Clinical experience with recombinant factor VIIa

Blood Coagulation and Fibrinolysis

Volumn 9, Issue 2, 1998, Pages 119-128

  Lusher, J ^a,b   Ingerslev, J ^a,b   Roberts, H ^a,b,c,d  

^a WAYNE STATE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b AARHUS UNIVERSITY HOSPITAL   (Denmark)

^c UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^d NOVO NORDISK A S   (Denmark)

Author keywords

Haemophilia; Inhibitors; Recombinant factor VIIa

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8;

DRUG EFFICACY; DRUG SAFETY; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; PRIORITY JOURNAL; RETROPERITONEAL HEMORRHAGE; REVIEW;

EID: 0031954881     PISSN: 09575235     EISSN: None     Source Type: Journal    
DOI: 10.1097/00001721-199803000-00002     Document Type: Review

References (58)

1. Shapiro SS, Hultin M. Acquired inhibitors to the blood coagulation factors. Semin Thromb Haemost 1975; 1: 336-385.
2. Addiego J, Kasper C, Abildgaard C, Hilgartner M, Lusher J, Glader B, et al. Frequency of inhibitor development in haemophiliacs treated with low purity factor VIII. Lancet 1993; 342: 462-464.
3. Ehrenforth S, Kreuz W, Scharrer I, Linde R, Funk M, Güngör T, et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet 1992; 339: 594-598.
4. Lusher JM, Arkin S, Abildgaard CF, Schwanz RS. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia. New Engl JMed 1993; 328: 453-459.
5. Katz J. Prevalence of factor IX inhibitors among patients with haemophilia B -results of a large-scale North American survey. Haemophilia 1996; 2: 28-31.
6. Hay CRM, Colvin BT, Ludlam CA, Hill FGH, Preston FE. Recommendations for the treatment of factor VIII inhibitors: from the UK Haemophilia Centre Directors' Organisation Inhibitor Working Party. Blood Coag Fibrinol 1996; 7: 134-138.
7. Green D, Lechner K. A survey of 214 non-haemophiliac patients with inhibitors to factor VIII. Thromb Haemost 1981; 45: 200-203.
8. Margolius A, Jackson DP, Ratnoff OD. Circulating anticoagulants: a study of 40 cases and review of the literature. Medicine 1961; 40: 145-202.
9. Nilsson IM, Berntorp E, Zettervall O. Induction of immune tolerance in patients with haemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide and factor VIII. N Engl] Med 1988; 318: 947-950.
10. Brackmann HH. The treatment of inhibitors against factor VIII by continuous treatment with factor VIII and activated prothrombin complex concentrates. In: Mariani G, Russo MA, Mandelli F, (editors): Activated prothrombin complex concentrates. Managing hemophilia with factor VIII inhibitor. New York: Praeger 1982; pp. 194-205.
11. Wensley RT, Burn AM, Reading OM. Induction of tolerance to factor VIII in haemophilia A patients with inhibitors using low doses of human factor VIII. Thromb Haemost 1985; 54: 227.
12. van Leeuwen EF, Mauser-Bunschoten EP, van Dijken PJ, Kok AJ, Sjamsoedin-Visser EJ, Sixma JJ. Disappearance of factor VIII:C antibodies in patients with haemophilia A upon frequent administration of factor VIII in intermediate or low dose. BrJ Haematol 1986; 64:291-297.
13. Aznar JA, Jorqueeraa JL, Peiro A. Suppression of inhibitors in haemophilia with corticoids and factor VIII. Thromb Haemost 1983; 49: 241-243.
14. Mauser-Bunschoten EP, Niewenhuis HK, Roosendaal G, van den Berg HM. Low-dose immune tolerance induction in hemophilia A patients with inhibitors. Blood 1995; 86: 983-988.
15. Mariani G, Ghirardini A, Belloco R. Immune tolerance in haemophilia. Principal results from the international registry. Report of the factor VIII and IX Subcommittee. Thromb Haemost 1994; 72: 155-158.
16. Brettler DB, Shopnick R, Bolivar E, Dumas B. The price of immune tolerance program for factor VIII inhibitors in the New England area, are they worth it? MW1994; 84: 201.
17. Kernoff PB A, Thomas ND, Lilley PA, Matthews KB, Goldman E, Tuddenham EGD. Clinical experience with polyelectrolyte-fractionated porcine factor VIII concentrate in the treatment of hemophiliacs with antibodies to factor VIII. Blood 1984; 63: 31-41.
