Semin Liver Dis 1998;18(3):227-35

Liver and biliary problems in cystic fibrosis

Colombo C, Battezzati PM, Strazzabosco M, Podda M

Abstract
Department of Pediatrics, University of Sassari, Italy.

Liver disease associated with cystic fibrosis has been increasingly diagnosed
during recent years probably due to the combined effect of systematic hepatic
assessment and reduced death from extra-hepatic causes of CF patients. In a
group of 173 CF patients regularly followed at our Center, cumulative incidence
of liver disease was 17% over a mean period of 10 years. Although it generally
runs a mild course, it is considered a major complication of CF which may limit
survival and quality of life of affected patients. CF-associated liver disease
should be considered as the first inherited liver disorder in which the primary
defect affects cholangiocyte transport systems. Although data assessing the
effects of defective CFTR on cholangiocyte pathobiology are not yet available,
the impaired secretory function of the biliary epithelium is considered
responsible for reduced biliary fluidity and alkalinity and for subsequent bile
duct damage by cytotoxic compounds or infectious agents. No clear association
with specific CFTR mutations has been observed. Treatment with ursodeoxycholic
acid, aimed at improving biliary secretion in terms of bile viscosity and bile
acid composition, is currently the most useful therapeutic approach in
CF-associated liver disease. Beneficial effects on liver biochemistry, hepatic
excretory function, liver histology, and essential fatty acid status have been
reported, but no long-term data exist on its effectiveness on clinically
relevant outcomes, such as death or need for transplantation. The effectiveness
of bile acid therapy may be higher if started in patients with early stage liver
disease, before symptoms have become clinically evident. Early diagnosis and
identification of CF patients who are more liable to develop liver disease
should be actively pursued