International recommendations on the diagnosis and treatment of patients with Acquired hemophilia a

Haematologica

Volumn 94, Issue 4, 2009, Pages 566-575

  Huth Kühne, Angela ^a,j   Baudo, Francesco ^b   Collins, Peter ^c   Ingerslev, Jørgen ^d   Kessler, Craig M ^e   Lévesque, Hervé ^f   Castellano, Maria Eva Mingot ^g   Shima, Midori ^h   St Louis, Jean ⁱ  

^a Hemophilia Center   (Germany)

^b OSPEDALE NIGUARDA CA GRANDA   (Italy)

^c UNIVERSITY HOSPITAL OF WALES   (United Kingdom)

^d AARHUS UNIVERSITY HOSPITAL   (Denmark)

^e GEORGETOWN UNIVERSITY HOSPITAL   (United States)

^f Dept de Médecine Interne Ctr Hosp Univ Rouen Boisguillaume   (France)

^g HOSPITAL REGIONAL UNIVERSITARIO DE MÁLAGA   (Spain)

^h NARA MEDICAL UNIVERSITY   (Japan)

ⁱ MAISONNEUVE ROSEMONT HOSPITAL   (Canada)

^j SRH Kurpfalzkrankenhaus and Hemophilia Center Heidelberg gGmbH   (Germany)

more..

Author keywords

Acquired hemophilia; Bleeding; Inhibitors; Recommendations; Treatment

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; AUTOANTIBODY; AZATHIOPRINE; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 INHIBITOR; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN; DESMOPRESSIN; IMMUNOGLOBULIN; IMMUNOGLOBULIN G; LUPUS ANTICOAGULANT; MYCOPHENOLIC ACID; PREDNISOLONE; PREDNISONE; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RITUXIMAB; SEPHAROSE; TRANEXAMIC ACID; VINCRISTINE;

ALOPECIA; ANTIBODY TITER; ARTERY THROMBOSIS; BLOOD CLOTTING TEST; CLINICAL TRIAL; CONTINUOUS INFUSION; CONVULSION; CORTICOSTEROID THERAPY; CYTOPENIA; DISSEMINATED INTRAVASCULAR CLOTTING; EMBOLISM; FOLLOW UP; HEMOPHILIA A; HEMOSTASIS; HUMAN; HYPONATREMIA; IMMUNOADSORPTION; IMMUNOLOGICAL TOLERANCE; IMMUNOSUPPRESSIVE TREATMENT; IMMUNOTHERAPY; INFECTION COMPLICATION; NEUTROPENIA; PARTIAL THROMBOPLASTIN TIME; PLASMAPHERESIS; PRACTICE GUIDELINE; PROPHYLAXIS; RECOMMENDED DRUG DOSE; REVIEW; SHEEP; SIDE EFFECT; THROMBOSIS; TOXIC HEPATITIS; VEIN THROMBOSIS; WATER RETENTION;

DRUG THERAPY, COMBINATION; GERMANY; HEMOPHILIA A; HEMORRHAGE; HUMANS; INTERNATIONAL COOPERATION; PARTIAL THROMBOPLASTIN TIME;

EID: 66349094306     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2008.001743     Document Type: Review

References (104)

