This page contains a Flash digital edition of a book.
Keeling_subbed 12/2/09 15:19 Page 33
Immunological Aspects of Inhibitor Development in Haemophilia
Haemost, 2003;1:456–63. IX concentrates, Thrombosis Research, 1996;83:279–86. haemophilia A, Thromb Haemost, 2008;100:429–34.
23. Fijnvandraat K, Turenhout EAM, van den Brink EN, et al. The 45. Gill JC, The role of genetics in inhibitor formation, Thromb 69. Martinowitz U, Lalezari S, Luboshitz, et al., Infusion rates of
missense mutation Arg593 ‡ Cys is related to antibody Haemost, 1999;82:500–504. recombinant FVIII-FS withi PEGylated liposomes in haemophilia
formation in a patient with mild haemophilia A, Blood, 46. Astermark J, Berntorp E, White GC, et al.; MIBS Study Group, A, Haemophilia, 2008;14:1122–4.
1997;89:4371–7. The Malmö International Brother Study (MIBS): further support 70. Powell JS, Nugent DJ, Harrison JA, et al., Safety and
24. Jacquemin M, Benhida A, Peerlinck K, et al., A human antibody for genetic predisposition to inhibitor development in pharmacokinetics of a recombinant factor VIII with pegylated
directed to the factor VIII C1 domain inhibits factor VIII cofactor hemophilia patients, Haemophilia, 2001;7(3):267–72. liposomes in severe hemophilia A, J Thromb Haemost,
activity and binding to von Willebrand factor, Blood, 2000;95: 47. Aledort LM, Dimichele DM, Inhibitors occur more frequently in 2008;6:277–83.
156–63. African-American and Latino haemophiliacs, Haemophilia, 71. Morado M, Villar A, Jiménez Yuste V, et al., Prophylactic
25. Suzuki H, Shima M, Arai M et al. Factor VIII Ise (R2159C) in a 1998;4:68. treatment effects on inhibitor risk: experience in one centre,
patient with mild haemophilia A, an abnormal factor VIII with 48. Oldenburg J, Schröder J, Brackmann HH, et al., Environmental Haemophilia, 2005;11:79–83.
retention of function but modification of C2 epitopes, Thromb and genetic factors influencing inhibitor development, Semin 72. Matzinger P, The danger model: a renewed sense of self,
Haemost, 1997;77:862–7. Hematol, 2004;41(Suppl. 1):82–8. Science, 2002;296:301–5.
26. d’Oiron R, Lavergne JM, Lavend’homme R et al., Deletion of 49. Gouw SC, van der Bom JG, Auerswald Gunter, et al., 73. Sharathkumar A, Lillicrap D, Blanchette VS, et al., Intensive
alanine 2201 in the FVIII C2 domain results in mild haemophilia Recombinant versus plasma-derived factor VIII products and the exposure to factor VIII is a risk factor for inhibitor development
A by impairing FVIII binding to vWF and phospholipids and development of inhibitors in previously untreated patients with in mild hemophilia A, J Thromb Haemost, 2003;1(6):1228–36.
destroys a major FVIII antigenic determinant involved in severe hemophilia A: the CANAL cohort study, Blood, 74. Bray GL, Kroner BL, Arkin S, et al., Loss of high-responder
inhibitor development, Blood, 2004;103:155–7. 2007;109:4693–7. inhibitors in patients with severe hemophilia A and human
27. Peerlinck K, Jacquemin MG, Arnout J, et al. Antifactor VIII 50. Santagostino E, Mancuso ME, Rocino A et al., Environmental risk immunodeficiency virus type 1 infection: a report from the
antibody inhibiting allogeneic but not autologous factor VIII in factors for inhibitor development in children with haemophilia A: Multi-Center Hemophilia Cohort Study, Am J Hematol,
patients with mild hemophilia A, Blood, 1999;93:2267–73. a case-control study, Br J Haematol, 2005;130:422–7. 1993;42:375–9.
