English Polski
Tom 16, Nr 4 (2023)
Artykuł przeglądowy
Opublikowany online: 2024-02-09

dostęp otwarty

Wyświetlenia strony 133
Wyświetlenia/pobrania artykułu 63
Pobierz cytowanie

Eksport do Mediów Społecznościowych

Eksport do Mediów Społecznościowych

Mimetyki aktywnego czynnika VIII (FVIIIa) i ich wpływ na testy laboratoryjne hemostazy

Edyta Odnoczko1
Journal of Transfusion Medicine 2023;16(4):269-277.

Streszczenie

Wprowadzenie do leczenia hemofilii A długodziałających, podskórnie podawanych mimetyków naśladujących aktywny czynnik VIII (FVIIIa) jest bez wątpienia przełomem w leczeniu profilaktycznym tej grupy chorych. Nowatorski charakter i sposób działania cząsteczek mimetyków FVIIIa wiąże się ze znaczącymi implikacjami w dotychczasowych algorytmach laboratoryjnych  monitorowania leczenia hemofilii. Monitorowanie stężenia leku w celu dostosowania dawki nie jest wymagane, jednak w praktyce klinicznej kontrolowanie laboratoryjne hemostazy może być przydatne na przykład w przypadku konieczności przeprowadzenia interwencji chirurgicznej lub wystąpienia krwawień mimo stosowanej profilaktyki. Wówczas świadomość wpływu stosowania terapii mimetykami FVIIIa na testy laboratoryjne hemostazy jest warunkiem koniecznym do sprawnego posługiwania się nimi i interpretowania uzyskanych wyników.