1. Collins PW, Hirsch S, Baglin TP, Dolan G, Hanley J, Makris M, et al. Acquired hemophilia A in the United Kingdom: a 2-year national surveil- lance study by the United Kingdom Haemophilia Centre Doctors' Organisation. Blood 2007;109:1870-7.
2. Green D, Lechner K. A survey of 215 non-hemophilic patients with inhibitors to Factor VIII. Thromb Haemost 1981;45:200-3.
3. Morrison AE, Ludlam CA, Kessler C. Use of porcine factor VIII in the treat- ment of patients with acquired hemo- philia. Blood 1993;81:1513-20.
4. Franchini M, Gandini G, Di Paolantonio T, Mariani G. Acquired hemophilia A: a concise review. Am J Hematol 2005;80:55-63.
5. Grunewald M, Beneke H, Guthner C, Germowitz A, Brommer A, Griesshammer M. Acquired haemophilia: experiences with a stan- dardized approach. Haemophilia 2001;7:164-9.
6. Green D. The management of acquired haemophilia. Haemophilia 2006;12 (Suppl 5):32-6.
7. Guyatt G, Gutterman D, Baumann MH, Addrizzo-Harris D, Hylek EM, Phillips B, et al. Grading strength of recommendations and quality of evi- dence in clinical guidelines: Report from an American College of Chest Physicians task force. Chest 2006; 129:174-81.
8. Ma AD, Carrizosa D. Acquired factor VIII inhibitors: pathophysiology and treatment. Hematology Am Soc Hematol Educ Program 2006:432-7.
9. Collins PW. Management of acquired haemophilia A-more questions than answers. Blood Coagul Fibrinolysis 2003;14 (Suppl 1):S23-7.
10. Lossing TS, Kasper CK, Feinstein DI. Detection of factor VIII inhibitors with the partial thromboplastin time. Blood 1977;49:793-7.
11. Kasper CK. Laboratory tests for factor VIII inhibitors, their variation, significance and interpretation. Blood Coagul Fibrinolysis 1991; 2 (Suppl 1): 7-10.
12. Kasper CK, Aledort L, Aronson D, Counts R, Edson JR, van Eys J, et al. Proceedings: A more uniform meas- urement of factor VIII inhibitors. Thromb Diath Haemorrh 1975; 34:612.
13. Verbruggen B, Novakova I, Wessels H, Boezeman J, van den Berg M, Mauser-Bunschoten E. The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitors: improved specificity and reliability. Thromb Haemost 1995;73:247-51.
14. Gawryl MS, Hoyer LW. Inactivation of factor VIII coagulant activity by two different types of human antibodies. Blood 1982;60:1103-9.
15. Kazmi MA, Pickering W, Smith MP, Holland LJ, Savidge GF. Acquired haemophilia A: errors in the diagno- sis. Blood Coagul Fibrinolysis 1998;9: 623-8.
16. Greaves M, Cohen H, MacHin SJ, Mackie I. Guidelines on the investiga- tion and management of the antiphospholipid syndrome. Br J Haematol 2000;109:704-15.
17. Sahud MA, Pratt KP, Zhukov O, Qu K, Thompson AR. ELISA system for detection of immune responses to FVIII: a study of 246 samples and cor- relation with the Bethesda assay. Haemophilia 2007;13:317-22.
18. Astermark J, Voorberg J, Lenk H, DiMichele D, Shapiro A, Tjonnfjord G, et al. Impact of inhibitor epitope profile on the neutralizing effect against plasma-derived and recombi- nant factor VIII concentrates in vitro. Haemophilia 2003;9:567-72.
19. Lottenberg R, Kentro TB, Kitchens CS. Acquired hemophilia. A natural history study of 16 patients with fac- tor VIII inhibitors receiving little or no therapy. Arch Intern Med 1987;147: 1077-81.
20. Kessler CM. New perspectives in hemophilia treatment. Hematology Am Soc Hematol Educ Program 2005: 429-35.
21. Delgado J, Jimenez-Yuste V, Hernandez-Navarro F, Villar A. Acquired haemophilia: review and meta-analysis focused on therapy and prognostic factors. Br J Haematol 2003;121:21-35.
