Academic Journal

Impact of Elexacaftor/Tezacaftor/Ivacaftor on Systemic Antibiotic Utilization in Pediatric Patients With Cystic Fibrosis.

Λεπτομέρειες βιβλιογραφικής εγγραφής
Τίτλος: Impact of Elexacaftor/Tezacaftor/Ivacaftor on Systemic Antibiotic Utilization in Pediatric Patients With Cystic Fibrosis.
Συγγραφείς: Fava J; Department of Pharmacy, Nationwide Children's Hospital, Columbus, Ohio, USA., Stephan E; Department of Pharmacy, Nationwide Children's Hospital, Columbus, Ohio, USA., Tansmore J; Department of Pharmacy, Nationwide Children's Hospital, Columbus, Ohio, USA., Sheikh S; Section of Pulmonary Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA., Eisner M; Center for Biostatistics, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.; Biostatistics Resource at Nationwide Children's Hospital, Abigail Wexner Research Institute and Nationwide Children's Hospital, Columbus, OH, USA., Novak KJ; Department of Pharmacy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Πηγή: Pediatric pulmonology [Pediatr Pulmonol] 2026 Aug; Vol. 61 (8), pp. e71749.
Τύπος έκδοσης: Journal Article
Γλώσσα: English
Στοιχεία περιοδικού: Publisher: Wiley-Liss Country of Publication: United States NLM ID: 8510590 Publication Model: Print Cited Medium: Internet ISSN: 1099-0496 (Electronic) Linking ISSN: 10990496 NLM ISO Abbreviation: Pediatr Pulmonol Subsets: MEDLINE
Imprint Name(s): Publication: <2005-> : Hoboken, NJ : Wiley-Liss
Original Publication: [Philadelphia, PA] : W.B. Saunders, [c1985-
Ιατρικοί όροι (MeSH): Cystic Fibrosis*/drug therapy , Cystic Fibrosis*/complications , Anti-Bacterial Agents*/therapeutic use , Indoles*/therapeutic use , Aminophenols*/therapeutic use , Benzodioxoles*/therapeutic use , Quinolones*/therapeutic use , Pyrrolidines*/therapeutic use , Respiratory Tract Infections*/drug therapy , Respiratory Tract Infections*/etiology , Pyridines*/therapeutic use , Pyrazoles*/therapeutic use, Tetrazoles/therapeutic use ; Humans ; Child ; Retrospective Studies ; Female ; Adolescent ; Male ; Drug Combinations ; Quinolines
Περίληψη: Introduction: Cystic fibrosis (CF) is an autosomal recessive disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Recurrent respiratory infections are a common problem, and patients receive frequent antibiotics. Treatment of CF involves both symptomatic and underlying disease-targeting therapies. Elexacaftor/tezacaftor/ivacaftor (ETI), a disease-targeting therapy, combines two correctors and a potentiator to increase functioning of the CFTR channel. With better functioning channels, symptomatic improvement is expected in the form of decreased respiratory infections.
Objective: To evaluate the change in antibiotic use following the initiation of ETI therapy in pediatric patients.
Methods: This is a single-center, retrospective chart review conducted at Nationwide Children's Hospital (NCH) in Columbus, Ohio. Patients served as self-controls, compared before and after initiating ETI. The primary outcome was change in antibiotic days per patient-year. Secondary outcomes included change in use of specific antibiotics, enteral versus intravenous (IV) therapy, and different classes of antibiotics. The study was approved by the NCH institutional review board (IRB).
Results: The study included 139 patients aged 6-18 years. The median antibiotic days per patient-year was 17 days pre-ETI and 6 days post-ETI (p < 0.001). There was a significant decrease in antibiotic days per patient-year for subgroups of IV antibiotics, enteral antibiotics, IV vancomycin, linezolid and tedizolid, anti-methicillin-resistant Staphylococcus aureus (MRSA) tetracyclines, other anti-MRSA medications (clindamycin and trimethoprim/sulfamethoxazole), aminoglycosides, anti-Pseudomonas aeruginosa (Psae) beta-lactams, and fluoroquinolones. There was no significant difference for carbapenems or non-anti-Psae beta-lactams.
