Current Updates on Pediatric Cystic Fibrosis Care and Outcomes

A special issue of Children (ISSN 2227-9067). This special issue belongs to the section "Pediatric Pulmonary and Sleep Medicine".

Deadline for manuscript submissions: closed (20 November 2022) | Viewed by 2458

Special Issue Editors


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Guest Editor
Division of Pediatric Pulmonology, First Department of Pediatrics, National and Kapodistrian University of Athens School of Medicine and Aghia Sophia Children's Hospital, Thivon and Papadiamantopoulou St., 115.27 Athens, Greece
Interests: sleep apnea; sleep-disordered breathing; cystic fibrosis; bronchiolitis; spirometry

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Co-Guest Editor
Cystic Fibrosis Department, Agia Sofia Children’s Hospital Thivon and Papadiamantopoulou St., 115.27 Athens, Greece
Interests: pediatric pulmonology; cystic fibrosis; bronchiolitis; spirometry; lung diseases; respiratory physiology

Special Issue Information

Dear Colleagues,

Tremendous progress has been achieved in the diagnosis and management of cystic fibrosis (CF) over the last few years. Highly effective CFTR modulators have become available, “adding tomorrows” to the lives of people with CF. Patients who are not eligible for modulators may benefit from the development of new CFTR function restoring therapies. Newborn screening and early diagnosis of the disease in the newborn period remains an area of intensive research. Lung infections and inflammation, CF-related diabetes, nutrition, hepatic and gastrointestinal disorders, and lung transplantation for advanced lung disease are additional exciting areas of CF research. The purpose of this Special Issue is to accommodate review articles describing the path to a cure for every individual with CF. We hope that contributors will cover as many topics as possible, reviewing the extensive body of published and ongoing research.

Dr. Athanasios Kaditis
Dr. Argyri Petrocheilou
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. Children is an international peer-reviewed open access monthly 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 2400 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

  • advanced lung disease
  • CFTR modulators
  • cystic fibrosis
  • cystic fibrosis related diabetes
  • lung transplantation
  • newborn screening
  • pulmonary exacerbations

Published Papers (1 paper)

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Research

11 pages, 722 KiB  
Communication
Nutritional Status and Circulating Levels of Fat-Soluble Vitamins in Cystic Fibrosis Patients: A Cohort Study and Evaluation of the Effect of CFTR Modulators
by Michela Francalanci, Vito Terlizzi, Cristina Fevola, Giulia Di Rosa, Valentina Pierattini, Elena Roselli, Paolo Bonomi, Maria Chiara Cavicchi, Valeria Galici, Anna Silvia Neri, Chiara Bianchimani, Silvia Campana, Daniela Dolce, Novella Ravenni, Erica Camera, Tommaso Orioli and Giovanni Taccetti
Children 2023, 10(2), 252; https://doi.org/10.3390/children10020252 - 30 Jan 2023
Cited by 9 | Viewed by 2006
Abstract
Background: Improved therapy in CF has led to an overall improvement in nutritional status. The objectives of our study are: to cross-sectionally assess nutritional status and serum levels of fat-soluble vitamins; to retrospectively evaluate the efficacy of modulators on nutritional status and fat-soluble [...] Read more.
Background: Improved therapy in CF has led to an overall improvement in nutritional status. The objectives of our study are: to cross-sectionally assess nutritional status and serum levels of fat-soluble vitamins; to retrospectively evaluate the efficacy of modulators on nutritional status and fat-soluble vitamin levels. Methods: In patients younger than 2 years of age, we evaluated growth, in patients aged 2–18 years, we assessed BMI z-scores, and in adults, we assessed absolute BMI values. Levels of 25(OH)D, vitamins A, and E were measured. Results: A cross-sectional analysis was conducted on 318 patients, 109 (34.3%) with pancreatic sufficiency. Only three patients were under 2 years old. In 135 patients aged 2–18 years, the median BMI z-score was 0.11, and 5 (3.7%) patients had malnutrition (z-score ≤ 2SD). In 180 adults, the median BMI was 21.8 kg/m2. Overall, 15 (13.7%) males (M) and 18 (25.3%) females (F) were underweight (18 < BMI > 20); 3 (2.7%) M and 5 (7.0%) F had a BMI < 18. Suboptimal 25(OH)D levels were found in patients with pancreatic insufficiency. The prevalence of deficiency of vitamins A and E is low. After one year of treatment with modulators, the increase in BMI was more consistent (M: 1.58 ± 1.25 kg/m2 F: 1.77 ± 1.21 kg/m2) in elexacaftor/tezacaftor/ivacaftor (ETI)-treated patients compared with other modulators, with a significant increase in levels of all fat-soluble vitamins. Conclusions: Malnutrition is present in a limited number of subjects. The prevalence of subjects with suboptimal 25(OH)D levels is high. ETI showed a beneficial effect on nutritional status and circulating levels of fat-soluble vitamins. Full article
(This article belongs to the Special Issue Current Updates on Pediatric Cystic Fibrosis Care and Outcomes)
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