Calthorpe Rebecca J, Saumtally Hisham A, Howells Laura M, Goodchild Natalie J, Evans Bethinn C, Elliott Zoe, Hayee Bu'Hussain, Carr Siobhán B, Elston Caroline M, Horsley Alexander A R, Peckham Daniel G, Barr Helen L, Major Giles A D, Stewart Iain D, Thomas Kim S, Smyth Alan R
School of Medicine and NIHR Nottingham Biomedical Research Centre, Nottingham, United Kingdom.
Leeds Institute of Medical Research, University of Leeds, United Kingdom.
Mayo Clin Proc Digit Health. 2025 Mar 3;3(2):100203. doi: 10.1016/j.mcpdig.2025.100203. eCollection 2025 Jun.
To develop a cystic fibrosis (CF)-specific patient-reported outcome measure (PROM) to measure the daily burden of gastrointestinal symptoms for people with cystic fibrosis (pwCF) aged 12 years and older and address the lack of validated outcome measures for gastrointestinal symptoms in CF.
CF Tummy Tracker was developed through a 5-stage approach in accordance with regulatory guidance. This included development and refinement of a conceptual framework; item generation; refinement; reduction; selection; and initial PROM testing. A mixed-methods approach, consisting of expert panel discussions, a focus group, interviews, and an online survey, was used. In initial testing, participants completed the PROM daily for 14 days via a smartphone application. This study was performed from March 14, 2022, December 12, 2023.
The CF community were involved throughout the development via a focus group (n=7 pwCF), interviews (n=11 pwCF), and an online survey (n=180 pwCF). A formative model was confirmed for the PROM. The final PROM, CF Tummy Tracker, consists of 10 items capturing gastrointestinal symptom burden, tested in 151 pwCF. The PROM reported no floor or ceiling effects, high test-retest reliability (intra-class correlation coefficient=0.94), and strong correlation with the anchor question.
CF Tummy Tracker aims to address the gap in validated CF-specific PROMs for daily completion. Further testing of the psychometric properties of the PROM are planned in a new patient cohort to validate its use in clinical trials and support its use in both electronic and paper formats to increase accessibility.
开发一种针对囊性纤维化（CF）的患者报告结局测量工具（PROM），以测量12岁及以上囊性纤维化患者（pwCF）胃肠道症状的日常负担，并解决CF中缺乏经过验证的胃肠道症状结局测量工具的问题。
CF腹部追踪器根据监管指南通过五阶段方法开发。这包括概念框架的开发和完善；条目生成；完善；缩减；选择；以及PROM初始测试。采用了混合方法，包括专家小组讨论、焦点小组、访谈和在线调查。在初始测试中，参与者通过智能手机应用程序连续14天每天完成该PROM。本研究于2022年3月14日至2023年12月12日进行。
CF群体通过焦点小组（n = 7名pwCF）、访谈（n = 11名pwCF）和在线调查（n = 180名pwCF）全程参与开发。为该PROM确认了一个形成性模型。最终的PROM，即CF腹部追踪器，由10个捕捉胃肠道症状负担的条目组成，在151名pwCF中进行了测试。该PROM没有地板效应或天花板效应，重测信度高（组内相关系数 = 0.94），并且与锚定问题有很强的相关性。
CF腹部追踪器旨在填补经过验证的特定于CF的每日完成的PROM的空白。计划在新的患者队列中对该PROM的心理测量特性进行进一步测试，以验证其在临床试验中的应用，并支持其以电子和纸质格式使用，以提高可及性。
