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The effect of elexacaftor-tezacaftor-ivacaftor on liver stiffness in children with cystic fibrosis

Diemer, Stefanie LU ; Elidottir, Helga LU orcid ; Eklund, Erik LU ; Påhlman, Lisa LU and Rönne Hansen, Christine LU (2025) In Journal of Pediatric Gastroenterology and Nutrition
Abstract
Objectives: Cystic fibrosis hepato-biliary involvement (CFHBI) is a common comorbidity in patients with CF and is associated with increased morbidity and mortality. The effect of the new and highly potent CF transmembrane conductance regulator modulator therapy, elexacaftor-tezacaftor-ivacaftor (ETI), on CFHBI, is still unclear. This study aimed to investigate the impact of ETI on liver stiffness in children with CF, as measured using two-dimensional (2D) shear wave elastography (SWE).

Methods: Twenty-one children with CF were included in this retrospective study at the CF centre, Skåne University Hospital, Lund, Sweden. Twelve children of our cohort had CFHBI; none had advanced CF liver disease. 2D SWE data from annual... (More)
Objectives: Cystic fibrosis hepato-biliary involvement (CFHBI) is a common comorbidity in patients with CF and is associated with increased morbidity and mortality. The effect of the new and highly potent CF transmembrane conductance regulator modulator therapy, elexacaftor-tezacaftor-ivacaftor (ETI), on CFHBI, is still unclear. This study aimed to investigate the impact of ETI on liver stiffness in children with CF, as measured using two-dimensional (2D) shear wave elastography (SWE).

Methods: Twenty-one children with CF were included in this retrospective study at the CF centre, Skåne University Hospital, Lund, Sweden. Twelve children of our cohort had CFHBI; none had advanced CF liver disease. 2D SWE data from annual assessments, clinical data and liver enzymes were analysed.

Results: We found a significant reduction in liver stiffness after starting treatment with ETI in the total cohort. This reduction in liver stiffness could even be seen in children with CFHBI. Liver enzymes were within the normal range in both pre- and post-ETI therapy in the total cohort. In children with CFHBI, a decline in aspartate aminotransferase activity was observed after ETI was initiated. Lung function and lung clearance index improved significantly after ETI treatment commenced.

Conclusion: ETI treatment could positively affect CFHBI in children with CF, as demonstrated by reduced liver stiffness during treatment. (Less)
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author
; ; ; and
organization
publishing date
type
Contribution to journal
publication status
epub
subject
in
Journal of Pediatric Gastroenterology and Nutrition
publisher
Lippincott Williams & Wilkins
external identifiers
  • pmid:40264362
  • scopus:105003119527
ISSN
1536-4801
DOI
10.1002/jpn3.70050
language
English
LU publication?
yes
id
09ae4550-8b0f-42d8-bbc6-98e8d45c0a54
date added to LUP
2025-05-01 10:57:12
date last changed
2025-05-05 08:28:45
@article{09ae4550-8b0f-42d8-bbc6-98e8d45c0a54,
  abstract     = {{Objectives: Cystic fibrosis hepato-biliary involvement (CFHBI) is a common comorbidity in patients with CF and is associated with increased morbidity and mortality. The effect of the new and highly potent CF transmembrane conductance regulator modulator therapy, elexacaftor-tezacaftor-ivacaftor (ETI), on CFHBI, is still unclear. This study aimed to investigate the impact of ETI on liver stiffness in children with CF, as measured using two-dimensional (2D) shear wave elastography (SWE).<br/><br/>Methods: Twenty-one children with CF were included in this retrospective study at the CF centre, Skåne University Hospital, Lund, Sweden. Twelve children of our cohort had CFHBI; none had advanced CF liver disease. 2D SWE data from annual assessments, clinical data and liver enzymes were analysed.<br/><br/>Results: We found a significant reduction in liver stiffness after starting treatment with ETI in the total cohort. This reduction in liver stiffness could even be seen in children with CFHBI. Liver enzymes were within the normal range in both pre- and post-ETI therapy in the total cohort. In children with CFHBI, a decline in aspartate aminotransferase activity was observed after ETI was initiated. Lung function and lung clearance index improved significantly after ETI treatment commenced.<br/><br/>Conclusion: ETI treatment could positively affect CFHBI in children with CF, as demonstrated by reduced liver stiffness during treatment.}},
  author       = {{Diemer, Stefanie and Elidottir, Helga and Eklund, Erik and Påhlman, Lisa and Rönne Hansen, Christine}},
  issn         = {{1536-4801}},
  language     = {{eng}},
  month        = {{04}},
  publisher    = {{Lippincott Williams & Wilkins}},
  series       = {{Journal of Pediatric Gastroenterology and Nutrition}},
  title        = {{The effect of elexacaftor-tezacaftor-ivacaftor on liver stiffness in children with cystic fibrosis}},
  url          = {{http://dx.doi.org/10.1002/jpn3.70050}},
  doi          = {{10.1002/jpn3.70050}},
  year         = {{2025}},
}