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Cystic Fibrosis: Pathophysiology, Complications and Therapy

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

Deadline for manuscript submissions: closed (20 May 2022) | Viewed by 8386

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Dr. Meir Mei-Zahav

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Guest Editor

1. Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
2. Schneider Children’s Medical Center of Israel, Tel Aviv, Israel
3. Pulmonary Institute, Tel Aviv, Israel
Interests: cystic fibrosis; pediatric pulmonary medicine; pediatrics

Special Issue Information

Dear Colleagues,

Cystic fibrosis (CF), the most common genetic life-shortening disease, results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsible for the formation of an epithelial chloride channel. These mutations lead to the dysfunction of the chloride channel, causing chronic infections and inflammation in the airways and a loss of exocrine pancreatic function. Most patients die at an early age from chronic progressive lung disease. Until recently, the only potentially curative treatment was lung transplantation; however, the emergence of new therapies such as CFTR modulators has led to major changes in CF prognosis and will potentially change the outcomes for most CF patients.

In this issue of the journal, we will address several aspects of CF. We will discuss the pathophysiology of the disease and its influence on the clinical manifestations of CF. CF lung disease is a major cause of morbidity and mortality in CF, and clinicians are struggling to find outcome measures for assessing lung disease severity and parameters for predicting pulmonary deterioration. We will present methods for assessing lung disease severity and discuss other CF complications, new CF treatments and the future of CF care.

Dr. Meir Mei-Zahav
Guest Editor

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Keywords

CF care
CF lung disease assessment
CF therapy
CF epidemiology
CF genetics
CF pathophysiology.

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Published Papers (4 papers)

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Research

11 pages, 2134 KiB

Open AccessArticle

Dynamic Hyperinflation While Exercising—A Potential Predictor of Pulmonary Deterioration in Cystic Fibrosis

by Einat Shmueli, Yulia Gendler, Patrick Stafler, Hagit Levine, Guy Steuer, Ophir Bar-On, Hannah Blau, Dario Prais and Meir Mei-Zahav

J. Clin. Med. 2023, 12(18), 5834; https://doi.org/10.3390/jcm12185834 - 8 Sep 2023

Cited by 1 | Viewed by 1046

Abstract

Background: Lung function deterioration in cystic fibrosis (CF) is typically measured by a decline in the forced expiratory volume in one second (FEV₁%), which is thought to be a late marker of lung disease. Dynamic hyperinflation (DH) is seen in obstructive lung diseases while exercising. Our aim was to assess whether DH could predict pulmonary deterioration in CF; a secondary measure was the peak VO₂. Methods: A retrospective study was conducted of people with CF who performed cardiopulmonary exercise tests (CPETs) during 2012–2018. The tests were classified as those demonstrating DH non-DH. Demographic, genetic, and clinical data until 12.2022 were extracted from patient charts. Results: A total of 33 patients aged 10–61 years performed 41 valid CPETs with valid DH measurements; sixteen (39%) demonstrated DH. At the time of the CPETs, there was no difference in the FEV₁% measurements between the DH and non-DH groups (median 83.5% vs. 87.6%, respectively; p = 0.174). The FEV₁% trend over 4 years showed a decline in the DH group compared to the non-DH group (p = 0.009). A correlation was found between DH and the lung clearance index (LCI), as well as the FEV₁% (r = 0.36 and p = 0.019 and r = −0.55 and p = 0.004, respectively). Intravenous (IV) antibiotic courses during the 4 years after the CPETs were significantly more frequent in the DH group (p = 0.046). The peak VO₂ also correlated with the FEV₁% and LCI (r = 0.36 and p = 0.02 and r = −0.46 and p = 0.014, respectively) as well as with the IV antibiotic courses (r = −0.46 and p = 0.014). Conclusions: In our cohort, the DH and peak VO₂ were both associated with lung function deterioration and more frequent pulmonary exacerbations. DH may serve as a marker to predict pulmonary deterioration in people with CF. Full article

(This article belongs to the Special Issue Cystic Fibrosis: Pathophysiology, Complications and Therapy)

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11 pages, 1386 KiB

Open AccessArticle

Lung Clearance Index in Children with Cystic Fibrosis during Pulmonary Exacerbation

by Katarzyna Walicka-Serzysko, Magdalena Postek, Justyna Milczewska and Dorota Sands

