Safety and efficacy of recombinant factor VIIa by pediatric age cohort: reassessment of compassionate use and trial data supporting US label

Pediatric Blood and Cancer

Volumn 63, Issue 10, 2016, Pages 1822-1828

  Croteau, Stacy E ^a   Nakar, Charles ^b   Neufeld, Ellis J ^a   Shapiro, Amy ^b   Cooper, David L ^c  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b INDIANA HEMOPHILIA AND THROMBOSIS CENTER   (United States)

^c NOVO NORDISK INC   (United States)

Author keywords

hemophilia; hemostasis; inhibitors; rFVIIa

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 7A;

ADOLESCENT; ADULT; ARTICLE; BLEEDING; BODY TEMPERATURE; CHILD; COMPASSIONATE USE; CONGENITAL HEMOPHILIA; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG SAFETY; EDEMA; FEVER; GROUPS BY AGE; HEADACHE; HEMOPHILIA; HEMOSTASIS; HUMAN; HYPERTENSION; MAJOR CLINICAL STUDY; MALE; NAUSEA; PAIN; PERIOPERATIVE BLEEDING; PRIORITY JOURNAL; PRURITUS; RASH; SIDE EFFECT; SURGICAL TECHNIQUE; THROMBOSIS; TREATMENT RESPONSE; VOMITING;

EID: 84983313760     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.26082     Document Type: Article

References (22)

1. Jurlander B, Thim L, Klausen NK, et al. Recombinant activated factor VII (rFVIIa): Characterization, manufacturing, and clinical development. Semin Thromb Hemost. 2001;27:373–384.
2. NovoSeven RT [package insert]. Bagsvaerd, Denmark: NovoNordisk A/S; 2015.
3. Hedner U, Glazer S, Pingel K, et al. Successful use of recombinant factor VIIa in patient with severe haemophilia A during synovectomy. Lancet. 1988;2:1193.
4. Hedner U, Lee CA. First 20 years with recombinant FVIIa (NovoSeven). Haemophilia. 2011;17:e172–e182.
5. Bech RM. Recombinant factor VIIa in joint and muscle bleeding episodes. Haemostasis. 1996;26(Suppl 1):135–138.
6. Shapiro AD. Recombinant factor VIIa in the treatment of bleeding in hemophilic children with inhibitors. Semin Thromb Hemost. 2000;26:413–419.
7. Arkin S, Blei F, Fetten J, 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.
8. 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.
9. 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.
10. Lusher JM, Roberts HR, Davignon G, et al. A randomized, double-blind 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.
11. Lusher JM. Recombinant factor VIIa (NovoSeven) in the treatment of internal bleeding in patients with factor VIII and IX inhibitors. Haemostasis. 1996;26(Suppl 1):124–130.
12. Lusher J, Ingerslev J, Roberts H, Hedner U. Clinical experience with recombinant factor VIIa. Blood Coagul Fibrinolysis. 1998;9:119–128.
13. Shapiro AD, Gilchrist GS, Hoots WK, Cooper HA, Gastineau DA. Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in haemophilia patients with inhibitors undergoing surgery. Thromb Haemost. 1998;80:773–778.
14. Neufeld EJ, Saxena K, Kessler CM, Cooper DL, Investigators H. Dosing, efficacy, and safety of recombinant factor VIIa (rFVIIa) in pediatric versus adult patients: The experience of the Hemostasis and Thrombosis Research Society (HTRS) Registry (2004-2008). Pediatr Blood Cancer. 2013;60:1178–1183.
15. Shapiro AD. Single-dose recombinant activated factor VII for the treatment of joint bleeds in hemophilia patients with inhibitors. Clin Adv Hematol Oncol. 2008;6:579–586.
16. 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.
17. 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.
18. 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;4:367–371.
19. Kenet G, Lubetsky A, Luboshitz J, Martinowitz U. A new approach to treatment of bleeding episodes in young hemophilia patients: A single bolus megadose of recombinant activated factor VII (NovoSeven). J Thromb Haemost. 2003;1:450–455.
20. 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.
21. 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.
22. 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.