Study data set.

BackgroundGastrointestinal (GI) complications are the second most common disorders in persons with cystic fibrosis (PwCF). There is limited data on how having a dedicated CF-GI clinic and cystic fibrosis transmembrane conductance regulator (CFTR) modulators may affect rates of GI complications. Our aim was to assess the effect of the CF-GI clinic and CFTR modulators on GI complications with incidence of distal intestinal obstructive syndrome (DIOS).MethodsThis was a retrospective study of adult PwCF who were seen in a CF-GI clinic from 2000–2023. Comparisons were made between the numbers of admissions and emergency department (ED) visits for DIOS at three years before and after CFTR modulator use and the first CF-GI clinic visit.ResultsOf the 1,076 PwCF identified, 242 were seen in CF-GI clinic. Of this, 126 (52.1%) were female, with a median age of 40 (IQR: 30–47) years. There were 146 (60.3%) with regular use of laxatives. Of the 59 PwCF with CF-GI clinic visits for constipation, hospital admissions decreased in 16, were unchanged in 32, and increased in 11 (p = 0.402) while ED visits decreased in 9, remained the same in 40, and increased in 10 (p = 0.862). Of the 125 PwCF with CFTR modulator use, DIOS-related hospital admissions decreased in 15 patients, remained unchanged in 89, and increased in 21 (p = 0.021) while ED visits were fewer in 8, unchanged in 97, and increased in 20 (p = 0.587).ConclusionPwCF had high burden of constipation with a majority of patients regularly using laxatives, and almost half had a history of DIOS. CFTR modulator use and CF-GI clinic were not associated with a decrease of DIOS incidence.

研究背景：胃肠道（Gastrointestinal, GI）并发症是囊性纤维化（cystic fibrosis, CF）患者（persons with cystic fibrosis, PwCF）第二常见的病症。目前关于专属CF-GI门诊与囊性纤维化跨膜传导调节因子（cystic fibrosis transmembrane conductance regulator, CFTR）调节剂如何影响GI并发症发生率的相关数据较为有限。本研究旨在评估CF-GI门诊与CFTR调节剂对胃肠道并发症，尤其是远端肠梗阻综合征（distal intestinal obstructive syndrome, DIOS）发病率的影响。
研究方法：本研究为回顾性研究，纳入2000年至2023年间于专属CF-GI门诊就诊的成年囊性纤维化患者。分别对比了患者使用CFTR调节剂前后3年，以及首次于CF-GI门诊就诊前后3年中，因DIOS入院及急诊科（emergency department, ED）就诊的人次。
研究结果：本研究共纳入1076例确诊囊性纤维化患者，其中242例于CF-GI门诊就诊。在该242例患者中，126例（52.1%）为女性，中位年龄为40岁（四分位距（interquartile range, IQR）：30~47岁）；146例（60.3%）规律服用泻药。在59例因便秘于CF-GI门诊就诊的患者中，16例入院人次减少、32例无变化、11例入院人次增加（p = 0.402）；急诊科就诊人次方面，9例减少、40例无变化、10例增加（p = 0.862）。在125例使用CFTR调节剂的患者中，15例DIOS相关入院人次减少、89例无变化、21例增加（p = 0.021）；急诊科就诊人次方面，8例减少、97例无变化、20例增加（p = 0.587）。
研究结论：囊性纤维化患者便秘负担较重，多数患者规律服用泻药，且近半数患者存在DIOS病史。使用CFTR调节剂与就诊于CF-GI门诊均未显著降低DIOS的发病率。