Experience with recombinant-activated factor VII in 30 patients with congenital factor VII deficiency

Hematology

Volumn 12, Issue 1, 2007, Pages 55-62

  Brenner, Benjamin ^a   Wiis, Jørgen ^b  

^a RAMBAM MEDICAL CENTER   (Israel)

^b Rigshospitalet   (Denmark)

Author keywords

Congenital factor VII deficiency; Haemorrhage; Recombinant activated factor VII; Replacement therapy; rFVIIa

Indexed keywords

4 AMINOBENZOIC ACID; 4 AMINOMETHYLBENZOIC ACID; ANTIBIOTIC AGENT; ETAMSYLATE; FIBRIN GLUE; GLUCOCORTICOID; HYPERTENSIVE AGENT; LOW MOLECULAR WEIGHT HEPARIN; RECOMBINANT BLOOD CLOTTING FACTOR 7A; TRANEXAMIC ACID;

ADOLESCENT; ADULT; ARTICLE; BLEEDING; BLOOD CLOTTING FACTOR 7 DEFICIENCY; CHILD; CHILDBIRTH; CLINICAL ARTICLE; CLINICAL DATA REPOSITORY; DISSEMINATED INTRAVASCULAR CLOTTING; DRUG EFFICACY; DRUG INDICATION; DRUG SAFETY; ELECTIVE SURGERY; EMERGENCY SURGERY; EPISTAXIS; EUROPEAN UNION; FEMALE; HEMARTHROSIS; HEMATOMA; HEMATURIA; HEMOSTASIS; HUMAN; INFANT; INTERNATIONAL NORMALIZED RATIO; INTERNET; MALE; MENORRHAGIA; PRIORITY JOURNAL; PROPHYLAXIS; SUTURE REMOVAL;

ADOLESCENT; ADULT; AGED; ANTIFIBRINOLYTIC AGENTS; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; FACTOR VII; FACTOR VII DEFICIENCY; FEMALE; HEMARTHROSIS; HEMATOMA; HEMORRHAGE; HEMOSTATICS; HUMANS; INFANT; INFANT, NEWBORN; INFANT, PREMATURE; INFANT, PREMATURE, DISEASES; MALE; MIDDLE AGED; POSTOPERATIVE HEMORRHAGE; RECOMBINANT PROTEINS; REGISTRIES; RETROSPECTIVE STUDIES; THROMBOSIS; TREATMENT OUTCOME; WOUNDS AND INJURIES;

EID: 33947155799     PISSN: 10245332     EISSN: 16078454     Source Type: Journal    
DOI: 10.1080/10245330601111573     Document Type: Article

References (49)