Conclusion: ETI therapy was associated with decreased antibiotic days for many therapeutic classes of both enteral and IV antibiotics.
(© 2026 Wiley Periodicals LLC.)
References: “2023 Annual Data Report,” Cystic Fibrosis Foundation (2024), https://www.cff.org/about-us/2023-annual-report.
M. M. Rafeeq and H. A. S. Murad, “Cystic Fibrosis: Current Therapeutic Targets and Future Approaches,” Journal of Translational Medicine 15, no. 1 (2017): 84.
K. De Boeck, “Cystic Fibrosis in the Year 2020: A Disease With a New Face,” Acta Paediatrica 109, no. 5 (2020): 893–899.
T. Ong and B. W. Ramsey, “Cystic Fibrosis: A Review,” Journal of the American Medical Association 329, no. 21 (2023): 1859–1871.
A. Z. Uluer, G. MacGregor, P. Azevedo, et al., “Safety and Efficacy of Vanzacaftor‐Tezacaftor‐Deutivacaftor in Adults With Cystic Fibrosis: Randomised, Double‐Blind, Controlled, Phase 2 Trials,” Lancet Respiratory Medicine 11, no. 6 (2023): 550–562, https://doi.org/10.1016/S2213-2600(22)00504-5.
P. G. Middleton, M. A. Mall, P. Dřevínek, et al., “Elexacaftor‐Tezacaftor‐Ivacaftor for Cystic Fibrosis With a Single Phe508del Allele,” New England Journal of Medicine 381, no. 19 (2019): 1809–1819.
H. G. M. Heijerman, E. F. McKone, D. G. Downey, et al., “Efficacy and Safety of the Elexacaftor Plus Tezacaftor Plus Ivacaftor Combination Regimen in People With Cystic Fibrosis Homozygous for the F508del Mutation: A Double‐Blind, Randomised, Phase 3 Trial,” Lancet 394, no. 10212 (2019): 1940–1948.
E. T. Zemanick, J. L. Taylor‐Cousar, J. Davies, et al., “A Phase 3 Open‐Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age With Cystic Fibrosis and at Least One F508del Allele,” American Journal of Respiratory and Critical Care Medicine 203, no. 12 (2021): 1522–1532.
J. L. Goralski, J. E. Hoppe, M. A. Mall, et al., “Phase 3 Open‐Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2‐5 Years With Cystic Fibrosis and at Least One F508del Allele,” American Journal of Respiratory and Critical Care Medicine 208, no. 1 (2023): 59–67.
“Trikafta Package Insert,” Vertex 2019, https://pi.vrtx.com/files/uspi&#95;elexacaftor&#95;tezacaftor&#95;ivacaftor.pdf.
G. Livnat, A. Dagan, M. Heching, et al., “Treatment Effects of Elexacaftor/Tezacaftor/Ivacaftor in People With CF Carrying non‐F508del Mutations,” Journal of Cystic Fibrosis 22, no. 3 (2023): 450–455.
A. C. Miller, L. M. Harris, J. E. Cavanaugh, et al., “The Rapid Reduction of Infection‐Related Visits and Antibiotic Use Among People With Cystic Fibrosis After Starting Elexacaftor‐Tezacaftor‐Ivacaftor,” Clinical Infectious Diseases 75, no. 7 (2022): 1115–1122.
E. Walter and J. L. Bass, “The Effect of Elexacaftor/Tezacaftor/Ivacaftor on Hospitalizations and Intravenous Antibiotic Use,” Permanente Journal 26, no. 1 (2022): 73–79.
