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2024 年炎症性肠病研究面临的挑战:精准医学。

Challenges in IBD Research 2024: Precision Medicine.

机构信息

Department of Pediatrics, University of Virginia, Charlottesville, VA, USA.

Patient representative for Crohn's & Colitis Foundation, New York, NY, USA.

出版信息

Inflamm Bowel Dis. 2024 May 23;30(Suppl 2):S39-S54. doi: 10.1093/ibd/izae084.

Abstract

Precision medicine is part of 5 focus areas of the Challenges in IBD Research 2024 research document, which also includes preclinical human IBD mechanisms, environmental triggers, novel technologies, and pragmatic clinical research. Building on Challenges in IBD Research 2019, the current Challenges aims to provide a comprehensive overview of current gaps in inflammatory bowel diseases (IBDs) research and deliver actionable approaches to address them with a focus on how these gaps can lead to advancements in interception, remission, and restoration for these diseases. The document is the result of multidisciplinary input from scientists, clinicians, patients, and funders, and represents a valuable resource for patient-centric research prioritization. In particular, the precision medicine section is focused on the main research gaps in elucidating how to bring the best care to the individual patient in IBD. Research gaps were identified in biomarker discovery and validation for predicting disease progression and choosing the most appropriate treatment for each patient. Other gaps were identified in making the best use of existing patient biosamples and clinical data, developing new technologies to analyze large datasets, and overcoming regulatory and payer hurdles to enable clinical use of biomarkers. To address these gaps, the Workgroup suggests focusing on thoroughly validating existing candidate biomarkers, using best-in-class data generation and analysis tools, and establishing cross-disciplinary teams to tackle regulatory hurdles as early as possible. Altogether, the precision medicine group recognizes the importance of bringing basic scientific biomarker discovery and translating it into the clinic to help improve the lives of IBD patients.

摘要

精准医学是《2024 年炎症性肠病研究挑战》研究文件五个重点领域之一,其他四个领域分别为临床前人类 IBD 机制、环境触发因素、新技术和实用临床研究。在《2019 年炎症性肠病研究挑战》的基础上,目前的挑战旨在全面概述炎症性肠病(IBD)研究中的当前差距,并提供可行的方法来解决这些差距,重点关注这些差距如何为这些疾病的干预、缓解和恢复带来进展。该文件是来自科学家、临床医生、患者和资助者的多学科投入的结果,是患者为中心的研究优先事项的宝贵资源。特别是,精准医学部分专注于阐明如何为 IBD 患者提供最佳个体护理方面的主要研究差距。在发现和验证预测疾病进展和为每个患者选择最合适治疗的生物标志物方面存在研究差距。在充分利用现有患者生物样本和临床数据、开发分析大型数据集的新技术以及克服监管和支付障碍以实现生物标志物的临床应用方面也存在其他差距。为了解决这些差距,工作组建议专注于彻底验证现有候选生物标志物,使用一流的数据生成和分析工具,并尽早建立跨学科团队来解决监管障碍。总的来说,精准医学小组认识到将基础科学生物标志物发现转化为临床实践,以帮助改善 IBD 患者生活的重要性。

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