Prevention of arthropathy: May it be extrapolated from patients without inhibitors to patients with inhibitors?

Haemophilia

Volumn 14, Issue SUPPL. 6, 2008, Pages 23-27

  Hedner, U ^a,b,c  

^a University of Lund ^*  (Sweden)

^b LUND UNIVERSITY   (Sweden)

^c NOVO NORDISK A S   (Denmark)

Author keywords

Haemophilia; Inhibitors; Prophylaxis

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; AUTOANTIBODY; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 ANTIBODY; BLOOD CLOTTING FACTOR 9 CONCENTRATE; RECOMBINANT BLOOD CLOTTING FACTOR 7A; UNCLASSIFIED DRUG;

ARTHROPATHY; ARTICLE; CLINICAL TRIAL; DISEASE SEVERITY; DOSAGE SCHEDULE COMPARISON; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG HALF LIFE; DRUG MEGADOSE; DRUG SAFETY; HEMOPHILIA; HEMOSTASIS; HOME CARE; HUMAN; IMMUNOLOGICAL TOLERANCE; JOINT CONTRACTURE; KNEE; OPTIMAL DRUG DOSE; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; SINGLE DRUG DOSE; THROMBOEMBOLISM; TRACTION THERAPY;

ADULT; BLOOD COAGULATION FACTOR INHIBITORS; BLOOD COAGULATION FACTORS; CHILD; COAGULANTS; DRUG ADMINISTRATION SCHEDULE; FACTOR VIIA; HEMARTHROSIS; HEMOPHILIA A; HUMANS; MALE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECOMBINANT PROTEINS;

EID: 53249134603     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2008.01886.x     Document Type: Article

References (41)

