• Nazan Çobanoğlu, Uğur Özçelik, Erkan Çakır, Şişmanlar EyüboğluTuğbaT0000-0001-7284-4999Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Sevgi Pekcan, Güzin Cinel, Ebru Yalçın, Nural Kiper, Nagehan Emiralioğlu, Velat Şen, Hadice Selimoğlu Şen, Ömür Ercan, Haluk Çokuğraş, Ayşe Ayzıt Kılınç, Lina Muhammed Al Shadfan, Hakan Yazan, Derya Ufuk Altıntaş, Dilek Karagöz, Esen Demir, Gökçen Kartal Öztürk, Ayşen Bingöl, Abdurrahman Erdem Başaran, Nihat Sapan, Şükrü Çekiç, Ebru Çelebioğlu, Ayşe Tana Aslan, Tuğba Ramaslı Gürsoy, Gökçen Tuğcu, Ali Özdemir, Koray Harmancı, Gonca Kılıç Yıldırım, Mehmet Köse, Melih Hangül, Zeynep Tamay, Ayşe Süleyman, Hasan Yüksel, Özge Yılmaz, Gizem Özcan, Erdem Topal, Demet Can, Korkmaz EkrenPervinPDepartment of Pulmonology, Faculty of Medicine, Ege University, İzmir, Turkey., Gönül Çaltepe, Mehmet Kılıç, Şebnem Özdoğan, and Deniz Doğru.
• Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey.
• Pediatr. Pulmonol. 2020 Sep 1; 55 (9): 2302-2306.

BackgroundA 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.MethodsData 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.ResultsOf 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).ConclusionsThe 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.© 2020 Wiley Periodicals LLC.

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