一个令人担忧的问题：调节剂时代囊性纤维化患者胃肠道症状的调查
A grumbling concern: A survey of gastrointestinal symptoms in cystic fibrosis in the modulator era.
作者信息
Calthorpe Rebecca J, Goodchild Natalie, Gleetus Vigilius, Premakumar Vinishaa, Hayee Bu, Elliott Zoe, Evans Bethinn, Rowbotham Nicola J, Carr Siobhán B, Barr Helen, Horsley Alexander, Peckham Daniel, Smyth Alan R
机构信息
University of Nottingham School of Medicine & NIHR Nottingham Biomedical Research Centre, Nottingham, UK.
Patient and Public representative, N/A, UK.
出版信息
NIHR Open Res. 2024 Feb 5;3:18. doi: 10.3310/nihropenres.13384.1. eCollection 2023.
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.
背景
囊性纤维化（CF）患者的胃肠道症状很常见，且会干扰日常生活。缓解胃肠道症状被确定为一项重要的研究重点，此前在2018年的一项国际调查中对此进行了探讨。然而，随着2019年囊性纤维化跨膜传导调节因子（CFTR）调节剂的广泛应用，CF的治疗格局发生了变化。我们重复进行了一项在线调查，以进一步描述CFTR调节剂时代的胃肠道症状及其对生活质量（QoL）的影响。
方法
2022年3月至4月期间，通过社交媒体和专业网络开展了为期一个月的电子调查，调查内容包括封闭式问题和自由文本回复。邀请CF患者（pwCF）、他们的家人和朋友以及医疗保健专业人员（HCPs）参与。
结果
共有164名受访者：88名pwCF（54%）、22名（13%）家属和54名（33%）医疗保健专业人员（HCPs）。共有89/110（81%）的pwCF或家庭成员报告接受了CFTR调节剂治疗。在调节剂组和非调节剂组中，最常报告的症状都是肠胃胀气和肚子咕噜咕噜响（腹鸣），此外还有腹泻（调节剂组）和腹胀（非调节剂组）。腹痛和腹胀对生活质量的影响最大。对于使用CFTR调节剂的患者，报告所有调查症状“无变化”或“恶化”的pwCF比例高于报告症状改善的比例。对于一些症状，如胃痛和食欲下降，HCPs认为症状改善的情况比pwCF报告的更为常见。在引入调节剂后，28/35（80%）的HCPs建议通过饮食改变来管理胃肠道症状，38/76（50%）的非专业受访者报告了这种情况。19/35（54%）的HCPs建议改变药物治疗，44/76（58%）的患者和家庭成员报告了这种情况。
结论
这项调查表明，在CFTR调节剂时代，pwCF的胃肠道症状仍然普遍存在，尽管这些症状的性质可能已经发生了变化。更好地了解这些症状的潜在病理生理学至关重要。未来的临床研究应侧重于改善症状和生活质量。
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