Nicolas Henao-Romero, Lingxiu Susan Liu, Amirala O M Nazari, Dain Kim, Xiaojie Luan, Kash Desai, Julian S Tam, Juan P Ianowski, Verónica A Campanucci
{"title":"Dysmotility in the ileum of CFTR null swine.","authors":"Nicolas Henao-Romero, Lingxiu Susan Liu, Amirala O M Nazari, Dain Kim, Xiaojie Luan, Kash Desai, Julian S Tam, Juan P Ianowski, Verónica A Campanucci","doi":"10.1016/j.jcf.2025.07.007","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.07.007","url":null,"abstract":"<p><strong>Background: </strong>Gastrointestinal (GI) complications are a common source of morbidity for people with cystic fibrosis (pwCF). The pathobiology of these clinical presentations is not fully understood, but there is evidence that gut dysmotility may be a primary contributor.</p><p><strong>Methods: </strong>We studied gut motility in ileum samples from CF (CFTR-/-) and wild-type (WT) swine at birth (P0) and one week of post-natal life (P7) using organ bath assays.</p><p><strong>Results: </strong>Ileal samples from both WT and CF swine displayed spontaneous peristalsis. CF swine presented with reduced basal amplitude of the peristaltic waves compared to WT swine. Stimulating the ileal samples with increasing concentrations of acetylcholine (ACh) resulted in four main findings: 1) ACh increased the amplitude of smooth muscle contraction in all ileal samples in a dose-dependent manner. 2) At P7, ACh stimulation caused a significant increase in the maximum smooth muscle contraction in the WT but not in the CF samples. 3) Increasing doses of ACh caused fatigue-like contracting decline in smooth muscle from WT samples at both ages, but not in samples from CF swine. 4) ACh stimulation had no effect on the frequency of smooth muscle contraction in either genotype.</p><p><strong>Conclusions: </strong>Our results show ileal dysmotility in the CF swine characterized by a decrease in basal peristalsis and weaker smooth muscle contraction. Our data suggest that GI dysmotility would impact chyme transit through the GI tract, which may predispose pwCF to intestinal manifestations associated with the disease.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144682670","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}
Ieuan Es Evans, Christine M Duplancic, Timothy A Kidd, Emma L Ballard, Rachel M Thomson, Claire E Wainwright, Scott C Bell
{"title":"The changing face of cystic fibrosis research: challenges of multi-centre microbiology cohort studies.","authors":"Ieuan Es Evans, Christine M Duplancic, Timothy A Kidd, Emma L Ballard, Rachel M Thomson, Claire E Wainwright, Scott C Bell","doi":"10.1016/j.jcf.2025.07.002","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.07.002","url":null,"abstract":"<p><strong>Introduction: </strong>Infection is a primary cause of progressive pulmonary disease in people with cystic fibrosis (pwCF). Researchers frequently utilize data registries or centre-based cohort studies to study infections in CF. We examine challenges encountered in conducting two multi-centre non-interventional investigator-led cohort studies undertaken a decade apart.</p><p><strong>Methods: </strong>The Australian Clonal Pseudomonas Study (ACPinCF, 1294 participants) and the National NTM in CF study (1314 participants) leveraged clinical practice, with data was supplied by clinics and the Australian CF Data Registry. Diagnostic laboratories performed cultures according to local protocols.</p><p><strong>Results: </strong>Site approval times were lengthy; 16 months for the ACPinCF study and between 21-44 months for the National NTM in CF study. The latter required individual contract negotiation with each clinical site as a requirement of governance approval. Changing clinical demands and unforeseen challenges such as the SARS-CoV-2 pandemic significantly impacted the NTM study. Despite these challenges, participant retention remained robust, with minimal loss to follow-up. Changes in CF centre leadership and health service policy on cost-recovery for research participation further exacerbates the challenges of maintaining longitudinal cohort CF studies.</p><p><strong>Conclusions: </strong>Conducting and sustaining longitudinal non-interventional studies present significant challenges. Even after costs for centre participation, sample processing, data curation, data analysis and results output are covered by grant funding, these studies will still heavily depend on significant support provided by the centres without substantial funding.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144667724","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}
