Serrano Daniel, Uzumcu Alyssa, Gerstein Maya, Ayasse Nicolai D, Engstrom Ella, Barnes Frederick B, Iaconangelo Charles, Thorat Teja, McGarry Lisa J, Sermet-Gaudelus Isabelle
Pharmerit International-An OPEN Health Company, Bethesda, MD, United States of America.
The Psychometrics Team, Sheridan, WY, United States of America.
PLoS One. 2025 Jan 24;20(1):e0317775. doi: 10.1371/journal.pone.0317775. eCollection 2025.
The Cystic Fibrosis (CF) Impact Questionnaire (CF-IQ) was qualitatively developed to assess the impact of CF in the context of treatment advancements and increased longevity. This study reports the CF-IQ validation. In this noninterventional validation study, people with CF completed the 40-item CF-IQ and validating patient-reported outcome measures (PROMs) via electronic diaries at enrollment (baseline) and at the 4-week follow-up. Validation consisted of modern methods and focus groups to finalize structural validity, and classical methods to assess internal consistency [1-3], test-retest reliability [4,5], concurrent validity [5], and known-groups validity [5] of the CF-IQ. At baseline, 214 adults completed the survey; 193 completed the follow-up survey. Unidimensional item response theory (IRT) models were separately fit to 5 prespecified domains (Control and Burden of CF Treatment Impacts, Physical Activity Impacts, Social Activity Impacts, Emotional Impacts, and Work/School Limitation Impacts). IRT local dependence (LD) statistics identified 17 redundant items. Two independent CF-patient focus groups (14 total patients) confirmed these findings, and the 17 items were dropped. Each domain defined on the final 23 items achieved the criterion of exact model fit as measured by the root mean squared error of approximation (RMSEA, values = 0), Internal consistency (Cronbach's α) values ranged from 0.81 to 0.89, 4 of 5 domains achieved acceptable test-retest reliability, with intraclass correlation coefficient (ICC) values ≥ 0.7, acceptable concurrent validity was achieved for all domains, and known-groups validity was established. The novel CF-IQ is a psychometrically robust PROM capturing patient-centric impacts of CF in the context of the current standard of care.
囊性纤维化（CF）影响问卷（CF-IQ）是为评估在治疗进展和预期寿命延长背景下CF的影响而进行定性开发的。本研究报告了CF-IQ的验证情况。在这项非干预性验证研究中，CF患者在入组时（基线）和4周随访时通过电子日记完成了40项CF-IQ以及验证患者报告结局指标（PROMs）。验证包括采用现代方法和焦点小组来确定结构效度，以及采用经典方法来评估CF-IQ的内部一致性[1-3]、重测信度[4,5]、同时效度[5]和已知群体效度[5]。在基线时，214名成年人完成了调查；193人完成了随访调查。单维项目反应理论（IRT）模型分别拟合5个预先指定的领域（CF治疗影响的控制与负担、身体活动影响、社交活动影响、情绪影响以及工作/学校限制影响）。IRT局部依赖性（LD）统计识别出17个冗余项目。两个独立的CF患者焦点小组（共14名患者）证实了这些发现，这17个项目被删除。由最终的23个项目定义的每个领域均达到了近似误差均方根（RMSEA，值 = 0）所衡量的精确模型拟合标准，内部一致性（Cronbach's α）值范围为0.81至0.89，5个领域中的4个达到了可接受的重测信度，组内相关系数（ICC）值≥0.7，所有领域均达到了可接受的同时效度，并确立了已知群体效度。新型CF-IQ是一种心理测量学上稳健的PROM，在当前护理标准背景下捕捉以患者为中心的CF影响。
