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Respiratory Outcomes of Interrupted Modulator Therapies in Children with Cystic Fibrosis
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  • Berrak Oztosun,
  • Azer Kilic Baskan,
  • Huseyin Arslan,
  • Cigdem Korkmaz,
  • Albulhamit Collak,
  • Haluk Cokugras,
  • Ayse Ayzit Kilinc
Berrak Oztosun
Istanbul Universitesi-Cerrahpasa

Corresponding Author:[email protected]

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Azer Kilic Baskan
Istanbul Universitesi-Cerrahpasa
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Huseyin Arslan
Istanbul Universitesi-Cerrahpasa
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Cigdem Korkmaz
Istanbul Universitesi-Cerrahpasa
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Albulhamit Collak
Istanbul Universitesi-Cerrahpasa
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Haluk Cokugras
Istanbul Universitesi-Cerrahpasa
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Ayse Ayzit Kilinc
Istanbul Universitesi-Cerrahpasa
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Abstract

Background Cystic fibrosis (CF) is a multisystemic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene, resulting in defective synthesis or function of the CFTR protein. Historically, CF treatment focused on managing symptoms and complications. Fortunately, modulator drugs are now available to directly target the defective CFTR protein. However, in some countries, such as Turkey, these drugs are not covered by social insurance. Consequently, many CF patients face barriers to accessing modulatory therapies or must interrupt their treatment. This study demonstrates the impact of interrupting modulator therapy on pulmonary function, emphasizing the need for uninterrupted continuous treatment. Methods In this study, 39 CF patients receiving elexacaftor-tezacaftor-ivacaftor (ETI) at our clinic were retrospectively analyzed. Among the patients, 18 experienced one or more interruptions, ranging from 15 to 210 days during ETI treatment. We analyzed pulmonary function test results from 27 interruption periods. Results At the beginning of the interruption, the mean percent predicted FEV1 (ppFEV1) was 69.59 ± 25.87%, which decreased to 64.96 ± 24.52% by the end of the interruption. There was a significant decrease with a mean change of 4.62 ± 8.49 (p = 0.008). However, no significant correlation was found between the interruption duration and FEV1 change. Conclusion Our results demonstrate that pulmonary functions are adversely affected by interruption periods, regardless of their duration. Even short interruptions have a significant impact on pulmonary functions. This underscores the need for uninterrupted continuation of modulatory treatment and for improved policies to ensure equitable access to treatment.
04 Aug 2024Submitted to Pediatric Pulmonology
05 Aug 2024Submission Checks Completed
05 Aug 2024Assigned to Editor
05 Aug 2024Review(s) Completed, Editorial Evaluation Pending
04 Sep 2024Reviewer(s) Assigned
01 Oct 2024Editorial Decision: Revise Minor
03 Oct 20241st Revision Received
05 Oct 2024Review(s) Completed, Editorial Evaluation Pending
05 Oct 2024Submission Checks Completed
05 Oct 2024Assigned to Editor
05 Oct 2024Reviewer(s) Assigned
27 Oct 2024Editorial Decision: Accept