Forum on: The role of recombinant factor VIII in children with severe haemophilia A

Haemophilia

Volumn 15, Issue 2, 2009, Pages 578-586

  Franchini, M ^a   Coppola, Antonio ^b   Molinari, A C ^c   Santoro, C ^d   Schinco, P ^e   Speciale, V ^f   Tagliaferri, A ^a  

^a UNIVERSITY HOSPITAL OF PARMA   (Italy)

^b UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^c G GASLINI INSTITUTE   (Italy)

^d SAPIENZA UNIVERSITY OF ROME   (Italy)

^e MOLINETTE HOSPITAL   (Italy)

^f San Giacomo Hospital   (Italy)

Author keywords

Children; Haemophilia; Inhibitor; Prophylaxis; Recombinant FVIII; Safety

Indexed keywords

BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 CONCENTRATE; RECOMBINANT BLOOD CLOTTING FACTOR 8;

ARTHROPATHY; BLOOD CLOTTING AND BLOOD CLOTTING FACTORS; CHILD; CLINICAL TRIAL; CONFERENCE PAPER; CONTINUOUS INFUSION; DISEASE COURSE; DISEASE SEVERITY; DISEASE TRANSMISSION; DOSAGE SCHEDULE COMPARISON; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERANCE; GENE MUTATION; GENETIC POLYMORPHISM; HEALTH CARE COST; HEMARTHROSIS; HEMOPHILIA A; HUMAN; JOINT DESTRUCTION; MORBIDITY; MORTALITY; OPTIMAL DRUG DOSE; PRIORITY JOURNAL; PROPHYLAXIS; QUALITY OF LIFE; VASCULAR ACCESS DEVICE;

BLOOD COAGULATION FACTOR INHIBITORS; CHILD; DRUG ADMINISTRATION SCHEDULE; FACTOR VIII; HEMARTHROSIS; HEMOPHILIA A; HUMANS; ISOANTIBODIES; JOINT DISEASES;

EID: 63349111371     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2008.01975.x     Document Type: Conference Paper

References (57)

