Textbook of Hemophilia

Content: 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 Bjorkman    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  Pal Andre 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 Bjorn Lundin    35 Physiotherapy in the management of hemophilia, 247  Sebastien 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. Barrowcliffe    45 Global laboratory assays in hemophilia, 328  Benny Sorensen and Guy Young    PART XIV: Women and bleeding disorders    46 Obstetrics and gynecology: hemophilia, 337  Rezan A. Kadir and Christine A. Lee    47 Women and von Willebrand disease, 345  Peter A. Kouides    PART XV: von Willebrand disease    48 von Willebrand disease: molecular aspects, 355  Daniel Hampshire and Anne Goodeve    49 von Willebrand disease: epidemiology, 362  Francesco Rodeghiero and Giancarlo Castaman    50 von Willebrand disease: biological diagnosis, 370  Veronica H. Flood and Robert R. Montgomery    51 Classification and clinical aspects of von Willebrand disease, 377  Augusto B. Federici    52 Treatment of von Willebrand disease: desmopressin, 386  Pier M. Mannucci    53 Treatment of von Willebrand disease: therapeutic concentrates, 390  Erik E. Berntorp    PART XVI: Rare bleeding disorders    54 Factor II, 399  Jan Astermark    55 Factor V and combined factor V and VIII deficiencies, 403  Flora Peyvandi and Marzia Menegatti    56 Congenital factor VII deficiency, 413  Angelika Batorova    57 Factor X and factor X deficiency, 421  David J. Perry    58 Factor XI deficiency, 428  Paula H.B. Bolton-Maggs and Uri Seligsohn    59 Factor XIII deficiency, 436  Diane Nugent and Loan Hsieh    60 Fibrinogen deficiency, 445  Michael Laffan    61 Miscellaneous rare bleeding disorders, 452  Frederico Xavier and Amy D. Shapiro    PART XVII: Emergency medicine    62 Emergency management of hemophilia, 463  W. Keith Hoots    PART XVIII: Evaluation of hemophilia    63 Clinical trials and other methodologies, 473  Sharyne M. Donfield and Alice E. Lail    64 Quality of life in hemophilia, 478  Sylvia von Mackensen and Alessandro Gringeri    65 The economics of hemophilia treatment, 489  Katarina Steen Carlsson and Erik E. Berntorp    PART XIX: Comprehensive care and delivery of care    66 Hemophilia databases, 497  Charles R.M. Hay    67 Comprehensive care and delivery of care: the developed world, 502  Christopher A. Ludlam and Cedric R.J.R. Hermans    68 Comprehensive care and delivery of care in hemophilia: the developing world, 508  Alok Srivastava and Auro Viswabandya    69 Comprehensive care and delivery of care: the global perspective, 515  Mark W. Skinner and Alison M. Street    Index, 523    Color plate section can be found facing page 202