Retrospective Study
Copyright ©The Author(s) 2021. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Clin Cases. May 16, 2021; 9(14): 3273-3286
Published online May 16, 2021. doi: 10.12998/wjcc.v9.i14.3273
Pediatric Wilson disease presenting as acute liver failure: Prognostic indices
Wei-Yuan Fang, Kuerbanjiang Abuduxikuer, Peng Shi, Yi-Ling Qiu, Jing Zhao, Yu-Chuan Li, Xue-Yuan Zhang, Neng-Li Wang, Xin-Bao Xie, Yi Lu, A S Knisely, Jian-She Wang
Wei-Yuan Fang, Kuerbanjiang Abuduxikuer, Yi-Ling Qiu, Jing Zhao, Yu-Chuan Li, Xue-Yuan Zhang, Neng-Li Wang, Xin-Bao Xie, Yi Lu, Jian-She Wang, Center for Pediatric Liver Diseases, Children’s Hospital of Fudan University, Shanghai 201102, China
Peng Shi, Medical Statistics Department, Children’s Hospital of Fudan University, Shanghai 201102, China
A S Knisely, Institut für Pathologie, Medizinische Universität Graz, Graz 8010, Austria
Author contributions: Fang WY and Abuduxikuer K contributed equally to this work and were in charge of data acquisition, analysis, interpretation, and writing; Wang JS and Lu Y contributed to the study design and critical revision; Abuduxikuer K, Shi P, and Qiu YL contributed to the statistical analyses; Knisely AS contributed to the manuscript review; Zhao J, Li YC, Zhang XY, Wang NL, and Xie XB contributed to the patient’s medical management; All authors have read and approved the final manuscript.
Institutional review board statement: The study was reviewed and approved by the Children's Hospital of Fudan University Institutional Review Board (Approval No. 2020-402).
Informed consent statement: Patients and their parents were not required to give informed consent to the study because the analysis used anonymous clinical data that were obtained after each patient and their parents agreed to treatment by written consent.
Conflict-of-interest statement: All authors have declared no conflicts of interest.
Data sharing statement: No additional data are available.
STROBE statement: The authors have read the STROBE Statement-checklist of items, and the manuscript was prepared and revised according to the STROBE Statement-checklist of items.
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: Yi Lu, MD, Associate Chief Physician, Center for Pediatric Liver Diseases, Children's Hospital of Fudan University, No. 399 Wanyuan Road, Shanghai 201102, China. luyi@fudan.edu.cn
Received: January 12, 2021
Peer-review started: January 12, 2021
First decision: February 11, 2021
Revised: February 28, 2021
Accepted: March 17, 2021
Article in press: March 17, 2021
Published online: May 16, 2021
Processing time: 106 Days and 16.3 Hours
Core Tip

Core Tip: This study determined outcomes among 41 recently seen pediatric patients with well-documented Wilson disease (WD) with acute liver failure (ALF) (WDALF) (most confirmed genetically, WD presenting as ALF) and assessed mortality model performances. Mortality was low, showing that WDALF children, including those with milder encephalopathy, can survive and recover without liver transplant (LT) when given D-penicillamine and zinc-salt therapy with or without plasmapheresis. Sensitivity of seven different prognostic scoring systems was good in our patients but without optimal positive predictive values, indicating that pediatric WD with higher prognostic scores may recover from ALF even after enlisting for LT.