Evidence supporting the use of recombinant activated factor VII in congenital bleeding disorders

Drug Design, Development and Therapy

Volumn 4, Issue , 2010, Pages 107-116

  Johansson, Pär I ^a   Ostrowski, Sisse R ^a  

^a UNIVERSITY OF COPENHAGEN   (Denmark)

Author keywords

Coagulation factor IX; Coagulation factor VIII; FEIBA; Hemophilia; Hemostasis; Inhibitors; Novoseven; RCT; rFVIIa

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; BLOOD CLOTTING FACTOR 7A INHIBITOR; RECOMBINANT BLOOD CLOTTING FACTOR 7A; THROMBIN; TRANEXAMIC ACID; BLOOD CLOTTING FACTOR 7A; HEMOSTATIC AGENT; RECOMBINANT FVIIA; RECOMBINANT PROTEIN;

BLEEDING; BLOOD CLOTTING TEST; BOLUS INJECTION; CONTINUOUS INFUSION; DOSAGE SCHEDULE COMPARISON; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; EVIDENCE BASED MEDICINE; HEMARTHROSIS; HEMOPHILIA; HEMOSTASIS; HOME CARE; HOSPITAL CARE; HUMAN; LOW DRUG DOSE; OPTIMAL DRUG DOSE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; SECONDARY PREVENTION; SINGLE DRUG DOSE; THROMBIN TIME; THROMBOELASTOGRAPHY; THROMBOEMBOLISM; THROMBOSIS; VEIN THROMBOSIS; BIOSYNTHESIS; BLOOD CLOTTING; BODY WEIGHT; DRUG EFFECT; HEMOPHILIA A; PATHOPHYSIOLOGY; TREATMENT OUTCOME;

BLOOD COAGULATION; BODY WEIGHT; FACTOR VIIA; HEMOPHILIA A; HEMOSTATICS; HUMANS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECOMBINANT PROTEINS; THROMBIN; TREATMENT OUTCOME;

EID: 79952025130     PISSN: 11778881     EISSN: None     Source Type: Journal    
DOI: 10.2147/dddt.s11764     Document Type: Review

References (76)

