肠易激综合征临床试验的研究设计考量
Study design considerations for irritable bowel syndrome clinical trials.
作者信息
Miller Larry E
出版信息
Ann Gastroenterol. 2014;27(4):338-345.
Abstract
Clinical trials of therapies intended to alleviate symptoms of irritable bowel syndrome (IBS) are prevalent. However, the ideal study design remains elusive since there is no obvious pathophysiological target and no universally accepted endpoint to assess symptom improvement in IBS. The purpose of this paper is to identify and discuss the most problematic issues in the design of clinical trials intended to evaluate the effectiveness of treatments for IBS symptoms. Lack of standardized diagnostic criteria, symptom variability, heterogeneous subject characteristics, large placebo effects, lack of statistical power, inappropriate endpoint selection, and poorly selected study design are the most critical issues that may confound study outcomes in IBS clinical trials.
摘要
旨在缓解肠易激综合征（IBS）症状的治疗方法的临床试验很普遍。然而，理想的研究设计仍然难以捉摸，因为没有明显的病理生理靶点，也没有普遍接受的终点来评估IBS症状的改善情况。本文的目的是识别并讨论旨在评估IBS症状治疗效果的临床试验设计中最具问题的问题。缺乏标准化诊断标准、症状变异性、受试者特征异质性、安慰剂效应大、统计效力不足、终点选择不当以及研究设计选择不佳是可能混淆IBS临床试验研究结果的最关键问题。
相似文献
1
Study design considerations for irritable bowel syndrome clinical trials.肠易激综合征临床试验的研究设计考量
Ann Gastroenterol. 2014;27(4):338-345.
2
Therapist telephone-delivered CBT and web-based CBT compared with treatment as usual in refractory irritable bowel syndrome: the ACTIB three-arm RCT.电话式认知行为疗法和基于网络的认知行为疗法联合常规治疗与常规治疗对照治疗难治性肠易激综合征的 ACTIB 三臂 RCT 研究。
Health Technol Assess. 2019 Apr;23(17):1-154. doi: 10.3310/hta23170.
3
Controlled treatment trials in the irritable bowel syndrome: a critique.肠易激综合征的对照治疗试验：一项批判性分析。
Gastroenterology. 1988 Jul;95(1):232-41. doi: 10.1016/0016-5085(88)90319-8.
4
Biofeedback for treatment of irritable bowel syndrome.生物反馈疗法治疗肠易激综合征
Cochrane Database Syst Rev. 2019 Nov 12;2019(11):CD012530. doi: 10.1002/14651858.CD012530.pub2.
5
Tegaserod for the treatment of irritable bowel syndrome and chronic constipation.替加色罗用于治疗肠易激综合征和慢性便秘。
Cochrane Database Syst Rev. 2007 Oct 17(4):CD003960. doi: 10.1002/14651858.CD003960.pub3.
6
Effectiveness of probiotics in irritable bowel syndrome: Updated systematic review with meta-analysis.益生菌在肠易激综合征中的有效性：更新的系统评价与荟萃分析
World J Gastroenterol. 2015 Mar 14;21(10):3072-84. doi: 10.3748/wjg.v21.i10.3072.
7
Different therapies of Chinese herbal medicine for diarrhea-predominant irritable bowel syndrome: A network meta-analysis of double-blinded, placebo-controlled trials.中药治疗腹泻型肠易激综合征的不同疗法：一项双盲、安慰剂对照试验的网络荟萃分析。
J Ethnopharmacol. 2023 Dec 5;317:116672. doi: 10.1016/j.jep.2023.116672. Epub 2023 Jun 14.
8
Tegaserod for the treatment of irritable bowel syndrome.替加色罗用于治疗肠易激综合征。
Cochrane Database Syst Rev. 2004(1):CD003960. doi: 10.1002/14651858.CD003960.pub2.
9
Efficacy and indication optimization of Chinese medicine (Tiao-Chang Ke-Min granules) for diarrhea-predominant irritable bowel syndrome: study protocol for a randomized controlled trial.中药（调肠克敏颗粒）治疗腹泻型肠易激综合征的疗效及适应证优化：一项随机对照试验的研究方案
Trials. 2018 Jul 11;19(1):367. doi: 10.1186/s13063-018-2754-9.
10
Homeopathy for treatment of irritable bowel syndrome.顺势疗法治疗肠易激综合征
Cochrane Database Syst Rev. 2013 Nov 13(11):CD009710. doi: 10.1002/14651858.CD009710.pub2.
引用本文的文献
1
Network meta-analysis of probiotics, prebiotics, and synbiotics for the treatment of chronic constipation in adults.益生菌、益生元和合生元治疗成人慢性便秘的网状荟萃分析。
Eur J Nutr. 2024 Sep;63(6):1999-2010. doi: 10.1007/s00394-024-03410-1. Epub 2024 May 2.
2
Acacia fiber or probiotic supplements to relieve gastrointestinal complaints in patients with constipation-predominant IBS: a 4-week randomized double-blinded placebo-controlled intervention trial.金合欢纤维或益生菌补充剂缓解便秘为主的 IBS 患者的胃肠道不适：为期 4 周的随机双盲安慰剂对照干预试验。
Eur J Nutr. 2024 Aug;63(5):1983-1994. doi: 10.1007/s00394-024-03398-8. Epub 2024 Apr 23.
3
An 8-Week Course of Bifidobacterium longum 35624 Is Associated with a Reduction in the Symptoms of Irritable Bowel Syndrome.服用长双歧杆菌35624八周疗程可减轻肠易激综合征的症状。
Probiotics Antimicrob Proteins. 2025 Feb;17(1):315-327. doi: 10.1007/s12602-023-10151-w. Epub 2023 Sep 13.
