Lysy PA, Najimi M, Stéphenne X, Bourgois A, Smets F, Sokal EM. Liver cell transplantation for Crigler-Najjar syndrome type I: Update and perspectives. World J Gastroenterol 2008; 14(22): 3464-3470 [PMID: 18567072 DOI: 10.3748/wjg.14.3464]
Corresponding Author of This Article
Etienne M Sokal, Pediatric Hepatology and Cell Therapy, Université Catholique de Louvain, Cliniques Saint Luc, 10 av. Hippocrate, Brussels B-1200, Belgium. sokal@pedi.ucl.ac.be
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Review
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World J Gastroenterol. Jun 14, 2008; 14(22): 3464-3470 Published online Jun 14, 2008. doi: 10.3748/wjg.14.3464
Liver cell transplantation for Crigler-Najjar syndrome type I: Update and perspectives
Philippe A Lysy, Mustapha Najimi, Xavier Stéphenne, Annick Bourgois, Françoise Smets, Etienne M Sokal
Philippe A Lysy, Mustapha Najimi, Xavier Stéphenne, Annick Bourgois, Françoise Smets, Etienne M Sokal, Université Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium
Author contributions: Sokal EM, Smets F, and Lysy PA designed research; Lysy PA, Bourgois A, Smets F and Sokal EM performed research; Lysy PA, Najimi M, Bourgois A, Smets F and Sokal EM analyzed data; and Lysy PA, Stéphenne X, Smets F, Sokal EM wrote the paper.
Correspondence to: Etienne M Sokal, Pediatric Hepatology and Cell Therapy, Université Catholique de Louvain, Cliniques Saint Luc, 10 av. Hippocrate, Brussels B-1200, Belgium. sokal@pedi.ucl.ac.be
Telephone: +32-2-7641387
Fax: +32-2-7648909
Received: December 3, 2007 Revised: March 19, 2008 Accepted: March 26, 2008 Published online: June 14, 2008
Abstract
Liver cell transplantation is an attractive technique to treat liver-based inborn errors of metabolism. The feasibility and efficacy of the procedure has been demonstrated, leading to medium term partial metabolic control of various diseases. Crigler-Najjar is the paradigm of such diseases in that the host liver is lacking one function with an otherwise normal parenchyma. The patient is at permanent risk for irreversible brain damage. The goal of liver cell transplantation is to reduce serum bilirubin levels within safe limits and to alleviate phototherapy requirements to improve quality of life. Preliminary data on Gunn rats, the rodent model of the disease, were encouraging and have led to successful clinical trials. Herein we report on two additional patients and describe the current limits of the technique in terms of durability of the response as compared to alternative therapeutic procedures. We discuss the future developments of the technique and new emerging perspectives.
