Modern haemophilia care

The Lancet

Volumn 379, Issue 9824, 2012, Pages 1447-1456

  Berntorp, Erik ^a   Shapiro, Amy D ^b  

^a LUND UNIVERSITY   (Sweden)

^b INDIANA HEMOPHILIA AND THROMBOSIS CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; ATALUREN; CYCLOPHOSPHAMIDE; DESMOPRESSIN ACETATE; HYDROCORTISONE; IMMUNOGLOBULIN; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; TRANEXAMIC ACID;

BLEEDING; DRUG SAFETY; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HEPATITIS; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; NONHUMAN; PRIORITY JOURNAL; REVIEW; VASCULAR ACCESS; VIRUS TRANSMISSION;

EID: 84859596442     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(11)61139-2     Document Type: Review

References (131)

1. GC White 2nd, F Rosendaal, LM Aledort, JM Lusher, C Rothschild, J Ingerslev Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis Thromb Haemost 85 2001 560
2. Stonebraker JS, Bolton-Maggs PH, Soucie JM, Walker I, Brooker M. A study of variations in the reported haemophilia A prevalence around the world. Haemophilia 16: 20-32.
3. CD Forbes The early history of hemophilia CD Forbes L Aledort R Madhok Hemophilia 1997 Chapman and Hall London 3 6
4. GI Ingram The history of haemophilia J Clin Pathol 29 1976 469 479
5. EI Rogaev, AP Grigorenko, G Faskhutdinova, ELW Kittler, YK Moliaka Genotype analysis identifies the cause of the "royal disease" Science 326 2009 817
6. G Roosendaal, FP Lafeber Blood-induced joint damage in hemophilia Semin Thromb Hemost 29 2003 37 42
7. O Ramgren Haemophilia in Sweden. III. Symptomatology, with special reference to differences between haemophilia A and B Acta Med Scand 171 1962 237 242
8. R Biggs, AS Douglas, RG Macfarlane et al. Christmas disease: a condition previously mistaken for haemophilia BMJ 2 1952 1378 1382
9. PM Mannucci, EG Tuddenham The hemophilias - from royal genes to gene therapy N Engl J Med 344 2001 1773 1779
10. NS Key, C Negrier Coagulation factor concentrates: past, present, and future Lancet 370 2007 439 448
11. U Martinowitz, S Schulman, S Gitel, H Horozowski, M Heim, D Varon Adjusted dose continuous infusion of factor VIII in patients with haemophilia A Br J Haematol 82 1992 729 734
12. S Lethagen Desmopressin in mild hemophilia A: indications, limitations, efficacy, and safety Semin Thromb Hemost 29 2003 101 106
13. AM Hvas, HT Sørensen, L Norengaard, K Christiansen, J Ingerslev, B Sorensen Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A J Thromb Haemost 5 2007 2408 2414
14. PM Mannucci Hemostatic drugs N Engl J Med 339 1998 245 253
15. U Martinowitz, S Schulman, H Horoszowski, M Heim Role of fibrin sealants in surgical procedures on patients with hemostatic disorders Clin Orthop Relat Res 328 1996 65 75
16. V Mathews, A Viswabandya, S Baidya et al. Surgery for hemophilia in developing countries Semin Thromb Hemost 31 2005 538 543
17. A Srivastava Dose and response in haemophilia - optimization of factor replacement therapy Br J Haematol 127 2004 12 25
18. H Chambost, R Ljung Changing pattern of care of boys with haemophilia in western European centres Haemophilia 11 2005 92 99
19. S Donadel-Claeyssens Current co-ordinated activities of the PEDNET (European Paediatric Network for Haemophilia Management) Haemophilia 12 2006 124 127
20. S Ota, M McLimont, MD Carcao et al. Definitions for haemophilia prophylaxis and its outcomes: the Canadian consensus study Haemophilia 13 2007 12 20
