Regeneron Pharmaceuticals, Tarrytown, NY, USA.
Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
J Patient Rep Outcomes. 2023 Nov 27;7(1):120. doi: 10.1186/s41687-023-00654-z.
Eosinophilic esophagitis (EoE) has a detrimental effect on health-related quality of life (HRQOL). The Eosinophilic Esophagitis Impact Questionnaire (EoE-IQ) is a novel patient-reported outcome (PRO) measure assessing the impact of EoE on HRQOL. To assess suitability of the EoE-IQ, its measurement properties were evaluated.
Using baseline and week 24 data from the pivotal, randomized, placebo-controlled, multinational phase 3 R668-EE-1774 trial (NCT03633617) of dupilumab, we evaluated EoE-IQ's measurement properties (including reliability, construct and known-groups validity, and ability to detect change) and established the threshold for change in scores that can be considered clinically meaningful.
The analysis population comprised 239 adults and adolescents with EoE. Mean age was 28.1 (standard deviation, 13.14) years; 63.6% were male, and 90.4% were White. Reliability estimates for the EoE-IQ average score exceeded acceptable thresholds for patients who were stable as indicated by ratings of Patient Global Impression of Severity (PGIS) and Change (PGIC) (intraclass correlation coefficients, 0.75 and 0.81). Construct validity correlations with other EoE-specific PRO scores were moderate at baseline (|r|= 0.44-0.60) and moderate to strong at week 24 (|r|= 0.61-0.72). In known-groups analysis, EoE-IQ average score discriminated among groups of patients at varying EoE severity levels defined by PGIS scores. A ≥ 0.6-point reduction in EoE-IQ average score (where scores range from 1 to 5, with higher scores indicating worse HRQOL) from baseline to week 24 can be considered clinically meaningful.
The EoE-IQ's measurement properties are acceptable, making it a valid, reliable measure of the HRQOL impacts of EoE among adults and adolescents.
ClinicalTrials.gov, NCT03633617. Registered August 14, 2018, https://clinicaltrials.gov/study/NCT03633617 .
嗜酸性食管炎 (EoE) 对健康相关生活质量 (HRQOL) 有不利影响。嗜酸性食管炎影响问卷 (EoE-IQ) 是一种新的患者报告结局 (PRO) 测量工具，用于评估 EoE 对 HRQOL 的影响。为了评估 EoE-IQ 的适用性，对其测量特性进行了评估。
使用关键性、随机、安慰剂对照、多中心 3 期 R668-EE-1774 试验（NCT03633617）的基线和第 24 周数据，我们评估了 EoE-IQ 的测量特性（包括可靠性、结构和已知组有效性，以及检测变化的能力），并确定了可以认为是临床有意义的评分变化阈值。
分析人群包括 239 名患有 EoE 的成年人和青少年。平均年龄为 28.1（标准差，13.14）岁；63.6%为男性，90.4%为白人。根据患者总体严重程度评分（PGIS）和变化评分（PGIC）的稳定评分，EoE-IQ 平均得分的可靠性估计值超过了可接受的阈值（组内相关系数分别为 0.75 和 0.81）。与其他 EoE 特异性 PRO 评分的结构有效性相关性在基线时为中度（|r|=0.44-0.60），在第 24 周时为中度至强度（|r|=0.61-0.72）。在已知组分析中，EoE-IQ 平均得分可以区分不同严重程度的 EoE 患者组，这些患者的严重程度是根据 PGIS 评分来定义的。从基线到第 24 周，EoE-IQ 平均得分下降≥0.6 分（得分范围为 1 到 5，得分越高表示 HRQOL 越差）可被认为具有临床意义。
EoE-IQ 的测量特性是可以接受的，这使其成为一种成人和青少年中评估 EoE 对 HRQOL 影响的有效、可靠的测量工具。
ClinicalTrials.gov，NCT03633617。注册于 2018 年 8 月 14 日，网址为 https://clinicaltrials.gov/study/NCT03633617。
