Textbook of Hemophilia

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 Björkman 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 Pål Andrè 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 Björn Lundin 35 Physiotherapy in the management of hemophilia 247 Sébastien 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 Sørensen 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