21. E Berntorp, V Boulyjenkov, D Brettler et al. Modern treatment of haemophilia Bull World Health Organ 73 1995 691 701
22. E Berntorp, J Astermark, S Bjorkman et al. Consensus perspectives on prophylactic therapy for haemophilia: summary statement Haemophilia 9 suppl 1 2003 1 4
23. S Donadel-Claeyssens Current co-ordinated activities of the PEDNET (European Paediatric Network for Haemophilia Management) Haemophilia 12 2006 124 127
24. IM Nilsson, E Berntorp, T Lofqvist, H Pettersson Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B J Intern Med 232 1992 25 32
25. HM van den Berg, K Fischer, EP Mauser-Bunschoten et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia Br J Haematol 112 2001 561 565
26. M Carlsson, E Berntorp, S Bjorkman, K Lindvall Pharmacokinetic dosing in prophylactic treatment of hemophilia A Eur J Haematol 51 1993 247 252
27. BM Feldman, M Pai, GE Rivard et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study J Thromb Haemost 4 2006 1228 1236
28. K Steen Carlsson, S Hojgard, A Glomstein et al. On-demand vs prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome Haemophilia 9 2003 555 566
29. MJ Manco-Johnson, TC Abshire, AD Shapiro et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia N Engl J Med 357 2007 535 544
30. S van Creveld Prophylaxis in haemophilia Lancet 297 1971 450
31. K Fischer, J Astermark, JG van der Bom et al. Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen Haemophilia 8 2002 753 760
32. MS Gilbert Prophylaxis: musculoskeletal evaluation Semin Hematol 30 1993 3 6
33. H Pettersson, A Ahlberg, IM Nilsson A radiologic classification of hemophilic arthropathy Clin Orthop Relat Res 149 1980 153 159
34. J Astermark, P Petrini, L Tengborn, S Schulman, R Ljung, E Berntorp Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized Br J Haematol 105 1999 1109 1113
35. A Gringeri, B Lundin, S von Mackensen, L Mantovani, PM Mannucci A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study) J Thromb Haemost 9 2011 700 710
36. RC Ljung Aspects of haemophilia prophylaxis in Sweden Haemophilia 8 suppl 2 2002 34 37
37. TT Yee, K Beeton, A Griffioen et al. Experience of prophylaxis treatment in children with severe haemophilia Haemophilia 8 2002 76 82
38. ME Mancuso, L Berardinelli, C Beretta, M Raiteri, E Pozzoli, E Santagostino Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-up Haematologica 94 2009 687 692
39. LM Aledort, RH Haschmeyer, H Pettersson A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group J Intern Med 236 1994 391 399
40. K van Dijk, K Fischer, JG van der Bom, DE Grobbee, HM van den Berg Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed Haemophilia 11 2005 438 443
41. C Escuriola Ettingshausen, S Halimeh, K Kurnik et al. Symptomatic onset of severe hemophilia A in childhood is dependent on the presence of prothrombotic risk factors Thromb Haemost 85 2001 218 220
42. M Carlsson, S Bjorkman, E Berntorp Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis Haemophilia 4 1998 83 88
43. BM Feldman, S Funk, B Lundin, AS Doria, R Ljung, V Blanchette Musculoskeletal measurement tools from the International Prophylaxis Study Group (IPSG) Haemophilia 14 suppl 3 2008 162 169
44. BM Feldman, P Babyn, AS Doria et al. Proceedings of the International Haemophilia Prophylaxis Study Group Meeting, November 2003, Montreal, PQ, Canada Haemophilia 11 2005 58 63
45. K Beeton, P De Kleijn, P Hilliard et al. Recent developments in clinimetric instruments Haemophilia 12 suppl 3 2006 102 107
