Patients with cystic fibrosis who could not receive the CFTR modulator treatment: What did they lose in 1 year?

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dc.authorid0000-0002-6209-196Xen_US
dc.authorid0000-0002-7301-3692en_US
dc.authorid0000-0003-1261-7393en_US
dc.authorid0000-0002-9232-5982en_US
dc.authorid0000-0002-2597-6646en_US
dc.contributor.authorUytun, Salih
dc.contributor.authorCinel, Güzin
dc.contributor.authorPolat, Sanem Eryılmaz
dc.contributor.authorTabakçı, Satı Özkan
dc.contributor.authorKiper, Nural
dc.contributor.authorYalçın, Ebru
dc.contributor.authorŞen, Velat
dc.contributor.authorŞen, Hadice Selimoğlu
dc.date.accessioned2023-10-16T06:31:27Z
dc.date.available2023-10-16T06:31:27Z
dc.date.issued2023en_US
dc.departmentDicle Üniversitesi, Tıp Fakültesi, Dahili Tıp Bilimleri Bölümü, Çocuk Sağlığı ve Hastalıkları Ana Bilim Dalıen_US
dc.description.abstractBackground: Cystic fibrosis (CF) is an autosomal recessive disorder caused by CF transmembrane conductance regulator (CFTR) genetic variants. CFTR modulators improve pulmonary function and reduce respiratory infections in CF. This study investigated the clinical and laboratory follow-up parameters over 1 year in patients with CF who could not receive this treatment. Methods: This retrospective cohort study included 2018 and 2019 CF patient data from the CF registry of Turkey. Demographic and clinical characteristics of 294 patients were assessed, who had modulator treatment indications in 2018 but could not reach the treatment. Results: In 2019, patients younger than 18 years had significantly lower BMI z-scores than in 2018. During the 1-year follow-up, forced expiratory volumes (FEV1) and FEV1 z-scores a trend toward a decrease. In 2019, chronic Staphylococcus aureus colonization, inhaled antipseudomonal antibiotic use for more than 3 months, oral nutritional supplement requirements, and oxygen support need increased. Conclusions: Patients who had indications for modulator treatments but were unable to obtain them worsened even after a year of follow-up. This study emphasized the importance of using modulator treatments for patients with CF in our country, as well as in many countries worldwide.en_US
dc.identifier.citationUytun, S., Cinel, G., Polat, S. E., Tabakçı, S. Ö., Kiper, N., Yalçın, E. ve diğerleri. (2023). Patients with cystic fibrosis who could not receive the CFTR modulator treatment: What did they lose in 1 year?. Pediatric Pulmonology, (58), 2505-2512.en_US
dc.identifier.doi10.1002/ppul.26535
dc.identifier.endpage2512en_US
dc.identifier.issn8755-6863
dc.identifier.issue58en_US
dc.identifier.pmid37278544
dc.identifier.scopus2-s2.0-85161522932
dc.identifier.scopusqualityQ1
dc.identifier.startpage2505en_US
dc.identifier.urihttps://onlinelibrary.wiley.com/doi/epdf/10.1002/ppul.26535
dc.identifier.urihttps://hdl.handle.net/11468/12813
dc.identifier.wosWOS:001001347000001
dc.identifier.wosqualityN/A
dc.indekslendigikaynakWeb of Science
dc.indekslendigikaynakScopus
dc.indekslendigikaynakPubMed
dc.institutionauthorŞen, Velat
dc.institutionauthorŞen, Hadice Selimoğlu
dc.language.isoenen_US
dc.publisherJohn Wiley and Sons Incen_US
dc.relation.ispartofPediatric Pulmonology
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/embargoedAccessen_US
dc.subjectBody mass indexen_US
dc.subjectCFTR modulatoren_US
dc.subjectCystic fibrosisen_US
dc.subjectForced expiratory volumeen_US
dc.subjectz-scoresen_US
dc.titlePatients with cystic fibrosis who could not receive the CFTR modulator treatment: What did they lose in 1 year?en_US
dc.titlePatients with cystic fibrosis who could not receive the CFTR modulator treatment: What did they lose in 1 year?
dc.typeArticleen_US

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