22. Novo Nordisk Health Care AG. NovoSeven Summary of Product Characteristics 2006.
23. Baxter Corp. FEIBA VH Anti-inhibitor coagulant complex Vapor Heated Summary of Product Characteristics 2005.
24. Sorensen B, Ingerslev J. Tailoring haemostatic treatment to patient requirements - an update on monitor- ing haemostatic response using thrombelastography. Haemophilia. 2005;11 (Suppl 1):1-6.
25. Hedner U. Dosing with recombinant factor viia based on current evidence. Semin Hematol 2004;41 (Suppl 1): 35-9.
26. Abshire T, Kenet G. Recombinant fac- tor 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.
27. Croom KF, McCormack PL. Recombinant factor VIIa (eptacog α): a review of its use in congenital hemophilia with inhibitors, acquired hemophilia, and other congenital bleeding disorders. BioDrugs 2008; 22:121-36.
28. Franchini M. Recombinant factor VIIa: a review on its clinical use. Int J Hematol 2006;83:126-38.
29. Parameswaran R, Shapiro AD, Gill JC, Kessler CM. Dose effect and effi- cacy of rFVIIa in the treatment of haemophilia patients with inhibitors: analysis from the Hemophilia and Thrombosis Research Society Registry. Haemophilia 2005;11:100-6.
30. Roberts HR, Monroe DM, White GC. The use of recombinant factor VIIa in the treatment of bleeding disorders. Blood 2004;104:3858-64.
31. Sumner MJ, Geldziler BD, Pedersen M, Seremetis S. Treatment of acquired haemophilia with recombi- nant activated FVII: a critical apprais- al. Haemophilia 2007;13:451-61.
32. Hay CR, Negrier C, Ludlam CA. The treatment of bleeding in acquired haemophilia with recombinant factor VIIa: a multicentre study. Thromb Haemost 1997;78:1463-7.
33. Baudo F, de Cataldo F. Acquired hemophilia: a critical bleeding syn- drome. Haematologica 2004;89:96-100.
34. 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.
35. Ingerslev J, Christiansen K, Sorensen B. Inhibitor to factor VII in severe fac- tor VII deficiency: detection and course of the inhibitory response. J Thromb Haemost 2005;3:799-800.
36. Dimichele D, Negrier C. A retrospec- tive postlicensure survey of FEIBA efficacy and safety. Haemophilia 2006;12:352-62.
37. Leissinger CA, Becton DL, Ewing NP, Valentino LA. Prophylactic treatment with activated prothrombin complex concentrate (FEIBA) reduces the fre- quency of bleeding episodes in paedi- atric patients with haemophilia A and inhibitors. Haemophilia 2007;13:249-55.
38. Tjonnfjord GE, Holme PA. Factor eight inhibitor bypass activity (FEIBA) in the management of bleeds in hemophilia patients with high-titer inhibitors. Vasc Health Risk Manag 2007;3:527-31.
39. Sallah S. Treatment of acquired haemophilia with factor eight inhibitor bypassing activity. Haemophilia 2004;10:169-73.
40. Goudemand J. Treatment of bleeding episodes occurring in patients with acquired haemophilia with FEIBA: the French experience. Haemophilia 2004;10:14.
41. Holme PA, Brosstad F, Tjonnfjord GE. Acquired haemophilia: management of bleeds and immune therapy to eradicate autoantibodies. Haemophilia 2005;11:510-5.
42. Yee TT, Taher A, Pasi KJ, Lee CA. A survey of patients with acquired haemophilia in a haemophilia centre over a 28-year period. Clin Lab Haematol 2000;22:275-8.
43. Aledort LM. Factor VIII inhibitor bypassing activity (FEIBA) - address- ing safety issues. Haemophilia 2008; 14:39-43.
44. Hilgartner MW, Knatterud GL. The use of factor eight inhibitor by-pass- ing activity (FEIBA immuno) product for treatment of bleeding episodes in hemophiliacs with inhibitors. Blood 1983;61:36-40.