18. Gringeri A, Santagostino E, Tradati F, Giangrande PLF, Mannucci PM. Adverse effects of treatment with porcine FVIII. Thromb Haemost 1991; 65: 245-247.
19. Lusher JM, Blatt PM, Penner JA, Aledort LM, Levine PH, White GC, et al. Autoplex versus Proplex: a controlled, double-blind study of effectiveness in acute hemarthroses in hemophiliacs with inhibitors to factor VIII. Blood 1983; 62: 1135-1138.
20. Lusher JM, Shapiro SS, Palascak JE, Rao AV, Levine PH, Blatt PM, the Hemophilia Study Group. Efficacy of prothrombin-complex concentrates in haemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. N EnglJ Med 1980; 303: 421-425.
21. Sjamsoedin LJM, Heijnen L, Mauser-Bunschoten EP, van Geijlswijk JL, van Houwelingen H, van Asten P, et al. The effect of activated prothrombin complex concentrate (FEIBA) on joint and muscle bleeding in patients with haemophilia A and antibodies to factor VIII. N EnglJ Med 1981; 305: 717-758.
22. Abildgaard CF. Hazards of prothrombin complex concentrates in the treatment of haemophilia. New Engl J Med 1981; 304: 670-671.
23. Campbell EW, Neff S, Browdler AJ. Therapy with factor IX concentrate resulting in DIG and thromboembolic phenomena. Transfusion 1978; 18: 94-97.
24. Lusher JM. Thrombogenicity associated with factor IX complex concentrates. Semin Hematol 1991; 28: 3-5.
25. Schimpf K, Zeltsch C, Zeltsch P. Myocardial infarction complicating activated prothrombin complex concentrate substitution in a patient with haemophilia A. Lancet 1982; ii: 1043.
26. Chavin SL, Siegel DM, Rocco TA Jr, Oison JP. Acute myocardial infarction during treatment with an activated prothrombin complex concentrate in a patient with factor VIII deficiency and a factor VIII inhibitor. Am J Med 1988; 85: 245-249.
27. Ohga S, Saito M, Matsukazi A, Kai T, Ueda K. Disseminated intravascular coagulation in a patient with haemophilia B during factor IX replacement therapy. BrJ Haematol 1993; 84: 343-345.
28. Rao LVM, Rapaport SI. Activation of factor VII bound to tissue factor: a key early step in the tissue factor pathway of coagulation. Proc Natl Acad Sei 1988; 85: 6687-6691.
29. Nemerson Y, Repke D. Tissue factor accelerates the activation of coagulation factor VII: the role of a bifunctional coagulation cofactor. Tbromb Res 1985; 49: 351-358.
30. Rapaport SI, Rao LVM. The tissue factor pathway: how it has become a "prima ballerina". Tbromb Haemost 1995; 74: 7-17.
31. Rapaport SI, Rao LVM. Initiation and regulation of tissue factor-dependent blood coagulation. Arterioscler Thromb 1992; 10: 1111-1121.
32. Rao LV, Rapaport SI. Factor Vila-catalysed activation of factor X independent of tissue factor. Its possible significance for control of hémophilie bleeding by infused factor Vila. Blood 1990; 75: 1069-1073.
33. Hoffman M, Monroe DM, Roberts HR. Human monocytes support factor X activation by factor Vila, independent of tissue factor: implications for the therapeutic mechanism of high dose factor Vila in hemophilia. Blood 1994; 83: 38-42.
34. ten Gate H, Bauer KA, Lev! M, Edington TS, Sublett RD, Barzegar S, et al. The activation of factor X and prothrombin by recombinant factor Vila in vivo is mediated by tissue factor. / Clin Invest 1993; 92: 1207-1212.
35. Weiss HJ, Turitto VT, Baumgartner HR, Nemerson Y, Hoffmann T. Evidence for the presence of tissue factor activity on subendothelium. Blood 1989; 73: 968-975.
36. Drake TA, Morrissey JH, Edgington TS. Selective cellular expression of tissue factor in human tissues: implications for disorders of hemostasis and thrombosis. AmJPathol 1989; 134: 1087-1097.
37. Fleck RA, Rao LVM, Rapaport SI, Varki N. Localization of human tissue factor antigen by immunostaining with monospecific, polyclonal anti-human tissue factor antibody. Thromb Res 1990; 57: 765-781.