Seyda Karabulut, Velat Sen, Beste Özsezen, Ali Özdemir, Melih Hangül, Suat Savas, Hadice Selimoğlu Sen, Gaye Inal, Huseyin Arslan, Mahir Serbes, Pelin Asfuroglu, Ezgi Cay, Erdem Topal, Neval Metin Çakar, Almala Pınar Ergenekon, Ela Erdem Eralp, Sedat Oktem, Zeynep Seda Uyan, Fazilet Karakoc, Bulent Karadag, Yasemin Gokdemir
{"title":"After the earthquake: Unmet needs in people with cystic fibrosis in Turkiye- multicenter study.","authors":"Seyda Karabulut, Velat Sen, Beste Özsezen, Ali Özdemir, Melih Hangül, Suat Savas, Hadice Selimoğlu Sen, Gaye Inal, Huseyin Arslan, Mahir Serbes, Pelin Asfuroglu, Ezgi Cay, Erdem Topal, Neval Metin Çakar, Almala Pınar Ergenekon, Ela Erdem Eralp, Sedat Oktem, Zeynep Seda Uyan, Fazilet Karakoc, Bulent Karadag, Yasemin Gokdemir","doi":"10.1016/j.jcf.2025.06.008","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.06.008","url":null,"abstract":"<p><strong>Background: </strong>We aimed to assess unmet needs of pwCF in the earthquake zone by income level.</p><p><strong>Methods: </strong>Following the February 6, 2023 Earthquake in Turkiye, the shorter version of the 'Your Current Life Situation' (YCLS) survey was adapted for post-earthquake conditions. The adapted YCLS was administered through face-to-face interviews at participants in seven earthquake-affected provinces to determine the insecurity areas and unmet needs in pwCF. Parents completed the survey for those pwCF under 18 years old; those over 18 completed it themselves.</p><p><strong>Results: </strong>Among 255 participants, 91.7% (n=234) had incomes below the poverty threshold and 71.8% (n=183) below the hunger threshold. Post-earthquake, 69% (n=176) lived in overcrowded conditions and 37.6% (n=96) relocated to temporary housing. Under these challenging circumstances, 34.5% (n=88) of pwCF experienced disruptions in routine visits, and 20.8% (n=53) reported disruption in daily CF care routine. Financial and food insecurities were prevalent, with 77.3% (n=197) and 53.3% (n=136) of participants affected, respectively. The potential impact of earthquake-induced relocation on the participants' insecurity status was analyzed. Among those who relocated, financial, transportation, and housing insecurity appeared to be more prevalent (p<0.001).</p><p><strong>Conclusion: </strong>This is the first study to analyze association between income level and unmet needs among pwCF living in earthquake-affected zone. The study revealed significant financial and food insecurity among pwCF in these areas. The findings emphasize the need for disaster-specific emergency action plans to address these vulnerabilities, ensuring access to healthcare and basic needs during crises.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144659359","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}
Cian Greaney, Ellen McCarthy, Lauren O'Brien, Sarah Tecklenborg, Ciara Howlett, Karen Cronin, Clodagh Landers, Mary Connolly, Derbhla O'Sullivan, Aoife Whiston, Katie Robinson, Audrey Tierney
{"title":"Dietary intakes and quality of Irish adults with cystic fibrosis: Comparisons to nutrition guidelines and HEI-2020.","authors":"Cian Greaney, Ellen McCarthy, Lauren O'Brien, Sarah Tecklenborg, Ciara Howlett, Karen Cronin, Clodagh Landers, Mary Connolly, Derbhla O'Sullivan, Aoife Whiston, Katie Robinson, Audrey Tierney","doi":"10.1016/j.jcf.2025.07.004","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.07.004","url":null,"abstract":"<p><strong>Background: </strong>With evolving Cystic Fibrosis (CF) phenotypes resulting from changes to clinical management, healthier dietary practices are warranted for many people with CF. Whilst diet composition is reported, diet quality data is lacking in CF. This study aims to evaluate dietary intakes and diet quality in adults with CF via guideline comparison and a validated diet quality index.