US Guidelines for immune tolerance induction in patients with haemophilia a and inhibitors

Haemophilia

Volumn 21, Issue 5, 2015, Pages 559-567

  Valentino, L A ^a   Kempton, C L ^b,e   Kruse Jarres, R ^c   Mathew, P ^d   Meeks, S L ^e   Reiss, U M ^f  

^a RUSH UNIVERSITY MEDICAL CENTER   (United States)

^b EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c PUGET SOUND BLOOD CENTER   (United States)

^d UNIVERSITY OF NEW MEXICO   (United States)

^e CHILDREN'S HEALTHCARE OF ATLANTA   (United States)

^f ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

Guidelines; Haemophilia A; Immune tolerance induction; Inhibitors

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 INHIBITOR;

BLEEDING; ETHNICITY; EVIDENCE BASED PRACTICE; HEMOPHILIA A; HUMAN; IMMUNE TOLERANCE INDUCTION; IMMUNITY; IMMUNOMODULATION; MONITORING; PRACTICE GUIDELINE; PREDICTION; PRIORITY JOURNAL; REVIEW; TREATMENT DURATION; COHORT ANALYSIS; HEALTH CARE PLANNING; IMMUNOLOGICAL TOLERANCE; IMMUNOLOGY; REGISTER; UNITED STATES;

COHORT STUDIES; FACTOR VIII; HEALTH PLANNING GUIDELINES; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; PRACTICE GUIDELINES AS TOPIC; REGISTRIES; UNITED STATES;

EID: 84942190975     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12730     Document Type: Review

References (66)

