Journal of Cystic Fibrosis最新文献

{"title":"Feasibility of multiple-breath washout in the clinical setting and prediction of its duration in children and adults with cystic fibrosis","authors":"Christine Allomba ,&nbsp;Leonie Busack ,&nbsp;Niklas Ziegahn ,&nbsp;Charlotte O. Pioch ,&nbsp;Alexandra N. Schnorr ,&nbsp;Bent R. Fuhlrott ,&nbsp;Eva Steinke ,&nbsp;Marcus A. Mall ,&nbsp;Mirjam Stahl","doi":"10.1016/j.jcf.2025.08.005","DOIUrl":"10.1016/j.jcf.2025.08.005","url":null,"abstract":"<div><h3>Background</h3><div>Pulmonary function tests play an important role in diagnosis, management and treatment of people with cystic fibrosis (pwCF). Multiple-breath washout (MBW) is a sensitive method to detect ventilation inhomogeneities and is established in children with CF. Despite promising data in adults with pulmonary diseases, its feasibility remains questioned in adults due to longer examination times and limited added value over spirometry. We aimed to analyse feasibility of MBW in pwCF and healthy controls (HC) and evaluate factors influencing test duration.</div></div><div><h3>Methods</h3><div>We analysed 1,395 MBW measurements from pwCF (n = 1,158) and HC (n = 237). PwCF were divided according to percent predicted forced expiratory volume in one second (ppFEV₁; high, &gt;90 %; mid, 40-90 %; low, &lt;40 %) and age groups. Test feasibility was defined as two acceptable trials. The impact of constitutional and disease severity factors was analysed.</div></div><div><h3>Results</h3><div>Test feasibility was 100.0 % in HC and 94.3 % in pwCF, with higher feasibility in children than adults (96.0 % vs. 92.5 %, p = 0.01), and in those pwCF with higher ppFEV₁ (high, 96.6 %; mid, 94.9 %; low, 82.6 %; p &lt; 0.001). The higher the ppFEV₁, the larger is the influence of constitutional factors over the impact of disease severity on trial time. Mean single trial time was 2.0 min, 2.7 min respectively 5.2 min in high, mid and low ppFEV₁.</div></div><div><h3>Conclusion</h3><div>This study suggests extending MBW to adults with CF. Despite concerns about time efficiency, this study shows a good feasibility of MBW (92.5 %) in adults. Based on correlating factors, this study provides a decision-making tool for clinicians forecasting trial time.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 982-990"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144955686","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":"Lung clearance index in patients with cystic fibrosis: Can we avoid repeating the test three times?","authors":"Amany F. Elbehairy ,&nbsp;Doone Y. Boorman ,&nbsp;Cassandra MacNaughton ,&nbsp;Andrew M. Jones ,&nbsp;Anirban Maitra ,&nbsp;Francis Gilchrist ,&nbsp;Alex Horsley","doi":"10.1016/j.jcf.2025.07.016","DOIUrl":"10.1016/j.jcf.2025.07.016","url":null,"abstract":"<div><div>Multiple breath washout (MBW) testing and its derived measurements (e.g., lung clearance index (LCI)) are useful for measuring ventilation heterogeneity in patients with cystic fibrosis, monitoring disease progression, and assessing the response to treatments. However, the use of MBW in routine clinical practice is limited by the long washout time and total test duration, especially in patients with more advanced disease. Recommendations are to complete three MBW repeats. In this analysis, we examine whether two acceptable repeats would provide an accurate assessment of the LCI and could therefore avoid the need for a third measurement, thus reducing total test time.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 979-981"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144768700","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":"Evidence for altered immune-structural cell crosstalk in cystic fibrosis revealed by single cell transcriptomics","authors":"Marijn Berg ,&nbsp;Lisette Krabbendam ,&nbsp;Esmee K. van der Ploeg ,&nbsp;Menno van Nimwegen ,&nbsp;Tjeerd van der Veer ,&nbsp;Martin Banchero ,&nbsp;Orestes A. Carpaij ,&nbsp;Remco Hoogenboezem ,&nbsp;Maarten van den Berge ,&nbsp;Eric Bindels ,&nbsp;Joachim G.J.V. Aerts ,&nbsp;Antoine Collin ,&nbsp;Pascal Barbry ,&nbsp;Lieke S. Kamphuis ,&nbsp;Rudi W. Hendriks ,&nbsp;Martijn C. Nawijn ,&nbsp;Ralph Stadhouders","doi":"10.1016/j.jcf.2025.01.016","DOIUrl":"10.1016/j.jcf.2025.01.016","url":null,"abstract":"<div><h3>Background</h3><div>Chronic pulmonary inflammation strongly contributes to respiratory failure and mortality in patients with cystic fibrosis (pwCF). Effective anti-microbial immunity and maintaining lung homeostasis require continuous structural-immune cell communication. Whether and how this crosstalk is altered in CF remains poorly understood, obscuring potential new angles for therapy development to restore airway homeostasis in pwCF.