Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators.
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Nayır Büyükşahin Halime, Emiralioğlu Nagehan, Yalçın Ebru, Şen Velat, Selimoğlu Şen Hadice, Arslan Hüseyin, Başkan Azer Kılıç, Çakır Fatma Betül, Koray Cem Fırat, Yılmaz Aslı İmran, Ercan Fatih, Altıntaş Derya Ufuk, Serbes Mahir, Keskin Özlem, Arık Elif, Gülen Figen, Barlık Meral, Karcıoğlu Oğuz, Damadoğlu Ebru, Köse Mehmet, Ersoy Ali, Bingöl Ayşen, Başaran Erdem, Çakır Eylül Pınar, Aslan Ayşe Tana, Canıtez Yakup, Korkmaz Merve, Özdemir Ali, Harmancı Koray, Soydaş Şule Selin, Hangül Melih, Yüksel Hasan, Özcan Gizem, Korkmaz Pervin, Kılıç Mehmet, Gayretli Aydın Zeynep Gökçe, Çaltepe Gönül, Can Demet, Doğru Sibel, Kartal Öztürk Gökçen, Süleyman Ayşe, Topal Erdem, Özsezen Beste, Hızal Mina, Demirdöğen Ezgi, Ogun Hamza, Börekçi Şermin, Yazan Hakan, Çakır Erkan, Şişmanlar Eyüboğlu Tuğba, Çobanoğlu Nazan, Cinel Güzin, Pekcan Sevgi, Özçelik Uğur, Doğru Deniz
Аннотация
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.