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囊性纤维化跨膜电导调节因子(CFTR)调节剂时代的囊性纤维化肝病。

Cystic fibrosis liver disease in the new era of cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

机构信息

Department of Gastroenterology and Clinical Nutrition, Royal Children's Hospital, Melbourne, Australia.

Department of Gastroenterology and Clinical Nutrition, Royal Children's Hospital, Melbourne, Australia; Department of Paediatrics, Faculty of Medicine, The University of Melbourne, Melbourne, Australia.

出版信息

Paediatr Respir Rev. 2024 Jun;50:54-61. doi: 10.1016/j.prrv.2023.12.005. Epub 2023 Dec 28.

Abstract

Cystic fibrosis liver disease (CFLD) is characterised by a wide heterogenity of manifestations and severity. It represents a major cause of morbidity in people with cystic fibrosis (PwCF), which will be of increasing relevance as survival increases in the new era of cystic fibrosis care. No medical therapy currently available has evidence to treat or prevent progression of liver disease. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators may be transformative on pulmonary, nutritional and quality of life, but direct effect on long term liver disease outcomes is not yet established. Drug-associated hepatic adverse effects may be common, and clinician familiarity with drug-monitoring recommendations is essential. Longitudinal studies are required to understand the effect of CFTR modulators on the incidence and natural history of CFLD, including with early treatment initiation, in established advanced liver disease, and post liver transplantation.

摘要

囊性纤维化肝病(CFLD)的表现和严重程度具有广泛的异质性。它是囊性纤维化患者(PwCF)发病的主要原因,随着囊性纤维化治疗新时代中患者生存时间的延长,这一问题将变得更加重要。目前尚无可用的医学疗法有证据表明可以治疗或预防肝病的进展。囊性纤维化跨膜电导调节因子(CFTR)调节剂可能对肺部、营养和生活质量产生变革性影响,但对长期肝病结局的直接影响尚未确定。与药物相关的肝不良反应可能很常见,临床医生熟悉药物监测建议至关重要。需要进行纵向研究,以了解 CFTR 调节剂对 CFLD 的发生率和自然史的影响,包括早期治疗在已建立的晚期肝病和肝移植后的启动。

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