</div></div><div><h3>Methods</h3><div>We performed droplet-based single cell RNA-sequencing on bronchial biopsies from pwCF to investigate structural-immune cell crosstalk. Computational analyses were used to compare these data to samples obtained from healthy controls.</div></div><div><h3>Results</h3><div>CF airway wall biopsies showed lower proportions and altered transcriptomes of basal cells, submucosal gland cells and endothelial cells, and a higher abundance of ciliated cells, monocytes, macrophages and T cells. Both B and T lymphocytes displayed aberrantly activated phenotypes with transcriptional changes linked to hypoxia and vascular endothelial growth factor signaling, indicative of crosstalk with endothelial cells. The CF lung displayed unique changes in intercellular communication potential involving ionocytes, macrophages, endothelial cells and lymphocytes. This included interactions between HLA-E on structural cells and the druggable CD94/NKG2A immune checkpoint on CD8⁺ <em>T</em> cells.</div></div><div><h3>Conclusions</h3><div>We report the first single cell transcriptome atlas of the CF lung containing the full spectrum of structural and immune cells, providing a valuable resource for investigating changes to cellular composition, phenotypes and crosstalk linked to CF. Our analyses highlight dysregulated basal cell function and adaptive immunity in pwCF – despite favorable responses to CFTR modulator therapy. We identify novel aspects of CF pathophysiology and potential entry points for therapeutic strategies.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 849-860"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143414369","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":"Trends in Medicare versus Medicaid spending on CFTR modulator therapy – an economic evaluation","authors":"Wanjae Cho ,&nbsp;Michael D. Parkins ,&nbsp;Christina S. Thornton ,&nbsp;Stephen E. Congly","doi":"10.1016/j.jcf.2025.04.008","DOIUrl":"10.1016/j.jcf.2025.04.008","url":null,"abstract":"<div><div>The introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has changed the landscape of therapy for persons with cystic fibrosis. However, the steep cost of targeted therapy poses significant financial burden for individuals and health systems. We aimed to determine the trends in Medicare and Medicaid spending on CFTR modulators between the years 2015 and 2022 through retrospective analysis of the Medicare and Medicaid claims data. The outcome measures included total dosage units prescribed, number of claims, spending per claim, and total spending on CFTR modulators for Medicare and Medicaid between 2015 to 2022. Average annual percentage changes (AAPC) were calculated for all outcome measures. Our results show that from 2015 to 2022, Medicaid consistently had higher total dosage units prescribed, number of claims, spending per claim, and overall spending on CFTR modulators compared to Medicare. Total spending for both Medicaid [AAPC 38.9, 95 % confidence interval [CI] 27.2–51.6, <em>p</em> &lt; 0.01] and Medicare [AAPC 39.2, 95 % CI 30.2–55.1, <em>p</em> &lt; 0.01] increased significantly during this period. Increases in CFTR spending was accelerated beginning in 2019, when the triple combination CFTR modulator therapy, elexacaftor/tezacaftor/ivacaftor, was introduced to the US market. This increase has been accompanied by a reduction in spending and use of other CFTR agents. This study evaluated the increases in spending for CFTR modulators over the years and the main drivers behind them, which may help inform future negotiations between healthcare systems and pharmaceutical companies, as well as policy makers and stakeholders.