1. Hunault, M. and Bauer, KA (2000) Recombinant factor VIIa for the treatment of congenital factor VII deficiency. Semin Thromb Hemost, 26, pp. 401-405.
2. Hoffman, M. and Monroe 3rd, DM (2001) A cell-based model of hemostasis. Thromb Haemost, 85, pp. 958-965.
3. Cooper, DN, Millar, DS, Wacey, A., Banner, DW and Tuddenham, EG (1997) Inherited factor VII deficiency: Molecular genetics and pathophysiology. Thromb Haemost, 78, pp. 151-160.
4. Herrmann, FH, Wulff, K. Auberger, K. Aumann, V. Bergmann, F., Bergmann, K. Bratanoff, E. Franke, D. Grundeis, M. Kreuz, W. Lenk, H. Losonczy, H. Maak, B. Marx, G. Mauz-Korholz, C. Pollmann, H. Serban, M. Sutor, A. Syrbe, G. Vogel, G. Weinstock, N. Wenzel, E. and Wolf, K. (2000) Molecular biology and clinical manifestation of hereditary factor VII deficiency. Semin Thromb Hemost, 26, pp. 393-400.
5. Mariani G., Herrmann, FH, Dolce, A., Batorova, A. Etro, D., Peyvandi, F. Wulff, K. Schved, JF, Auerswald, G. Ingerslev, J. and Bernardi, F. (2005) International factor VII deficiency study group. Clinical phenotypes and factor VII genotype in congenital factor VII deficiency. Thromb Haemost, 93, pp. 481-487.
6. Bolton-Maggs, PH, Perry, DJ, Chalmers, EA, Parapia, LA, Wilde, JT, Williams, MD, Collins, PW, Kitchen, S., Dolan, G. and Mumford, AD (2004) The rare coagulation disorders-review with guidelines for management from the United Kingdom Haemophilia Centre Doctors' Organisation. Haemophilia, 10, pp. 593-628.
7. Peyvandi, F., Mannucci, PM, Asti, D., Abdoullahi, M., Rocco, N Di and Sharifian, R. (1997) Clinical manifestations in 28 Italian and Iranian patients with severe factor VII deficiency. Haemophilia, 3, pp. 242-246.
8. Peyvandi, F. and Mannucci, PM (1999) Rare coagulation disorders. Thromb Haemost, 82, pp. 1207-1214.
9. Roberts, HR and Lefkowitz, JB (1994) Inherited disorders of prothrombin conversion, pp. 206-208. Lippincott JB, Philadelphia, USA
10. Mariani, G., Testa, MG, Paolantonio, T Di, Bech, R Molskov and Hedner, U. (1999) Use of recombinant, activated factor VII in the treatment of congenital factor VII deficiencies. Vox Sanguinis, 77, pp. 131-136.
11. Ragni, MV, Lewis, JH, Spero, JA and Hasiba, U. (1981) Factor VII deficiency. Am J Hematol, 10, pp. 79-88.
12. Acharya, SS, Coughlin, A. and Dimichele, DM (2004) Rare bleeding disorder registry: Deficiencies of factors II, V, VII, X, XIII, fibrinogen and dysfibrinogenemias. J Thromb Haemost, 2, pp. 248-256.
13. Ingerslev, J. Knudsen, L. Hvid, I. Tange, MR Fredberg, U. and Sneppen, O. (1997) Use of recombinant factor VIIa in surgery in factor-VII-deficient patients. Haemophilia, 3, pp. 215-218.
14. Paola, J Di, Nugent, D. and Young, G. (2001) Current therapy for rare factor deficiencies. Haemophilia, 7, pp. 16-22.
15. Mannucci, PM, Duga, S. and Peyvandi, F. (2004) Recessively inherited coagulation disorders. Blood, 104, pp. 1243-1252.
16. Ingerslev, J. and Kristensen, HL (1998) Clinical picture and treatment strategies in factor VII deficiency. Haemophilia, 4, pp. 689-696.
17. Kohler, M. Hellstern, P. Pindur, G. Wenzel, E. and von Blohn, G. (1989) Factor VII half-life after transfusion of a steam-treated prothrombin complex concentrate in a patient with homozygous factor VII deficiency. Vox Sanguinis, 56, pp. 200-201.
18. White, GC, Lundblad, RL and Kingdon, HS (1979) Prothrombin complex concentrates: Preparation, properties, and clinical uses. Curr Topics Hematol, 2, pp. 203-244.
19. United Kingdom Haemophilia Centre Doctors' Organisation (2003) Guidelines on the selection and use of therapeutic products to treat haemophilia and other hereditary bleeding disorders. Haemophilia, 9, pp. 1-23.
20. Scharrer, I. (1999) Recombinant factor VIIa for patients with inhibitors to factor VIII or IX or factor VII deficiency. Haemophilia, 5, pp. 253-259.
21. Almeida, AM, Khair, K., Hann, I. and Liesner, R. (2003) The use of recombinant factor VIIa in children with inherited platelet function disorders. Br J Haematol, 121, pp. 477-481.
22. Chuansumrit, A., Sangkapreecha, C. and Hathirat, P. (1999) Successful epistaxis control in a patient with Glanzmann thrombasthenia by increased bolus injection dose of recombinant factor VIIa. Thromb Haemost, 82, p. 1778.
23. Poon, MC, Demers, C., Jobin, F. and Wu, JW (1999) Recombinant factor VIIa is effective for bleeding and surgery in patients with Glanzmann thrombasthenia. Blood, 94, pp. 3951-3953.
24. Poon, MC, D'Oiron, R., Von Depka, M., Khair, K., Negrier, C., Karafoulidou, A., Huth-Kuehne, A. and Morfini, M. (2004) International data collection on recombinant factor VIIa and congenital platelet disorders study group. Prophylactic and therapeutic recombinant factor VIIa administration to patients with Glanzmann's thrombasthenia: Results of an international survey. J Thromb Haemost, 2, pp. 1096-1103.
25. Tengborn, L. and Petruson, B. (1996) A patient with Glanzmann thrombasthenia and epistaxis successfully treated with recombinant factor VIIa. Thromb Haemost, 75, pp. 981-982.
