The Childhood Liver Disease Research Network's prospective characterization of pediatric primary sclerosing cholangitis.

BACKGROUND: Primary sclerosing cholangitis (PSC) is a rare biliary fibrosing disease that leads to significant morbidity and the need for liver transplantation. The Childhood Liver Disease Research Network is conducting a prospective, observational longitudinal study to define the natural history of pediatric PSC. The aim of this report is to characterize the clinical phenotypes of the first 175 children enrolled in the PSC study and to determine correlations of specific phenotypes with clinical and laboratory characteristics. METHODS: Data collected included a history of inflammatory bowel disease (IBD), autoimmune hepatitis (AIH), laboratories, autoantibodies, liver stiffness measurements (LSM), medications, and IBD activity. Comparison of phenotypes with/without IBD and/or AIH, and small-duct versus large-duct PSC was performed. RESULTS: The median age at enrollment was 16.1 years, 60% were male, 78% had large-duct PSC, and the median duration of PSC was 2.2 years. The predominant phenotype was PSC/IBD, and 96% had quiescent/mild IBD. The entire cohort had evidence of mild liver disease based on biochemistries and LSMs; <10% had evidence of portal hypertension. Higher LSMs were associated with elevated liver biochemistries. There were no significant characteristics that differentiated the phenotypes; however, there was more frequent autoantibody positivity in PSC/IBD and higher LSMs in PSC/AIH overlap syndrome. CONCLUSIONS: This prospective cohort of pediatric PSC revealed mild liver disease in the majority. Laboratory biomarkers of liver injury and fibrosis were associated with LSM severity, suggesting LSM as a reliable tool to monitor PSC progression. This dataset encompasses a well-phenotyped cohort of children with PSC that will be followed prospectively for longitudinal assessment of progression of disease, as well as a model for stratification within future treatment trials.

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Journal
PubMed
Published
2026-10-01
DOI
https://doi.org/10.1097/hc9.0000000000001049
Primary Topic
Liver Diseases and Immunity
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article
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article

The Childhood Liver Disease Research Network's prospective characterization of pediatric primary sclerosing cholangitis.

Kasper Wang, Amanda Ricciuto, Ronald J. Sokol, Saul J. Karpen et al.
PubMed
Liver Diseases and Immunity
article

The Childhood Liver Disease Research Network's prospective characterization of pediatric primary sclerosing cholangitis.

Kasper Wang, Amanda Ricciuto, Ronald J. Sokol, Saul J. Karpen, Alexander Gerhard Miethke, Mary Elizabeth M. Tessier, Pamela L. Valentino, Cara L. Mack, Estella M. Alonso, Shikha S. Sundaram, Kathleen M. Loomes, Binita M. Kamath, John C. Magee, Simon Horslen, Nitika A. Gupta, Molly Bozic, Kieran Hawthorne, Phillip Rosenthal, Stephen Guthery, Lisa Henn, Rohit Kohli, Benjamin Shneider, Mark Deneau
article en

Abstract

BACKGROUND: Primary sclerosing cholangitis (PSC) is a rare biliary fibrosing disease that leads to significant morbidity and the need for liver transplantation. The Childhood Liver Disease Research Network is conducting a prospective, observational longitudinal study to define the natural history of pediatric PSC. The aim of this report is to characterize the clinical phenotypes of the first 175 children enrolled in the PSC study and to determine correlations of specific phenotypes with clinical and laboratory characteristics. METHODS: Data collected included a history of inflammatory bowel disease (IBD), autoimmune hepatitis (AIH), laboratories, autoantibodies, liver stiffness measurements (LSM), medications, and IBD activity. Comparison of phenotypes with/without IBD and/or AIH, and small-duct versus large-duct PSC was performed. RESULTS: The median age at enrollment was 16.1 years, 60% were male, 78% had large-duct PSC, and the median duration of PSC was 2.2 years. The predominant phenotype was PSC/IBD, and 96% had quiescent/mild IBD. The entire cohort had evidence of mild liver disease based on biochemistries and LSMs; <10% had evidence of portal hypertension. Higher LSMs were associated with elevated liver biochemistries. There were no significant characteristics that differentiated the phenotypes; however, there was more frequent autoantibody positivity in PSC/IBD and higher LSMs in PSC/AIH overlap syndrome. CONCLUSIONS: This prospective cohort of pediatric PSC revealed mild liver disease in the majority. Laboratory biomarkers of liver injury and fibrosis were associated with LSM severity, suggesting LSM as a reliable tool to monitor PSC progression. This dataset encompasses a well-phenotyped cohort of children with PSC that will be followed prospectively for longitudinal assessment of progression of disease, as well as a model for stratification within future treatment trials.

PubMedVol. 10(10)
Northwestern University (US), University of Southern California (US), Cincinnati Children's Hospital Medical Center (US), Lurie Children's Hospital (US), Seattle Children's Hospital (US), Primary Children's Hospital (US), Children's Hospital of Philadelphia (US), Children's Hospital of Los Angeles (US), Emory University (US), University of Pittsburgh (US), University of California, San Francisco (US), Baylor College of Medicine (US), Virginia Commonwealth University (US), University of Toronto (CA), University of Washington (US), Medical College of Wisconsin (US), University of Utah (US), University of Michigan (US), Hospital for Sick Children (CA), Riley Hospital for Children (US), Children's Hospital Colorado (US), Michigan Medicine (US), Children's Hospital of Pittsburgh (US), University of Pittsburgh Medical Center (US), Arbor Research Collaborative for Health (US), Texas Children's Hospital (US), Indiana University School of Medicine, University of Colorado Anschutz Medical Campus (US), University of Cincinnati (US), University of Pennsylvania (US)
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