Textbook of Hemophilia

The Textbook of Hemophilia has become the definitive resource on all aspects of hemophilia, including diagnosis, management and treatment. It covers all the common and rare bleeding disorders, both in terms of clinical management as well as the genetic, laboratory, financial and psychological aspects. Complications of hemophilia care are well covered in chapters on inhibitors, musculoskeletal problems and transfusion-transmitted disease. This new edition includes all the latest developments in the field of hemophilia, including the molecular basis of the disease, the role of factors in coagulation, epidemiology, pharmacokinetics, treatment inhibitors and musculoskeletal aspects of the disease. It contains a new chapter on chronic pain management and coverage of new developments such as gene therapy, and highlights controversial issues and provides advice for everyday clinical questions. Christine A. Lee , MA, MD, DSc (Med), FRCP, FRCPath, Professor of Haemophilia, Director and Consultant Haematologist, Haemophilia Centre and Haemostasis Unit, The Royal Free Hospital, London, UK - Erik E. Berntop, 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 CONTENTS List of Contributors. Historical introduction ( Christine A. Lee ). Part I: Introduction. 1 Overview of Hemostasis ( Kathleen Brummel Ziedins and Kenneth G. Mann ). 2 Cellular processing of Factor VIII and Factor IX ( Michael U. Callaghan and Randal J. Kaufman ). Part II: Hemophilia A. 3 Molecular Basis of Hemophilia A ( Geoffrey Kemball-Cook and Keith Gomez ). 4 Phenotypic/genotypic relationship ( Kathelijn Fischer and H. Marijke van den Berg ). 5 Prophylaxis ( Kathelijn Fischer and H. Marijke van den Berg ). 6 Continuous Infusion of Coagulation Products in Hemophilia ( Angelika Batorova and Uri Martinowitz ). Part III: Inhibitors to Factor VIII. 7 Inhibitors to Factor VIII - Immunology ( Jean-Marie R. Saint-Remy and Marc G. Jacquemin ). 8 Genetic and environmental risk factors for FVIII inhibitor development ( Jan Astermark ). 9 Inhibitors to Factor VIII - Mild and Moderate Hemophilia ( Kathelijne Peerlinck and Marc Jacquemin ). 10 Inhibitors to Factor VIII/IX: Immune Tolerance ( Donna DiMichele ). 11 Inhibitors to factor VIII: treatment of acute bleeds ( Claude Negrier ). Part IV: Acquired Hemophilia. 12 Acquired Inhibitors to Factor VIII ( Suman L. Sood and Craig M. Kessler ). Part V: Hemophilia B. 13 Hemophilia B - Molecular Basis ( Keith Gomez ). 14 Factor IX Inhibitors in Hemophilia B ( Jeanne M. Lusher and Meera Chitlur ). 15 Treatment of Inhibitors in Hemophilia B ( Anand Tandra and Amy D. Shapiro ). Part VI: Pharmacokinetics of Factors VIII & IX. 16 Pharmacokinetics ( Sven Björkman and Erik E. Berntorp ). Part VII: Hemophilia - birth to old age. 17 The Neonate with Hemophilia ( Angela E. Thomas and Elizabeth Chalmers ). 18 Work-up of a Bleeding Child ( Manuel D. Carcao and Victor S. Blanchette ). 19 Care of the Child with Hemophilia ( Rolf Ljung ). 20 Adolescence ( Martti A Siimes ). 21 Old age medicine and hemophilia ( Evelien P. Mauser-Bunschoten and R.E.G. Schutgens ). Part VIII: Products used to Treat Hemophilia. 22 Products Used to Treat Hemophilia: Recombinant Products ( Akira Yoshioka ). 23 Products Used to Treat Hemophilia: Plasma-Derived Coagulation Factor Concentrates ( Paul Giangrande ). 