Safety update on the use of recombinant activated factor VII in approved indications

Blood Reviews

Volumn 29, Issue S1, 2015, Pages S34-S41

  Neufeld, Ellis J ^a   Négrier, Claude ^b   Arkhammar, Per ^c   Benchikh el Fegoun, Soraya ^d   Simonsen, Mette Duelund ^a   Rosholm, Anders ^a   Seremetis, Stephanie ^e  

^a DANA FARBER CANCER INSTITUTE   (United States)

^b UNIVERSITÉ LYON 1   (France)

^c NOVO NORDISK A S   (Denmark)

^d Novo Nordisk FemCare AG   (Switzerland)

^e NOVO NORDISK INC   (United States)

Author keywords

Inhibitors; NovoSevensup sup; Recombinant activated factor VII; Safety; Thrombotic events

Indexed keywords

NEUTRALIZING ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 7A; BLOOD CLOTTING FACTOR 7A; RECOMBINANT FVIIA; RECOMBINANT PROTEIN;

ACQUIRED HEMOPHILIA; ANTIBODY TITER; ARTERY THROMBOSIS; ARTICLE; BLOOD CLOTTING; BLOOD CLOTTING FACTOR 7 DEFICIENCY; CONGENITAL FACTOR VII DEFICIENCY; CONGENITAL HEMOPHILIA WITH INHIBITOR; DRUG INDICATION; DRUG SAFETY; GLANZMANN DISEASE; HEMOPHILIA; HUMAN; IMMUNOGENICITY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHASE 4 CLINICAL TRIAL (TOPIC); POSTMARKETING SURVEILLANCE; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SYSTEMATIC REVIEW; THROMBOSIS; VEIN THROMBOSIS; SAFETY;

FACTOR VIIA; HUMANS; RECOMBINANT PROTEINS; SAFETY;

EID: 84931429109     PISSN: 0268960X     EISSN: 15321681     Source Type: Journal    
DOI: 10.1016/S0268-960X(15)30006-0     Document Type: Article

References (81)

