{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,14]],"date-time":"2026-11-14T01:26:39Z","timestamp":1794619599885,"version":"4.9.1"},"reference-count":18,"publisher":"National Institute for Health and Care Research","license":[{"start":{"date-parts":[[2022,5,9]],"date-time":"2022-05-09T00:00:00Z","timestamp":1652054400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100009128","name":"Research for Patient Benefit Programme","doi-asserted-by":"publisher","award":["PB-PG-1217-20018"],"award-info":[{"award-number":["PB-PG-1217-20018"]}],"id":[{"id":"10.13039\/501100009128","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["openresearch.nihr.ac.uk"],"crossmark-restriction":false},"short-container-title":["NIHR Open Res"],"abstract":"<ns4:p>\n                    <ns4:bold>Background:<\/ns4:bold>\n                    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.\n                  <\/ns4:p>\n                  <ns4:p>\n                    <ns4:bold>Methods:<\/ns4:bold>\n                    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).\n                  <\/ns4:p>\n                  <ns4:p>\n                    <ns4:bold>Results:<\/ns4:bold>\n                    Among 101 participants, the median reported treatment burden by the CFQ-R subscale was 55.5 (IQR 33.3 \u2013 66.6), the CFQoL subscale was 66.6 (IQR 46.6 \u2013 86.6), and the MTBQ reversed global score was 84.6 (IQR 73.1 \u2013 92.3). No correlation was found between respondents\u2019 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.\n                  <\/ns4:p>\n                  <ns4:p>\n                    <ns4:bold>Conclusions:<\/ns4:bold>\n                    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.\n                  <\/ns4:p>","DOI":"10.3310\/nihropenres.13260.1","type":"journal-article","created":{"date-parts":[[2022,5,9]],"date-time":"2022-05-09T09:20:09Z","timestamp":1652088009000},"page":"36","update-policy":"https:\/\/doi.org\/10.3310\/nihropenres.crossmark-policy","source":"Crossref","is-referenced-by-count":13,"title":["Exploring the nature of perceived treatment burden: a study to compare treatment burden measures in adults with cystic fibrosis"],"prefix":"10.3310","volume":"2","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8626-5269","authenticated-orcid":false,"given":"Rana","family":"Altabee","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0580-2478","authenticated-orcid":false,"given":"Siobhan B.","family":"Carr","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janice","family":"Abbott","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7442-0935","authenticated-orcid":false,"given":"Rory","family":"Cameron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Office","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessie","family":"Matthews","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Simmonds","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca","family":"Cosgriff","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1689-4147","authenticated-orcid":false,"given":"David","family":"Turner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Whitty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"6221","published-online":{"date-parts":[[2022,5,9]]},"reference":[{"key":"ref-1","first-page":"1","article-title":"UK CF Registry annual report 2020","year":"2021"},{"key":"ref-2","doi-asserted-by":"publisher","first-page":"218-227","DOI":"10.1016\/j.jcf.2017.11.019","article-title":"Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: A longitudinal study using UK patient registry data.","volume":"17","author":"R Keogh","year":"2018","journal-title":"J Cyst Fibros."},{"key":"ref-3","doi-asserted-by":"publisher","first-page":"91-6","DOI":"10.1016\/j.jcf.2008.09.007","article-title":"High treatment burden in adults with cystic fibrosis: challenges to disease self-management.","volume":"8","author":"G Sawicki","year":"2009","journal-title":"J Cyst Fibros."},{"key":"ref-4","doi-asserted-by":"publisher","first-page":"39-49","DOI":"10.2147\/PROM.S34681","article-title":"Building a measurement framework of burden of treatment in complex patients with chronic conditions: a qualitative study.","volume":"3","author":"D Eton","year":"2012","journal-title":"Patient Relat Outcome Meas."},{"key":"ref-5","doi-asserted-by":"publisher","first-page":"b2803","DOI":"10.1136\/bmj.b2803","article-title":"We need minimally disruptive medicine.","volume":"339","author":"C May","year":"2009","journal-title":"BMJ."