</p><p><strong>Methods: </strong>Cross-sectional study of Irish adults with CF. Demographic questionnaires and three-day food diaries were completed. Healthy Eating Index - 2020 (HEI-2020) assessed diet quality. Data was statistically analysed in SPSS®.</p><p><strong>Results: </strong>Of n = 68 participants (female: 58.8 %, age: 35.2 ± 10.1 years, FEV<sub>1</sub>%: 77.4 ± 25.1 %), 36.8 % were overweight/obese and 77.6 % pancreatic insufficient. While median (interquartile range) percentage estimated average requirement (EAR) was 110.1 (45.3) %, 50.0 % of participants were below CF energy requirements (110 % EAR). Mean percentage total energy intake (%TEI) protein (18.0 ± 3.9 %) aligned to dietary reference values (DRV). %TEI carbohydrates (44.1 ± 6.5 %) was below, and %TEI fat (37.1 ± 5.4 %), saturated fat (14.1 ± 3.3 %) and sugar (17.4 ± 5.6 %) exceeded DRV. Median vitamin A intake was adequate [936.6 (1005.2) µg], but vitamin D [3.6 (4.3) µg], E (9.6 ± 5.2 mg) and K<sub>1</sub> [31.1 (71.2) µg] intakes were insufficient without supplementation. Regarding Irish healthy eating guidelines, 95.6 % of participants overconsumed energy-dense nutrient poor (EDNP) foods, with 76.5 % below vegetables, salad and fruit intake guidelines. Participants' mean HEI-2020 score (0-100) was 59.3 ± 12.4.</p><p><strong>Conclusion: </strong>Findings indicate suboptimal diet quality. Despite reliance on EDNP foods, many did not achieve energy targets. Moving forward, emphasis on diet quality is of paramount importance to improve overall health in people with CF.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144618132","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}
G Tamer, H M van Santen, Hgm Arets, D Snoek, C K van der Ent, H J van der Kamp
{"title":"Bone mineral density in children and adolescents with cystic fibrosis: a longitudinal study.","authors":"G Tamer, H M van Santen, Hgm Arets, D Snoek, C K van der Ent, H J van der Kamp","doi":"10.1016/j.jcf.2025.06.009","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.06.009","url":null,"abstract":"<p><strong>Background: </strong>Adults with cystic fibrosis (CF) show a higher rate of osteoporosis compared to healthy adults. Achieving proper adult bone mass is a process starting in childhood. We aimed to evaluate the prevalence, course in time and risk factors for decreased bone mineral density (BMD) in youth with CF.</p><p><strong>Methods: </strong>Anthropometry, dual-energy X-ray absorptiometry (DXA), and endocrine data were collected in 106 children and adolescents with CF. BMD Z-scores were adjusted for height (BMD Z-score). At risk and low BMD were defined as Z-scores ≤ -1 and ≤ -2, respectively. Multivariable analyses were performed for change in BMD Z-scores in 65 patients with a second DXA available.</p><p><strong>Results: </strong>At baseline, overall normal median (IQR) BMD Z-scores were found (-0.02 (-0.85 - 0.55) and -0.01 (-0.72 - 0.82) for lumbar spine (LS) and total body less head (TBLH) respectively). In 6.6 % and 2.8 % of patients low BMD Z-scores were found for LS and TBLH. At follow-up, significant decreases in BMD Z-scores at both LS and TBLH were identified (p = 0.003 and p < 0.001). The decrease in TBLH BMD Z-scores was higher in boys than girls (p < 0.001). New onset of CFRD (β = -0.493, p = 0.002) was a negative predictor for the change in TBLH BMD Z-score.</p><p><strong>Conclusion: </strong>Most children and adolescents with CF have normal BMD Z-scores. However a significant decrease in BMD Z-scores over time is already seen during childhood, especially in boys and in those with newly onset CFRD.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144618131","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}
Natalia Smirnova, Scott Gillespie, Jane Lowers, Andrew Jergel, Elisabeth P Dellon, Alexandre Cammarata-Mouchtouris, Anne Fitzpatrick, Dio Kavalieratos