26. Ciavarella, N., Schiavoni, M., Valenzano, E., Mangini, F. and Inchingolo, F. (1996) Use of recombinant factor VIIa (NovoSeven) in the treatment of two patients with type III von Willebrand's disease and an inhibitor against von Willebrand factor. Haemostasis, 26, pp. 150-154.
27. Grossmann, RE, Geisen, U., Schwender, S. and Keller, F. (2000) Continuous infusion of recombinant factor VIIa (NovoSeven) in the treatment of a patient with type III von Willebrand's disease and alloantibodies against von Willebrand factor. Thromb Haemost, 83, pp. 633-634.
28. Meijer, K., Peters, FT and van der Meer, J. (2001) Recurrent severe bleeding from gastrointestinal angiodysplasia in a patient with von Willebrand's disease, controlled with recombinant factor VIIa. Blood Coag Fibrinol, 12, pp. 211-213.
29. Ozelo, MC, Svirin, P. and Larina, L. (2005) Use of recombinant factor VIIa in the management of severe bleeding episodes in patients with Bernard-Soulier syndrome. Ann Hematol
30. Peters, M. and Heijboer, H. (1998) Treatment of a patient with Bernard-Soulier syndrome and recurrent nosebleeds with recombinant factor VIIa. Thromb Haemost, 80, p. 352.
31. Poon, MC and d'Oiron, R. (2000) Recombinant activated factor VII (NovoSeven) treatment of platelet-related bleeding disorders. International registry on recombinant factor VIIa and congenital platelet disorders group. Blood Coag Fibrinol, 11, pp. S55-S68.
32. Billon, S. Niger, C Le, Escoffre-Barbe, M., Vicariot, M. and Abgrall, JF (2001) The use of recombinant factor VIIa (NovoSeven) in a patient with factor XI deficiency and a circulating anticoagulant. Blood Coag Fibrinol, 12, pp. 551-553.
33. Lawler, P., White, B., Pye, S., Hermans, C., Riddell, A., Costello, C., Brown, S. and Lee, CA (2002) Successful use of recombinant factor VIIa in a patient with inhibitor secondary to severe factor XI deficiency. Haemophilia, 8, pp. 145-148.
34. Billio, A., Pescosta, N., Rosanelli, C., Amaddii, G., Fontanellia, F. and Coser, P. (1997) Successful short-term oral surgery prophylaxis with rFVIIa in severe congenital factor VII deficiency. Blood Coag Fibrinol, 8, pp. 249-250.
35. Chuansumrit, A., Visanuyothin, N., Puapunwattana, S., Chaivisuth, A., Rasmidat, P., Charoenkwan, P. and Chiemchanya, S. (2002) Outcome of intracranial hemorrhage in infants with congenital factor VII deficiency. J Med Assoc Thailand, 85, pp. S1059-S1064.
36. Eskandari, N., Feldman, N. and Greenspoon, JS (2002) Factor VII deficiency in pregnancy treated with recombinant factor VIIa. Obst Gynecol, 99, pp. 935-937.
37. Huang, WY, Kruskall, MS, Bauer, KA, Uhl, L. and Shaz, BH (2004) The use of recombinant activated factor VII in three patients with central nervous system hemorrhages associated with factor VII deficiency. Transfusion, 44, pp. 1562-1566.
38. Maimon, M., Bernstein, T., Kenet, G. and Kapelushnik, J. (2003) Recombinant factor VIIa for treatment of a child with severe factor VII deficiency and coarctation of the aorta. J Pediat Hematol/Oncol, 25, p. 591.
39. Mathijssen, NC, Masereeuw, R., Verbeek, K., Lavergne, JM, Costa, JM, van Heerde, WL and Novakova, IR (2004) Prophylactic effect of recombinant factor VIIa in factor VII deficient patients. Br J Haematol, 125, pp. 494-499.
40. Niikura, T., Nishikawa, T., Saegusa, Y., Fujishiro, T., Yoshiya, S. and Kurosaka, M. (2005) Total hip arthroplasty in severe congenital factor VII deficiency: Successful use of recombinant activated factor VII for hemostasis. J Arthroplast, 20, pp. 396-400.
41. Tcheng, WY, Donkin, J., Konzal, S. and Wong, WY (2004) Recombinant factor VIIa prophylaxis in a patient with severe congenital factor VII deficiency. Haemophilia, 10, pp. 295-298.
42. White, B., Martin, M., Kelleher, S., Browne, P., McCann, SR and Smith, OP (1999) Successful use of recombinant FVIIa (NovoSeven) in the management of pulmonary haemorrhage secondary to Aspergillus infection in a patient with leukaemia and acquired FVII deficiency. Br J Haematol, 106, pp. 254-255.
43. White, B., O'Connor, H. and Smith, OP (2000) Successful use of recombinant VIIa (NovoSeven) and endometrial ablation in a patient with intractable menorrhagia secondary to FVII deficiency. Blood Coag Fibrinol, 11, pp. 155-157.
44. Mariani, G., Konkle, BA and Ingerslev, J. (2006) Congenital factor VII deficiency: Therapy with recombinant activated factor VII-a critical appraisal. Haemophilia, 12, pp. 19-27.
45. Kessler, C. (2004) Haemostasis.com: Clinical experiences in the investigational use of rFVIIa in the management of severe haemorrhage. Br J Haematol, 127, p. 230.
46. Ziedins, K Brummel, Rivard, GE, Pouliot, RL, Butanas, S., Gissel, M., Parhami-Seren, B. and Mann, KG (2004) Factor VIIa replacement therapy in factor VII deficiency. J Thromb Haemost, 2, pp. 1735-1744.
47. Mariani, G. and Mazzucconi, MG (1983) Factor VII congenital deficiency. Clinical picture and classification of the variants. Haemostasis, 13, pp. 169-177.
48. Abshire, T. and Kenet, G. (2004) Recombinant factor VIIa: Review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors. J Thromb Haemost, 2:6, pp. 899-909.
49. Roberts, HR (2001) Recombinant factor VIIa (NovoSeven®) and the safety of treatment. Semin Haematol, 38, pp. 48-50.