TY - JOUR
T1 - Outcome of liver disease in children with Alagille syndrome
T2 - A study of 163 patients
AU - Lykavieris, P.
AU - Hadchouel, M.
AU - Chardot, C.
AU - Bernard, O.
PY - 2001/9/12
Y1 - 2001/9/12
N2 - Background and aims - Various opinions have been expressed as to the long term prognosis of liver disease associated with Alagille syndrome (AGS). Patients and methods - We reviewed the outcome of 163 children with AGS and liver involvement, investigated from 1960 to 2000, the end point of the study (median age 10 years (range 2 months to 44 years)) being death, liver transplantation, or the last visit. Results - At the study end point, of the 132 patients who presented with neonatal cholestatic jaundice, 102 remained jaundiced, 112 had poorly controlled pruritus, and 40 had xanthomas; cirrhosis was found in 35/76 livers, varices in 25/71 patients, and liver transplantation had been carried out in 44 patients (33%). Forty eight patients died, 17 related to complications of liver disease. Of 31 patients who did not present with neonatal cholestatic jaundice, five were jaundiced at the study end point, 17 had well controlled pruritus, and none had xanthomas; cirrhosis was found in 6/18 patients, varices in 4/11, and none underwent liver transplantation. Nine patients died, two of liver disease. In the whole series, actuarial survival rates with native liver were 51% and 38% at 10 and 20 years, respectively, and overall survival rates were 68% and 62%, respectively. Neonatal cholestatic jaundice was associated with poorer survival with native liver (p=0.0004). Conclusions - The prognosis of liver disease in AGS 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.
AB - Background and aims - Various opinions have been expressed as to the long term prognosis of liver disease associated with Alagille syndrome (AGS). Patients and methods - We reviewed the outcome of 163 children with AGS and liver involvement, investigated from 1960 to 2000, the end point of the study (median age 10 years (range 2 months to 44 years)) being death, liver transplantation, or the last visit. Results - At the study end point, of the 132 patients who presented with neonatal cholestatic jaundice, 102 remained jaundiced, 112 had poorly controlled pruritus, and 40 had xanthomas; cirrhosis was found in 35/76 livers, varices in 25/71 patients, and liver transplantation had been carried out in 44 patients (33%). Forty eight patients died, 17 related to complications of liver disease. Of 31 patients who did not present with neonatal cholestatic jaundice, five were jaundiced at the study end point, 17 had well controlled pruritus, and none had xanthomas; cirrhosis was found in 6/18 patients, varices in 4/11, and none underwent liver transplantation. Nine patients died, two of liver disease. In the whole series, actuarial survival rates with native liver were 51% and 38% at 10 and 20 years, respectively, and overall survival rates were 68% and 62%, respectively. Neonatal cholestatic jaundice was associated with poorer survival with native liver (p=0.0004). Conclusions - The prognosis of liver disease in AGS 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.
KW - Alagille syndrome
KW - Cholestasis
KW - End stage liver disease
KW - Liver transplantation
UR - http://www.scopus.com/inward/record.url?scp=0034877469&partnerID=8YFLogxK
U2 - 10.1136/gut.49.3.431
DO - 10.1136/gut.49.3.431
M3 - Article
C2 - 11511567
AN - SCOPUS:0034877469
SN - 0017-5749
VL - 49
SP - 431
EP - 435
JO - Gut
JF - Gut
IS - 3
ER -