Case Report
Copyright ©The Author(s) 2020. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Oct 28, 2020; 26(40): 6295-6303
Published online Oct 28, 2020. doi: 10.3748/wjg.v26.i40.6295
Compromised therapeutic value of pediatric liver transplantation in ethylmalonic encephalopathy: A case report
Guang-Peng Zhou, Wei Qu, Zhi-Jun Zhu, Li-Ying Sun, Lin Wei, Zhi-Gui Zeng, Ying Liu
Guang-Peng Zhou, Wei Qu, Zhi-Jun Zhu, Lin Wei, Zhi-Gui Zeng, Ying Liu, Liver Transplantation Center, National Clinical Research Center for Digestive Diseases, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China
Li-Ying Sun, Liver Transplantation Center, Intensive Care Unit, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China
Author contributions: Zhu ZJ contributed to conception and design of the study and performed the operation for the patient; Zhou GP collected the data, performed the data analysis, and wrote the initial draft of the manuscript; Qu W, Sun LY, Wei L, Zeng ZG, and Liu Y participated in the clinical management and follow-up of the patient and analysis and interpretation of data; All authors contributed to manuscript drafting and revision and issued final approval for the submitted version.
Supported by Beijing Municipal Science & Technology Commission, No. Z181100001718220, and Capital's Funds for Health Improvement and Research, No. 2020-1-2024.
Informed consent statement: Informed written consent was obtained from the patient for publication of this report and any accompanying images.
Conflict-of-interest statement: The authors declare that they have no conflicts of interest.
CARE Checklist (2016) statement: The authors have read the CARE Checklist (2016), and the manuscript was prepared and revised according to the CARE Checklist (2016).
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:
Corresponding author: Zhi-Jun Zhu, MD, PhD, Chief Doctor, Professor, Surgeon, Liver Transplantation Center, Beijing Friendship Hospital, Capital Medical University, No. 95 Yong-an Road, Xicheng District, Beijing 100050, China.
Received: May 12, 2020
Peer-review started: May 12, 2020
First decision: June 18, 2020
Revised: June 25, 2020
Accepted: September 18, 2020
Article in press: September 18, 2020
Published online: October 28, 2020
Core Tip

Core Tip: Liver transplantation (LT) is emerging as a novel therapeutic option for ethylmalonic encephalopathy (EE). However, worldwide experience is still limited. Our case of LT for EE, to the best of our knowledge, is the first case in Asian-Pacific countries. The case suggests that despite only a partial cure, LT does have a definite role in defending against the devastating natural progression of EE. Early establishment of diagnosis and initiation of conventional treatment pre-transplant, timely LT, and continuous administration of metabolism-correcting medications post-transplant may be helpful in minimizing the neurologic impairment and maximizing the therapeutic value of LT in EE.