Journal of Cystic Fibrosis最新文献

{"title":"Extrapulmonary benefits and pharmacogenomics in lung transplant recipients with Cystic Fibrosis on Elexacaftor/Tezacaftor/Ivacaftor (ETI) therapy.","authors":"John C Kennedy, Aderajew A Taddesse, Antonio Coppolino, Stella E Lee, Suzanne E Dahlberg, Ryan C Perkins, Rebecca E Cagnina, Adil Sheikh, Manuela Cernadas, Selvin S Jacob, Kathryn Kear, Sarah Kubiak, Yiyang Zhang, Mohammed A Keshk, Amy E Hackmann, Stefi F Lee, Elizabeth Fieg, Asma Algarawi, Ethan K Ito, Benjamin J Kerman, Ann E Woolley, George R Washko, Hilary J Goldberg, Benjamin A Raby, Ahmet Z Uluer","doi":"10.1016/j.jcf.2026.09.007","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.09.007","url":null,"abstract":"<p><p>Elexacaftor/Tezacaftor/Ivacaftor (ETI) and other highly effective modulator therapies have transformed outcomes for people with cystic fibrosis (pwCF), yielding significant pulmonary and extrapulmonary benefits. Although lung transplant (LTx) rates have declined dramatically in the post ETI era, a substantial number of transplant recipients with CF remain who may benefit from the extrapulmonary effects of these therapies. Unfortunately, available post market data regarding ETI safety, efficacy, and tolerability following LTx are limited and conflicting, raising concerns for Drug-Drug Interactions (DDI) and long-term graft function. In this single center retrospective cohort analysis, we report sustained improvements in sinonasal disease and weight maintenance with preserved spirometric values over a mean follow up of 35.1 months on ETI compared to those not on therapy. We additionally present pharmacogenomic testing in a subset of our cohort as proof-of-concept for predicting DDIs at time of initiation of ETI.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149061412","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Heterozygous CFTR variants associated with low-phospholipid-associated cholelithiasis phenotypes expand hepatobiliary disorders spectrum.","authors":"Camille Giannetti, Louis Carmarans, Patrice Bourgeois, René Gerolami, Patrick Borentain, Diane Lorenzo, Solene Hoibian, Alexandre Fabre, Catherine Badens, Christophe Buffat","doi":"10.1016/j.jcf.2026.09.009","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.09.009","url":null,"abstract":"<p><p>Low-phospholipid-associated cholelithiasis (LPAC) syndrome is mainly associated with ABCB4 variants, although up to half of cases remain genetically unexplained. Given the role of CFTR in biliary homeostasis and its involvement in CFTR-related pancreatobiliary disorders, we investigated whether CFTR variants may contribute to LPAC phenotypes. We conducted a monocentric study including 34 patients fulfilling LPAC diagnostic criteria who underwent whole-exome sequencing with analysis restricted to an extended hepatobiliary gene panel including CFTR. Variants of interest were identified in 14/34 patients (41%), including 4 ABCB4 variant carriers (12%) and 10 CFTR variant carriers (29%). Patients carrying CFTR variants presented with classical LPAC features, including cholelithiasis, recurrent biliary symptoms, and cholangitis, while pancreatic involvement was observed in half of carriers. Compared with the CFTR carrier frequency estimated from the French newborn screening program (∼2.6%), CFTR variant carriers were significantly enriched in the cohort (11.5-fold enrichment, 95% CI 5.9-18.6, p < 0.001), suggesting a contributory role in a subset of LPAC phenotypes. These findings support consideration of CFTR in the genetic evaluation of selected patients with LPAC and reinforce the concept of hepatobiliary transporter disorders as a phenotypic continuum extending beyond ABCB4 deficiency.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149061428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neuropsychiatric adverse effects following switch to vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: early experience from a regional centre.","authors":"A P Sawant, N Shaw, I J Clifton, H Saumtally, D G Peckham","doi":"10.1016/j.jcf.2026.09.008","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.09.008","url":null,"abstract":"<p><p>Vanzacaftor-tezacaftor-deutivacaftor (VTD) is the newest triple-combination CFTR modulator, licensed as an alternative to elexacaftor-tezacaftor-ivacaftor (ETI). Neuropsychiatric adverse effects are recognised with ETI and now carry a specific post-marketing warning for VTD. At our regional adult