46. AS Doria, B Lundin, RF Kilcoyne et al. Reliability of progressive and additive MRI scoring systems for evaluation of haemophilic arthropathy in children: expert MRI Working Group of the International Prophylaxis Study Group Haemophilia 11 2005 245 253
47. B Lundin, P Babyn, AS Doria et al. Compatible scales for progressive and additive MRI assessments of haemophilic arthropathy Haemophilia 11 2005 109 115
48. AS Doria, B Lundin, S Miller et al. Reliability and construct validity of the compatible MRI scoring system for evaluation of elbows in haemophilic children Haemophilia 14 2008 303 314
49. BM Feldman, L Aledort, M Bullinger et al. The economics of haemophilia prophylaxis: governmental and insurer perspectives. Proceedings of the Second International Prophylaxis Study Group (IPSG) symposium Haemophilia 13 2007 745 749
50. AS Doria, PS Babyn, B Lundin et al. Reliability and construct validity of the compatible MRI scoring system for evaluation of haemophilic knees and ankles of haemophilic children. Expert MRI working group of the international prophylaxis study group Haemophilia 12 2006 503 513
51. MJ Manco-Johnson, H Pettersson, P Petrini et al. Physical therapy and imaging outcome measures in a haemophilia population treated with factor prophylaxis: current status and future directions Haemophilia 10 suppl 4 2004 88 93
52. K van Dijk, K Fischer, JG van der Bom, E Scheibel, J Ingerslev, HM van den Berg Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands Br J Haematol 130 2005 107 112
53. SC Darby, SW Kan, RJ Spooner et al. Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV Blood 110 2007 815 825
54. AA Sharathkumar, JM Soucie, B Trawinski, A Greist, AD Shapiro Prevalence and risk factors of cardiovascular disease (CVD) events among patients with haemophilia: experience of a single haemophilia treatment centre in the United States (US) Haemophilia 17 2011 597 604
55. W Miesbach, S Krekeler, S Alesci Treatment of haemophilia in the elderly Hamostaseologie 29 suppl 1 2009 S29 S31
56. M Franchini, PM Mannucci Co-morbidities and quality of life in elderly persons with haemophilia Br J Haematol 148 2010 522 533
57. SM Siboni, PM Mannucci, A Gringeri et al. Health status and quality of life of elderly persons with severe hemophilia born before the advent of modern replacement therapy J Thromb Haemost 7 2009 780 786
58. PM Mannucci, RE Schutgens, E Santagostino, EP Mauser-Bunschoten How I treat age-related morbidities in elderly persons with hemophilia Blood 114 2009 5256 5263
59. R Webster, E Didier, P Harris et al. PEGylated proteins: evaluation of their safety in the absence of definitive metabolism studies Drug Metab Dispos 35 2007 9 16
60. M Baru, L Carmel-Goren, Y Barenholz et al. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy Thromb Haemost 93 2005 1061 1068
61. J Spira, OP Plyushch, TA Andreeva, Y Andreev Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII reconstituted with pegylated liposomes Blood 108 2006 3668 3673
62. J Spira, OP Plyushch, TA Andreeva, RN Khametova Evaluation of liposomal dose in recombinant factor VIII reconstituted with pegylated liposomes for the treatment of patients with severe haemophilia A Thromb Haemost 100 2008 429 434
63. B Mei, C Pan, H Jiang et al. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment Blood 116 2010 270 279
64. H Rottensteiner, PL Turecek, R Pendu et al. PEGylation or polysialation reduces FVIII binding to LRP resulting in prolonged half-life in murine models Blood 11 2007 abstract 3150.
