Special lectures in haemophilia management

Haemophilia

Volumn 16, Issue SUPPL. 5, 2010, Pages 22-28

  Batorova, A ^a   High, K A ^b   Gringeri, A ^c  

^a UNIVERSITY HOSPITAL BRATISLAVA   (Slovakia)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c UNIVERSITY OF MILAN   (Italy)

Author keywords

Gene therapy; Haemophilia; Inhibitors; Risk factors; SIPPET

Indexed keywords

BAY 794980; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; LIPOSOME; MACROGOL; PARVOVIRUS VECTOR; POLYSIALIC ACID; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; UNCLASSIFIED DRUG;

CLINICAL TRIAL; DRUG EFFICACY; DRUG FORMULATION; DRUG SAFETY; GENE THERAPY; GENE TRANSFER; HEMOPHILIA; HUMAN; IMMUNOGENICITY; IMMUNOLOGICAL TOLERANCE; MEDICAL EDUCATION; NONHUMAN; PATIENT CARE; PREVALENCE; PRIORITY JOURNAL; RECURRENT DISEASE; REVIEW; TRANSGENE;

BLOOD COAGULATION FACTOR INHIBITORS; BLOOD COAGULATION FACTORS; GENE THERAPY; HEMOPHILIA A; HEMORRHAGE; HUMANS; RECOMBINANT PROTEINS; RISK FACTORS;

EID: 77955028466     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2010.02289.x     Document Type: Review

References (63)

