Patients with cystic fibrosis who could not receive the CFTR modulator treatment: What did they lose in 1 year?

Autor: Uytun S; Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey., Cinel G; Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey.; Division of Pediatric Pulmonology, Faculty of Medicine, Ankara Yıldırım Beyazıt University, Ankara, Turkey., Eryılmaz Polat S; Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey., Özkan Tabakçı S; Division of Pediatric Pulmonology, Ankara City Hospital, Ankara, Turkey., Kiper N; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Yalçın E; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Ademhan Tural D; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Özsezen B; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Şen V; Division of Pediatric Pulmonology, Faculty of Medicine, Dicle University, Diyarbakır, Turkey., Selimoğlu Şen H; Department of Pulmonology, Faculty of Medicine, Dicle University, Diyarbakır, Turkey., Ufuk Altıntaş D; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Çukurova University, Adana, Turkey., Çokuğraş H; Division of Pediatric Pulmonology, Cerrahpaşa Faculty of Medicine, İstanbul University-Cerrahpaşa, İstanbul, Turkey., Kılınç AA; Division of Pediatric Pulmonology, Cerrahpaşa Faculty of Medicine, İstanbul University-Cerrahpaşa, İstanbul, Turkey., Başkan AK; Division of Pediatric Pulmonology, Cerrahpaşa Faculty of Medicine, İstanbul University-Cerrahpaşa, İstanbul, Turkey., Yazan H; Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem Vakıf University, İstanbul, Turkey., Çollak A; Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem Vakıf University, İstanbul, Turkey., Uzuner S; Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem Vakıf University, İstanbul, Turkey., Ünal G; Division of Pediatric Pulmonology, Meram Faculty of Medicine, Necmettin Erbakan University, Konya, Turkey., Yılmaz Aİ; Division of Pediatric Pulmonology, Meram Faculty of Medicine, Necmettin Erbakan University, Konya, Turkey., Çağlar HT; Division of Pediatric Pulmonology, Meram Faculty of Medicine, Necmettin Erbakan University, Konya, Turkey., Damadoğlu E; Department of Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Irmak İ; Department of Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Demir E; Division of Pediatric Pulmonology, Faculty of Medicine, Ege University, İzmir, Turkey., Kartal Öztürk G; Division of Pediatric Pulmonology, Faculty of Medicine, Ege University, İzmir, Turkey., Bingöl A; Division of Pediatric Pulmonology, Faculty of Medicine, Akdeniz University, Antalya, Turkey., Başaran E; Division of Pediatric Pulmonology, Faculty of Medicine, Akdeniz University, Antalya, Turkey., Sapan N; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Uludağ University, Bursa, Turkey., Canıtez Y; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Uludağ University, Bursa, Turkey., Tana Aslan A; Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Asfuroğlu P; Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Harmancı K; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Eskişehir Osmangazi University, Eskişehir, Turkey., Köse M; Division of Pediatric Pulmonology, Faculty of Medicine, Erciyes University, Kayseri, Turkey., Hangül M; Division of Pediatric Pulmonology, Faculty of Medicine, Erciyes University, Kayseri, Turkey., Özdemir A; Division of Pediatric Pulmonology, Mersin City Training and Research Hospital, Mersin, Turkey., Çobanoğlu N; Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey., Özcan G; Division of Pediatric Pulmonology, Faculty of Medicine, Ankara University, Ankara, Turkey., Keskin Ö; Division of Pediatric Allergy and Immunology, Faculty of Medicine, Gaziantep University, Gaziantep, Turkey., Yüksel H; Division of Pediatric Pulmonology, Allergy and Immunology, Faculty of Medicine, Celal Bayar University, Manisa, Turkey., Özdoğan Ş; Division of Pediatric Pulmonology, Sarıyer Hamidiye Etfal Training and Research Hospital, İstanbul, Turkey., Topal E; Division of Pediatric Allergy and Immunology, Faculty of Medicine, İnönü University, Malatya, Turkey., Çaltepe G; Division of Pediatric Gastroenterology, Faculty of Medicine, Ondokuz Mayıs University, Samsun, Turkey., Can D; Division of Pediatric Pulmonology, Faculty of Medicine, Balıkesir University, Balıkesir, Turkey., Korkmaz Ekren 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., Emiralioğlu N; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Şişmanlar Eyüboğlu T; Division of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey., Pekcan S; Division of Pediatric Pulmonology, Meram Faculty of Medicine, Necmettin Erbakan University, Konya, Turkey., Çakır E; Division of Pediatric Pulmonology, Faculty of Medicine, Bezmialem Vakıf University, İstanbul, Turkey., Özçelik U; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Doğru D; Division of Pediatric Pulmonology, Faculty of Medicine, Hacettepe University, Ankara, Turkey.
Jazyk: angličtina
Zdroj: Pediatric pulmonology [Pediatr Pulmonol] 2023 Sep; Vol. 58 (9), pp. 2505-2512. Date of Electronic Publication: 2023 Jun 06.
DOI: 10.1002/ppul.26535
Abstrakt: Background: Cystic fibrosis (CF) is an autosomal recessive disorder caused by CF transmembrane conductance regulator (CFTR) genetic variants. CFTR modulators improve pulmonary function and reduce respiratory infections in CF. This study investigated the clinical and laboratory follow-up parameters over 1 year in patients with CF who could not receive this treatment.
Methods: This retrospective cohort study included 2018 and 2019 CF patient data from the CF registry of Turkey. Demographic and clinical characteristics of 294 patients were assessed, who had modulator treatment indications in 2018 but could not reach the treatment.
Results: In 2019, patients younger than 18 years had significantly lower BMI z-scores than in 2018. During the 1-year follow-up, forced expiratory volumes (FEV1) and FEV1 z-scores a trend toward a decrease. In 2019, chronic Staphylococcus aureus colonization, inhaled antipseudomonal antibiotic use for more than 3 months, oral nutritional supplement requirements, and oxygen support need increased.
Conclusions: Patients who had indications for modulator treatments but were unable to obtain them worsened even after a year of follow-up. This study emphasized the importance of using modulator treatments for patients with CF in our country, as well as in many countries worldwide.
(© 2023 Wiley Periodicals LLC.)
Databáze: MEDLINE
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