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Cystic Fibrosis: Management Strategies and Patient Outcomes
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Cystic Fibrosis: Management Strategies and Patient Outcomes

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Respiratory Medicine".

Deadline for manuscript submissions: 25 June 2026 | Viewed by 1258

Special Issue Editor

Prof. Dr. Marta Rachel

E-Mail Website
Guest Editor
Institute of Medical Sciences, Medical College of Rzeszow University, 35-310 Rzeszow, Poland
Interests: asthma; cystic fibrosis; quality of life; patient outcomes

Special Issue Information

Dear Colleagues,

Cystic fibrosis (CF) is a complex, multisystem genetic disorder that requires lifelong, multidisciplinary care. In recent years, advances in diagnostics, personalized medicine, and targeted therapies have significantly improved patient outcomes and life expectancy. This Special Issue aims to explore contemporary strategies in CF management, including pharmacological innovations, nutritional support, physiotherapy, and psychological interventions. We welcome contributions that address both pediatric and adult populations, reducing complications and enhancing quality of life. Additionally, we encourage submissions that focus on patient-centered approaches and the impact of emerging technologies such as telemedicine and AI in CF care. By bringing together diverse perspectives, this issue seeks to provide a comprehensive overview of current practices and future directions in CF management.

Prof. Dr. Marta Rachel
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • cystic fibrosis
  • multidisciplinary care
  • personalized medicine
  • patient outcomes
  • telemedicine
  • pediatric pulmonology
  • CFTR modulators
  • chronic disease management

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Published Papers (1 paper)

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Research

20 pages, 650 KB  
Article
Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis: A 24-Month Italian National Registry Study
by Donatello Salvatore, Giuseppe Campagna, Rita Padoan, Angela Pepe, Annalisa Amato and Marco Salvatore
J. Clin. Med. 2026, 15(7), 2699; https://doi.org/10.3390/jcm15072699 - 2 Apr 2026
Viewed by 1042
Abstract
Background: The CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) has transformed cystic fibrosis (CF) care, but national-level real-world data on long-term effectiveness, durability of response, and treatment de-escalation remain limited. Methods: We conducted a nationwide longitudinal study using the Italian Cystic Fibrosis Registry. People with CF [...] Read more.
Background: The CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) has transformed cystic fibrosis (CF) care, but national-level real-world data on long-term effectiveness, durability of response, and treatment de-escalation remain limited. Methods: We conducted a nationwide longitudinal study using the Italian Cystic Fibrosis Registry. People with CF aged ≥6 years who initiated ETI between October 2019 and December 2022 and received ≥3 months of continuous therapy were included. Lung function (percent predicted FEV1, ppFEV1), nutritional status (BMI or BMI z-score), hospital days, complications, microbiology, and chronic treatments were assessed during the two years before and up to two years after ETI initiation. Longitudinal changes were analyzed using generalized estimating equations with multiple imputation for missing data. Results: The cohort included 2276 individuals (mean age 27.9 ± 13.3 years; 49% female). Mean ppFEV1 declined during the pre-ETI period but increased by 9.9 percentage points at 12 months after ETI initiation (p < 0.001) and remained 6.8 percentage points above baseline at 24 months. A decline between 12 and 24 months was observed overall, except in individuals with severe baseline lung disease (ppFEV1 < 40%), who maintained stable improvements. Mean annual hospital days decreased by approximately 65% and remained low throughout follow-up. Nutritional status improved, with a mean BMI increase of approximately 1.05 kg/m2 compared with immediate pre-treatment in adults and a BMI z-score increase of 0.2 SD compared with pre-treatment timepoints in children. Use of most standard CF therapies declined substantially, particularly among individuals with ppFEV1 ≥ 40%. The prevalence of allergic bronchopulmonary aspergillosis decreased, while liver disease prevalence increased modestly, largely reflecting transient elevations in liver enzymes. Conclusions: In this nationwide real-world cohort, ETI was associated with sustained improvements in lung function, nutritional status, and hospitalization burden. The attenuation of lung function gains after the first year, particularly in less severe disease, supports the need for individualized monitoring and cautious treatment de-escalation in the ETI era. Full article
(This article belongs to the Special Issue Cystic Fibrosis: Management Strategies and Patient Outcomes)
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