1. ® Summary of Product Characteristics [Accessed March 3, 2015]. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000074/WC500030873.pdf.
2. ® Product Monograph [Accessed April 8, 2015]. http://webprod5.hc-sc.gc.ca/dpd-bdpp/start-debuter.do?lang=eng.
3. ® RT Highlights of Prescribing Information [Accessed April 8, 2015]. http://www.fda.gov/downloads/.../ucm056954.pdf.
4. Nedergaard H, Vestergaard S, Jensen PT, Kristiansen MW, Jensen MB, Østergaard PB, et al. In vitro stability of lyophilized and reconstituted recombinant activated factor VII formulated for storage at room temperature. Clin Ther 2008, 30:1309-1315.
5. Bysted BV, Scharling B, Møller T, Hansen BL A randomized, double-blind trial demonstrating bioequivalence of the current recombinant activated factor VII formulation and a new robust 25 degrees C stable formulation. Haemophilia 2007, 13:527-532.
6. Shapiro AD, Hedner U Advances in bypassing agent therapy for hemophilia patients with inhibitors to close care gaps and improve outcomes. Ther Adv Drug Saf 2011, 2:213-225.
7. 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.
8. 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.
9. Shapiro AD, Neufeld EJ, Blanchette V, Salaj P, Gut RZ, Cooper DL Safety of recombinant activated factor VII (rFVIIa) in patients with congenital haemophilia with inhibitors: overall rFVIIa exposure and intervals following high (240 μg kgsup-1/sup) rFVIIa doses across clinical trials and registries. Haemophilia 2014, 20:e23-e31.
10. Hoffman M, Monroe DM A cell-based model of hemostasis. Thromb Haemost 2001, 85:958-965.
11. Jurlander B, Thim L, Klausen NK, Persson E, Kjalke M, Rexen P, et al. Recombinant activated factor VII (rFVIIa): characterization, manufacturing, and clinical development. Semin Thromb Hemost 2001, 27:373-384.
12. 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.
13. Monroe DM, Hoffman M, Oliver JA, Roberts HR A possible mechanism of action of activated factor VII independent of tissue factor. Blood Coagul Fibrinolysis 1998, 9 Suppl 1:S15-S20.
14. Monroe DM, Hoffman M What does it take to make the perfect clot?. Arterioscler Thromb Vasc Biol 2006, 26:41-48.
15. Diness V, Bregengaard C, Erhardtsen E, Hedner U Recombinant human factor VIIa (rFVIIa) in a rabbit stasis model. Thromb Res 1992, 67:233-241.
16. Gallistl S, Cvirn G, Muntean W Recombinant factor VIIa does not induce hypercoagulability in vitro. Thromb Haemost 1999, 81:245-249.
17. de Paula EV, Kavakli K, Mahlangu J, Ayob Y, Lentz SR, Morfini M, et al. Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial. J Thromb Haemost 2012, 10:81-89.
18. Lentz SR, Ehrenforth S, Karim FA, Matsushita T, Weldingh KN, Windyga J, et al. Recombinant factor VIIa analog in the management of hemophilia with inhibitors: results from a multicenter, randomized, controlled trial of vatreptacog alfa. J Thromb Haemost 2014, 12:1244-1253.
19. Morfini M, Bjerre J Pharmacokinetics and safety of a 270 mcg kg-1 dose of room temperature stable recombinant activated factor VII in patients with haemophilia. Haemophilia 2011, 17:860-866.
20. Ljung R, Karim FA, Saxena K, Suzuki T, Arkhammar P, Rosholm A, et al. 40K glycoPEGylated, recombinant FVIIa: 3-month, double-blind, randomized trial of safety, pharmacokinetics and preliminary efficacy in hemophilia patients with inhibitors. J Thromb Haemost 2013, 11:1260-1268.
21. Lijfering WM, Flinterman LE, Vandenbroucke JP, Rosendaal FR, Cannegieter SC Relationship between venous and arterial thrombosis: a review of the literature from a causal perspective. Semin Thromb Hemost 2011, 37:885-896.
22. Lowe GD Common risk factors for both arterial and venous thrombosis. Br J Haematol 2008, 140:488-495.
23. Previtali E, Bucciarelli P, Passamonti SM, Martinelli I Risk factors for venous and arterial thrombosis. Blood Transfus 2011, 9:120-138.
24. Badimon L, Vilahur G Thrombosis formation on atherosclerotic lesions and plaque rupture. J Intern Med 2014, 276:618-632.
25. Bentzon JF, Otsuka F, Virmani R, Falk E Mechanisms of plaque formation and rupture. Circ Res 2014, 114:1852-1866.