1. Oldenburg J, Dolan G, Lemm G. Haemophilia care then, now and in the future. Haemophilia. 2009;15(Suppl 1):S2-S7.
2. Dimichele D. Inhibitors: resolving diagnostic and therapeutic dilemmas. Haemophilia. 2002;8:280-287.
3. Yee TT, Pasi KJ, Lilley PA, Lee CA. Factor VIII inhibitors in haemophiliacs: a single-centre experience over 34 years, 1964-97. Br J Haematol. 1999;104:909-914.
4. Kreuz W, Ettingshausen CE, Zyschka A, et al. Inhibitor development in previously untreated patients with hemophilia A: a prospective long- term follow-up comparing plasma-derived and recombinant products. Semin Thromb Hemost. 2002;28:285-290.
5. Goodeve A. The incidence of inhibitor development according to specific mutations - and treatment? Blood Coagul Fibrinolysis. 2003;14 (Suppl 1):S17-S21.
6. Morfini M, Haya S, Tagariello G, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia. 2007;13:606-612.
7. Sjamsoedin LJ, Heijnen L, Mauser-Bunschoten EP, et al. The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII. A double-blind clinical trial. N Engl J Med. 1981;305: 717-721.
8. Hedner U. Mechanism of action, development and clinical experience of recombinant FVIIa. J Biotechnol. 2006;124:747-757.
9. Pan-Petesch B, Laguna P, Mital A, et al. Single-dose (270 microg kg(-1)) recombinant activated factor VII for the treatment and prevention of bleeds in haemophilia A patients with inhibitors: experience from seven European haemophilia centres. Haemophilia. 2009;15:760-765.
10. 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; 80:912-918.
11. Lusher JM, Roberts HR, Davignon G, et al. A randomized, doubleblind comparison of two dosage levels of recombinant factor VIIa in the treatment of joint, muscle and mucocutaneous haemorrhages in persons with haemophilia A and B, with and without inhibitors. rFVIIa study group. Haemophilia. 1998;4:790-798.
12. Laurian Y, Goudemand C, Négrier C, et al. Use of recombinant activated factor VII as first-line therapy for bleeding episodes in haemophiliacs with factor VIII or IX inhibitors (NOSEPAC study). Blood Coagul Fibrinolysis. 1998;9:S155-S156.
13. Monroe DM, Hoffman M, Oliver JA, Roberts HR. Platelet activity of high-dose factor VIIa is independent of tissue factor. Br J Haematol. 1997;99:542-547.
14. Lisman T, Mosnier LO, Lambert T, et al. Inhibition of fibrinolysis by recombinant factor VIIa in plasma from patients with severe hemophilia A. Blood. 2002;99:175-179.
15. Shapiro AD, Gilchrist GS, Hoots WK, et al. Prospective, randomized trial of two doses of rFVIIa (NovoSeven) in haemophilia patients with inhibitors undergoing surgery. Thromb Haemost. 1998;80:773-778.
16. Santagostino E, Mancuso ME, Rocino A, et al. 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;4:367-371.
17. 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;95:600-605.
18. 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;109:546-551.
19. Konkle BA, Ebbesen LS, Erhardtsen E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost. 2007;5:1904-1913.
20. Pruthi RK, Mathew P, Valentino LA, et al. Haemostatic efficacy and safety of bolus and continuous infusion of recombinant factor VIIa are comparable in haemophilia patients with inhibitors undergoing major surgery. Results from an open-label, randomized, multicenter trial. Thromb Haemost. 2007;98:726-732.
21. Young G, Shafer FE, Rojas P, Seremetis S. Single 270 microg kg(-1)-dose rFVIIa vs standard 90 microg kg(-1)-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison. Haemophilia. 2008;14:287-294.
22. Obergfell A, Auvinen MK, Mathew P. Recombinant activated factor VII for haemophilia patients with inhibitors undergoing orthopaedic surgery: a review of the literature. Haemophilia. 2008;14: 233-241.
23. Solimeno LP, Perfetto OS, Pasta G, Santagostino E. Total joint replacement in patients with inhibitors. Haemophilia. 2006;12 (Suppl 3):113-116.
24. Rodriguez-Merchan EC, Wiedel JD, Wallny T, et al. Elective orthopaedic surgery for inhibitor patients. Haemophilia. 2003;9:625-631.
25. 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: 22-26.
26. Goldstein B, Geldziler B, Bjerre J, Seremetis S. Evidence-based use of recombinant FVIIa (NovoSeven, NiaStase) for the treatment of hemophilia with inhibitors in children and adolescents. Transfus Apher Sci. 2008;38:25-32.
27. Hotchkiss RS, Tinsley KW, Swanson PE, Karl IE. Endothelial cell apoptosis in sepsis. Crit Care Med. 2002;30:S225-S228