cystic fibrosis centre (454 patients under follow-up), 87 have been commenced on VTD, mainly for elevated sweat chloride on ETI and dosing preference. Three individuals with pre-existing mental health conditions were proactively started on a reduced dose (one tablet daily); two of three showed sweat chloride improvement below 40 mmol/L, with no reported adverse effects. Among the remaining patients, four (4.6%) developed new-onset or worsening psychiatric symptoms at standard dose. Three had no prior psychiatric history; one had pre-existing depression on long-term sertraline that markedly worsened. Onset ranged from two weeks to four months. Three of four showed a good physiological response, with further sweat chloride reduction and stable or improved lung function; the fourth switched back too rapidly for retesting. Three patients required switching back to ETI, with resolution of symptoms within days; one continued VTD with resolution by three months. This rate is comparable to previously reported real-world ETI rates (7% and 7.9%), though more of our cases involved suicidal ideation and required full discontinuation. Cases were identified clinically rather than through systematic screening, which may affect detection rates. These findings highlight the need for early mental health screening after VTD initiation and a low threshold for psychiatric review. All cases have been reported to the MHRA Yellow Card scheme.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149008643","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Epigenetic regulation and chromatin organization in cystic fibrosis airways.","authors":"Agustín De Ganzó, Brian Chan, Michael Rubinstein, Kenichi Okuda, Carla M P Ribeiro","doi":"10.1016/j.jcf.2026.08.013","DOIUrl":"10.1016/j.jcf.2026.08.013","url":null,"abstract":"<p><p>Cystic fibrosis (CF) is caused by pathogenic variants of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CF in the airways is associated with dehydrated and hyperconcentrated mucus, impaired mucociliary transport, and inflammation. Although CF is a genetic disorder, CFTR sequence variations alone may not fully explain its phenotypic variation. Additionally, while pharmacological CFTR modulators that correct CFTR protein function greatly improve clinical outcomes, people with CF treated with modulators can still exhibit abnormal mucus and persistent inflammation compared to non-CF individuals. As such, understanding the regulation of mucus and inflammation remains crucial to full characterization and management of CF. We suggest that non-DNA-sequence-based mechanisms that regulate CFTR, mucus, and inflammation contribute to CF pathogenesis. This review highlights three features of transcriptional regulation known to modify gene expression (DNA methylation, histone modifications, and three-dimensional chromatin organization) and their role in CF airways. For each feature, we discuss current knowledge of their role in regulating CFTR, gel-forming mucin genes, and inflammation in CF contexts. We also highlight exploratory efforts to target epigenetic mechanisms as CF treatment strategies and suggest that epigenetics and chromatin organization offer a rich space for future CF research.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13578995/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148976955","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing the impact of elexacaftor/tezacaftor/ivacaftor on work productivity and activity impairment in people living with cystic fibrosis in Canada.","authors":"Grace G Kim, Taryn Wong, Jonathan Rayment, Lara Bilodeau, Elizabeth Tullis, Larry C Lands, Winnie M Leung, Grace Y Lam, Felix Ratjen, Bradley S Quon","doi":"10.1016/j.jcf.2026.09.005","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.09.005","url":null,"abstract":"<p><strong>Background: </strong>Elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with substantial improvements in lung function and quality of life in people with cystic fibrosis (pwCF). Its longer-term impact on work productivity and daily activity remains less well characterized.</p><p><strong>Methods: </strong>We analyzed data from the CAN-IMPACT-CF and REAL-ETI-CF observational studies. Work productivity and activity impairment (WPAI) outcomes were compared at baseline and after 1 year of ETI using paired t-tests. Absenteeism represents % of work missed and presenteeism represents % impairment at work. Associations between changes in WPAI outcomes and domains of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) were assessed using Spearman's correlation.