Prophylaxis in haemophilia with inhibitors: Update from international experience

Haemophilia

Volumn 16, Issue SUPPL. 2, 2010, Pages 16-23

  Carcao, M ^a   Lambert, T ^b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b BICÊTRE HOSPITAL   (France)

Author keywords

APCC; Bypassing agent; Immune tolerance induction; ITI; RFVIIa

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; RECOMBINANT BLOOD CLOTTING FACTOR 7A;

ARTHROPATHY; BLEEDING; CLINICAL TRIAL; DEVICE INFECTION; DRUG EFFICACY; DRUG HALF LIFE; DRUG INDICATION; DRUG SAFETY; ERADICATION THERAPY; HEMARTHROSIS; HEMOPHILIA; HEMOPHILIA A; HUMAN; IMMUNOLOGICAL TOLERANCE; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW; THROMBOSIS; TREATMENT DURATION; TREATMENT RESPONSE; VASCULAR ACCESS DEVICE;

ADOLESCENT; ADULT; BLOOD COAGULATION FACTOR INHIBITORS; BLOOD COAGULATION FACTORS; CHILD; CHILD, PRESCHOOL; DRUG ADMINISTRATION SCHEDULE; FACTOR IX; FACTOR VIIA; FACTOR VIII; HEMARTHROSIS; HEMOPHILIA A; HUMANS; INFANT; RECOMBINANT PROTEINS; YOUNG ADULT;

EID: 76749125487     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2009.02198.x     Document Type: Review

References (39)

