Condition-Specific Growth Charts for Children With Alagille Syndrome

Koen Huysentruyt; Shannon M Vandriel; Mathieu Roelants; David A Piccoli; Kathleen M Loomes; Elizabeth B Rand; Noelle H Ebel; Jeffrey A Feinstein; Irena Jankowska; Piotr Czubkowski; Dorota Gliwicz-Miedzinska; Emmanuel M Gonzales; Emmanuel Jacquemin; Jérôme Bouligand; Saul J Karpen; Rene Romero; Henry C Lin; Björn Fischler; Henrik Arnell; Li-Ting Li; Jian-She Wang; Rima Fawaz; Silvia Nastasio; Kyung Mo Kim; Seak Hee Oh; Lorenzo D'Antiga; Emanuele Nicastro; Ryan T Fischer; Susan M Siew; Michael Stormon; Chatmanee Lertudomphonwanit; Winita Hardikar; Sahana Shankar; James E Squires; Shikha S Sundaram; Catherine Larson-Nath; Deirdre A Kelly; Jane Hartley; Pinar Bulut; M Kyle Jensen; Catalina Jaramillo; Amin J Roberts; Helen M Evans; Étienne M Sokal; Tanguy Demaret; Henkjan J Verkade; Richard J Thompson; Bettina E Hansen; Tim J Cole; Binita M Kamath; Dominique Debray; Florence Lacaille; Jernej Brecelj; Nehal M El-Koofy; Mohamed A Elmonem; Way Seah Lee; Maria Camila Sanchez; Maria Lorena Cavalieri; Christina Hajinicolaou; Kathleen B Schwarz; Elisa Carvalho; Nathalie Rock; Wikrom Karnsakul; Ruben E Quiros-Tejeira; Seema Alam; Gabriella Nebbia; Yael Mozer-Glassberg; Pamela L Valentino; Ermelinda Santos-Silva; Zerrin Önal; Antal Dezsofi-Gottl; Melina Melere; María Legarda Tamara; John Eshun; Aglaia Zellos; Giuseppe Indolfi; Maria Rogalidou; Niviann Blondet; Pier Luigi Calvo; Marisa Beretta; Andréanne N Zizzo; Cigdem Arikan; Mureo Kasahara; Nanda Kerkar; Amal A Aqul; Victorien M Wolters; Raquel Borges Pinto; Jesus Quintero Bernabeu; Jennifer Garcia; Sabina Wiecek; Christos Tzivinikos; Quais Mujawar; Carolina Jimenez-Rivera; Cristina Molera Busoms; Cristina Gonçalves; Luis Bujanda; Global Alagille Alliance (GALA) Study Group

doi:10.1001/jamanetworkopen.2025.45294

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Condition-Specific Growth Charts for Children With Alagille Syndrome

Journal article

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Peer reviewed

Condition-Specific Growth Charts for Children With Alagille Syndrome

Koen Huysentruyt, Shannon M Vandriel, Mathieu Roelants, David A Piccoli, Kathleen M Loomes, Elizabeth B Rand, Noelle H Ebel, Jeffrey A Feinstein, Irena Jankowska, Piotr Czubkowski, …