</p><p><strong>Results: </strong>This analysis included 146 pwCF. Among working participants (n = 75), absenteeism decreased from 13.1% to 3.3% (p = 0.006), presenteeism from 24.9% to 8.8% (p = 0.0002), and overall work productivity loss from 30% to 10.8% (p = 0.0002) following 1 year of ETI. Reductions in productivity loss were most strongly correlated with relative improvements in CFQ-R Role Functioning (ρ=-0.66), Physical Functioning (ρ=-0.63), and Respiratory (ρ=-0.59) domains (all p = 0.0003). Overall activity impairment decreased from 30.1% to 15.1% (p = 0.0002) and similarly was most strongly correlated with improvements in CFQ-R Role Functioning (ρ=-0.53), Physical Functioning (ρ=-0.57), and Respiratory (ρ=-0.54) domains (all p = 0.0003).</p><p><strong>Conclusions: </strong>In this real-world study, ETI was associated with significant improvements in work productivity and activity impairment. These functional gains closely mirror improvements in physical, role, and respiratory domains, highlighting the broader socioeconomic and quality-of-life benefits of ETI beyond traditional clinical endpoints.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148968796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CFTR modulation alter pancreatic cancer cell growth and signaling: implications for cancer risk in cystic fibrosis.","authors":"Yunxia O'Malley, Henry Keen, Ian M Thornell, Zachary Campbell, Anna J Prouty, Carlos H F Chan, Andrew W Norris, Prabhat Goswami, Aliye Uc","doi":"10.1016/j.jcf.2026.08.012","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.08.012","url":null,"abstract":"<p><strong>Background: </strong>People with cystic fibrosis (PwCF) have an increased risk of pancreatic cancer, likely related to chronic pancreatic inflammation and CFTR dysfunction. With the widespread use of CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI), understanding their impact on pancreatic ductal cell biology is critical.</p><p><strong>Methods: </strong>We studied human pancreatic ductal adenocarcinoma cell lines (PANC-1, CFPAC-1) and primary pig pancreatic duct epithelial cells with wild-type, F508del, or null CFTR. CFTR function was restored using adenoviral transfection (Ad5CMVCFTR-GFP) or ETI treatment. Proliferation was monitored longitudinally; cell cycle kinetics and active DNA synthesis were analyzed via Brdu/propidium iodide flow cytometry; cellular motility was assessed via scratch wound healing assays; apoptosis was measured by flow cytometry; CFTR activity was evaluated using halide-sensitive YFP; and gene expression was assessed by RT-qPCR and RNA sequencing. Human pancreas cancer CFTR expression was evaluated using public datasets.</p><p><strong>Results: </strong>CFTR expression was reduced in pancreas cancer tissues, cell lines, and CF pancreatic duct epithelial cells compared to controls. CFTR-deficient cells exhibited increased baseline proliferation and migration. ETI treatment dynamically downshifted cell cycle distribution kinetics, without affecting apoptosis. Transcriptomic analysis revealed ETI downregulated key cell cycle regulators (CCNDs, CDKs, MCMs) and suppressed Wnt pathway activators while upregulating inhibitors, consistent with growth restriction.</p><p><strong>Conclusions: </strong>CFTR restoration not only improves channel function but also reprograms transcriptional networks to act as a synchronized brake on epithelial replication velocity and migration. These findings suggest that CFTR-targeting therapies may fundamentally influence pancreatic cancer biology and have implications for cancer risk management in PwCF.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148955736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A functional comparison of readthrough agent ELX-02 across a wide range of nonsense CFTR variants.","authors":"Marlies Destoop, Anabela S Ramalho, Iris A L Silva, Annelotte M Vonk, Sylvia Suen, Marlou C Bierlaagh, Sylvia F Boj, Robert G J Vries, Jeffrey M Beekman, Kris De Boeck, Cornelis K van der Ent, Margarida D Amaral, Francois Vermeulen, Sacha Spelier","doi":"10.1016/j.jcf.2026.09.004","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.09.004","url":null,"abstract":"<p><strong>Background: </strong>Nonsense variants in CFTR account for ∼10% of cystic fibrosis (CF) variants and cannot be treated with available CFTR modulators. Translational readthrough agents such as ELX-02 offer a potential therapeutic strategy for CFTR nonsense variants, but clinical trials, conducted primarily in individuals carrying the G542X variant, have demonstrated limited efficacy. Our study aimed to evaluate ELX-02-mediated CFTR rescue across a broad range of nonsense variants using patient-derived intestinal organoids (PDIOs) to define variant-specific determinants of readthrough efficacy and assess its potential across a genetically diverse CF population.