| Title: |
Condition-Specific Growth Charts for Children With Alagille Syndrome |
| Authors: |
Huysentruyt,Koen; Vandriel,Shannon M; Roelants,Mathieu; Piccoli,David A; Loomes,Kathleen M; Rand,Elizabeth B; Ebel,Noelle H; Feinstein,Jeffrey A; Jankowska,Irena; Czubkowski,Piotr; Gliwicz-Miedzinska,Dorota; Gonzales,Emmanuel M; Jacquemin,Emmanuel; Bouligand,Jérôme; Karpen,Saul J; Romero,Rene; Lin,Henry C; Fischler,Björn; Arnell,Henrik; Li,Li-Ting; Wang,Jian-She; Fawaz,Rima; Nastasio,Silvia; Kim,Kyung Mo; Oh,Seak Hee; D'Antiga,Lorenzo; Nicastro,Emanuele; Fischer,Ryan T; Siew,Susan M; Stormon,Michael; Lertudomphonwanit,Chatmanee; Hardikar,Winita; Shankar,Sahana; Squires,James E; Sundaram,Shikha S; Larson-Nath,Catherine; Kelly,Deirdre A; Hartley,Jane; Bulut,Pinar; Jensen,M Kyle; Jaramillo,Catalina; Roberts,Amin J; Evans,Helen M; Sokal,Étienne M; Demaret,Tanguy; Verkade,Henkjan J; Thompson,Richard J; Hansen,Bettina E; Cole,Tim J; Wolters, Victorien M; Global ALagille Alliance (GALA) Study Group; MDL; Child Health |
| Publication Year: |
2025 |
| Subject Terms: |
Alagille Syndrome/physiopathology; Body Height; Body Weight; Child; Preschool; Female; Growth Charts; Humans; Infant; Newborn; Male; Journal Article; Multicenter Study |
| Description: |
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 ... |
| Document Type: |
article in journal/newspaper |
| File Description: |
application/pdf |
| Language: |
English |
| ISSN: |
2574-3805 |
| Relation: |
https://dspace.library.uu.nl/handle/1874/468417 |
| Availability: |
https://dspace.library.uu.nl/handle/1874/468417 |
| Rights: |
info:eu-repo/semantics/OpenAccess |
| Accession Number: |
edsbas.F3CBBD13 |
| Database: |
BASE |