Cecchetti Martina, Scarallo Luca, Lionetti Paolo, Ooi Chee Y, Terlizzi Vito
Department of Health Sciences, University of Florence, Florence, Italy; Meyer Children Hospital IRCCS, Florence, Italy.
Gastroenterology and Nutrition Unit, Meyer Children's Hospital IRCCS, Florence, Italy; Department of NEUROFARBA, University of Florence, Florence, Italy.
Paediatr Respir Rev. 2025 Jun;54:70-75. doi: 10.1016/j.prrv.2024.07.004. Epub 2024 Sep 20.
Highly effective modulator therapy (HEMT), particularly the triple combination elexacaftor-tezacaftor-ivacaftor (ETI), significantly improved clinical outcomes and quality of life in people with Cystic Fibrosis (pwCF). This review analyzes current knowledge on the impact of HEMTs on gastrointestinal (GI) symptoms and features in pwCF. A descriptive review of English literature until February 29, 2024, was conducted using medical databases. Observational studies and clinical trials addressing GI reflux disease (GERD), lower GI symptoms and pancreatic disease were considered. Studies report positive effects of HEMTs on pH levels and bicarbonate secretion as well as improvement on intestinal inflammation. HEMTs also demonstrated positive effects on GERD and lower GI symptoms or conditions CF related such as dysbiosis. Taking ETI during pregnancy could also allow resolution of meconium ileus in fetuses with CF. The best benefits were observed in pancreatic function, potentially delaying CF-related diabetes and recovering pancreatic function in some children on ETI. Larger trials, particularly in pediatric populations, need to confirm these findings and explore long-term effects.
高效调节剂疗法（HEMT），特别是三联组合药物依列卡福-替扎卡福-艾伐卡福（ETI），显著改善了囊性纤维化患者（pwCF）的临床结局和生活质量。本综述分析了目前关于HEMTs对pwCF胃肠道（GI）症状和特征影响的知识。使用医学数据库对截至2024年2月29日的英文文献进行了描述性综述。纳入了针对胃食管反流病（GERD）、下消化道症状和胰腺疾病的观察性研究和临床试验。研究报告了HEMTs对pH值水平和碳酸氢盐分泌的积极影响以及对肠道炎症的改善作用。HEMTs对GERD和与CF相关的下消化道症状或病症（如菌群失调）也显示出积极影响。孕期服用ETI还可使患有CF的胎儿胎粪性肠梗阻得到缓解。在胰腺功能方面观察到了最佳益处，这可能会延迟与CF相关的糖尿病，并使一些接受ETI治疗的儿童恢复胰腺功能。需要开展更大规模的试验，尤其是针对儿科人群的试验，以证实这些发现并探索长期影响。