{"title":"Symptom factors and their clinical correlates among adults with cystic fibrosis.","authors":"Natalia Smirnova, Scott Gillespie, Jane Lowers, Andrew Jergel, Elisabeth P Dellon, Alexandre Cammarata-Mouchtouris, Anne Fitzpatrick, Dio Kavalieratos","doi":"10.1016/j.jcf.2025.07.001","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.07.001","url":null,"abstract":"<p><strong>Background: </strong>Despite cystic fibrosis transmembrane conductance (CFTR) modulator therapy that may dramatically alter the course of disease, many people living with cystic fibrosis (PwCF) experience co-occurring symptoms that may be interrelated and may synergistically degrade quality of life. We sought to identify symptom factors, or groups of correlated symptoms, connected by underlying latent variable(s). We then examined demographic and clinical characteristics associated with these groups among PwCF.</p><p><strong>Methods: </strong>Using baseline data from a trial of specialist palliative care in PwCF, we assessed symptoms with the Memorial Symptom Assessment Scale-CF. We used exploratory factor analysis followed by confirmatory factor analysis to identify and validate symptom factors. For each factor (using only the symptoms identified in the factor), we then created a binary variable (Symptom Factor Severity) that identified patients as Low versus High Severity, by mean splitting the sum of each patient's severity symptom scores for that factor. Multivariable logistic regression was used to examine associations between demographic and clinical characteristics with each split Symptom Factor Severity score.</p><p><strong>Results: </strong>Among 262 participants, median age was 33 years, and 78 % were prescribed a CFTR modulator. We identified three symptom factors: respiratory-energy, mood-gastrointestinal irritability, and pain-gastrointestinal dysmotility. High symptom severity in each factor was associated with specific demographic and clinical characteristics.</p><p><strong>Conclusions: </strong>CF symptom management strategies have historically focused on single-symptom approaches. Findings from this study may prompt clinicians to consider co-occurring symptoms, and ensure their assessment and management is tailored to the unique experiences of PwCF.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144591306","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":"Isolated hyperphosphatasemia in adults with cystic fibrosis - a case series.","authors":"Blair Lenhan, Katie Kaput, HollyAnn Jacobs, Laurie LeClair","doi":"10.1016/j.jcf.2025.06.004","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.06.004","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575524","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}
Enrico Costa, Hendrika A van den Ham, Hubert G M Leufkens
{"title":"Challenges and opportunities of adding CFTR modulators for cystic fibrosis to the WHO essential medicines list.","authors":"Enrico Costa, Hendrika A van den Ham, Hubert G M Leufkens","doi":"10.1016/j.jcf.2025.06.006","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.06.006","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567438","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}
Tim Lee, Kate Hill, Daan Caudri, Pierluigi Ciet, Gwyneth Davies, Jane C Davies, Anna-Maria Dittrich, Anders Lindblad, Paul McNally, Philippe Reix, Clare Saunders, Isabelle Sermet-Gaudelus, Mirjam Stahl, Harm A W M Tiddens, Hettie M Janssens
{"title":"Pulmonary endpoints in clinical trials for children with cystic fibrosis under two years of age.","authors":"Tim Lee, Kate Hill, Daan Caudri, Pierluigi Ciet, Gwyneth Davies, Jane C Davies, Anna-Maria Dittrich, Anders Lindblad, Paul McNally, Philippe Reix, Clare Saunders, Isabelle Sermet-Gaudelus, Mirjam Stahl, Harm A W M Tiddens, Hettie M Janssens","doi":"10.1016/j.jcf.2025.06.003","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.06.003","url":null,"abstract":"<p><p>Cystic fibrosis is a lifelong progressive disease in which lung disease is the main prognostic factor, where starting early treatment is crucial for improving long-term outcomes. Therefore, new treatment should be available as early as possible. However, choosing appropriate and feasible clinical trial endpoints in children under 2 years of age presents significant challenges. Most studies in