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Consensual guidelines of the Czech National Haemophilia Programme (CNHP) for the diagnosis and treatment of patients with congenital haemophilia and inhibitors to FVIII/FIX


Authors: P. Smejkal 1;  J. Blatný 2;  A. Hluší 3;  R. Hrdličková 4;  V. Komrska 5;  M. Penka 1;  J. Šlechtová 6;  J. Blatný 2;  B. Blažek 7;  Z. Čermáková 4;  Z. Černá 8;  P. Ďulíček 9;  J. Hak 10;  A. Hluší 3;  R. Hrdličková 4;  V. Komrska 5;  P. Ovesná 11;  D. Pospíšilová 12;  D. Procházková 13;  P. Smejkal 1;  J. Šlechtová 6;  P. Timr 14;  J. Ullrychová 15;  I. Vonke 16;  L. Walterová 17;  M. Penka 1
Authors‘ workplace: Oddělení klinické hematologie, Fakultní nemocnice Brno 1;  Oddělení dětské hematologie, Fakultní nemocnice Brno 2;  Hemato-onkologická klinika, Fakultní nemocnice Olomouc 3;  Klinika hematoonkologie a Krevní centrum, Fakultní nemocnice Ostrava 4;  Klinika dětské hematologie a onkologie, Fakultní nemocnice v Motole, Praha 5;  Ústav klinické biochemie a hematologie, Fakultní nemocnice Plzeň 6;  Klinika dětského lékařství, Fakultní nemocnice Ostrava 7;  Dětská klinika, Fakultní nemocnice Plzeň 8;  IV. Interní hematologická klinika, Fakultní nemocnice Hradec Králové 9;  Dětská klinika, Fakultní nemocnice Hradec Králové 10;  Institut biostatistiky a analýz Lékařské fakulty, Masarykova univerzita, Brno 11;  Dětská klinika, Fakultní nemocnice Olomouc 12;  Dětská klinika, Krajská zdravotní a. s., Masarykova nemocnice v Ústí nad Labem 13;  Dětské oddělení, Nemocnice České Budějovice 14;  Oddělení klinické hematologie, Krajská zdravotní a. s., Masarykova nemocnice v Ústí nad Labem 15;  Oddělení klinické hematologie, Nemocnice České Budějovice 16;  Oddělení klinické hematologie, Krajská nemocnice Liberec 17
Published in: Transfuze Hematol. dnes,22, 2016, No. 1, p. 44-54.
Category: Comprehensive Reports, Original Papers, Case Reports

Pozn:
*Pracovní skupina ČNHP pro standardy
**Jménem Koordinační rady a Výkonné rady ČNHP

Overview

The aim of these guidelines is to set-up and standardized diagnostic as well as therapeutic approaches to patients suffering from congenital haemophilia and factor VIII or factor IX inhibitors.

Key words:
haemophilia – guidelines – treatment – inhibitor – surgery


Sources

1. Astermark J. Basic aspects of inhibitors to factors VIII and IX and the influence of non-genetic risk factors. Haemophilia 2006; 12 (Suppl. 6): 8–14.

2. Kasper C, Aledort L, Counts R, et al. A more uniform measurement of factor VIII inhibitors. Thrombos Diathes Haemorrh 1975; 34: 869–872.

3. White GC, Rozendal F, Aledort L, et al. Definitions in Hemophilia. Thromb Haemost 2001; 85: 560.

4. Kasper C. Human factor VIII for bleeding in patients with inhibitors. Vox Sang 1999; 77 (Suppl 1): 47–48.

5. Astermark J, Morado M, Rocino A, et al. Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors. Haemophilia 2006; 12: 363–371.

6. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003; 9: 418–435.

7. Blatny J, Komrska V, Blazek B, Penka M, Ovesna P. Czech National Haemophilia Programme. Blood Coagul Fibrinolysis 2015; 26(6): 673–678.

8. DiMichele DM, Hoots WK, Pipe SW, et al. International workshop on immune tolerance induction: consensus recommendations. Haemophilia 2007; 13(Suppl. 1): 1–22.

9. Astermark J. Overview of inhibitors. Semin Hematol 2006; 43 (Suppl. 4): 3–7.

10. Darby SC, Keeling DM, Spooner RJD, et al. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977–99. J Thromb Haemost 2004; 2: 1047–1054.

11. Peerlinck K, Jacquemin M. Characterictics of inhibitors in mild/moderate haemophilia A. Haemophilia 2006; 12 (Suppl. 6): 43–47.

