Cited by 230Liver Diseases and ImmunityPediatric Hepatobiliary Diseases and TreatmentsIgG4-Related and Inflammatory Diseases

The natural history of primary sclerosing cholangitis in 781 children: a multicenter, international collaboration

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Deneau, Mark R., El‐Matary, Wael, Valentino, Pamela L., Abdou, Reham, Alqoaer, Khaled, Amin, Mansi, Amir, Achiya Z., Auth, Marcus +38 more·2017·Hepatology

Abstract

There are limited data on the natural history of primary sclerosing cholangitis (PSC) in children. We aimed to describe the disease characteristics and long‐term outcomes of pediatric PSC. We retrospectively collected all pediatric PSC cases from 36 participating institutions and conducted a survival analysis from the date of PSC diagnosis to dates of diagnosis of portal hypertensive or biliary complications, cholangiocarcinoma, liver transplantation, or death. We analyzed patients grouped by disease phenotype and laboratory studies at diagnosis to identify objective predictors of long‐term outcome. We identified 781 patients, median age 12 years, with 4,277 person‐years of follow‐up; 33% with autoimmune hepatitis, 76% with inflammatory bowel disease, and 13% with small duct PSC. Portal hypertensive and biliary complications developed in 38% and 25%, respectively, after 10 years of disease. Once these complications developed, median survival with native liver was 2.8 and 3.5 years, respectively. Cholangiocarcinoma occurred in 1%. Overall event‐free survival was 70% at 5 years and 53% at 10 years. Patient groups with the most elevated total bilirubin, gamma‐glutamyltransferase, and aspartate aminotransferase‐to‐platelet ratio index at diagnosis had the worst outcomes. In multivariate analysis PSC–inflammatory bowel disease and small duct phenotypes were associated with favorable prognosis (hazard ratios 0.6, 95% confidence interval 0.5‐0.9, and 0.7, 95% confidence interval 0.5‐0.96, respectively). Age, gender, and autoimmune hepatitis overlap did not impact long‐term outcome. Conclusion: PSC has a chronic, progressive course in children, and nearly half of patients develop an adverse liver outcome after 10 years of disease; elevations in bilirubin, gamma‐glutamyltransferase, and aspartate aminotransferase‐to‐platelet ratio index at diagnosis can identify patients at highest risk; small duct PSC and PSC–inflammatory bowel disease are more favorable disease phenotypes. (H epatology 2017;66:518–527).

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  • In a large, multicentre international retrospective study including 781 children and young people diagnosed with PSC, 67% fulfilled the diagnostic criteria for AIH, in contrast to 6.6% in a similar study including 7,121 adults, suggesting higher prevalence in the paediatric population.

    “In a large, multicentre international retrospective study including 781 children and young people diagnosed with PSC, 67% fulfilled the diagnostic criteria for AIH, in contrast to 6.6% in a similar study including 7,121 adults, suggesting higher prevalence in the paediatric population.6,427”

    From EASL Clinical Practice Guidelines on sclerosing cholangitis (2022) · cites this paper as [427]

  • In a large, multicentre international retrospective study including 781 children and young people diagnosed with PSC, CCA was diagnosed in 1% and all patients with large duct involvement were >15 years.

    “In a large, multicentre international retrospective study including 781 children and young people diagnosed with PSC, CCA was diagnosed in 1%all patients with large duct involvement were >15 years.427”

    From EASL Clinical Practice Guidelines on sclerosing cholangitis (2022) · cites this paper as [427]

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  1. Weismuller TJ, Hansen BE, Trivedi PJ, Imam M, Lenzen H, Ponsioen CY, et al. Gender and IBD phenotype are independent predictors of death or transplantation and of malignancy in primary sclerosing cholangitis-a multicenter retrospective study of the International PSC Study Group (IPSCSG) [Abstract]. HEPATOLOGY 2015;62:246A.

