Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators

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dc.authoridCakir, Erkan/0000-0002-1438-7854
dc.authoridNayir Buyuksahin, Halime/0000-0002-6909-7993
dc.authoridArik, Elif/0000-0002-7319-8535
dc.authoridSerbes, Mahir/0000-0001-6422-2639
dc.authoridKartal Ozturk, Gokcen/0000-0002-0793-9710
dc.authoridkarcioglu, oguz/0000-0002-6071-0851
dc.contributor.authorBuyuksahin, Halime Nayir
dc.contributor.authorEmiralioglu, Nagehan
dc.contributor.authorYalcin, Ebru
dc.contributor.authorSen, Velat
dc.contributor.authorSen, Hadice Selimoglu
dc.contributor.authorArslan, Huseyin
dc.contributor.authorBaskan, Azer Kilic
dc.date.accessioned2025-02-22T14:09:09Z
dc.date.available2025-02-22T14:09:09Z
dc.date.issued2024
dc.departmentDicle Üniversitesien_US
dc.description.abstractIntroduction: Cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs target the underlying defect and improve CFTR function. They are a part of standard care in many countries, but not all patients are eligible for these drugs due to age and genotype. Here, we aimed to determine the characteristics of non-eligible patients for CFTR modulators in the CF registry of Turkey (CFRT) to highlight their clinical needs. Methods: This retrospective cohort study included CF patient data from the CFRT in 2021. The decision of eligibility for the CFTR modulator was determined according to the 'Vertex treatment-Finder' on the Vertex (R) website. Demographic and clinical characteristics of patients were compared between eligible (group 1) and ineligible (group 2) groups for CFTR modulators. Results: Among the study population (N = 1527), 873 (57.2%) were in group 1 and 654 (42.8%) were in group 2. There was no statistical difference between groups regarding sex, meconium ileus history, diagnoses via newborn screening, FEV1 z-score, CF-associated complications, organ transplant history, and death. Patients in group 2 had a higher incidence of pancreatic insufficiency (87.7% vs. 83.2%, p = .010), lower median height z-scores (-0.87 vs. -0.55, p < .001), lower median body mass index z-scores (-0.65 vs. -0.50, p < .001), longer days receiving antibiotics due to pulmonary exacerbation (0 [interquartile range, IQR: 0-2] vs. 0 [IQR: 0-7], p = 0.001), and more non-invasive ventilation support (2.6% vs. 0.9%, p = 0.008) than patients in group 1. Conclusion: The ineligible group had worse clinical outcomes than the eligible group. This highlights their need for life-changing drugs to improve clinical outcomes.en_US
dc.description.sponsorshipNoneen_US
dc.description.sponsorshipWe would like to thank the Cystic Fibrosis Registry of Turkey for supplying access to patient data and individual center representatives for allowing the use of their data.en_US
dc.identifier.doi10.1002/ppul.27051
dc.identifier.endpage2506en_US
dc.identifier.issn8755-6863
dc.identifier.issn1099-0496
dc.identifier.issue10en_US
dc.identifier.pmid38771207en_US
dc.identifier.scopus2-s2.0-85193687493en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage2499en_US
dc.identifier.urihttps://doi.org/10.1002/ppul.27051
dc.identifier.urihttps://hdl.handle.net/11468/29813
dc.identifier.volume59en_US
dc.identifier.wosWOS:001228233000001en_US
dc.identifier.wosqualityQ1en_US
dc.indekslendigikaynakWeb of Science
dc.indekslendigikaynakScopus
dc.indekslendigikaynakPubMed
dc.language.isoenen_US
dc.publisherWileyen_US
dc.relation.ispartofPediatric Pulmonologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.snmzKA_WOS_20250222
dc.subjectCFTR modulatorsen_US
dc.subjectclinical featuresen_US
dc.subjectcystic fibrosisen_US
dc.subjecteligibilityen_US
dc.subjectregistryen_US
dc.titleComparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulatorsen_US
dc.typeArticleen_US

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