1. Ahlberg Å. Haemophilia in Sweden VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand 1965; 77 (Suppl.): 7-80.
2. Blombäck B, Blombäck M. Purification of human and bovine fibrinogen. Ark Kemi 1956; 10: 415-43.
3. Nilsson IM, Hedner U, Ahlberg Å. Haemophilia prophylaxis in Sweden. Acta Paediatr Scand 1976; 65: 129-35.
4. Fischer K, Astermark J, van der Bom JG et al. Prophylactic treatment for severe haemophilia: Comparison of an intermediate-dose to a high-dose regimen. Haemophilia 2002; 8: 753-60.
5. Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25-32.
6. Brettler DB, Forsberg AD, O'Connell FD, Cederbaum AI, Chaitman AK, Levine PH. A long-term study of hemophilic arthropathy of the knee joint on a program of Factor VIII replacement given at time of each hemarthrosis. Am J Hematol 1985; 18: 13-8.
7. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357: 535-44.
8. Fekete L, Holst S, Peetoom F, deVeber LL. "Auto"factor IX concentrate: A new therapeutic approach to treatment of hemophilia A patients with inhibitors. Paper presented at the XIV International Congress of Hematology, São Paulo, July 16-21, 1972.
9. Kurczynski EM, Penner JA. Activated prothrombin concentrate for patients with factor VIII inhibitors. N Engl J Med 1974; 291: 164-7.
10. Lusher JM. Thrombogenicity associated with factor IX complex concentrates. Semin Hematol 1991; 28: 3-5.
11. White GC II, Roberts HR, Kingdon HS, Lundblad RL. Prothrombin complex concentrates: Potentially thrombogenic materials and clues to the mechanism of thrombosis in vivo. Blood 1977; 49: 159-70.
12. Kasper CK, Hemophilia Study Group. Effect of prothrombin complex concentrates on factor VIII inhibitor levels. Blood 1979; 54: 1358-68.
13. Onder O, Hoyer LW. FVIII coagulant antigen in factor IX complex concentrates. Thromb Res 1979; 15: 569-72.
14. Bray GL, Kingdon H, Lundblad R et al. Characterization of active enzymes in Autoplex (abstract). Blood 1995; 10 (Suppl. 1): 195a. Abstract no. 766.
15. Sjamsoedin LJM, Heijnen L, Mauser-Bunschoten EP et al. The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII. A double-blind clinical trial. N Engl J Med 1981; 305: 717-21.
16. Lusher JM, Shapiro SS, Palascak JE et al. Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: A multicenter therapeutic trial. N Engl J Med 1980; 303: 421-5.
17. Young G, Shafer FE, Rojas P, Seremetis S. Single 270μg/kg-dose rFVIIa vs. standard 90μg/kg-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: A randomized comparison. Haemophilia 2008; 14: 287-94.
18. Hedner U, Schulman S, Alberts KA et al. Successful use of rFVIIa in a patient with severe hemophilia A subjected to synovectomy. Lancet 1988; 2: 1193.
19. Hedner U, Ingerslev J. Clinical use of recombinant FVIIa (rFVIIa). Transfus Sci 1998; 19: 163-76.
20. 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: 773-78.
21. 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: 912-8.
22. Fridberg MJ, Hedner U, Roberts HR, Erhardtsen E. A study of the pharmacokinetics and safety of recombinant activated factor VII in healthy Caucasian and Japanese subjects. Blood Coagul Fibrinolysis 2005; 16: 259-66.
23. Glazer S, Hedner U, Falch JF. Clinical update on the use of recombinant factor VIIa. In: Aledort LM, Hoyer LW, Lusher JM, Reisner HM, White GC II, eds. Inhibitors to Coagulation Factors. New York: Plenum Press, 1995. 163-74.
24. Villar A, Aronis S, Morfini M, Santagostino E, Auerswald G, Thomsen H.F, Erhardtsen E, Giangrande P.L.F. Pharmacokinetics of activated recombinant coagulation factor VII (NovoSeven ®) in children vs. adults with haemophilia A. Haemophilia 2004; 10: 352-9.
25. Sumner WT, Monroe DM, Hoffman M. Variability in platelet procoagulant activity in healthy volunteers. Thromb Res 1996; 81: 533-43.
26. Kavakli K, Makris M, Zulfikar B, Erhardtsen E, Abrams ZS, Kenet G. 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: 600-5.
27. Cooper HA, Jones CP, Campion E, Roberts HR, Hedner U. Rationale for the use of high dose rFVIIa in a high-titre inhibitor patient with haemophilia B during major arthopaedic procedures. Haemophilia 2001; 7: 517-22.
28. Saxon BR, Shanks D, Jory CB, Williams V. Effective prophylaxis with daily recombinant factor VIIa (rFVIIa-NovoSeven) in a child with high titre inhibitors and a target joint. Thromb Haemost 2001; 86: 1126-7.
29. Blatny J, Kohlerove S, Zapletal O, Fiamoli V, Penka M. rFVIIa is effective alternative to aPCC in prophylaxis of bleeding during immune tolerance protocol in haemophiliac with inhibitor (abstract). Abstract no. P-M-166, XXIst ISTH Congress, Geneva, July 6-11, 2007.
30. Brackmann HH, Effenberger E, Hess L, Schwaab R, Oldenburg J. NovoSeven in immune tolerance therapy. Blood Coagul Fibrinol 2000; 11 (Suppl. 1): 39-44.
31. Lopez Fernandez M, Andon Saavedra C, Amor Otero MA, Battle J. "Primary"prophylactic treatment with recombinant activated factor VII (rFVIIa) during immune tolerance in a haemophilic child (abstract). Abstract no. P-M-161, XXIst ISTH Congress, Geneva, July 6-11, 2007.
32. Marquès-Verdier A, Chaleteix C, Soulié B, Bay JO. Secondary prophylaxis with recombinant activated factor VII (rFVIIa) in a severe haemophilia A patient with anti-factor VIII inhibitors (abstract). Abstract no. P-T-168, XXIst ISTH Congress, Geneva, July 6-11, 2007.
33. Molina MQ, Bello IF, Yuste VJ et al. Prophylaxis with recombinant FVIIa (abstract). Abstract no. P-T-170, XXIst ISTH Congress, Geneva, July 6-11, 2007.
34. Tagliaferri A, Rivolta G, De Fanti A, Pattacini C, Negri N, Izzi G. Prophylaxis with rFVIIa during immune-tolerance treatment in a haemophiliac child with a target joint (abstract). Haemophilia 2006; 12 (Suppl. 2): Abstract no. 23 PO 718.
35. Young G, McDaniel M, Nugent DJ. Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors. Haemophilia 2005; 11: 203-7.
36. Morfini M, Auerswald G, Kobelt RA et al. Prophylactic treatment of haemophilia patients with inhibitors: Clinical experience with recombinant factor VIIa in European Haemophilia Centres. Haemophilia 2007; 13: 502-7.
37. 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: 1904-13.
38. Steen Carlsson K, Höjgård S, Glomstein A et al. On-demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome. Haemophilia 2003; 9: 555-66.
39. Nilsson IM, Blombäck M, Ahlberg Å. Our experience in Sweden with prophylaxis on haemophilia. Proceedings of the 5th Congress World Federation of Hemophilia, Montreal. Bibl Haemat 1968; 34: 111-24 (Karger, Basel/New York 1970).
40. Hilgartner MW, Makipernaa A, DiMichele DM. Long-term FEIBA prophylaxis does not prevent progression of existing joint disease. Haemophilia 2003; 9: 261-8.
41. Aronstam A, Arblaster PG, Rainsford SG et al. Prophylaxis in haemophilia: A double-blind controlled trial. Br J Haemat 1976; 33: 81-90.