Flood Emuella, Silberg Debra G, Romero Beverly, Beusterien Kathleen, Erder M Haim, Cuffari Carmen
ICON plc, 820 W Diamond Ave, Ste 100, Gaithersburg, MD, 20878, USA.
Shire, Wayne, PA, USA.
BMC Res Notes. 2017 Sep 25;10(1):491. doi: 10.1186/s13104-017-2814-3.
The purpose of this study is to develop patient-reported (PRO) and observer-reported (ObsRO) outcome measures of ulcerative colitis (UC) signs/symptoms in children aged 5-17 with mild/moderate UC. The daily ulcerative colitis signs and symptoms scale (DUCS) was developed in two phases. Phase I involved concept elicitation interviews with patients and healthcare providers, review of website posts and item generation. Phase II involved cognitive debriefing and assessment of usability and feasibility of the eDiaries. Participants were recruited from five US clinical sites, a research recruitment agency, and internet advertising. Thematic and content analysis was performed to identify concepts from Phase I. The Phase II cognitive debriefing interviews were analyzed iteratively to identify problems with clarity and relevance of eDiary content. The US Food and Drug Administration (FDA) also reviewed and provided feedback on the eDiaries.
Phase I included 32 participants (22 remission; 10 active disease). Phase II included 38 participants (22 remission; 16 active disease). A core set of seven signs and symptoms emerged that were reported by at least 30% of the patients interviewed: abdominal pain, blood in stool, frequent stools, diarrhea, stool urgency, nighttime stools, and tiredness. Participant input influenced changes such as refinement of item wording, revision of graphics, and selection of response scales. Revisions suggested by FDA included simplifying the response scale and adding questions to capture symptoms during sleeping hours.
The findings of instrument development suggest that the DUCS PRO and ObsRO eDiaries are content-valid instruments for capturing the daily signs and symptoms of pediatric patients with mild to moderate UC in a clinical trial setting.
本研究的目的是开发针对5至17岁轻度/中度溃疡性结肠炎（UC）患儿的患者报告（PRO）和观察者报告（ObsRO）的UC体征/症状结局指标。每日溃疡性结肠炎体征和症状量表（DUCS）分两个阶段开发。第一阶段包括与患者和医疗服务提供者进行概念激发访谈、审查网站帖子以及生成条目。第二阶段包括认知反馈以及对电子日记的可用性和可行性进行评估。参与者从美国的五个临床地点、一家研究招募机构和互联网广告中招募。进行了主题和内容分析以从第一阶段识别概念。对第二阶段的认知反馈访谈进行了迭代分析，以识别电子日记内容在清晰度和相关性方面的问题。美国食品药品监督管理局（FDA）也对电子日记进行了审查并提供了反馈。
第一阶段包括32名参与者（22名缓解期；10名活动期疾病患者）。第二阶段包括38名参与者（22名缓解期；16名活动期疾病患者）。出现了一组由至少30%的受访患者报告的七个核心体征和症状：腹痛、便血、大便频繁、腹泻、便急、夜间排便和疲劳。参与者的意见影响了一些变化，如条目的措辞细化、图形的修订以及反应量表的选择。FDA建议的修订包括简化反应量表并添加问题以捕捉睡眠时间的症状。
量表开发的结果表明，DUCS PRO和ObsRO电子日记是在临床试验环境中捕捉轻度至中度UC儿科患者每日体征和症状的内容有效的工具。
