TEXTBOOK OF HEMOPHILIA

TABLE OF CONTENTS 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 Prophylaxis Kathelijn Fischer and H. Marijke van den Berg Part III: Inhibitors to factor VIII 5 Inhibitors to factor VIII: immunology Jean-Marie R. Saint-Remy and Marc G. Jacquemin 6 Genetic and environmental risk factors for FVIII inhibitor development Jan Astermark 7 Epidemiology of inhibitors in hemophilia Alfonso Iorio 8 Inhibitors to factor VIII: mild and moderate hemophilia Kathelijne Peerlinck and Marc G. Jacquemin 9 Inhibitors to factor VIII/IX: immune tolerance Donna M. DiMichele 10 Prophylaxis in inhibitor patients Alessandro Gringeri 11 Inhibitors to factor VIII: treatment of acute bleeds Claude Negrier Part IV: Acquired hemophilia 12 Acquired inhibitors to Factor VIII Craig M. Kessler Part V: Hemophilia B 13 Hemophilia B: molecular basis Keith Gomez, Pratima Chowdary 14 Factor IX inhibitors in hemophilia B Meera B. Chitlur and Jeanne M. Lusher 15 Treatment of inhibitors in hemophilia B Anand Tandra and Amy D. Shapiro Part VI: Pharmacokinetics of factors VIII & IX 16 Pharmacokinetics Sven Björkman 17 Individualised dosing Peter W. Collins Part VII: Hemophilia - birth to old age 18 The neonate with hemophilia Angela E. Thomas and Elizabeth A. Chalmers 19 Work-up of a bleeding child Manuel D. Carcao and Victor S. Blanchette 20 Care of the child with hemophilia Rolf Ljung 21 Adolescence Pia Petrini 22 Old age medicine and hemophilia Evelien P. Mauser-Bunschoten and Roger E.G. Schutgens Part VIII: Products used to treat hemophilia 23 Products used to treat hemophilia: recombinant products Midori Shima and Akira Yoshioka 24 Products used to treat hemophilia: plasma-derived coagulation factor concentrates Paul Giangrande 25 Products used to treat hemophilia: dosing Miguel A. Escobar 26 Products used to treat hemophilia: regulation Albert Farrugia 27 New drugs in the pipeline: From concept to clinic Leonard A. Valentino Part IX: Surgical management 28 General surgical management of patients with hemophilia Cindy Leissinger and Rebecca Kruse-Jarres 29 Continuous infusion of coagulation products in hemophilia Angelika Batorova and Uri Martinowitz 30 Surgery in inhibitor patients Pål Andrè Holme Part X: Musculoskeletal 31 Joint replacement Nicholas Goddard 32 Medical synovectomy (synoviorthesis) in hemophilia: Radiosynovectomy and chemical synovectomy E. Carlos Rodriguez-Merchan 33 Pseudotumors in patients with hemophilia Michael Heim and Uri Martinowitz 34 Imaging modalities for assessment of hemophilic arthropathy Andrea S. Doria and Björn Lundin 35 Physiotherapy in the management of hemophilia Sébastien Lobet and David Stephensen 36 Outcome assessment in hemophilia Pradeep M. Poonnoose and Alok Srivastava Part XI: Transfusion-transmitted Disease 37 Viral hepatitis and hemophilia Michael Makris and Geoffrey Dusheiko 38 Transfusion-transmitted disease: Emerging infections Thomas R. Kreil 39 vCJD and hemophilia Carolyn Millar Part XII: Gene therapy 40 Hemophilia gene therapy: an overview David Lillicrap 41 Gene therapy trials in hemophilia A and B Katherine A. High 42 Gene therapy: molecular engineering of factor VIII and Factor IX Sundar R. Selvaraj and Steven W. Pipe Part XIII: Laboratory 43 Laboratory and quality control of assays Steve Kitchen 44 Standardization of assays in hemophilia Sanj Raut and Trevor W. Barrowcliffe 45 Global laboratory assays in hemophilia Benny Sørensen and Guy Young Part XIV: Women and bleeding disorders 46 Obstetrics and gynecology: hemophilia Rezan A. Kadir and Christine A. Lee 47 Women and von Willebrand disease Peter A. Kouides Part XV: von Willebrand disease 48 von Willebrand disease: molecular aspects Daniel Hampshire and Anne Goodeve 49 von Willebrand disease: epidemiology Francesco Rodeghiero and Giancarlo Castaman 50 von Willebrand disease: biological diagnosis Veronica H. Flood and Robert R. Montgomery 51 Classification and clinical aspects of von Willebrand disease Augusto B. Federici 52 Treatment of von Willebrand disease: desmopression Pier M. Mannucci 53 Treatment of von Willebrand disease: therapeutic concentrates Erik Berntorp Part XVI: Rare bleeding disorders 54 Factor II Jan Astermark 55 Factor V and combined Factor V and VIII deficiencies Flora Peyvandi and Marzia Menegatti 56 Congenital factor VII deficiency Angelika Batorova 57 Factor X and Factor X Deficiency David J. Perry 58 Factor XI deficiency Paula H.B. Bolton-Maggs and Uri Seligsohn 59 Factor XIII deficiency Diane Nugent and Loan Hsieh 60 Fibrinogen deficiency Michael Laffan 61 Miscellaneous rare bleeding disorders Frederico Xavier and Amy D. Shapiro Part XVII: Emergency medicine 62 Emergency medicine and inherited blood disorders W. Keith Hoots Part XVIII: Evaluation of hemophilia 63 Clinical trials and other methodologies Sharyne M. Donfield and Alice E. Lail 64 Quality of life in hemophilia Sylvia von Mackensen and Alessandro Gringeri 65 The economics of hemophilia treatments Katarina Steen Carlsson and Erik Berntorp Part XIX: Comprehensive care and delivery of care 66 Hemophilia databases Charles R.M. Hay 67 Comprehensive care and the delivery of care: the developed world Christopher A. Ludlam and Cedric R.J.R. Hermans 68 Comprehensive care and delivery of care in hemophilia: the developing world Alok Srivastava and Auro Viswabandya 69 Comprehensive care and delivery of care: the global perspective Mark W. Skinner and Alison M. Street