24 Products Used to Treat Hemophilia: Recombinant Factor VIIa ( Ulla Hedner ). 25 Products Used to Treat Hemophilia: Dosing ( Miguel A. Escobar ). 26 Products Used to Treat Hemophilia: Regulation ( Albert Farrugia ). Part IX: Musculoskeletal. 27 Joint Replacement ( Nicholas Goddard ). 28 Synovioarthesis in Hemophilia ( E. Carlos Rodriguez-Merchan ). 29 Pseudotumours in patients with Hemophilia ( Michael Heim and Uri Martinowitz ). 30 Imaging modalities for assessment of hemophilic arthropathy ( Andrea S. Doria and Bjorn Lundin ). 31  Physiotherapy in the Management of Hemophilia ( Karen Beeton and Jane Padkin ). 32 Clinimetric Instruments in Haemophilia ( Pradeep M. Poonnoose and Frank van Genderen ). Part X: Transfusion-Transmitted Disease. 33 Hepatitis C Virus Infection and Liver Transplantation ( Michael Makris and Geoffrey Dusheiko ). 34 Emerging Infections ( Thomas R Kreil ). Part XI: Gene Therapy. 35 Hemophilia Gene Therapy: An overview ( David Lillicrap ). 36 Gene Therapy Trials ( Kathy High ). 37 Gene Therapy: Molecular Engineering of Factor VIII and Factor IX ( Steven W. Pipe ). Part XII: Laboratory. 38 Laboratory Assays in Hemophilia ( Jorgen Ingerslev ). 39 Standardization of Assays ( Sanj Raut and Trevor W. Barrowcliffe ). 40 Global laboratory assays in Haemophilia ( Benny Sørensen and Guy Young ). Part XIII: Obstetrics & Gynaecology. 41 Obstetrics and Gynecology: Hemophilia ( Rezan A Kadir and Christine A. Lee ). Part XIV: Von Willebrand Disease. 42 von Willebrand Disease: Molecular Aspects ( Anne Goodeve ). 43 von Willebrand Disease: Epidemiology ( Francesco Rodeghiero and Giancarlo Castaman ). 44 von Willebrand Disease: Biological Diagnosis ( Bob Montgomery and Veronica Flood ). 45 Classification and Clinical Aspects of von Willebrand Disease ( Augusto Federici ). Part XV: Treatment of Von Willebrand Disease. 46 Treatment of von Willebrand Disease: Desmopressin ( Pier Mannuccio Mannucci ). 47 Treatment of von Willebrand Disease: Therapeutic Concentrates ( Peter Collins ). 48 Women & Von Willebrand Disease ( Peter Kouides ). Part XVI: Rare Bleeding Disorders. 49 Factor II ( Jan Astermark ). 50 Factor V and combined Factor V and VIII Deficiencies ( Flora Peyvandi and Marta Spreafico ). 51 Congenital Factor VII Deficiency ( Guglielmo Mariani and Alberto Dolce ). 52 Factor X  Deficiency ( David J Perry ). 53 Factor XI Deficiency ( Uri Seligsohn and Paula Bolton Maggs ). 54  Factor XIII Deficiency ( Diane Nugent and Loan Hsieh ). 55 Fibrinogen Deficiency ( Michael Laffan ). 56 Miscellaneous Rare Bleeding Disorders ( Anjali A. Sharathkumar and Amy D. Shapiro ). Part XVII: Emergency medicine. 57 Emergency Medicine and Inherited Blood Disorders ( W. Keith Hoots ). Part XVIII: Quality of Life. 58 Quality of Life in Hemophilia ( Sylvia v. Mackensen and Alessandro Gringeri ). Part XVIII: Economics. 59 The Economics of Hemophilia Treatments ( Katarina Steen Carlsson and Erik Berntorp ). Part XVIV: Comprehensive Care and Delivery of Care. 60 National Haemophilia Databases ( Charles R.M. Hay ). 61 Comprehensive Care and the Delivery of Care: The Developed World ( Christopher A Ludlam ). 62 Comprehensive care and delivery of care in haemophilia: The Developing World ( Alok Srivastava and Auro Viswabandya ). 63 Comprehensive Care and Delivery of Care: The Global Perspective ( Bruce L. Evatt and Alison Street ). Index