New Developments in Pediatric Cystic Fibrosis Therapy: Current Trends and Future Possibilities

A special issue of Children (ISSN 2227-9067).

Deadline for manuscript submissions: closed (25 June 2025) | Viewed by 234

Special Issue Editors


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Guest Editor
Cystic Fibrosis Unit, Hospital Universitario Central de Asturias, Oviedo, Spain
Interests: cystic fibrosis; nutrition; gastroenterology

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Guest Editor
Servicio de Gastroenterología y Nutrición Pediátrica, Hospital Infantil Miguel Servet, Zaragoza, Spain
Interests: cystic fibrosis

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Guest Editor Assistant
Hospital Universitario Virgen del Rocío, Sevilla, Spain
Interests: cystic fibrosis; nutrition; inflammatory bowel disease; gastroenterology

Special Issue Information

Dear Colleagues,

CFTR modulators have revolutionized the treatment of patients with cystic fibrosis, significantly improving their survival and quality of life. The early initiation of treatment in childhood with highly effective modulators opens a window of opportunity to improve patient prospects beyond the lung.

This Special Issue is interested in research articles and literature reviews that discuss the safety and efficacy of treatment with modulators in the pediatric age group. We wish to address the multisystemic nature of this disease; these research areas may include (but are not limited to) the following topics:

  • Pulmonary topics: lung function, functional and radiological tests, etc.;
  • Digestive topics: pancreatic insufficiency, acute pancreatitis, cystic fibrosis liver disease, gut inflammation and microbiota, motility disorders, etc.;
  • Nutrition topics: body composition, fat-soluble vitamins, risk of obesity, bone health, etc.;
  • Endocrine topics: cystic fibrosis-related diabetes, metabolic syndrome, etc.;
  • Mental health and quality of life topics: depression, anxiety, etc.;
  • Renal topics: lithiasis, renal failure, etc.

Dr. David González Jiménez
Dr. Ruth García-Romero
Guest Editors

Dr. Alejandro Rodríguez Martínez
Guest Editor Assistant

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Keywords

  • cystic fibrosis
  • cystic fibrosis modulators
  • lung function
  • sweat test
  • nutrition and growth
  • cystic fibrosis-related diabetes
  • cystic fibrosis-related liver disease

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Published Papers (1 paper)

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Review

13 pages, 261 KiB  
Review
Treatment with CFTR Modulators for Cystic Fibrosis: What aPediatric Gastroenterologist Needs to Know
by David Gonzalez Jimenez, Ruth García Romero, Alejandro Rodríguez Martínez and Saioa Vicente Santamaria
Children 2025, 12(9), 1104; https://doi.org/10.3390/children12091104 - 22 Aug 2025
Abstract
Background: Cystic fibrosis (CF) is a multisystemic disorder caused by CFTR gene mutations, leading to impaired protein function and affecting pulmonary, gastrointestinal, hepatobiliary, skeletal, and nutritional health. The advent of CFTR modulators—especially the triple therapy elexacaftor/tezacaftor/ivacaftor (ETI)—has revolutionized clinical management, offering genotype-specific benefits [...] Read more.
Background: Cystic fibrosis (CF) is a multisystemic disorder caused by CFTR gene mutations, leading to impaired protein function and affecting pulmonary, gastrointestinal, hepatobiliary, skeletal, and nutritional health. The advent of CFTR modulators—especially the triple therapy elexacaftor/tezacaftor/ivacaftor (ETI)—has revolutionized clinical management, offering genotype-specific benefits beyond pulmonary outcomes. Pediatric gastroenterologists must now recognize and address emerging gastrointestinal and nutritional challenges introduced by modulator therapy. Methods: A narrative review was conducted to assess the impact of CFTR modulators on gastrointestinal function, nutritional status, bone health, and hepatobiliary involvement in pediatric patients. A structured literature search was performed using PubMed, EMBASE, and Scopus databases. Filters included articles in English or Spanish. Following full-text review based on relevance and quality, 68 articles were selected for inclusion in this review. Results: CFTR modulators have demonstrated potential improvements in gastrointestinal function, nutrient absorption, weight gain, and bone mineral density. In pediatric populations, ETI therapy has been associated with early increases in lean mass, enhanced vitamin levels, and promising trends in bone microarchitecture. However, variable outcomes regarding liver function and bone mineral density highlight the need for careful monitoring. Conclusions: While CFTR modulators present novel opportunities for systemic improvement in CF, their long-term impact on digestive and skeletal health in children remains under investigation. Pediatric gastroenterologists play a pivotal role in monitoring nutritional and hepatobiliary outcomes, optimizing treatment plans, and guiding personalized care strategies in the era of CFTR modulation. Full article
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