The researchers reviewed the outcome of liver disease in 163 children with Alagille syndrome, and reported their findings in the September issue of Gut.
All subjects were investigated between 1960 and 2000. The study end-points were death, liver transplantation, or the last visit (median age of 10 years; range 2 months to 44 years).
Of 132 patients who presented with neonatal cholestatic jaundice, 102 remained jaundiced, 112 had poorly controlled pruritus, and 40 had xanthomas. In addition, cirrhosis was found in 35/76 livers, and varices in 25/71 patients. Liver transplantation was carried out in 44 patients (33%).
A total of 48 of these patients died - 17 related to complications of liver disease.
Of the 31 patients who did not present with neonatal cholestatic jaundice, 5 were jaundiced at the study end-point, 17 had well-controlled pruritus, and none had xanthomas. Cirrhosis was found in 6/18 patients and varices in 4/11. No subjects underwent liver transplantation.
|Overall survival rates|
10 years: 68%
20 years: 62%
Nine of these patients died - 2 of liver disease.
The investigators found that, in the whole series, actuarial survival rates with native liver were 51% and 38%, at 10 and 20 years respectively. Overall survival rates were 68% and 62% respectively.
Neonatal cholestatic jaundice was found to be associated with poorer survival with native liver.
Author P. Lykavieris, of the BicÍtre Hospital, said on behalf of fellow colleagues, "The prognosis of liver disease in Alagille syndrome is worse in children who present with neonatal cholestatic jaundice.
"However, severe liver complications are possible even after late onset of liver disease, demanding follow-up throughout life," it was concluded.