Manchester Adult Cystic Fibrosis Centre, Wythenshawe Hospital, Manchester University NHS Foundation Trust, Southmoor Road, Manchester M23 9LT, UK; Division of Immunology, Immunity to Infection & Respiratory Medicine, University of Manchester, Manchester, UK.
Division of Diabetes, Endocrine and Gastroenterology, University of Manchester, Manchester, UK.
J Cyst Fibros. 2024 Mar;23(2):349-353. doi: 10.1016/j.jcf.2023.09.006. Epub 2023 Sep 19.
There are limited studies to date on the effects of elexacaftor/tezacaftor/ivacaftor (E/T/I) on markers of liver fibrosis in adults with cystic fibrosis (CF). This study aims to analyse changes in makers of liver fibrosis before and after initiation of E/T/I in CF adults.
Outcome measures of liver fibrosis, including liver stiffness measurement (LSM) using FibroScan, AST-to-platelet-ratio index (APRI) and gamma-GT-to-platelet-ratio (GPR) were available in 74 CF adults following initiation of E/T/I. This was compared to historical data collected in 2018 prior to UK availability of E/T/I.
The median duration of E/T/I therapy at the time liver fibrosis markers were repeated was 21 (IQR: 17-25) months. There was an increase in APRI from historical measurement to follow-up but no change in LSM or GPR. There were no differences in change in fibrosis markers according to CF liver disease (CFLD) status, although those with a raised LSM at baseline (>6.8 kPa) (n = 14) had a significant reduction in LSM from historical measurement to follow-up versus those with a normal historical value (-3.3 kPa vs 0.25 kPa, p < 0.01).
Apart from APRI, we found no changes in liver fibrosis outcomes after initiation of E/T/I in adults with CF. Those with a historical diagnosis of CFLD had no significant worsening or improvement of liver fibrosis markers. We did observe a reduction in LSM in those with liver nodularity, with an initial highest result suggesting a potential positive treatment effect of E/T/I in this category of those with severe CFLD.
目前关于 elexacaftor/tezacaftor/ivacaftor(E/T/I)对成人囊性纤维化(CF)患者肝纤维化标志物影响的研究有限。本研究旨在分析 CF 成人开始 E/T/I 治疗前后肝纤维化标志物的变化。
74 例 CF 成人开始 E/T/I 治疗后,可获得肝纤维化的结局测量指标,包括使用 FibroScan 进行的肝硬度测量(LSM)、天冬氨酸氨基转移酶与血小板比值指数(APRI)和 γ-谷氨酰转肽酶与血小板比值(GPR)。这与 2018 年在英国 E/T/I 可用之前收集的历史数据进行了比较。
在重复肝纤维化标志物时,E/T/I 治疗的中位持续时间为 21(IQR:17-25)个月。APRI 从历史测量值到随访时有所增加,但 LSM 或 GPR 没有变化。根据 CF 肝病(CFLD)状态,纤维化标志物的变化没有差异,尽管那些基线 LSM 升高(>6.8 kPa)(n=14)与基线时的历史正常值相比,LSM 从历史测量值到随访时显著降低(-3.3 kPa 比 0.25 kPa,p<0.01)。
除了 APRI,我们发现 CF 成人开始 E/T/I 治疗后,肝纤维化结局没有变化。那些有 CFLD 历史诊断的患者,肝纤维化标志物没有明显的恶化或改善。我们确实观察到那些有肝结节的患者 LSM 降低,最初的最高结果表明 E/T/I 在这一类严重 CFLD 患者中可能有积极的治疗效果。