65. T Weimer, W Wormsbacher, U Kronthaler, W Lang, U Liebing, S Schulte Prolonged in-vivo half-life of factor VIIa by fusion to albumin Thromb Haemost 99 2008 659 667
66. RT Peters, SC Low, GD Kamphaus et al. Prolonged activity of factor IX as a monomeric Fc fusion protein Blood 115 2010 2057 2064
67. J Astermark Overview of inhibitors Semin Hematol 43 suppl 4 2006 S3 S7
68. J Krudysz-Amblo, B Parhami-Seren, S Butenas et al. Quantitation of anti-factor VIII antibodies in human plasma Blood 113 2009 2587 2594
69. M Franchini, G Lippi, M Montagnana et al. Anaphylaxis in patients with congenital bleeding disorders and inhibitors Blood Coagul Fibrinolysis 20 2009 225 229
70. M Chitlur, I Warrier, M Rajpurkar, JM Lusher Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006) Haemophilia 15 2009 1027 1031
71. SC Gouw, JG van der Bom, H Marijke van den Berg Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study Blood 109 2007 4648 4654
72. E Santagostino, ME Mancuso, A Rocino, G Mancuso, MG Mazzucconi, A Tagliaferri et al. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study Br J Haematol 130 2005 422 427
73. T Calvez, Y Laurian Protective effect of prophylaxis on inhibitor development in children with haemophilia A: more convincing studies are required Br J Haematol 132 2006 798 800
74. KR Viel, A Ameri, TC Abshire et al. Inhibitors of factor VIII in black patients with hemophilia N Engl J Med 360 2009 1618 1627
75. I Scharrer, GL Bray, O Neutzling Incidence of inhibitors in haemophilia A patients - a review of recent studies of recombinant and plasma-derived factor VIII concentrates Haemophilia 5 1999 145 154
76. J Goudemand, C Rothschild, V Demiguel et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A Blood 107 2006 46 51
77. SC Gouw, JG van der Bom, G Auerswald, CE Ettinghausen, U Tedgard, HM van den Berg Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study Blood 109 2007 4693 4697
78. J Oldenburg, O El-Maarri, R Schwaab Inhibitor development in correlation to factor VIII genotypes Haemophilia 8 suppl 2 2002 23 29
79. J Astermark, X Wang, J Oldenburg, E Berntorp, AK Lefvert Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A J Thromb Haemost 5 2007 263 265
80. J Astermark, J Oldenburg, A Pavlova, E Berntorp, AK Lefvert Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A Blood 107 2006 3167 3172
81. J Astermark, J Oldenburg, J Carlson et al. Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A Blood 108 2006 3739 3745
82. A Pavlova, D Delev, S Lacroix-Desmazes et al. Impact of polymorphisms of the major histocompatibility complex class II, interleukin-10, tumor necrosis factor-alpha and cytotoxic T-lymphocyte antigen-4 genes on inhibitor development in severe hemophilia A J Thromb Haemost 7 2009 2006 2015
83. P Matzinger Tolerance, danger, and the extended family Annu Rev Immunol 12 1994 991 1045
84. R Vance Cutting edge: cutting edge commentary: a Copernican revolution? Doubts about the danger theory J Immunol 165 2000 1725 1728
85. M Ling, EM Duncan, SE Rodgers, AM Street, JV Lloyd Low detection rate of antibodies to non-functional epitopes on factor VIII in patients with hemophilia A and negative for inhibitors by Bethesda assay J Thromb Haemost 1 2003 2548 2553
86. F Dazzi, T Tison, F Vianello et al. High incidence of anti-FVIII antibodies against non-coagulant epitopes in haemophilia A patients: a possible role for the half-life of transfused FVIII Br J Haematol 93 1996 688 693
87. J Astermark, C Altisent, A Batorova et al. Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report Haemophilia 16 2010 747 766
88. HH Brackmann, J Gormsen Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder Lancet 310 1977 933