1. Valentino LA, Pipe SW, Tarantino MD, Ye X, Xiong Y, Luo MP. Healthcare resource utilization among haemophilia A patients in the United States. Haemophilia 2012; 18: 332-8.
2. Hay CR, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335-44.
3. Guyatt G, Gutterman D, Baumann MH et al. Grading strength of recommendations and quality of evidence in clinical guidelines: report from an american college of chest physicians task force. Chest 2006; 129: 174-81.
4. Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet 1977; 2: 933.
5. Brackmann HH, Oldenburg J, Schwaab R. Immune tolerance for the treatment of factor VIII inhibitors-twenty years' 'Bonn protocol'. Vox Sang 1996; 70(SUPPL. 1): 30-5.
6. Freiburghaus C, Berntorp E, Ekman M, Gunnarsson M, Kjellberg B, Nilsson IM. Tolerance induction using the Malmo treatment model 1982-1995. Haemophilia 1999; 5: 32-9.
7. Gruppo RA, Valdez LP, Stout RD. Induction of immune tolerance in patients with hemophilia A and inhibitors. Am J Pediatric Hematol/Oncol 1992; 14: 82-7.
8. Rocino A, Papa ML, Salerno E, Capasso F, Miraglia E, de Biasi R. Immune tolerance induction in haemophilia A patients with high-responding inhibitors to factor VIII: experience at a single institution. Haemophilia 2001; 7: 33-8.
9. Smith MP, Spence KJ, Waters EL et al. Immune tolerance therapy for haemophilia A patients with acquired factor VIII alloantibodies: comprehensive analysis of experience at a single institution. Thromb Haemost 1999; 81: 35-8.
10. Mauser-Bunschoten EP, Roosendaal G, van den Berg HM. Low-dose immune tolerance therapy: the van creveld model. Vox Sang 1996; 70(SUPPL. 1): 66-7.
11. DiMichele DM. Immune tolerance: critical issues of factor dose, purity and treatment complications. Haemophilia 2006; 12(SUPPL. 6): 81-6.
12. Mariani G, Ghirardini A, Bellocco R. Immune tolerance in hemophilia-principal results from the International Registry. Report of the factor VIII and IX Subcommittee. Thromb Haemost 1994; 72: 155-8.
13. DiMichele DM, Kroner BL. The North American Immune Tolerance Registry: practices, outcomes, outcome predictors. Thromb Haemost 2002; 87: 52-7.
14. Lenk H. The German Registry of immune tolerance treatment in hemophilia-1999 update. Haematologica 2000; 85: 45-7.
15. Haya S, Lopez MF, Aznar JA, Batlle J. Immune tolerance treatment in haemophilia patients with inhibitors: the Spanish Registry. Haemophilia 2001; 7: 154-9.
16. Ewing NP, Sanders NL, Dietrich SL, Kasper CK. Induction of immune tolerance to factor VIII in hemophiliacs with inhibitors. JAMA 1988; 259: 65-8.
17. Mauser-Bunschoten EP, Nieuwenhuis HK, Roosendaal G, van den Berg HM. Low-dose immune tolerance induction in hemophilia A patients with inhibitors. Blood 1995; 86: 983-8.
18. Kroner BL. Comparison of the international immune tolerance registry and the North American immune tolerance registry. Vox Sang 1999; 77(SUPPL. 1): 33-7.
19. Coppola A, Margaglione M, Santagostino E et al. Factor VIII gene (F8) mutations as predictors of outcome in immune tolerance induction of hemophilia A patients with high-responding inhibitors. J Thromb Haemost 2009; 7: 1809-15.
20. Ragni MV, Ojeifo O, Feng J et al. Risk factors for inhibitor formation in haemophilia: a prevalent case-control study. Haemophilia 2009; 15: 1074-82.
21. Damiano ML, Hutter JJ Jr. Immune tolerance for haemophilia patients with inhibitors: analysis of the western United States experience. The Tri-Regional Nursing Group. Haemophilia 2000; 6: 526-32.
22. Ter Avest PC, Fischer K, Gouw SC, Van Dijk K, Mauser-Bunschoten EP. Successful low dose immune tolerance induction in severe haemophilia A with inhibitors below 40 Bethesda Units. Haemophilia 2010; 16: 71-9.
23. Callaghan MU, Rajpurkar M, Chitlur M, Warrier I, Lusher J. Immune tolerance induction in 31 children with haemophilia A: is ITI less successful in African Americans? Haemophilia 2011; 17: 483-9.
24. Oldenburg J, Schwaab R, Brackmann HH. Induction of immune tolerance in haemophilia A inhibitor patients by the 'Bonn Protocol': predictive parameter for therapy duration and outcome. Vox Sang 1999; 77: 49-54.
25. Salviato R, Belvini D, Radossi P et al. F8 gene mutation profile and ITT response in a cohort of Italian haemophilia A patients with inhibitors. Haemophilia 2007; 13: 361-72.
26. Peyvandi F, Jayandharan G, Chandy M et al. Genetic diagnosis of haemophilia and other inherited bleeding disorders. Haemophilia 2006; 12(SUPPL. 3): 82-9.
27. 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.
28. Mariani G, Siragusa S, Kroner BL. Immune tolerance induction in hemophilia A: a review. Semin Thromb Hemost 2003; 29: 69-76.
29. DiMichele DM, Kroner BL. Analysis of the North American Immune Tolerance Registry (NAITR) 1993-1997: current practice implications. ISTH Factor VIII/IX Subcommittee Members. Vox Sang 1999; 77(SUPPL. 1): 31-2.
30. Unuvar A, Warrier I, Lusher JM. Immune tolerance induction in the treatment of paediatric haemophilia A patients with factor VIII inhibitors. Haemophilia 2000; 6: 150-7.
31. Barnes C, Rivard GE, Poon MC 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.
32. Kreuz W, Escuriola-Ettingshausen C, Auerswald G. Immune tolerance induction in haemophilia A patients with inhibitors: the choice of concentrate affecting success. Haematologica 2001; 86(SUPPL. 4): 16-20.
33. 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.
34. Orsini F, Rothschild C, Beurrier P, Faradji A, Goudemand J, Polack B. Immune tolerance induction with highly purified plasma-derived factor VIII containing von Willebrand factor in hemophilia A patients with high-responding inhibitors. Haematologica 2005; 90: 1288-90.