C. L. Williams, J. Billings, H. McGowan, et al., “Impact of Elexacaftor/Tezacaftor/Ivacaftor on Microbiology and Antibiotic Utilization in People With Cystic Fibrosis,” Pediatric Pulmonology 60, no. 3 (2025): e71038.
C. Gushue, M. Eisner, S. Bai, et al., “Impact of Elexacaftor‐Tezacaftor‐Ivacaftor on Lung Disease in Cystic Fibrosis,” Pediatric Pulmonology 58, no. 8 (2023): 2308–2316.
E. L. Guenther, K. S. McCoy, M. Eisner, et al., “Impact of Chronic Medication De‐Escalation in Patients With Cystic Fibrosis Taking Elexacaftor, Tezacaftor, Ivacaftor: A Retrospective Review,” Journal of Cystic Fibrosis 23, no. 1 (2024): 32–37.
E. M. Byerley, K. S. McCoy, M. Eisner, T. Johnson, K. J. Novak, and E. M. Stephan, “De‐Escalation of Medications After Initiation of Elexacaftor‐Tezacaftor‐Ivacaftor in Children 6 to 11 Years of Age With Cystic Fibrosis,” Journal of Pediatric Pharmacology and Therapeutics, ahead of print, May 15, 2026, https://doi.org/10.5863/JPPT-25-00051.
D. Salvatore, P. Iacotucci, A. Pepe, et al., “Real‐World Effectiveness and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients With Phe508del ‐Gating and ‐Residual Function Genotypes,” Respiratory Medicine 245 (2025): 108221, https://doi.org/10.1016/j.rmed.
“2022 Annual Data Report,” Cystic Fibrosis Foundation (2023), https://www.cff.org/about-us/2022-annual-report.
A. C. Blanchard and V. J. Waters, “Microbiology of Cystic Fibrosis Airway Disease,” Seminars in Respiratory and Critical Care Medicine 40, no. 6 (2019): 727–736.
D. Keating, G. Marigowda, L. Burr, et al., “VX‐445‐Tezacaftor‐Ivacaftor in Patients With Cystic Fibrosis and One or Two Phe508del Alleles,” New England Journal of Medicine 379, no. 17 (2018): 1612–1620.
D. Appelt, G. Steinkamp, S. Sieber, and H. Ellemunter, “Early and Sustained Improvements of Lung Clearance Index From Two to Sixteen Weeks of Elexacaftor/Tezacaftor/Ivacaftor Therapy in Patients With Cystic Fibrosis‐A Real World Study,” Frontiers in Pharmacology 14 (2023): 1125853.
Grant Information: UL1TR002733 The Ohio State University Center for Clinical and Translational Science (National Center for Advancing Translational Sciences); MCCOY19RO Cystic Fibrosis Foundation (Research Development Program); Cure CF Columbus Translational Core (C3TC) supported by the Division of Pediatric Pulmonary Medicine, the Biopathology Center Core, and the Data Collaboration Team at Nationwide Children's Hospital
Contributed Indexing: Keywords: antibiotics; cystic fibrosis; elexacaftor/tezacaftor/ivacaftor; infection; respiratory
Substance Nomenclature: 0 (Anti-Bacterial Agents)
0 (Indoles)
0 (Aminophenols)
0 (Benzodioxoles)
0 (Drug Combinations)
0 (Quinolones)
0 (Pyrrolidines)
0 (Pyridines)
0 (elexacaftor, ivacaftor, tezacaftor drug combination)
0 (Pyrazoles)
0 (Tetrazoles)
8RW88Y506K (tezacaftor)
0 (Quinolines)
Entry Date(s): Date Created: 20260728 Date Completed: 20260729 Latest Revision: 20260730
Update Code: 20260730
PubMed Central ID: PMC13411607
DOI: 10.1002/ppul.71749
PMID: 42517727
Βάση Δεδομένων: MEDLINE
Περιγραφή
ISSN:1099-0496
DOI:10.1002/ppul.71749