Eligibility of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapies: cohort of cystic fibrosis registry of Türkiye
Only half of patients registered in CFrT were eligible for CFTRms, which is a significant difference from the CFTR variant profile seen in USA and Europe. However, access to treatment is hampered for some patients whose genes are not analysed. Further studies in CF populations, where rare mutations are relatively more common, will contribute to the field of CFTR modulator treatments for such rare mutations.
Authors
- Tuğba Şişmanlar Eyüboğlu (ORCID: https://orcid.org/0000-0001-7284-4999)
- Meltem Akgül Erdal (ORCID: https://orcid.org/0000-0002-7507-0770)
- Erdem Topal (ORCID: https://orcid.org/0000-0002-4439-2689)
- Haluk Cezmi Çokuğraş (ORCID: https://orcid.org/0000-0002-0086-3936)
- Deni̇z Doğru (ORCID: https://orcid.org/0000-0001-9931-9473)
- Hasan Yüksel (ORCID: https://orcid.org/0000-0001-9577-8445)
- Gökçen Kartal Öztürk (ORCID: https://orcid.org/0000-0002-0793-9710)
- Mahir Serbes (ORCID: https://orcid.org/0000-0001-6422-2639)
- Elif Arık (ORCID: https://orcid.org/0000-0002-7319-8535)
- Ali Özdemir (ORCID: https://orcid.org/0000-0001-7340-0409)
- Gizem Özcan (ORCID: https://orcid.org/0000-0001-9063-4063)
- Yakup Canıtez (ORCID: https://orcid.org/0000-0001-8929-679X)
- Halime Nayır Büyükşahin (ORCID: https://orcid.org/0000-0002-6909-7993)
- Mina Hızal (ORCID: https://orcid.org/0000-0002-6922-4948)
- Derya Ufuk Altıntaş (ORCID: https://orcid.org/0000-0003-2090-5248)
- Nagehan Emiralioğlu (ORCID: https://orcid.org/0000-0002-1405-8401)
- Güzide Doğan (ORCID: https://orcid.org/0000-0003-4291-7282)
- Ayşe Ayzıt Kılınç (ORCID: https://orcid.org/0000-0002-2879-8910)
- Mehmet Mustafa Özaslan (ORCID: https://orcid.org/0000-0003-0611-0852)
- Mehmet Kılıç (ORCID: https://orcid.org/0000-0002-1089-1316)
- Erkan Çakır (ORCID: https://orcid.org/0000-0002-1438-7854)
- Şermin Börekçi (ORCID: https://orcid.org/0000-0002-0089-1312)
- Aslı İmran Yılmaz (ORCID: https://orcid.org/0000-0003-2689-7904)
- Koray Harmancı (ORCID: https://orcid.org/0000-0002-8494-648X)
- Gökçen Ünal (ORCID: https://orcid.org/0000-0002-4380-7643)
- Ezgi Demirdöğen (ORCID: https://orcid.org/0000-0002-7400-9089)
- Abdurrahman Erdem Başaran (ORCID: https://orcid.org/0000-0002-9092-6936)
- Melih Hangül (ORCID: https://orcid.org/0000-0001-6226-0340)
- Gönül Çaltepe (ORCID: https://orcid.org/0000-0001-8525-6352)
- Salih Uytun (ORCID: https://orcid.org/0000-0002-7301-3692)
- Mehmet Köse (ORCID: https://orcid.org/0000-0002-3003-918X)
- Oğuz Karcıoğlu (ORCID: https://orcid.org/0000-0002-6071-0851)
- Sanem Eryılmaz Polat (ORCID: https://orcid.org/0000-0003-2309-7952)
- Sibel Doğru (ORCID: https://orcid.org/0000-0002-0578-5025)
- Hamza Ogün (ORCID: https://orcid.org/0000-0002-4799-9670)
- Velat Şen (ORCID: https://orcid.org/0000-0002-8411-4499)
- Zeynep Gökçe Gayretli Aydın (ORCID: https://orcid.org/0000-0003-4291-1067)
- Ayşe Süleyman (ORCID: https://orcid.org/0000-0002-9190-6849)
- Hakan Yazan (ORCID: https://orcid.org/0000-0002-7680-4000)
- Pervin Korkmaz Ekren (ORCID: https://orcid.org/0000-0002-6913-9970)
- Beste Özsezen (ORCID: https://orcid.org/0000-0002-0052-8361)
- Uğur Özçelik (ORCID: https://orcid.org/0000-0003-1587-5216)
- Sevgi Pekcan (ORCID: https://orcid.org/0000-0002-8059-902X)
- Ayşegül Doğan Demir (ORCID: https://orcid.org/0000-0002-2536-1422)
- Özlem Keskin (ORCID: https://orcid.org/0000-0002-3061-7832)
- Demet Can (ORCID: https://orcid.org/0000-0002-1258-9348)
- Hadice Selimoğlu Şen (ORCID: https://orcid.org/0000-0002-2597-6646)
- Eylül Pınar Çakır (ORCID: https://orcid.org/0000-0003-2067-3256)
Institutions
- Dicle University (TR)
- Ministry of Health (TR)
- Bursa Uludağ Üni̇versi̇tesi̇ (TR)
- Ankara University (TR)
- University of Turku (FI)
- Karadeniz Technical University (TR)
- Manisa Celal Bayar University (TR)
- Bursa Technical University (TR)
- Akdeniz University (TR)
- Inonu University (TR)
- Mardin Artuklu University (TR)
- Ondokuz Mayıs University (TR)
- Bezmiâlem Vakıf Üniversitesi (TR)
- Ege University (TR)
- Dr. Behçet Uz Çocuk Hastalıkları Hastanesi (TR)
- Gaziantep Children's Hospital (TR)
- Memorial Ankara Hospital (TR)
- Biruni University (TR)
- Necmettin Erbakan University (TR)
- Ümraniye Eğitim ve Araştırma Hastanesi (TR)
- Istanbul University-Cerrahpaşa (TR)
- Dr Sami Ulus Çocuk Sağlığı ve Hastalıkları Eğitim ve Araştırma Hastanesi (TR)
- Istinye University (TR)
- Education Training And Research (US)
- S.B.Ü. Bursa Yüksek İhtisas Eğitim ve Araştırma Hastanesi (TR)
- Eskişehir Osmangazi University (TR)
- Mersin Üniversitesi (TR)
- Cukurova University (TR)
- Gaziantep University (TR)
- Hacettepe University (TR)
- Istanbul University (TR)
- Erciyes University (TR)
- Gazi University (TR)
Publication Details
- Journal
- The Turkish Journal of Pediatrics
- Published
- 2025-02-20
- DOI
- https://doi.org/10.24953/turkjpediatr.2025.4680
- Citations
- 3
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 4.56