TY - JOUR
T1 - Development and validation of a novel personalized electronic patient-reported outcome measure to assess quality of life (Q-LIFE)
T2 - a prospective observational study in people with Cystic Fibrosis
AU - Muilwijk, Danya
AU - van Paridon, Tessa J.
AU - van der Heijden, Doris C.
AU - Faber-Bisschop, Brenda M.
AU - Zomer-van Ommen, Domenique D.
AU - Heijerman, Harry G.M.
AU - van der Ent, Cornelis K.
N1 - Funding Information:
This project received funding from the Dutch Cystic Fibrosis Foundation (CFOS 2019 and CFOS 2020) and Health Holland (project number TKI-LSH-DT2019-NCFS).
Funding Information:
Dutch Cystic Fibrosis Foundation, Health-Holland.We are thankful to all Dutch people with CF and parents of children with CF who were involved in the development process of the Q-Life app; the people with CF across Europe who were involved in the development and testing of the international versions of the Q-Life app; and those who participated in this study and provided important and valuable feedback for further development and implementation. We also thank the Dutch Cystic Fibrosis Foundation and CF Europe for their support and collaboration in this project; Solviteers for the development and hosting of the Q-Life app; and the CF clinical research team of the Wilhelmina Children's Hospital and UMC Utrecht for their help with the organization and data collection during the cohort study. This project received funding from the Dutch Cystic Fibrosis Foundation (CFOS 2019 and CFOS 2020) and Health Holland (project number TKI-LSH-DT2019-NCFS).
Publisher Copyright:
© 2023 The Author(s)
PY - 2023/8
Y1 - 2023/8
N2 - Background: 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. Methods: 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. Findings: 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). Interpretation: 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. Funding: Dutch Cystic Fibrosis Foundation, Health-Holland.
AB - Background: 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. Methods: 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. Findings: 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). Interpretation: 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. Funding: Dutch Cystic Fibrosis Foundation, Health-Holland.
KW - Cystic Fibrosis
KW - Patient-reported outcome measures
KW - Personalized medicine
KW - Quality of life
UR - https://www.scopus.com/pages/publications/85165920310
U2 - 10.1016/j.eclinm.2023.102116
DO - 10.1016/j.eclinm.2023.102116
M3 - Article
C2 - 37554124
AN - SCOPUS:85165920310
SN - 2589-5370
VL - 62
JO - EClinicalMedicine
JF - EClinicalMedicine
M1 - 102116
ER -