89. IM Nilsson, E Berntorp, O Zettervall Induction of immune tolerance in patients with hemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII N Engl J Med 318 1988 947 950
90. EP Mauser-Bunschoten, HK Nieuwenhuis, G Roosendaal, HM van den Berg Low-dose immune tolerance induction in hemophilia A patients with inhibitors Blood 86 1995 983 988
91. J Oldenburg, R Schwaab, HH Brackmann Induction of immunetolerance in haemophilia A inhibitor patients by the 'Bonn Protocol': predictive parameter for therapy duration and outcome Vox Sang 77 suppl 1 1999 49 54
92. J Wight, S Paisley, C Knight Immune tolerance induction in patients with haemophilia A with inhibitors: a systematic review Haemophilia 9 2003 436 463
93. G Mariani, S Siragusa, BL Kroner Immune tolerance induction in hemophilia A: a review Semin Thromb Hemost 29 2003 69 76
94. M Makris Systematic review of the management of patients with haemophilia A and inhibitors Blood Coagul Fibrinolysis 15 suppl 1 2004 S25 S27
95. J Astermark, M Morado, A Rocino et al. Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors Haemophilia 12 2006 363 371
96. D Dimichele Immune tolerance therapy for factor VIII inhibitors: moving from empiricism to an evidence-based approach J Thromb Haemost 5 suppl 1 2007 143 150
97. CE Ettingshausen, W Kreuz Role of von Willebrand factor in immune tolerance induction Blood Coagul Fibrinolysis 16 suppl 1 2005 S27 S31
98. DM Dimichele, CR Hay The international immune tolerance study: a multicenter prospective randomized trial in progress J Thromb Haemost 4 2006 2271 2273
99. J Astermark, SM Donfield, DM DiMichele et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study Blood 109 2007 546 551
100. G Young, FE Shafer, P Rojas, S Seremetis Single 270 microg kg(-1)-dose rFVIIa vs. standard 90 microg kg(-1)-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison Haemophilia 14 2008 287 294
101. E Santagostino, ME Mancuso, A Rocino, G Mancuso, F Scaraggi, PM Mannucci A prospective randomized trial of high and standard dosages of recombinant factor VIIa for treatment of hemarthroses in hemophiliacs with inhibitors J Thromb Haemost 4 2006 367 371
102. K Kavakli, M Makris, B Zulfikar, E Erhardtsen, ZS Abrams, G Kenet Home treatment of haemarthroses using a single dose regimen of recombinant activated factor VII in patients with haemophilia and inhibitors. A multi-centre, randomised, double-blind, cross-over trial Thromb Haemost 95 2006 600 605
103. AD Shapiro, GS Gilchrist, WK Hoots, HA Cooper, DA Gastineau Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in haemophilia patients with inhibitors undergoing surgery Thromb Haemost 80 1998 773 778
104. I Scharrer Recombinant factor VIIa for patients with inhibitors to factor VIII or IX or factor VII deficiency Haemophilia 5 1999 253 259
105. EC Rodriguez-Merchan, J Wiedel Jd, T Walluy et al. Elective orthopedic surgery for hemophilia patients with inhibitors: new opportunities Semin Hematol 41 suppl 1 2004 109 116
106. M Quintana-Molina, F Martinez-Bahamonde, E Gonzalez-Garcia et al. Surgery in haemophilic patients with inhibitor: 20 years of experience Haemophilia 10 suppl 2 2004 30 40
107. J Ingerslev Efficacy and safety of recombinant factor VIIa in the prophylaxis of bleeding in various surgical procedures in hemophilic patients with factor VIII and factor IX inhibitors Semin Thromb Hemost 26 2000 425 432
108. GE Tjonnfjord Surgery in patients with hemophilia and inhibitors: a review of the Norwegian experience with FEIBA Semin Hematol 43 suppl 4 2006 S18 S21
109. S Rangarajan, T Yee, J Wilde Experience of four UK comprehensive care centres using FEIBA® for surgeries in patients with inhibitors Haemophilia 17 2011 28 34
