Hemophilia Care in the New Millennium

1. Front Matter

   Pages i-xiv

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2. Infectious Disease Risk

   1. The Transfusion-Transmitted Viruses in Blood Transfusion

      • Naomi L. C. Luban, Rima F. Jubran

      Pages 1-12

3. Unexplained Aspects of Hemophilia and Hemostasis

   1. Are Patients with Glanzmann Thrombasthenia and the Bernard-Soulier Syndrome Protected Against Atherosclerosis?

      • Alan T. Nurden, Paquita Nurden, James N. George

      Pages 13-29

   2. Factor V: Dr. Jeckyll and Mr. Hyde

      • Michael Kalafatis, Kenneth G. Mann

      Pages 31-43

4. Novel Approaches to Therapy

   1. Gene Therapy for Hemophilia B: Aav-Mediated Transfer of The Gene for Coagulation Factor IX to Human Muscle

      • Peter J. Larson, Katherine A. High

      Pages 45-57

   2. Use of Prophylaxis to Prevent Complications of Hemophilia

      • P. M. Mannucci, L. Mendolicchio, A. Gringeri

      Pages 59-64

   3. Factor Viii Inhibitors

      • Pete Lollar, John F. Healey, Rachel T. Barrow, Ernest T. Parker

      Pages 65-73

   4. Use of High Dose Factor Vila in Hemophilia Patients

      • Ulla Hedner

      Pages 75-88

5. Inhibitor Development in Hemophilia Patients

   1. The Incidence of Inhibitors in Hemophilia A and the Induction of Immune Tolerance

      • Ernest Briët, Marjolein Peters

      Pages 89-97

   2. Mechanisms of B-cell Tolerance

      • Marc G. Jacquemin, Beatrijs Vanzieleghem, Jean-Marie R. Saint-Remy

      Pages 99-108

   3. Mechanisms of Unresponsiveness:T- And B-Cell Mediated Mechanisms of Anergy

      • Roberta Greenwood, Jeffrey Frelinger

      Pages 109-117

   4. CD4+ T Cells Specific for Factor VIII as a Target for Specific Suppression of Inhibitor Production

      • Mark T. Reding, Huiyun Wut, Mark Krampft, David K. Okita, Brenda M. Diethelm-Okita, Nigel S. Key et al.

      Pages 119-134

   5. CD40L Pathway Blockade as an Approach to Immunotherapy

      • Linda C. Burkly

      Pages 135-152

6. Back Matter

   Pages 153-182

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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.

• blood
• hemophilia
• immunotherapy
• muscle

• University of North Carolina School of Medicine, Chapel Hill, USA

  Dougald M. Monroe, Gilbert C. White

• Novo Nordisk A/S, Målov, Denmark

  Ulla Hedner

• University of Lund, Lund, Sweden

  Ulla Hedner

• Duke University and Durham VA Medical Centers, Durham, USA

  Maureane R. Hoffman

• Hôpital Edouard Herriot, Lyon, France

  Claude Negrier

• Guys’ and St. Thomas’s Trust, London, UK

  Geoffrey F. Savidge

Policies and ethics