Educational aims

The reader will come to appreciate that:

Severe cystic fibrosis liver disease (CLFD) commonly presents in early childhood years, and affects up to 10 % of people with cystic fibrosis by age 30 years.

United States CF Foundation (USCFF) guidelines have recently further defined CF hepatobiliary involvement (CFHBI) and advanced CF liver disease (aCFLD).

Familiarity with Cystic Fibrosis Transmembrane Conductance Receptor (CFTR) modulators monitoring guidelines is essential in clinical practice, particularly while their role in aCFLD is not established.

Summary

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 Receptor (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.

中文翻译：

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囊性纤维化跨膜电导受体（CFTR）调节剂新时代的囊性纤维化肝病

教育目标

读者将会意识到：

严重囊性纤维化肝病 (CLFD) 通常出现在儿童早期，并且在 30 岁时影响高达 10% 的囊性纤维化患者。

美国CF基金会（USCFF）指南最近进一步定义了CF肝胆受累（CFHBI）和晚期CF肝病（aCFLD）。

熟悉囊性纤维化跨膜电导受体 (CFTR) 调节剂监测指南在临床实践中至关重要，特别是在其在 aCFLD 中的作用尚未确定的情况下。

概括

囊性纤维化肝病（CFLD）的特点是表现形式和严重程度存在广泛的异质性。它是囊性纤维化 (PwCF) 患者发病的一个主要原因，随着囊性纤维化护理新时代生存率的提高，这一点将变得越来越重要。目前没有可用的药物治疗有证据可以治疗或预防肝病的进展。囊性纤维化跨膜电导受体 (CFTR) 调节剂可能会对肺、营养和生活质量产生变革，但对长期肝病结果的直接影响尚未确定。药物相关的肝脏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。需要进行纵向研究来了解 CFTR 调节剂对 CFLD 发病率和自然史的影响，包括早期治疗开始、已确诊的晚期肝病和肝移植后。