Operating Properties of Disease Activity Indices in Pediatric Inflammatory Bowel Disease: A Systematic Review.

IF 4.5 3区医学 Q1 GASTROENTEROLOGY & HEPATOLOGY

Inflammatory Bowel Diseases Pub Date : 2025-01-06 DOI:10.1093/ibd/izae060

Ruben J Colman, Virginia Solitano, John K MacDonald, Christopher Ma, Anne M Griffiths, Vipul Jairath, Eileen Crowley

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引用次数: 0

Abstract

Background: Accurate, reliable, and responsive disease activity indices are important to streamline drug approval and treatment modalities for pediatric inflammatory bowel disease (pIBD). We aimed to identify all scoring indices used in pIBD randomized controlled trials (RCTs) and to evaluate their operating properties.

Methods: MEDLINE, EMBASE, and CENTRAL were searched on December 6, 2022, to identify studies evaluating clinical, endoscopic, imaging, or patient-reported outcome measures (PROMs) in pIBD including Crohn's disease (CD) and ulcerative colitis (UC). Validity, reliability, responsiveness, and feasibility were summarized.

Results: Seventy RCTs evaluating pIBD indices were identified. Forty-one studies reported on the operating properties of 14 eligible indices (n = 9 CD, n = 5 UC). The Pediatric Crohn's Disease Activity Index (PCDAI) varied widely in terms of validity and reliability and was less feasible overall. In contrast, the Mucosal Inflammation Noninvasive Index, which includes fecal calprotectin, had better operating properties than the PCDAI. The Simplified Endoscopic Mucosal Assessment of Crohn's Disease appears more feasible and had similar operating properties than the longer Simple Endoscopic Score for Crohn's Disease. The Pediatric Ulcerative Colitis Activity Index was feasible, valid, and reliable, but responsiveness needs to be evaluated further. The Endoscopic Mayo score and the Ulcerative Colitis Endoscopic Index of Severity were reliable, but validity and responsiveness need to be evaluated further. Imaging and PROMs/quality of life indices need further evaluation.

Conclusions: The operating properties of pIBD clinical trial end points varied widely. These results highlight the need for further validation and development of novel indices.

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小儿炎症性肠病疾病活动指数的操作特性：系统回顾

背景：准确、可靠、反应灵敏的疾病活动性指数对于简化药物审批和小儿炎症性肠病（pIBD）的治疗方法非常重要。我们旨在确定所有在小儿炎症性肠病随机对照试验（RCT）中使用的评分指标，并评估其操作特性：方法：于 2022 年 12 月 6 日检索了 MEDLINE、EMBASE 和 CENTRAL，以确定评估包括克罗恩病（CD）和溃疡性结肠炎（UC）在内的 pIBD 临床、内窥镜、影像学或患者报告结果测量指标（PROMs）的研究。对有效性、可靠性、响应性和可行性进行了总结：结果：确定了 70 项评估 pIBD 指数的 RCT。41项研究报告了14个符合条件的指数的运行特性（n = 9 CD，n = 5 UC）。小儿克罗恩病活动指数（PCDAI）在有效性和可靠性方面差异很大，总体而言可行性较低。相比之下，包括粪钙蛋白在内的粘膜炎症无创指数的操作特性优于 PCDAI。克罗恩病简易内镜黏膜评估似乎更可行，其操作特性与较长的克罗恩病简易内镜评分相似。小儿溃疡性结肠炎活动指数是可行的、有效的和可靠的，但响应性还需要进一步评估。内镜下梅奥评分和溃疡性结肠炎内镜下严重程度指数是可靠的，但有效性和响应性需要进一步评估。成像和PROMs/生活质量指数需要进一步评估：结论：pIBD临床试验终点的操作属性差异很大。这些结果凸显了进一步验证和开发新型指数的必要性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Inflammatory Bowel Diseases 医学-胃肠肝病学

CiteScore

9.70

自引率

6.10%

发文量

462

审稿时长

1 months

期刊介绍： Inflammatory Bowel Diseases® supports the mission of the Crohn''s & Colitis Foundation by bringing the most impactful and cutting edge clinical topics and research findings related to inflammatory bowel diseases to clinicians and researchers working in IBD and related fields. The Journal is committed to publishing on innovative topics that influence the future of clinical care, treatment, and research.