JAMA network open, Vol.8(11), p.e2545294

2025-11-03

DOI: https://doi.org/10.1001/jamanetworkopen.2025.45294

PMID: 41284294

Appears in Miller School of Medicine - Latest Publications

Abstract

Alagille Syndrome - complications

Alagille Syndrome - physiopathology

Body Height

Body Weight

Child

Child, Preschool

Female

Growth Charts

Humans

Infant

Infant, Newborn

Male

Different degrees of growth delay have been reported in children with Alagille syndrome (ALGS), yet these patients are routinely evaluated using standard growth charts. To develop condition-specific growth charts for ALGS using modern statistical approaches. This case series used data from the international, multicenter Global Alagille Alliance (GALA) study accrued between May 14, 2018, and March 20, 2023. Children born at full term between January 1, 1997, and August 31, 2019, with a clinically and/or genetically confirmed ALGS diagnosis and their native liver were included. Data from children with a known history of prematurity were excluded for the development of the growth charts. Data were analyzed from March 25, 2023, to December 30, 2024. Growth of children with Alagille syndrome. Generalized additive models for location scale and shape were fitted to generate percentile plots for weight and height relative to age and superimposed on US Centers for Disease Control and Prevention (CDC) growth charts to illustrate differences in growth patterns compared with children with typical development. Data from 1204 children with ALGS in overlapping cohorts (median [IQR] gestational age, 38 [37-39] weeks) were analyzed (1204 in the weight cohort; 695 boys [57.7%]; 9855 weight observations; 995 with neonatal cholestasis [82.6%]; 306 receiving a liver transplant [25.4%]; 98 deaths [8.1%] and 1106 in the height cohort, 635 boys [57.4%]; 8464 height observations; 906 with neonatal cholestasis [81.9%]; 287 receiving a liver transplant [25.9%]; 86 deaths [7.8%]) were included for the modeling of the weight-for-age and height-for-age charts, respectively. The median birth weight was 2.8 kg (IQR, 2.5-3.0 kg) for boys and 2.6 kg (IQR, 2.4-2.9 kg) for girls. The median birth length was 48.0 cm (IQR, 46.0-50.0 cm) for boys and 47.0 cm (IQR, 45.0-49.0 cm) for girls. The weight-for-age and height-for-age growth charts for boys and girls with AGLS differed significantly from CDC growth charts. The estimated height at age 18 years corresponded to the 50th percentile was 171.5 cm for boys and 156.5 cm for girls on the condition-specific charts vs 176 cm and 163 cm, respectively, on the CDC growth charts. These findings suggest that condition-specific growth charts for ALGS may provide a crucial tool for clinicians to evaluate growth and aid in decision-making around listing children for liver transplant.

