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

IF 2.6 3区医学 Q3 GASTROENTEROLOGY & HEPATOLOGY

Journal of Pediatric Gastroenterology and Nutrition Pub Date : 2025-07-01 Epub Date: 2025-04-23 DOI:10.1002/jpn3.70050

Stefanie Diemer, Helga Elidottir, Erik A Eklund, Lisa I Påhlman, Christine Hansen

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引用次数: 0

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 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.

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松解因子对囊性纤维化患儿肝僵硬的影响。

目的：囊性纤维化累及肝胆（CFHBI）是CF患者常见的合并症，与发病率和死亡率增加有关。新型高效CF跨膜电导调节剂（elexacaftor- tezactor -ivacaftor， ETI）对CFHBI的影响尚不清楚。本研究旨在探讨ETI对CF患儿肝脏硬度的影响，采用二维（2D）横波弹性成像（SWE）测量。方法：在瑞典隆德sk大学医院CF中心对21名CF患儿进行回顾性研究。我们的队列中有12个孩子患有CFHBI；没有人有晚期CF肝病。分析来自年度评估、临床数据和肝酶的2D SWE数据。结果：我们发现在整个队列中，开始使用ETI治疗后肝脏僵硬度显著降低。这种肝脏硬度的降低甚至可以在CFHBI患儿中看到。在整个队列中，肝酶在eti治疗前和治疗后都在正常范围内。在CFHBI患儿中，ETI启动后观察到天冬氨酸转氨酶活性下降。ETI治疗后肺功能和肺清除率指数明显改善。结论：ETI治疗对CF患儿CFHBI有积极影响，治疗期间肝脏僵硬度降低。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Pediatric Gastroenterology and Nutrition 医学-胃肠肝病学

CiteScore

5.30

自引率

13.80%

发文量

467

审稿时长

3-6 weeks

期刊介绍： The Journal of Pediatric Gastroenterology and Nutrition (JPGN) provides a forum for original papers and reviews dealing with pediatric gastroenterology and nutrition, including normal and abnormal functions of the alimentary tract and its associated organs, including the salivary glands, pancreas, gallbladder, and liver. Particular emphasis is on development and its relation to infant and childhood nutrition.