25 years in comprehensive care and treatment of haemophilia

Czech version

Authors: A. Bátorová
Authors‘ workplace: Klinika hematológie a transfuziológie LFUK, a SZU, Univerzitná nemocnica, Bratislava
Published in: Transfuze Hematol. dnes,25, 2019, No. 1, p. 50-55.
Category: Review/Educational Papers

Overview

Over the past 25 years, significant progress has been made in the treatment and comprehensive care for haemophilia and other congenital bleeding disorders in Slovakia and the Czech Republic. The era of modern haemophilia treatment began in the early 1990s. The most significant factor that led to a radical change in treatment was the introduction of safe viral-inactivated concentrates and home therapy in all subjects with severe haemophilia. The gradually increasing availability of concentrates, in Slovakia this is currently up to 8.0 IU/kg /year of FVIII, has helped improve comprehensive care, including elective surgery; introduce prophylaxis and manage inhibitors. The significant progress achieved has brought current haemophilia care in our countries to a level comparable with other developed countries in Europe.

Keywords:

haemophilia – comprehensive care – Prophylaxis – inhibitors

Sources

1. Hrubišková K, Filová A, Bátorová A. Dispenzárna starostlivosť o vrodené krvácavé ochorenia na Slovensku. Vnitř Lék 1984;30(7): 633–641.

2. Bátorová A, Filová A, Makai F, et al. Development of haemophilia care in Slovakia – The rapid progress in the 1990´s. Haemophilia 1998;4(3):201.

3. WFH Global Survey 2016. World Federation of Hemophilia, 2017. Dostupné na www: www.wfh.org.

4. Nilsson IM, Hedner U, Ahlberg A. Haemophilia prophylaxis in Sweden. Acta Pediatr Scand 1976;65:129–135.

5. van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long term outcome of individualised prophylactic treatment of children with severe haemophilia. Br J Haematol 2001;107:561–565.

6. 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;357:535–544.

7. Tagliaferri A, Franchini M, Coppola A, et al. Effects of secondary prophylaxis started in adolescent andadult haemophiliacs. Haemophilia 2008;14:945–951.

8. Berntorp E, Dolan G, Hay C, et al. European retrospective study of real-life haemophilia treatment. Haemophilia 2017;23:105–114.

9. Richards M, Altisent C, Batorova A, et al. Should prophylaxis be used in adolescent and adult patients with severe hemophilia. An European survey of practice and outcome data. Haemophilia 2007;13(5):473–479.

10. Evatt BL, Black C, Batorova A, Street A, Srivastava A. Comprehensive care for haemophilia around the world. Haemophilia 2004;10(Suppl. 4):9–13.

11. Makris M, Calizzani G, Fischer K, et al. The European Haemophilia Network (EUHANET). Blood Transfus 2014;12(Suppl 3):s515–s518.

12. EUHANET certified Centres. Dostupné na www: https://www.euhass.org/aspxpages/certcentres.aspx. Last update 16/04/1208.

13. Bátorová A, Jankovičová D, Žarnovičanová M, et al. Národné štandardné postupy pre liečbu hemofílie a iných vrodených koagulopatií na Slovensku. Lek Obzor 2008;7–8:330–340.

14. Odborné usmernenie Ministerstva zdravotníctva Slovenskej republiky na poskytovanie zdravotnej starostlivosti pacientom s hemofíliou a inými vrodenými koagulopatiami. Vestník MZ-SR z dňa 29.5.2009, roč. 57, čiastka 22–24:158–161.

15. Hermans C, Altisent C, Batorova A, et al. Replacement therapy for invasive procedures in patients with haemophilia: Literature review, European survey and recommendations. Haemophilia 2009;15:639–658.

16. Smejkal P, Blatný J, Hluší A, a spol. Konsenzuální doporučení Českého národního hemofilického programu (ČNHP) pro diagnostiku a léčbu pacientů s vrozenou hemofilií a s inhibitorem FVIII/FIX. Transfuze Hematol dnes 2016;22(1):44–54.

17.Hrdličková R, Smejkal P, Blatný J, et al. Konsenzuální doporučení českého národního hemofilického programu (ČNHP) pro zajištění chirurgických a invazivních výkonů u pacientů s hemofilií. Transfuze Hematol dnes 2016;22(1):39–43.

