Successful immune tolerance induction with high-dose coagulation factor VIII and intravenous immunoglobulins in a patient with congenital hemophilia and high-titer inhibitor of coagulation factor VIII despite unfavorable prognosis for the therapy

Medical Science Monitor

Volumn 15, Issue 6, 2009, Pages

  Kubisz, Peter ^a,b   Plameňová, Ivana ^a   Hollý, Pavol ^a   Staško, Ján ^a  

^a University Hospital in Martin   (Slovakia)

^b FACULTY HOSPITAL   (Slovakia)

Author keywords

Coagulation factor VIII inhibitor; Hemophilia A; Immune tolerance induction; Intravenous immunoglobulins; Recombinant activated factor VII

Indexed keywords

ALLOANTIBODY; BERIATE P; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 INHIBITOR; FANHDI; IMMUNOGLOBULIN; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 8A; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR;

ANTIBODY TITER; ARTICLE; BLEEDING; CASE REPORT; DRUG MEGADOSE; HEMOPHILIA; HEMOPHILIA A; HUMAN; IMMUNOLOGICAL TOLERANCE; MALE; NEWBORN; PARTIAL THROMBOPLASTIN TIME; PROGNOSIS; PROPHYLAXIS; SEPSIS; TREATMENT OUTCOME;

BLOOD COAGULATION FACTOR INHIBITORS; CHILD; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG ADMINISTRATION SCHEDULE; FACTOR VIII; HEMOPHILIA A; HEMOSTASIS; HUMANS; IMMUNE TOLERANCE; IMMUNOGLOBULINS, INTRAVENOUS; IMMUNOTHERAPY; PROGNOSIS; TIME FACTORS;

EID: 66749108874     PISSN: 12341010     EISSN: 16433750     Source Type: Journal    
DOI: None     Document Type: Article

References (31)

