Liver transplantation and cell therapies for inborn errors of metabolism
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SSIEM SYMPOSIUM 2012
Liver transplantation and cell therapies for inborn errors of metabolism Patrick McKiernan
Received: 29 November 2012 / Revised: 16 December 2012 / Accepted: 20 December 2012 / Published online: 8 January 2013 # SSIEM and Springer Science+Business Media Dordrecht 2013
Abstract Liver transplantation is now very successful with >85 % long term survival into adult life. When considering the impact of liver transplantation for metabolic disease two independent factors need to be considered; whether or not the defect causes liver disease and whether or not it is confined to the liver. When considering transplantation many factors need to be considered including the local success of transplantation, the impact of the metabolic disease on the patient and family and the potential for future therapeutic developments. Where transplantation is undertaken for a liver based defect there is a lifelong complete correction of the defect. Where there is a residual extrahepatic defect this will have an impact on the outcome of liver transplantation and the severity of this defect must be considered as part of the transplant assessment process. Access to a multi-disciplinary team with expertise in metabolic disease, liver disease and other relevant organ based specialists is crucial. Most children will receive transplantation from cadaveric donor but living related transplantation from a heterozygote parent is usually safe and effective. Auxiliary liver transplantation has a small but useful role where partial correction of the defect is helpful and there is a future prospect of gene therapy. The first-generation of hepatocyte transplants have shown proof of principle but to date have had a rather modest and temporary metabolic effect. Stem cells may have the potential to produce a more sustained and Communicated by: K. Michael Gibson Presented at the Annual Symposium of the SSIEM, Birmingham, UK, September 4-7, 2012 P. McKiernan (*) Liver Unit, Birmingham Children’s Hospital, B4 6NH Birmingham, UK e-mail: [email protected]
significant metabolic correction, but must be shown to be effective in controlled trials.
Introduction Liver transplantation is one of the outstanding successes of high technology medicine. Paediatric liver transplantation can now offer a 1 year survival of >90 % of whom the vast majority will survive into adulthood with a good quality of life(McKiernan 2011). Since the introduction of liver transplantation, inherited metabolic disorders have been the indication in approximately 15 % of cases(Arnon et al 2010). These indications however are never static. Liver transplantation is not a “cure” but rather a disorder in itself with a defined acute mortality and a future attrition rate mostly related to the lifelong need for immunosuppression. In an individual child and family the therapeutic decision has to incorporate the contemporary local success of transplantation, the impact of the metabolic defect on the child and the family, the natural history of the defect and whether any new th
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