Pediatric liver transplantation in metabolic disease: Clinical decision making

被引:28
作者
Shneider, BL [1 ]
机构
[1] Mt Sinai Med Ctr, Dept Pediat, New York, NY 10029 USA
关键词
gene therapy; hyperoxaluria; inherited disease; mitochondria; valproate;
D O I
10.1034/j.1399-3046.2002.1p057.x
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
Proper utilization of liver transplantation in the management of pediatric metabolic diseases requires a comprehensive understanding of both metabolic disease and the risk and benefits of transplantation. This brief review focuses on issues that pertain to the treatment of tyrosinemia type 1, bile acid biosynthesis disorders, primary hyperoxaluria, Crigler-Najjar Type 1, and mitochondrial diseases. These entities are used as prototypes to illustrate many of the principles that are applied in a more general sense to the management of metabolic diseases. The natural history of these disorders are considered in the context of the risks of liver transplantation. Indications, contraindications, and both current and future alternatives to transplantation, are considered.
引用
收藏
页码:25 / 29
页数:5
相关论文
共 23 条
[1]   Combined liver kidney transplantation in primary hyperoxaluria type 1 [J].
Cochat, P ;
Gaulier, JM ;
Nogueira, PCK ;
Feber, J ;
Jamieson, NV ;
Rolland, MO ;
Divry, P ;
Bozon, D ;
Dubourg, L .
EUROPEAN JOURNAL OF PEDIATRICS, 1999, 158 (Suppl 2) :S75-S80
[2]   Tyrosinemia type 1 should be suspected in infants with severe coagulopathy even in the absence of other signs of liver failure [J].
Croffie, JM ;
Gupta, SK ;
Chong, SKF ;
Fitzgerald, JF .
PEDIATRICS, 1999, 103 (03) :675-678
[3]   RESOLUTION OF LIVER-BIOPSY ALTERATIONS IN 3 SIBLINGS WITH BILE-ACID TREATMENT OF AN INBORN ERROR OF BILE-ACID METABOLISM (DELTA(4)-3-OXOSTEROID 5-BETA-REDUCTASE DEFICIENCY) [J].
DAUGHERTY, CC ;
SETCHELL, KDR ;
HEUBI, JE ;
BALISTRERI, WF .
HEPATOLOGY, 1993, 18 (05) :1096-1101
[4]  
Delarue A, 2000, Pediatr Transplant, V4, P67, DOI 10.1034/j.1399-3046.2000.00090.x
[5]   Preemptive liver transplantation from a living related donor for primary hyperoxaluria type I [J].
Gruessner, RW .
NEW ENGLAND JOURNAL OF MEDICINE, 1998, 338 (26) :1924-1924
[6]  
HOLME E, 1995, CURR OPIN PEDIATR, V7, P716
[7]   TREATMENT OF CHRONIC LIVER-DISEASE CAUSED BY 3-BETA-HYDROXY-DELTA-5-C27-STEROID DEHYDROGENASE-DEFICIENCY WITH CHENODEOXYCHOLIC ACID [J].
ICHIMIYA, H ;
NAZER, H ;
GUNASEKARAN, T ;
CLAYTON, P ;
SJOVALL, J .
ARCHIVES OF DISEASE IN CHILDHOOD, 1990, 65 (10) :1121-1124
[8]   Diagnosis and management of Crigler-Najjar syndrome [J].
Jansen, PLM .
EUROPEAN JOURNAL OF PEDIATRICS, 1999, 158 (Suppl 2) :S89-S94
[9]   Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide [J].
Kren, BT ;
Parashar, B ;
Bandyopadhyay, P ;
Chowdhury, NR ;
Chowdhury, JR ;
Steer, CJ .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1999, 96 (18) :10349-10354
[10]   TREATMENT OF HEREDITARY TYROSINEMIA TYPE-I BY INHIBITION OF 4-HYDROXYPHENYLPYRUVATE DIOXYGENASE [J].
LINDSTEDT, S ;
HOLME, E ;
LOCK, EA ;
HJALMARSON, O ;
STRANDVIK, B .
LANCET, 1992, 340 (8823) :813-817