1. Cystic fibrosis liver disease in the new era of cystic fibrosis transmembrane conductance regulator (CFTR) modulators.
- Author
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Eldredge, Jessica A., Oliver, Mark R., and Ooi, Chee Y.
- Subjects
CYSTIC fibrosis transmembrane conductance regulator ,HEPATIC fibrosis ,CYSTIC fibrosis ,LIVER diseases - Abstract
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 Regulator (CFTR) modulators monitoring guidelines is essential in clinical practice, particularly while their role in aCFLD is not established. 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. [ABSTRACT FROM AUTHOR]
- Published
- 2024
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