Long-term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma-derived FVIII/VWF product: the long-term ITI study

Haemophilia

Volumn 22, Issue 6, 2016, Pages 859-865

  Jimenez Yuste V ^a   Oldenburg, J ^b   Rangarajan, S ^c   Peiro Jordan R ^d   Santagostino, E ^e  

^a HOSPITAL UNIVERSITARIO LA PAZ   (Spain)

^b UNIVERSITY HOSPITAL BONN   (Germany)

^c BASINGSTOKE AND NORTH HAMPSHIRE HOSPITAL   (United Kingdom)

^d I3 Research   (Spain)

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

Author keywords

coagulation factor VIII; factor concentrate; FVIII inhibitor; haemophilia; immune tolerance; long term

Indexed keywords

BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 PLUS VON WILLEBRAND FACTOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8; F8 PROTEIN, HUMAN; VON WILLEBRAND FACTOR;

ADULT; ARTICLE; CONTROLLED STUDY; DISEASE CLASSIFICATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; FOLLOW UP; HEMOPHILIA A; HOSPITALIZATION; HUMAN; IMMUNOLOGICAL TOLERANCE; IMMUNOMODULATION; MAJOR CLINICAL STUDY; MEDICAL RECORD; OBSERVATIONAL STUDY; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RETROSPECTIVE STUDY; TREATMENT INDICATION; CLINICAL TRIAL; FEMALE; GENETICS; MALE; MULTICENTER STUDY; TREATMENT OUTCOME; YOUNG ADULT;

ADULT; FACTOR VIII; FEMALE; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; MALE; RETROSPECTIVE STUDIES; TREATMENT OUTCOME; VON WILLEBRAND FACTOR; YOUNG ADULT;

EID: 84978473786     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12986     Document Type: Article

References (24)

1. Montgomery RR, Gill JC, Scott JP. Hemophilia and von Willebrand Disease. In: Nathan DG, Orkin SH, Ginsberg D, Look AT eds. Hematology of Infancy and Childhood. Philadelphia: WB Saunders, 2003: 1548–9.
2. Ehrenforth S, Kreuz W, Scharrer I et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet 1992; 339: 594–8.
3. 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: 485–95.
4. 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.
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. 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.
7. 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.
8. Rangarajan S, Yee TT, Roy A. Immune tolerance induction in adult severe haemophilia A patients with a single FVIII/VWF product: the UK experience. J Thromb Haemost 2013; 11(Suppl 2): 1081.
9. 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.
10. Ettingshausen CE, Kreuz W. Role of von Willebrand factor in immune tolerance induction. Blood Coagul Fibrinolysis 2005; 16(Suppl 1): S27–31.
11. Kreuz W, Escuriola EC, Vdovin V et al. First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study. Haemophilia 2016; 22: 87–95.
12. Robertson JD, Higgins P, Price J, Dunkley S, Barrese G, Curtin J. Immune tolerance induction using a factor VIII/von Willebrand factor concentrate (BIOSTATE), with or without immunosuppression, in Australian paediatric severe haemophilia A patients with high titre inhibitors: a multicentre, retrospective study. Thromb Res 2014; 134: 1046–51.
13. Wight J, Paisley S, Knight C. Immune tolerance induction in patients with haemophilia A with inhibitors: a systematic review. Haemophilia 2003; 9: 436–63.
14. 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.
15. Rangarajan S, Jimenez-Yuste V, Santagostino E. Adult haemophilia A patients with inhibitors: successful immune tolerance induction with a single FVIII/VWF product. Haemophilia 2014; 20: e414–7.
16. Hilliard P, Funk S, Zourikian N et al. Hemophilia joint health score reliability study. Haemophilia 2006; 12: 518–25.
17. Arnold WD, Hilgartner MW. Hemophilic arthropathy. Current concepts of pathogenesis and management. J Bone Joint Surg Am 1977; 59: 287–305.
18. Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia 2014; 20: 459–63.
19. Aznar JA, Moret A, Ibanez F et al. Inhibitor development after switching of FVIII concentrate in multitransfused patients with severe haemophilia A. Haemophilia 2014; 20: 624–9.
20. Hay CR, Palmer BP, Chalmers EA et al. The incidence of factor VIII inhibitors in severe haemophilia A following a major switch from full-length to B-domain-deleted factor VIII: a prospective cohort comparison. Haemophilia 2015; 21: 219–26.
21. 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.
22. Ettingshausen CE, Kreuz W. The immune tolerance induction (ITI) dose debate: does the International ITI Study provide a clearer picture? Haemophilia 2013; 19(Suppl 1): 12–7.
23. Hay CR, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335–44.
24. Antun A, Monahan PE, Manco-Johnson MJ et al. Inhibitor recurrence following immune tolerance induction: a multicenter retrospective cohort study. J Thromb Haemost 2015; 13: 1980–8.