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 916-919"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144023109","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":"Be it resolved airway clearance cannot and should not be replaced by exercise in the era of CFTR modulators–Summary of a Pro/Con debate","authors":"G. Stanford ,&nbsp;K. von Berg ,&nbsp;C. Smith ,&nbsp;M. Richmond ,&nbsp;J. Walzel ,&nbsp;L. Morrison","doi":"10.1016/j.jcf.2025.05.001","DOIUrl":"10.1016/j.jcf.2025.05.001","url":null,"abstract":"<div><div><span>Airway clearance to clear excessive sputum has long been a key part of cystic fibrosis care, however the introduction of highly effective modulator medications where many people with CF are experiencing reduced sputum loads, has raised a question about its necessity. Specifically, questions are being asked as to if exercise, historically an adjunct to </span>airway clearance, could become a replacement. This short communication summarizes a debate that was held at the 2024 North American Cystic Fibrosis Conference, summarizing the key arguments for and against the replacement of traditional airway clearance with exercise.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 954-956"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093870","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":"Shallow metagenomic shotgun sequencing improves detection of pathogenic species in cystic fibrosis respiratory samples","authors":"Eline Cauwenberghs ,&nbsp;Ilke De Boeck ,&nbsp;Lize Delanghe ,&nbsp;Tim Van Rillaer ,&nbsp;Thomas Demuyser ,&nbsp;Irina Spacova ,&nbsp;Stijn Verhulst ,&nbsp;Kim Van Hoorenbeeck ,&nbsp;Sarah Lebeer","doi":"10.1016/j.jcf.2025.07.011","DOIUrl":"10.1016/j.jcf.2025.07.011","url":null,"abstract":"<div><h3>Background</h3><div>Chronic infection and inflammation of the lungs contribute significantly to disease progression in persons with cystic fibrosis (pwCF). Treatment regimens are largely based on isolating the putative causative pathogen(s) from respiratory samples using basic culturing methods. While this strategy has shown to be highly valuable in the management of CF, the approach is time-consuming and often misses detection of pathogenic microbes that are more difficult to culture, including <em>Mycobacterium</em> spp.</div></div><div><h3>Methods</h3><div>In our proof-of-concept study, we evaluated shallow metagenomic shotgun sequencing to detect potential infection-causing pathogens at species level in sputum, oropharyngeal and salivary samples of pwCF (<em>n</em> = 13), and compared it to culture results from the clinic and standard 16S rRNA V4 amplicon sequencing.</div></div><div><h3>Results</h3><div>Shallow shotgun sequencing improved the detection of pathogenic species in respiratory samples compared to culture methods. In particular, shallow shotgun sequencing could detect pathogenic species associated with CF, specifically <em>Staphylococcus aureus, Pseudomonas aeruginosa, Stenotrophomonas maltophilia, Achromobacter xylosoxidans, Haemophilus influenzae</em> and <em>Mycobacterium</em> spp. in sputum, oropharyngeal and/or salivary samples. Notably, <em>Mycobacterium</em> spp. was not detected based on 16S rRNA amplicon sequencing. Moreover, our approach was able to distinguish <em>S. aureus</em> from <em>S. epidermidis</em> and <em>H. influenzae</em> from <em>H. parainfluenzae</em>. This is not possible with 16S amplicon sequencing, but highly valuable in a clinical setting.</div></div><div><h3>Conclusions</h3><div>The improved detection of CF pathogens and other critical microbiome members as well as insights into their relative abundance within the community, could provide more knowledge on patient’s disease status leading to more personalized medicine and ultimately benefit patient care.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 909-915"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144753508","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":"“Mind the gap”- will we ever see equal median predicted survival for males and females in cystic fibrosis?","authors":"Jamie Duckers,&nbsp;Sarah L. Clarke,&nbsp;Susan C. Charman,&nbsp;Siobhan Carr,&nbsp;Nicholas J. Simmonds,&nbsp;Raksha Jain","doi":"10.1016/j.jcf.2025.04.001","DOIUrl":"10.1016/j.jcf.2025.04.001","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 823-824"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143968896","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":"Elexacaftor/tezacaftor/ivacaftor prescription in lung transplant recipients with cystic fibrosis in the US","authors":"Nora C. Burdis ,&nbsp;Miranda C. Bradford ,&nbsp;Sonya L. Heltshe ,&nbsp;Tijana Milinic ,&nbsp;Oliver J. McElvaney ,&nbsp;Erika D. Lease ,&nbsp;Christopher H. Goss ,&nbsp;Siddhartha G. Kapnadak ,&nbsp;Brandon L. Guthrie ,&nbsp;Kathleen J. Ramos","doi":"10.1016/j.jcf.2025.06.005","DOIUrl":"10.1016/j.jcf.2025.06.005","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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&lt;18.5 kg/m² (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 <em>F508del</em> alleles (OR 0.18, 95 % CI 0.07–0.49; 1 or 2 alleles: reference group) were negatively associated with ETI prescription after LTx.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 929-934"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","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}

