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Published on: June 26, 2018
Paediatric-onset autoimmune liver disease: Insights from a monocentric experience
Franco Curci1, Chiara Rubino2, Mariangela Stinco2
1NEUROFARBA Department, University of Florence, viale Gaetano Pieraccini 6, 50139 Firenze, Italy.
Insights
Childhood autoimmune liver diseases (AILD) show a progression with increasing age at onset for autoimmune hepatitis (AIH), autoimmune sclerosing cholangitis (ASC), and primary sclerosing cholangitis (PSC). A third of patients experienced liver complications, indicating a progressive disease course.
Area of Science:
- Pediatric Gastroenterology and Hepatology
- Autoimmune Liver Diseases
- Clinical Research
Background:
- Autoimmune liver disease (AILD) includes autoimmune hepatitis (AIH), autoimmune sclerosing cholangitis (ASC), and primary sclerosing cholangitis (PSC).
- A unified disease process evolving over time through these entities has been recently suggested.
- This study investigates childhood-onset AILD characteristics and progression.
Purpose of the Study:
- To compare the characteristics of childhood-onset AILD at baseline.
- To analyze medium-to-long term follow-up data for pediatric AILD patients.
- To identify trends and outcomes in pediatric autoimmune liver diseases.
Main Methods:
- Retrospective review of pediatric-onset AILD cases diagnosed between 1992 and 2023.
- Inclusion of 55 patients: 20 AIH, 22 ASC, 13 PSC.
- Clinical updates collected from patients transitioned to adult care.
Main Results:
- AIH, ASC, and PSC showed increasing age at onset (p < 0.01).
- Gamma-glutamyltranspeptidase (GGT) and ALP/AST ratio predicted sclerosing cholangitis with 86% sensitivity and 94% specificity.
- After a median follow-up of 5.8 years, 33% developed portal hypertension, 4% underwent liver transplantation, and no deaths occurred.
Conclusions:
- Childhood-onset AILD exhibits a temporal trend with progressively older ages for AIH, ASC, and PSC.
- Elevated GGT and high ALP/AST ratio are predictive of sclerosing cholangitis.
- One-third of patients experiencing adverse events highlights the progressive nature of pediatric AILD.
Background:
Autoimmune liver disease (AILD) encompasses autoimmune hepatitis (AIH), autoimmune sclerosing cholangitis (ASC) and primary sclerosing cholangitis (PSC). A unified disease process evolving over time through these entities has been recently suggested. From this perspective, this study aimed to compare the characteristics of childhood-onset AILD at baseline and after a medium-to-long term follow-up period.
Methods:
Paediatric-onset cases of AILD diagnosed between 1992 and 2023 at a tertiary-care centre were reviewed. Patients transitioned to adult-care by the time of data collection were asked for clinical updates.
Results:
Fifty-five patients were included (AIH = 20, ASC =22, PSC =13). AIH, ASC and PSC exhibited increasing age at the onset (AIH to PSC, p < 0.01). The area under the receiver operating characteristic curve for gamma-glutamyltranspeptidase (GGT) combined with alkaline phosphatase/aspartate aminotransferase (ALP/AST) ratio in predicting sclerosing cholangitis was 0.94, with a sensitivity of 86 % and a specificity of 94 %. At the last follow-up (median duration 5,8 years, interquartile range [IQR] 2,9-10,2, n = 45), 15 patients (33 %) developed portal hypertension, 2 patients (4 %) underwent liver transplantation, no patient died.
Conclusion:
A cohort of childhood-onset AILD managed at a single centre reveals a temporal trend in the onset of AIH, ASC and PSC, with progressively older ages. Elevated GGT levels combined with a high ALP/AST ratio predict the diagnosis of sclerosing cholangitis. The occurrence of liver-related adverse events in one-third of patients highlights the progressive nature of paediatric-onset AILD.
