Exploring the nature of perceived treatment burden: a study to compare treatment burden measures in adults with cystic fibrosis

Background: Despite the importance of reducing treatment burden for people with cystic fibrosis (CF), it has not been fully understood as a concept. This study aims to quantify the treatment burden perceived by CF adults and explore the association between different validated treatment burden measures. Methods: This is a cross-sectional observational study of CF adults attending a single large UK adult center. Participants completed an online survey that contained three different treatment burden scales; CF Questionnaire-Revised (CFQ-R) subscale, CF Quality of Life (CFQoL) subscale, and the generic multimorbidity treatment burden questionnaire (MTBQ). Results: Among 101 participants, the median reported treatment burden by the CFQ-R subscale was 55.5 (IQR 33.3 – 66.6), the CFQoL subscale was 66.6 (IQR 46.6 – 86.6), and the MTBQ reversed global score was 84.6 (IQR 73.1 – 92.3). No correlation was found between respondents’ demographic or clinical variables and treatment burden measured via any of the three measures. All treatment burden measures showed correlations against each other. More treatments were associated with high treatment burden as measured by the CFQ-R, CFQoL subscales, and the MTBQ. However, longer treatment time and more complex treatment plans were correlated with high treatment burden as measured by the CFQ-R and CFQoL subscales, but not with the MTBQ. Conclusions: Treatment burden is a substantial issue in CF. Currently, the only available way to evaluate it is with the CF-specific quality of life measure treatment burden subscales (CFQ-R and CFQoL); both indicated that treatment burden increases with more treatments, longer treatment time, and more complex treatments

Exploring the nature of perceived treatment burden: a study to compare treatment burden measures in adults with cystic fibrosis [version 1; peer review: 2 approved]

Background: Despite the importance of reducing treatment burden for people with cystic fibrosis (CF), it has not been fully understood as a concept. This study aims to quantify the treatment burden perceived by CF adults and explore the association between different validated treatment burden measures. Methods: This is a cross-sectional observational study of CF adults attending a single large UK adult center. Participants completed an online survey that contained three different treatment burden scales; CF Questionnaire-Revised (CFQ-R) subscale, CF Quality of Life (CFQoL) subscale, and the generic multimorbidity treatment burden questionnaire (MTBQ). Results: Among 101 participants, the median reported treatment burden by the CFQ-R subscale was 55.5 (IQR 33.3 – 66.6), the CFQoL subscale was 66.6 (IQR 46.6 – 86.6), and the MTBQ reversed global score was 84.6 (IQR 73.1 – 92.3). No correlation was found between respondents’ demographic or clinical variables and treatment burden measured via any of the three measures. All treatment burden measures showed correlations against each other. More treatments were associated with high treatment burden as measured by the CFQ-R, CFQoL subscales, and the MTBQ. However, longer treatment time and more complex treatment plans were correlated with high treatment burden as measured by the CFQ-R and CFQoL subscales, but not with the MTBQ. Conclusions: Treatment burden is a substantial issue in CF. Currently, the only available way to evaluate it is with the CF-specific quality of life measure treatment burden subscales (CFQ-R and CFQoL); both indicated that treatment burden increases with more treatments, longer treatment time, and more complex treatments

Understanding treatment burden in cystic fibrosis, its association with health-related quality of life and implications for the economic evaluation

Background: The continuous advancements in cystic fibrosis (CF) treatments have improved the longevity of people with CF (pwCF). Eventually, the increased number and complexity of treatments elevate treatment burden. Decision-making bodies, such as NICE, prefer using EQ-5D as the generic health-related quality of life (HRQoL) measure in economic evaluations, however, limited data exist on its use in CF. Despite the importance of reducing treatment burden for pwCF, it remains an underexplored concept and is not yet considered in economic evaluation. This thesis aimed to provide evidence to enhance the focus on patient-centred outcomes through measuring HRQoL and treatment burden for pwCF to support economic evaluation and resource allocation. Systematic review: A systematic search explored methods used to measure CF-related treatment burden. Seventeen studies underwent narrative synthesis to identify the most utilised burden measures. Methods: The thesis consisted of four studies. Study 1 described HRQoL using EQ-5D-5L and its discriminatory ability toward CF disease severities. Study 2 assessed EQ-5D-5L convergent validity with the condition-specific CFQoL; evaluated HRQoL and well-being differences before and during COVID-19 pandemic. Study 3 described the nature of perceived treatment burden using three measures. Study 4 explored treatment burden association with EQ-5D utility score and considered how treatment burden might be captured in economic evaluations. Results: The EQ-5D-5L discriminated between CF disease severities and demonstrated convergent validity with CFQoL. High levels of perceived treatment burden among pwCF, were associated with increased daily treatments, treatment time, and treatment complexity. The EQ-5D utility score partially captures treatment burden. Conclusion: Reducing treatment burden is the desirable outcome for pwCF. This research revealed high levels of burden perceived by pwCF, emphasising the importance of addressing this issue. By incorporating treatment burden in economic evaluation, decision-makers can make more informed cost-effectiveness decisions which reduce burden, optimise resource allocation, and improve HRQoL for pwCF

Exploring the nature of perceived treatment burden: a study to compare treatment burden measures in adults with cystic fibrosis.

Background: Despite the importance of reducing treatment burden for people with cystic fibrosis (CF), it has not been fully understood as a concept. This study aims to quantify the treatment burden perceived by CF adults and explore the association between different validated treatment burden measures. Methods: This is a cross-sectional observational study of CF adults attending a single large UK adult center. Participants completed an online survey that contained three different treatment burden scales; CF Questionnaire-Revised (CFQ-R) subscale, CF Quality of Life (CFQoL) subscale, and the generic multimorbidity treatment burden questionnaire (MTBQ). Results: Among 101 participants, the median reported treatment burden by the CFQ-R subscale was 55.5 (IQR 33.3 – 66.6), the CFQoL subscale was 66.6 (IQR 46.6 – 86.6), and the MTBQ reversed global score was 84.6 (IQR 73.1 – 92.3). No correlation was found between respondents’ demographic or clinical variables and treatment burden measured via any of the three measures. All treatment burden measures showed correlations against each other. More treatments were associated with high treatment burden as measured by the CFQR, CFQoL subscales, and the MTBQ. However, longer treatment time and more complex treatment plans were correlated with high treatment burden as measured by the CFQ-R and CFQoL subscales, but not with the MTBQ. Conclusions: Treatment burden is a substantial issue in CF. Currently, the only available way to evaluate it is with the CF-specific quality of life measure treatment burden subscales (CFQ-R and CFQoL); both indicated that treatment burden increases with more treatments, longer treatment time, and more complex treatments