Journal of Inherited Metabolic Disease

, Volume 29, Issue 2, pp 431–435

Hepatocyte transplantation for liver-based metabolic disorders

SSIEM SYMPOSIUM 2005

DOI: 10.1007/s10545-006-0245-8

Cite this article as:
Dhawan, A., Mitry, R.R. & Hughes, R.D. J Inherit Metab Dis (2006) 29: 431. doi:10.1007/s10545-006-0245-8

Summary

Hepatocyte transplantation is being investigated as an alternative to orthotopic liver transplantation in patients with liver-based metabolic disorders. The progress made in this field to date is reviewed. Protocols have been developed using collagenase perfusion to isolate human hepatocytes from unused donor liver tissue. Hepatocytes with a high viability can often be obtained and can be cryopreserved for later use, though with loss of function on thawing. For clinical use, hepatocytes must be prepared in clean GMP conditions with cells meeting criteria of function and lack of microbial contamination before patient use. Hepatocytes are infused intraportally into the patient's liver, where a proportion of cells will engraft and replace the deficient metabolic function without the need for major surgery. Twenty patients have now received hepatocyte transplantation, including eight children at King's College Hospital. There was a range of aetiologies of liver disease: familial hypercholesterolaemia, Crigler–Najjar syndrome type 1, urea cycle defects, infantile Refsum disease, glycogen storage disease type Ia, inherited factor VII deficiency and progressive familial intrahepatic cholestasis type 2. Clinical improvement and partial correction of the metabolic abnormality was observed in most cases. Considerable progress has been made in developing the technique, but hepatocyte transplantation is limited by the available supply of liver tissue. Hepatocytes derived from stem cells could provide alternative sources of cells in the future.

Copyright information

© SSIEM and Springer 2006

Authors and Affiliations

  • Anil Dhawan
    • 1
    • 2
  • Ragai R. Mitry
    • 2
  • Robin D. Hughes
    • 2
  1. 1.Paediatric Liver ServiceKing's College HospitalLondonUK
  2. 2.Institute of Liver StudiesKing's College London School of Medicine, King's College HospitalLondonUK

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