1. th ed. Philadelphia: Lippincott Williams & Wilkins, 2006; 887-904
2. Wight J, Paisley S: The epidemiology of inhibitors in haemophilia A: a systemic review. Haemophilia, 2003; 9: 418-435 (Pubitemid 36874391)
3. DiMichele DM: Inhibitors in hemophilia: A primer. June 2004. Available from: http://www.wfh.org/2/docs/Publications/Inhibitors/TOH-7-English- Inhibitors.pdf
4. Oldenburg J, Schwaab R, Brackmann HH: Induction of immune tolerance in haemophilia A inhibitor patients by the "Bonn protocol": predictive parameter for therapy duration and outcome. Vox Sang, 1999; 77: 49-54
5. Rocino A, Santagostino E, Mancuso ME, Mannucci PM: Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors. Haematologica, 2006; 91: 558-561
6. Mauz-Korholz C, Korholz D, Gobel U: Rapid elimination of a high-titered F VIII inhibitor by high dose recombinant F VIII combined with high dose immunoglobulin infusion. Thromb Haemost, 1997; 78: 959 (Pubitemid 27365601)
7. Giles AR, Verbruggen B, Roivard GE et al, and the Association of Haemophilia Centre of Canada: A detailed comparison of the performance of the standard versus Nijmegen modification of the Bethesda assay in detecting factor FVIII inhibitors in the hemophilia population of Canada. Thromb Haemost, 1998; 79: 872-875 (Pubitemid 28163870)
8. Saint-Remy J-MR, Lacroix-Desmazes S, Oldenburg J: Inhibitors in haemophilia: pathophysiology. Haemophilia, 2004; 10: 146-151 (Pubitemid 40050864)
9. DiMichele D, Rivard G, Hay C, Antunes S: Inhibitors in haemophilia: clinical aspects. Haemophilia, 2004; 10(S4): 140-145 (Pubitemid 40045433)
10. White II GC, Kempton CL, Grimsley A et al: Cellular immune responses in hemophilia: why do inhibitors develop in some, but not all hemophiliacs? J Thromb Haemost, 2005; 3: 1676-1681
11. Gilles JGG: Nature autoantibodies and antiidiotypes. Sem Thromb Hemost, 2000; 26: 151-155
12. Sapir T, Blank M, Shoefeld Y: Immunomodulatory effects of intravenous immunoglobulins as a treatment for autoimmune diseases, cancer, and recurrent pregnancy loss. Ann NY Acad Sci, 2005; 1051: 743-778 (Pubitemid 41362801)
13. Von Depka M: Managing acute bleeds in the patient with haemophilia and inhibitors: option, efficacy and safety. Haemophilia, 2005; 11(S1): 18-23 (Pubitemid 41631606)
14. Berntorp E: Options for treating acute bleeds in addition to bypassing agents: extracorporeal immunoadsorption, FVIII/FIX, desmopressin and antifibrinolytics. Haemophilia, 2006; 12(S6): 62-66 (Pubitemid 44741504)
15. Rivard GE, St Louis J, Lacroix S et al: Immunoadsorption for coagulation factor inhibitors: a retrospective critical appraisal of 10 consecutive cases from a single institution. Haemophilia, 2003; 9: 711-716 (Pubitemid 37540811)
16. Wiestner A, Cho HJ, Asch AS et al: Rituximab in the treatment of acquired factor VIII inhibitors. Blood, 2002; 100: 199-205 (Pubitemid 35217098)
17. Stasi R, Brunetti M, Stipa E, Amadori S: Selective B-cell depletion with rituximab for the treatment of patients with acquired hemophilia. Blood, 2004; 103: 4424-4428 (Pubitemid 38745966)
18. Kessler CM: New perspectives in hemophilia treatment. Am Soc Hematol Educ Prog, 2005: 429-435
19. Franchini M, Capra F, Nicolini N et al: Drug-induced anti-factro VIII antibodies: A systemic review. Med Sci Monit, 2007; 13(4): RA55-61 (Pubitemid 46557763)
20. Collins PW: Novel therapies for immune tolerance in haemophilia A. Haemophilia, 2006; 12(S6): 94-101 (Pubitemid 44741509)
21. Mariani G, Kroner BL: International Immune Tolerance Registry, 1997 update. Vox Sang, 1999; 77: 25-27
22. Hay CRM, Brown S, Collins PW et al: The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation. Br J Haematol, 2006; 133: 591-605 (Pubitemid 43742773)
23. DiMichele DM, Kroner BL: The North American Immune Tolerance Registry: practises, outcomes, outcome predictors. Thromb Haemost, 2002; 87: 52-57 (Pubitemid 34092575)
24. Lenk H: The German Registry of immune tolerance treatment in hemophilia - 1999 update. Haematologica, 2000; 85: 45-47
25. Mariani G, Kroner B: Immune tolerance in hemophilia with factor VIII inhibitors: predictors of success. Haematologica, 2001; 86: 1186-1193 (Pubitemid 33063865)
26. Kreuz W, Mentzer D, Auerswald G et al: Successful immunotolerance therapy of FVIII inhibitor in children after changing from high to intermediate purity FVIII concentrate. XXII International Congress of the World Federation of Hemophilia, Dublin. Haemophilia, 1996; 1(S2): 19 (Abstract)
27. Lillicrap D: The role of immunomodulation in the management of factor VIII inhibitors. Am Soc Hematol Educ Prog, 2006; 421-425
28. Gringeri A, Mannucci PM: Italian guidelines for the diagnosis and treatment of patients with haemophilia and inhibitors. Haemophilia, 2005; 11: 611-619 (Pubitemid 41631596)
29. Makris M: Systematic review of the management of patients with haemophilia A and inhibitors. Blood Coagulation & Fibrinolysis, 2004; 15(Suppl.1): S25-27
30. Abshire T, Kenet G: Recombinant factor FVIIa: review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors. J Thromb Haemsot, 2004; 2: 899-909 (Pubitemid 40185737)
31. Teitel JM: Recombinant factor VIIa versus aPCCs in haemophiliacs with inhibitors: treatment and cost considerations. Haemophilia, 1999; 5(s3): 43-49 (Pubitemid 29497720)