28. Jacquemin M, Vantomme V, Buhot C, et al., CD4+ T-cell clones 51. Chalmers EA, Brown SA, Keeling D, et al., Early factor VIII 75. Werwitzke S, Tiede A, Stoll M, et al., Immune reconstitution
specific for wild-type factor VIII: a molecular mechanism exposure and subsequent inhibitor development in children inflammatory syndrome (IRIS) as a cause for inhibitor
responsible for a higher incidence of inhibitor formation in with severe haemophilia A, Haemophilia, 2007;13:149–55. development in hemophilia, J Thromb Haemost, 2004;2:193–4.
mild/moderate haemophilia A, Blood, 2003;101:1351–8. 52. Gouw SC, van der Bom JG, van den Berg M for the CANAL 76. Hay CRM, Lee CA, Inhibitors in mild and moderate haemophilia
29. Bril WS, MacLean PE, Kaijen PH, et al., HLA class II genotype Study Group, Treatment-related risk factors of inhibitor A. In: Rodriguez-Merchan EC, Lee CA (eds), Inhibitors in
and factor VIII inhibitors in mild haemophilia A patients with an development in previously untreated patients with hemophilia Patients with Haemophilia, Oxford, UK: Blackwell Publishing,
Arg593 to Cys mutation, Haemophilia, 2004;10(5):509–14. A: the CANAL cohort study, Blood, 2007;109:4648–54. 2002;92–7.
30. Hay CRM, Ollier W, Pepper L et al. HLA class II profile: a weak 53. Green P, A Database of Point Mutations and Short Additions 77. Peerlinck K, Jacquemin M, Characteristics of inhibitors in
determinant of factor VIII inhibitor development in severe and Deletions in the Factor IX Gene. Haemophilia B Mutation mild/moderate haemophilia A, Haemophilia, 2006;12(Suppl. 6):
haemophilia A, Thromb Haemost, 1997;77:234–7. Database, King’s College London, University of London, 43–7.
31. Oldenburg J, Picard JK, Schwaab R, et al., HLA genotype of London, Version 13, 2004. Available at: www.kcl.ac.uk/ip/ 78. Hay CR, The epidemiology of factor VIII inhibitors, Haemophilia,
patients with severe haemophilia A due to intron 22 inversion petergreen/haemBdatabase.html (accessed 12 January 2009. 2006;12(Suppl 6):23–9.
with and without inhibitors of factor VIII, Thromb Haemost, 54. Christophe OD, Lenting PJ, Cherel G, et al., Functional mapping 79. Von Auer CH, Oldenburg J, von Depka M, et al., Inhibitor
1997;77:238–42. of anti-factor IX inhibitors developed in patients with severe development in patients with hemophilia A after continuous
32. White GC 2nd, Kempton CL, Grimsley A, et al., Cellular immune haemophilia B, Blood, 2001;98:1416–1423. infusion of FVIII concentrates, Ann NY Acad Sci, 2005;1051:
responses in hemophilia: why do inhibitors develop in some, 55. Takahashi I, Mizumo S, Kamiya T, et al., Epitope mapping of 498–505.
but not all hemophiliacs?, J Thromb Haemost, 2005;3:1676–81. human factor IX inhibitor antibodies, Br J Haematol, 1994;88: 80. White B, Cotter M, Byrne M, et al., High responding factor VIII
33. Astermark J, Oldenburg J, Pavlova A, et al.; the MIBS Study 166–73. inhibitors in mild haemophilia: is there a link with recent
Group, Polymorphisms in the IL10 but not the IL-1b and IL-4 56. Schellekens H, Factors influencing the immunogenicity of changes in clinical practice?, Haemophilia, 2000;6:113–15.
genes are associated with inhibitor development in patients therapeutic proteins, Nephrol Dial Transplant, 2005;20 81. Bidlingmaier C, Deml MM, Kurnik K, Continuous infusion of
with haemophilia A, Blood, 2006;107:3167–72. (Suppl. 6):vi3–9. factor concentrates in children with haemophilia A in
34. Wieland I, Wermes C, Eifrig B, et al., Different HLA-types seem 57. Hermeling S, Crommelin DJ, Schellekens H, et al., comparison with bolus injections, Haemophilia, 2006;12:
to be involved in the inhibitor development in haemophilia A, Structure–immunogenicity relationships of therapeutic proteins, 212–17.
