Pediatric Pulmonology
Volume 59, Issue 10 pp. 2499-2506
ORIGINAL ARTICLE

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

Halime Nayır Büyükşahin MD, 

Corresponding Author

Halime Nayır Büyükşahin MD

Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey

Correspondence Halime Nayır Büyükşahin, MD, Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey.

Email: [email protected]

Contribution: Conceptualization, Methodology

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Nagehan Emiralioğlu MD, 

Nagehan Emiralioğlu MD

Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey

Contribution: Data curation, Writing - review & editing

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Ebru Yalçın MD, 

Ebru Yalçın MD

Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey

Contribution: Data curation, Writing - review & editing

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Velat Şen MD, 

Velat Şen MD

Division of Pulmonology, Department of Pediatrics, Dicle University Faculty of Medicine, Diyarbakır, Turkey

Contribution: Writing - review & editing, Data curation

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Hadice Selimoğlu Şen MD, 

Hadice Selimoğlu Şen MD

Department of Pulmonology, Dicle University Faculty of Medicine, Diyarbakır, Turkey

Contribution: Data curation, Writing - review & editing

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Hüseyin Arslan MD, 

Hüseyin Arslan MD

Division of Pediatric Allergy and Pulmonology, Department of Pediatrics, Cerrahpaşa Medicine Faculty, Istanbul University, Istanbul, Turkey

Contribution: Writing - review & editing

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Azer Kılıç Başkan MD, 

Azer Kılıç Başkan MD

Division of Pediatric Allergy and Pulmonology, Department of Pediatrics, Cerrahpaşa Medicine Faculty, Istanbul University, Istanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Fatma Betül Çakır MD, 

Fatma Betül Çakır MD

Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem University, Istanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Cem Fırat Koray MD, 

Cem Fırat Koray MD

Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem University, Istanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Aslı İmran Yılmaz MD, 

Aslı İmran Yılmaz MD

Division of Pediatric Pulmonology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey

Contribution: Data curation, Writing - review & editing

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Fatih Ercan MD, 

Fatih Ercan MD

Division of Pediatric Pulmonology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey

Contribution: Data curation, Writing - review & editing

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Derya Ufuk Altıntaş MD, 

Derya Ufuk Altıntaş MD

Division of Pediatric Allergy and Immunology, Faculty of Medicine, Çukurova University, Adana, Turkey

Contribution: Writing - review & editing, Data curation

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Mahir Serbes MD, 

Mahir Serbes MD

Division of Pediatric Allergy and Immunology, Faculty of Medicine, Çukurova University, Adana, Turkey

Contribution: Data curation, Writing - review & editing

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Özlem Keskin MD, 

Özlem Keskin MD

Division of Pediatric Allergy, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey

Contribution: Data curation, Writing - review & editing

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Elif Arık MD, 

Elif Arık MD

Division of Pediatric Allergy, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey

Contribution: Data curation, Writing - review & editing

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Figen Gülen MD, 

Figen Gülen MD

Division of Pulmonology, Department of Pediatrics, Ege University Faculty of Medicine, Diyarbakır, Turkey

Contribution: Data curation, Writing - review & editing

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Meral Barlık MD, 

Meral Barlık MD

Division of Pulmonology, Department of Pediatrics, Ege University Faculty of Medicine, Diyarbakır, Turkey

Contribution: Data curation, Writing - review & editing

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Oğuz Karcıoğlu MD, 

Oğuz Karcıoğlu MD

Department of Pulmonology, Hacettepe University Faculty of Medicine, Ankara, Turkey

Contribution: Data curation, Writing - review & editing

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Ebru Damadoğlu MD, 

Ebru Damadoğlu MD

Department of Pulmonology, Hacettepe University Faculty of Medicine, Ankara, Turkey

Contribution: Data curation, Writing - review & editing

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Mehmet Köse MD, 

Mehmet Köse MD

Division of Pediatric Pulmonology, Faculty of Medicine, Erciyes University, Kayseri, Turkey

Contribution: Data curation, Writing - review & editing

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Ali Ersoy MD, 

Ali Ersoy MD

Division of Pediatric Pulmonology, Faculty of Medicine, Erciyes University, Kayseri, Turkey

Contribution: Data curation, Writing - review & editing

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Ayşen Bingöl MD, 

Ayşen Bingöl MD

Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Akdeniz University, Antalya, Turkey

Contribution: Data curation, Writing - review & editing

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Erdem Başaran MD, 

Erdem Başaran MD

Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Akdeniz University, Antalya, Turkey

Contribution: Data curation, Writing - review & editing

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Eylül Pınar Çakır MD, 

Eylül Pınar Çakır MD

Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey

Contribution: Data curation, Writing - review & editing

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Ayşe Tana Aslan MD, 

Ayşe Tana Aslan MD

Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey

Contribution: Writing - review & editing, Data curation

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Yakup Canıtez MD, 

Yakup Canıtez MD

Division of Pediatric Allergy and Immunology, Faculty of Medicine, Bursa Uludağ University, Bursa, Turkey

Contribution: Data curation, Writing - review & editing

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Merve Korkmaz MD, 

Merve Korkmaz MD

Division of Pediatric Allergy and Immunology, Faculty of Medicine, Bursa Uludağ University, Bursa, Turkey

Contribution: Data curation, Writing - review & editing

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Ali Özdemir MD, 

Ali Özdemir MD

Division of Pediatric Pulmonology, Mersin City Training and Research Hospital, Mersin, Turkey