</p><p><strong>Method: </strong>The ex vivo response to ELX-02 was assessed in 206 PDIOs carrying heterogeneous nonsense variants. CFTR function was quantified using the forskolin-induced swelling (FIS) assay after 48-hour exposure to ELX-02. Responses were analysed by genotype and stop codon identity, with secondary validation performed in a selected subset of PDIOs (n = 60).</p><p><strong>Results: </strong>ELX-02-mediated CFTR rescue varied markedly, ranging from responses approaching those observed with approved CFTR modulators (LUM/IVA) to responses at or below detection limit. Overall, maximal responses were modest and at the lower end of the functional range for CFTR modulators. Rescue was dose-dependent and higher in PDIOs carrying two nonsense variants when compared with PDIOs carrying a single nonsense variant combined with a residual or minimal function variant. Nonsense variants in nucleotide-binding domain 1, including G542X, S466X, G550X and R553X, showed relatively higher responsiveness.</p><p><strong>Conclusion: </strong>ELX-02 induces limited and highly heterogeneous CFTR rescue across nonsense variants. PDIO-based functional screening provides a framework to guide patient selection and stratification for future readthrough therapy trials.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148944857","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"In Vitro Responsiveness of 417 CFTR Variants to Vanzacaftor/Tezacaftor/Ivacaftor.","authors":"Hermann Bihler, Andrey Sivachenko, Priyanka Bhatt, Ashley Baldwin, Jan Harrington, Violaine Bailey, Alexi Panagiotou, Divesh Patel, Michael J Mulligan, Rachel Valdez Misiolek, Kylie Arnold, Yi Cheng, Kevin Coote, Normand E Allaire, Joshua Conte, Karen S Raraigh, Garry R Cutting, Martin Mense","doi":"10.1016/j.jcf.2026.08.010","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.08.010","url":null,"abstract":"<p><strong>Background: </strong>This study sought to identify additional rare CFTR variants that show improved chloride (Cl⁻) transport in response to the modulator combination vanzacaftor/tezacaftor/ivacaftor (VNZ/TEZ/IVA; VTI). It builds on prior work evaluating 655 variants treated with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA; ETI). The findings may inform people with cystic fibrosis (pwCF) and caregivers pursuing off-label access to modulator therapies when genotype-based eligibility is limited.</p><p><strong>Methods: </strong>Fischer Rat Thyroid (FRT) cells, which do not express endogenous CFTR, were transfected with plasmids encoding wildtype (WT) CFTR or specific CFTR variants. After 24 h under control conditions or treatment with VNZ and TEZ, cells received an acute addition of IVA. CFTR activity was quantified using transepithelial current clamp conductance assays. For each variant, we measured basal forskolin (Fsk)/cAMP-stimulated Cl⁻ transport, potentiation by IVA, and responsiveness to the full VTI combination. CFTR maturation was assessed via Western blot.</p><p><strong>Results and conclusions: </strong>We evaluated 417 CFTR variants for VTI responsiveness, including 272 tested for the first time and 145 previously categorized as non-responsive (NR) or borderline-responsive (BR) to ETI. Overall, ∼76% of variants achieved ≥10% of WT improvement, the threshold for responsiveness. This included 87% of newly tested variants and 55% of ETI NR/BR variants. Notably, 50 variants classified as NR/BR to ETI reached the responsive threshold with VTI using empirical Bayes criteria. Compared with ETI, VTI demonstrated broader efficacy across this dataset. These results expand the number of CFTR variants likely to benefit from highly effective modulator therapy and highlight remaining variants that may require alternative therapeutic strategies.