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Cystic Fibrosis: Novel Strategies of Diagnosis and Treatments
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Cystic Fibrosis: Novel Strategies of Diagnosis and Treatments

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

Deadline for manuscript submissions: 31 May 2025 | Viewed by 1434

Special Issue Editors

Dr. Michal Gur

E-Mail Website
Guest Editor
1. Pediatric Pulmonary Institute, CF Center, Rappaport Children’s Hospital, Rambam Health Care Campus, Haifa 3109601, Israel
2. Rappaport Faculty of Medicine, Technion—Israel Institute of Technology, Haifa 3109601, Israel
Interests: cystic fibrosis; lung diseases; pediatric pulmonology
Prof. Dr. Lea Bentur

E-Mail Website1 Website2
Guest Editor
1. Pediatric Pulmonary Institute, CF Center, Rappaport Children’s Hospital, Rambam Health Care Campus, Haifa 3109601, Israel
2. Rappaport Faculty of Medicine, Technion—Israel Institute of Technology, Haifa 3109601, Israel
Interests: pediatric respirology; pediatric pulmonology; cystic fibrosis
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Cystic fibrosis (CF) is the most common fatal genetic disease in Caucasians, with over 2000 known mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR).

The introduction of CFTR modulators (CFTRm), approved by the FDA in 2019, has revolutionized the lives of CF patients, with improved pulmonary functions, nutritional status, and quality of life. However, a challenging group of patients remains those who are not eligible for CFTRm (especially patients with severe stop mutations, which are more prevalent in certain populations).

We are launching a Special Issue entitled “Cystic Fibrosis: Novel Strategies of Diagnosis and Treatments”. The aim of this subject is to highlight the diagnosis and treatment of CF in the era of CFTRm—methods to obtain sputum cultures in patients who do not expectorate sputum after the initiation of CFTRm; effects of CFTRm that have not been studied so far; novel therapies for those ineligible to CFTRm—gene therapy, mRNA therapy; and the diagnosis and treatment of challenging bacteria (such as non-tuberculous mycobacteria—NTM) in patients with and without CFTRm.

These topics will help more deeply understand the new face of CF diagnosis and treatments—major advances in the field versus the challenges that remain.

Dr. Michal Gur
Prof. Dr. Lea Bentur
Guest Editors

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 100 words) can be sent to the Editorial Office for announcement on this website.

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
  • CFTR modulators
  • stop mutations
  • gene therapy
  • mRNA therapy
  • novel therapies
  • rare pathogens

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Published Papers (2 papers)

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Research

10 pages, 863 KiB  
Article
The Association of Achromobacter xylosoxidans Airway Infection with Disease Severity in Cystic Fibrosis
by Ophir Bar-On, Meir Mei-Zahav, Hagit Levine, Huda Mussaffi, Hannah Blau, Haim Ben Zvi, Dario Prais and Patrick Stafler
J. Clin. Med. 2025, 14(7), 2437; https://doi.org/10.3390/jcm14072437 - 3 Apr 2025
Viewed by 273
Abstract
Background/Objectives: The prevalence of Achromobacter xylosoxidans is increasing in people with Cystic Fibrosis (pwCF), yet its clinical pathogenicity remains controversial. The objective of this study was to chart the longitudinal prevalence and examine clinical associations before and after infection. Methods: This [...] Read more.
Background/Objectives: The prevalence of Achromobacter xylosoxidans is increasing in people with Cystic Fibrosis (pwCF), yet its clinical pathogenicity remains controversial. The objective of this study was to chart the longitudinal prevalence and examine clinical associations before and after infection. Methods: This observational, retrospective study was conducted at a single CF center over a 14-year period. Data were collated from patient charts and clinic databases. Patients with Achromobacter sputum cultures were compared to those without the bacterium and analyzed according to whether they had single, intermittent, or chronic infections. Results: During the study period, an annual average of 124 pwCF were followed up at our clinic, with a median age of 13.6 years (IQR = 7.6–27.7). The Achromobacter detection rate increased from 0 to 6.1%. Twenty-three percent (29/124) of patients had at least one positive culture. The median age at acquisition was 17 years (IQR = 14.5–33). At the time of acquisition, the median FEV1 was 81% (IQR = 46–94), compared to 90% (IQR = 72–99) for patients without Achromobacter, p < 0.001. Patients with Achromobacter tended to demonstrate more chronic Pseudomonas (55% vs. 27%, p = 0.06) and pancreatic insufficiency (66% vs. 47%, p = 0.07). At two years post-acquisition, the median FEV1 for patients with intermittent and chronically infected decreased by 11.5% (IQR = −3.75–7.5), compared to 1.5% (IQR = −2.5–12.5) for those with a single positive culture, p = 0.03. Similarly, pulmonary exacerbations per year became more frequent post-acquisition in intermittent and chronically infected patients: Median (range) 2.5 (0–8) pre-, versus 3.0 (0–9) post-acquisition, p = 0.036. Conclusions: Chronic and intermittent infection with Achromobacter were associated with accelerated lung function decline and increased exacerbation frequency. Larger prospective studies are needed to confirm these findings and examine the effect of eradication on the clinical course. Full article
(This article belongs to the Special Issue Cystic Fibrosis: Novel Strategies of Diagnosis and Treatments)
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10 pages, 508 KiB  
Article
A Novel Medical Device for Airway Clearance
by Nir Helper, Moshe Ashkenazi, Gil Sokol, Adi Dagan and Ori Efrati
J. Clin. Med. 2025, 14(3), 907; https://doi.org/10.3390/jcm14030907 - 30 Jan 2025
Viewed by 821
Abstract
Background: Airway clearance techniques are a key element in the daily treatment of people with bronchiectasis. There are several methods and devices to assist in effective airway clearance. We investigated LibAirty, a novel medical device, and compared it with the common practice [...] Read more.
Background: Airway clearance techniques are a key element in the daily treatment of people with bronchiectasis. There are several methods and devices to assist in effective airway clearance. We investigated LibAirty, a novel medical device, and compared it with the common practice performed today. Methods: Twenty adults enrolled, and each one had three different treatments in a randomized order: a human respiratory physiotherapist, a High-Frequency Chest Wall Oscillator, and LibAirty with BiPAP. The outcome parameters were mucus weight and a questionnaire. Further studies were performed to investigate LibAirty with hypertonic saline (HS) inhalation and using the device as a standalone. Results: No adverse events were recorded. The sputum amount expectorated in all arms using LibAirty was 14.4 ± 11.1 g with BIPAP, 16.4 ± 7 g with HS, and 11.3 ± 4.1 g for the standalone treatment. For HFCWO, 4.45 ± 3.28 g was obtained, and for CPT, 15.9 ± 11.1 g was obtained. The amount obtained by LibAirty (all arms) was significantly higher than HFCWO. Conclusions: All arms of LibAirty were superior to HFCWO and similar to the human physiotherapist. Further studies should be performed to investigate the long-term effects of LibAirty. Full article
(This article belongs to the Special Issue Cystic Fibrosis: Novel Strategies of Diagnosis and Treatments)
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