On-demand treatment of bleeds in haemophilia patients with inhibitors: Strategies for securing and maintaining predictable efficacy with recombinant activated factor VII

Haemophilia

Volumn 18, Issue 2, 2012, Pages 255-262

  Sorensen B ^a   Dargaud, Y ^b   Kenet, G ^c   Lusher, J ^d   Mumford, A ^e   Pipe, S ^f   Tiede, A ^g  

^a ST THOMAS HOSPITAL   (United Kingdom)

^b EDOUARD HERRIOT HOSPITAL   (France)

^c SHEBA MEDICAL CENTER   (Israel)

^d CHILDREN'S HOSPITAL OF MICHIGAN   (United States)

^e BRISTOL HAEMATOLOGY AND ONCOLOGY CENTRE   (United Kingdom)

^f UNIVERSITY OF MICHIGAN   (United States)

^g HANNOVER MEDICAL SCHOOL   (Germany)

Author keywords

Haemophilia; Inhibitor; Recombinant activated factor VII; Treatment strategies

Indexed keywords

ANTIFIBRINOLYTIC AGENT; RECOMBINANT BLOOD CLOTTING FACTOR 7A; STEROID;

ADD ON THERAPY; ADOLESCENT; ADULT; ARTICLE; CHILD; CLINICAL ARTICLE; COLD TREATMENT; COMPRESSION THERAPY; DISEASE SEVERITY; DOSE CALCULATION; DRUG CLEARANCE; DRUG DOSE COMPARISON; DRUG EFFICACY; FOLLOW UP; HEMARTHROSIS; HEMOPHILIA; HEMOSTASIS; HOMEOPATHY; HUMAN; MUSCLE BLEEDING; PHYSIOTHERAPY; PRIORITY JOURNAL; RECOMMENDED DRUG DOSE; REST; SKIN BLEEDING;

ADULT; CHILD; EUROPE; FACTOR VIIA; HEMOPHILIA A; HEMORRHAGE; HUMANS; PRACTICE GUIDELINES AS TOPIC; QUESTIONNAIRES; RECOMBINANT PROTEINS; TIME FACTORS; UNITED STATES;

EID: 84857108099     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2011.02612.x     Document Type: Article

References (39)

