Recombinant porcine factor VIII for high-risk surgery in paediatric congenital haemophilia A with high-titre inhibitor

Haemophilia

Volumn 23, Issue 2, 2017, Pages e93-e98

  Croteau, S E ^a   Abajas, Y L ^b   Wolberg, A S ^b   Nielsen, B I ^b   Marx, G R ^a   Baird, C W ^a   Neufeld, E J ^a   Monahan, P E ^b,c  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

^c SHIRE   (United States)

Author keywords

bypassing agents; FEIBA; Obizur; recombinant activated factor VII; recombinant porcine factor VIII

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 INHIBITOR; BORTEZOMIB; DEXAMETHASONE; HEMOSTATIC AGENT; IMMUNOGLOBULIN; MYCOPHENOLIC ACID; PROTHROMBIN COMPLEX; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RITUXIMAB; RECOMBINANT FACTOR VIII SQ; RECOMBINANT PROTEIN;

AORTA COARCTATION; ARTICLE; CASE REPORT; CHILD; DRUG COST; DRUG MEGADOSE; HEART SURGERY; HEMOPHILIA A; HEMOSTASIS; HUMAN; IMMUNOLOGICAL TOLERANCE; IMMUNOSUPPRESSIVE TREATMENT; IMMUNOTHERAPY; MALE; PERIOPERATIVE PERIOD; PRESCHOOL CHILD; SURGICAL RISK; THROMBIN TIME; ANIMAL; PIG;

ANIMALS; CHILD, PRESCHOOL; FACTOR VIII; HEMOPHILIA A; HUMANS; MALE; RECOMBINANT PROTEINS; SWINE;

EID: 85015337934     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.13157     Document Type: Article

References (19)

1. Valentino LA, Cooper DL, Goldstein B. Surgical experience with rFVIIa (NovoSeven) in congenital haemophilia A and B patients with inhibitors to factors VIII or IX. Haemophilia 2011; 17: 579–89.
2. 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: 546–51.
3. Croteau SE, Nakar C, Neufeld EJ, Shapiro A, Cooper DL. Safety and efficacy of recombinant factor VIIa by pediatric age cohort: reassessment of compassionate use and trial data supporting US label. Pediatr Blood Cancer 2016; 63: 1822–8.
4. Astermark J, Santagostino E, Keith Hoots W. Clinical issues in inhibitors. Haemophilia 2010; 16(Suppl. 5): 54–60.
5. Hoffman M, Dargaud Y. Mechanisms and monitoring of bypassing agent therapy. J Thromb Haemost 2012; 10: 1478–85.
6. Kruse-Jarres R, St-Louis J, Greist A et al. Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A. Haemophilia 2015; 21: 162–70.
7. OBIZUR [package insert]. Antihemophilic Factor (Recombinant), Porcine Sequence. Westlake Village, CA: Baxter Healthcare Corporation, 2014.
8. Lillicrap D, Schiviz A, Apostol C et al. Porcine recombinant factor VIII (Obizur; OBI-1; BAX801): product characteristics and preclinical profile. Haemophilia 2016; 22: 308–17.
9. Kempton CL, Abshire TC, Deveras RA et al. Pharmacokinetics and safety of OBI-1, a recombinant B domain-deleted porcine factor VIII, in subjects with haemophilia A. Haemophilia 2012; 18: 798–804.
10. Dargaud Y, Lienhart A, Negrier C. Prospective assessment of thrombin generation test for dose monitoring of bypassing therapy in hemophilia patients with inhibitors undergoing elective surgery. Blood 2010; 116: 5734–7.
11. Chitlur M, Young G. Global assays in hemophilia. Semin Hematol 2016; 53: 40–5.
12. Hay CR. Porcine factor VIII: current status and future developments. Haemophilia 2002; 8(Suppl. 1): 24–7.
13. Beutel K, Hauch H, Rischewski J, Kordes U, Schneppenheim J, Schneppenheim R. ITI with high-dose FIX and combined immunosuppressive therapy in a patient with severe haemophilia B and inhibitor. Hamostaseologie 2009; 29: 155–7.
14. Machlus KR, Colby EA, Wu JR, Koch GG, Key NS, Wolberg AS. Effects of tissue factor, thrombomodulin and elevated clotting factor levels on thrombin generation in the calibrated automated thrombogram. Thromb Haemost 2009; 102: 936–44.
15. Khandelwal P, Davies SM, Grimley MS et al. Bortezomib for refractory autoimmunity in pediatrics. Biol Blood Marrow Transplant 2014; 20: 1654–9.
16. Lozier JN, Santagostino E, Kasper CK, Teitel JM, Hay CR. Use of porcine factor VIII for surgical procedures in hemophilia A patients with inhibitors. Semin Hematol 1993; 30: 10–21.
17. Toschi V. OBI-1, porcine recombinant factor VIII for the potential treatment of patients with congenital hemophilia A and alloantibodies against human factor VIII. Curr Opin Mol Ther 2010; 12: 617–25.
18. Brettler DB, Forsberg AD, Levine PH et al. The use of porcine factor VIII concentrate (Hyate:C) in the treatment of patients with inhibitor antibodies to factor VIII. A multicenter US experience. Arch Intern Med 1989; 149: 1381–5.
19. Gomperts E. Recombinant B domain deleted porcine factor VIII for the treatment of bleeding episodes in adults with acquired hemophilia A. Expert Rev Hematol 2015; 8: 427–32.