26. Nowak-Göttl U, Junker R, Kreuz W, von Eckardstein A, Kosch A, Nohe N, et al. Risk of recurrent venous thrombosis in children with combined prothrombotic risk factors. Blood 2001, 97:858-862.
27. Paschôa AF, Guillaumon AT Impact of screening on thrombophilia for patients with venous thrombosis. Int Angiol 2006, 25:52-59.
28. Goldhaber SZ, Morrison RB Cardiology patient pages. Pulmonary embolism and deep vein thrombosis. Circulation 2002, 106:1436-1438.
29. Tiede A, Friedrich U, Stenmo C, Allen G, Giangrande P, Goudemand J, et al. Safety and pharmacokinetics of subcutaneously administered recombinant activated factor VII (rFVIIa). J Thromb Haemost 2011, 9:1191-1199.
30. Trial ID: NN7128-380 Clinical Trial Report: Synopsis [Accessed March 25, 2015]. http://novonordisk-trials.com/Website/pdf/registry/bin_20130206-034941-231.pdf.
31. Nicolaisen EM Antigenicity of activated recombinant factor VII followed through nine years of clinical experience. Blood Coagul Fibrinolysis 1998, 9 Suppl 1:S119-S123.
32. Arkin S, Blei F, Fetten J, Foulke R, Gilchrist GS, Heisel MA, et al. Human coagulation factor FVIIa (recombinant) in the management of limb-threatening bleeds unresponsive to alternative therapies: results from the NovoSeven emergency-use programme in patients with severe haemophilia or with acquired inhibitors. Blood Coagul Fibrinolysis 2000, 11:255-259.
33. Hay CR, Négrier C, Ludlam CA The treatment of bleeding in acquired haemophilia with recombinant factor VIIa: a multicentre study. Thromb Haemost 1997, 78:1463-1467.
34. Mariani G, Herrmann FH, Schulman S, Batorova A, Wulff K, Etro D, et al. Thrombosis in inherited factor VII deficiency. J Thromb Haemost 2003, 1:2153-2158.
35. Girolami A, Ruzzon E, Tezza F, Scandellari R, Vettore S, Girolami B Arterial and venous thrombosis in rare congenital bleeding disorders: a critical review. Haemophilia 2006, 12:345-351.
36. Girolami A, Tezza F, Scandellari R, Vettore S, Girolami B Associated prothrombotic conditions are probably responsible for the occurrence of thrombosis in almost all patients with congenital FVII deficiency. Critical review of the literature. J Thromb Thrombolysis 2010, 30:172-178.
37. Mariani G, Konkle BA, Ingerslev J Congenital factor VII deficiency: therapy with recombinant activated factor VII - a critical appraisal. Haemophilia 2006, 12:19-27.
38. Rice KM, Savidge GF NovoSeven (recombinant factor VIIa) in central nervous systems bleeds. Haemostasis 1996, 26(Suppl. 1):131-134.
39. Scharrer I Recombinant factor VIIa for patients with inhibitors to factor VIII or IX or factor VII deficiency. Haemophilia 1999, 5:253-259.
40. Sumner MJ, Geldziler BD, Pedersen M, Seremetis S Treatment of acquired haemophilia with recombinant activated FVII: a critical appraisal. Haemophilia 2007, 13:451-461.
41. Mariani G, Lapecorella M, Dolce A Steps towards an effective treatment strategy in congenital factor VII deficiency. Semin Hematol 2006, 43:S42-S47.
42. Mariani G, Dolce A, Batorova A, Auerswald G, Schved JF, Siragusa S, et al. Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation - the surgical STER. Br J Haematol 2011, 152:340-346.
43. Tokgoz H, Caliskan U, Lavigne-Lissalde G, Giansily-Blaizot M Successful prophylactic use of recombinant activated factor VII (rFVIIa) in a patient with congenital FVII deficiency and inhibitors to FVII. Haemophilia 2012, 18:e25-e27.
44. Nicolaisen EM Long-term follow-up with regard to potential immunogenicity: clinical experience with NovoSeven (recombinant factor VIIa). Haemostasis 1996, 26(Suppl. 1):98-101.
45. Batorova A, Mariani G, Kavakli K, De Saez AR, Caliskan U, Karimi M, et al. Inhibitors to factor VII in congenital factor VII deficiency. Haemophilia 2014, 20:e188-e191.
46. Pruthi RK, Rodriguez V, Allen C, Slaby JA, Schmidt KA, Plumhoff EA Molecular analysis in a patient with severe factor VII deficiency and an inhibitor: report of a novel mutation (S103G). Eur J Haematol 2007, 79:354-359.