45. Lechner K, Nowotny C, Krinninger B, Zegner M, Deutsch E. Effect of treat- ment with activated prothrombin complex concentrate (FEIBA) on fac- tor VIII-antibody level. Thromb Haemost 1979;40:478-85.
46. Hay CR, Brown S, Collins PW, Keeling DM, Liesner R. 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.
47. Guillet B, Pinganaud C, Proulle V, Dreyfus M, Lambert T. Myocardial infarction occurring in a case of acquired haemophilia during the treatment course with recombinant activated factor VII. Thromb Haemost 2002;88:698-9.
48. Kasper CK. Human factor VIII for bleeding in patients with inhibitors. Vox Sang 1999;77 (Suppl 1):47-8.
49. Muhm M, Grois N, Kier P, Stumpflen A, Kyrle P, Pabinger I, et al. 1- Deamino-8-D-arginine vasopressin in the treatment of non-haemophilic patients with acquired factor VIII inhibitor. Haemostasis 1990;20:15-20.
50. de la Fuente B, Panek S, Hoyer LW. The effect of 1-deamino 8 D-arginine vasopressin (DDAVP) in a non- haemophilic patient with an acquired type II factor VIII inhibitor. Br J Haematol 1985;59:127-31.
51. Naorose-Abidi SM, Bond LR, Chitolie A, Bevan DH. Desmopressin therapy in patients with acquired factor VIII inhibitors. Lancet 1988;1:366.
52. Mudad R, Kane WH. DDAVP in acquired hemophilia A: case report and review of the literature. Am J Hematol 1993;43:295-9.
53. Hay CR, Lozier JN, Lee CA, Lafan M, Tradati H, Santagostino E, et al. Porcine factor VIII therapy in patients with congenital hemophilia and inhibitors: efficacy, patient selection, and side effects. Semin Hematol 1994;31 (Suppl 4):20-5.
54. Abshire T, Hoots K, Kessler C, Macfarlane D, Bergman G. Preclinical and clinical experiences with a recom- binant porcine FVIII product. Haemophilia 2006;12 (Suppl 2):644.
55. Mahlangu J, Andreeva T, Macfarlane D, Reding M, Walsh C, Ritchie B, et al. A Phase II open-label study evalu- ating hemostatic activity, pharmaco- kinetics and safety of recombinant Porcine Factor VIII (rpFVIII, OBI-1) in hemophilia A patients with alloanti- body inhibitors directed against human FVIII (hFVIII). Blood 2007; 110:Abstract #783.
56. Schneiderman J, Nugent DJ, Young G. Sequential therapy with activated prothrombin complex concentrate and recombinant factor VIIa in patients with severe haemophilia and inhibitors. Haemophilia 2004;10: 347-51.
57. Schneiderman J, Rubin E, Nugent DJ, Young G. Sequential therapy with activated prothrombin complex con- centrates and recombinant FVIIa in patients with severe haemophilia and inhibitors: update of our previous experience. Haemophilia 2007;13: 244-8.
58. Baxter Corp. Product Monograph: FEIBA VH Immuno: Anti-Inhibitor Coagulant Complex, Vapor Heated, Freeze-Dried Substance With Solvent for Intravenous Injection or Infusion Human Plasma Fraction with Factor VIII Inhibitor Bypassing Activity; 2005.
59. Erskine JG, Burnett AK, Walker ID, Davidson JF. Plasma exchange in non-haemophiliac patients with inhibitors to factor VIIIC. Br Med J 1981;283: 760.
60. Hambley H, Watkins R, Tansey PA, Walker ID, Davidson JF. Plasmapheresis and Factor VIIIC inhibitors. Lancet 1985;1:274.
61. Rivard GE, St Louis J, Lacroix S, Champagne M, Rock G. Immunoadsorption for coagulation factor inhibitors: a retrospective criti- cal appraisal of 10 consecutive cases from a single institution. Haemophilia 2003;9:711-6.
62. Huth-Kühne A, Lages P, Hampel H, Zimmermann R. Management of severe hemorrhage and inhibitor elimination in acquired hemophilia: The modified Heidelberg-Malmö protocol. Haematologica 2003;88:86-92.