1. Mannucci PM. Back to the future: a recent history of haemophilia treatment. Haemophilia 2008, 14(Suppl 3):10-8.
2. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992, 232:25-32.
3. Fischer K, Astermark J, van der Bom JG. Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen. Haemophilia 2002, 8:753-60.
4. Manco-Johnson MJ, Abshire TC, Shapiro AD. Prophylaxis vs. episodic treatment to prevent joint disease in boys with severe haemophilia. N J Engl Med 2007, 357:535-44.
5. Santagostino E, Manusco ME, Rocino A. Enviromental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol 2005, 130:422-7.
6. Kurnik K, Bidlingmaier C, Engl W. New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development. Haemophilia 2010, 16:256-62.
7. Richards M, Altisent C, Batorova A. Should prophylaxis be used in adolescent and adult patients with severe hemophilia? An European survey of practice and outcome data. Haemophilia 2007, 13:473-9.
8. Valentino LA. Controversies regarding the prophylactic management of adults with severe haemophilia A. Haemophilia 2009, 15(Suppl. 2):5-22.
9. Astermark J, Morado M, Rocino A. Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors. Haemophilia 2006, 12:363-71.
10. Konkle BA, Ebbesen LS, Erdhardtsen E. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in haemophilia patients with inhibitors. J Thromb Haemost 2007, 5:1904-13.
11. Valentino LA. Accessing the benefits of FEIBA prophylaxis in haemophilia patients with inhibitors. Haemophilia 2010, 2:263-71.
12. Powel JS. Recombinant factor VIII in the management of haemophilia: current and future promise. Ther Clin Risk Manag 2009, 5:391-402.
13. DiMinno G, Cerbone AM, Coppola A. Longer-acting factor VIII to overcome limitations in haemophilia management: the PEGylated liposomes formulation issue. Haemophilia 2010, 16(Suppl. 1):2-6.
14. Santagostino E, Manusco ME. Venous access in haemophilic children: choice and management. Haemophilia 2010, 16(Suppl.1):20-4.
15. Saenko EL, Pipe SW. Strategies towards a longer acting factor VIII. Haemophilia 2006, 12:42-51. 3
16. Spira J, Plyusch OP, Andreeva TA, Andreev Y. Prolonged bleeding free period following prophylactic infusion of recombinant factor VIII reconstituted with pegylated liposomes. Blood 2006, 108:3668-73.
17. Powel JS, Nugent DJ, Harrison JA. Safety and pharmacokinetics of a recombinant factor VIII with pegylated liposomes in severe haemophilia A. J Thromb Haemost 2008, 6:277-83. 2
18. Sen P, Ghosh S, Ezban M. Effect of glycoPEGylation on factor VIIa binding and internationalization. Haemophilia 2010, 16:339-48.
19. Pipe SW, High KA, Ohashi K. Progress in the molecular biology of inherited bleeding disorders. Haemophilia 2008, 14(Suppl. 3):130-7.
20. Margaritis P, Roy E, Aljamali MN. Succesful treatment of canine haemophilia by continuous expression of canine FVIIa. Blood 2009, 113:3682-9.
21. Astermark J. Inhibitor development: patient-determined risk factors. Haemophilia 2009, 5:263-5.
22. Saint-Remy JM, Lacroix-Desmazes S, Oldenburg J. Inhibitors in haemophilia: patophysiology. Haemophilia 2004, 10(Suppl. 4):146-51.
23. Chambost H. Assessing risk factors: prevention of inhibitors in haemophilia. Haemophilia 2010, 16(Suppl 2):10-5.
24. Calvez T, Laurian Y, Goudemand J. Inhibitor incidence with recombinant vs plasma derived FVIII in previously untreated patients with severe hemophilia A: homogenous results from four published observational studies. J Thromb Haemost 2008, 6:390-2.
25. Gouw SC, van der Bom JG, Auerswald G. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe haemophilia A: the CANAL cohort study. Blood 2007, 109:4693-7.
26. Makris M. European Haemophilia Safety Surveillance projects (EUHASS): the first 12 months [Abstract]. Haemophilia 2001, 16:386.
27. Berntorp E, Shapiro A, Astermark J. Inhibitor treatment in haemophilias A and B: summary statement for the 2006 international consensus conference. Haemophilia 2006, 12(Suppl. 6):1-7.
28. Ragni MV, Ojeifo O, Feng J. Risk factors for inhibitor formation in haemophilia: a prevalent case-control study. Haemophilia 2009, 15:1074-82.
29. Franchini M, Lippi G. Recent improvements in the clinical treatment of coagulation factor inhibitors. Semin Thromb Hemost 2009, 35:806-13.
30. Mauser AE, Whitlark J, Whitney KM, Lothrop CD. A deletion mutation causes hemophilia B in Lhasa Apso dogs. Blood 1996, 88:3451-5.
31. Mount JD, Herzog RW, Tillson DM. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 2002, 99:2670-6.
32. Herzog RW, Mount JD, Arruda VR, High KA, Lothrop CD. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Mol Ther 2001, 4:192-200.
33. Brown BD, Sitia G, Annoni A. In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. Blood 2007, 109:2797-805.
34. Brown BD, Cantore A, Annoni A. A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 2007, 110:4144-52.
35. Follenzi A, Battaglia M, Lombardo A, Annoni A, Roncarolo MG, Naldini L. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood 2004, 103:3700-9.
36. Veron P, Allo V, Riviere C, Bernard J, Douar AM, Masurier C. Major subsets of human dendritic cells are efficiently transduced by self-complementary adeno-associated virus vectors 1 and 2. J Virol 2007, 81:5385-94.
37. Mingozzi F, Liu YL, Dobrzynski E. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest 2003, 111:1347-56.
38. Cao O, Dobrzynski E, Wang L. Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood 2007, 110:1132-40.
39. Matsui H, Shibata M, Brown B. A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells. Blood 2009, 114:677-85.
40. Manno CS, Pierce GF, Arruda VR. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006, 12:342-7.
41. Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E. Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood 2007, 110:2334-41.
42. Peng B, Ye P, Blazar BR. Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice. Blood 2008, 112:1662-72.
43. Peng B, Ye P, Rawlings DJ, Ochs HD, Miao CH. Anti-CD3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy. Blood 2009, 114:4373-82.
44. Faria AM, Weiner HL. Oral tolerance. Immunol Rev 2005, 206:232-59.
45. Herzog RW, Verma D, Moghimi B, Singh HD, LoDuca PA, Daniell H. Oral delivery of bioencapsulated factor IX protects from inhibitor formation and anaphylaxis in protein replacement therapy for hemophilia B. Blood 2009, 114:97. (Abstract #222)
46. Xu L, Gao C, Sands MS. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood 2003, 101:3924-32.
47. Xu L, Mei M, Haskins ME. Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice. Thromb Res 2007, 120:269-80.
48. Finn JD, Sabatino DE, Ozelo MC. Induction of immune tolerance to canine FVIII in hemophilia A dogs with inhibitors using AAV-mediated expression of canine FVIII. Blood 2008, 112:96. (abstract 243)
49. Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003, 102:2358-63.
50. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003, 9:418-35.
51. Schimpf K, Schwarz P, Kunschak M. Zero incidence of inhibitors in previously untreated patients who received intermediate purity factor VIII concentrate or factor IX complex. Thromb Haemost 1995, 73:553-5.
52. Guerois C, Laurian Y, Rothschild C. Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of highly purified plasma-derived concentrate. Thromb Haemost 1995, 73:215-8.
53. Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with haemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N Engl J Med 1993, 328:453-9.
54. Rothschild C, Laurian Y, Satre EP. French previously untreated patients with severe haemophilia A after exposure to recombinant factor VIII: incidence of inhibitor and evaluation of immune tolerance. Thromb Haemost 1998, 80:779-83.
55. Kreuz W, Gill JC, Rothschild C, Manco-Johnson MJ. Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A: results of an international clinical investigation. Thromb Haemost 2005, 93:457-67.
56. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously untreated patients. Semin Hematol 2001, 38(2 Suppl 4):52-9.
57. Goudemand J, Rothschild C, Demiguel V. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 2006, 107:46-51.
58. Chalmers EA, Brown SA, Keeling D. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007, 13:149-55.
59. Strauss T, Ravid B, Martinowitz U, Bashari D, Kenet G. Early exposure to recombinant factor concentrates may increase inhibitor development: a single center cohort study. Haemophilia 2008, 14(Suppl. 2):53. (abstract #61)
60. Scharrer I, Ehrlich HJ. Reported inhibitor incidence in FVIII PUP studies: comparing apples with oranges? Haemophilia 2004, 10:197-8.
61. Amano K, Arai M, Koshihara K, Suzuki T. Autoantibody to factor VIII that has less reactivity to factor VIII/von Willebrand factor complex. Am J Hematol 1995, 49:310-7.
62. Auerswald G, Spranger T, Brackmann HH. The role of plasma-derived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A patients. Haematologica 2003, 88:EREP05.
63. Gringeri A, Monzini M, Tagariello G, Scaraggi FA, Mannucci PM. Occurrence of inhibitors in previously untreated or minimally treated patients with haemophilia A after exposure to a plasma-derived solvent-detergent factor VIII concentrate. Haemophilia 2006, 12:128-32.