Contribution: Data curation, Writing - review & editing

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Koray Harmancı MD, 

Koray Harmancı MD

Division of Pediatric Allergy and Immunology, Faculty of Medicine, Eskişehir Osmangazi University, Eskişehir, Turkey

Contribution: Data curation, Writing - review & editing

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Şule Selin Soydaş MD, 

Şule Selin Soydaş MD

Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey

Contribution: Writing - review & editing, Data curation

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Melih Hangül MD, 

Melih Hangül MD

Division of Pediatric Pulmonology, Gaziantep Cengiz Gökçek Maternity and Children Hospital, Gaziantep, Turkey

Contribution: Data curation, Writing - review & editing

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Hasan Yüksel MD, 

Hasan Yüksel MD

Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Celal Bayar University, Manisa, Turkey

Contribution: Data curation, Writing - review & editing

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Gizem Özcan MD, 

Gizem Özcan MD

Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey

Contribution: Writing - review & editing, Data curation

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Pervin Korkmaz MD, 

Pervin Korkmaz MD

Department of Pulmonology, Faculty of Medicine, Ege University, İzmir, Turkey

Contribution: Data curation, Writing - review & editing

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Mehmet Kılıç MD, 

Mehmet Kılıç MD

Division of Pediatric Allergy and Immunology, Faculty of Medicine, Fırat University, Elazığ, Turkey

Contribution: Data curation, Writing - review & editing

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Zeynep Gökçe Gayretli Aydın MD, 

Zeynep Gökçe Gayretli Aydın MD

Division of Pediatric Infectious Disease, Faculty of Medicine, Karadeniz Technical University, Trabzon, Turkey

Contribution: Data curation, Writing - review & editing

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Gönül Çaltepe MD, 

Gönül Çaltepe MD

Division of Pediatric Gastroenterology, Hepatology and Nutrition, Faculty of Medicine, Ondokuz Mayıs University, Samsun, Turkey

Contribution: Data curation, Writing - review & editing

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Demet Can MD, 

Demet Can MD

Division of Pediatric Pulmonology, Faculty of Medicine, Balıkesir University, Balıkesir, Turkey

Contribution: Writing - review & editing, Data curation

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Sibel Doğru MD, 

Sibel Doğru MD

Department of Pulmonology, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey

Contribution: Data curation, Writing - review & editing

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Gökçen Kartal Öztürk MD, 

Gökçen Kartal Öztürk MD

Division of Pediatric Pulmonology, Dr.Behçet Uz Children's Hospital, Izmir, Turkey

Contribution: Data curation, Writing - review & editing

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Ayşe Süleyman MD, 

Ayşe Süleyman MD

Division of Pediatric Allergy and Immunology, Istanbul University/Istanbul Faculty of Medicine, Istanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Erdem Topal MD, 

Erdem Topal MD

Division of Pediatric Allergy and Immunology, Inönü University Faculty of Medicine, Malatya, Turkey

Contribution: Data curation, Writing - review & editing

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Beste Özsezen MD, 

Beste Özsezen MD

Division of Pediatric Pulmonology, Şanliurfa Training and Research Hospital, Şanliurfa, Turkey

Contribution: Data curation, Writing - review & editing

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Mina Hızal MD, 

Mina Hızal MD

Division of Pediatric Pulmonology, Dr Sami Ulus Maternity and Children Training and Research Hospital, Ankara, Turkey

Contribution: Data curation, Writing - review & editing

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Ezgi Demirdöğen MD, 

Ezgi Demirdöğen MD

Department of Pulmonology, Faculty of Medicine, Bursa Uludağ University, Bursa, Turkey

Contribution: Data curation, Writing - review & editing

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Hamza Ogun MD, 

Hamza Ogun MD

Department of Pulmonology, Faculty of Medicine, Bezmialem University, İstanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Şermin Börekçi MD, 

Şermin Börekçi MD

Department of Pulmonology, Cerrahpaşa Medicine Faculty, Istanbul University, Istanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Hakan Yazan MD, 

Hakan Yazan MD

Division of Pediatric Pulmonology, Istanbul Ümraniye Training and Research Hospital, Istanbul, Turkey

Contribution: Data curation, Writing - review & editing

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Erkan Çakır MD, 

Erkan Çakır MD

Division of Pediatric Pulmonology, Faculty of Medicine, İstinye University, İstanbul, Turkey

Contribution: Data curation, Writing - review & editing, Methodology

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Tuğba Şişmanlar Eyüboğlu MD, 

Tuğba Şişmanlar Eyüboğlu MD

Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey

Contribution: Methodology, Writing - review & editing, Data curation

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Nazan Çobanoğlu MD, 

Nazan Çobanoğlu MD

Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey

Contribution: Data curation, Methodology, Writing - review & editing

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Güzin Cinel MD, 

Güzin Cinel MD

Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey

Contribution: Methodology, Data curation, Writing - review & editing

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Sevgi Pekcan MD, 

Sevgi Pekcan MD

Division of Pediatric Pulmonology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey

Contribution: Methodology, Data curation, Writing - review & editing

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Uğur Özçelik MD, 

Uğur Özçelik MD

Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey

Contribution: Methodology, Data curation, Writing - review & editing

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Deniz Doğru MD, 

Deniz Doğru MD

Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey

Contribution: Data curation, Methodology, Writing - review & editing, Writing - original draft, Conceptualization

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First published: 21 May 2024
https://doi.org/10.1002/ppul.27051
Citations: 2

Abstract

Introduction

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® 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.

CONFLICT OF INTEREST STATEMENT

The authors declare no conflicts of interest.

DATA AVAILABILITY STATEMENT

The data that support the findings of this study are available from the corresponding author upon reasonable request.

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