Hämostaseologie, 2008;28(Suppl. 1):S26–28. Pharm Res, 2004;21:897–903. 82. Ljung R, Central venous catheters in children with haemophilia,
35. Wilson AG, di Giovine FS, Blakemore AI, et al., Single base 58. Purohit VS, Middaugh CR, Balasubramanian SV, Influence of Blood Reviews, 2004;18:93–100.
polymorphism in the human tumour necrosis factor alpha (TNF aggregation on immunogenicity of recombinant human factor 83. Blanchette VS, Al Musa A, Stain AM, et al., Central venous
alpha) gene detectable by NcoI restriction of PCR product, VIII in hemophilia A mice, J Pharm Sci, 2006;95:358–71. access catheters in children with haemophilia, Blood Coagul
Hum Mol Genet, 1992;1:353. 59. Purohit VS, Ramani K, Sarkar R, et al., Lower inhibitor Fibrinol, 1996;7(Suppl. 1):S39–44.
36. Bouma G, Crusius JB, Oudkerk Pool M, et al., Secretion of development in hemophilia A mice following administration of 84. Perkins JL, Johnson VA, Osip JM, et al., The use of implantable
tumour necrosis factor alpha and lymphotoxin alpha in relation recombinant factor VIII–O-phospho-l-serine complex, J Biol venous access devices (IVADs) in children with hemophilia,
to polymorphisms in the TNF genes and HLA-DR alleles. Chem, 2005;280:17593–600. J Pediatr Hematol Oncol, 1997;19:339–44.
Relevance for inflammatory bowel disease, Scand J Immunol, 60. Pratt KP, Qian J, Ellaban E, et al., Immunodominant T-cell 85. Ljung R, van den Berg M, Petrini P, et al., Port-A-Cath usage in
1996;43:456–63. epitopes in the factor VIII C2 domain are located within an children with haemophilia: experience of 53 cases, Acta
37. Huang DR, Pirskanen R, Matell G, et al., Tumour necrosis inhibitory antibody binding site, Thromb Haemost, 2004;92: Paediatr, 1998;87:1051–4.
factor-alpha polymorphism and secretion in myasthenia gravis, 522–8. 86. Miller K, Buchanan GR, Zappa S, et al., Implantable venous
J Neuroimmunol, 1999;94:165–71. 61. Reding MT, Okita DK, Diethelm-Okita BM, et al., Human CD4 + access devices in children with hemophilia: a report of low
38. Oldenburg J, Pavlova A, Genetic risk factors for inhibitors to T-cell epitope repertoire on the C2 domain of coagulation factor infection rates, J Pediatr, 1998;132:934–8.
factors VIII and IX, Haemophilia, 2006;12(Suppl. 6):15–22. VIII, J Thromb Haemost, 2003;1:1777–84. 87. McMahon C, Smith J, Khair K, et al., Central venous access
39. Astermark J, Oldenburg J, Carlson J, et al., Polymorphisms in 62. Purohit VS, Balasubramanian SV, Interaction of dicaproyl devices in children with congenital coagulation
the TNFA gene and the risk of inhibitor development in patients phosphatidylserine with recombinant factor VIII and its impact disorders;complications and long-term outcome, Brit J
with hemophilia A, Blood, 2006;108:3739–45. on immunogenicity, AAPS J, 2006;8:E362–70. Haematol, 2000;110:461–8.
