Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group

Cholestasis; Paediatrics

Detalles Bibliográficos
Autores: Murillo Perez, Carla Fiorella, GONZALES, Emmanuel, Li, Liting, She, Huiyu, Vandriel, Shannon, wang, jianshe, quintero, jesus
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Institución:Departament de Salut de la Generalitat de Catalunya (DS)
Repositorio:Scientia. Dipòsit d'Informació Digital del Departament de Salut
OAI Identifier:oai:dnet:scientia____::4384df93d6e46053cb5ac49ed92f3e6b
Acceso en línea:http://hdl.handle.net/11351/14582
Access Level:acceso abierto
Palabra clave:Infants
Colèstasi
Àcids biliars
Conductes biliars - Malalties
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis::Cholestasis, Intrahepatic::Alagille Syndrome
CHEMICALS AND DRUGS::Polycyclic Compounds::Fused-Ring Compounds::Steroids::Bile Acids and Salts
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis
NAMED GROUPS::Persons::Age Groups::Child
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis::colestasis intrahepática::síndrome de Alagille
COMPUESTOS QUÍMICOS Y DROGAS::compuestos policíclicos::compuestos con anillos de fusión::esteroides::ácidos y sales biliares
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis
DENOMINACIONES DE GRUPOS::personas::Grupos de Edad::niño
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oai_identifier_str oai:dnet:scientia____::4384df93d6e46053cb5ac49ed92f3e6b
network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
title Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
spellingShingle Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
Murillo Perez, Carla Fiorella
Infants
Colèstasi
Àcids biliars
Conductes biliars - Malalties
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis::Cholestasis, Intrahepatic::Alagille Syndrome
CHEMICALS AND DRUGS::Polycyclic Compounds::Fused-Ring Compounds::Steroids::Bile Acids and Salts
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis
NAMED GROUPS::Persons::Age Groups::Child
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis::colestasis intrahepática::síndrome de Alagille
COMPUESTOS QUÍMICOS Y DROGAS::compuestos policíclicos::compuestos con anillos de fusión::esteroides::ácidos y sales biliares
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis
DENOMINACIONES DE GRUPOS::personas::Grupos de Edad::niño
title_short Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
title_full Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
title_fullStr Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
title_full_unstemmed Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
title_sort Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group
dc.creator.none.fl_str_mv Murillo Perez, Carla Fiorella
GONZALES, Emmanuel
Li, Liting
She, Huiyu
Vandriel, Shannon
wang, jianshe
quintero, jesus
author Murillo Perez, Carla Fiorella
author_facet Murillo Perez, Carla Fiorella
GONZALES, Emmanuel
Li, Liting
She, Huiyu
Vandriel, Shannon
wang, jianshe
quintero, jesus
author_role author
author2 GONZALES, Emmanuel
Li, Liting
She, Huiyu
Vandriel, Shannon
wang, jianshe
quintero, jesus
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv Institut Català de la Salut
[Murillo Perez CF, Vandriel SM] Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Canada. [Gonzales EM] Pediatric Hepatology and Liver Transplantation Unit, National Reference Centre for Rare Pediatric Liver Diseases, FILFOIE, ERN RARE LIVER, Bicêtre Hospital, AP-HP, Université Paris-Saclay, Le Kremlin-Bicêtre, and Inserm U1193, Hepatinov, University of ParisSaclay, Orsay, France. [Wang JS, Li LT, She H] Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China. [Quintero Bernabeu J] Servei d’Hepatologia i Trasplantament Hepàtic Pediàtric, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Department of Liver and Gastrointestinal Diseases, Biodonostia Health Research Institute – Donostia University Hospital – University of the Basque Country (UPV/EHU), San Sebastian, Spain
Vall d'Hebron Barcelona Hospital Campus
dc.subject.none.fl_str_mv Infants
Colèstasi
Àcids biliars
Conductes biliars - Malalties
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis::Cholestasis, Intrahepatic::Alagille Syndrome
CHEMICALS AND DRUGS::Polycyclic Compounds::Fused-Ring Compounds::Steroids::Bile Acids and Salts
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis
NAMED GROUPS::Persons::Age Groups::Child
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis::colestasis intrahepática::síndrome de Alagille
COMPUESTOS QUÍMICOS Y DROGAS::compuestos policíclicos::compuestos con anillos de fusión::esteroides::ácidos y sales biliares
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis
DENOMINACIONES DE GRUPOS::personas::Grupos de Edad::niño
topic Infants
Colèstasi
Àcids biliars
Conductes biliars - Malalties
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis::Cholestasis, Intrahepatic::Alagille Syndrome
CHEMICALS AND DRUGS::Polycyclic Compounds::Fused-Ring Compounds::Steroids::Bile Acids and Salts
DISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis
NAMED GROUPS::Persons::Age Groups::Child
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis::colestasis intrahepática::síndrome de Alagille
COMPUESTOS QUÍMICOS Y DROGAS::compuestos policíclicos::compuestos con anillos de fusión::esteroides::ácidos y sales biliares
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis
