Diemer Stefanie, Elidottir Helga, Eklund Erik A, Påhlman Lisa I, Hansen Christine
Department of Paediatrics, Skåne University Hospital, Lund, Sweden.
Wallenberg Centre for Molecular Medicine, Lund, Sweden.
J Pediatr Gastroenterol Nutr. 2025 Jul;81(1):74-81. doi: 10.1002/jpn3.70050. Epub 2025 Apr 23.
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).
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.
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.
ETI treatment could positively affect CFHBI in children with CF, as demonstrated by reduced liver stiffness during treatment.
囊性纤维化肝胆受累（CFHBI）是囊性纤维化（CF）患者常见的合并症，与发病率和死亡率增加相关。新型高效的CF跨膜电导调节剂疗法，即依列卡福-替扎卡福-依伐卡福（ETI），对CFHBI的影响仍不明确。本研究旨在探讨ETI对CF儿童肝脏硬度的影响，采用二维（2D）剪切波弹性成像（SWE）进行测量。
瑞典隆德斯科讷大学医院CF中心的这项回顾性研究纳入了21名CF儿童。我们队列中的12名儿童患有CFHBI；均无晚期CF肝病。分析了年度评估的2D SWE数据、临床数据和肝酶。
我们发现，在整个队列中开始使用ETI治疗后，肝脏硬度显著降低。这种肝脏硬度的降低在患有CFHBI的儿童中甚至也能看到。在整个队列中，ETI治疗前后肝酶均在正常范围内。在患有CFHBI的儿童中，开始使用ETI后观察到天冬氨酸转氨酶活性下降。开始ETI治疗后，肺功能和肺清除指数显著改善。
ETI治疗可对CF儿童的CFHBI产生积极影响，治疗期间肝脏硬度降低证明了这一点。
