A grumbling concern: an international survey of gastrointestinal symptoms in cystic fibrosis in the modulator era [version 1; peer review: awaiting peer review]

Calthorpe, Rebecca J; Goodchild, Natalie; Gleetus, Vigilius; Premakumar, Vinuja; Hayee, Bu; Elliott, Zoe; Evans, Bethinn; Rowbotham, Nicola J; Carr, Siobhán B; Barr, Helen; Horsley, Alexander; Peckham, Daniel; Smyth, Alan R

Abstract

Background: Gastrointestinal symptoms in cystic fibrosis (CF) are common and intrusive to daily life. Relieving gastrointestinal symptoms was identified as an important research priority and previously explored in an international survey in 2018. However, following the widespread introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators in 2019, the landscape of CF treatment has changed. We repeated an online survey to further describe gastrointestinal symptoms and their effect on quality of life (QoL) in the CFTR modulator era. Methods: An electronic survey consisting of closed questions and free text responses was distributed via social media and professional networks for a period of one month between March - April 2022. People with CF (pwCF), their family and friends, and healthcare professionals (HCPs) were invited to take part. Results: There were 164 respondents: 88 pwCF (54%), 22 (13%) family, and 54 (33%) healthcare professionals (HCPs). A total of 89/110 (81%) pwCF or family members reported CFTR modulator treatment. The most commonly reported symptoms were wind / gas, rumbling stomach noises, loose motions (modulator) and bloating (no modulator). Abdominal pain and bloating had the greatest impact on QoL. For those on a CFTR modulator, the proportion of pwCF reporting “no change” or “worse” for all of the symptoms surveyed was greater than the proportion reporting an improvement. Following modulator introduction, dietary changes were recommended by 28/35 (80%) of HCPs and reported by 38/76 (50%) lay respondents. Changes in medication were recommended by 19/35 (54%) HCPs and reported by 44/76 (58%) of patients and family members. Conclusion This survey has shown that gastrointestinal symptoms remain prevalent in pwCF in the CFTR modulator era, though the nature of these symptoms may have changed. A better understanding of the underlying pathophysiology of these symptoms is essential. Future clinical studies should focus on improving symptoms and QoL.

Citation

Calthorpe, R. J., Goodchild, N., Gleetus, V., Premakumar, V., Hayee, B., Elliott, Z., Evans, B., Rowbotham, N. J., Carr, S. B., Barr, H., Horsley, A., Peckham, D., & Smyth, A. R. (2023). A grumbling concern: an international survey of gastrointestinal symptoms in cystic fibrosis in the modulator era [version 1; peer review: awaiting peer review]. NIHR Open Research, 3, Article 18. https://doi.org/10.3310/nihropenres.13384.1

• Journal Article Type: Article
• Acceptance Date: Apr 14, 2023
• Online Publication Date: Apr 14, 2023
• Publication Date: Apr 14, 2023
• Deposit Date: Apr 21, 2023
• Publicly Available Date: Apr 28, 2023
• Journal: NIHR Open Research
• Print ISSN: 2633-4402
• Publisher: F1000Research
• Peer Reviewed: Not Peer Reviewed
• Volume: 3
• Article Number: 18
• DOI: https://doi.org/10.3310/nihropenres.13384.1
• Public URL: https://nottingham-repository.worktribe.com/output/19788972
• Publisher URL: https://openresearch.nihr.ac.uk/articles/3-18/v1
• Additional Information: Referee status: Awaiting Peer Review; Grant Information: This project is funded by the National Institute for Health Research (NIHR) under its Programme Development Grant (Grant Reference Number: NIHR202952). The views expressed are those of the author(s) and not necessarily those of the NIHR or the Department of Health and Social Care. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.; Copyright: 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.