Artykuł dostępny w formacie PDF

Pokaż PDF Pobierz plik PDF

Referencje

  1. Lewandowska M, Nasr S, Shapiro AD. Therapeutic and technological advancements in haemophilia care: Quantum leaps forward. Haemophilia. 2022; 28 Suppl 4: 77–92.
  2. Windyga J, Chojnowski K, Klukowska A, et al. Część I: Wytyczne postępowania w hemofilii A i B niepowikłanej inhibitorem czynnika VIII i IX (wydanie zaktualizowane). Acta Haematologica Polonica. 2016; 47(2): 86–114.
  3. Alcedo Andrade PE, Mannucci PM, Kessler CM. Emicizumab: the hemophilia A game changer. Haematologica. 2023 [Epub ahead of print].
  4. Müller J, Miesbach W, Prüller F, et al. Standing Commission Labor (STAEKOLA) of the Society of Thrombosis and Haemostasis Research (GTH). An Update on Laboratory Diagnostics in Haemophilia A and B. Hamostaseologie. 2022; 42(4): 248–260.
  5. Windyga J, Chojnowski K, Klukowska A, et al. Część I: Wytyczne postępowania w hemofilii A i B niepowikłanej inhibitorem czynnika VIII i IX (wydanie zaktualizowane). Acta Haematol Pol. 2016; 47(2): 86–114.
  6. Persson P, Amstrup AB, Coester HV, et al. Mim8, a novel factor VIIIa mimetic bispecific antibody, shows favorable safety and pharmacokinetics in healthy adults. Res Pract Thromb Haemost. 2023; 7(6): 102181.
  7. Ozelo MC, Yamaguti-Hayakawa GG. Impact of novel hemophilia therapies around the world. Res Pract Thromb Haemost. 2022; 6(3): e12695.
  8. Jenkins PV, Bowyer A, Burgess C, et al. Laboratory coagulation tests and emicizumab treatment A United Kingdom Haemophilia Centre Doctors' Organisation guideline. Haemophilia. 2020; 26(1): 151–155.
  9. Windyga J, Chojnowski K, Klukowska A, et al. Emicizumab (Hemlibra®) in hemophilia A patients with inhibitors against factor VIII — guidelines of the Group for Haemostasis of the Polish Society of Haematology and Transfusion Medicine. J Transf Med. 2020; 13(3): 165–175.
  10. Lippi G, Favaloro EJ. Emicizumab (ACE910): Clinical background and laboratory assessment of hemophilia A. Adv Clin Chem. 2019; 88: 151–167.
  11. Ellsworth P, Ma A. Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates? Hematology Am Soc Hematol Educ Program. 2021; 2021(1): 219–225.
  12. Peyvandi F, Kenet G, Pekrul I, et al. Laboratory testing in hemophilia: Impact of factor and non-factor replacement therapy on coagulation assays. J Thromb Haemost. 2020; 18(6): 1242–1255.
  13. Lowe A, Kitchen S, Jennings I, et al. Effects of Emicizumab on APTT, FVIII assays and FVIII Inhibitor assays using different reagents: Results of a UK NEQAS proficiency testing exercise. Haemophilia. 2020; 26(6): 1087–1091.
  14. Bowyer AE, Lowe AE, Tiefenbacher S. Laboratory issues in gene therapy and emicizumab. Haemophilia. 2021; 27 Suppl 3: 142–147.
  15. Bowyer AE, Kitchen S, Ezban M. The effect of a next generation factor VIII mimetic bispecific antibody (Mim8) on assays of factor VIII activity and thrombin generation. J Thromb Haemost. 2023; 21(3): 480–487.
  16. Lauritzen B, Bjelke M, Björkdahl O, et al. A novel next-generation FVIIIa mimetic, Mim8, has a favorable safety profile and displays potent pharmacodynamic effects: Results from safety studies in cynomolgus monkeys. J Thromb Haemost. 2022; 20(6): 1312–1324.
  17. Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021; 138(14): 1258–1268.
  18. Yamaguchi K, Soeda T, Sato M, et al. Pharmacology and Pharmacokinetics of NXT007; Emicizumab-Based Engineered Fixa/Fx Bispecific Antibody with Improved Properties. Blood. 2020; 136(Supplement 1): 19–19.
  19. Teranishi-Ikawa Y, Soeda T, Koga H, et al. A bispecific antibody NXT007 exerts a hemostatic activity in hemophilia A monkeys enough to keep a nonhemophilic state. J Thromb Haemost. 2023 [Epub ahead of print].
  20. López-Jaime FJ, Benítez O, Díaz Jordán BL, et al. Expert opinion paper on the treatment of hemophilia a with emicizumab. Hematology. 2023; 28(1): 2166334.
  21. Tripodi A, Chantarangkul V, Novembrino C, et al. Advances in the Treatment of Hemophilia: Implications for Laboratory Testing. Clin Chem. 2019; 65(2): 254–262.
  22. Holstein K, Albisetti M, Bidlingmaier C, et al. ‘Ständige Kommission Hämophilie’ (Haemophilia board) of the German, Swiss Austrian Society for Thrombosis Haemostasis Research (GTH). Practical Guidance of the GTH Haemophilia Board on the Use of Emicizumab in Patients with Haemophilia A. Hamostaseologie. 2020; 40(5): 561–571.
  23. Müller J, Miesbach W, Prüller F, et al. Standing Commission Labor (STAEKOLA) of the Society of Thrombosis and Haemostasis Research (GTH). An Update on Laboratory Diagnostics in Haemophilia A and B. Hamostaseologie. 2022; 42(4): 248–260.
  24. Lenting PJ. Laboratory monitoring of hemophilia A treatments: new challenges. Blood Adv. 2020; 4(9): 2111–2118.
  25. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020; 26 Suppl 6: 1–158.
  26. Castaman G, Santoro C, Coppola A, et al. ad hoc Working Group. Emergency management in patients with haemophilia A and inhibitors on prophylaxis with emicizumab: AICE practical guidance in collaboration with SIBioC, SIMEU, SIMEUP, SIPMeL and SISET. Blood Transfus. 2020; 18(2): 143–151.
  27. Bowyer AE, Hickey K, Kitchen S, et al. A next generation FVIII mimetic bispecific antibody, Mim8, the impact on non-factor VIII related haemostasis assays. Haemophilia. 2023; 29(6): 1633–1637.
  28. Adamkewicz JI, Chen DC, Paz-Priel I. Effects and Interferences of Emicizumab, a Humanised Bispecific Antibody Mimicking Activated Factor VIII Cofactor Function, on Coagulation Assays. Thromb Haemost. 2019; 119(7): 1084–1093.
  29. Nougier C, Jeanpierre E, Ternisien C, et al. With contributions from the Working Group on Biology of Haemorrhagic Disorders (BIMHO, Under the Aegis of GFHT). Emicizumab treatment: Impact on coagulation tests and biological monitoring of haemostasis according to clinical situations (BIMHO group proposals). Eur J Haematol. 2020; 105(6): 675–681.
  30. Bowyer AE, Maclean RM, Kitchen S. The combination of emicizumab and recombinant factor VIII in plasma: Which assays can we use for accurate measurement? Int J Lab Hematol. 2023; 45(3): 368–376.
  31. Escuriola-Ettingshausen C, Auerswald G, Königs C, et al. Optimizing the management of patients with haemophilia A and inhibitors in the era of emicizumab: Recommendations from a German expert panel. Haemophilia. 2021; 27(3): e305–e313.
  32. Bowyer A, Kitchen S, Maclean R. Effects of emicizumab on APTT, one-stage and chromogenic assays of factor VIII in artificially spiked plasma and in samples from haemophilia A patients with inhibitors. Haemophilia. 2020; 26(3): 536–542.
  33. Lund J, Jensen K, Burnier L, et al. In vitro effects of combining Mim8 with factor VIII, FVIIa, and activated prothrombin complex concentrates in thrombin generation assays. J Thromb Haemost. 2023; 21(6): 1493–1502.



Journal of Transfusion Medicine