35. Gringeri A, Musso R, Bernasconi S. Immunotolerance induction (ITI) with high purity FVIII/VWF concentrates in inhibitor patients with a high risk of a poor response to ITI: a prospective surveillance. J Thromb Haemost 2005; 3(SUPPL. 1): A207.
36. Kurth MAH, DiMichele D, Sexauer C et al. Immune tolerance therapy utilizing factor VIII/von Willebrand factor concentrate in haemophilia A patients with high titre factor VIII inhibitors. Haemophilia 2008; 14: 50-5.
37. Oldenburg J, Jimenez-Yuste V, Peiro-Jordan R, Aledort LM, Santagostino E. Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate. Haemophilia 2014; 20: 83-91.
38. van Velzen AS, Peters M, van der Bom JG, Fijnvandraat K. Effect of von Willebrand factor on inhibitor eradication in patients with severe haemophilia A: a systematic review. Br J Haematol 2014; 166: 485-495.
39. Collins PW, Mathias M, Hanley J et al. Rituximab and immune tolerance in severe hemophilia A: a consecutive national cohort. J Thromb Haemost 2009; 7: 787-94.
40. Franchini M, Mengoli C, Lippi G et al. Immune tolerance with rituximab in congenital haemophilia with inhibitors: a systematic literature review based on individual patients' analysis. Haemophilia 2008; 14: 903-12.
41. Leissinger C, Josephson CD, Granger S et al. Rituximab for treatment of inhibitors in haemophilia A. A Phase II Study. Thromb Haemost 2014; 112: 445-58.
42. DiMichele DM, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: consensus recommendations. Hemophilia 2007; 13(SUPPL. 1): 1-22.
43. Meeks SL, Chapman RL, Kempton C, Dunn AL. Late immune tolerance induction in haemophilia A patients. Haemophilia 2013; 19: 445-8.
44. Kruse-Jarres R. Current controversies in the formation and treatment of alloantibodies to factor VIII in congenital hemophilia A. Hematology 2011; 2011: 407-12.
45. Darby SC, Keeling DM, Spooner RJ et al. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. J Thromb Haemost 2004; 2: 1047-54.
46. Nakar CT, Manco-Johnson MJ, Lail A et al. Prompt immune tolerance induction at inhibitor diagnosis regardless of titer may increase overall success in hemophilia A with inhibitors: experience of two US centers. Blood 2013; 122: 575.
47. Collins PW, Chalmers E, Hart DP et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). Br J Haematol 2013; 160: 153-70.
48. Hay CR, Palmer B, Chalmers E et al. Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United Kingdom. Blood 2011; 117: 6367-70.
49. Tagariello G, Iorio A, Matino D et al. High rate of spontaneous inhibitor clearance during the long term observation study of a single cohort of 524 haemophilia A patients not undergoing immunotolerance. J Hematol Oncol 2013; 6: 63.
50. Caram C, de Souza RG, de Sousa JC et al. The long-term course of factor VIII inhibitors in patients with congenital haemophilia A without immune tolerance induction. Thromb Haemost 2011; 105: 59-65.
51. Mariani G, Kroner B. Immune tolerance in hemophilia with factor VIII inhibitors: predictors of success. Haematologica 2001; 86: 1186-93.
52. Hay CRM. Immune tolerance induction: current status. Hematol Educ 2013; 7: 1-6.
53. Dardikh M, Albert T, Masereeuw R et al. Low-titre inhibitors, undetectable by the Nijmegen assay, reduce factor VIII half-life after immune tolerance induction. J Thromb Haemost 2012; 10: 706-8.
54. Hay C. Annual Report 2012 and Bleeding Disorder Statistics for the Financial Year 2011/12. UKHCDO 2012. Available at www.ukhcdo.org. Accessed June 18, 2014.
55. National Hemophilia Foundation. MASAC [Medical and Scientific Advisory Council] Recommendation 179. MASAC Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding). Adopted November 4, 2007. Available at http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007.
56. Barnes C, Brown SA, Curtin J, Dunkley S. When is enough..enough? Developing consensus of definition of failure of immune tolerance induction in patients with haemophilia and inhibitors. Haemophilia 2014; 20: e275-9.
57. Srivastava A, Brewer AK, Mauser-Bunschoten EP et al. Guidelines for the management of hemophilia. Haemophilia 2013; 19: e1-47.
58. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. New England J Med 2007; 357: 535-44.
59. Manco-Johnson MJ, Kempton CL, Reding MT et al. Randomized, controlled, parallel-group trial of routine prophylaxis versus on-demand treatment with rFVIII-FS in adults with severe hemophilia A (SPINART). J Thromb Haemost 2013; 11: 1119-1127.
60. Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9: 700-10.
61. Antun A, Monahan P, Manco-Johnson M et al. Natural history of inhibitor recurrence following successful immune tolerance induction. Blood 2013; 122: Abstract 1106.
62. Astermark J, Donfield SM, DiMichele DM et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study. Blood 2007; 109: 546-51.
63. DiMichele D, Rivard G, Hay C, Antunes S. Inhibitors in haemophilia: clinical aspects. Haemophilia 2004; 10: 140-5.
64. Konkle BA, Ebbesen LS, Erhardtsen E et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007; 5: 1904-13.
65. Leissinger C, Gringeri A, Antmen B et al. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. New England J Med 2011; 365: 1684-92.
66. Antunes S, Tangada S, Stasyshyn O et al. A prospective, open-label-randomized, parallel study with AICC to evaluate the efficacy and safety of prophylactic versus on-demand treatment in hemophilia A or B subjects with inhibitors Haemophilia 2014; 20: 65-72.