Condition-Specific Growth Charts for Children With Alagille Syndrome
Year: 2025
Type: article
Abstract: Importance: Different degrees of growth delay have been reported in children with Alagille syndrome (ALGS), yet these patients are routinely evaluated using standard growth charts. Objective: To develop condition-specific growth charts for ALGS using modern statistical approaches. Design, Setting, and Participants: 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. Exposure: Growth of children with Alagille syndrome. Main Outcomes and Measures: 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. Results: 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. Conclusions and Relevance: 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.
Source: JAMA Network Open
Authors: 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-Miedzińska, 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, Global Alagille Alliance (GALA) Study Group, 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 Dezsőfi-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
Institutions: Vrije Universiteit Brussel, University of Toronto, Hospital for Sick Children, Universitair Ziekenhuis Brussel, KU Leuven, Children's Hospital of Philadelphia, University of Pennsylvania, Stanford Medicine, Stanford University, Lucile Packard Children's Hospital, Children's Memorial Health Institute, Inserm, Université Paris-Saclay, Assistance Publique – Hôpitaux de Paris, Bicêtre Hospital, Emory University, Virginia Commonwealth University, Children's Healthcare of Atlanta, Oregon Health & Science University, Karolinska University Hospital, Karolinska Institutet, Fudan University, Children's Hospital of Fudan University, Yale University, Boston Children's Hospital, Harvard University, Asan Medical Center, University of Ulsan, Ospedale Papa Giovanni XXIII, University of Milano-Bicocca, Children's Mercy Hospital, Children's Hospital at Westmead, Mahidol University, Ramathibodi Hospital, Royal Children's Hospital, Narayana Health, Mazumdar Shaw Medical Centre, University of Pittsburgh, Children's Hospital Colorado, University of Colorado Anschutz, University of Minnesota, Birmingham Women’s and Children’s NHS Foundation Trust, University of Birmingham, Phoenix Children's Hospital, Primary Children's Hospital, University of Utah, Starship Children's Health, Cliniques Universitaires Saint-Luc, University Medical Center Groningen, University of Groningen, King's College London, University Health Network, Toronto General Hospital, University College London, Hôpital Necker-Enfants Malades, Université Paris Cité, Ljubljana University Medical Centre, Cairo University, University of Malaya, Hospital Italiano de Buenos Aires, University of the Witwatersrand, Chris Hani Baragwanath Hospital, University of California San Diego, Rady Children's Hospital-San Diego, University of Geneva, University Hospital of Geneva, Johns Hopkins University, University of Nebraska Medical Center, Institute of Liver and Biliary Sciences, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Schneider Children's Medical Center, Seattle Children's Hospital, University of Washington, Universidade do Porto, Centro Hospitalar do Porto, Istanbul University-Cerrahpaşa, Istanbul University, Semmelweis University, Universidade Federal de Ciências da Saúde de Porto Alegre, Hospital de Cruces, University of Tennessee Health Science Center, Le Bonheur Children's Hospital, Mitera Hospital, Meyer Children's Hospital, National and Kapodistrian University of Athens, Children's Hospital Agia Sophia, Azienda Ospedaliera Citta' della Salute e della Scienza di Torino, Ospedale Regina Margherita, Wits University Donald Gordon Medical Centre, Western University, London Health Sciences Centre, Children's Hospital of Western Ontario, Koç University, National Center For Child Health and Development, University of Rochester Medicine, Massachusetts General Hospital, University of Rochester, The University of Texas Southwestern Medical Center, University Medical Center Utrecht, Grupo Hospitalar Conceição, Hospital Universitario La Paz, Azienda Ospedaliera San Gerardo, Vall d'Hebron Hospital Universitari, ERN TRANSPLANT-CHILD, University of Miami, Miami Transplant Institute, Medical University of Silesia, Mohammed Bin Rashid University of Medicine and Health Sciences, University of Manitoba, Children's Hospital of Eastern Ontario, University of the Basque Country, Biogipuzkoa Health Research Institute, Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas
Language: English
Subfield: Surgery
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,000
Awards: G6942828855, G7117951472
OpenAlex ID: W4416591177