Ursodeoxycholic acid treatment in patients with cystic fibrosis at risk for liver disease.
BACKGROUND
Meconium ileus has been detected as a risk factor for development of liver disease in cystic fibrosis, with influence on morbidity and mortality.


OBJECTIVE
To evaluate the effect of early treatment with ursodeoxycholic acid in patients with cystic fibrosis and meconium ileus to prevent chronic hepatic involvement and to explore the potential role of therapy on clinical outcomes.


METHODS
26 cystic fibrosis patients with meconium ileus (16 M, mean age 8,4 years, range 3,5-9) were assigned to two groups: group 1 (14 patients) treated early with ursodeoxycholic acid (UDCAe); group 2 (12 patients) treated with ursodeoxycholic acid at the onset of cystic fibrosis liver disease (UDCAd). Anthropometric data, pulmonary function tests, pancreatic status, complications such as diabetes, hepatic involvement and Pseudomonas aeruginosa colonisation were compared among groups.


RESULTS
A higher prevalence of cystic fibrosis chronic liver disease was observed in the UDCAd group with a statistically significant difference at 9 years of age (p<0.05). Chronic infection by P. aeruginosa was found in 7% of UDCAe and 33% of UDCAd (p<0.05). No differences were observed in nutritional status and other complications.


CONCLUSIONS
Early treatment with ursodeoxycholic acid may be beneficial in patients at risk of developing cystic fibrosis chronic liver disease such as those with meconium ileus. Multicentre studies should be encouraged to confirm these data.
Siano
Maria
M
Department of Pediatrics, University of Naples Federico II, Via S. Pansini, 5, 80131 Naples, Italy.
De Gregorio
Fabiola
F
Boggia
Bartolo
B
Sepe
Angela
A
Ferri
Pasqualina
P
Buonpensiero
Paolo
P
Di Pasqua
Antonio
A
Raia
Valeria
V
D002648:Child; D002675:Child, Preschool; D002756:Cholagogues and Choleretics / Q000008:administration & dosage / Q000627:therapeutic use*; D002908:Chronic Disease; D003550:Cystic Fibrosis / Q000150:complications / Q000188:drug therapy* / Q000453:epidemiology; D004305:Dose-Response Relationship, Drug; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007558:Italy / Q000453:epidemiology; D008107:Liver Diseases / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control*; D008297:Male; D009017:Morbidity / Q000639:trends; D012189:Retrospective Studies; D015996:Survival Rate / Q000639:trends; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000008:administration & dosage / Q000627:therapeutic use*
D002756:Cholagogues and Choleretics; D014580:Ursodeoxycholic Acid