1. Livnat T, Martinowitz U, Zivelin A, Seligsohn U. Effects of factor VIII inhibitor bypassing activity (FEIBA), recombinant factor VIIa or both on thrombin generation in normal and haemophilia A plasma. Haemophilia 2008, 14:782-6.
2. Morfini M, Haya S, Tagariello G. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007, 13:606-12.
3. Hilgartner MW, Makipernaa A, DiMichele DM. Long-term FEIBA prophylaxis does not prevent progression of existing joint disease. Haemophilia 2003, 9:261-8.
4. Berntorp E, Shapiro A, Astermark J. Inhibitor treatment in haemophilias A and B: summary statement for the 2006 international consensus conference. Haemophilia 2006, 12(Suppl 6):1-7.
5. Tjønnfjord GE, Holme PA. Factor eight inhibitor bypass activity (FEIBA) in the management of bleeds in hemophilia patients with high-titer inhibitors. Vasc Health Risk Manag 2007, 3:527-31.
6. DiMichele DM. Management of factor VIII inhibitors. Int J Hematol 2006, 83:119-25.
7. 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.
8. Hedner U. Recombinant factor VIIa: its background, development and clinical use. Curr Opin Hematol 2007, 14:225-9.
9. Leissinger CA. Prevention of bleeds in hemophilia patients with inhibitors: emerging data and clinical direction. Am J Hematol 2004, 77:187-93.
10. White GC, Rosendaal F, Aledort LM. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 2001, 85.
11. Kempton CL, White GC. How we treat a hemophilia A patient with a factor VIII inhibitor. Blood 2009, 113:11-7.
12. Leissinger CA. Use of prothrombin complex concentrates and activated prothrombin complex concentrates as prophylactic therapy in haemophilia patients with inhibitors. Haemophilia 1999, 5(Suppl 3):25-32.
13. Ho AY, Height SE, Smith MP. Immune tolerance therapy for haemophilia. Drugs 2000, 60:547-54.
14. Carcao MD. Side-effects and venous access issues with immune tolerance therapy. Haemophilia 2009, 15:494-500.
15. Valentino LA, Ewenstein B, Navickis RJ, Wilkes MM. Central venous access devices in haemophilia. Haemophilia 2004, 10:134-46.
16. Ewenstein BM, Valentino LA, Journeycake JM. Consensus recommendations for use of central venous access devices in haemophilia. Haemophilia 2004, 10:629-48.
17. Petrini P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia 2007, 13(Suppl 2):16-22.
18. Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet 1977, 2:933.
19. 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.
20. Kreuz W, Escuriola-Ettingshausen C, Mentzer D. Factor VIII inhibitor bypass activity (FEIBA) for prophylaxis during immune tolerance induction (ITI) in patients with high-responding inhibitors. Blood 2000, 96. Abstract 1141
21. Valentino L. FEIBA prophylaxis in hemophilia A patients with inhibitors results in a 95% reduction in bleeding episodes. Haemophilia 2004, 10:115. XXVIth International Congress of the World Federation of Haemophilia, Bangkok, Thailand, Abstract 22 PO 18
22. Leissinger CA, Becton DL, Ewing NP, Valentino LA. Prophylactic treatment with activated prothrombin complex concentrate (FEIBA) reduces the frequency of bleeding episodes in paediatric patients with haemophilia A and inhibitors. Haemophilia 2007, 13:249-55.
23. Valentino LA. Assessing the benefits APCC prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2009, 7(Suppl 2). Abstract PP-WE-583
24. Shapiro AD, Gilchrist GS, Hoots WK, Cooper HA, Gastineau DA. Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in haemophilia patients with inhibitors undergoing surgery. Thromb Haemost 1998, 80:773-8.
25. Lindley CM, Sawyer WT, Macik BG. Pharmacokinetics and pharmacodynamics of recombinant factor VIIa. Clin Pharmacol Ther 1994, 55:638-48.
26. Morfini M, Auerswald G, Kobelt RA. Prophylactic treatment of haemophilia patients with inhibitors: clinical experience with recombinant factor VIIa in European Haemophilia Centres. Haemophilia 2007, 13:502-7.
27. Konkle BA, Ebbesen LS, Erhardtsen E. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007, 5:1904-13.
28. Hoots WK. Arthropathy in inhibitor patients: differences in the joint status. Semin Hematol 2008, 2(Suppl 1):S42-9.
29. Young G, McDaniel M, Nugent DJ. Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors. Haemophilia 2005, 11:203-7.
30. Saxon BR, Shanks D, Jory CB, Williams V. Effective prophylaxis with daily recombinant factor VIIa (rFVIIa-Novoseven) in a child with high titre inhibitors and a target joint. Thromb Haemost 2001, 86:1126-7.
31. Cooper HA, Jones CP, Campion E, Roberts HR, Hedner U. Rationale for the use of high dose rFVIIa in a high-titre inhibitor patient with haemophilia B during major orthopaedic procedures. Haemophilia 2001, 7:517-22.
32. Auerswald G. [Long-term prophylaxis in congenital haemophilia with inhibitors - experiences with rFVIIa]. Hamostaseologie 2007, 27:123-8.
33. Blatny J, Kohlerova S, Zapletal O, Fiamoli V, Penka M, Smith O. Prophylaxis with recombinant factor VIIa for the management of bleeding episodes during immune tolerance treatment in a boy with severe haemophilia A and high-response inhibitors. Haemophilia 2008, 14:1140-2.
34. Jiménez-Yuste V, Quintana M, Alvarez MT, Martin-Salces M, Hernandez-Navarro F. ''Primary prophylaxis'' with rFVIIa in a patient with severe haemophilia a and inhibitor. Blood Coagul Fibrinolysis 2008, 19:719-20.
35. Jiménez-Yuste V, Alvarez MT, Martin-Salces M. Prophylaxis in 10 patients with severe haemophilia A and inhibitor: different approaches for different clinical situations. Haemophilia 2009, 15:203-9.
36. Balsam LB, Timek TA, Pelletier MP. Factor eight inhibitor bypassing activity (FEIBA) for refractory bleeding in cardiac surgery: review of clinical outcomes. J Card Surg 2008, 23:614-21.
37. FEIBA® Summary of Product Characteristics 2007, Baxter AG, Vienna, Austria
38. NovoSeven® Summary of Product Characteristics 2009, NovoNordisk, Bagsvaerd, Denmark
39. Rodriguez-Merchan EC. The Haemophilia Joints: New Perspectives 2003, Oxford, UK, Blackwell Publishing Limited,