},{"key":"ref-6","doi-asserted-by":"publisher","first-page":"24","DOI":"10.1186\/s40900-019-0159-x","article-title":"Adapting the James Lind Alliance priority setting process to better support patient participation: an example from cystic fibrosis.","volume":"5","author":"N Rowbotham","year":"2019","journal-title":"Res Involv Engagem."},{"key":"ref-7","doi-asserted-by":"publisher","first-page":"461-7","DOI":"10.1016\/j.jcf.2012.12.009","article-title":"Treatment complexity in cystic fibrosis: trends over time and associations with site-specific outcomes.","volume":"12","author":"G Sawicki","year":"2013","journal-title":"J Cyst Fibros."},{"key":"ref-8","doi-asserted-by":"publisher","first-page":"262","DOI":"10.1186\/s12877-019-1222-z","article-title":"A systematic literature review of the assessment of treatment burden experienced by patients and their caregivers.","volume":"19","author":"O Sheehan","year":"2019","journal-title":"BMC Geriatr."},{"key":"ref-9","doi-asserted-by":"publisher","first-page":"312-24","DOI":"10.1111\/hex.12046","article-title":"Burden of treatment for chronic illness: a concept analysis and review of the literature.","volume":"18","author":"A Sav","year":"2015","journal-title":"Health Expect."},{"key":"ref-10","doi-asserted-by":"publisher","first-page":"946-54","DOI":"10.1136\/thorax.55.11.946","article-title":"Development of a disease specific health related quality of life measure for adults and adolescents with cystic fibrosis.","volume":"55","author":"L Gee","year":"2000","journal-title":"Thorax."},{"key":"ref-11","doi-asserted-by":"publisher","first-page":"2347-54","DOI":"10.1378\/chest.128.4.2347","article-title":"Development and validation of The Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis.","volume":"128","author":"A Quittner","year":"2005","journal-title":"Chest."},{"key":"ref-12","doi-asserted-by":"publisher","first-page":"e019413","DOI":"10.1136\/bmjopen-2017-019413","article-title":"Development and validation of the Multimorbidity Treatment Burden Questionnaire (MTBQ).","volume":"8","author":"P Duncan","year":"2018","journal-title":"BMJ Open."},{"key":"ref-13","doi-asserted-by":"publisher","first-page":"109","DOI":"10.1186\/1741-7015-12-109","article-title":"Adaptation and validation of the Treatment Burden Questionnaire (TBQ) in English using an internet platform.","volume":"12","author":"V Tran","year":"2014","journal-title":"BMC Med."},{"key":"ref-14","article-title":"VALU-CF study questionnaire.","author":"R Altabee","year":"2022","journal-title":"figshare."},{"key":"ref-15","doi-asserted-by":"publisher","first-page":"373-384","DOI":"10.1007\/s40271-015-0118-z","article-title":"Sample Size Requirements for Discrete-Choice Experiments in Healthcare: a Practical Guide.","volume":"8","author":"E de Bekker-Grob","year":"2015","journal-title":"Patient."},{"key":"ref-16","doi-asserted-by":"publisher","first-page":"139-142","DOI":"10.1057\/jt.2009.5","article-title":"The correlation coefficient: Its values range between +1\/\u22121, or do they?","volume":"17","author":"B Ratner","year":"2009","journal-title":"J Target Meas Anal Mark."},{"key":"ref-17","doi-asserted-by":"publisher","first-page":"63-76","DOI":"10.1023\/a:1022037320039","article-title":"Development of the Cystic Fibrosis Questionnaire (CFQ) for Assessing Quality of Life in Pediatric and Adult Patients.","volume":"12","author":"H Bernadette","year":"2003","journal-title":"Qual Life Res."},{"key":"ref-18","doi-asserted-by":"publisher","first-page":"140","DOI":"10.1186\/s12874-017-0411-8","article-title":"Measuring the burden of treatment for chronic disease: implications of a scoping review of the literature.","volume":"17","author":"A Sav","year":"2017","journal-title":"BMC Med Res Methodol."}],"container-title":["NIHR Open Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1\/xml","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1\/pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1\/iparadigms","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,15]],"date-time":"2023-08-15T11:51:10Z","timestamp":1692100270000},"score":1,"resource":{"primary":{"URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,5,9]]},"references-count":18,"URL":"https:\/\/doi.org\/10.3310\/nihropenres.13260.1","relation":{"has-review":[{"id-type":"doi","id":"10.3310\/nihropenres.14376.r28651","asserted-by":"subject"},{"id-type":"doi","id":"10.3310\/nihropenres.14376.r28841","asserted-by":"subject"},{"id-type":"doi","id":"10.3310\/nihropenres.14376.r28841","asserted-by":"object"},{"id-type":"doi","id":"10.3310\/nihropenres.14376.r28651","asserted-by":"object"}]},"ISSN":["2633-4402"],"issn-type":[{"value":"2633-4402","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,5,9]]},"assertion":[{"value":"Indexed","URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1#article-reports","order":0,"name":"referee-status","label":"Referee status","group":{"name":"current-referee-status","label":"Current Referee Status"}},{"value":"10.3310\/nihropenres.14376.r28651, Gary Latchford, Leeds Institute of Health Sciences, University of Leeds, Leeds, UK, 01 Jun 2022, version 1, indexed","URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1#referee-response-28651","order":0,"name":"referee-response-28651","label":"Referee Report","group":{"name":"article-reports","label":"Article Reports"}},{"value":"<b>Rana Altabee<\/b>; \n<i>Posted: 08 Jun 2022<\/i>; We thank the reviewer for his kind and helpful comments.  