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Authors
Deneau, M. R., El‐Matary, W., Valentino, P. L., Abdou, R., Alqoaer, K., Amin, M., Amir, A. Z., Auth, M., Bazerbachi, F., Broderick, A., Chan, A., Cotter, J., Doan, S., El‐Youssef, M., Ferrari, F., Furuya, K. N., Gottrand, M., Gottrand, F., Gupta, N., Homan, M., Kamath, B. M., Kim, K. M., Kolho, K., Konidari, A., Koot, B., Iorio, R., Ledder, O., Mack, C., Martinez, M., Miloh, T., Mohan, P., O'Cathain, N., Papadopoulou, A., Ricciuto, A., Saubermann, L., Sathya, P., Shteyer, E., Smolka, V., Tanaka, A., Varier, R., Venkat, V., Vitola, B., Vos, M. B., Woynarowski, M., Yap, J., & Jensen, M. K.
Affiliations
  • University of Utah (US)
  • University of Manitoba (CA)
  • Yale University (US)
  • University at Buffalo, State University of New York (US)
  • North West Armed Forces Hospital (SA)
  • University of California, San Francisco (US)
  • Tel Aviv University (IL)
  • Alder Hey Children's Hospital (GB)
  • Mayo Clinic (US)
  • University College Dublin (IE)
  • University of Rochester Medicine (US)
  • Alfred I. duPont Hospital for Children (US)
  • Lille University Hospital of Lille,Lille,France
  • Emory University (US)
  • University of Ljubljana,Ljubljana,Slovenia
  • University of Toronto (CA)
  • University of Helsinki (FI)
  • University of Liverpool (GB)
  • Amsterdam UMC Location University of Amsterdam (NL)
  • Shaare Zedek Medical Center (IL)
  • Columbia University College of Physicians and Surgeons,New York,NY
  • Children's National (US)
  • University College Dublin,Dublin,Ireland
  • National and Kapodistrian University of Athens (GR)
  • Memorial University of Newfoundland (CA)
  • University of Pittsburgh Medical Center (US)
  • Medical College of Wisconsin (US)
  • University of Alberta (CA)
Corresponding author
Deneau, Mark R.
Venue
Hepatology
Published
2017-06-26
Volume
66
Issue
2
Pages
518-527
ISSN
  • 0270-9139 (ISSN-L)
  • 1527-3350
OpenAlex
W2604766632
Cited by
230
References
39
Citations by year
2026: 22 · 2025: 21 · 2024: 28 · 2023: 27 · 2022: 19 · 2021: 34 · 2020: 20 · 2019: 26 · 2018: 23 · 2017: 8
FWCI
13.01
Type
journal article
Language
en
Open access
Locations
  • Hepatology (publishedVersion)
  • PubMed (publishedVersion)
  • IRIS Research product catalog (Sapienza University of Rome) (submittedVersion, other-oa)
  • Pure Amsterdam UMC (submittedVersion, other-oa)
  • Data Archiving and Networked Services (DANS) (submittedVersion)
  • Institutional Repositories DataBase (IRDB) (submittedVersion)
Funders
  • Gilead Sciences
  • Alexion Pharmaceuticals
  • Allergan
  • Research Committee, Aristotle University of Thessaloniki
Topics
  • Health Sciences › Medicine › Hepatology › Liver Diseases and Immunity
  • Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
  • Health Sciences › Medicine › Rheumatology › IgG4-Related and Inflammatory Diseases
Keywords
primary sclerosing cholangitis, natural history (disease), long-term outcomes, portal hypertension, biliary complications, cholangiocarcinoma, liver transplantation, inflammatory bowel disease, autoimmune hepatitis, prognostic factors, aspartate aminotransferase to platelet ratio index, gamma-glutamyl transferase, bilirubin
MeSH terms
Analysis of Variance, Biopsy, Needle, Child, Female, Follow-Up Studies, Humans, Immunohistochemistry, Japan, Liver Function Tests, Male, Predictive Value of Tests, Retrospective Studies, Severity of Illness Index, Cholangitis, Sclerosing, Cohort Studies, Multivariate Analysis, Proportional Hazards Models, Survival Analysis, Liver Transplantation, Disease Progression, Risk Assessment, Disease-Free Survival, Internationality

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