12. Abbonizio F, Giampaolo A, Coppola A, et al. Therapeutic management and cost of severe haemophilia A patients with inhibitors in Italy. Haemophilia 2014; 20: e243–e250.

13. Hay CRM. The epidemiology of factor VIII inhibitors. Haemophilia 2006; 12 (Suppl. 6): 23–29.

14. Gouw SC, van der Bom JG, van den Berg HM. Treatment–related risk factors of inhibitor development in previously untreated pa-tients with hemophilia A: the CANAL cohort study. Blood 2007; 109: 4648–4654.

15. Calvez T, Chambost H,Claeyssens-Donadel S, et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A. Blood 2014; 124(23): 3398–3408.

16. Collins PW, Palmer BP, Chalmers AE, et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000–2011. Blood 2014; 124(23): 3389–3397.

17. Fischer K, Lassila R, Peyvandi F, et al. Inhibitor development in hae-mophilia according to concentrate. Four-year results from the European HAemophila Safety Surveilance (EUHASS) project. Thromb Haemost 2015; 113: 968–975.

18. Xi M, Makris M, Marcuci M, et al. Inhibitor development in previously treated hemophilia A patients: a systematic review, meta-analysis, and meta-regression. J Thromb Haemost 2013;11: 1655–1662.

19. Giles AR, Verbruggen B, Rivard GE, et al. A detailed comparison of the performance of the standard versus the Nijmegen modification of the Bethesda assay in detecting factor VIII:C inhibitors in the haemophilia A population of Canada. Thromb Haemost 1998; 79: 872–875.

20. DiMichele D. Inhibitor treatment in haemophilias A and B: inhibitor diagnosis. Haemophilia 2006; 12 (Suppl. 6): 37–42.

21. Verbruggen B, Novakova I, Wessels H, et al. The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitor. Improved specifity and reliability. Thromb Haemost 1995; 73: 247–251.

22. Gringeri A, Mannucci PM. Italian guidelines for the diagnosis and treatment of patients with haemophilia and inhibitors. Haemophilia 2005; 11: 611–619.

23. 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.

24. Collins PW, Chlamers E, Hart DP, et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). Br J Haematol 2013; 160: 153-170.

25. Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia 2013;19: e1–e47.

26. Lusher JM. Natural history of inhibitor development in children with severe hemophilia A treated with factor VIII products. In: Lee CA, Berntorp E, Hoots WK. Textbook of Hemophilia. 1. vyd. Malden, Oxford, Carlton: Blackwell Publishing 2005: 34–38.

27. Astermark J. Why do inhibitors develop? Principles of and factors influencing the risk for inhibitor development in haemophilia. Haemophilia 2006; 12(Suppl. 3): 52–60.

28. Astermark J, Altisent C, Batorova A, et al. Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report. Haemophilia 2010; 16: 747–766.

29. Oldenburg J, Pavlova A. Genetic risk factors for inhibitors to factors VIII and IX. Haemophilia 2006; 12(Suppl. 6): 15–22.

30. Goudemand J, Rothschild C, Demiguel V, et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophila A. Blood 2006; 107: 46–51.

31. Peerlinck K, Hermans C. Epidemiology of inhibitor formation with recombinant factor VIII replacement therapy. Haemophilia 2006; 12: 579–590.

32. Gouw SC, van der Born JG, Auerswald G, et al. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood 2007; 109: 4693–4697.

33. Batorova A, Holme P, Gringeri A, et al. Continuous infusion in haemophilia: current practice in Europe. Haemophilia 2012;18: 753–759.

34. Eckhardt EL, van Velzen AS, Peters M, et al. Factor VIII gene (F8) mutation risk of inhibitor development in nonsevere hemophilia A. Blood 2013; 122(11): 1954–1962.

35. Auerswald G, Bidlingmaier C, Kurnik K. Early prophylaxis/FVIII tolerization that avoids immunological ganger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients – long-term follow-up and continuing experience. Haemophilia 2012; 18: e18–e20.

36. Eckhardt CL, Menke LA, van Ommen CH, et al. Intensive peri-operative use of factor VIII and the Arg593® Cys station are risk factors for inhibitor development in mild/moderate hemophilia A. J Thromb Haemost 2009; 7: 930–937.

37. Chalmers EA, Brown SA, Keeling D, et al. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007; 13: 149–155.

38. Mancuso ME, Mannucci PM, Rocino A, et al. Source and purity of factor VIII produst as risk factors for inhibitor development in patients with hemophilia A. J Thromb Haemost 2012; 10: 781–790.

