Textbook of Hemophilia

"It has been more than 20 years since the publication of a textbook that focused on hemophilia and, consequently, this book seeks to and successfully fills an obvious void." ("New England Journal of Medicine") "The excellent reviews in this book will make it useful to a wide audience." ("New England Journal of Medicine") "This textbook not only will be a compulsory text for all haemophilia centres but will also serve as a valuable reference book for medical and biomedical scientists in training in haematology." ("British Journal of Biomedical Science") ."..this is the best haemophilia textbook on the market today." ("Clinical and Laboratory Haematology") "The target audience is trainees, as well as practising haematologists and haemophilia specialists. All members of the multidisciplinary team delivering haemophilia care including nurses, physiotherapists, laboratory technicians, and data managers will also appreciate the book. This is a book each haemophilia centre should have available and accessible. In practise, it is likely that most specialists will wish to purchase their own copy." ("Clinical and Laboratory Haematology")

Christine A. Lee, MA, MD, DSc, FRCP, FRCPath, Emeritus Professor of Haemophilia, University of London; Honorary Consultant Haematologist, Oxford Haemophilia & Thrombosis Centre, Oxford, UK. Erik E. Berntorp, MD, PhD, Professor of Hemophilia, Lund University; Director, Department of Coagulation Disorders, Malmo University Hospital, Malmo, Sweden. W. Keith Hoots, MD, Professor of Pediatrics, University of Texas M.D. Anderson Cancer Center; Professor of Pediatrics and Internal Medicine, University of Texas Medical School at Houston; Medical Director, Gulf States Hemophilia and Thrombophilia Center, Houston, Texas, USA.

Contributors ix Historical introduction xv Christine A. Lee Part I: Introduction 1 Overview of hemostasis 3 Kathleen Brummel Ziedins and Kenneth G. Mann 2 Cellular processing of factor VIII and factor IX 9 Michael U. Callaghan and Randal J. Kaufman Part II: Hemophilia A 3 Molecular basis of hemophilia A 23 Geoffrey Kemball-Cook and Keith Gomez 4 Prophylaxis 33 Kathelijn Fischer and H. Marijke van den Berg Part III: Inhibitors to Factor VIII 5 Inhibitors to factor VIII: immunology 43 Jean-Marie R. Saint-Remy and Marc G. Jacquemin 6 Genetic and Environmental Risk Factors for Factor VIII inhibitor development 48 Jan Astermark 7 Epidemiology of inhibitors in hemophilia 53 Alfonso Iorio 8 Inhibitors to factor VIII: mild and moderate hemophilia 59 Kathelijne Peerlinck and Marc Jacquemin 9 Inhibitors to factor VIII/IX: immune tolerance 64 Donna M. DiMichele 10 Prophylaxis in inhibitor patients 72 Alessandro Gringeri 11 Inhibitors to factor VIII: treatment of acute bleeds 78 Claude Negrier Part IV: Acquired Hemophilia 12 Acquired inhibitors to factor VIII 87 Craig M. Kessler Part V: Hemophilia B 13 Hemophilia B: molecular basis 97 Keith Gomez and Pratima Chowdary 14 Factor IX inhibitors in hemophilia B 103 Meera B. Chitlur and Jeanne M. Lusher 15 Treatment of inhibitors in hemophilia B 107 Anand Tandra and Amy D. Shapiro Part VI: Pharmacokinetics of Factors VIII and IX 16 Pharmacokinetics 117 Sven Bjrkman 17 Individualized dosing 123 Peter W. Collins Part VII: Hemophilia: Birth to Old Age 18 Neonate with hemophilia 131 Angela E. Thomas and Elizabeth A. Chalmers 19 Work-up of a bleeding child 138 Manuel D. Carcao and Victor S. Blanchette 20 Care of the child with hemophilia 145 Rolf C.R. Ljung 21 Hemophilia in adolescence 150 Pia Petrini 22 Old age medicine and hemophilia 154 Evelien P. Mauser-Bunschoten and Roger E.G. Schutgens Part VIII: Products Used to Treat Hemophilia 23 Products used to treat hemophilia: recombinant products 165 Midori Shima and Akira Yoshioka 24 Products used to treat hemophilia: plasma-derived coagulation factor concentrates 174 Paul L.F. Giangrande 25 Products used to treat hemophilia: dosing 180 Miguel A. Escobar 26 Products used to treat hemophilia: regulation 185 Albert Farrugia 27 New drugs in the pipeline: from concept to clinic 192 Leonard A. Valentino Part IX: Surgical management 28 General surgical management of patients with hemophilia 199 Cindy Leissinger and Rebecca Kruse-Jarres 29 Continuous infusion of coagulation products in hemophilia 204 Angelika Batorova and Uri Martinowitz 30 Surgery in inhibitor patients 213 Pl Andr Holme Part X: Musculoskeletal 31 Joint replacement in patients with hemophilia 221 Nicholas Goddard 32 Medical synovectomy (synoviorthesis) in hemophilia: radiosynovectomy and chemical synovectomy 228 E. Carlos Rodriguez-Merchan 33 Pseudotumors in patients with hemophilia 233 Michael Heim and Uri Martinowitz 34 Imaging modalities for assessment of hemophilic arthropathy 237 Andrea S. Doria and Bjrn Lundin 35 Physiotherapy in the management of hemophilia 247 Sbastien Lobet and David Stephensen 36 Outcome assessment in hemophilia 253 Pradeep M. Poonnoose and Alok Srivastava Part XI: Transfusion-transmitted Disease 37 Viral hepatitis and hemophilia 265 Michael Makris and Geoffrey Dusheiko 38 Transfusion-transmitted disease: emerging infections 272 Thomas R. Kreil 39 vCJD and hemophilia 277 Carolyn M. Millar Part XII: Gene Therapy 40 Hemophilia gene therapy: an overview 285 David Lillicrap 41 Gene therapy trials in hemophilia A and B 291 Katherine A. High 42 Gene therapy: molecular engineering of factor VIII and factor IX 298 Sundar R. Selvaraj and Steven W. Pipe Part XIII: Laboratory 43 Laboratory and quality control of assays 311 Steve Kitchen 44 Standardization of assays in hemophilia 318 Sanj Raut and Trevor W.