TITLE

Liver transplantation in maple syrup urine disease

AUTHOR(S)
Wendel, U.; Saudubray, J. M.; Bodner, A.; Schadewaldt, P.
PUB. DATE
December 1999
SOURCE
European Journal of Pediatrics;1999, Vol. 158 Issue 14, pS060
SOURCE TYPE
Academic Journal
DOC. TYPE
Article
ABSTRACT
Abstract Maple syrup urine disease (MSUD) is an autosomal recessive disorder. Impaired activity of the branched-chain 2-oxoacid dehydrogenase complex (BCOA-DH) causes accumulation of branched-chain L-amino (BCAA) and 2-oxoacids (BCOA) which may exert neurotoxic effects. Treatment comprises dietary management with strictly reduced quantities of protein and BCAA as well as aggressive intervention during acute neonatal and subsequent metabolic complications. MSUD is regarded as a metabolic disorder with potentially favourable outcome when the patients are kept on a carefully supervised long-term therapy. Up to now, three MSUD patients, exhibiting the classical form of the disease, have received orthotopic whole liver transplantation (OLT). Liver replacement resulted in a clear increase in whole body BCOA-DH activity to at least the level of very mild MSUD variants. These patients no longer require protein restricted diets and the risk of metabolic decompensation during catabolic events is apparently abolished.
ACCESSION #
4681739

 

Related Articles

  • Liver transplantation in metabolic disorders: summary of the general discussion. Burdelski, M.; Ullrich, K. // European Journal of Pediatrics;1999, Vol. 158 Issue 14, pS095 

    Presents a summary of discussion about liver transplantation in metabolic disorders. Antitrypsin deficiency; Neonatal haemochromatosis; Methylmalenic acidaemia.

  • Decellularized human liver as a natural 3D-scaffold for liver bioengineering and transplantation. Mazza, Giuseppe; Rombouts, Krista; Rennie Hall, Andrew; Urbani, Luca; Vinh Luong, Tu; Al-Akkad, Walid; Longato, Lisa; Brown, David; Maghsoudlou, Panagiotis; Dhillon, Amar P.; Fuller, Barry; Davidson, Brian; Moore, Kevin; Dhar, Dipok; De Coppi, Paolo; Malago, Massimo; Pinzani, Massimo // Scientific Reports;8/7/2015, p13079 

    Liver synthetic and metabolic function can only be optimised by the growth of cells within a supportive liver matrix. This can be achieved by the utilisation of decellularised human liver tissue. Here we demonstrate complete decellularization of whole human liver and lobes to form an...

  • Sodium excretion in relation to calcium and hydroxyproline... Itoh, Roichi; Suyama, Yasuo // American Journal of Clinical Nutrition;May96, Vol. 63 Issue 5, p735 

    Studies the relation between calcium excretion and sodium excretion of Japanese to evaluate whether habitual excess sodium intake is a significant risk factor of calcium loss. Anthropometric measures of the subjets; Changes in excretion of sodium, calcium, and hydroxyproline by age and sex;...

  • Serum Cr May Assist in Selecting Liver Transplant Candidates.  // Kidney;May/Jun98, Vol. 7 Issue 3, p109 

    Discusses the abstract of the research paper entitled 'Pretransplant renal dysfunction predicts poorer outcome in liver transplantation,' by R.A. Lafayette, G. Pare et al and published in a 1997 issue of the journal 'Clinical Nephrology.'

  • Liver transplantation after paracetamol overdose. O'Grady, John G.; Wendon, Julia; Tan, K.C.; Potter, Dennis; Cottam, Simon; Cohen, Alexander T.; Gimson, Alexander E.S.; Williams, Roger // BMJ: British Medical Journal (International Edition);7/27/91, Vol. 303 Issue 6796, p221 

    Evaluates the role of liver transplantation after paracetamol overdose in London, England. Influence of paracetamol overdose on fulminant hepatic failure; Identification of patients who would merit a liver transplant; Description of the prognostic criteria.

  • Sublethal Cd-induced cellular damage and metabolic changes in the freshwater crab Sinopotamon henanense. Xuan, Ruijing; Wu, Hao; Li, Yingjun; Wang, Jinxiang; Wang, Lan // Environmental Science & Pollution Research;Feb2014, Vol. 21 Issue 3, p1738 

    To explore whether sublethal cadmium (Cd) exposure causes branchial cellular damages and affects the metabolic activity in brachyuran crustaceans, the freshwater crab Sinopotamon henanense was exposed to 0.71, 1.43, and 2.86 mg/L Cd for 3 weeks. Gill morphology, metabolic activity (activities of...

  • Preface. Burdelski, M.; Ullrich, K. // European Journal of Pediatrics. Supplement;1999, Vol. 158, pS33 

    Presents a preface on the international symposium on "Indication for Liver Transplantation in Metabolic Disorders" held in November 1997 in Fulda, Germany. Presence of experts in the field of transplantation and metabolic disorders from all over Europe and the U.S.; Inclusion of the reviews...

  • Ex Vivo Liver--Directed Gene Therapy for the Treatment of Metabolic Diseases: Advances in Hepatocyte Transplantation and Retroviral Vectors. Tuan Huy Nguyen; Mainot, Sylvie; Lainas, Panagiotis; Groyer-Picard, Marie-Th�r�se; Franco, Dominique; Dagher, Ibrahim; Weber, Anne // Current Gene Therapy;Apr2009, Vol. 9 Issue 2, p136 

    Transplantation of hepatocytes, whether genetically modified or not, has become an alternative to orthotopic liver transplantation for the treatment of patients with metabolic disease. However, more than ten years after the first clinical trial of ex vivo gene therapy to treat patients with...

  • Pediatric liver transplantation in India: Its time has come. D'Cruz, Ashley L. J. // Journal of Indian Association of Pediatric Surgeons;Jan-Mar2011, Vol. 16 Issue 1, p1 

    The author reflects on the practice of pediatric liver transplantation for end-stage liver disease (ESLD) treatment in India. She says that pediatric liver transplants in India was first performed in 1998 wherein most of the centers use live donor transplants. She adds that in India, 280 ESLD...

Share

Read the Article

Courtesy of THE LIBRARY OF VIRGINIA

Sorry, but this item is not currently available from your library.

Try another library?
Sign out of this library

Other Topics