Journal of Inherited Metabolic Disease

, Volume 29, Issue 2–3, pp 426–430 | Cite as

Liver transplantation for inborn errors of liver metabolism



Liver transplantation brings complete recovery from end-stage liver disease, and full correction of liver based inborn errors of metabolism.


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  1. Cutillo L, Najimi M, Smets F, et al (2006) Safety of living related liver transplantation for progressive familial intrahepatic cholestasis. Pediatr Transplant (in press).Google Scholar
  2. Darwish AA, Sokal E, Stephenne X, et al (2004) Permanent access to the portal system for cellular transplantation using an implantable port device. Liver Transpl 10: 1213–1215.PubMedCrossRefGoogle Scholar
  3. Fox IJ, Chowdhury JR, Kaufman SS, et al (1998) Treatment of the Crigler–Najjar syndrome type I with hepatocyte transplantation. N Engl J Med 338: 1422–1426.PubMedCrossRefGoogle Scholar
  4. Ganschow R, Grabhorn E, Schulz A, et al (2005) Long-term results of basiliximab induction immunosuppression in paediatric liver transplant recipients. Pediatr Transplant 9: 741–745.PubMedCrossRefGoogle Scholar
  5. Goncalves I, Hermans D, Chretien D, et al (1995) Mitochondrial respiratory chain defect: a new etiology for neonatal cholestasis and early liver insufficiency. J Hepatol 23: 290–294.PubMedGoogle Scholar
  6. Jacquemin E, Hermans D, Myara A, et al (1997) Ursodeoxycholic acid therapy in paediatric patients with progressive familial intrahepatic cholestasis. Hepatology 25: 519–523.PubMedCrossRefGoogle Scholar
  7. Jain AB, Marcos A, Pokharna R, et al (2005) Rituximab (chimeric anti-CD20 antibody) for posttransplant lymphoproliferative disorder after solid organ transplantation in adults: long-term experience from a single center. Transplantation 80: 1692–1698.PubMedCrossRefGoogle Scholar
  8. Kaibori M, Egawa H, Inomata Y, et al (1998) Strategy used to overcome graft atrophy in auxiliary partial orthotopic livertransplantation from a living donor for ornithine transcarbamylase deficiency. Transplant Proc 30: 3221–3222.PubMedCrossRefGoogle Scholar
  9. Leonard JV (1995) The management and outcome of propionic and methylmalonic acidaemia. J Inherit Metab Dis 18: 430–434.PubMedCrossRefGoogle Scholar
  10. Lerut JP, Ciccarelli O, Sempoux C, et al (2003) Glycogenosis storage type I diseases and evolutive adenomatosis: an indication for liver transplantation. Transpl Int 16: 879–884.PubMedCrossRefGoogle Scholar
  11. Nagarajan S, Enns GM, Millan MT, et al (2005) Management of methylmalonic acidaemia by combined liver-kidney transplantation. J Inherit Metab Dis 28: 517–524.PubMedCrossRefGoogle Scholar
  12. Reding R, Gras J, Sokal E, et al (2003) Steroid-free liver transplantation in children. Lancet 362: 2068–2070.PubMedCrossRefGoogle Scholar
  13. Reding R, Bourdeaux C, Gras J, et al (2004) The paediatric liver transplantation program at the Université catholique de Louvain. Acta Gastroenterol Belg 67: 176–178.PubMedGoogle Scholar
  14. Rela M, Muiesan P, Vilca-Melendez H, et al (1999) Auxiliary partial orthotopic liver transplantation for Crigler–Najjar syndrome type I. Ann Surg 229: 565–569.PubMedCrossRefGoogle Scholar
  15. Santos Silva EE, Sarles J, Buts JP, et al (1996) Successful medical treatment of severely decompensated Wilson disease. J Pediatr 128: 285–287.PubMedCrossRefGoogle Scholar
  16. Saudubray JM, Touati G, Delonlay P, et al (1999) Liver transplantation in propionic acidaemia. Eur J Pediatr 158(Supplement 2): S65–S69.PubMedCrossRefGoogle Scholar
  17. Scheers I, Bachy V, Stephenne X, et al (2005) Risk of hepatocellular carcinoma in liver mitochondrial respiratory chain disorders. J Pediatr 146: 414–417.PubMedCrossRefGoogle Scholar
  18. Smets F, Sokal EM (2002) Epstein–Barr virus-related lymphoproliferation in children after liver transplant: role of immunity, diagnosis, and management. Pediatr Transplant 6: 280–287.PubMedCrossRefGoogle Scholar
  19. Smets F, Bodeus M, Goubau P, et al (2000a) Characteristics of Epstein–Barr virus primary infection in paediatric liver transplant recipients. J Hepatol 32: 100–104.CrossRefGoogle Scholar
