Natural history of liver disease in cystic fibrosis

Abstract

The median age of the population with cystic fibrosis (CF) has increased worldwide, which has led to the suggestion that the prevalence of liver disease would increase. The aim of this study was to evaluate the natural history of CF‐associated liver disease over a 15‐year period in a well‐controlled population of patients with CF. During the years 1976 through 1993, 124 patients were followed up by yearly liver function tests (LFTs). Fifteen patients were followed up with liver biopsies throughout the whole study period. More than 50% of the patients had pathological LFTs in infancy, later being normalized. Approximately 25% of children 4 years of age or older had biochemical markers of liver disease during the study period. In about 10% of the patients, cirrhosis or advanced fibrosis was confirmed at biopsy and 4% of patients had cirrhosis with clinical liver disease. Severe liver disease developed mainly during prepuberty and puberty. Of the 15 patients prospectively followed up with liver biopsies, only 3 had progressive fibrosis. No specific risk factor was identified, but deficiency of essential fatty acids was found more often in patients with marked steatosis (P < .05). No patient developed clinical liver disease in adulthood and the histological changes in the liver biopsies were usually not progressive. Liver disease was no more frequent at the end of the study period although the median age of the patient population had increased. Modern treatment might positively influence liver disease because it seemed less common, less progressive, and less serious than previously reported.

Cystic Fibrosis Liver Disease: To Transplant or Not to Transplant?

“…Cystic fibrosis (CF) is complicated by liver disease in 4-10% of cases (1)(2)(3). Focal biliary cirrhosis is characteristic and is complicated frequently by portal hypertension and chronic liver failure (4,5).…”

“…Although most patients with CF present with liver disease in their first decade (1,8,9), a small proportion are adults when decompensated cirrhosis develops. A role for liver transplantation in end-stage liver disease complicating CF in childhood is well established (6,10,11), but the longterm outcome of liver transplantation for adults with CFrelated liver disease is less certain.…”

Fibrogenesis in pediatric cholestatic liver disease: Role of taurocholate and hepatocyte-derived monocyte chemotaxis protein-1 in hepatic stellate cell recruitment #

“…[1][2][3][4] Disrupted bile flow via intrahepatic or extrahepatic biliary obstruction results in variably progressive liver fibrosis. In CFLD, this process is slow, causing significant morbidity from focal biliary cirrhosis in about 20% of patients by the second decade of life.…”

“…In CFLD, this process is slow, causing significant morbidity from focal biliary cirrhosis in about 20% of patients by the second decade of life. 3,4 The apical localization of the cystic fibrosis transmembrance conductance regulator (CFTR) in cholangiocytes 5 suggests that abnormal ion transport leads to cholestasis, with focal fibrosing destruction of intrahepatic bile ducts. 6 The resulting pathognomonic pattern of injury is focal biliary cirrhosis, for which there is no known treatment and which may evolve into multilobular biliary cirrhosis and liver failure.…”

Liver Disease in Cystic Fibrosis - Presentation in Early Childhood: Report of Two Cases

“…(6) The new medications and better comprehension of Cystic Fibrosis increased survival of the patients and the occurrence of other complications such as hepatobiliary manifestations, which became more frequent. (4) The liver disease affects one third of the patients (7) and it is the third cause of death in cystic fibrosis (8) despite the lack of association with severity of the pulmonary disease. (2) Most studies describe the beginning of liver manifestations in cystic fibrosis at the first decade of life, mostly between the fourth and sixth year.…”