炎症性肠病的个性化(N-of-1)临床试验:机遇与挑战。

IF 11.6 1区 医学 Q1 GASTROENTEROLOGY & HEPATOLOGY Clinical Gastroenterology and Hepatology Pub Date : 2024-09-18 DOI:10.1016/j.cgh.2024.08.028
Sailish Honap, Guangyong Zou, Silvio Danese, Laurent Peyrin-Biroulet, Vipul Jairath
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引用次数: 0

摘要

背景和目的:炎症性肠病治疗效果的异质性意味着许多个体或患者亚群偏离平均水平,传统随机试验的结果对他们可能不适用。N-of-1试验是一种对单个患者进行长期随访的设计,不同时期的治疗方法是随机的,目的是找到对该患者最有效的治疗方法。本研究旨在调查N-of-1试验在IBD中的实用性:为确定本范围综述的相关文章,我们在PubMed平台上使用 "炎症性肠病"、"克罗恩病"、"溃疡性结肠炎"、"N-of-1试验"、"单病例设计"、"个性化试验 "等检索词对MEDLINE上发表的英文文章进行了文献检索:在更多个性化医疗和以患者为中心的医疗保健需求的推动下,N-of-1试验在多个医学学科中再次兴起,但在IBD中的应用却很少,仅发现了三项试验。涉及多项N-of-1试验的研究可为每位参与者提供可靠的证据以及平均效应估计值。N-of-1试验可用于研究被随机试验排除在外或代表性不足的IBD患者,如肠道外表现、小袋炎和直肠炎患者。虽然从方法学上讲,N-of-1试验是合理的,而且类似于RCT试验的严谨性,但研究设计中固有的交叉期可能会被患者和研究人员视为负担:N-of-1试验设计提供了一种以患者为中心的方法,可以客观地确定个体对治疗的反应。
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Personalized (N-of-1) Clinical Trials for Inflammatory Bowel Disease: Opportunities and Challenges.

Background & aims: Heterogeneity of treatment effects in inflammatory bowel disease (IBD) means that many individuals or patient subgroups depart from the average for whom the outcomes from traditional randomized trials may not be applicable. The N-of-1 trial is a design in which a single patient is followed over time with the treatments being randomized from period to period with the intention of finding the most effective treatment for that patient. The aim was to investigate the utility of N-of-1 trials in IBD.

Methods: To identify relevant articles for this scoping review, a MEDLINE literature search was conducted through the PubMed platform for articles published in the English language using the search terms "inflammatory bowel disease," "Crohn's disease," "ulcerative colitis," "N-of-1 trials," "single case designs," and "personalized trials."

Results: N-of-1 trials have seen a resurgence across several medical disciplines, driven by a need for more personalized medicine and patient-centered health care; their use in IBD is scarce with only 3 trials identified. Studies involving multiple N-of-1 trials can generate robust evidence for each participant and average effect estimates. The N-of-1 trial may hold potential for studying patients with IBD that are excluded from or underrepresented by randomized trials, such as those with extraintestinal manifestations, pouchitis, and proctitis. Although methodologically sound and akin to the rigor of a randomized controlled trial, the crossover periods inherent to the study design can be perceived as burdensome by patients and researchers.

Conclusions: The N-of-1 trial design provides a patient-centered means of objectively determining individual response to therapy.

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来源期刊
CiteScore
16.90
自引率
4.80%
发文量
903
审稿时长
22 days
期刊介绍: Clinical Gastroenterology and Hepatology (CGH) is dedicated to offering readers a comprehensive exploration of themes in clinical gastroenterology and hepatology. Encompassing diagnostic, endoscopic, interventional, and therapeutic advances, the journal covers areas such as cancer, inflammatory diseases, functional gastrointestinal disorders, nutrition, absorption, and secretion. As a peer-reviewed publication, CGH features original articles and scholarly reviews, ensuring immediate relevance to the practice of gastroenterology and hepatology. Beyond peer-reviewed content, the journal includes invited key reviews and articles on endoscopy/practice-based technology, health-care policy, and practice management. Multimedia elements, including images, video abstracts, and podcasts, enhance the reader's experience. CGH remains actively engaged with its audience through updates and commentary shared via platforms such as Facebook and Twitter.
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