Calthorpe, R.J., Goodchild, N., Gleetus, V. et al. (10 more authors) (2024) A grumbling concern: A survey of gastrointestinal symptoms in cystic fibrosis in the modulator era. NIHR Open Research, 3. ISSN 2633-4402
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 and rumbling stomach noises (borborygmi) in both the modulator and non-modulator groups in addition to loose motions (modulator group) and bloating (no modulator group). 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. For some symptoms such as stomach pains and reduced appetite, improvements were perceived more commonly in HCPs than what was reported by pwCF. Following modulator introduction, dietary changes to manage GI symptoms 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.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © Crown copyright, 2024 Calthorpe RJ et al.. This open access work is licensed under the Open Government Licence v3.0 |
Keywords: | Respiratory, cystic fibrosis, gastrointestinal symptoms, CFTR modulators |
Dates: |
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Institution: | The University of Leeds |
Academic Units: | The University of Leeds > Faculty of Medicine and Health (Leeds) > School of Medicine (Leeds) > Leeds Institute of Medical Research (LIMR) > Division of Molecular Medicine |
Funding Information: | Funder Grant number NIHR National Inst Health Research 21GCS001 |
Depositing User: | Symplectic Publications |
Date Deposited: | 08 Jul 2024 10:48 |
Last Modified: | 08 Jul 2024 10:48 |
Published Version: | http://dx.doi.org/10.3310/nihropenres.13384.2 |
Status: | Published |
Publisher: | National Institute for Health and Care Research |
Identification Number: | 10.3310/nihropenres.13384.2 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:214018 |