63. Hay CR. Acquired haemophilia. Baillieres Clin Haematol 1998;11: 287-303.
64. Collins PW. Treatment of acquired hemophilia A. J Thromb Haemost 2007;5:893-900.
65. Baudo F, de Cataldo F. Acquired haemophilia in the elderly. In: Balducci L, Ershler W, de Gaetano G, eds. Blood Disorders in the Elderly. Cambridge: Cambridge University Press, 2007. p. 389-407.
66. Kessler CM, Asatiani E. Acquired Inhibitors to Factor VIII. In: Lee CA, Berntorp EE, Hoots WK, Aledort LM, eds. Textbook of Hemophilia, 2007: 86-90.
67. Green D. Cytotoxic suppression of acquired factor VIII:C inhibitors. Am J Med 1991;91:14S-9S.
68. Green D, Rademaker AW, Briet E. A prospective, randomized trial of prednisone and cyclophosphamide in the treatment of patients with fac- tor VIII autoantibodies. Thromb Haemost 1993;70:753-7.
69. Aggarwal A, Grewal R, Green RJ, Boggio L, Green D, Weksler BB, et al. Rituximab for autoimmune haemophilia: a proposed treatment algorithm. Haemophilia 2005;11:13-9.
70. Sperr WR, Lechner K, Pabinger I. Rituximab for the treatment of acquired antibodies to factor VIII. Haematologica 2007;92:66-71.
71. Wiestner A, Cho HJ, Asch AS, Michelis MA, Zeller JA, Peerschke EI, et al. Rituximab in the treatment of acquired factor VIII inhibitors. Blood 2002;100:3426-8.
72. Maillard H, Launay D, Hachulla E, Goudemand J, Lambert M, Morell- Dubois S, et al. Rituximab in postpar- tum-related acquired hemophilia. Am J Med 2006;119:86-8.
73. Kain S, Copeland TS, Leahy MF. Treatment of refractory autoimmune (acquired) haemophilia with anti- CD20 (rituximab). Br J Haematol 2002;119:578.
74. Franchini M. Rituximab in the treat- ment of adult acquired hemophilia A: a systematic review. Crit Rev Oncol Hematol 2007;63:47-52.
75. Franchini M, Lippi G. Acquired factor VIII inhibitors. Blood 2008;112: 250-5.
76. Sohngen D, Specker C, Bach D, Kuntz BM, Burk M, Aul C, et al. Acquired factor VIII inhibitors in nonhemophilic patients. Ann Hematol 1997;74:89-93.
77. Lian EC, Larcada AF, Chiu AY. Combination immunosuppressive therapy after factor VIII infusion for acquired factor VIII inhibitor. Ann Intern Med 1989;110:774-8.
78. Eisert S, Mosler K, Laws HJ, Gobel U. Successful use of mycophenolate mofetil and prednisone in a 14-year- old girl with acquired hemophilia A. Thromb Haemost 2005;93:792-3.
79. Schulman S, Langevitz P, Livneh A, Mortinowitz U, Seligsohn U, Varon D. Cyclosporine therapy for acquired factor VIII inhibitor in a patient with systemic lupus erythematosus. Thromb Haemost 1996;76: 344-6.
80. Brox AG, Laryea H, Pelletier M. Successful treatment of acquired fac- tor VIII inhibitors with cyclosporin. Am J Hematol 1998;57:87-8.
81. Petrovic M, Derom E, Baele G. Cyclosporine treatment of acquired hemophilia due to factor VIII anti- bodies. Haematologica 2000;85:895-6.
82. Saxena R, Mishra DK, Kashyap R, Choudhry VP, Mahapatra M, Bhargava M. Acquired haemophilia - a study of ten cases. Haemophilia 2000;6:78-83.
83. Borg JY, Levesque H, for the SACHA study group. Epidemiology and one- year outcomes in patients with acquired hemophilia in France. J Thromb Haemost 2007;5 (Suppl 1):O-M-062.
84. Stasi R, Brunetti M, Stipa E, Amadori S. Selective B-cell depletion with rit- uximab for the treatment of patients with acquired hemophilia. Blood 2004;103:4424-8.
85. Huth-Kühne A, Findeklee J, Bajanova M, Lages P, Zimmermann R. Successful inhibitor-elimination with rituximab in one patient with a carri- er state for hemophilia A and 4 patients with acquired hemophilia A. J Thromb Haemost 2007;5 (Suppl 2):[P-T-231].