this age group have extrapolated pulmonary efficacy from older age groups, focusing on safety, pharmacokinetics, and biomarker response. As lung health is near normal in infants, demonstrating absence of pulmonary decline requires large sample sizes and extended study duration, which may not be feasible for standard regulatory trials. To address this gap, the European Cystic Fibrosis Society Clinical Trials Network developed a consensus document evaluating direct pulmonary endpoints for therapeutic pulmonary studies in this young age group. The pulmonary endpoints evaluated include multiple-breath washout (MBW); chest computed tomography (CT); chest magnetic resonance imaging (MRI); airway infection and inflammation. Relevant literature, pitfalls, practice guidelines, and recommendations are presented. None of the pulmonary endpoints evaluated are currently suitable to serve as a primary efficacy endpoint in children below 2 years of age, as this will require large numbers and long follow-up. For clinical trials in infants with CF, pharmacokinetics, pharmacodynamics, safety and tolerability should remain the primary endpoints, with pulmonary endpoints as secondary or exploratory outcomes. Post authorization studies are essential to evaluate long-term pulmonary benefits, including MBW, structural lung assessment (e.g. CT and MRI), and markers of pulmonary inflammation to fully understand the impact of early therapy initiation in this young population.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144564820","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}
Nora C Burdis, Miranda C Bradford, Sonya L Heltshe, Tijana Milinic, Oliver J McElvaney, Erika D Lease, Christopher H Goss, Siddhartha G Kapnadak, Brandon L Guthrie, Kathleen J Ramos
{"title":"Elexacaftor/tezacaftor/ivacaftor prescription in lung transplant recipients with cystic fibrosis in the US.","authors":"Nora C Burdis, Miranda C Bradford, Sonya L Heltshe, Tijana Milinic, Oliver J McElvaney, Erika D Lease, Christopher H Goss, Siddhartha G Kapnadak, Brandon L Guthrie, Kathleen J Ramos","doi":"10.1016/j.jcf.2025.06.005","DOIUrl":"https://doi.org/10.1016/j.jcf.2025.06.005","url":null,"abstract":"<p><strong>Background: </strong>Elexacaftor/tezacaftor/ivacaftor (ETI) has dramatically improved pulmonary and extrapulmonary manifestations of cystic fibrosis (CF). ETI clinical trials excluded lung transplant (LTx) recipients and current post-transplant prescribing practices are not evidence-based. We sought to identify the prevalence of new ETI prescriptions among CF LTx recipients in the United States (U.S.) and factors associated with ETI prescription after LTx.</p><p><strong>Methods: </strong>We performed a retrospective cross-sectional study of LTx recipients through December 2022 using the Cystic Fibrosis Foundation Patient Registry. Recipients were alive as of October 2019, had an ETI-eligible genotype, and were not prescribed ETI before LTx. Logistic regression was used to assess factors associated with ETI prescription. CF Center prescribing patterns were categorized based on the proportion of LTx recipients who were prescribed ETI after LTx at each center.</p><p><strong>Results: </strong>Overall, 488/1666 (29.3 %) of patients were prescribed ETI after LTx. The presence of sinus disease (OR 2.12, 95 % CI 1.51-2.99) and BMI<18.5 kg/m<sup>2</sup> (OR 1.52, 95 % CI 1.13-2.04) were positively associated with ETI prescription after LTx. CF center prescribing pattern [\"middle prescribing\": OR 0.19, 95 % CI 0.14-0.26; \"low prescribing\": OR 0.02, 95 % CI 0.01-0.04; \"high prescribing\": reference group] and zero F508del alleles (OR 0.18, 95 % CI 0.07-0.49; 1 or 2 alleles: reference group) were negatively associated with ETI prescription after LTx.</p><p><strong>Conclusions: </strong>ETI was newly prescribed to almost 30 % of eligible LTx recipients in the U.S. Even when including patient clinical characteristics in the model, CF center prescribing pattern was one of the strongest factors associated with ETI prescription after LTx.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144553604","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}