Talbot Advisors LLC, PO Box 2121, Anna Maria, FL, 34217, USA.
Evidera, Bethesda, MD, USA.
Health Qual Life Outcomes. 2019 May 3;17(1):77. doi: 10.1186/s12955-019-1142-9.
The purpose of the current study was to determine the final content validation, psychometric characteristics, clinically meaningful improvement, and responder thresholds of the Clostridium difficile infection (CDI)-Daily Symptoms (CDI-DaySyms™) patient-reported outcome (PRO) questionnaire.
This validation study was part of two phase III studies (NCT01987895 and NCT01983683) conducted in patients with mild-to-moderate or severe CDI who completed the CDI-DaySyms™ daily throughout the treatment period. The questionnaire was evaluated in three stages: final PRO item content validation (Stage I); psychometric evaluation of reliability and construct validity (Stage II); and determination of clinically meaningful improvement and responder thresholds using distribution-based methods (Stage III).
The analysis included 168 patients. Most patients were female and Caucasian with mild-to-moderate CDI. The mean age was 57.1 years. Initial item analysis supported by confirmatory factor analysis demonstrated the relevance of 10 items grouped into three distinct domains (Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms). Domain scores demonstrated acceptable internal consistency and test-retest reliability, were sensitive to change, and correlated in expected directions with other relevant symptom and disease-severity measures. Responder thresholds were defined as score changes of - 1.00, - 0.80, and - 0.70 in the Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms domains, respectively.
The CDI-DaySyms™ is a valid measure of patient-reported CDI symptoms, with good measurement properties, which supports its utility as an endpoint in clinical studies. Further studies confirming responder thresholds based on anchor-based methods are required.
NCT01987895 , registered November 20, 2013; NCT01983683 , registered November 14, 2013.
本研究旨在确定艰难梭菌感染（CDI）-日常症状（CDI-DaySyms™）患者报告结局（PRO）问卷的最终内容验证、心理测量特征、临床意义改善以及应答者阈值。
这项验证性研究是两项 III 期研究（NCT01987895 和 NCT01983683）的一部分，入组了在治疗期间完成 CDI-DaySyms™日常记录的轻中度或重度 CDI 患者。问卷在三个阶段进行评估：最终 PRO 项目内容验证（阶段 I）；可靠性和结构有效性的心理测量评估（阶段 II）；使用基于分布的方法确定临床意义改善和应答者阈值（阶段 III）。
分析纳入了 168 例患者。大多数患者为女性，白种人，患有轻中度 CDI。平均年龄为 57.1 岁。初始项目分析和验证性因子分析均支持 10 项组成 3 个不同领域（腹泻症状、腹部症状和全身/其他症状）的相关性。领域评分显示出可接受的内部一致性和重测信度，对变化敏感，并与其他相关症状和疾病严重程度测量指标呈预期方向相关。应答者阈值定义为腹泻症状、腹部症状和全身/其他症状领域的评分变化分别为-1.00、-0.80 和-0.70。
CDI-DaySyms™是一种评估患者报告的 CDI 症状的有效工具，具有良好的测量特性，支持其作为临床研究终点的应用。需要进一步的研究来确认基于锚定方法的应答者阈值。
NCT01987895，于 2013 年 11 月 20 日注册；NCT01983683，于 2013 年 11 月 14 日注册。