1. Berntorp E, Shapiro A, Astermark J et al. Inhibitor treatment in haemophilias A and B: summary statement for the 2006 international consensus conference. Haemophilia 2006; 12(Suppl. 6): 1-7.
2. Gringeri A, Mannucci PM, Italian Association of Haemophilia Centres. Italian guidelines for the diagnosis and treatment of patients with haemophilia and inhibitors. Haemophilia 2005; 11: 611-9.
3. Hay CR, Baglin TP, Collins PW, Hill FG, Keeling DM. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the UK Haemophilia Centre Doctors' Organization (UKHCDO). Br J Haematol 2000; 111: 78-90.
4. Hay CR, Brown S, Collins PW, Keeling DM, Liesner R. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation. Br J Haematol 2006; 133: 591-605.
5. Mathew P. Current opinion on inhibitor treatment options. Semin Hematol 2006; 43(Suppl. 4): S8-13.
6. Paisley S, Wight J, Currie E, Knight C. The management of inhibitors in haemophilia A: introduction and systematic review of current practice. Haemophilia 2003; 9: 405-17.
7. Perez Bianco R, Ozelo MC, Villaca PR et al. Diagnosis and treatment of congenital hemophilia with inhibitors a Latin American perspective. Medicina (B Aires) 2008; 68: 227-42.
8. Teitel J, Berntorp E, Collins P et al. A systematic approach to controlling problem bleeds in patients with severe congenital haemophilia A and high-titre inhibitors. Haemophilia 2007; 13: 256-63.
9. Key NS, Aledort LM, Beardsley D et al. Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors. Thromb Haemost 1998; 80: 912-8.
10. Kavakli K, Makris M, Zulfikar B et al. Home treatment of haemarthroses using a single dose regimen of recombinant activated factor VII in patients with haemophilia and inhibitors. A multi-centre, randomised, double-blind, cross-over trial. Thromb Haemost 2006; 95: 600-5.
11. Santagostino E, Mancuso ME, Rocino A, Mancuso G, Scaraggi F, Mannucci PM. A prospective randomized trial of high and standard dosages of recombinant factor VIIa for treatment of hemarthroses in hemophiliacs with inhibitors. J Thromb Haemost 2006; 4: 367-71.
12. -1-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison. Haemophilia 2008; 14: 287-94.
13. 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.
14. Croom KF, McCormack PL. Recombinant factor VIIa (eptacog alfa): a review of its use in congenital hemophilia with inhibitors, acquired hemophilia, and other congenital bleeding disorders. BioDrugs 2008; 22: 121-36.
15. Shapiro A. Inhibitor treatment: state of the art. Dis Mon 2003; 49: 22-38.
16. 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.
17. Lusher JM. Early treatment with recombinant factor VIIa results in greater efficacy with less product. Eur J Haematol Suppl 1998; 63: 7-10.
18. Lusher JM. Recombinant activated factor VII for treatment of intramuscular haemorrhages: a comparison of early versus late treatment. Blood Coagul Fibrinolysis 1998; 9(Suppl. 1): S111-4.
19. Lusher JM. Acute hemarthroses: the benefits of early versus late treatment with recombinant activated factor VII. Blood Coagul Fibrinolysis 2000; 11(Suppl. 1): S45-9.
20. Salaj P, Brabec P, Penka M et al. Effect of rFVIIa dose and time to treatment on patients with haemophilia and inhibitors: analysis of HemoRec registry data from the Czech Republic. Haemophilia 2009; 15: 752-9.
21. Abshire TC. Dose optimization of recombinant factor VIIa for control of mild to moderate bleeds in inhibitor patients: improved efficacy with higher dosing. Semin Hematol 2004; 41(Suppl. 1): 3-7.
22. Santagostino E, Gringeri A, Mannucci PM. Home treatment with recombinant activated factor VII in patients with factor VIII inhibitors: the advantages of early intervention. Br J Haematol 1999; 104: 22-6.
23. Escobar MA. Health economics in haemophilia: a review from the clinician's perspective. Haemophilia 2010; 16(Suppl. 3): 29-34.
24. ®) in children vs. adults with haemophilia A. Haemophilia 2004; 10: 352-9.
25. Tengborn L. Fibrinolytic Inhibitors in the Management of Bleeding Disorders. Canada: WFH publications, Treatment of Hemophilia; No. 42 (April 2007).
26. Beyer R, Ingerslev J, Sorensen B. Muscle bleeds in professional athletes - diagnosis, classification, treatment and potential impact in patients with haemophilia. Haemophilia 2010; 16: 858-65.
27. Beyer R, Ingerslev J, Sorensen B. Current practice in the management of muscle haematomas in patients with severe haemophilia. Haemophilia 2010; 16: 926-31.
28. Young G, Blain R, Nakagawa P, Nugent DJ. Individualization of bypassing agent treatment for haemophilic patients with inhibitors utilizing thromboelastography. Haemophilia 2006; 12: 598-604.
29. Nair SC, Dargaud Y, Chitlur M, Srivastava A. Tests of global haemostasis and their applications in bleeding disorders. Haemophilia 2010; 16(Suppl. 5): 85-92.
30. Levi MM, Vink R, de Jonge E. Management of bleeding disorders by prohemostatic therapy. Int J Hematol 2002; 76(Suppl. 2): 139-44.
31. Hedner U. Mechanism of action, development and clinical experience of recombinant FVIIa. J Biotechnol 2006; 124: 747-57.
32. Abshire T, Kenet G. Safety update on the use of recombinant factor VIIa and the treatment of congenital and acquired deficiency of factor VIII or IX with inhibitors. Haemophilia 2008; 14: 898-902.
33. Lindley CM, Sawyer WT, Macik BG et al. Pharmacokinetics and pharmacodynamics of recombinant factor VIIa. Clin Pharmacol Ther 1994; 55: 638-48.
34. Soucie JM, Nuss R, Evatt B et al. Mortality among males with hemophilia: relations with source of medical care. The Hemophilia Surveillance System Project Investigators. Blood 2000; 96: 437-42.
35. Monroe DM. Further understanding of recombinant activated factor VII mode of action. Semin Hematol 2008; 45(Suppl. 1): S7-11.
36. Ovanesov MV, Panteleev MA, Sinauridze EI et al. Mechanisms of action of recombinant activated factor VII in the context of tissue factor concentration and distribution. Blood Coagul Fibrinolysis 2008; 19: 743-55.
37. Pruthi RK, Schmidt KA, Slaby JA, Rodriguez V. Platelet dysfunction induced by herbal supplements in a patient with mild hemophilia A. J Thromb Haemost 2007; 5: 2556-8.
38. 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.
39. Valentino LA, Ewenstein B, Navickis RJ, Wilkes MM. Central venous access devices in haemophilia. Haemophilia 2004; 10: 134-46.