110. E Rodriguez-Merchan, V Jimenez-Yuste, P Gomez-Cardero, M Alvarez-Roman, M Martin-Salces, A Rodriguez de la Rua Surgery in haemophilia patients with inhibitors, with special emphasis on orthopaedics: Madrid experience Haemophilia 16 2010 84 88
111. D Dimichele, C Negrier A retrospective postlicensure survey of FEIBA efficacy and safety Haemophilia 12 2006 352 362
112. BA Konkle, LS Ebbesen, E Erhardtsen et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors J Thromb Haemost 5 2007 1904 1913
113. WK Hoots, LS Ebbesen, BA Konkle et al. Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors Haemophilia 14 2008 466 475
114. LA Valentino The benefits of prophylactic treatment with APCC in patients with haemophilia and high-titre inhibitors: a retrospective case series Haemophilia 15 2009 733 742
115. CA Leissinger, DL Becton, NP Ewing, LA Valentino Prophylactic treatment with activated prothrombin complex concentrate (FEIBA) reduces the frequency of bleeding episodes in paediatric patients with haemophilia A and inhibitors Haemophilia 13 2007 249 255
116. V Jimenez-Yuste, EC Rodriguez-Merchan, MT Alvarez, M Quintana, M Martin-Salces, F Hernandez-Navarro Experiences in the prevention of arthropathy in haemophila patients with inhibitors Haemophilia 14 suppl 6 2008 28 35
117. MW Hilgartner, A Makipernaa, DM Dimichele Long-term FEIBA prophylaxis does not prevent progression of existing joint disease Haemophilia 9 2003 261 268
118. Ettingshausen CE, Kreuz W. Early long-term FEIBA prophylaxis in haemophilia A patients with inhibitor after failing immune tolerance induction: a prospective clinical case series. Haemophilia 16: 90-100.
119. Leissinger C, Berntorp E, Biasioli C, et al. Prophylactic dosing of anti-inhibitor coagulant complex (FEIBA) reduces bleeding frequency in hemophilia A patients with inhibitors: results of the Pro-FEIBA study. 52nd Amerian Society of Hematology annual meeting; Orlando, FL; Dec 4-7, 2010. Abstract number 720.
120. PC ter Avest, K Fischer, ME Mancuso et al. Risk stratification for inhibitor development at first treatment for severe hemophilia A: a tool for clinical practice J Thromb Haemost 6 2008 2048 2054
121. JF Healey, ET Parker, RT Barrow, TJ Langley, WR Church, P Lollar The comparative immunogenicity of human and porcine factor VIII in haemophilia A mice Thromb Haemost 102 2009 35 41
122. AB Colowick, RL Bohn, J Avorn, BM Ewenstein Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper Blood 96 2000 1698 1702
123. GF Pierce, D Lillicrap, SW Pipe, T Vandendriessche Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment J Thromb Haemost 5 2007 901 906
124. RK Paleyanda, WH Velander, TK Lee et al. Transgenic pigs produce functional human factor VIII in milk Nat Biotechnol 15 1997 971 975
125. KE Van Cott, SP Butler, CG Russell et al. Transgenic pigs as bioreactors: a comparison of gamma-carboxylation of glutamic acid in recombinant human protein C and factor IX by the mammary gland Genet Anal 15 1999 155 160
126. D Verma, B Moghimi, PA LoDuca et al. Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice Proc Natl Acad Sci USA 107 2010 7101 7106
127. RR Montgomery, Q Shi Alternative strategies for gene therapy of hemophilia Hematology Am Soc Hematol Educ Program 2010 2010 197 202
128. S Yokoyama, A Bartlett, FS Dar et al. Outcome of liver transplantation for haemophilia HPB (Oxford) 13 2011 40 45
129. A Srivastava Factor replacement therapy in haemophilia - are there models for developing countries? Haemophilia 9 2003 391 396
130. KE Van Cott, PE Monahan, TC Nichols, WH Velander Haemophilic factors produced by transgenic livestock: abundance that can enable alternative therapies worldwide Haemophilia 10 suppl 4 2004 70 76
131. K Bowman, R Sarkar, S Raut, KW Leong Gene transfer to hemophilia A mice via oral delivery of FVIII-chitosan nanoparticles J Control Release 132 2008 252 259