DENOMINACIONES DE GRUPOS::personas::Grupos de Edad::niño
description Cholestasis; Paediatrics
publishDate 2025
dc.date.none.fl_str_mv 2025
2026
2026
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/11351/14582
url http://hdl.handle.net/11351/14582
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Liver International;45(12)
https://doi.org/10.1111/liv.70423
dc.rights.none.fl_str_mv Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Wiley
publisher.none.fl_str_mv Wiley
dc.source.none.fl_str_mv Scientia
reponame:Scientia. Dipòsit d'Informació Digital del Departament de Salut
instname:Departament de Salut de la Generalitat de Catalunya (DS)
instname_str Departament de Salut de la Generalitat de Catalunya (DS)
reponame_str Scientia. Dipòsit d'Informació Digital del Departament de Salut
collection Scientia. Dipòsit d'Informació Digital del Departament de Salut
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869422733655277568
spelling Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study GroupMurillo Perez, Carla FiorellaGONZALES, EmmanuelLi, LitingShe, HuiyuVandriel, Shannonwang, jianshequintero, jesusInfantsColèstasiÀcids biliarsConductes biliars - MalaltiesDISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::Cholestasis::Cholestasis, Intrahepatic::Alagille SyndromeCHEMICALS AND DRUGS::Polycyclic Compounds::Fused-Ring Compounds::Steroids::Bile Acids and SaltsDISEASES::Digestive System Diseases::Biliary Tract Diseases::Bile Duct Diseases::CholestasisNAMED GROUPS::Persons::Age Groups::ChildENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasis::colestasis intrahepática::síndrome de AlagilleCOMPUESTOS QUÍMICOS Y DROGAS::compuestos policíclicos::compuestos con anillos de fusión::esteroides::ácidos y sales biliaresENFERMEDADES::enfermedades del sistema digestivo::enfermedades de la vía biliar::enfermedades de los conductos biliares::colestasisDENOMINACIONES DE GRUPOS::personas::Grupos de Edad::niñoCholestasis; PaediatricsColèstasi; PediatriaColestasis; PediatríaBackground and Aim Alagille syndrome (ALGS) is a rare disorder characterised by cholestasis and extrahepatic manifestations. Given the current era of ileal bile acid transporter (IBAT) inhibitor therapies that reduce serum bile acid (SBA) levels, we evaluated whether SBA predicts liver disease outcomes in ALGS. Methods Patients were ascertained from the Global ALagille Alliance (GALA) cohort. A prognostic threshold of SBA 102 μmol/L was assessed as a time-dependent covariate in Cox regression analyses for native liver survival (NLS) and event-free survival (EFS), while adjusting for total bilirubin (TB) levels. Results 570 GALA patients were included (348 [61%] male). There was a moderate positive correlation between SBA and TB (Pearson correlation = 0.47, p < 0.001). SBA below 102 μmol/L was a significant predictor of outcomes (NLS: HR = 3.78, 95% CI 2.39–5.99, p < 0.001; EFS: HR = 3.44, 95% CI 2.35–5.04, p < 0.001). SBA remained a significant predictor for improved EFS after adjusting for TB clearance at 1 year (TB < 2 mg/dL; HR = 2.00, 95% CI 1.10–3.65, p = 0.02). Median SBA in the first year of life above 102 μmol/L, predicted lower NLS (67.2% vs. 83.5% at 7 years p = 0.05) and EFS (63.4% vs. 80.9% at 7 years, p = 0.02). Conclusion Lower SBA in children with ALGS liver disease predicts improved NLS and EFS. SBA is also associated with NLS in children with ALGS who clear their bilirubin, that is, those with anicteric cholestasis. Although the patients studied here did not receive IBAT inhibition, these data suggest that lowering SBA may improve important clinical outcomes.This work was supported by the Alagille Syndrome Alliance, Mirum Pharmaceuticals Inc., and Ipsen, who provided unrestricted educational grants to the Hospital for Sick Children (SickKids Foundation). The National Natural Science Foundation of China (81873543 and 81570468) provided funding to the Children's Hospital of Fudan University, the Center for Paediatric Liver Diseases, Shanghai, China.WileyInstitut Català de la Salut[Murillo Perez CF, Vandriel SM] Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Canada. [Gonzales EM] Pediatric Hepatology and Liver Transplantation Unit, National Reference Centre for Rare Pediatric Liver Diseases, FILFOIE, ERN RARE LIVER, Bicêtre Hospital, AP-HP, Université Paris-Saclay, Le Kremlin-Bicêtre, and Inserm U1193, Hepatinov, University of ParisSaclay, Orsay, France. [Wang JS, Li LT, She H] Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China. [Quintero Bernabeu J] Servei d’Hepatologia i Trasplantament Hepàtic Pediàtric, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Department of Liver and Gastrointestinal Diseases, Biodonostia Health Research Institute – Donostia University Hospital – University of the Basque Country (UPV/EHU), San Sebastian, SpainVall d'Hebron Barcelona Hospital Campus202620262025info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/11351/14582Scientiareponame:Scientia. Dipòsit d'Informació Digital del Departament de Salutinstname:Departament de Salut de la Generalitat de Catalunya (DS)InglésLiver International;45(12)https://doi.org/10.1111/liv.70423Attribution-NonCommercial 4.0 Internationalhttp://creativecommons.org/licenses/by-nc/4.0/info:eu-repo/semantics/openAccessoai:dnet:scientia____::4384df93d6e46053cb5ac49ed92f3e6b2026-06-12T09:38:37Z
score 15,812429

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