High rate of spontaneous inhibitor clearance during the long term observation study of a single cohort of 524 haemophilia A patients not undergoing immunotolerance

Journal of Hematology and Oncology

Volumn 6, Issue 1, 2013, Pages

  Tagariello, Giuseppe ^a   Iorio, Alfonso ^b   Matino, Davide ^c   Belvini, Donata ^a   Salviato, Roberta ^a   Sartori, Roberto ^a   Radossi, Paolo ^a  

^a Castelfranco Veneto Hospital   (Italy)

^b MCMASTER UNIVERSITY   (Canada)

^c UNIVERSITY OF PERUGIA   (Italy)

Author keywords

Hemophilia A; Inhibitors; ITI

Indexed keywords

ALLOANTIBODY; BLOOD CLOTTING FACTOR 8 INHIBITOR;

ADOLESCENT; ADULT; ARTICLE; CHILD; DISEASE SEVERITY; DRUG CLEARANCE; GENE MUTATION; HEMOPHILIA A; HUMAN; IMMUNOLOGICAL TOLERANCE; INCIDENCE; MAJOR CLINICAL STUDY; OBSERVATIONAL STUDY; PRESCHOOL CHILD; RETROSPECTIVE STUDY; SCHOOL CHILD; SYNOVECTOMY;

ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; FACTOR VIII; FEMALE; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; MALE; MIDDLE AGED; RETROSPECTIVE STUDIES; YOUNG ADULT;

EID: 84883188534     PISSN: None     EISSN: 17568722     Source Type: Journal    
DOI: 10.1186/1756-8722-6-63     Document Type: Article

References (30)