1. Mannucci PM. Back to the future: A recent history of haemophilia treatment. Haemophilia 2008; 14(Suppl. 3): 10-8.
2. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: A systematic review. Haemophilia 2003; 9: 418-35.
3. Berntorp E, Boulyjenkov V, Brettler D et al. Modern treatment of haemophilia. Bull World Health Organ 1995; 73: 691-701.
4. National Hemophilia Foundation. MASAC Recommendations concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Document 179. Available at: http://www.hemophilia.org/ NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007 (last accessed on 20 October 2008).
5. 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.
6. Astermark J, Petrini P, Tengborn L, Schulman S, Ljung R, Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 1999; 105: 1109-13.
7. van den Berg HM, Fischer K, Mauser-Bunschoten EP et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol 2001; 112: 561-5.
8. Berntorp E, Astermark J, Bjorkman S et al. Consensus perspectives on prophylaxis therapy for haemophilia: Summary statement. Haemophilia 2003; 9(Suppl. 1): 1-4.
9. Donadel-Claeyssens S. Current co-ordinated activities of the PEDNET (European Paediatric Network for Haemophilia Management). Haemophilia 2006; 12: 124-7.
10. Valentino LA. Secondary prophylaxis therapy: What are benefits, limitations and unknown? Haemophilia 2004; 10: 147-57.
11. Stobart K, Iorio A, Wu JK. Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B. Cochrane Database Syst Rev 2006; 2: CD003429.
12. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
13. Doria AS, Lundin B, Kilcoyne RF 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 2005; 11: 245-53.
14. Gringeri A, Lundin B, von Mackensen S et al. A 10-yr, randomized clinical trial on prophylaxis vs. on demand treatment in children with haemophilia A: The E.S.P.R.I.T. study. Haemophilia 2008; 14(Suppl. 2): 99. [abstract].
15. Fischer K, Astermark J, van der Bom JG et al. Prophylactic treatment for severe haemophilia: Comparison of an intermediate-dose to a high-dose regimen. Haemophilia 2002; 8: 753-60.
16. Petrini P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia 2007; 13(Suppl. 2): 16-22.
17. van den Berg HM, de Groot PHG, Fischer K. Phenotypic heterogeneity in severe hemophilia. J Thromb Haemost 2007; 5(Suppl. 1): 151-6.
18. Petrini P. What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B? Haemophilia 2001; 7: 99-102.
19. Feldman BN, Pai M, Rivard GE 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 2006; 4: 1228-36.
20. Geraghty S, Dunkley T, Harrington C et al. Practice patterns in haemophilia A therapy - global progress towards optimal care. Haemophilia 2006; 12: 75-81.
21. Fischer K, van der Bom JG, Prejs R et al. Discontinuation of prophylactic therapy in severe haemophilia: Incidence and effects on outcome. Haemophilia 2001; 7: 544-50.
22. van Dijk K, Fischer K, van der Bom JG, Scheibel E, Ingerslev J, van den Berg HM. Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands. Br J Haematol 2005; 130: 107-12.
23. Aledort L, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 1994; 236: 391-9.
24. 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.
25. Lee CA, Lillicrap D, Astermark J. Inhibitor development in hemophiliacs: the roles of genetic versus environmental factors. Semin Thromb Hemost 2006; 32 (Suppl. 2): 10-4.
26. Schwaab R, Brackmann HH, Meyer C et al. Haemophilia A: Mutation type determines the risk of inhibitor formation. Thromb Haemost 1995; 74: 1402-6.
27. Astermark J, Oldenburg J, Pavlova A, Berntorp E, Lefvert AK; MIBS Study Group. Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. Blood 2006; 107: 3167-72.
28. Astermark J, Oldenburg J, Carlson J et al. Polymorphisms in the TNFA gene and risk of inhibitor development in patients with hemophilia A. Blood 2006; 108: 3739-45.
29. Astermark J, Wang X, Oldenburg J, Berntorp E, Lefvert AK; MIBS Study Group. Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A. J Thromb Haemost 2007; 5: 263-5.
30. Astermark J, Lacroix-Desmazes S, Reding MT. Inhibitor development. Haemophilia 2008; 14(Suppl. 3): 36-42.
31. Santagostino E, Mancuso ME, Rocino A et al. Environmental risk factors for inhibitor development in children with haemophilia A: A case-control study. Br J Haematol 2005; 130: 422-7.
32. Kreuz W, Gill JC, Rothschild C et al. International Kogenate-FS Study Group. 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.
33. Gouw SC, van der Bom JG, van den Berg HM. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: The CANAL cohort study. Blood 2007; 109: 4648-54.
34. Gouw SC, van den Berg HM, le Cessie S, van der Bom JG. Treatment characteristics and the risk of inhibitor development: A multi-center cohort study among previously untreated patients with severe hemophilia A. J Thromb Haemost 2007; 5: 1383-90.
35. Gouw SC, van der Bom JG, Auerswald G, Ettinghausen CE, Tedgård U, van den Berg HM. 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 2007; 109: 4693-7.
36. Goudemand J, Rothschild C, Demiguel V et al.; FVIII-LFB and Recombinant FVIII study groups. 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.
37. Chalmers EA, Brown SA, Keeling D et al.; Paediatric Working Party of UKHCDO. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007; 13: 149-55.
38. Peerlinck K, Hermans C. Epidemiology of inhibitor formation with recombinant factor VIII replacement therapy. Haemophilia 2006; 12: 579-90.
39. DiMichele D, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: Consensus recommendations. Haemophilia 2007; 13 (Suppl. 1): 1-22.
40. Mariani G, Ghirardini A, Bellocco R. Immune tolerance in hemophilia - principal results from the International Registry. Thromb Haemost 1994; 72: 155-8.
41. DiMichele D, Kroner BL. The North American Immune Tolerance Registry: practices, outcomes, outcome predictors. Thromb Haemost 2002; 87: 52-7.
42. Lenk H. The German Registry of immune tolerance treatment in hemophilia-1999 update. Haematologica 2000; 10 (Suppl): 45-7.
43. Kroner BL. Comparison of the international immune tolerance registry and the North American immune tolerance registry. Vox Sang 1999; 77 (Suppl. 1): 33-7.
44. Rocino A, Santagostino E, Mancuso ME, Mannucci PM. Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors. Haematologica 2006; 91: 558-61.
45. Barnes C, Rivard GE, Poon M-C et al. Canadian multi-institutional survey of immune tolerance therapy (ITT) - experience with the use of recombinant factor VIII for ITT. Haemophilia 2006; 12: 1-6.
46. DiMichele DM, Hay CR. The international immune tolerance study: A multicenter prospective randomized trial in progress. J Thromb Haemost 2006; 4: 2271-3.
47. Gringeri A. VWF/FVIII concentrates in high-risk immunotolerance: The RESIST study. Haemophilia 2007; 13(Suppl. 5): 73-7.
48. Komvilaisak P, Connolly B, Naqvi A, Blanchette V. Overview of the use of implantable venous access devices in the management of children with inherited bleeding disorders. Haemophilia 2006; 12 (Suppl. 6): 87-93.
49. Santagostino E, Gringeri A, Berardinelli L, Beretta C, Muça-Perja M, Mannucci PM. Long-term safety and feasibility of arteriovenous fistulae as vascular accesses in children with haemophilia: A prospective study. Br J Haematol 2003; 123: 502-6.
50. McCarthy WJ, Valentino LA, Bonilla AS et al. Arteriovenous fistola for long-term venous access for boys with hemophilia. J Vasc Surg 2007; 45: 986-90.
51. Llewelyn CA, Hewitt PE, Knight RS et al. Possible transmission of variant Creutzfeldt-Jakob disease by blood transfusion. Lancet 2004; 363: 417-21.
52. Santagostino E, Mannucci PM, for the Italian Association of Haemophilia Centres (AICE). Guidelines on replacement therapy for haemophilia and inherited coagulation disorders in Italy. Haemophilia 2000; 6: 1-10.
53. Keeling D, Tait C, Makris M. Guideline on the selection and use of therapeutic products to treat haemophilia and other hereditary bleeding disorders. Haemophilia 2008; 14: 671-84.
54. Pipe SW. Recombinant clotting factors. Thromb Haemost 2008; 99: 840-50.
55. Musso R, Santagostino E, Faradji A et al.; Kogenate Bayer European PMS Study Group. Safety and efficacy of sucrose-formulated full-length recombinant factor VIII: Experience in the standard clinical setting. Thromb Haemost 2008; 99: 52-8.
56. Delumeau JC, Ikegawa C, Yokoyama C, Haupt V. An observational study of sucrose-formulated recombinant factor VIII for Japanese patients with haemophilia A. Thromb Haemost 2008; 100: 32-7.
57. Rubinger M, Lillicrap D, Rivard GE et.al. A prospective surveillance study of factor VIII inhibitor development in the Canadian haemophilia A population following the switch to a recombinant factor VIII product formulated with sucrose. Haemophilia 2008; 14: 281-6.