28. Doshi SN, Marmur JD. Evolving role of tissue factor and its pathway inhibitor. Crit Care Med. 2002;30:S241-S250.
29. Ludlam CA, Smith MP, Morfini M, et al. A prospective study of recombinant activated factor VII administered by continuous infusion to inhibitor patients undergoing elective major orthopaedic surgery: a pharmacokinetic and efficacy evaluation. Br J Haematol. 2003;120:808-813.
30. Santagostino E, Morfini M, Rocino A, et al. Relationship between factor VII activity and clinical efficacy of recombinant factor VIIa given by continuous infusion to patients with factor VIII inhibitors. Thromb Haemost. 2001;86:954-958.
31. Smith MP, Ludlam CA, Collins PW, et al. Elective surgery on factor VIII inhibitor patients using continuous infusion of recombinant activated factor VII: plasma factor VII activity of 10 IU/mL is associated with an increased incidence of bleeding. Thromb Haemost. 2001;86:949-953.
32. Negrier C, Goudemand J, Sultan Y, et al. Multicenter retrospective study on the utilization of FEIBA in France in patients with factor VIII and factor IX inhibitors. French FEIBA Study Group. Factor eight bypassing activity. Thromb Haemost. 1997;77:1113-1119.
33. Dimichele D, Negrier C. A retrospective postlicensure survey of FEIBA efficacy and safety. Haemophilia. 2006;12:352-362.
34. Smejkal P, Brabec P, Matyskova M, et al. FEIBA in treatment of acute bleeding episodes in patients with haemophilia A and factor VIII inhibitors: a retrospective survey in regional haemophilia centre. Haemophilia. 2009;15:743-751.
35. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs.The Orthopaedic Outcome Study Group. J Intern Med. 1994;236:391-399.
36. Szucs TD, Offner A, Kroner B, et al. Resource utilisation in haemophiliacs treated in Europe: results from the European study on socioeconomic aspects of haemophilia care. The European Socioeconomic Study Group. Haemophilia. 1998;4:498-501.
37. Cooper HA, Jones CP, Campion E, et al. Rationale for the use of high dose rFVIIa in a high-titre inhibitor patient with haemophilia B during major orthopaedic procedures. Haemophilia. 2001;7:517-522.
38. Young G, McDaniel M, Nugent DJ. Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors. Haemophilia. 2005;11:203-207.
39. Morfini M, Auerswald G, Kobelt RA, et al. Prophylactic treatment of haemophilia patients with inhibitors: clinical experience with recombinant factor VIIa in European haemophilia centres. Haemophilia. 2007;13:502-507.
40. Aledort LM. Comparative thrombotic event incidence after infusion of recombinant factor VIIa versus factor VIII inhibitor bypass activity. J Thromb Haemost. 2004;2:1700-1708.
41. O'Connell NM, Riddell AF, Pascoe G, et al. Recombinant factor VIIa to prevent surgical bleeding in factor XI deficiency. Haemophilia. 2008;14:775-781.
42. 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.
43. 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.
44. Franchini M. The use of recombinant activated factor VII in platelet disorders: a critical review of the literature. Blood Transfus. 2009;7:24-28.
45. Davie EW, Ratnoff OD. Waterfall sequence for intrinsic blood clotting. Science. 1964;145:1310-1312.
46. MacFarlane RG. An enzyme cascade in the blood clotting mechanism, and its function as a biochemical amplifier. Nature. 1964;202:498-499.
47. Aoki N, Wall MJ, Demsar J, et al. Predictive model for survival at the conclusion of a damage control laparotomy. Am J Surg. 2000; 180:540-544.
48. Brohi K, Singh J, Heron M, Coats T. Acute traumatic coagulopathy. J Trauma. 2003;54:1127-1130.
49. MacLeod JB, Lynn M, McKenney MG, et al. Early coagulopathy predicts mortality in trauma. J Trauma. 2003;55:39-44.
50. Martini WZ, Cortez DS, Dubick MA, et al. Thrombelastography is better than PT, aPTT, and activated clotting time in detecting clinically relevant clotting abnormalities after hypothermia, hemorrhagic shock and resuscitation in pigs. J Trauma. 2008;65:535-543.
51. Plotkin AJ, Wade CE, Jenkins DH, et al. A reduction in clot formation rate and strength assessed by thrombelastography is indicative of transfusion requirements in patients with penetrating injuries. J Trauma. 2008;64(Suppl 2):S64-S68.
52. Gravlee GP, Arora S, Lavender SW, et al. Predictive value of blood clotting tests in cardiac surgical patients. Ann Thorac Surg. 1994; 58:216-221.