J. Clin. Med. 2021, 10(21), 4884; https://doi.org/10.3390/jcm10214884 - 23 Oct 2021

Cited by 13 | Viewed by 1844

Abstract

(1) Background: Pulmonary exacerbation (PEx) is one of the main factors affecting the quality of life and life expectancy in patients with cystic fibrosis (CF). Our study aimed to evaluate the change in selected pulmonary function parameters, including lung clearance index (LCI), in patients with CF diagnosed with PEx. (2) Methods: We enrolled 40 children with CF aged 6–17. They performed spirometry and multiple breath nitrogen washout (MBNW) tests during a stable condition period at the beginning and the end of intravenous antibiotic treatment. (3) Results: LCI increased by 65% and FEV₁ decreased by ≥10% in 40% of patients with CF during PEx. An absolute change in LCI between a stable condition period and PEx was 1.05 (±1.92) units, which corresponds to a relative change of 11.48% (±18.61) of the baseline. The relative decrease in FEV₁ was −9.22% (±12.00) and the z-score was −0.67 (±1.13). After the PEx treatment, FEV₁ increased by 11.05% (±9.04) on average, whereas LCI decreased by 1.21 ± 1.59 units on average, which represented 9.42% ± 11.40 compared to the value at the beginning of PEx. (4) Conclusions: The change in LCI captures a higher proportion of events with functional impairment than FEV₁ in school-age children with CF. Full article

(This article belongs to the Special Issue Cystic Fibrosis: Pathophysiology, Complications and Therapy)

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7 pages, 377 KiB

Open AccessArticle

The Association between IgG and Disease Severity Parameters in CF Patients

by Michal Gur, Yael Ben-David, Moneera Hanna, Anat Ilivitzki, Adi Weichhendler, Ronen Bar-Yoseph, Yazeed Toukan, Kamal Masarweh and Lea Bentur

J. Clin. Med. 2021, 10(15), 3316; https://doi.org/10.3390/jcm10153316 - 28 Jul 2021

Cited by 1 | Viewed by 1885

Abstract

Assessing disease severity in patients with cystic fibrosis (CF) is essential when directing therapies. Serum immunoglobulin G (IgG) levels increase with disease severity. Lung clearance index (LCI) is recognized as an outcome measure for CF clinical trials. Our aim was to evaluate the correlations between IgG and disease severity markers. This was a single-center retrospective study, evaluating association between IgG and markers of severity in CF patients (including clinical characteristics, lung spirometry, LCI, clinical scores and computed tomography (CT) scores) during stable conditions. There were 69 patients, age 20.5 ± 11.6 years. Nineteen (27.5%) patients had elevated IgG. IgG correlated positively with LCI (r = 0.342, p = 0.005). IgG was higher in pancreatic insufficient (PI) and patients with liver disease (1504.3 ± 625.5 vs. 1229 ± 276.1 mg/dL in PI vs. PS, p = 0.023, and 1702.6 ± 720.3 vs. 1256.2 ± 345.5 mg/dL with vs. without liver disease, p = 0.001, respectively). IgG also correlated positively with CRP, CT score, and days with antibiotics in the previous year (r = 0.38, p = 0.003; r = 0.435, p = 0.001; and r = 0.361, p = 0.002, respectively), and negatively with FEV1% and SK score (r = −0.527, p < 0.001 and r = −0.613, p < 0.001, respectively). IgG correlated with clinical parameters, pulmonary functions, and imaging. However, this is still an auxiliary test, complementing other tests, including lung function and imaging tests. Larger multi-center longitudinal studies are warranted. Full article

(This article belongs to the Special Issue Cystic Fibrosis: Pathophysiology, Complications and Therapy)

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8 pages, 513 KiB

Open AccessArticle

Computed Tomographic Changes in Patients with Cystic Fibrosis Treated by Combination Therapy with Lumacaftor and Ivacaftor

by François Arnaud, Nathalie Stremler-Le Bel, Martine Reynaud-Gaubert, Julien Mancini, Jean-Yves Gaubert and Guillaume Gorincour

J. Clin. Med. 2021, 10(9), 1999; https://doi.org/10.3390/jcm10091999 - 7 May 2021

Cited by 13 | Viewed by 2532

Abstract

Background: As Cystic Fibrosis (CF) treatments drastically improved in recent years, tools to assess their efficiency need to be properly evaluated, especially cross-sectional imaging techniques. High-resolution computed tomography (HRCT) scan response to combined lumacaftor- ivacaftor therapy (Orkambi^®) in patients with homozygous for F508del CFTR has not yet been assessed. Methods: We conducted a retrospective observational study in two French reference centers in CF in Marseille hospitals, including teenagers (>12 years old) and adults (>18 years) who had received lumacaftor–ivacaftor and for whom we had at disposal at least two CT scans, one at before therapy and one at least six months after therapy start. CT scoring was performed by using the modified version of the Brody score. Results: 34 patients have been included. The mean age was 26 years (12–56 years). There was a significant decrease in the total CT score (65.5 to 60.3, p = 0.049) and mucous plugging subscore (12.3 to 8.7, p = 0.009). Peribronchial wall thickening (PWT) was significantly improved only in the adult group (29.1 to 27.0, p = 0.04). Improvements in total score, peribronchial thickening, and mucous pluggings were significantly correlated with improvement in FEV1 (forced expiratory volume in 1 s). Conclusions: Treatment with lumacaftor–ivacaftor was associated with a significant improvement in the total CT score, which was mainly related to an improvement in mucous pluggings. Full article

(This article belongs to the Special Issue Cystic Fibrosis: Pathophysiology, Complications and Therapy)

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