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Title: Condition-Specific Growth Charts for Children With Alagille Syndrome
Creators: Koen Huysentruyt - University of Toronto
Shannon M Vandriel - University of Toronto
Mathieu Roelants - KU Leuven
David A Piccoli - University of Pennsylvania
Kathleen M Loomes - University of Pennsylvania
Elizabeth B Rand - University of Pennsylvania
Noelle H Ebel - Stanford University School of Medicine
Jeffrey A Feinstein - Stanford University School of Medicine
Irena Jankowska - Children's Memorial Health Institute
Piotr Czubkowski - Children's Memorial Health Institute
Dorota Gliwicz-Miedzinska - Children's Memorial Health Institute
Emmanuel M Gonzales - Université Paris-Saclay
Emmanuel Jacquemin - Inserm
Jérôme Bouligand - Assistance Publique – Hôpitaux de Paris
Saul J Karpen - Virginia Commonwealth University
Rene Romero - Emory University School of Medicine
Henry C Lin - University of Portland
Björn Fischler - Karolinska University Hospital
Henrik Arnell - Karolinska University Hospital
Li-Ting Li - Children's Hospital of Fudan University
Jian-She Wang - Children's Hospital of Fudan University
Rima Fawaz - Yale University
Silvia Nastasio - Boston Children's Hospital
Kyung Mo Kim - University of Ulsan
Seak Hee Oh - Asan Medical Center
Lorenzo D'Antiga - University of Milano-Bicocca
Emanuele Nicastro - Ospedale Papa Giovanni XXIII
Ryan T Fischer - Sisters of Mercy Health System
Susan M Siew - Children's Hospital at Westmead
Michael Stormon - Children's Hospital at Westmead
Chatmanee Lertudomphonwanit - Ramathibodi Hospital
Winita Hardikar - Royal Children's Hospital
Sahana Shankar - Narayana Health
James E Squires - University of Pittsburgh School of Medicine
Shikha S Sundaram - Children's Hospital Colorado
Catherine Larson-Nath - University of Minnesota
Deirdre A Kelly - University of Birmingham
Jane Hartley - University of Birmingham
Pinar Bulut - Phoenix Children's Hospital
M Kyle Jensen - University of Utah
Catalina Jaramillo - University of Utah
Amin J Roberts - Starship Children's Health
Helen M Evans - Starship Children's Health
Étienne M Sokal - Cliniques Universitaires Saint-Luc
Tanguy Demaret - Cliniques Universitaires Saint-Luc
Henkjan J Verkade - University Medical Center Groningen
Richard J Thompson - King's College London
Bettina E Hansen - University Health Network
Tim J Cole - Great Ormond Street Hospital
Binita M Kamath - University of Toronto
Dominique Debray - Pediatric Liver Unit, National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis), French Network for Rare Liver Disease, European Reference Network on Hepatological Diseases, Necker-Enfants Malades Hospital, University of Paris, Paris, France
Florence Lacaille - Hôpital Necker-Enfants Malades
Jernej Brecelj - Pediatric Gastroenterology, Hepatology and Nutrition and Department of Pediatrics, Faculty of Medicine, University Medical Center Ljubljana, Ljubljana, Slovenia
Nehal M El-Koofy - Cairo University
Mohamed A Elmonem - Cairo University
Way Seah Lee - University of Malaya
Maria Camila Sanchez - Hospital Italiano de Buenos Aires
Maria Lorena Cavalieri - Hospital Italiano de Buenos Aires
Christina Hajinicolaou - Chris Hani Baragwanath Hospital
Kathleen B Schwarz - University of California San Diego
Elisa Carvalho - Pediatric Gastroenterology Department, Hospital da Crianca de Brasilia, Centro Universitario de Brasilia, Brasilia, Federal District, Brazil
Nathalie Rock - University of Geneva
Wikrom Karnsakul - Johns Hopkins Medicine
Ruben E Quiros-Tejeira - University of Nebraska Medical Center
Seema Alam - Institute of Liver and Biliary Sciences
Gabriella Nebbia - Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Yael Mozer-Glassberg - Schneider Children's Medical Center
Pamela L Valentino - University of Washington
Ermelinda Santos-Silva - Universidade do Porto
Zerrin Önal - Istanbul University
Antal Dezsofi-Gottl - Semmelweis University
Melina Melere - Universidade Federal de Ciências da Saúde de Porto Alegre
María Legarda Tamara - Paediatric Gastroenterology Unit, Cruces University Hospital, Bilbao, Spain
John Eshun - University of Tennessee Health Science Center
Aglaia Zellos - Mitera Hospital
Giuseppe Indolfi - Meyer Children's Hospital
Maria Rogalidou - National and Kapodistrian University of Athens
Niviann Blondet - University of Washington
Pier Luigi Calvo - Pediatric Gastroenterology Unit, Regina Margherita Children's Hospital, Azienda Ospedaliera-Universitaria Citta' della Salute e della Scienza, Turin, Italy
Marisa Beretta - University of the Witwatersrand
Andréanne N Zizzo - University of London
Cigdem Arikan - Koç University
Mureo Kasahara - National Center For Child Health and Development
Nanda Kerkar - University of Rochester Medical Center
Amal A Aqul - The University of Texas Southwestern Medical Center
Victorien M Wolters - University Medical Center Utrecht
Raquel Borges Pinto - Grupo Hospitalar Conceição
Jesus Quintero Bernabeu - ERN TRANSPLANT-CHILD
Jennifer Garcia - Division of Pediatric Gastroenterology, Hepatology and Nutrition, Miami Transplant Institute, University of Miami, Miami, Florida
Sabina Wiecek - Medical University of Silesia
Christos Tzivinikos - Mohammed Bin Rashid University of Medicine and Health Sciences
Quais Mujawar - University of Manitoba
Carolina Jimenez-Rivera - Children's Hospital of Eastern Ontario
Cristina Molera Busoms - Pediatric Gastroenterology Hepatology and Nutrition Unit, Hospital Sant Joan de Deu, Esplugues de Llobregat, Spain
Cristina Gonçalves - Pediatric Gastroenterology/Hepatology Center, European Reference Network on Hepatological Diseases, Lisbon, Portugal
Luis Bujanda - Department of Hepatology and Gastroenterology, Biodonostia Health Research Institute-Donostia University Hospital, Universidad del Pais Vasco, Centro de Investigacion Biomedica en Red de Enfermedades Hepaticas y Digestivas, San Sebastian, Spain
Global Alagille Alliance (GALA) Study Group
Publication Details: JAMA network open, Vol.8(11), p.e2545294
Publisher: AMER MEDICAL ASSOC; CHICAGO
Number of pages: 15
Grant note: Hospital for Sick Children (SickKids Foundation) from the Alagille Syndrome AllianceIpsenCenter for Pediatric Liver Diseases, Children's Hospital of Fudan University: 81873543, 81570468 National Natural Science Foundation of China
This work was funded by unrestricted educational grants to the Hospital for Sick Children (SickKids Foundation) from the Alagille Syndrome Alliance, Mirum Pharmaceuticals, and Ipsen. Funding to the Center for Pediatric Liver Diseases, Children's Hospital of Fudan University, was provided by grants 81873543 and 81570468 from the National Natural Science Foundation of China.
Academic Unit: UMMG Dept of Pediatrics - Gastroenterology; Miller School of Medicine; UMMG Department of Pediatrics
Language: English
Resource Type: Journal article
PMID: 41284294
Record Identifier: 991032880900102976