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功能性胃肠疾病治疗试验的设计

Design of treatment trials for functional gastrointestinal disorders.

作者信息

Irvine E Jan, Whitehead William E, Chey William D, Matsueda Kei, Shaw Michael, Talley Nicholas J, Veldhuyzen van Zanten Sander J O

机构信息

Division of Gastroenterology, St. Michael's Hospital and University of Toronto, Toronto, Ontario, Canada.

出版信息

Gastroenterology. 2006 Apr;130(5):1538-51. doi: 10.1053/j.gastro.2005.11.058.

Abstract

This document addresses the design of trials to assess the efficacy of new treatments for functional gastrointestinal disorders (FGID), emphasizing trials in irritable bowel syndrome and dyspepsia, because most research has been undertaken in these conditions. The double-blind, randomized, placebo-controlled, parallel group trial remains the preferred design. Randomized withdrawal designs, although encouraged by the European Agency for the Evaluation of Medicinal Products, have the same potential disadvantages as a crossover design, including carryover effects, unmasking (unblinding), and overestimation of the potential benefit for clinical practice. Innovative trial designs that evaluate intermittent (on demand) treatment are likely to become more common in the future. Investigators should include as broad a spectrum of patients as possible and should report recruitment strategies, inclusion/exclusion criteria, and attrition data. The primary analysis should be based on the proportion of patients in each treatment arm who satisfy an a priori treatment responder definition, or a prespecified clinically meaningful change in a patient-reported symptom improvement measure. Such measures of improvement are psychometrically validated subjective global assessments or a change from baseline in a validated symptom severity questionnaire. It is unethical to change the responder definition after a trial begins. Data analysis should address all patients enrolled, using an intention-to-treat principle. Reporting of results should follow the Consolidated Standards for Reporting Trials guidelines and include an analysis of harms data and secondary outcome measures to support or explain the primary outcome. Trials should be registered in a public location, prior to initiation, and should be published even if the results are negative or inconclusive.

摘要

本文件论述了评估功能性胃肠疾病(FGID)新疗法疗效的试验设计,重点是肠易激综合征和消化不良的试验,因为大多数研究都是在这些病症中进行的。双盲、随机、安慰剂对照、平行组试验仍然是首选设计。随机撤药设计虽然得到了欧洲药品评估局的鼓励,但与交叉设计有相同的潜在缺点,包括遗留效应、揭盲(解除盲法)以及对临床实践潜在益处的高估。评估间歇性(按需)治疗的创新试验设计未来可能会变得更加普遍。研究者应纳入尽可能广泛的患者群体,并应报告招募策略、纳入/排除标准以及损耗数据。主要分析应基于每个治疗组中满足预先定义的治疗反应者定义的患者比例,或患者报告的症状改善测量指标中预先指定的具有临床意义的变化。这种改善的测量指标是经过心理测量学验证的主观整体评估,或经过验证的症状严重程度问卷中相对于基线的变化。在试验开始后改变反应者定义是不道德的。数据分析应采用意向性分析原则,涵盖所有入组患者。结果报告应遵循《试验报告统一标准》指南,包括对危害数据和次要结局指标的分析,以支持或解释主要结局。试验应在启动前在公共平台注册,即使结果为阴性或无定论也应发表。

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