Immune tolerance induction in patients with haemophilia a and inhibitors: Effectiveness and cost analysis in an European Cohort (The ITER Study)

Haemophilia

Volumn 22, Issue 1, 2016, Pages 96-102

  Rocino, A ^a   Cortesi, P A ^b,c   Scalone, L ^b,c   Mantovani, L G ^b,c   Crea, R ^d   Gringeri, A ^e   Altisent, C ^f   Astermark, J ^g   Diniz, M J ^h   Fijnvandraat, K ⁱ   Klamroth, R ^j   Lambert, T ^k   Lavigne Lissalde, G ^l   Lopez Fernandez, M F ^m   Morfini, M ⁿ  

^a San Giovanni Bosco Hospital   (Italy)

^b UNIVERSITY OF MILANO BICOCCA   (Italy)

^c CHARTA Foundation   (Italy)

^d Baxalta Italia Srl   (Italy)

^e Baxalta Innovations GmbH   (Austria)

^f HOSPITAL UNIVERSITARI VALL D HEBRON   (Spain)

^g LUND UNIVERSITY HOSPITAL   (Sweden)

^h HOSPITAL DE SÃO JOSÉ   (Portugal)

ⁱ EMMA CHILDREN S HOSPITAL   (Netherlands)

^j Vivantes Klinikum in Friedrichhain Klinik fuer Innere Medizin Haemophilie zentrum   (Germany)

^k BICÊTRE HOSPITAL   (France)

^l DEPARTMENT OF NEUROLOGY   (France)

^m HOSPITAL JUAN CANALEJO   (Spain)

ⁿ CAREGGI UNIVERSITY HOSPITAL   (Italy)

more..

Author keywords

Costs; Haemophilia A; Immunotolerance; Inhibitors; Success predictors

Indexed keywords

BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 8; NEUTRALIZING ANTIBODY;

ADOLESCENT; ADULT; ARTICLE; CHILD; COST EFFECTIVENESS ANALYSIS; COST OF ILLNESS; ERADICATION THERAPY; HEMOPHILIA A; HUMAN; IMMUNE TOLERANCE INDUCTION; IMMUNOLOGICAL TOLERANCE; INFANT; MAJOR CLINICAL STUDY; PRIORITY JOURNAL; QUALITY OF LIFE; TREATMENT DURATION; TREATMENT OUTCOME; COHORT ANALYSIS; COST; ECONOMICS; EUROPE; IMMUNOLOGY; PRESCHOOL CHILD; RETROSPECTIVE STUDY; YOUNG ADULT;

ADOLESCENT; ADULT; ANTIBODIES, NEUTRALIZING; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; COSTS AND COST ANALYSIS; EUROPE; FACTOR VIII; HEMOPHILIA A; HUMANS; INFANT; QUALITY OF LIFE; RETROSPECTIVE STUDIES; YOUNG ADULT;

EID: 84958891367     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12780     Document Type: Article

References (28)

1. Gringeri A, Mantovani LG, Scalone L, Mannucci PM, COCIS. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS Study Group. Blood 2003; 102: 2358-63.
2. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003; 9: 418-35.
3. Ullman M, Hoots WK. Assessing the costs for clinical care of patients with high responding factor VIII and IX inhibitors. Haemophilia 2006; 12(Suppl 6): 74-80.
4. Leissinger C, Gringeri A, Antmen B et al. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med 2011; 365: 1684-92.
5. Abbonizio F, Giampaolo A, Coppola A; Italian Association of Haemophilia Centres, Arcieri R, Hassan HJ. Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. Haemophilia 2014; 20: e243-50.
6. Srivastava A, Brewer AK, Mauser-Bunschoten EP et al. Guidelines for the management of hemophilia. Haemophilia 2013; 19: e1-47.
7. Colvin BT, Astermark J, Fischer K et al. Inter disciplinary working group European principles of haemophilia care. Haemophilia 2008; 14: 361-74.
8. Collins PW, Chalmers E, Hart DP et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition), UK Haemophilia Centre Doctors Organization. Br J Haematol 2013; 160: 153-70.
9. Rocino A, Coppola A, Franchini M, Castaman G, Santoro C, Zanon E; Italian Association of Haemophilia Centres (AICE) Working Party. Principles of treatment and update of recommendations for the management of haemophilia and congenital bleeding disorders in Italy. Blood Transfus 2014; 12: 575-98.
10. Astermark J, Morado M, Rocino A, van den Berg HM, von Depka M, Gringeri A, EHTSB. Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors. Haemophilia 2006; 12: 363-71.
11. 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.
12. 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.
13. Mariani G, Kroner B; Immune Tolerance Study Group (ITSG). Immune tolerance in hemophilia with factor VIII inhibitors: predictors of success. Haematologica 2001; 86: 1186-93.
14. Lenk H, the Study Group of the German Haemophilia Centres. The German national immune tolerance registry, 1997 update. Vox Sang 1999; 77: 28-30.
15. Haya S, López MF, Aznar JA, Batlle J; Spanish Immune Tolerance Group. Immune tolerance treatment in haemophilia patients with inhibitors: the Spanish Registry. Haemophilia 2001; 7: 154-9.
16. Di Michele DM, Kroner BL, North American Immune Tolerance Study Group. The North American Immune Tolerance Registry: practices, outcomes, outcome predictors. Thrombost Haemost 2002; 87: 52-7.
17. Coppola A, Margaglione M, Santagostino E et al. AICE PROFIT Study Group. 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.
18. Hay CR, DiMichele DM; International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335-44.
19. Di Minno G, Coppola A. Management of patients with long-term inhibitors: is immune tolerance an underestimated life-long solution? Haemophilia 2013; 19(Suppl 1): 18-23.
20. Dixon WJ, Massey FJ. Introduction to Statistical Analysis. 4th ed. New York: McGraw-Hill, 1983: 105-7.
21. Brackmann HH, Oldenburg J, Schwaab R. Immune tolerance for the treatment of factor VIII inhibitors - twenty years of the Bonn Protocol. Vox Sang 1996; 70: 30-5.
22. Auerswald G, von Depka Prondzinski M, Ehlken B et al. Treatment patterns and cost-of-illness of severe haemophilia in patients with inhibitors in Germany. Haemophilia 2004; 10: 499-508.
23. Knight C. Health economics of treating haemophilia A with inhibitors. Haemophilia 2005; 11(Suppl 1): 11-17.
24. Oldenburg J, Austin SK, Kessler CM. ITI choice for the optimal management of inhibitor patients - from a clinical and pharmacoeconomic perspective. Haemophilia 2014; 20(Suppl 6): 17-26.
25. Colowick AB, Bohn RL, Avorn J, Ewenstein BM. Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper. Blood 2000; 96: 1698-702.
26. Farruggia A, Hermans C, Franchini M. Assessing options for treating haemophilia with inhibitors. Haemophilia 2015; 21: 307-9.
27. Earnshaw SR, Graham CN, McDade CL, Spears JB, Kessler CM. Factor VIII alloantibody inhibitors: cost analysis of immune tolerance induction vs. prophylaxis and on-demand with bypass treatment. Haemophilia 2015; 21: 310-19.
28. Villarrubia R, Oyagüez I, Álvarez-Román MT, Mingot-Castellano ME, Parra R, Casado MA. Cost analysis of prophylaxis with activated prothrombin complex concentrate vs. on-demand therapy with activated factor VII in severe haemophilia A patients with inhibitors, in Spain. Haemophilia 2015; 21: 320-9.