We hope that this study would highlight the importance of capturing treatment burden in people with CF, especially with the new era of modulators.&nbsp;Since the CF-specific treatment burden subscales were the most adequate in capturing treatment burden in CF, it would be great to see future studies developing a new treatment burden instrument for CF.","URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1#referee-comment-3206","order":1,"name":"referee-comment-3206","label":"Referee Comment","group":{"name":"article-reports","label":"Article Reports"}},{"value":"10.3310\/nihropenres.14376.r28841, Nataliya Kashirskaya, Research Centre for Medical Genetics, Moscow, Russian Federation, 10 Nov 2022, version 1, indexed","URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1#referee-response-28841","order":2,"name":"referee-response-28841","label":"Referee Report","group":{"name":"article-reports","label":"Article Reports"}},{"value":"<b>Rana Altabee<\/b>; \n<i>Posted: 14 Nov 2022<\/i>; We thank the reviewer for her helpful and valuable feedback.  Currently, the CF-specific quality of life measures (CFQ-R and CFQoL) treatment burden subscales are the only available methods used to capture treatment burden from the pwCF perspective. However, treatment burden is much bigger in concept than what\u2019s covered by the 3-itemed subscales. Therefore, we hope with this study to highlight the importance of treatment burden for the CF population, especially with the availability of the CFTR modulators.","URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1#referee-comment-3219","order":3,"name":"referee-comment-3219","label":"Referee Comment","group":{"name":"article-reports","label":"Article Reports"}},{"value":"<b>Rana Altabee<\/b>; \n<i>Posted: 01 Jul 2022<\/i>; We thank Hoo and Wildman for their interesting thoughts about our study. Nobody has yet defined what treatment burden is for people with CF. In order to do that, a comprehensive qualitative study needs to explain what treatment burden means for people with CF. Treatment time, complexity, and the number of treatments are all different aspects of the overall treatment burden. The CFQ-R and CFQoL subscales are the only available instruments that claim to subjectively capture treatment burden for people with CF.&nbsp;  We looked at the internal consistency of the CFQ-R and the CFQoL treatment burden domains in our study and found them both acceptable (CFQ-R Cronbach\u2019s alpha= 0.73 and CFQoL Cronbach\u2019s alpha= 0.88). However, treatment burden is bigger than to be captured in a three items subscale. Thus, we assessed the performance of the 13-item generic MTBQ compared to the condition-specific subscale, but unfortunately, it underperformed the other two subscales in people with CF. Therefore, we believe that it is greatly important to develop a treatment burden measure that looks into all the aspects that affect treatment burden from the CF population\u2019s perspective.","URL":"https:\/\/openresearch.nihr.ac.uk\/articles\/2-36\/v1#article-response-3213","order":4,"name":"article-response-3213","label":"Article Comment","group":{"name":"article-comments","label":"Article Comments"}},{"value":"This project is funded by the National Institute for Health Research (NIHR) under its Research for Patient Benefit (RfPB) Programme (Grant Reference Number PB-PG-1217-20018). The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care. RA is funded by a PhD studentship from King Saud bin Abdulaziz University for Health Sciences (KSAU-HS). JW and RC involvement were also supported by the National Institute of Health Research (NIHR) Applied Research Collaboration East of England (ARC EoE) program. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.","order":5,"name":"grant-information","label":"Grant Information"},{"value":"This is an open access article distributed under the terms of the Creative Commons Attribution Licence, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.","order":0,"name":"copyright-info","label":"Copyright"}]}}