Muilwijk Danya, van Paridon Tessa J, van der Heijden Doris C, Faber-Bisschop Brenda M, Zomer-van Ommen Domenique D, Heijerman Harry G M, van der Ent Cornelis K
Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Lundlaan 6, 3584 EA, Utrecht, the Netherlands.
Department of Pulmonology, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX, Utrecht, the Netherlands.
EClinicalMedicine. 2023 Jul 27;62:102116. doi: 10.1016/j.eclinm.2023.102116. eCollection 2023 Aug.
Generic and disease-specific patient-reported outcome measures (PROMs) may lack relevance and sensitivity on a patient-level in chronic diseases with differential disease expression and high individual variability, such as Cystic Fibrosis (CF). This study aimed to develop and validate a novel personalized electronic PROM (ePROM) that captures relevant aspects of quality of life in individuals with CF.
The Q-Life app was developed as a short personalized ePROM to assess individual quality of life. Psychometric properties were assessed in a single-center cross-sectional study between September 2019 and September 2021 and in a prospective cohort study between September 2021 and September 2022.
Combined studies included 223 participants (median age: 24 years, IQR: 19.0-32.5 years, range: 12.0-58.0 years). Internal consistency (Cronbach's alpha: 0.83-0.90) and test-retest reliability (intraclass correlation coefficient: 0.90; 95% CI: 0.65-0.92; p < 0.001) of quality of life (Q-Life) scores were strong. Q-Life scores were associated with overall Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores (ρ = 0.71; p < 0.001), CFQ-R respiratory domain scores (ρ = 0.57; p < 0.001) and forced expiratory volume in 1s (ρ = 0.41; p < 0.001). Furthermore, Q-Life scores improved from 65.0 (IQR: 45.0-63.3) at baseline to 84.2 (IQR: 75.0-95.0) and 87.5 (IQR: 75.0-100.0) after 3 and 6 months of elexacaftor/tezacaftor/ivacaftor treatment (change: 20.8; 95% CI: 17.5-25.0; p < 0.001), comparable to CFQ-R respiratory domain scores (change: 22.2, 95% CI: 19.4-25.0, p < 0.001).
The Q-Life app is a reliable, valid and sensitive personalized ePROM to measure all aspects of quality of life that really matter to individuals with Cystic Fibrosis. This patient-centered approach could provide important advantages over generic and disease-specific PROMs in the era of personalized medicine and value-based healthcare.
Dutch Cystic Fibrosis Foundation, Health-Holland.
在诸如囊性纤维化（CF）这种疾病表现存在差异且个体变异性高的慢性疾病中，一般的和特定疾病的患者报告结局测量指标（PROMs）在患者层面可能缺乏相关性和敏感性。本研究旨在开发并验证一种新型的个性化电子患者报告结局测量指标（ePROM），以捕捉CF患者生活质量的相关方面。
开发了Q-Life应用程序作为一种简短的个性化ePROM，用于评估个体生活质量。在2019年9月至2021年9月期间的一项单中心横断面研究以及2021年9月至2022年9月期间的一项前瞻性队列研究中对其心理测量特性进行了评估。
综合研究纳入了223名参与者（中位年龄：24岁，四分位间距：19.0 - 32.5岁，范围：12.0 - 58.0岁）。生活质量（Q-Life）评分的内部一致性（Cronbach's α系数：0.83 - 0.90）和重测信度（组内相关系数：0.90；95%置信区间：0.65 - 0.92；p < 0.001）很强。Q-Life评分与总体囊性纤维化问卷修订版（CFQ-R）评分（ρ = 0.71；p < 0.001）、CFQ-R呼吸领域评分（ρ = 0.57；p < 0.001）以及一秒用力呼气量（ρ = 0.41；p < 0.001）相关。此外，在接受依列卡福/替扎卡福/依伐卡福治疗3个月和6个月后，Q-Life评分从基线时的65.0（四分位间距：45.0 - 63.3）提高到84.2（四分位间距：75.0 - 95.0）和87.5（四分位间距：75.0 - 100.0）（变化：20.8；95%置信区间：17.5 - 25.0；p < 0.001），与CFQ-R呼吸领域评分相当（变化：22.2，95%置信区间：19.4 - 25.0，p < 0.001）。
Q-Life应用程序是一种可靠、有效且敏感的个性化ePROM，可用于测量对囊性纤维化患者真正重要的生活质量的各个方面。在个性化医疗和基于价值的医疗保健时代，这种以患者为中心的方法可能比一般的和特定疾病的PROMs具有重要优势。
荷兰囊性纤维化基金会，荷兰健康促进组织。
