Inserm, U954 et Service d'Hepato-Gastroenterologie, Hôpital Universitaire de Nancy, Université Henri Poincaré 1, Vandoeuvre-lès-Nancy, France.
Division of Gastroenterology, University of California San Diego, La Jolla, California.
Clin Gastroenterol Hepatol. 2014 Aug;12(8):1246-56.e6. doi: 10.1016/j.cgh.2014.02.016. Epub 2014 Feb 15.
The Food and Drug Administration (FDA) is moving from the Crohn's Disease Activity Index to patient-reported outcomes (PROs) and objective measures of disease, such as findings from endoscopy. PROs will become an important aspect of assessing activity of inflammatory bowel disease (IBD) and for labeling specific drugs for this disease. PROs always have been considered in the management of patients with rheumatoid arthritis or multiple sclerosis, and have included measurements of quality of life, disability, or fatigue. Several disease-specific scales have been developed to assess these PROs and commonly are used in clinical trials. Outcomes reported by patients in clinical trials of IBD initially focused on quality of life, measured by the Short-Form 36 questionnaire or disease-specific scales such as the Inflammatory Bowel Disease Questionnaire or its shorter version. Recently considered factors include fatigue, depression and anxiety, and work productivity, as measured by the Functional Assessment Chronic Illness Therapy-Fatigue, the Hospital Anxiety Depression, and the Work Productivity Activity Impairment Questionnaire, respectively. However, few data are available on how treatment affects these factors in patients with IBD. Although disability generally is recognized in patients with IBD, it is not measured. The international IBD disability index currently is being validated. None of the PROs currently used in IBD were developed according to FDA guidance for PRO development. PROs will be a major primary end point of future trials. FDA guidance is needed to develop additional PROs for IBD that can be incorporated into trials, to better compare patients' experience with different therapies.
美国食品和药物管理局（FDA）正在从克罗恩病活动指数转向患者报告的结果（PROs）和疾病的客观指标，例如内窥镜检查结果。PROs 将成为评估炎症性肠病（IBD）活动的重要方面，并为该疾病的特定药物贴上标签。PROs 在治疗类风湿关节炎或多发性硬化症患者时一直被考虑在内，包括生活质量、残疾或疲劳的衡量标准。已经开发了几种针对这些 PROs 的特定疾病量表，并通常在临床试验中使用。IBD 临床试验中报告的患者结果最初侧重于生活质量，通过短格式 36 问卷或炎症性肠病问卷或其较短版本等特定疾病量表进行衡量。最近考虑的因素包括疲劳、抑郁和焦虑以及工作效率，分别通过功能评估慢性疾病治疗-疲劳、医院焦虑抑郁和工作效率活动障碍问卷进行衡量。然而，关于治疗如何影响 IBD 患者的这些因素的数据很少。尽管残疾在 IBD 患者中普遍被认识到，但它没有被测量。国际 IBD 残疾指数目前正在验证中。目前在 IBD 中使用的 PROs 都不是根据 FDA 对 PRO 开发的指南开发的。PROs 将成为未来试验的主要主要终点。需要 FDA 指南来开发可纳入试验的用于 IBD 的额外 PROs，以更好地比较不同治疗方法对患者体验的影响。
