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儿童炎症性肠病的目标治疗:证据说了什么?

Treat-to-Target in Pediatric Inflammatory Bowel Disease: What Does the Evidence Say?

机构信息

Department of Women's and Children's Health, Pediatric Gastroenterology and Liver Unit, Sapienza University of Rome, Viale Regina Elena 324, 00161, Rome, Italy.

出版信息

Paediatr Drugs. 2020 Oct;22(5):463-472. doi: 10.1007/s40272-020-00406-2.

Abstract

The traditional management of inflammatory bowel disease, based on treatment intensification guided by clinical activity alone, has been revised in the last 10 years and a treat-to-target approach has been proposed and is currently under evaluation as a disease-modifying strategy. Treat-to-target focuses on objective and scheduled measures to monitor intestinal damage, with consequent therapeutic adjustments in case of failure to achieve pre-defined targets. Identification of targets has been set out by the Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) committee in 2015. Mucosal healing is universally accepted as the main target both in Crohn's disease and ulcerative colitis, given its proven association with better long-term outcomes than clinical remission alone. Equally important is to ensure patients' clinical remission and improve patient-reported outcomes. Transmural healing (for Crohn's disease) and histological remission (for ulcerative colitis), listed as adjunctive targets, are likely to become primary targets in the near future. The ultimate goal of this approach is to modify the natural history of inflammatory bowel diseases by trying to block bowel damage progression, with interventions in the pre-clinical stage. In this review, we will discuss the current recommended therapeutic targets, as well as those that are considered adjunctive targets, with a focus on the limited pediatric literature available. Prospective long-term trials are warranted in order to identify the most appropriate target for the pediatric population and its specific issues. Identification of reliable predictors of disease course, outcome, and response to treatment will help to individually adapt each step of this monitoring algorithm and consequent therapeutic decision.

摘要

在过去的 10 年中,传统的炎症性肠病管理方法已经发生了变化,不再仅仅基于临床活动的治疗强化,而是提出了一种针对目标的治疗方法,并正在作为一种疾病修饰策略进行评估。针对目标的治疗方法侧重于监测肠道损伤的客观和定期措施,如果未能达到预先定义的目标,则进行相应的治疗调整。2015 年,炎症性肠病选择治疗靶点(STRIDE)委员会确定了目标的选择。鉴于黏膜愈合与临床缓解单独相比可带来更好的长期结果,其已被普遍接受为克罗恩病和溃疡性结肠炎的主要目标。同样重要的是要确保患者的临床缓解和改善患者报告的结果。作为辅助目标列出的透壁愈合(用于克罗恩病)和组织学缓解(用于溃疡性结肠炎)可能在不久的将来成为主要目标。这种方法的最终目标是通过尝试阻止肠道损伤的进展来改变炎症性肠病的自然病史,在临床前阶段进行干预。在这篇综述中,我们将讨论当前推荐的治疗目标,以及那些被认为是辅助目标的目标,重点是有限的儿科文献。需要进行前瞻性长期试验,以确定儿科人群及其特定问题的最佳目标。识别疾病过程、结果和治疗反应的可靠预测因素将有助于根据个人情况调整这种监测算法和随后的治疗决策的每一步。

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