53. Segal JB, Dzik WH. Paucity of studies to support that abnormal coagulation test results predict bleeding in the setting of invasive procedures: an evidence-based review. Transfusion. 2005;45:1413-1425.
54. Murray D, Pennell B, Olson J. Variability of prothrombin time and activated partial thromboplastin time in the diagnosis of increased surgical bleeding. Transfusion. 1999;39:56-62.
55. Shapiro S, Sherwin B, Gordimer H. Postoperative thromboembolization: the platelet count and the prothrombin time after surgical operations: a simple method for detecting reductions and elevations of the prothrombin concentration (or activity) of the blood plasma. Ann Surg. 1942;116:175-183.
56. Proctor RR, Rapaport SI. The partial thromboplastin time with kaolin. A simple screening test for first stage plasma clotting factor deficiencies. Am J Clin Pathol. 1961;36:212-219.
57. Monroe DM, Roberts HR, Hoffman M. Platelet procoagulant complex assembly in a tissue factor-initiated system. Br J Haematol. 1994;88:364-371.
58. Monroe DM, Hoffman M. What does it take to make the perfect clot? Arterioscler Thromb Vasc Biol. 2006;26:41-48
59. Wolberg AS. Thrombin generation and fibrin clot structure. Blood Rev. 2007;21:131-142.
60. Kang YG, Martin DJ, Marquez J, et al. Intraoperative changes in blood coagulation and thrombelastographic monitoring in liver transplantation. Anesth Analg. 1985;64:888-896.
61. McNicol PL, Liu G, Harley ID, et al. Patterns of coagulopathy during liver transplantation: experience with the first 75 cases using thrombelastography. Anaesth Intensive Care. 1994;22:659-665.
62. Spiess BD, Gillies BS, Chandler W, Verrier E. Changes in transfusion therapy and reexploration rate after institution of a blood management program in cardiac surgical patients. J Cardiothorac Vasc Anesth. 1995;9:168-173.
63. Shore-Lesserson L, Manspeizer HE, Deper io M, et al. Thromboelastography-guided transfusion algorithm reduces transfusions in complex cardiac surgery. Anesth Analg. 1999;88:312-319.
64. Manikappa S, Mehta Y, Juneja R, Trehan N. Changes in transfusion therapy guided by thromboelastograph in cardiac surgery. Ann Card Anaesth. 2001;4:21-27.
65. Anderson L, Quasim I, Soutar R, et al. An audit of red cell and blood product use after the institution of thromboelastometry in a cardiac intensive care unit. Transfus Med. 2006;16:31-39.
66. Welsby IJ, Jiao K, Ortel TL, et al. The kaolin-activated thrombelastograph predicts bleeding after cardiac surgery. J Cardiothorac Vasc Anesth. 2006;20:531-535.
67. Kaufmann CR, Dwyer KM, Crews JD, et al. Usefulness of thrombelastography in assessment of trauma patient coagulation. J Trauma. 1997;42:716-720.
68. Johansson PI. Treatment of massively bleeding patients: introducing real-time monitoring, transfusion packages and thrombelastography (TEGR). ISBT Sci Ser. 2007;2:159-167.
69. Johansson PI, Bochsen L, Stensballe J, Secher NH. Transfusion packages for massively bleeding patients: the effect on clot formation and stability as evaluated by Thrombelastograph (TEG(R)). Transfus Apher Sci. 2008;39:3-8.
70. Johansson PI, Stensballe J. Effect of haemostatic control resuscitation on mortality in massively bleeding patients: a before and after study. Vox Sang. 2009;96:111-118.
71. Johansson PI, Stissing T, Bochsen L, Ostrowski SR. Thrombelastography and tromboelastometry in assessing coagulopathy in trauma. Scand J Trauma Resusc Emerg Med. 2009;17:45.
72. Rivard GE, Brummel-Ziedins KE, Mann KG, et al. Evaluation of the profile of thrombin generation during the process of whole blood clotting as assessed by thrombelastography. J Thromb Haemost. 2005;3:2039-2043.
73. Yoshioka A, Nishio K, Shima M. Thrombelastgram as a hemostatic monitor during recombinant factor VIIa treatment in hemophilia A patients with inhibitor to factor VIII. Haemostasis. 1996;26:139-142.
74. Sorensen B, Ingerslev J. Tailoring haemostatic treatment to patient requirements - an update on monitoring haemostatic response using thrombelastography. Haemophilia. 2005;11(Suppl 1):1-6.
75. Bassus S, Wegert W, Krause M, et al. Platelet-dependent coagulation assays for factor VIII efficacy measurement after substitution therapy in patients with haemophilia A. Platelets. 2006;17:378-384.
76. Trowbridge CC, Stammers AH, Ciccarelli N, Klayman M. Dose titration of recombinant factor VIIa using thromboelastograph monitoring in a child with hemophilia and high titer inhibitors to factor VIII: a case report and brief review. J Extra Corpor Technol. 2006;38:254-259.