47. Biere-Rafi S, Zwiers M, Peters M, van der Meer J, Rosendaal FR, Buller HR, et al. The effect of haemophilia and von Willebrand disease on arterial thrombosis: a systematic review. Neth J Med 2010, 68:207-214.
48. Bilora F, Zanon E, Petrobelli F, Cavraro M, Prandoni P, Pagnan A, et al. Does hemophilia protect against atherosclerosis? A case-control study. Clin Appl Thromb Hemost 2006, 12:193-198.
49. Gailani D, Renné T Intrinsic pathway of coagulation and arterial thrombosis. Arterioscler Thromb Vasc Biol 2007, 27:2507-2513.
50. Rosendaal FR, Briet E, Stibbe J, van Herpen G, Leuven JA, Hofman A, et al. Haemophilia protects against ischaemic heart disease: a study of risk factors. Br J Haematol 1990, 75:525-530.
51. Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, et al. Guidelines for the management of hemophilia. Haemophilia 2013, 19:e1-47.
52. de Raucourt E, Roussel-Robert V, Zetterberg E Prevention and treatment of atherosclerosis in haemophilia - how to balance risk of bleeding with risk of ischaemic events. Eur J Haematol 2015, 94 Suppl. 77:23-29.
53. Staritz P, de Moerloose P., Schutgens R, Dolan G, ADVANCE Working Group Applicability of the European Society of Cardiology guidelines on management of acute coronary syndromes to people with haemophilia - an assessment by the ADVANCE Working Group. Haemophilia 2013, 19:833-840.
54. Schutgens RE, Tuinenburg A, Fischer K, Mauser-Bunschoten EP Anti-coagulation therapy in haemophilia. Managing the unknown. Hamostaseologie 2013, 33:299-304.
55. Giansily-Blaizot M, Marty S, Chen SW, Pellequer JL, Schved JF Is the coexistence of thromboembolic events and Factor VII deficiency fortuitous?. Thromb Res 2012, 130(Suppl. 1):S47-S49.
56. Girolami A, Bertozzi I, Rigoni I, Muzzolon R, Vettore S Congenital FVII deficiency and thrombotic events after replacement therapy. J Thromb Thrombolysis 2011, 32:362-367.
57. Girolami A, Berti de Marinis G, Vettore S, Girolami B Congenital FVII deficiency and pulmonary embolism: a critical appraisal of all reported cases. Clin Appl Thromb Hemost 2013, 19:55-59.
58. Marty S, Barro C, Chatelain B, Fimbel B, Tribout B, Reynaud J, et al. The paradoxical association between inherited factor VII deficiency and venous thrombosis. Haemophilia 2008, 14:564-570.
59. Bleeding disorder statistics for April 2013 to March 2014. A report from the National Haemophilia Database [Accessed March 25, 2015]. http://www.ukhcdo.org/docs/AnnualReports/2014/Bleeding_Disorder_Statistics_For_Website_2013-2014.pdf.
60. Dolan G, Bjerre J, Hay CRM Real-life use of activated recombinant FVII (rFVIIa) in elderly patients with haemophilia with inhibitors - data from the UK National Haemophilia Database. J Thromb Haemost 2013, 11(Suppl. 2):980.
61. Birschmann I, Klamroth R, Eichler H, Schenk J, Kirchmaier CM, Halimeh S Results of the WIRK prospective, non-interventional observational study of recombinant activated factor VII (rFVIIa) in patients with congenital haemophilia with inhibitors and other bleeding disorders. Haemophilia 2013, 19:679-685.
62. Chambost H, Santagostino E, Laffan M, Kavakli K, ONE Registry Steering Committee on behalf of the investigators Real-world outcomes with recombinant factor VIIa treatment of acute bleeds in haemophilia patients with inhibitors: results from the international ONE registry. Haemophilia 2013, 19:571-577.
63. Di Minno G, d'Oiron R, Zotz R, Poon M-C Treatment modalities and outcomes in 870 non-surgical bleeds in 184 Glanzmann's thrombasthenia patients: the International Prospective Glanzmann's Thrombasthenia Registry. Haematologica 2015;May 22, pii:haematol.2014.121475.
64. Hay CRM, Bjerre J, Dolan G Real-life use of high and standard initial doses of activated recombinant factor VII (rFVIIa) in patients with haemophilia A and B with inhibitors - data from the UKHCDO/NHD registry. J Thromb Haemost 2013, 11(Suppl. 2):718.
65. Kavakli K, Arkhammar P, Benson G, Chambost H, De Martis F, Rosholm A Interim results from the prospective observational study on NovoSevensup®/sup room temperature stable (VII25) in patients with hemophilia A or B. J Thromb Haemost 2014, 12(Suppl. 1):63-64.
