Comparison of interrupted and continuous modulator therapy in cystic fibrosis: a real life experience in Turkey
Year: 2025
Type: article
Study design: Observational Study
Abstract: Introduction Cystic fibrosis (CF) transmembrane conductance regulator protein (CFTR) modulators have significantly improved health outcomes in patients with cystic fibrosis (pwCF). However, in Turkey, access is limited due to lack of insurance coverage, and treatment is only granted in 3-month periods via court rulings. This study aimed to compare clinical outcomes between patients receiving continuous vs. intermittent modulator therapy. Methods In this retrospective multicenter study, data from 229 CF patients across 14 centers in Turkey who received highly effective modulator therapy (HEMT) for at least six months were analyzed. Patients were grouped based on whether they received treatment continuously (Group 1) or with interruptions (Group 2). Changes in percent predicted forced expiratory volume in one second (ppFEV₁) and body mass index (BMI) were evaluated at baseline, 3 months, and 6 months. For Group 2, ppFEV₁ was also assessed during interruption periods. Results Of the 229 patients, 38.4% received continuous treatment while 61.5% experienced treatment interruptions. Both groups showed significant improvements in ppFEV₁ over six months (p < 0.001). However, Group 2 experienced a significant decline during interruption periods (p < 0.001), followed by recovery upon reinitiation. BMI also increased significantly in both groups (p < 0.05). Patients with baseline ppFEV₁ < 70% showed greater improvement compared to those with milder disease. Conclusion Short-term clinical outcomes in ppFEV₁ and BMI were similar between continuous and intermittent treatment. However, treatment interruptions may reduce cumulative benefits, potentially impacting long-term outcomes. Ensuring uninterrupted access to HEMT is essential, especially in low- and middle-income countries.
Source: Frontiers in Pediatrics
Authors: Almala Pinar Ergenekon, Merve Selcuk, Gokcen Unal, Gamzegul Gozen Bayramoğlu, Cansu Altuntas, Mehmet Kose, Abdurrahman Erdem Basaran, Sinem Can Oksay, Salih Uytun, Sedat Oktem, Zeynep Seda Uyan, Yakup Canitez, Esen Demir, Velat Sen, Veysel Karakulak, Ela Erdem Eralp, Bulent Karadag, Fazilet Karakoc, Sevgi Pekcan, Tugba Sismanlar Eyuboğlu, Erkan Cakır, Yasemin Gokdemir
Institutions: Marmara University, Necmettin Erbakan University, Gazi University, Istinye University, Erciyes University, Akdeniz University, Istanbul Medeniyet University, Ankara Bilkent City Hospital, Istanbul Medipol University, Koç University, Bursa Uludağ Üni̇versi̇tesi̇, Ege University, Dicle University, Cukurova University
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,801
OpenAlex ID: W4415096959