Liver cell transplantation for the treatment of inborn errors of metabolism

• Published in: Journal of inherited metabolic disease Vol. 31; no. 2; pp. 164 - 172
• Main Authors: Meyburg, J., Hoffmann, G. F.
• Format: Journal Article Conference Proceeding
• Language: English
• Published: Dordrecht Springer Netherlands 01.04.2008; Springer; Blackwell Publishing Ltd
• Subjects: Adolescent; Biochemistry; Biological and medical sciences; Child; Child, Preschool; Genetic Therapy - adverse effects; Genetic Therapy - methods; Graft Rejection - etiology; Graft Survival; Hepatocytes - transplantation; Human Genetics; Humans; Infant; Infant, Newborn; Internal Medicine; Liver - enzymology; Liver - surgery; Liver Transplantation - adverse effects; Liver Transplantation - methods; Liver, biliary tract, pancreas, portal circulation, spleen; Medical genetics; Medical sciences; Medicine; Medicine & Public Health; Metabolic Diseases; Metabolism, Inborn Errors - enzymology; Metabolism, Inborn Errors - genetics; Metabolism, Inborn Errors - surgery; Middle Aged; Pediatrics; SSIEM Symposium 2007; Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases; Surgery of the digestive system; Treatment Outcome; Inborn Error; Glycogen Storage Disease Type; Orthotopic Liver Transplantation; Portal Vein Thrombosis; Maple Syrup Urine Disease; Treatment; Nutrition; Homograft; Graft; Genetics; Metabolic diseases; Enzymopathy; Congenital disease; Genetic disease; Liver transplantation
• ISSN: 0141-8955; 1573-2665
• DOI: 10.1007/s10545-008-0829-6

Summary Over the last 15 years, liver cell transplantation (LCT) has developed from an experimental laboratory technique to a potentially life-saving therapeutic option. Because of its minimally invasive nature, the method is especially attractive for (small) children. In children with liver-based inborn errors of metabolism, this transfer of enzyme activity can be regarded as a gene therapy, which can be installed independently and additionally to conservative treatment concepts. To date 14 children with inherited metabolic diseases have undergone LCT in various centres. Although individual results are encouraging, different treatment protocols, difficulties in the objective assessment of function of the transplant, and finally the lack of a controlled study make it difficult to judge the overall significance of LCTin the treatment of metabolic diseases and call forcollaborative clinical research.