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148944863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reducing multiple-breath washout test burden in cystic fibrosis: agreement between the first acceptable trial and mean lung clearance index.","authors":"Natalia S Escobar, Elad Ben-Meir, Renee Jensen, Scott Sagel, Nicole Hamblett, Michelle Shaw, Felix Ratjen","doi":"10.1016/j.jcf.2026.09.002","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.09.002","url":null,"abstract":"<p><strong>Background: </strong>Lung clearance index (LCI) derived from multiple-breath washout (MBW) is a sensitive marker of early lung disease in cystic fibrosis (CF), but testing is limited by the need for repeated technically acceptable trials. Whether a single technically acceptable trial provides information comparable to conventional mean-based reporting remains uncertain.</p><p><strong>Methods: </strong>We performed a retrospective pooled analysis of MBW test occasions from three multicenter cohorts (TRACK, SIMPLIFY, and PROSPECT), including children and adults with CF and healthy controls. Agreement between the first technically acceptable LCI and mean LCI from two and three acceptable trials was assessed using intraclass correlation coefficients and Bland-Altman analysis. Concordance relative to age-dependent upper limits of normal was also evaluated.</p><p><strong>Results: </strong>A total of 1960 MBW test occasions from 466 participants were included. Allowing a single technically acceptable trial increased overall test acceptability by 3.6%, recovering 78 additional test occasions. Agreement between the first technically acceptable trial and mean LCI from two trials was high (ICC 0.992; mean bias -0.03 LCI units; limits of agreement -0.67 to 0.60). Similar agreement was observed versus the mean of three trials (ICC 0.989; bias -0.06; limits of agreement -0.80 to 0.68). Findings were consistent across cohorts, age groups, and clinical state.</p><p><strong>Conclusions: </strong>Across three independent cohorts, the first technically acceptable MBW trial showed close agreement with mean-based LCI reporting. A single-trial approach may reduce MBW test burden while preserving clinical interpretability in selected contexts.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148944875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association between the childhood opportunity index and healthcare utilization in cystic fibrosis.","authors":"Ashley Han, Matt Hall, Elizabeth Cromwell, Ronald L Gibson, Thida Ong, A Jay Freeman, Clement L Ren, Don B Sanders, Jonathan D Cogen","doi":"10.1016/j.jcf.2026.08.009","DOIUrl":"https://doi.org/10.1016/j.jcf.2026.08.009","url":null,"abstract":"<p><strong>Background: </strong>Among people with cystic fibrosis (CF), limited data exists describing how area-level socioeconomic factors influence in-hospital, emergency room, and ambulatory surgery healthcare utilization. Using the Childhood Opportunity Index (COI) as an area-level composite measure, we aimed to describe the healthcare utilization patterns among children with CF (CwCF) by COI and determine if COI is associated with increased healthcare utilization.</p><p><strong>Methods: </strong>Retrospective cohort study that utilized the CF Foundation Patient Registry-Pediatric Health Information System dataset. The COI version 3.0 was used. Primary study outcomes included the number of hospital encounters (hospitalizations, emergency room visits, and ambulatory surgeries) between 2009-2022, compared among five COI quintiles. Generalized estimating equations were used to regress hospital encounter on COI quintile with a Poisson distribution and clustered on hospital.</p><p><strong>Results: </strong>8177 CwCF <22 years contributed 65,038 hospital encounters for analysis. Compared to the very high quintile, CwCF in the very low, low, and moderate quintiles had a higher hospitalization risk (very low Relative Risk (RR) 1.31, 95% Confidence Interval (CI) 1.17-1.47; p < 0.001, low RR 1.16, 95%CI 1.04-1.30; p = 0.008, and moderate RR 1.15, 95%CI 1.05-1.25; p = 0.001). In addition, CwCF in the very low group had a higher respiratory-related hospitalization risk (RR 1.60, 95%CI 1.30-1.90; p < 0.01).</p><p><strong>Conclusions: </strong>CwCF in the lower COI quintiles had a higher hospitalization risk. Future research should focus on underlying area-level drivers of hospitalizations to reduce inpatient utilization.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.5,"publicationDate":"2026-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148891879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}