Liver transplantation for pediatric metabolic disease

被引：81

作者：

Mazariegos, George ^{[1
,2
]}

Shneider, Benjamin ^{[3
]}

Burton, Barbara ^{[5
]}

Fox, Ira J. ^{[1
,2
,6
]}

Hadzic, Nedim ^{[7
]}

Kishnani, Priya ^{[8
]}

Morton, D. Holmes ^{[9
]}

Mcintire, Sara ^{[10
]}

Sokol, Ronald J. ^{[11
,12
]}

Summar, Marshall ^{[13
]}

White, Desiree ^{[14
]}

Chavanon, Vincent ^{[15
]}

Vockley, Jerry ^{[4
,16
,17
]}

机构：

[1] UPMC, Childrens Hosp Pittsburgh, Hillman Ctr Pediat Transplantat, Pittsburgh, PA 15224 USA

[2] Univ Pittsburgh, UPMC, Sch Med, Dept Surg,Thomas E Starzl Transplantat Inst, Pittsburgh, PA 15261 USA

[3] UPMC, Childrens Hosp Pittsburgh, Rangos Res Ctr, Div Pediat Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15224 USA

[4] Univ Pittsburgh, Sch Med, Dept Pediat, Pittsburgh, PA 15224 USA

[5] Northwestern Univ, Feinberg Sch Med, Ann & Robert H Lurie Childrens Hosp Chicago, Dept Pediat, Chicago, IL 60611 USA

[6] Univ Pittsburgh, McGowan Inst Regenerat Med, Pittsburgh, PA 15224 USA

[7] Kings Coll Hosp London, Paediat Liver Ctr, London, England

[8] Duke Univ, Med Ctr, Dept Pediat, Div Med Genet, Durham, NC 27710 USA

[9] Franklin & Marshall Coll, Clin Special Children, Strasburg, PA 17579 USA

[10] Univ Pittsburgh, Childrens Hosp Pittsburgh, UPMC, Sch Med,Dept Pediat,Paul C Gaffney Diagnost Refer, Pittsburgh, PA 15224 USA

[11] Univ Colorado, Sch Med, Dept Pediat, Aurora, CO 80045 USA

[12] Childrens Hosp Colorado, Sect Gastroenterol Hepatol & Nutr, Aurora, CO 80045 USA

[13] George Washington Univ, Childrens Natl Med Ctr, Med Genet Res Ctr, Div Genet & Metab, Washington, DC 20010 USA

[14] Washington Univ, Dept Psychol, St Louis, MO 63130 USA

[15] Mt Sinai Hosp, Div Plast & Reconstruct Surg, New York, NY 10029 USA

[16] Univ Pittsburgh, Grad Sch Publ Hlth, Dept Human Genet, Pittsburgh, PA 15261 USA

[17] UPMC, Childrens Hosp Pittsburgh, Bongos Res Ctr, Div Med Genet, Pittsburgh, PA 15224 USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2014年 / 111卷 / 04期

关键词：

Inborn errors of metabolism; Liver transplant; Hepatocyte transplant; Liver failure; Organic acidemia; Amino aciduria; GLYCOGEN-STORAGE-DISEASE; SYRUP-URINE-DISEASE; UREA CYCLE DISORDERS; PROPIONIC ACIDEMIA; HEPATOCYTE TRANSPLANTATION; METHYLMALONIC ACIDEMIA; CELL TRANSPLANTATION; GRAFT FIBROSIS; MITOCHONDRIAL HEPATOPATHIES; KIDNEY TRANSPLANTATION;

D O I：

10.1016/j.ymgme.2014.01.006

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Liver transplantation (LTx) was initially developed as a therapy for liver diseases known to be associated with a high risk of near-term mortality but is based upon a different set of paradigms for inborn metabolic diseases. As overall outcomes for the procedure have improved, LTx has evolved into an attractive approach for a growing number of metabolic diseases in a variety of clinical situations. No longer simply life-saving, the procedure can lead to a better quality of life even if not all symptoms of the primary disorder are eliminated. Juggling the risk-benefit ratio thus has become more complicated as the list of potential disorders amenable to treatment with LTx has increased. This review summarizes presentations from a recent conference on metabolic liver transplantation held at the Children's Hospital of Pittsburgh of UPMC on the role of liver or hepatocyte transplantation in the treatment of metabolic liver disease.

引用

页码：418 / 427

页数：10

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