66. Mariani G, Dolce A, Napolitano M, Ingerslev J, Giansily-Blaizot M, Di Minno MD, et al. Invasive procedures and minor surgery in factor VII deficiency. Haemophilia 2012, 18:e63-e65.
67. Napolitano M, Giansily-Blaizot M, Dolce A, Schved JF, Auerswald G, Ingerslev J, et al. Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER). Haematologica 2013, 98:538-544.
68. Poon M-C, d'Oiron R, Zotz R, Di Minno G Treatment modalities and outcomes in 204 surgical procedures in 96 Glanzmann's thrombasthenia (GT) patients: the International Prospective Glanzmann's Thrombasthenia Registry (GTR). Haematologica 2015;May 22, pii:haematol.2014.121384.
69. Bolton-Maggs PH, Chalmers EA, Collins PW, Harrison P, Kitchen S, Liesner RJ, et al. A review of inherited platelet disorders with guidelines for their management on behalf of the UKHCDO. Br J Haematol 2006, 135:603-633.
70. Collins PW, Chalmers E, Hart DP, Liesner R, Rangarajan S, Talks K, et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). UK Haemophilia Centre Doctors Organization. Br J Haematol 2013, 160:153-170.
71. Collins W, Chalmers E, Hart D, Jennings I, Liesner R, Rangarajan S, et al. Diagnosis and management of acquired coagulation inhibitors: a guideline from UKHCDO. Br J Haematol 2013, 162:758-773.
72. Mumford AD, Ackroyd S, Alikhan R, Bowles L, Chowdary P, Grainger J, et al. Guideline for the diagnosis and management of the rare coagulation disorders: a United Kingdom Haemophilia Centre Doctors' Organization guideline on behalf of the British Committee for Standards in Haematology. Br J Haematol 2014, 167:304-326.
73. Al-Mondhiry HAB, Ma A, Kessler CM, Fisher M, Gut RZ, Cooper DL US experience with recombinant factor VIIa (rFVIIa) for surgery in acquired hemophilia (AH): analysis from the Hemostasis and Thrombosis Research Society (HTRS) registry. Blood 2012, 120:3372.
74. Ma A, Kessler CM, Gut RZ, Cooper DL Recombinant factor VIIa (rFVIIa) is safe and effective when used to treat acute bleeding episodes and to prevent bleeding during surgery in patients with acquired hemophilia: updated assessment from the Hemostasis and Thrombosis Research Society (HTRS) registry AH database. Blood 2011, 118:3374.
75. Ma AD, Kessler C, Al-Mondhiry HAB, Fisher M, Gut RZ, Cooper DL Use of recombinant factor VIIa (rFVIIa) for acute bleeding episodes in acquired haemophilia: final analysis from the Hemostasis and Thrombosis Research Society (HTRS) registry AH study. Blood 2012, 120:4264.
76. Parameswaran R, Shapiro AD, Gill JC, Kessler CM, HTRS Registry Investigators Dose effect and efficacy of rFVIIa in the treatment of haemophilia patients with inhibitors: analysis from the Hemophilia and Thrombosis Research Society Registry. Haemophilia 2005, 11:100-106.
77. Takedani H, Shima M, Horikoshi Y, Koyama T, Fukutake K, Kuwahara M, et al. Ten-year experience of recombinant activated factor VII use in surgical patients with congenital haemophilia with inhibitors or acquired haemophilia in Japan. Haemophilia 2014 Dec 18, [Epub ahead of print].
78. Young G, Shapiro AD, Walsh CE, Gruppo RA, Gut RZ, Cooper DL Patient/caregiver-reported recombinant factor VIIa (rFVIIa) dosing: home treatment of acute bleeds in the Dosing Observational Study in Hemophilia (DOSE). Haemophilia 2012, 18:392-399.
79. Young G, Auerswald G, Jimenez-Yuste V, Lambert T, Morfini M, Santagostino E, et al. PRO-PACT: retrospective observational study on the prophylactic use of recombinant factor VIIa in hemophilia patients with inhibitors. Thromb Res 2012, 130:864-870.
80. Belhani MF, Hadj Khalifa H, Wali YAS, Alzoebie A, Saad HA, Benchikh el Fegoun S Home treatment of haemarthrosis with activated recombinant factor VII (rFVIIa) in haemophilia A and B patients with inhibitors: a prospective observational study. Haemophilia 2013, 19(Suppl. 2):43.
81. Shapiro AD, Akins S, Cooper DL Long-term outcomes from orthopaedic surgery in haemophilia: are we measuring success and documenting and assessing complications?. Haemophilia 2014, 20:e367-e371.