Patients eligible for modulator drugs: Data from cystic fibrosis registry of Turkey

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dc.authoridtopal, erdem/0000-0002-4439-2689
dc.authoridYilmaz, Ozge/0000-0001-6051-5020
dc.authoridTugcu, Gokcen Dilsa/0000-0002-9804-1200
dc.authoridCekic, Sukru/0000-0002-9574-1842
dc.authoridRamasli Gursoy, Tugba/0000-0002-7064-7585
dc.authoridSismanlar Eyuboglu, Tugba/0000-0001-7284-4999
dc.authoridEmiralioglu, Nagehan/0000-0002-1405-8401
dc.authorwosidtopal, erdem/ABI-7545-2020
dc.authorwosidYilmaz, Ozge/A-1463-2013
dc.authorwosidTugcu, Gokcen Dilsa/ABG-5531-2021
dc.authorwosidCekic, Sukru/L-1933-2017
dc.authorwosidCinel, Güzin/AAW-5222-2021
dc.authorwosidRamasli Gursoy, Tugba/AAA-7773-2020
dc.authorwosidSismanlar Eyuboglu, Tugba/AAK-5467-2020
dc.contributor.authorCobanoglu, Nazan
dc.contributor.authorOzcelik, Ugur
dc.contributor.authorCakir, Erkan
dc.contributor.authorEyuboglu, Tugba Sismanlar
dc.contributor.authorPekcan, Sevgi
dc.contributor.authorCinel, Guzin
dc.contributor.authorYalcin, Ebru
dc.date.accessioned2024-08-04T20:47:22Z
dc.date.available2024-08-04T20:47:22Z
dc.date.issued2020
dc.departmentİnönü Üniversitesien_US
dc.description.abstractBackground A better understanding of cystic fibrosis transmembrane conductance regulator biology has led to the development of modulator drugs such as ivacaftor, lumacaftor-ivacaftor, tezacaftor-ivacaftor, and elexacaftor-tezacaftor-ivacaftor. This cross-sectional study evaluated cystic fibrosis (CF) patients eligible for modulator drugs. Methods Data for age and genetic mutations from the Cystic Fibrosis Registry of Turkey collected in 2018 were used to find out the number of patients who are eligible for modulator therapy. Results Of registered 1488 CF patients, genetic analysis was done for 1351. The numbers and percentages of patients and names of the drugs, that the patients are eligible for, are as follows: 122 (9.03%) for ivacaftor, 156 (11.54%) for lumacaftor-ivacaftor, 163 (11.23%) for tezacaftor-ivacaftor, and 57 (4.21%) for elexacaftor-tezacaftor-ivacaftor. Among 1351 genotyped patients total of 313 (23.16%) patients are eligible for currently licensed modulator therapies (55 patients were shared by ivacaftor and tezacaftor-ivacaftor, 108 patients were shared by lumacaftor-ivacaftor and tezacaftor-ivacaftor, and 22 patients were shared by tezacaftor-ivacaftor and elexacaftor-tezacaftor-ivacaftor groups). Conclusions The present study shows that approximately one-fourth of the registered CF patients in Turkey are eligible for modulator drugs. As, frequent mutations that CF patients have in Turkey are different from North American and European CF patients, developing modulator drugs effective for those mutations is necessary. Furthermore, as modulator drugs are very expensive currently, financial support of the government in developing countries like Turkey is noteworthy.en_US
dc.identifier.doi10.1002/ppul.24854
dc.identifier.endpage2306en_US
dc.identifier.issn8755-6863
dc.identifier.issn1099-0496
dc.identifier.issue9en_US
dc.identifier.pmid32453906en_US
dc.identifier.scopus2-s2.0-85085547506en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage2302en_US
dc.identifier.urihttps://doi.org/10.1002/ppul.24854
dc.identifier.urihttps://hdl.handle.net/11616/99313
dc.identifier.volume55en_US
dc.identifier.wosWOS:000535323700001en_US
dc.identifier.wosqualityQ2en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
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.subjectcystic fibrosisen_US
dc.subjectmodulator drugsen_US
dc.subjectnational registryen_US
dc.titlePatients eligible for modulator drugs: Data from cystic fibrosis registry of Turkeyen_US
dc.typeArticleen_US

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