Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease: Technique, safety, and metabolic follow-up

Etienne M. Sokal, Françoise Smets, Annick Bourgois, Lionel Van Maldergem, Jean Paul Buts, Raymond Reding, Jean Bernard Otte, Veerle Evrard, Dominique Latinne, Marie Françoise Vincent, Anne Moser, Humberto E. Soriano

Research output: Contribution to journalArticle

Abstract

Hepatocyte transplantation is an investigational alternative to orthotopic liver transplantation to treat liver based inborn errors of metabolism. We report successful hepatocyte transplantation in a 4-year-old girl with infantile Refsum disease. Hepatocytes were isolated from the left liver segment of two male donors using a classic two-step perfusion method. Fresh cells were transplanted first and then cryopreserved cells, for a total of 2 billion cells. Total bile acids and abnormal dihydroxycoprostanoïc acid markedly decreased in the patient's serum, indicating resolution of cholestasis and re-population of liver cells. Pipecholic acid decreased by 40% and c26:c22 fatty acid ratio by 36% after 18 months. Donor chromosomes sequences were detected on biopsy posttransplant, indicating engraftment. Hepatocyte transplantation is a safe and promising technique in the treatment of rare inborn errors of metabolism. Future improvements of cell viability and prevention of apoptosis may increase engraftment and subsequent re-population.

Original languageEnglish (US)
Pages (from-to)735-738
Number of pages4
JournalTransplantation
Volume76
Issue number4
DOIs
StatePublished - Aug 27 2003

ASJC Scopus subject areas

  • Transplantation

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    Sokal, E. M., Smets, F., Bourgois, A., Van Maldergem, L., Buts, J. P., Reding, R., Otte, J. B., Evrard, V., Latinne, D., Vincent, M. F., Moser, A., & Soriano, H. E. (2003). Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease: Technique, safety, and metabolic follow-up. Transplantation, 76(4), 735-738. https://doi.org/10.1097/01.TP.0000077420.81365.53