University of California San Diego, La Jolla, CA, USA.
University of Pennsylvania, Philadelphia, PA, USA.
J Crohns Colitis. 2022 Mar 14;16(3):444-451. doi: 10.1093/ecco-jcc/jjab161.
Patient-reported outcomes are recommended endpoints in Crohn's disease [CD] trials. The association between patient-reported general well-being relative to symptoms of diarrhoea and abdominal pain [AP] in patients with moderate to severe CD was explored.
Patients from three randomized, placebo-controlled, double-blind adalimumab or upadacitinib studies with average daily very soft/liquid stool frequency [SF] ≥4 and/or AP score ≥2 at baseline were included. Using electronic diaries, patients reported general well-being [seven-point Likert scale; 1 = worst; 7 = best] in item 10 of the Inflammatory Bowel Disease Questionnaire [IBDQ]. Changes in well-being and clinical outcomes of SF and AP from baseline to week 12 or 16, and the relationship between well-being and clinical outcomes were evaluated using cumulative distribution function and probability density function curves.
In total, 858 patients with CD were included [adalimumab, n = 695; upadacitinib, n = 163]. Patients who achieved clinical remission [SF ≤2.8, AP score ≤1.0, neither worse than baseline] were more likely than those not in clinical remission to report IBDQ item 10 response in the 6-7 group category but not IBDQ categories ≤5. Higher IBDQ score for item 10 [6-7] was associated with lower SF and AP score. Greater point increases in IBDQ item 10 were associated with a greater percentage decrease in clinical parameters; a ≥25-30% decrease in SF or AP was associated with a ≥1-point improvement in IBDQ.
An association between improvements in patient-reported general well-being and clinical remission/response was observed using outcomes of SF and AP, supporting the clinical remission/response endpoint definitions used in clinical studies of CD. Clinical Trial Registrations [ClinicalTrials.gov]: NCT00077779 [CHARM]; NCT00348283 [EXTEND]; NCT02365649 [CELEST].
患者报告的结局被推荐为克罗恩病（CD）试验的终点。本研究旨在探索中重度 CD 患者中，与腹泻和腹痛（AP）症状相比，患者报告的一般健康状况与一般健康状况的相关性。
纳入三项随机、安慰剂对照、双盲阿达木单抗或乌帕替尼治疗研究的患者，这些患者在基线时有平均每天非常软/液体粪便频率（SF）≥4 和/或 AP 评分≥2。使用电子日记，患者报告了炎症性肠病问卷（IBDQ）第 10 项的一般健康状况（7 分李克特量表；1=最差；7=最佳）。从基线到第 12 或 16 周，评估 SF 和 AP 的健康状况变化和临床结局，以及健康状况与临床结局之间的关系。
共纳入 858 例 CD 患者（阿达木单抗，n=695；乌帕替尼，n=163）。与未达到临床缓解的患者相比，达到临床缓解（SF≤2.8，AP 评分≤1.0，均不较基线差）的患者更有可能报告 IBDQ 第 10 项的 6-7 分组类别反应，但不报告 IBDQ 类别≤5。IBDQ 第 10 项（6-7）评分较高与 SF 和 AP 评分较低相关。IBDQ 第 10 项的积分增加与临床参数的百分比下降幅度更大相关；SF 或 AP 下降≥25-30%与 IBDQ 提高≥1 分相关。
使用 SF 和 AP 的结果观察到患者报告的一般健康状况改善与临床缓解/反应之间存在关联，这支持 CD 临床研究中使用的临床缓解/反应终点定义。临床试验注册：NCT00077779[CHARM]；NCT00348283[EXTEND]；NCT02365649[CELEST]。