18. Blatný J, Blažek B, Čermáková Z. Konsenzuální doporučení Českého národního hemofilického programu pro diagnostiku a léčbu pacientů s hemofilií. Vydání 2. Transfuze Hematol dnes 2017;23(2):82–99.

19. Colvin BT, Astermark J, Fischer K, et al. on behalf of the IDWG. European principles of haemophilia care. Haemophilia 2008;14(2):361–374.

20. Giangrande PLF, Peyvandi F, O’Mahony B, et al. Kreuth IV: European consensus proposals for treatment of haemophilia with coagulation factor concentrates. Haemophilia 2017;23:370–337.

21. Batorova A, Martinowitz U. Intermittent injections vs. continuous infusion of factor VIII in haemophilia patient undergoing major surgery. Br J Haematol 2000;110:715–720.

22. Holme PA, Tjønnfjord GE, Batorova A. Continuous infusion of coagulation factor concentrates during intensive treatment. Haemophilia 2018;24(1):24–32.

23.Jankovičová D, Šteňo B, Prigancová T, et al. Manažment veľkých operácií u pacientov s hemofíliou A kontinuálnou infúziou a bolusovými injekciami FVIII – farmakokinetický prístup k liečbe. Transfuze Hematol dnes 2017;23(2):61–72.

24. Morfini M, Lee M, Messori A. The design and analysis of half-life and recovery studies for factor VIII and factor IX. Thromb Haemost 1991;66:384–386.

25. Iorio A, Blanchette V, Blatny J, et al. Estimating and interpreting the pharmacokinetic profiles of individual patients with hemophilia A or B using a population pharmacokinetic approach: communication from the SSC of the ISTH. J Thromb Haemost 2017;15:2461–2465.

26. Collins P, Chalmers E, Chowdary P, et al.The use of enhanced half-life coagulation factor concentrates in routine clinical practice: guidance from UKHCDO. Haemophilia 2016;22:487–498.

27. Peyvandi F, Ettingshausen CE, Goudemand J, et al. New findings on inhibitor development: from registries to clinical studies. Haemophilia 2017;23(Suppl.1):4–13.

28. Oldendurg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med 2017;377:809–818.

29. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med 2016;374:2054–2064.

30. Collins PW, Liesner R, Makris M, et al. Treatment of bleeding episodes in hemophilia A complicated by a F VIII inhibitor in patients receiving emicizumab. Interim Guidance from UKHCDO Inhibitor Working Party and Executive Committee. Haemophilia 2018;24:344–347.

31. Batorova A, Jankovicova D, Morongova A, et al. Inhibitors in severe hemophilia A: 25-year experience in Slovakia. Semin Thromb Hemost 2016;42(5):550–562.

32. Calvez T, Chambost H, d‘Oiron R, et al. Analyses of the FranceCoag cohort support differences in immunogenicity among one plasma-derived and two recombinant factor VIII brands in boys with severe hemophilia A. Haematologica 2018;103(1):179–189.

33. Fischer K, Lassila R, Peyvandi F, et al. EUHASS participants. Inhibitor development in haemophilia according to concentrate. Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project. Thromb Haemost 2015;113(5):968–975.

34. Blatny J, Komrska V, Blazek B, et al. Czech National Haemophilia Programme. Inhibitors incidence rate in Czech previously untreated patients with haemophilia A has not increased since introduction of recombinant factor VIII treatment in 2003. Blood Coagul Fibrinolysis 2015;26(6):673–678.

35. Marcucci M, Mancuso EM, Santagostino E, et al. Type and intensity of FVIII exposure on inhibitor development in PUPs with haemophilia A. Thromb Haemost 2015;113:958–967.

36. van den Berg HM, Hashemi SM, Fischer K, et al. Increased inhibitor incidence in severe haemophilia A since 1990 attributable to more low titre inhibitors. Thromb Haemost 2016;115:729–737.

37. Kurnik K, Bidlingmaier C, Engl W, et al. New early prophylaxis regimen that avids immunological danger signals can reduce FVIII inhibitor development. Haematologia 2010;16:256–262.

38. George LA, Sullivan SK, Giermasz A, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med 2017;377(23):2215–2227.

39. Rangarajan S, Walsh L, Perry D, Pasi J. AAV5–Factor VIII gene transfer in severe hemophilia A. N Engl J Med 2017;377:2519–2530.