86. Huth-Kühne A, Krause M, Zimmermann R, Findeklee J, Lages P. Successful inhibitor-elimination with rituximab in one patient with a carri- er state for hemophilia A and 9 patients with acquired hemophilia A. Haemophilia. 2008;14 (Suppl 2):[01- PO-7].
87. Shaffer LG, Phillips MD. Successful treatment of acquired hemophilia with oral immunosuppressive thera- py. Ann Intern Med 1997;127:206-9.
88. Lian EC, Villar MJ, Noy LI, Ruiz- Dayao Z. Acquired factor VIII inhibitor treated with cyclophos- phamide, vincristine, and pred- nisone. Am J Hematol 2002;69:294-5.
89. Bossi P, Cabane J, Ninet J, Dhote R, Hanslik T, Chosidow O, et al. Acquired hemophilia due to factor VIII inhibitors in 34 patients. Am J Med 1998;105:400-8.
90. Huang YW, Saidi P, Philipp C. Acquired factor VIII inhibitors in non-haemophilic patients: clinical experience of 15 cases. Haemophilia 2004;10:713-21.
91. Green D. Spontaneous inhibitors of FVIII. Br J Haematol 1968;15:57-75.
92. Nemes L, Pitlik E. New protocol for immune tolerance induction in acquired hemophilia. Haematologica 2000;85 (10 Suppl):64-8.
93. Zeitler H, Ulrich-Merzenich G, Hess L, Konsek E, Unkrig C, Walger P, et al. Treatment of acquired hemophilia by the Bonn-Malmo Protocol: documen- tation of an in vivo immunomodulat- ing concept. Blood 2005;105:2287-93.
94. Zeitler H, Ulrich-Merzenich G, Walger P, Dusing R, Vetter H, Brackmann HH. [The modified Bonn Malmo protocol (MBMP) in the treat- ment of acquired haemophilia A]. Dtsch Med Wochenschr 2006; 131:141-7.
95. Freedman J, Rand ML, Russell O, Davis C, Cheatley PL, Blanchette V, et al. Immunoadsorption may pro- vide a cost-effective approach to management of patients with inhibitors to FVIII. Transfusion 2003; 43:1508-13.
96. Guillet B, Kriaa F, Huysse MG, Proulle V, George C, Tchernia G, et al. Protein A sepharose immunoad- sorption: immunological and haemo- static effects in two cases of acquired haemophilia. Br J Haematol 2001; 114:837-44.
97. Brzoska M, Krause M, Geiger H, Betz C. Immunoadsorption with single- use columns for the management of bleeding in acquired hemophilia A: a series of nine cases. J Clin Apher 2007;22:233-40.
98. Sultan Y, Kazatchkine MD, Maisonneuve P, Nydegger UE. Anti- idiotypic suppression of autoanti- bodies to factor VIII (antihaemophilic factor) by high-dose intravenous gammaglobulin. Lancet 1984;2:765-8.
99. Schwartz RS, Gabriel DA, Aledort LM, Green D, Kessler CM. A prospective study of treatment of acquired (autoimmune) factor VIII inhibitors with high-dose intra- venous gammaglobulin. Blood 1995; 86:797-804.
100. Crenier L, Ducobu J, des Grottes JM, Cerny J, Delaunoit C, Capel P. Low response to high-dose intravenous immunoglobulin in the treatment of acquired factor VIII inhibitor. Br J Haematol 1996;95:750-3.
101. Marie I, Maurey G, Herve F, Hellot MF, Levesque H. Intravenous immunoglobulin-associated arterial and venous thrombosis; report of a series and review of the literature. Br J Dermatol 2006;155:714-21.
102. Nicolaides AN, Breddin HK, Carpenter P, Coccheri S, Conard J, De Stefano V, et al. Thrombophilia and venous thromboembolism. International consensus statement. Guidelines according to scientific evi- dence. Int Angiol 2005;24:1-26.
103. Kyrle PA. High factor VIII and the risk of venous thromboembolism. Hamostaseologie 2003;23:41-4.
104. Geerts WH, Pineo GF, Heit JA, Bergqvist D, Lassen MR, Colwell CW, et al. Prevention of venous thromboembolism: the Seventh ACCP Conference on Antithrombotic and Thrombolytic Therapy. Chest 2004;126 (3 Suppl): 338S-400S.