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Liver transplantation for pediatric inherited metabolic liver diseases.

ABSTRACT: Liver transplantation (LT) remains the gold standard treatment for end stage liver disease in the pediatric population. For liver based metabolic disorders (LBMDs), the decision for LT is predicated on a different set of paradigms. With improved outcomes post-transplantation, LT is no longer merely life saving, but has the potential to also significantly improve quality of life. This review summarizes the clinical presentation, medical treatment and indications for LT for some of the common LBMDs. We also provide a practical update on the dilemmas and controversies surrounding the indications for transplantation, surgical considerations and prognosis and long terms outcomes for pediatric LT in LBMDs. Important progress has been made in understanding these diseases in recent years and with that we outline some of the new therapies that have emerged.

SUBMITTER: Vimalesvaran S

PROVIDER: S-EPMC8568579 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature

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Liver transplantation for pediatric inherited metabolic liver diseases.

Vimalesvaran Sunitha S Dhawan Anil A

World journal of hepatology 20211001 10

Liver transplantation (LT) remains the gold standard treatment for end stage liver disease in the pediatric population. For liver based metabolic disorders (LBMDs), the decision for LT is predicated on a different set of paradigms. With improved outcomes post-transplantation, LT is no longer merely life saving, but has the potential to also significantly improve quality of life. This review summarizes the clinical presentation, medical treatment and indications for LT for some of the common LBMD ...[more]

PMID: 34786171

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Inherited metabolic diseases are a potent risk factor for cytomegalovirus infection in pediatric living donor liver transplantation.

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Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice.

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