Evidence map›Paper›PMID 42758866›Full record

Observational studyHepatology communications2026

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

Cara L Mack, Alexander G Miethke, Kieran Hawthorne, Lisa Henn, Simon Horslen, Estella M Alonso, Nitika A Gupta, Shikha S Sundaram, Rohit Kohli, Molly Bozic and 14 more

Abstract readObservational Study
In one paragraph

Observational study in Hepatology communications, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

24 authors.

Cara L MackDivision of Pediatric Gastroenterology, Hepatology & Nutrition, Department of Pediatrics, Medical College of Wisconsin, Children's Wisconsin, Milwaukee, Wisconsin, USA.ORCID 0000-0002-5760-3836
Alexander G MiethkeDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.ORCID 0000-0003-1395-9475
Kieran HawthorneArbor Research Collaborative for Health, Ann Arbor, Michigan, USA.
Lisa HennArbor Research Collaborative for Health, Ann Arbor, Michigan, USA.ORCID 0000-0002-5075-8077
Simon HorslenDivision of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA.ORCID 0000-0001-5949-7363
Estella M AlonsoDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.ORCID 0000-0002-4056-7752
Nitika A GuptaDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA.
Shikha S SundaramSection of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, Colorado, USA.
Rohit KohliDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Children's Hospital Los Angeles, University of Southern California, Keck School of Medicine, Los Angeles, California, USA.ORCID 0000-0002-0198-7703
Molly BozicDivision of Gastroenterology and Hepatology, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana, USA.ORCID 0000-0002-3644-5381
Mark DeneauDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Intermountain Primary Children's Hospital, University of Utah, Salt Lake City, Utah, USA.ORCID 0000-0003-0459-9404
Stephen GutheryDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Intermountain Primary Children's Hospital, University of Utah, Salt Lake City, Utah, USA.ORCID 0000-0003-1484-301
Kathleen M LoomesDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-1539-6672
Pamela L ValentinoDivision of Gastroenterology and Hepatology, Department of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, Washington, USA.ORCID 0000-0002-8227-2004
Mary Elizabeth M TessierDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.ORCID 0000-0002-0341-5442
Benjamin ShneiderDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.ORCID 0000-0002-7208-0949
John C MageeDepartment of Surgery, Section of Transplant Surgery, University of Michigan Medical School, Ann Arbor, Michigan, USA.ORCID 0000-0001-8416-7905
Saul KarpenStravitz-Sanyal Institute for Metabolic Health and Liver Disease, Virginia Commonwealth University, Richmond, Virginia, USA.ORCID 0000-0002-3379-7592
Phillip RosenthalDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of California San Francisco, San Francisco, California, USA.
Kasper WangDivision of General and Thoracic Surgery, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.ORCID 0000-0003-0487-6416
Amanda RicciutoDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.ORCID 0000-0001-9538-3005
Ronald J SokolSection of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, Colorado, USA.ORCID 0000-0001-7433-4095
Binita M KamathDivision of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-9982-5023
Childhood Liver Disease Research Network (ChilLDReN)

Funding

Phenotypic Diversity in COVID-19UL1TR001878 · NCATS · UNIVERSITY OF PENNSYLVANIA · PI FITZGERALD, GARRET A · 2016 to 2025
$102.4M
NIH Prior Approval Process ProfessionalUL1TR002535 · NCATS · UNIVERSITY OF COLORADO DENVER · PI SOKOL, RONALD J. · 2018 to 2022
$51.1M
Colorado Clinical and Translational Sciences Institute (CCTSI)UM1TR004399 · NCATS · UNIVERSITY OF COLORADO DENVER · PI JANINE A HIGGINS, RONALD J. SOKOL · 2023 to 2026
$30.7M
Childhood Liver Disease Research Network Data Coordinating CenterU24DK062456 · NIDDK · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI JOHN C MAGEE · 2019 to 2026
$18.9M
Etiology and Treatment of Biliary Atresia and INHU01DK062453 · NIDDK · UNIVERSITY OF COLORADO DENVER · PI RONALD J. SOKOL · 2002 to 2026
$13.5M
Clinical Center for Cholestatic Liver Disease in ChildrenU01DK062497 · NIDDK · CINCINNATI CHILDRENS HOSP MED CTR · PI Alexander Miethke · 2002 to 2026
$13.2M
VCU-Atlanta Center for the Childhood Liver Disease Research Network (ChiLDReN)U01DK062470 · NIDDK · VIRGINIA COMMONWEALTH UNIVERSITY · PI SAUL J. KARPEN · 2005 to 2026
$12.7M
Mid-Atlantic BANH Consortium SiteU01DK062481 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI Binita Maya Kamath, Kathleen Mary Loomes · 2002 to 2026
$9.2M
Macrophage Cell Subset Specific Biomarkers for Disease Prognosis in Biliary AtresiaU01DK062436 · NIDDK · LURIE CHILDREN'S HOSPITAL OF CHICAGO · PI ALONSO, ESTELLA M. · 2002 to 2023
$7.0M
The Pittsburgh Cholestatic Liver Disease ConsortiumU01DK062466 · NIDDK · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI HORSLEN, SIMON · 2002 to 2023
$6.3M
Continuation of ChiLDReN, the Childhood Liver Disease Research Network: Indiana UniversityU01DK084536 · NIDDK · INDIANA UNIVERSITY INDIANAPOLIS · PI MOLLESTON, JEAN PAPPAS · 2009 to 2023
$5.9M
Continuation of the Childhood Liver Disease Research Network Seattle Clinical CenterU01DK084575 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Pamela Valentino · 2009 to 2026
$5.2M
NCATS NIH HHS UL1 TR001878NCATS NIH HHS UL1 TR002535NCATS NIH HHS UM1 TR004399NIDDK NIH HHS U01 DK062436NIDDK NIH HHS U01 DK062453NIDDK NIH HHS U01 DK062466NIDDK NIH HHS U01 DK062470NIDDK NIH HHS U01 DK062481NIDDK NIH HHS U01 DK062497NIDDK NIH HHS U01 DK062500NIDDK NIH HHS U01 DK084536NIDDK NIH HHS U01 DK084538NIDDK NIH HHS U01 DK084575NIDDK NIH HHS U01 DK103140NIDDK NIH HHS U01 DK103149NIDDK NIH HHS U24 DK062456
6 · The paper itself

Abstract

backgroundPrimary 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.

methodsData 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.

resultsThe 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.

conclusionsThis 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.

Indexed as

Cholangitis, SclerosingAdolescentAutoantibodiesChildChild, PreschoolDisease ProgressionElasticity Imaging TechniquesFemaleHepatitis, AutoimmuneHumansInflammatory Bowel DiseasesLiverLongitudinal StudiesMalePhenotypeProspective StudiesAutoantibodiesautoantibodyautoimmune hepatitisinflammatory bowel diseaseSCOPE indextransient elastography

Identifiers

PMID42758866
PMCPMC13592951

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.