Liver transplantation as potential curative method in severe hemophilia A: case report and literature review

Česká verzia

Authors: Pavel Polák ¹; Petr Smejkal ^1,2; Gabriela Romanová ¹; Jiřina Zavřelová ^1,2; Radomíra Hrdličková ³; Šárka Blahutová ³; Libuše Husová ^4,5; Marta Zvarová ⁶; Miroslav Penka ^1,2
Authors place of work: Oddělení klinické hematologie FN Brno, pracoviště Bohunice ¹; Katedra laboratorních metod LF MU Brno ²; Krevní centrum FN Ostrava ³; Interní gastroenterologická klinika LF MU a FN Brno, pracoviště Bohunice ⁴; Centrum kardiovaskulární a transplantační chirurgie Brno ⁵; Oddělení klinické hematologie FN u sv. Anny Brno ⁶
Published in the journal: Vnitř Lék 2020; 66(5): 85-89
Category: Case Report

Summary

The authors present clinical case of orthotopic liver transplantation for cirhosis due to chronic viral hepatitis C in a subject with severe hemophilia A. Preoperatively performed pharmacokinetic study with recombinant F VIII confirmed satisfactory in vivo recovery of 2.1 %. A bolus application of 52 units F VIII/kg body weight with target F VIII activity over 100.0 % was administred shortly before the transplantation started. Totally, 30 000 units of recombinant F VIII, 3 thrombocyte concentrates, 2 erythrocyte concentrates, 5 units of virally inactivated plasma, 1 unit of fresh frozen plasma and 3 500 antithrombin units were used. There were no perioperative or postoperative bleeding complications, F VIII substitution was stopped on postoperative day 3. The patient was discharged on twentieth postoperative day.

Keywords:

liver transplantation – hemophilia A – chronic viral hepatitis C

Zdroje

1. Matýšková M, Penka M. Hemofilie. Vnitř Lék 1991; 37: 397–403.

2. Maier KP. Hepatitis‑Hepatitisfolgen. 6. Aufl. Bern: Verlag Hans Huber 2010, 457 s.

3. Chorba TL, Holman RC, Clarke MJ, et al. Effects of HIV infection on age and cause of death for persons with hemophilia A in the United States. Am J Hematol 2001; 66: 229–240.

4. Lewis JH, Bontempo FA, Spero JA, et al. Liver transplantation in a hemophiliac. N Engl J Med 1985; 312: 1189–1190.

5. Norman JC, Lambilliotte JP, Kojima Y, et al. Antihemophilic factor release by perfused liver and spleen: relationship to hemophilia. Science 1967; 158: 1060–1061.

6. Webster WP, Zukoski CF, Hutchin P, et al. Plasma factor VIII synthesis and control as revealed by canine organ transplantation. Am J Physiol 1971; 220: 1147–1154.

7. Stel HV, van der Kwast TH, Veerman EC. Detection of factor VIII/coagulant antigen in human liver tissue. Nature 1983; 303: 530–532.

8. Webster WP, Mandel SR, Reddick RL, et al. Orthotopic liver transplantation in canine hemophilia B. Am J Physiol 1974; 226: 496–500.

9. Kleeberger W, Rothamel T, Glockner S, et al. High frequency of epithelial chimerism in liver transplants demonstrated by microdissection and STR‑analysis. Hepatology 2002; 35: 110–116.

10. Zanolini D, Merlin S, Feola M et, al. Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A. Haematologica 2015; 100: 881–892.

11. Groth CG, Ringdén O. Transplantation in relation to the treatment of inherited disease. XXXXXXXXX XXXX; XXX: XXX–XXX.

12. Gupta E, Finn L, Johns G, et al. Correction of factor XI deficiency by liver transplantation. Blood Coagul Fibrinolysis 2015; 26: 961–963.

13. Gallastegui N, Kimble EL, Harrington TJ. Resolution of fibrinogen deficiency in a patient with congenital afibrinogenemia after liver transplantation. Haemophilia 2016; 22: e36–e79.

14. Alonso Madrigal C, Dobón Rebollo M, Laredo de la Torre V, et al. Trasplante hepático en hemofilia A y enfermedad de von Willebrand tipo 3. Manejo perioperatorio y evolución postrasplante. Rev Esp Enferm Dig 2018; 110: 522–526.

15. Suzuki Y, Kakisaka K, Matsumoto T, et al. Orthotopic liver transplantation for haemophilia A may not always lead to a phenotypic cure of haemophilia A: A case report. Haemophilia 2018; 24: e420-e422.

16. Murthy V, Murray D, Hebballi S, et al. Outcome of liver transplantation in patients with hereditary bleeding disorders: a single centre UK experience. Haemophilia 2016; 22: e139-e144.

17. Khakhar AK, Chan NG, Allan DS, et al. Catastrophic microangiopathy induced by high‑titre factor VIII inhibitors after liver transplantation for haemophilia A with cirrhosis. Haemophilia 2005; 11: 623–628.

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

19. Merlen C, Zourikian N, Bonnefoy A, et al. Effect of ABO blood group on haemostatic parameters in severe haemophilia A patients performing acute moderate‑intensity exercise. Blood Coagul Fibrinolysis 2018; 29: 626–635.

20. Shapiro AD, Korth‑Bradley J, Poon MC. Use of pharmacokinetics in the coagulation factor treatment of patients with haemophilia. Haemophilia 2005; 11: 571–582.

21. Björkman S, Folkesson A, Berntorp E. In vivo recovery of factor VIII and factor IX: intraand interindividual variance in a clinical setting. Haemophilia 2007; 13: 2–8.

22. Hazendonka HCAM, van Moorta I, Mathô RAA, et al. Setting the stage for individualized therapy in hemophilia: What role can pharmacokinetics play? Blood Rev 2018; 32: 265–271.

23. Togashi J, Akamatsu N, Tanaka T, et al. Living donor liver transplantation for hemophilia with special reference to the management of perioperative clotting factor replacement. Liver Transplant 2016; 22: 366–370.

24. Ragni MV, Devera ME, Roland ME, et al. Liver transplant outcomes in HIV+ haemophilic men. Haemophilia 2013; 19: 134–140.

25. Yokoyama S, Bartlett A, Dar FS, et al. Outcome of liver transplantation for haemophilia. HPB (Oxford) 2011; 13: 40–45.

26. Goldmann G, Zeitler H, Marquardt N, et al. Long‑term outcome of liver transplantation in HCV/HIV coinfected haemophilia patients. A single centre study of 10 patients. Hamostaseologie 2015; 35: 175–180.

27. Park YK, Kim HC, Kim BW, et al. Auxiliary partial orthotopic liver transplantation as a treatment for hemophilia a: a case report. Transplant Proc 2015; 47: 161–164.

28. Charvát J, Vychodil P, Adamec M, et al. Vyléčení těžké formy hemofilie A transplantací jater – kazuistika. Trombóza a hemostáza. Hradec Králové: HK Credit 2007, 114 s.