This was a very large retrospective study (with more than 90 authors) with 1433 children.
Key findings:
Only 40.3% of children reach adulthood with their native liver; 54.4% had their native liver at 10 years of life
“It is noteworthy that bile duct paucity was reported in only 65% of liver biopsies performed during the first 3 months of life, the period during which there are diagnostic challenges with distinguishing ALGS from syndromic BA.” Thus, with a liver biopsy, there is a significant risk of misdiagnosis
The all‐cause mortality rate was 8.5%
The total bilirubin level between 6-12 months of life had significant predictive value. In the associated editorial: “The authors reported that 79% of patients with median TB of <5.0 mg/dL..reached adulthood with their native livers, whereas only 31.6% and 18.2% of patients with median TB levels between” 5-10 and >10 mg/dL survived into adulthood with their native livers.
The editorial makes the point that this data will be helpful and ongoing studies will be needed to determine the effectiveness of novel treatments (e.g. IBAT inhibitors)
My take: This is a very useful study in understanding the long term outcomes of Alagille syndrome.