1. The natural history of factor VIII inhibitors in patients with hemophilia A. Gill FM, Prog Clin Biol Res 1984 150 19 29 6431427
2. The epidemiology of factor VIII inhibitors. Hay CR, Haemophilia 2006 12 Suppl 6 23 8 17123390
3. The epidemiology of inhibitors in haemophilia A: a systematic review. Wight J, Paisley S, Haemophilia 2003 9 4 418 35 10.1046/j.1365-2516.2003.00780.x 12828678 (Pubitemid 36874391)
4. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. Darby SC, Keeling DM, Spooner RJ, Wan Kan S, Giangrande PL, Collins PW, Hill FG, Hay CR, UK Haemophilia Centre Doctors' Organisation, J Thromb Haemost 2004 2 7 1047 54 10.1046/j.1538-7836.2004.00710.x 15219185 (Pubitemid 40186075)
5. Inhibitors in young boys with haemophilia. Lusher JM, Baillieres Best Pract Res Clin Haematol 2000 13 3 457 468 10.1053/beha.2000.0088 11030045
6. Transient inhibitors in the Recombinate PUP study. Rothschild C, Gill J, Scharrer I, Bray G, Thromb Haemost 2000 84 1 145 146 10928491 (Pubitemid 30433689)
7. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Hay CR, Dimichele DM, Blood 2012 119 6 1335 1344 10.1182/blood-2011-08-369132 22101900
8. Effects of factor VIII replacement on the level of FVIII antibodies in haemophiliacs. Rizza CR, Mattews JM, Br J Haematol 1982 52 1 13 24 10.1111/j.1365-2141.1982.tb03857.x 6810911 (Pubitemid 12028602)
9. The long-term course of factor VIII inhibitors in patients with congenital haemophilia A without immune tolerance induction. Caram C, De Souza RG, De Sousa JC, Araújo Pereira T, Do Amaral Cerqueira AM, Van Der Bom JG, Rezende SM, Thromb Haemost 2011 105 1 59 65 21057702
10. The molecular mechanisms of immunomodulation and tolerance induction to factor VIII. Waters B, Lillicrap D, J Thromb Haemost 2009 7 9 1446 1456 10.1111/j.1538-7836.2009.03538.x 19583822
11. Treatment of haemophilia A and B and von Willebrand's disease: summary and conclusions of a systematic review as part of a Swedish health-technology assessment. Berntorp E, Astermark J, Baghaei F, Bergqvist D, Holmström M, Ljungberg B, Norlund A, Palmblad J, Petrini P, Stigendal L, Säwe J, Haemophilia 2012 18 2 158 65 10.1111/j.1365-2516.2011.02723.x 22151198
12. Treatment Guidelines Working Group The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A, Haemophilia 2013 19 1 1 47 10.1111/j.1365-2516. 2012.02909 22776238
13. Economic modelling of different treatment strategies for haemophilia A with high-responding inhibitors. Knight C, Paisley J, Wight J, Jones ML, Haemophilia 2003 9 4 521 540 10.1046/j.1365-2516.2003.00783.x 12828681 (Pubitemid 36874394)
14. Definitions in Hemophilia. White GC, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J, on behalf of the Factor VIII and Factor IX Subcommittee, Thromb & Haemost 2001 85 3 560 23998557
15. A more uniform measurement of Factor VIII inhibitors. Kasper CK, Aledort LM, Counts RB, Edson JR, Fratantoni J, Green D, Hampton JW, Hilgartner MW, Lazerson J, Levine PH, McMillan CW, Pool JG, Shapiro SS, Shulman NR, Van Eys J, Thromb Diath Haemorrh 1975 34 2 869 872 1209552
16. The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype. Margaglione M, Castaman G, Morfini M, Rocino A, Santagostino E, Tagariello G, Tagliaferri AR, Zanon E, Bicocchi MP, Castaldo G, Peyvandi F, Santacroce R, Torricelli F, Grandone E, Mannucci PM, AICE-Genetics Study Group, Haematologica 2008 93 5 722 728 10.3324/haematol.12427 18387975 (Pubitemid 351669312)
17. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Ehrenforth S, Kreuz W, Scharrer I, Linde R, Funk M, Güngör T, Krackhardt B, Kornhuber B, Lancet 1992 339 8808 594 8 1347102
18. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. Iorio A, Halimeh S, Holzhauer S, Goldenberg N, Marchesini E, Marcucci M, Young G, Bidlingmaier C, Brandao LR, Ettingshausen CE, Gringeri A, Kenet G, Knöfler R, Kreuz W, Kurnik K, Manner D, Santagostino E, Mannucci PM, Nowak-Göttl U, J Thromb Haemost 2010 8 6 1256 1265 10.1111/j.1538-7836. 2010.03823.x 20345722
19. Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United Kingdom. Hay CR, Palmer B, Chalmers E, Liesner R, Maclean R, Rangarajan S, Williams M, Collins PW, on behalf of United Kingdom Haemophilia Centre Doctors' Organisation (UKHCDO), Blood 2011 117 23 6367 6370 10.1182/blood-2010-09-308668 21471523
20. Factor VIII products and inhibitor development in severe hemophilia A. Gouw SC, van der Bom JG, Ljung R, Escuriola C, Cid AR, Claeyssens-Donadel S, Van Geet C, Kenet G, Mäkipernaa A, Molinari AC, Muntean W, Kobelt R, Rivard G, Santagostino E, Thomas A, van den Berg HM, PedNet and RODIN Study Group, N Engl J Med 2013 368 3 231 239 10.1056/NEJMoa1208024 23323899
21. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Gouw SC, Van Der Bom JG, Van Den M, Berg H, Blood 2007 109 11 4648 4654 10.1182/blood-2006-11- 056291 17289808 (Pubitemid 46827754)
22. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Santagostino E, Mancuso ME, Rocino A, Mancuso G, Mazzucconi MG, Tagliaferri A, Messina M, Mannucci PM, Br J Haematol 2005 130 3 422 427 10.1111/j.1365-2141.2005.05605.x 16042693 (Pubitemid 43899592)
23. The international registry of immune tolerance: 1994 update. Ghirardini A, Puopolo M, Chiarotti F, Mariani G, Vox Sang 1996 70 Suppl 1 42 6 8869468 (Pubitemid 26055994)
24. The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Dimichele D, Haemophilia 2009 15 1 320 8 10.1111/j.1365-2516.2008.01880.x 18976249
25. Immune tolerance in hemophilia with factor VIII inhibitors: predictors of success. Mariani G, Kroner B, for the Immune Tolerance Study Group (ITSG), Haematologica 2001 86 11 1186 1193 11694405 (Pubitemid 33063865)
26. Haemophilia A: mutation type determines risk of inhibitor formation. Schwaab R, Brackmann HH, Meyer C, Seehafer J, Kirchgesser M, Haack A, Olek K, Tuddenham EGD, Oldenburg J, Thromb & Haemost 1995 74 6 1402 1406 23998557 (Pubitemid 26018063)
27. Risk factors for inhibitor development in hemophilia A. Oldenburg J, Brackmann HH, Schwaab R, Haematologica 2000 85 10 7 14 11187876
28. F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis. Gouw SC, van den Berg HM, Oldenburg J, Astermark J, De Groot PG, Margaglione M, Thompson AR, Van Heerde W, Boekhorst J, Miller CH, Le Cessie S, van der Bom JG, Blood 2012 119 12 2922 34 10.1182/blood-2011-09-379453 22282501
29. F8 gene mutation profile and ITT response in a cohort of Italian haemophilia A patients with inhibitors. Salviato R, Belvini D, Radossi P, Sartori R, Pierobon F, Zanotto D, Zanon E, Castaman G, Gandini G, Tagariello G, Haemophilia 2007 13 6 361 72 17610549 (Pubitemid 47044706)
30. Factor VIII gene (F8) mutations as predictors of outcome in immune tolerance induction of hemophilia A patients with high-responding inhibitors. Coppola A, Margaglione M, Santagostino E, Rocino A, Grandone E, Mannucci PM, Di Minno G, AICE PROFIT,Study Group, J Thromb Haemost 2009 7 11 1809 1815 10.1111/j.1538-7836.2009.03615.x 19740093