Table 1

Clinical signs and outcome at the study end point in 163 patients with Alagille syndrome and chronic liver disease. The study end point was defined as either death, liver transplantation, or last follow up

Patients with neonatal cholestatic jaundicePatients with no clinically patent neonatal jaundice
No patients13231
Median age (range)9 y (2 mos–29 y)14 y (3–44)p Value
No positive/No patients studied No positive/No patients studied
Jaundice102/132 5/31<0.0001
Pruritus112/1321-150 17/281-152  0.0003
Xanthomas 40/132 0/31 0.0004
Hepatomegaly115/13211/31<0.0001
Splenomegaly 62/132 6/29 0.0095
Cirrhosis 35/761-151  6/181-154 NS
Oesophageal varices 25/711-153  4/111-153 NS
GI bleeding  5/132 1/29NS
Cholelithiasis  6/132 1/29NS
Hepacellular carcinoma  0/132 1/31NS
Liver transplantation 44/132 0/31 0.0002
Death related to liver disease 17/132 2/31NS
Dead 48/132 9/31NS
Median (range)/No patients studied Median (range)/No patients studied
ASAT (IU/l)162 (23–650)/106 12 (25–186)/23 0.0004
γGT (IU/)408 (13–2288)/92332 (28–787)/18NS
Serum cholesterol (mmol/l) 9.9 (1.3–50.5)/126 7.4 (4.8–8)/26NS
  • 1-150 Variably controlled under medical treatment.

  • 1-152 Well controlled under medical treatment.

  • 1-151 32 patients who underwent liver biopsy after one year of age and 44 patients whose liver was removed at LT.

  • 1-154 18 patients who underwent liver biopsy after one year of age.

  • 1-153 Number of patients who underwent endoscopy during follow up.