Primary and rescue immune tolerance induction in children and adults: A multicentre international study with a VWF-containing plasma-derived FVIII concentrate

Haemophilia

Volumn 20, Issue 1, 2014, Pages 83-91

  Oldenburg, J ^a   Jimenez Yuste V ^b   Peiro Jordan R ^c   Aledort, L M ^d   Santagostino, E ^e  

^a UNIVERSITY HOSPITAL BONN   (Germany)

^b HOSPITAL UNIVERSITARIO LA PAZ   (Spain)

^c Instituto Grifols SA   (Spain)

^d MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^e FONDAZIONE IRCCS CA GRANDA OSPEDALE MAGGIORE POLICLINICO   (Italy)

Author keywords

Coagulation factor VIII; Factor concentrate; FVIII inhibitor; Haemophilia; Immune tolerance; Von Willebrand factor

Indexed keywords

BLOOD CLOTTING FACTOR 8; FANHDI; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR;

ADOLESCENT; ADULT; ARTICLE; CHILD; GERMANY; HEMOPHILIA A; HUMAN; IMMUNOLOGICAL TOLERANCE; INFANT; ITALY; MAJOR CLINICAL STUDY; PRESCHOOL CHILD; PRIORITY JOURNAL; PROGNOSIS; SCHOOL CHILD; SPAIN; TREATMENT OUTCOME; TREATMENT RESPONSE;

COAGULATION FACTOR VIII; FACTOR CONCENTRATE; FVIII INHIBITOR; HAEMOPHILIA; IMMUNE TOLERANCE; VON WILLEBRAND FACTOR;

ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; FACTOR VIII; GERMANY; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; INFANT; INFANT, NEWBORN; ITALY; MIDDLE AGED; RETROSPECTIVE STUDIES; SPAIN; TREATMENT OUTCOME; VON WILLEBRAND FACTOR; YOUNG ADULT;

EID: 84890858676     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12263     Document Type: Article

References (35)

1. Jones PK, Ratnoff OD. The changing prognosis of classic hemophilia (factor VIII "deficiency"). Ann Intern Med 1991; 114: 641-8.
2. Farshid M. Viral safety evaluation of plasma-derived therapeutic products. Dev Biol (Basel) 2004; 118: 11-5.
3. Mannucci PM, Mancuso ME, Santagostino E. How we choose factor VIII to treat hemophilia. Blood 2012; 119: 4108-14.
4. Addiego J, Kasper C, Abildgaard C et al. Frequency of inhibitor development in haemophiliacs treated with low-purity factor VIII. Lancet 1993; 342: 462-4.
5. Kreuz W, Ettingshausen CE, Auerswald G et al. Epidemiology of inhibitors and current treatment strategies. Haematologica 2003; 88: EREP04.
6. Abshire T, Kenet G. Recombinant factor VIIa: review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors. J Thromb Haemost 2004; 2: 899-909.
7. 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.
8. Brackmann HH, Effenberger E, Hess L, Schwaab R, Oldenburg J. NovoSeven in immune tolerance therapy. Blood Coagul Fibrinolysis 2000; 11(Suppl. 1): S39-44.
9. 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.
10. 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.
11. Coppola A, Di Minno MN, Santagostino E. Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence-based approaches. Br J Haematol 2010; 150: 515-28.
12. Haya S, Casaña P, Moret A et al. Immunotolerance induction treatments in hemophilia. J Coagul Disord 2009; 1: 1-6.
13. Kroner BL. Comparison of the international immune tolerance registry and the North American immune tolerance registry. Vox Sang 1999; 77(Suppl. 1): 33-7.
14. Mariani G, Scheibel E, Nogao T et al. Immunetolerance as treatment of alloantibodies to factor VIII in hemophilia. The International Registry of Immunetolerance Protocols. Semin Hematol 1994; 31: 62-4.
15. 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(Suppl. 1): 49-54.
16. Kreuz W. The role of VWF for the success of immune tolerance induction. Thromb Res 2008; 122(Suppl. 2): S7-12.
17. Gringeri A, Musso R, Mazzucconi MG et al. Immune tolerance induction with a high purity von Willebrand factor/VIII complex concentrate in haemophilia A patients with inhibitors at high risk of a poor response. Haemophilia 2007; 13: 373-9.
18. Greninger DA, Saint-Remy JM, Jacquemin M, Benhida A, DiMichele DM. The use of factor VIII/von Willebrand factor concentrate for immune tolerance induction in haemophilia A patients with high-titre inhibitors: association of clinical outcome with inhibitor epitope profile. Haemophilia 2008; 14: 295-302.
19. Bidlingmaier C, Kurnik K, Escuriola-Ettingshausen C et al. Immune tolerance induction with a factor VIII concentrate containing von Willebrand factor (Haemoctin SDH(R)) in 14 patients with severe haemophilia A. Haemophilia 2011; 17: e837-40.
20. DiMichele DM, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: consensus recommendations. Haemophilia 2007; 13(Suppl. 1): 1-22.
21. Gringeri A, Musso R, Bernasconi S et al. Immune tolerance induction (ITI) with a high purity VWF concentrates in inhibitor patients with a high risk of a poor response to ITI: a prospective surveillance. J Thromb Haemost 2005; 3: A207.
22. Dimichele D. The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 2009; 15: 320-8.
23. Haya S, Lopez MF, Aznar JA, Batlle J. Immune tolerance treatment in haemophilia patients with inhibitors: the Spanish Registry. Haemophilia 2001; 7: 154-9.
24. Lenk H. The German Registry of immune tolerance treatment in hemophilia-1999 update. Haematologica 2000; 85: 45-7.
25. Dasgupta S, Repessé Y, Bayry J et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007; 109: 610-2.
26. Kurth M, Puetz J, Kouides P et al. The use of a single von Willebrand factor-containing, plasma-derived FVIII product in hemophilia A immune tolerance induction: the US experience. J Thromb Haemost 2011; 9: 2229-34.
27. Orsini F, Rotschild 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.
28. ®) P as immune tolerance induction in patients with severe haemophilia A who failed previous induction attempts: a multicentre observational study. Haemophilia 2013; 19: 281-6.
29. Hay CR, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335-44.
30. Wight J, Paisley S, Knight C. Immune tolerance induction in patients with haemophilia A with inhibitors: a systematic review. Haemophilia 2003; 9: 436-63.
31. Carcao M, St Louis J, Poon MC et al. Rituximab for congenital haemophiliacs with inhibitors: a Canadian experience. Haemophilia 2006; 12: 7-18.
32. Fox RA, Neufeld EJ, Bennett CM. Rituximab for adolescents with haemophilia and high titre inhibitors. Haemophilia 2006; 12: 218-22.
33. Kubisz P, Plamenova I, Holly P, Stasko J. Successful immune tolerance induction consisting of high-dose factor VIII rich in von Willebrand factor and pulsed intravenous immunoglobulin: a case report. J Med Case Rep 2012; 6: 350.
34. Nilsson IM, Berntorp E, Zettervall O. Induction of immune tolerance in patients with hemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII. N Engl J Med 1988; 318: 947-50.
35. Tarantino MD, Lail A, Donfield SM et al. Surveillance of infectious complications associated with central venous access devices in children with haemophilia. Haemophilia 2003; 9: 588-92.