Cross Reacting Material (CRM) positive, negative, and reduced. Dr. Front Matter....Pages i-xiv The Transfusion-Transmitted Viruses in Blood Transfusion....Pages 1-12 Are Patients with Glanzmann Thrombasthenia and the Bernard-Soulier Syndrome Protected Against Atherosclerosis?....Pages 13-29 Factor V: Dr. Jeckyll and Mr. Hyde....Pages 31-43 Gene Therapy for Hemophilia B: Aav-Mediated Transfer of The Gene for Coagulation Factor IX to Human Muscle....Pages 45-57 Use of Prophylaxis to Prevent Complications of Hemophilia....Pages 59-64 Factor Viii Inhibitors....Pages 65-73 Use of High Dose Factor Vila in Hemophilia Patients....Pages 75-88 The Incidence of Inhibitors in Hemophilia A and the Induction of Immune Tolerance....Pages 89-97 Mechanisms of B-cell Tolerance....Pages 99-108 Mechanisms of Unresponsiveness:T- And B-Cell Mediated Mechanisms of Anergy....Pages 109-117 CD4+ T Cells Specific for Factor VIII as a Target for Specific Suppression of Inhibitor Production....Pages 119-134 CD40L Pathway Blockade as an Approach to Immunotherapy....Pages 135-152 Back Matter....Pages 153-182 These are the proceedings of the international symposium to honor Dr. Harold R. Roberts, held in Chapel Hill, North Carolina in April of 1999. The volume covers prions and viral transmissions, unexplained aspects of hemophilia and hemostasis, novel approaches to therapy, and inhibitor development in hemophilia patients.