  20. Smets F, Vajro P, Cornu G, et al (2000b) Indications and results of chemotherapy in children with posttransplant lymphoproliferative disease after liver transplantation. Transplantation 69: 982–984.CrossRefGoogle Scholar
  21. Smets F, Latinne D, Bazin H, et al (2002) Ratio between Epstein–Barr viral load and anti-Epstein–Barr virus specific T-cell response as a predictive marker of posttransplant lymphoproliferative disease. Transplantation 73: 1603–1610.PubMedCrossRefGoogle Scholar
  22. Sokal EM, Van Hoof F, Alberti D, et al (1992a) Progressive cardiac failure following orthotopic liver transplantation for type IV glycogenosis. Eur J Pediatr 151: 200–203.CrossRefGoogle Scholar
  23. Sokal EM, Bustos R, Van Hoof F, et al (1992b) Liver transplantation for hereditary tyrosinemia – early transplantation following the patient's stabilization. Transplantation 54: 937–939.Google Scholar
  24. Sokal EM, Lopez-Silvarrey A, Buts JP, et al (1993a) Orthotopic liver transplantation for type I glycogenosis unresponsive to medical therapy. J Pediatr Gastroenterol Nutr 16: 465–467.CrossRefGoogle Scholar
  25. Sokal EM, Ulla L, Harvengt C, et al (1993b) Liver transplantation for familial hypercholesterolemia before the onset of cardiovascular complications. Transplantation 55: 432–433.Google Scholar
  26. Sokal EM, Silva ES, Hermans D, et al (1995) Orthotopic liver transplantation for Crigler–Najjar type I disease in six children. Transplantation 60: 1095–1098.PubMedGoogle Scholar
  27. Sokal EM, Sokol R, Cormier V, et al (1999) Liver transplantation in mitochondrial respiratory chain disorders. Eur J Pediatr 158(Supplement 2): S81–S84.PubMedCrossRefGoogle Scholar
  28. Sokal EM, Smets F, Bourgois A, et al (2003) Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease: technique, safety, and metabolic follow-up. Transplantation 76: 735–738.PubMedCrossRefGoogle Scholar
  29. Stéphenne X, Najimi M, Smets F, et al (2005) Cryopreserved liver cell transplantation controls ornithine transcarbamylase deficient patient while awaiting liver transplantation. Am J Transplant 5: 2058–2061.PubMedCrossRefGoogle Scholar
  30. Stéphenne X, Najimi M, Sibille C, Nassogne M, Smets F, Sokal EM (2006) Sustained engraftment and tissue enzyme activity after liver cell transplantation for argininosuccinate lyase deficiency. Gastroenterology (in press).Google Scholar
  31. Strom SC, Chowdhury JR, Fox IJ (1999) Hepatocyte transplantation for the treatment of human disease. Semin Liver Dis 19: 39–48.PubMedCrossRefGoogle Scholar
  32. Van Maldergem L, Moser AB, Vincent MF, et al (2005) Orthotopic liver transplantation from a living-related donor in an infant with a peroxisome biogenesis defect of the infantile Refsum disease type. J Inherit Metab Dis 28: 593–600.PubMedCrossRefGoogle Scholar
  33. van't Hoff W, McKiernan PJ, Surtees RA, et al (1999) Liver transplantation for methylmalonic acidaemia. Eur J Pediatr 158(Supplement 2): S70–S74.CrossRefGoogle Scholar
  34. Verloes A, Temple IK, Hubert AF, et al (1996) Recurrence of neonatal haemochromatosis in half sibs born of unaffected mothers. J Med Genet 33: 444–449.PubMedGoogle Scholar
  35. Wanty C, Joomye R, Van Hoorebeek N, et al (2004) Fifteen years single center experience in the management of progressive familial intrahepatic cholestasis of infancy. Acta Gastroenterol Belg 67: 313–319.PubMedGoogle Scholar
  36. Wendel U, Saudubray JM, Bodner A, et al (1999) Liver transplantation in maple syrup urine disease. Eur J Pediatr 158(Supplement 2): S60–S64.PubMedCrossRefGoogle Scholar
  37. Yorifuji T, Kawai M, Mamada M, et al (2004) Living-donor liver transplantation for propionic acidaemia. J Inherit Metab Dis 27: 205–210.PubMedCrossRefGoogle Scholar

Copyright information

© SSIEM and Springer 2006

Authors and Affiliations

  1. 1.cliniques St Luc, Département de pédiatrieUniversité catholique de LouvainBrusselsBelgium

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