39. Iorio A, Halimeh S, Holzhauer S, et al. Rate of inhibitor development in previously untreated hemophila A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost 2010; 8: 1256–1265.

40. Franchini M, Tagliaferri A, Mengoli C, Cruciani M. Cumulative inhibitor incidence in previously untreated patient with severe hemophilia A treated with plasma-derived versus recombinant factor VIII concentrates: A critical systemic review. Critical Reviews in Oncology/Hematology 2012; 8: 82–93.

41. Jones ML, Wight J, Paisley S, Knight C. Control of bleeding in patients with haemophilia A with inhibitors: a systematic review. Haemophilia 2003; 9: 464–520.

42. Teitel J, Berntorp E, Collins P, et al. A systematic approach to controlling problem bleeds in patients with severe congenital haemophilia A and high-titre inhibitors. Haemophilia 2007; 13: 256–263.

43. Mehta R, Parameswaran R, Shapiro AD. An overview of the history, clinical practice concerns, comparative studies and strategies to optimize therapy of bypassing agents. Haemophilia 2006; 12(Suppl. 6): 54–61.

44. Astermark J, Rocino A, von Depka M, et al. Current use of by-passing agents in Europe in the management of acute bleeds in patients with haemophilia and inhibitors. Haemophilia 2007; 13: 38–45.

45. Key NS. Inhibitors in congenital coagulation disorders. Br J Haematol 2004; 127: 379–391.

46. Santagostino E, Mancuso ME, Rocino A, et al. 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.

47. Kavakli K, Makris M, Zulfikar B, et al. Home treatment of haemarthroses using a single dose regiment of recombinant activated factor VII in patiens with haemophilia and inhibitors. Thromb Haemost 2006; 95: 600–605.

48. 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: 502–507.

49. 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.

50. Young G, McDaniel M, Nugent DJ. Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors. Haemophilia 2005; 11: 203–207.

51. Valentino LA, Cooper DL, Goldstein B. Surgical experience with rVIIa (NovoSeven) in congenital haemophilia A nad B patiens with inhibitors to factors VIII or IX. Haemophilia 2011; 17: 579–589.

52. Quintana-Molina M, Martínez-Bahamonde F, González-García E, et al. Surgery in haemophilic patiens with inhibitor: 20 years of experience. Haemophilia 2004; 10(Suppl. 2): 30–40.

53. Rodriguez-Merchan EC, Rocino A, Ewenstein B, et al. Consensus perspectives on surgery in haemophilia patients with inhibitors: summary statement. Haemophilia 2004; 10(Suppl. 2): 50–52.

54. Rodriguez-Merchan EC, Wiedel JD, Salony T, et al. Elective orthopaedic surgery for inhibitor patients. Haemophilia 2003; 9: 625–631.

55. Giangrande PLF, Wilde JT, Madan B, et al. Consensus protocol for the use of recombinant activated factor VII(eptacog alfa (activated); NovoSeven) in elective orthopaedic surgery in haemophilic patients with inhibitors. Haemophilia 2009; 15: 501–508.

56. Kulkarni R. Comprehensive care of the patient with haemophilia and inhibitors undergoing surgery: practical aspects. Haemophilia 2013; 19: 2–10.

57. Obergfell A, Auvinen MK, Mathew P. Recombinant activated factor VII for haemophilia patients with inhibitors undergoing orthopaedic surgery: a review of the literature. Haemophilia 2008; 14: 233–241.

58. Ewenstein BM. Continuous infusion of recombinant factor VIIa: Continue or not? Thromb Haemost 2001: 856: 942–944.

59. Negrier C, Gomperts ED, Oldenburg J. The history of FEIBA: a lifetime of success in the treatment of haemophilia complicated by an inhibitor. Haemophilia 2006; 12 (Suppl. 5): 4–13.

60. Tran HTT, Sorensen B, Rea CJ, et al. Tranexamic acid as adjunct therapy to bypassing agents in haemophilia A patients with inhibitors. Haemophilia 2014; 20: 369–375.

61. Rangarajan S, Austin S, Goddard NJ, et al. Consensus recommendation for the use of FEIBA® in haemophilia A patients with inhibitors undergoing elective orthopaedic and non-orthopaedic surgery. Haemophilia 2013; 19: 294–303.

62. Young G, Cooper DL, Gut RZ. Dosing and effectiveness of recombinant activated factor VII (rFVIIA) in congenital haemophilia with inhibitors by bleed type and location: the experience of the Haemophilia and Thrombosis Research Society (HTRS) Registry (2004-2008). Haemophilia 2012; 18: 990–996.

