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World J Hepatol. Oct 27, 2021; 13(10): 1351-1366
Published online Oct 27, 2021. doi: 10.4254/wjh.v13.i10.1351
Liver transplantation for pediatric inherited metabolic liver diseases
Sunitha Vimalesvaran, Anil Dhawan
Sunitha Vimalesvaran, Anil Dhawan, Paediatric Liver GI and Nutrition Center, King's College Hospital, London SE5 9RS, United Kingdom
Author contributions: Vimalesvaran S and Dhawan A wrote the review; Dhawan A provided guidance and insightful comments on the review.
Conflict-of-interest statement: Authors declare no conflict of interests for this article.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Anil Dhawan, BM BCh, MD, Professor, Paediatric Liver GI and Nutrition Center, King's College Hospital, Denmark Hill, London SE5 9RS, United Kingdom. anil.dhawan@kcl.ac.uk
Received: May 4, 2021
Peer-review started: May 4, 2021
First decision: June 15, 2021
Revised: June 23, 2021
Accepted: August 20, 2021
Article in press: August 20, 2021
Published online: October 27, 2021
Processing time: 171 Days and 11.9 Hours
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.

Keywords: Pediatric metabolic liver disease; Liver transplantation; Liver based metabolic disorders; Inherited; Cell therapy; Gene therapy

Core Tip: The decision for liver transplantation (LT) in liver based metabolic disorders (LBMDs) is not straightforward. As outcomes from pediatric LT continue to improve, transplantation is no longer merely life saving, but also potentially significantly improves the child’s quality of life. We herein discuss the clinical presentation, medical and surgical treatment for some of the common LBMDs. We provide a practical update on the indications, dilemmas and controversies for LT and the long-term outcomes for children with LBMDs.