38. Callander NS, Varki N, Rao LVM. Immunohistochemical identification of tissue factor in solid tumours. Cancer 1992; 670: 1192-1201.
39. Wilcox JN, Smith KM, Schwanz SM, Gordon D. Localization of tissue factor in the normal vessel wall and in the atherosclerotic plaque. Proc Natl Acad Set 1989; 89: 2839-2843.
40. Diness V, Bregengaard C, Erhardtsen E, Hedner U. Recombinant human factor Vila (rFVIIa) in a rabbit stasis model. Thromb Res 1992; 67: 233-241.
41. Diness V. rFVIIa in an endotoxin model and in a Wessler model. In: Hedner U, Roberts HR, (editor): New aspects of haemophilia treatment. Bussum, The Netherlands: Medicom Europe: 1991; pp. 147-150.
42. Hedner U, Schulman S, Alberts KA, Blombäck M, Johnsson H, Glazer S, et al. Successful use of rFVIIa in a patient with severe haemophilia A subjected to synovectomy. Lancet 1988; ii: 1193.
43. Lusher JM, Roberts HR, Davignon G, Joist JH, Smith H, Shapiro A, et al. A randomised double-blind comparison of two dosage levels of recombinant factor Vila in the treatment of joint, muscle and mucocutaneous haemorrhages in persons with haemophilia A and B, with and without inhibitors. Haemophilia, in press.
44. Gringeri A, Santagostino E, Mannucci PM. Failure of recombinant activated factor VII during surgery in a hemophiliac with high-titer factor VIII antibody. Haemostasis 1991; 21: 1-4.
45. Ingerslev J, Freidman D, Gastineau D, Gilchrist G, Johnsson H, Lucas G, et al. Major surgery in haemophilic patients with inhibitors using recombinant factor Vila. Haemostasis 1996; 26 (suppl 1): 118-1123.
46. Glazer S, Hedncr U, Falch FJ. Clinical update on the use of recombinant factor Vila. In: Aledort LM, White GC, (editors): Proc 2nd International Symposium on Inhibitors to Coagulation Factors. New York: Plenum Publishing Corporation, 1993.
47. Stein SF, Duncan A, Cutler D, Glazer S. Disseminated intravascular coagulation (DIG) in a haemophiliac treated with recombinant factor Vila. Abstract presented at the American Society of Hemotology 32nd Annual Meeting, Boston, MA, 1990.
48. Molskov-Bech R, Nicolaisen EM, Andersen PM, Glazer S, Hedner U. Recombinant factor Vila for the treatment of congenital factor VII deficiency [abstract]. Thromb Haemost 1995; 73: 983.
49. Hedner U, Glazer S. Management of hemophilia patients with inhibitors. Hematol Oncol Clin North Am 1992; 6: 1035-1046.
50. Lund-Hansen T. NovoSeven -virus safety. Haemostasis 1996; 26 (suppl 1): 96-97.
51. Nicolaisen EM, Hansen LL, Poulsen F, Glazer S, Hedner U. Immunological aspects of recombinant factor Vila (rFVIIa) in clinical use. Thromb Haemost 1996; 76: 200-204.
52. Mariani G, Arcieri P, Testa MG, Hedner U for the NovoSeven Study Group. Clinical use of recombinant activated factor Vll in congenital factor VII deficiency [abstract]. Haemophilia 1996; 2 (suppl): 63.
53. Brinkhous KM, Hedner U, Garris JB, Diness V, Read M. Effect of recombinant factor Vila on the hemostatic defect in dogs with hemophilia A, hemophilia B and von Willebrand disease. Proc Natl Acad Sei USA 1989; 86: 1382-1386.
54. Lindley CM, Sawyer WT, Macik BG, Lusher J, Harrison JF, Baird-Cox K, et al. Pharmacokinetics and pharmacodynamics of recombinant factor Vila. Clin Pbarmacol Tber 1994; 55: 638-648.
55. Hedner U. Dosing and monitoring NovoSeven treatment. Haemostasis 1996; 26 (suppl 1): 102-108.
56. Morrissey JH, Macik BG, Neuenschwander PF, Comp PC. Quantitation of activated factor VII levels in plasma using a tissue factor mutant selectively deficient in promoting factor VII activation. Blood 1993; 81: 734-744.
57. Wildgoose P, Nemerson Y, Hausen LL, Nielsen FE, Glazer S, Hedner U. Measurement of basal levels of factor Vila in hemophilia A and B patients. Blood 1992; 80: 25-28.
58. Hansen L, Nielsen FE, Hedner U. Validation of method for determination of recombinant FVIIa coagulant activity in plasma using a one-stage clotting assay [abstract]. Tbromb Haemost 1993; 69: 1008.