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

Autor: Nayır Büyükşahin H; Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey., Emiralioğlu N; Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey., Yalçın E; Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey., Şen V; Division of Pulmonology, Department of Pediatrics, Dicle University Faculty of Medicine, Diyarbakır, Turkey., Selimoğlu Şen H; Department of Pulmonology, Dicle University Faculty of Medicine, Diyarbakır, Turkey., Arslan H; Division of Pediatric Allergy and Pulmonology, Department of Pediatrics, Cerrahpaşa Medicine Faculty, Istanbul University, Istanbul, Turkey., Başkan AK; Division of Pediatric Allergy and Pulmonology, Department of Pediatrics, Cerrahpaşa Medicine Faculty, Istanbul University, Istanbul, Turkey., Çakır FB; Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem University, Istanbul, Turkey., Koray CF; Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem University, Istanbul, Turkey., Yılmaz Aİ; Division of Pediatric Pulmonology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey., Ercan F; Division of Pediatric Pulmonology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey., Altıntaş DU; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Çukurova University, Adana, Turkey., Serbes M; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Çukurova University, Adana, Turkey., Keskin Ö; Division of Pediatric Allergy, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey., Arık E; Division of Pediatric Allergy, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey., Gülen F; Division of Pulmonology, Department of Pediatrics, Ege University Faculty of Medicine, Diyarbakır, Turkey., Barlık M; Division of Pulmonology, Department of Pediatrics, Ege University Faculty of Medicine, Diyarbakır, Turkey., Karcıoğlu O; Department of Pulmonology, Hacettepe University Faculty of Medicine, Ankara, Turkey., Damadoğlu E; Department of Pulmonology, Hacettepe University Faculty of Medicine, Ankara, Turkey., Köse M; Division of Pediatric Pulmonology, Faculty of Medicine, Erciyes University, Kayseri, Turkey., Ersoy A; Division of Pediatric Pulmonology, Faculty of Medicine, Erciyes University, Kayseri, Turkey., Bingöl A; Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Akdeniz University, Antalya, Turkey., Başaran E; Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Akdeniz University, Antalya, Turkey., Çakır EP; Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Aslan AT; Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Canıtez Y; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Bursa Uludağ University, Bursa, Turkey., Korkmaz M; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Bursa Uludağ University, Bursa, Turkey., Özdemir A; Division of Pediatric Pulmonology, Mersin City Training and Research Hospital, Mersin, Turkey., Harmancı K; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Eskişehir Osmangazi University, Eskişehir, Turkey., Soydaş ŞS; Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey., Hangül M; Division of Pediatric Pulmonology, Gaziantep Cengiz Gökçek Maternity and Children Hospital, Gaziantep, Turkey., Yüksel H; Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Celal Bayar University, Manisa, Turkey., Özcan G; Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey., Korkmaz P; Department of Pulmonology, Faculty of Medicine, Ege University, İzmir, Turkey., Kılıç M; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Fırat University, Elazığ, Turkey., Gayretli Aydın ZG; Division of Pediatric Infectious Disease, Faculty of Medicine, Karadeniz Technical University, Trabzon, Turkey., Çaltepe G; Division of Pediatric Gastroenterology, Hepatology and Nutrition, Faculty of Medicine, Ondokuz Mayıs University, Samsun, Turkey., Can D; Division of Pediatric Pulmonology, Faculty of Medicine, Balıkesir University, Balıkesir, Turkey., Doğru S; Department of Pulmonology, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey., Kartal Öztürk G; Division of Pediatric Pulmonology, Dr.Behçet Uz Children's Hospital, Izmir, Turkey., Süleyman A; Division of Pediatric Allergy and Immunology, Istanbul University/Istanbul Faculty of Medicine, Istanbul, Turkey., Topal E; Division of Pediatric Allergy and Immunology, Inönü University Faculty of Medicine, Malatya, Turkey., Özsezen B; Division of Pediatric Pulmonology, Şanliurfa Training and Research Hospital, Şanliurfa, Turkey., Hızal M; Division of Pediatric Pulmonology, Dr Sami Ulus Maternity and Children Training and Research Hospital, Ankara, Turkey., Demirdöğen E; Department of Pulmonology, Faculty of Medicine, Bursa Uludağ University, Bursa, Turkey., Ogun H; Department of Pulmonology, Faculty of Medicine, Bezmialem University, İstanbul, Turkey., Börekçi Ş; Department of Pulmonology, Cerrahpaşa Medicine Faculty, Istanbul University, Istanbul, Turkey., Yazan H; Division of Pediatric Pulmonology, Istanbul Ümraniye Training and Research Hospital, Istanbul, Turkey., Çakır E; Division of Pediatric Pulmonology, Faculty of Medicine, İstinye University, İstanbul, Turkey., Şişmanlar Eyüboğlu T; Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Çobanoğlu N; Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey., Cinel G; Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey., Pekcan S; Division of Pediatric Pulmonology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey., Özçelik U; Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey., Doğru D; Division of Pulmonology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Jazyk: angličtina
Zdroj: Pediatric pulmonology [Pediatr Pulmonol] 2024 Oct; Vol. 59 (10), pp. 2499-2506. Date of Electronic Publication: 2024 May 21.
DOI: 10.1002/ppul.27051
Abstrakt: 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.
(© 2024 Wiley Periodicals LLC.)
Databáze: MEDLINE
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