Academic Journal
Gastrointestinal and Pancreatic Manifestations of Cystic Fibrosis in the Era of Highly Effective Modulators.
| Title: | Gastrointestinal and Pancreatic Manifestations of Cystic Fibrosis in the Era of Highly Effective Modulators. |
|---|---|
| Authors: | Abi-Aad SJ; Department of Pediatrics, Division of Gastroenterology, Hepatology and Nutrition, University of South Florida Morsani College of Medicine, 13101 Bruce B. Downs Boulevard, Tampa, FL 33612, USA., Narayanan S; Department of Pediatrics, University of South Florida Morsani College of Medicine, 13101 Bruce B. Downs Boulevard, Tampa, FL 33612, USA., Khalaf RT; Department of Pediatrics, Division of Gastroenterology, Hepatology and Nutrition, University of South Florida Morsani College of Medicine, 13101 Bruce B. Downs Boulevard, Tampa, FL 33612, USA. Electronic address: khalafr@usf.edu. |
| Source: | Advances in pediatrics [Adv Pediatr] 2026 Aug; Vol. 73 (1), pp. 179-190. Date of Electronic Publication: 2026 May 19. |
| Publication Type: | Journal Article; Review |
| Language: | English |
| Journal Info: | Publisher: Mosby Country of Publication: United States NLM ID: 0370436 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-1926 (Electronic) Linking ISSN: 00653101 NLM ISO Abbreviation: Adv Pediatr Subsets: MEDLINE |
| Imprint Name(s): | Publication: Chicago Il : Mosby Original Publication: New York, Interscience [etc.] |
| MeSH Terms: | Cystic Fibrosis*/complications , Cystic Fibrosis*/drug therapy , Cystic Fibrosis*/physiopathology , Gastrointestinal Diseases*/etiology , Gastrointestinal Diseases*/drug therapy , Gastrointestinal Diseases*/physiopathology, Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Quinolones/therapeutic use ; Indoles/therapeutic use ; Benzodioxoles/therapeutic use ; Aminophenols/therapeutic use ; Isoxazoles/therapeutic use ; Pyrrolidines/therapeutic use ; Pyrazoles/therapeutic use ; Quinazolinones/therapeutic use ; Pyridines/therapeutic use ; Chloride Channel Agonists/therapeutic use ; Humans ; Drug Combinations ; Quinolines |
| Abstract: | This review synthesizes emerging evidence on gastrointestinal (GI) disorders, specifically luminal and pancreatic function in the era of triple cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy. Elexacaftor-tezacaftor-ivacaftor has been associated with mild improvements in GI symptoms and disease burden in patients' reported outcomes and has shown a significant increase in growth and body mass index reflecting improved nutritional status, even among individuals who remain pancreatic insufficient. Long-term data on triple CFTR modulators remain limited, highlighting the need to understand their long-term impact on GI manifestations, as survival improves and chronic intestinal inflammation persists. (Copyright © 2025 Elsevier Inc. All rights reserved.) |
| Competing Interests: | Disclosure None. |
| Contributed Indexing: | Keywords: CFTR modulators; Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; Digestive diseases; Motility disorders; Pancreatic insufficiency |
| Substance Nomenclature: | 126880-72-6 (Cystic Fibrosis Transmembrane Conductance Regulator) 0 (Quinolones) 0 (Indoles) 0 (Benzodioxoles) 0 (Aminophenols) 0 (Isoxazoles) 0 (Drug Combinations) 0 (Pyrrolidines) 0 (elexacaftor, ivacaftor, tezacaftor drug combination) 0 (Pyrazoles) 0 (Quinazolinones) 0 (Pyridines) 0 (Chloride Channel Agonists) 0 (Quinolines) |
| Entry Date(s): | Date Created: 20260721 Date Completed: 20260721 Latest Revision: 20260721 |
| Update Code: | 20260722 |
| DOI: | 10.1016/j.yapd.2025.11.001 |
| PMID: | 42481089 |
| Database: | MEDLINE |
| ISSN: | 1878-1926 |
|---|---|
| DOI: | 10.1016/j.yapd.2025.11.001 |