克罗恩病的疗效评估:系统回顾随机临床试验,为多重结果框架提供依据。

IF 5.8 2区 医学 Q1 GASTROENTEROLOGY & HEPATOLOGY United European Gastroenterology Journal Pub Date : 2024-10-11 DOI:10.1002/ueg2.12679
Paula Leão Moreira, Axel Dignass, Maria Manuela Estevinho, Francisco Portal, João Mendes, Mafalda Santiago, Walter Reinisch, Bruce E Sands, Geert D'Haens, Gerassimos J Mantzaris, Silvio Danese, Laurent Peyrin-Biroulet, Vipul Jairath, Iris Dotan, Fernando Magro
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引用次数: 0

摘要

克罗恩病(CD)的长期疾病控制具有挑战性,需要了解治疗效果和结果评估。面对多种新型治疗方案,严格评估随机对照试验(RCT)的疗效对于指导临床实践至关重要。本研究系统地回顾了以 CD 结果为重点的随机对照试验,以阐明所报告结果和测量工具的广度和深度。我们在 MEDLINE 和 Scopus 上对 2000 年 1 月 1 日至 2023 年 1 月 31 日期间发表的 RCT 进行了系统检索。符合条件的研究包括至少有 50 名成年 CD 患者的全文文章。主要和次要结果及其测量工具根据风湿病学结果测量筛选器 2.1 框架进行分类。对 88 项纳入研究中的 393 项结果进行了分析。临床结果,如临床缓解和反应,最为普遍(50.6%);生物标志物(11.5%)和患者报告结果(10.2%)也得到了评估。其他结果包括疾病行为和并发症(2%)、内窥镜检查(10.4%)、组织学检查(0.5%)、放射学检查(1.3%)、医疗保健利用率(3.8%)和治疗相关安全性(6.9%)。综合结果呈上升趋势,反映了向综合评估的转变。88 项研究中有 21 项报告了主要终点,包括临床症状和粘膜炎症。本综述强调了 CD RCT 研究中结果评估的演变情况,强调了结果的日益复杂性。综合结果的突出强调了为捕捉 CD 的多维性所做的努力。这些发现将为第二阶段的两轮 e-Delphi 提供信息,该阶段旨在为 CD 研究中的 RCT 制定多成分结果时优先考虑关键领域和结果。
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Assessment of outcomes in Crohn's disease: A systematic review of randomized clinical trials to inform a multiple outcome framework.

Longstanding disease control in Crohn's disease (CD) is challenging and requires understanding treatment efficacy and outcomes assessment. With multiple novel therapeutic options, rigorous evaluation of outcomes in randomized controlled trials (RCTs) is crucial to inform clinical practice. This study systematically reviewed RCTs focusing on CD outcomes to elucidate the breadth and depth of reported outcomes and measurement instruments. A systematic search was conducted on MEDLINE and Scopus for RCTs published from 1 January 2000 to 31 January 2023. Eligible studies included full-text articles with at least 50 adult CD patients. Primary and secondary outcomes, along with their measurement instruments, were categorized according to the Outcome Measures in Rheumatology Filter 2.1 framework. From 88 included studies, 393 outcomes were analyzed. Clinical outcomes, such as clinical remission and response, were the most prevalent (50.6%); biomarkers (11.5%) and patient-reported outcomes (10.2%) were also assessed. Other outcomes included disease behavior and complications (2%), endoscopy (10.4%), histology (0.5%), radiology (1.3%), healthcare utilization (3.8%), and therapy-related safety (6.9%). Composite outcomes showed an increasing trend, reflecting a shift toward comprehensive evaluations. Coprimary endpoints, including clinical symptoms and mucosal inflammation, were reported in 21 of 88 studies. This review highlights the evolving landscape of outcome assessment in CD RCTs, emphasizing the increasing complexity of outcomes. The prominence of composite outcomes underscores efforts to capture the multidimensional nature of CD. These findings will inform the second stage of a two-round e-Delphi aimed at prioritizing key domains and outcomes for developing a multiple-component outcome for RCTs in CD research.

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来源期刊
United European Gastroenterology Journal
United European Gastroenterology Journal GASTROENTEROLOGY & HEPATOLOGY-
CiteScore
10.50
自引率
13.30%
发文量
147
期刊介绍: United European Gastroenterology Journal (UEG Journal) is the official Journal of the United European Gastroenterology (UEG), a professional non-profit organisation combining all the leading European societies concerned with digestive disease. UEG’s member societies represent over 22,000 specialists working across medicine, surgery, paediatrics, GI oncology and endoscopy, which makes UEG a unique platform for collaboration and the exchange of knowledge.
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