Hindryckx Pieter, Baert Filip, Hart Ailsa, Magro Fernando, Armuzzi Alessandro, Peyrin-Biroulet Laurent
Department of Gastroenterology, Ghent University Hospital, Ghent, Belgium.
Department of Gastroenterology, AZ Delta, Roeselare, Belgium.
J Crohns Colitis. 2015 Jul;9(7):580-8. doi: 10.1093/ecco-jcc/jjv074. Epub 2015 Apr 25.
The clinical trial landscape in ulcerative colitis has evolved significantly in recent decades. Study endpoints have been shifting from mere clinical response to mucosal healing. It has become clear that the choice of combined clinical and endoscopic outcome criteria leads to a reduction in placebo responses, especially when central reading of the endoscopic images is performed. Accumulating evidence suggests that histological remission yields better long-term outcomes for ulcerative colitis patients than mucosal healing alone, and clinical trials with prolonged follow-up will have to address whether histological remission should be the ultimate treatment goal in ulcerative colitis. In recent years there has also been increasing interest in the implementation of patient-reported outcomes in clinical practice and research, and the regulatory authorities have set up guidelines for the development of such outcomes. This paper aims to provide a comprehensive review of historical aspects of clinical trials in ulcerative colitis and to discuss challenges and perspectives for clinical trials in the near future. A thorough analysis of all available landmark literature (both original papers and reviews) on clinical trials in ulcerative colitis was performed.
近几十年来，溃疡性结肠炎的临床试验格局发生了显著变化。研究终点已从单纯的临床反应转向黏膜愈合。很明显，选择临床和内镜结果相结合的标准会减少安慰剂反应，尤其是在内镜图像进行中心阅片时。越来越多的证据表明，对于溃疡性结肠炎患者，组织学缓解比单纯黏膜愈合能产生更好的长期疗效，而进行长期随访的临床试验将不得不探讨组织学缓解是否应成为溃疡性结肠炎的最终治疗目标。近年来，临床实践和研究中对患者报告结局的应用也越来越受到关注，监管机构已制定了此类结局的开发指南。本文旨在全面回顾溃疡性结肠炎临床试验的历史方面，并讨论近期临床试验面临的挑战和前景。对所有关于溃疡性结肠炎临床试验的现有标志性文献（包括原始论文和综述）进行了深入分析。
