Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study

Autor: Shannon M, Vandriel, Li-Ting, Li, Huiyu, She, Jian-She, Wang, Melissa A, Gilbert, Irena, Jankowska, Piotr, Czubkowski, Dorota, Gliwicz-Miedzińska, Emmanuel M, Gonzales, Emmanuel, Jacquemin, Jérôme, Bouligand, Nancy B, Spinner, Kathleen M, Loomes, David A, Piccoli, Lorenzo, D'Antiga, Emanuele, Nicastro, Étienne, Sokal, Tanguy, Demaret, Noelle H, Ebel, Jeffrey A, Feinstein, Rima, Fawaz, Silvia, Nastasio, Florence, Lacaille, Dominique, Debray, Henrik, Arnell, Björn, Fischler, Susan, Siew, Michael, Stormon, Saul J, Karpen, Rene, Romero, Kyung Mo, Kim, Woo Yim, Baek, Winita, Hardikar, Sahana, Shankar, Amin J, Roberts, Helen M, Evans, M Kyle, Jensen, Marianne, Kavan, Shikha S, Sundaram, Alexander, Chaidez, Palaniswamy, Karthikeyan, Maria Camila, Sanchez, Maria Lorena, Cavalieri, Henkjan J, Verkade, Way Seah, Lee, James E, Squires, Christina, Hajinicolaou, Chatmanee, Lertudomphonwanit, Ryan T, Fischer, Catherine, Larson-Nath, Yael, Mozer-Glassberg, Cigdem, Arikan, Henry C, Lin, Jesus Quintero, Bernabeu, Seema, Alam, Deirdre A, Kelly, Elisa, Carvalho, Cristina Targa, Ferreira, Giuseppe, Indolfi, Ruben E, Quiros-Tejeira, Pinar, Bulut, Pier Luigi, Calvo, Zerrin, Önal, Pamela L, Valentino, Dev M, Desai, John, Eshun, Maria, Rogalidou, Antal, Dezsőfi, Sabina, Wiecek, Gabriella, Nebbia, Raquel Borges, Pinto, Victorien M, Wolters, María Legarda, Tamara, Andréanne N, Zizzo, Jennifer, Garcia, Kathleen, Schwarz, Marisa, Beretta, Thomas Damgaard, Sandahl, Carolina, Jimenez-Rivera, Nanda, Kerkar, Jernej, Brecelj, Quais, Mujawar, Nathalie, Rock, Cristina Molera, Busoms, Wikrom, Karnsakul, Eberhard, Lurz, Ermelinda, Santos-Silva, Niviann, Blondet, Luis, Bujanda, Uzma, Shah, Richard J, Thompson, Bettina E, Hansen, Binita M, Kamath
Přispěvatelé: Arıkan, Çiğdem (ORCID 0000-0002-0794-2741 & YÖK ID 240198), Vandriel, S.M., Li, L.T., She, H., Wang, J.S., Gilbert, M.A., Jankowska, I., Czubkowski, P., Gliwicz-Miedzi?ska, D., Gonzales, E.M., Jacquemin, E., Bouligand, J., Spinner, N.B., Loomes, K.M., Piccoli, D.A., D'Antiga, L., Nicastro, E., Sokal, É., Demaret, T., Ebel, N.H., Feinstein, J.A., Fawaz, R., Nastasio, S., Lacaille, F., Debray, D., Arnell, H., Fischler, B., Siew, S., Stormon, M., Karpen, S.J., Romero, R., Kim, K.M., Baek, W.Y., Hardikar, W., Shankar, S., Roberts, A.J., Evans, H.M., Jensen, M.K., Kavan, M., Sundaram, S.S., Chaidez, A., Karthikeyan, P., Sanchez, M.C., Cavalieri, M.L., Verkade, H.J., Lee, W.S., Squires, J.E., Hajinicolaou, C., Lertudomphonwanit, C., Fischer, R.T., Larson-Nath, C., Mozer-Glassberg, Y., Lin, H.C., Quintero, Bernabeu J., Alam, S., Kelly, D., Carvalho, E., Ferreira, C.T., Indolfi, G., Quiros-Tejeira, R.E., Bulut, P., Calvo, P.L., Önal, Z., Valentino, P.L., Desai, D.M., Eshun, J., Rogalidou, M., Dezs?fi, A., Wiecek, S., Nebbia, G., Borges Pinto, R., Wolters, V.M., Tamara, M.L., Zizzo, A.N., Garcia, J., Schwarz, K., Beretta, M., Sandahl, T.D., Jimenez-Rivera, C., Kerkar, N., Brecelj, J., Mujawar, Q., Rock, N., Busoms, C.M., Karnsakul, W., Lurz, E., Santos-Silva, E., Blondet, N., Bujanda, L., Shah, U., Thompson, R.J., Hansen, B.E., Kamath, B.M., Global ALagille Alliance (GALA) Study Group, Koç University Hospital, School of Medicine, UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, UCL - (SLuc) Service de gastro-entérologie et hépatologie pédiatrique
Rok vydání: 2022
Předmět:
Zdroj: Hepatology
Vandriel, S M, Li, L T, She, H, Wang, J S, Gilbert, M A, Jankowska, I, Czubkowski, P, Gliwicz-Miedzińska, D, Gonzales, E M, Jacquemin, E, Bouligand, J, Spinner, N B, Loomes, K M, Piccoli, D A, D'Antiga, L, Nicastro, E, Sokal, É, Demaret, T, Ebel, N H, Feinstein, J A, Fawaz, R, Nastasio, S, Lacaille, F, Debray, D, Arnell, H, Fischler, B, Siew, S, Stormon, M, Karpen, S J, Romero, R, Kim, K M, Baek, W Y, Hardikar, W, Shankar, S, Roberts, A J, Evans, H M, Jensen, M K, Kavan, M, Sundaram, S S, Chaidez, A, Karthikeyan, P, Sanchez, M C, Cavalieri, M L, Verkade, H J, Lee, W S, Squires, J E, Hajinicolaou, C, Lertudomphonwanit, C, Fischer, R T, Sandahl, T D & The Global ALagille Alliance (GALA) Study Group 2023, ' Natural history of liver disease in a large international cohort of children with Alagille syndrome : Results from the GALA study ', Hepatology, vol. 77, no. 2 . https://doi.org/10.1002/hep.32761
Hepatology, Vol. 77, no. 2, p. 512-529 (2023)
ISSN: 0270-9139
DOI: 10.1002/hep.32761
Popis: 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 = 5.0 and = 10.0 mg/dl had an 8.0-fold (95% CI, 3.4-18.4) increased risk of developing CEPH compared with those 10.0 mg/dl were associated with a 4.8 (95% CI, 2.4-9.7) and 15.6 (95% CI, 8.7-28.2) increased risk of transplantation relative to = 5.0 mg/dl, with 79% reaching adulthood with native liver (p < 0.001). Conclusions: in this large international cohort of ALGS, only 40.3% of children reach adulthood with their native liver. A TB
This study received funding support from the following agencies: The Alagille Syndrome Alliance, Mirum Pharmaceuticals Inc. and Albireo Pharma, Inc. who provided unrestricted educational grants to the Hospital for Sick Children (SickKids Foundation). The study sponsors were not involved in the conduct of the research study or preparation of the manuscript.
Databáze: OpenAIRE
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