Current Treatment Options for Cystic Fibrosis-Related Liver Disease.
Publisher DOI
PubMed ID
33202578
Abstract
Cystic Fibrosis-related liver disease (CFLD) has become a leading cause of morbidity and mortality in patients with Cystic Fibrosis (CF), and affects children and adults. The understanding of the pathogenesis of CFLD is key in order to develop efficacious treatments. However, it remains complex, and has not been clarified to the last. The search for a drug might be additionally complicated due to the diverse clinical picture and lack of a unified definition of CFLD. Although ursodeoxycholic acid has been used for decades, its efficacy in CFLD is controversial, and the potential of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators and targeted gene therapy in CFLD needs to be defined in the near future. This review focuses on the current knowledge on treatment strategies for CFLD based on pathomechanistic viewpoints.
Date Issued
2020-11-14
Publication Type
Article
Subject(s)
Subjects
CFTR modulators cystic fibrosis-related liver disease liver transplantation ursodeoxycholic acid
Language(s)
en
Additional Credits
Journal
International journal of molecular sciences
Publisher
MDPI
ISSN
1422-0067
Access(Rights)
open.access