{"title":"After the earthquake: Unmet needs in people with cystic fibrosis in Turkiye- multicenter study","authors":"Seyda Karabulut ,&nbsp;Velat Sen ,&nbsp;Beste Özsezen ,&nbsp;Ali Özdemir ,&nbsp;Melih Hangül ,&nbsp;Suat Savas ,&nbsp;Hadice Selimoğlu Sen ,&nbsp;Gaye Inal ,&nbsp;Huseyin Arslan ,&nbsp;Mahir Serbes ,&nbsp;Pelin Asfuroglu ,&nbsp;Ezgi Cay ,&nbsp;Erdem Topal ,&nbsp;Neval Metin Çakar ,&nbsp;Almala Pınar Ergenekon ,&nbsp;Ela Erdem Eralp ,&nbsp;Sedat Oktem ,&nbsp;Zeynep Seda Uyan ,&nbsp;Fazilet Karakoc ,&nbsp;Bulent Karadag ,&nbsp;Yasemin Gokdemir","doi":"10.1016/j.jcf.2025.06.008","DOIUrl":"10.1016/j.jcf.2025.06.008","url":null,"abstract":"<div><h3>Background</h3><div>We aimed to assess unmet needs of pwCF in the earthquake zone by income level.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>Among 255 participants, 91.7% (<em>n</em>=234) had incomes below the poverty threshold and 71.8% (<em>n</em>=183) below the hunger threshold. Post-earthquake, 69% (<em>n</em>=176) lived in overcrowded conditions and 37.6% (<em>n</em>=96) relocated to temporary housing. Under these challenging circumstances, 34.5% (<em>n</em>=88) of pwCF experienced disruptions in routine visits, and 20.8% (<em>n</em>=53) reported disruption in daily CF care routine. Financial and food insecurities were prevalent, with 77.3% (<em>n</em>=197) and 53.3% (<em>n</em>=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 (<em>p</em>&lt;0.001).</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 830-835"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","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}

{"title":"Validation of an artificial intelligence-based automated PRAGMA and mucus plugging algorithm in pediatric cystic fibrosis","authors":"Pranali Raut ,&nbsp;Yuxin Chen ,&nbsp;Ahmad Taleb ,&nbsp;Merlijn Bonte ,&nbsp;Eleni Rosalina Andrinopoulou ,&nbsp;Pierluigi Ciet ,&nbsp;Jean-Paul Charbonnier ,&nbsp;Claire E. Wainwright ,&nbsp;Harm Tiddens ,&nbsp;Daan Caudri","doi":"10.1016/j.jcf.2025.08.003","DOIUrl":"10.1016/j.jcf.2025.08.003","url":null,"abstract":"<div><h3>Background</h3><div>PRAGMA-CF is a clinically validated visual chest CT scoring method, quantifying relevant components of structural airway damage in CF. We aimed to validate a newly developed AI-based automated PRAGMA-AI and Mucus Plugging algorithm using the visual PRAGMA-CF as reference.</div></div><div><h3>Material and Methods</h3><div>The study included 363 retrospective chest CT’s of 178 CF patients (100 New-Zealand and Australian, 78 Dutch) with at least one inspiratory CT matching the image selection criteria. Eligible CT scans were analyzed using visual PRAGMA-CF, automated PRAGMA-AI and Mucus Plugging algorithm. Outcomes were compared using descriptive statistics, correlation, intra- and interclass correlation and Bland-Altman plots. Sensitivity analyses evaluated the impact of disease severity, study cohort, number of slices and convolution kernel (soft vs. hard).</div></div><div><h3>Results</h3><div>The algorithm successfully analyzed 353 (97 %) CT scans. A strong correlation between the methods was found for %bronchiectasis ( %BE) and %disease ( %DIS), but weak for %Airway wall thickening ( %AWT). The automated Mucus plugging outcomes showed strong correlation with visual %mucus plugging ( %MP). ICC’s between visual and automated sub-scores witnessed average agreement for %BE and %DIS, except for %AWT which was weak. Sensitivity analyses revealed that convolution kernel did not affect the correlation between visual and automated outcomes, but harder kernels yielded lower disease scores, especially for %BE and %AWT.</div></div><div><h3>Conclusion</h3><div>Our results show that AI-derived outcomes are not identical to visual PRAGMA-CF scores in size, but strongly correlated on measures of bronchiectasis, bronchial-disease and mucus plugging. They could therefore be a promising alternative for time-consuming visual scoring, especially in larger studies.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 5","pages":"Pages 970-978"},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144955845","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}