63. Chambost H, Santagostino E, Laffan M, et al. Real-world outcomes with recombinant factor VIIa treatment of acute bleeds in haemophilia patients with inhibitors: results from the international ONE registry. Haemophilia 2013; 571–577.

64. Birschmann I, Klamroth R, Eichler H, et al. Results of the WIRK prospective, non-interventional observational study of recombinant activated factor VII (rFVIIa) in patients with congenital haemophilia with inhibitors and other bleeding disorders. Haemophilia 2013; 19: 679–685.

65. 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: 546–551.

66. Schneiderman J, Rubin E, Nugent DJ, Zouny G. Sequential therapy with activated prothrombin complex concentrates and recombinant FVIIa in patients with severe haemophilia and inhibitors: update of our previous experience. Haemophilia 2007; 13: 244–248.

67. Gomperts ED, Astenmark J, Gringeri A, Teitel J. From theory to practice: applying current clinical knowledge and treatment strategies to the care of hemophilia A patients with inhibitors. Blood Rew 2008; 22 (Suppl. 1): 1–11.

68. Gringeri A, Fischer K, Karafoulidou A, et al. Sequential combined bypassing therapy is safe and effective in the treatment of unresponsive bleeding in adults and children with haemophilia and inhibitors. Haemophilia 2011; 17: 630–635.

69. Kasper CK. Diagnosis and management of inhibitors to factors VIII and IX. World Federation of Hemophilia 2004.

70. Bátorová A, Jankovičová D, Žarnovičanová M, et al. Národné štandardné postupy pre liečbu hemofilie a iných vrodených koagulopatií v Slovenskej republike. Lek Obz 2008; 56: 330–340.

71. Barrowcliffe TW. Monitoring inhibitor patients with the right assays. Semin Hematol 2008; 45 (Suppl. 1): 25–30.

72. Dargaud Y, Sorensen B, Shima M, et al. Global Haemostasis and point of care testing. Haemophilia 2012; 18 (Suppl. 4): 81–88.

73. Leissinger C, Gringeri A, Antmen B, et al. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med 2011; 365: 1684–1692.

74. Young G, Auerswald G, Jimenez-Yuste V, et al. When should prophylaxis therapy in inhibitor patients be consider? Haemophilia 2011; 17: e849–e857.

75. Antunes SV, Tangada S, Stasyshyn O, et al. Randomized comparison of propfylaxis and on-demand regress FEIBA NF in the treatment of haemophilia A and B with inhibitor. Haemophilia 2014; 20: 65–72.

76. Freiburghaus C, Berntorp E, Ekman M, et al. Immunoadsorption for removal of inhibitors: update on treatments in Malmö-Lund between 1980 and 1995. Haemophilia 1998; 4: 16–20.

77. Jansen M, Schmaldienst S, Banyal S, et al. Treatment of coagulation inhibitors with extracorporeal immunoadsorption (Ig-Theasorb). Br J Haematol 2001; 112: 91–97.

78. Nilsson IM, Berntorp E, Freiburghaus Ch. Treatment of patients with factor VIII and IX inhibitors. Thromb Haemost 1993; 70(1): 56–59.

79. DiMichele DM. Immune tolerance: critical issues of factor dose, purity and treatment complications. Haemophilia 2006; 12(Suppl. 6): 81–86.

80. Hay CRM, Recht M, Carcao M, Reipert B. Current and Future Approaches to Inhibitor Management and Aversion. Semin Tromb Hemost 2006; 32(Suppl. 2): 15–21.

81. Hay CRM, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335–1344.

82. Wight J, Paisley S, Knight C. Immune tolerance induction in patiens with haemophilia with inhibitors: a systematic review. Haemophilia 2003; 9: 436–463.

83. Lim MY, Nielsen B, Lee K, et al. Rituxibam as first-line treatment for the management of adult patients with non-severe hemophilia A and inhibitors. J Thromb Haemost 2014; 12: 897–901.

84. Castaman G, Bonetti E, Messina M, Morfini M, Rocino A, Scaraggi FA, Tagariello G on behalf of Italian Association of Hemophilia Centers. Inhibitors in haemophilia B: the Italian experience. Haemophilia 2013; 19: 686–690.

85. Batorova A, Morongova A, Tagariello G, Jankovicova D, Prigancova T, Horakova J. Challenges of the management of hemophilia B with inhibitor. Semin Thromb Hemost 2013; 39(7):767–771.

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