4
Efficacy and dose response of in diarrhea-predominant irritable bowel syndrome.在腹泻型肠易激综合征中的疗效和剂量反应。
World J Gastroenterol. 2023 Jul 28;29(28):4451-4465. doi: 10.3748/wjg.v29.i28.4451.
5
What Has Longitudinal 'Omics' Studies Taught Us about Irritable Bowel Syndrome? A Systematic Review.关于肠易激综合征，纵向“组学”研究告诉了我们什么？一项系统评价。
Metabolites. 2023 Mar 28;13(4):484. doi: 10.3390/metabo13040484.
6
Efficacy of Jawarish Shahi a herbal formulation in irritable bowel syndrome: An open-labeled single-arm clinical trial.草药配方贾瓦里什·沙希治疗肠易激综合征的疗效：一项开放标签单臂临床试验。
J Tradit Complement Med. 2022 Apr 22;12(6):529-535. doi: 10.1016/j.jtcme.2022.04.004. eCollection 2022 Nov.
7
Probiotics in irritable bowel syndrome - is the quest for the right strain over? Rapid review of existing guidelines and recommendations.肠易激综合征中的益生菌——寻找合适菌株的探索结束了吗？对现有指南和建议的快速回顾
Prz Gastroenterol. 2021;16(4):369-382. doi: 10.5114/pg.2021.111766. Epub 2021 Dec 19.
8
Targeted therapy of irritable bowel syndrome with anti-inflammatory cytokines.以抗炎细胞因子为靶点治疗肠易激综合征。
Clin J Gastroenterol. 2022 Feb;15(1):1-10. doi: 10.1007/s12328-021-01555-8. Epub 2021 Dec 4.
9
Prospective, double-blind diagnostic multicentre study of confocal laser endomicroscopy for wheat sensitivity in patients with irritable bowel syndrome.前瞻性、双盲、多中心诊断研究：共聚焦激光内镜检查在肠易激综合征患者小麦敏感性中的应用。
Gut. 2022 Aug;71(8):1567-1576. doi: 10.1136/gutjnl-2021-325181. Epub 2021 Sep 20.
10
Psychological and Gastrointestinal Symptoms of Patients with Irritable Bowel Syndrome Undergoing a Low-FODMAP Diet: The Role of the Intestinal Barrier.肠易激综合征患者行低 FODMAP 饮食治疗后出现的心理和胃肠道症状：肠道屏障的作用。
Nutrients. 2021 Jul 19;13(7):2469. doi: 10.3390/nu13072469.
本文引用的文献
1
Evaluating the functional net value of pharmacologic agents in treating irritable bowel syndrome.评估药物治疗肠易激综合征的功能净效值。
Aliment Pharmacol Ther. 2014 May;39(9):973-83. doi: 10.1111/apt.12692. Epub 2014 Mar 11.
2
Adverse events appear to unblind clinical trials in irritable bowel syndrome.
Neurogastroenterol Motil. 2014 Apr;26(4):482-8. doi: 10.1111/nmo.12289. Epub 2013 Dec 18.
3
Characteristics of functional bowel disorder patients: a cross-sectional survey using the Rome III criteria.功能性肠病患者的特征：使用罗马 III 标准的横断面调查。
Aliment Pharmacol Ther. 2014 Feb;39(3):312-21. doi: 10.1111/apt.12573. Epub 2013 Dec 5.
4
Validation of the Rome III criteria for the diagnosis of irritable bowel syndrome in secondary care.验证罗马 III 标准在二级医疗机构中对肠易激综合征的诊断价值。
Gastroenterology. 2013 Dec;145(6):1262-70.e1. doi: 10.1053/j.gastro.2013.08.048. Epub 2013 Aug 28.
5
Report from the multinational irritable bowel syndrome initiative 2012.2012 年多国肠易激综合征倡议报告。
Gastroenterology. 2013 Jun;144(7):e1-5. doi: 10.1053/j.gastro.2013.04.049. Epub 2013 Apr 30.
6
Patients suspected of irritable bowel syndrome--cross-sectional study exploring the sensitivity of Rome III criteria in primary care.疑诊为肠易激综合征的患者——一项在初级保健中探索罗马 III 标准敏感性的横断面研究。
Am J Gastroenterol. 2013 Jun;108(6):972-80. doi: 10.1038/ajg.2013.15. Epub 2013 Feb 19.
7
"Pre-cebo": an unrecognized issue in the interpretation of adequate relief during irritable bowel syndrome drug trials.“预给服”：在肠易激综合征药物试验中充分缓解解读方面的一个未被识别的问题。
J Clin Gastroenterol. 2012 Sep;46(8):686-90. doi: 10.1097/MCG.0b013e31825828a7.
8
Systematic review of diagnostic criteria for IBS demonstrates poor validity and utilization of Rome III.系统综述肠易激综合征的诊断标准表明罗马 III 标准的效度和实用性均较差。
Neurogastroenterol Motil. 2012 Sep;24(9):853-e397. doi: 10.1111/j.1365-2982.2012.01943.x. Epub 2012 May 28.
9
Effect of gender on prevalence of irritable bowel syndrome in the community: systematic review and meta-analysis.性别对社区中肠易激综合征患病率的影响：系统评价和荟萃分析。
Am J Gastroenterol. 2012 Jul;107(7):991-1000. doi: 10.1038/ajg.2012.131. Epub 2012 May 22.
10
Anxiety and depression in various functional gastrointestinal disorders: do differences exist?各种功能性胃肠疾病中的焦虑和抑郁：是否存在差异？
J Dig Dis. 2012 May;13(5):252-7. doi: 10.1111/j.1751-2980.2012.00581.x.
Zotero 插件