Less than half of kids with ALGS reach adulthood with native liver

Approximately only 40.3% of children with Alagille syndrome (ALGS) will reach adulthood with their native liver, according to a large international study designed to look at the natural history of liver disease in a contemporary cohort.

Better hepatic outcomes were associated with total bilirubin (TB) levels <5.0 mg/dL in patients between 6-and-12-months of age.

In this multicenter retrospective study, 1433 children with ALGS born between January 1997 to August 2019 were included.

Native liver survival (NLS) rates were 54.4% at 10 years and 40.3% at 18 years. By 10 years old, 51.5% of children had experienced ≥1 adverse liver-related event (clinically evident portal hypertension [CEPH], transplant or death); that number increased to 66% by 18 years old.

In children between 6 and 12 months there was a 4.1-fold and 8.0-fold increased risk of developing CEPH with median TB levels ≥5.0 and <10.0 mg/dL and those ≥10.0 mg/dL, respectively, compared with those <5.0 mg/dL.

Reference
Vandriel SM, Li LT, She H, et al; Global ALagille Alliance (GALA) Study Group. Natural History of Liver Disease in a Large International Cohort of Children with Alagille syndrome: Results from The GALA Study. Hepatology. 2022;doi: 10.1002/hep.32761. Epub ahead of print. PMID: 36036223.

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