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

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dc.contributor.authorBüyüksahin, HN
dc.contributor.authorEmiralioglu, N
dc.contributor.authorYalçin, E
dc.contributor.authorSen, V
dc.contributor.authorSen, HS
dc.contributor.authorArslan, H
dc.contributor.authorBaskan, AK
dc.contributor.authorÇakir, FB
dc.contributor.authorKoray, CF
dc.contributor.authorYilmaz, AI
dc.contributor.authorErcan, F
dc.contributor.authorAltintas, DU
dc.contributor.authorSerbes, M
dc.contributor.authorKeskin, Ö
dc.contributor.authorArik, E
dc.contributor.authorGülen, F
dc.contributor.authorBarlik, M
dc.contributor.authorKarcioglu, O
dc.contributor.authorDamadoglu, E
dc.contributor.authorKöse, M
dc.contributor.authorErsoy, A
dc.contributor.authorBingöl, A
dc.contributor.authorBasaran, E
dc.contributor.authorÇakir, EP
dc.contributor.authorAslan, AT
dc.contributor.authorCanitez, Y
dc.contributor.authorKorkmaz, M
dc.contributor.authorÖzdemir, A
dc.contributor.authorHarmanci, K
dc.contributor.authorSoydas, SS
dc.contributor.authorHangül, M
dc.contributor.authorYüksel, H
dc.contributor.authorÖzcan, G
dc.contributor.authorKorkmaz, P
dc.contributor.authorKiliç, M
dc.contributor.authorAydin, ZGG
dc.contributor.authorÇaltepe, G
dc.contributor.authorCan, D
dc.contributor.authorDogru, S
dc.contributor.authorÖztürk, GK
dc.contributor.authorSüleyman, A
dc.contributor.authorTopal, E
dc.contributor.authorÖzsezen, B
dc.contributor.authorHizal, M
dc.contributor.authorDemirdögen, E
dc.contributor.authorOgun, H
dc.contributor.authorBörekçi, S
dc.contributor.authorYazan, H
dc.contributor.authorÇakir, E
dc.contributor.authorEyüboglu, TS
dc.contributor.authorÇobanoglu, N
dc.contributor.authorCinel, G
dc.contributor.authorPekcan, S
dc.contributor.authorÖzçelik, U
dc.contributor.authorDogru, D
dc.date.accessioned2025-04-10T10:37:47Z
dc.date.available2025-04-10T10:37:47Z
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.
dc.identifier.e-issn1099-0496
dc.identifier.issn8755-6863
dc.identifier.urihttp://hdl.handle.net/20.500.14701/43206
dc.language.isoEnglish
dc.titleComparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators
dc.typeArticle

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