Retrospective Cohort Study
Copyright ©The Author(s) 2024. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Hepatol. May 27, 2024; 16(5): 791-799
Published online May 27, 2024. doi: 10.4254/wjh.v16.i5.791
Characteristics of patients with Wilson disease in the United States: An insurance claims database study
Thomas Daniel-Robin, Pradeep Kumar, Bernard Benichou, Jean-Philippe Combal
Thomas Daniel-Robin, Bernard Benichou, Jean-Philippe Combal, Vivet Therapeutics, Paris 75008, France
Pradeep Kumar, Clarivate, Noida 201301, Uttar Pradesh, India
Author contributions: Daniel-Robin T, Kumar P, Benichou B, Combal JP were involved in the conceptualisation, methodology and validation of the study and in the preparation of this article, and each made a significant scientific contribution to the research in the manuscript, approved its claims, and agreed to be an author.
Institutional review board statement: The data were de-identified at the patient level and extracted using an encrypted participant key, in compliance with the US Health Insurance Portability and Accountability Act. As the data was anonymised prior to extraction, ethics committee approval as not required.
Informed consent statement: The data were de-identified at the patient level and extracted using an encrypted participant key, in compliance with the US Health Insurance Portability and Accountability Act. Since the data was anonymised prior to extraction, informed consent is not required. The authors are unaware of the identity of the patients evaluated.
Conflict-of-interest statement: TDR, BB and JPC are employees and shareholder of Vivet Therapeutics, a biotechnology company involved in the development of gene therapies for rare diseases, including Wilson’s disease. PK is an employee of Clarivate, a company which owns the RWD database and which performed the analysis on behalf of Vivet Therapeutics.
Data sharing statement: The study data are the property of Clarivate RWD, who can be contacted to obtain access for fee.
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: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Jean-Philippe Combal, PharmD, PhD, CEO, Vivet Therapeutics, No. 80 Bd Haussmann, Paris 75008, France. jpcombal@vivet-therapeutics.com
Received: October 10, 2023
Peer-review started: October 10, 2023
First decision: November 27, 2023
Revised: March 7, 2024
Accepted: March 25, 2024
Article in press: March 25, 2024
Published online: May 27, 2024
Processing time: 224 Days and 23.7 Hours
Core Tip

Core Tip: This insurance claims database study in the United States evaluated data on 5376 patients with a reimbursement claim for Wilson disease (WD), although only patients with a documented treatment of WD can be unequivocally assumed to have the disease. For patients receiving a specific WD treatment, the mean age at death was 57 years, and the standard mortality ratio with respect to the general United States population was 2.19. These findings indicate an important unmet medical need for more effective treatment.