40. Llorente L, Zou W, Levy Y, et al. Role of interleukin 10 in the B 63. Goudemand J, Inhibitor development in haemophilia A: the role 88. Van den Berg HM, Fischer K, Roosendaal G, et al., The use of
lymphocyte hyperactivity and autoantibody production of of von Willebrand factor/factor VIII concentrates, Haemophilia, the Port-A-Cath in children with haemophilia – a review,
human systemic lupus erythematosus, J Exp Med, 1995;181: 2007;13(Suppl. 5):47–51. Haemophilia, 1998;4:418–20.
839–44. 64. Wight J, Paisley S, The epidemiology of inhibitors in 89. Lim SH, Smith MP, Machin SJ, et al., A prospective randomised
41. Huang D, Zhou Y, Xia SQ, et al., Markers in the promoter haemophilia A: a systematic review, Haemophilia, study of prophylactic teicoplanin to prevent early Hickman
region of interleukin-10 (IL-10) gene in myasthenia gravis: 2003;9:418–35. catheter-related sepsis in patients receiving intensive
implications on diverse effects of IL-10 in the pathogenesis of 65. Escurioloa Ettingshausen C, Kreuz W, Recombinant vs. Plasma- chemotherapy for hematological malignancies, Eur J Haematol
the disease, J Neuroimmunol, 1999;94:82–7. derived products, especially those with intact VWF, regarding Suppl, 1993;54:10–13.
42. Astermark J, Wang X, Oldenburg J, et al., MIBS Study Group, inhibitor development, Haemophilia, 2006;12:102–6. 90. Ranson MR, Oppenheim BA, Jackson A, et al., Double-blind
Polymorphisms in the CTLA-4 gene and inhibitor development 66. Veronese FM, Pasut G, PEGylation, successful approach to drug placebo controlled study of vancomycin prophylaxis for central
in patients with severe hemophilia A, J Thromb Haemost, delivery, Drug Discov Today, 2005;10(21):1451–8. venous catheter insertion in cancer patients, J Hosp Infect,
2007;5(2):263–5. 67. Spira J, Plyushch OP, Andreeva TA, et al., Prolonged bleeding- 1990;15:95–102.
43. Warrier I, Inhibitors in haemophilia B, 2005. In: Lee C, Berntorp free period following prophylactic infusion of recombinant 91. Ljung R, Chambost H, Stain AM, et al., Haemophilia in the first
E, Hoots KW (EDS), Textbook of Hemophilia, Oxford: Blackwell factor VIII reconstituted with pegylated liposomes, Blood, years of life, Haemophilia, 2008;14(Suppl. 3):188–95.
Publishers, 2005;97–100. 2006;108:3668–73. 92. ter Avest PC, Fischer K, Mancuso ME, et al., for the CANAL
44. Sawamoto Y, Shima M, Yamamoto M, et al., Measurement of 68. Spira J, Plyushch OP, Andreeva TA, et al., Evaluation of study group, Risk stratification for inhibitor development at first
anti-factor IX IGG subclasses in haemophilia B patients who liposomal dose in recombinant factor VIII reconstituted with treatment for severe hemophilia A: a tool for clinical practice,
developed inhibitors with episodes of allergic reactions to factor pegylated liposomes for the treatment of patients with severe J Thromb Haemost, 2008;6(12):2048–54.
EUROPEAN HAEMATOLOGY 33
Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64  |  Page 65  |  Page 66  |  Page 67  |  Page 68  |  Page 69  |  Page 70  |  Page 71  |  Page 72  |  Page 73  |  Page 74  |  Page 75  |  Page 76  |  Page 77  |  Page 78  |  Page 79  |  Page 80  |  Page 81  |  Page 82  |  Page 83  |  Page 84  |  Page 85  |  Page 86  |  Page 87  |  Page 88  |  Page 89  |  Page 90  |  Page 91  |  Page 92  |  Page 93  |  Page 94  |  Page 95  |  Page 96  |  Page 97  |  Page 98  |  Page 99  |  Page 100
Produced with Yudu - www.yudu.com