Maastricht University Medical Center+, Department Gastroenterology and Hepatology, Maastricht, The Netherlands.
Maastricht University Medical Center+, NUTRIM - School for Nutrition and Translational Research in Metabolism, Maastricht, The Netherlands.
J Crohns Colitis. 2019 Apr 26;13(5):555-563. doi: 10.1093/ecco-jcc/jjy196.
Patient-reported outcome measures [PROMs] assessing inflammatory bowel disease [IBD] activity are of interest for monitoring in clinical practice, telemedicine systems, or trials. Different PROMs for follow-up of disease activity are available; however, none was developed with endoscopy as gold standard. The objective of this study was to develop and validate a PROM to predict endoscopic disease activity, following the recommendations of the Food and Drug Administration.
During development, 178 IBD patients undergoing a colonoscopy were asked to fill out 13 clinical questions derived from the literature. During endoscopy, inflammation was assessed with the simplified endoscopic score for Crohn's disease [CD] and the Mayo endoscopic subscore for ulcerative colitis [UC]. Based on correlation with endoscopic inflammation, questions were reduced to a total of six for CD and five for UC. The newly developed Monitor IBD At Home questionnaire [MIAH] was validated in an independent cohort of 135 CD and 131 UC patients. Additionally, diagnostic accuracy of the MIAH combined with a calprotectin home test [CHT] was assessed.
The MIAH-CD includes questions on rectal bleeding, mucus, stool frequency, urgency, fatigue, and patient-reported disease activity. The MIAH-UC contains items on rectal bleeding, stool frequency, urgency, abdominal pain, and patient-reported disease activity. Both questionnaires showed to be valid, reliable, and responsive to changes. The MIAH and CHT combined had a sensitivity, specificity, negative predictive value [NPV], and positive predicitive value [PPV] of 96.7%, 66.7%, 94.7%, and 76.3% for CD and of 88.2%, 81.4%, 95.6%, and 60.0% for UC, respectively, compared with endoscopy.
The MIAH is the first PROM developed to predict endoscopic inflammation in IBD patients. A combination of this questionnaire and a CHT shows excellent diagnostic accuracy to screen for patients who need further assessment of disease activity, and can be used in daily practice, telemedicine systems, and trials.
评估炎症性肠病（IBD）活动的患者报告结局测量（PROM）在临床实践、远程医疗系统或试验中用于监测具有重要意义。目前已有多种用于疾病活动随访的 PROM，但均未以内镜作为金标准进行开发。本研究的目的是根据美国食品和药物管理局的建议，开发和验证一种用于预测内镜疾病活动的 PROM。
在开发过程中，对 178 名接受结肠镜检查的 IBD 患者进行了问卷调查，问卷内容来自文献中的 13 个临床问题。在内镜检查过程中，使用简化克罗恩病内镜评分（SES-CD）和溃疡性结肠炎内镜评分（MCS）评估炎症。根据与内镜炎症的相关性，将问题减少到 SES-CD 共 6 个，MCS 共 5 个。在独立的 135 例 CD 和 131 例 UC 患者队列中验证了新开发的 Monitor IBD At Home 问卷（MIAH）。此外，还评估了 MIAH 联合粪便钙卫蛋白检测（CHT）的诊断准确性。
MIAH-CD 包括直肠出血、黏液、粪便频率、便急、疲劳和患者报告的疾病活动等问题。MIAH-UC 包含直肠出血、粪便频率、便急、腹痛和患者报告的疾病活动等项目。两个问卷都具有有效性、可靠性和对变化的反应性。MIAH 和 CHT 联合检测 CD 的敏感性、特异性、阴性预测值（NPV）、阳性预测值（PPV）分别为 96.7%、66.7%、94.7%和 76.3%，UC 分别为 88.2%、81.4%、95.6%和 60.0%，与内镜相比。
MIAH 是首个用于预测 IBD 患者内镜炎症的 PROM。该问卷与 CHT 的联合检测具有极好的诊断准确性，可用于筛查需要进一步评估疾病活动的患者，可用于日常实践、远程医疗系统和临床试验。
