| Title: |
Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study |
| Authors: |
Vandriel, SM; Li, LT; She, H; Wang, JS; Gilbert, MA; Jankowska, I; Czubkowski, P; Gliwicz-Miedzińska, D; Gonzales, EM; Jacquemin, E; Bouligand, J; Spinner, NB; Loomes, KM; Piccoli, DA; D'Antiga, L; Nicastro, E; Sokal, A; Demaret, T; Ebel, NH; Feinstein, JA; Fawaz, R; Nastasio, S; Lacaille, F; Debray, D; Arnell, H; Fischler, B; Siew, S; Stormon, M; Karpen, SJ; Romero, R; Kim, KM; Baek, WY; Hardikar, W; Shankar, S; Roberts, AJ; Evans, HM; Jensen, MK; Kavan, M; Sundaram, SS; Chaidez, A; Karthikeyan, P; Sanchez, MC; Cavalieri, ML; Verkade, HJ; Lee, WS; Squires, JE; Hajinicolaou, C; Lertudomphonwanit, C; Fischer, RT; Larson-Nath, C; Mozer-Glassberg, Y; Arikan, C; Lin, HC; Bernabeu, JQ; Alam, S; Kelly, DA; Carvalho, E; Ferreira, CT; Indolfi, G; Quiros-Tejeira, RE; Bulut, P; Calvo, PL; Anal, Z; Valentino, PL; Desai, DM; Eshun, J; Rogalidou, M; Dezsőfi, A; Wiecek, S; Nebbia, G; Pinto, RB; Wolters, VM; Tamara, ML; Zizzo, AN; Garcia, J; Schwarz, K; Beretta, M; Sandahl, TD; Jimenez-Rivera, C; Kerkar, N; Brecelj, J; Mujawar, Q; Rock, N; Busoms, CM; Karnsakul, W; Lurz, E; Santos-Silva, E; Blondet, N; Bujanda, L; Shah, U; Thompson, RJ; Hansen, BE; Kamath, BM |
| Publisher Information: |
LIPPINCOTT WILLIAMS & WILKINS |
| Publication Year: |
2023 |
| Collection: |
The University of Melbourne: Digital Repository |
| Description: |
BACKGROUND AND AIMS: Alagille syndrome (ALGS) is a multisystem disorder, characterized by cholestasis. Existing outcome data are largely derived from tertiary centers, and real-world data are lacking. This study aimed to elucidate the natural history of liver disease in a contemporary, international cohort of children with ALGS. APPROACH AND RESULTS: This was a multicenter retrospective study of children with a clinically and/or genetically confirmed ALGS diagnosis, born between January 1997 and August 2019. Native liver survival (NLS) and event-free survival rates were assessed. Cox models were constructed to identify early biochemical predictors of clinically evident portal hypertension (CEPH) and NLS. In total, 1433 children (57% male) from 67 centers in 29 countries were included. The 10 and 18-year NLS rates were 54.4% and 40.3%. By 10 and 18 years, 51.5% and 66.0% of children with ALGS experienced ≥1 adverse liver-related event (CEPH, transplant, or death). Children (>6 and ≤12 months) with median total bilirubin (TB) levels between ≥5.0 and |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| ISSN: |
0270-9139 |
| Relation: |
https://hdl.handle.net/11343/335866 |
| Availability: |
https://hdl.handle.net/11343/335866 |
| Rights: |
https://creativecommons.org/licenses/by-nc/4.0 ; CC BY-NC |
| Accession Number: |
edsbas.64680D05 |
| Database: |
BASE |