Assessing the validity of adult-derived ...
Type de document :
Article dans une revue scientifique: Article original
PMID :
URL permanente :
Titre :
Assessing the validity of adult-derived prognostic models for primary sclerosing cholangitis outcomes in children
Auteur(s) :
Deneau, Mark [Auteur]
Valentino, Pamela [Auteur]
Mack, Cara [Auteur]
Alqoaer, Khaled [Auteur]
Amin, Mansi [Auteur]
Amir, Achiya [Auteur]
Aumar, Madeleine [Auteur]
Auth, Marcus [Auteur]
Broderick, Annemarie [Auteur]
Diguglielmo, Matthew [Auteur]
Draijer, Laura G. [Auteur]
El-Matary, Wael [Auteur]
Ferrari, Federica [Auteur]
Furuya, Katryn [Auteur]
gottrand, Fréderic [Auteur]
Lille Inflammation Research International Center (LIRIC) - U995
Gupta, Nitika [Auteur]
Homan, Matjaz [Auteur]
Jensen, M. Kyle [Auteur]
Kamath, Binita M. [Auteur]
Kim, Kyung Mo [Auteur]
Kolho, Kaija-Leena [Auteur]
Koot, Bart [Auteur]
Iorio, Raffaele [Auteur]
Martinez, Mercedes [Auteur]
Miloh, Tamir [Auteur]
Mohan, Parvathi [Auteur]
Palle, Sirish [Auteur]
Papadopoulou, Alexandra [Auteur]
Ricciuto, Amanda [Auteur]
Saubermann, Lawrence [Auteur]
Sathya, Pushpa [Auteur]
Shteyer, Eyal [Auteur]
Smolka, Vratislav [Auteur]
Tanaka, Atsushi [Auteur]
Varier, Raghu [Auteur]
Venkat, Veena [Auteur]
Vitola, Bernadette [Auteur]
Woynarowski, Marek [Auteur]
Guthery, Stephen [Auteur]
Valentino, Pamela [Auteur]
Mack, Cara [Auteur]
Alqoaer, Khaled [Auteur]
Amin, Mansi [Auteur]
Amir, Achiya [Auteur]
Aumar, Madeleine [Auteur]
Auth, Marcus [Auteur]
Broderick, Annemarie [Auteur]
Diguglielmo, Matthew [Auteur]
Draijer, Laura G. [Auteur]
El-Matary, Wael [Auteur]
Ferrari, Federica [Auteur]
Furuya, Katryn [Auteur]
gottrand, Fréderic [Auteur]
Lille Inflammation Research International Center (LIRIC) - U995
Gupta, Nitika [Auteur]
Homan, Matjaz [Auteur]
Jensen, M. Kyle [Auteur]
Kamath, Binita M. [Auteur]
Kim, Kyung Mo [Auteur]
Kolho, Kaija-Leena [Auteur]
Koot, Bart [Auteur]
Iorio, Raffaele [Auteur]
Martinez, Mercedes [Auteur]
Miloh, Tamir [Auteur]
Mohan, Parvathi [Auteur]
Palle, Sirish [Auteur]
Papadopoulou, Alexandra [Auteur]
Ricciuto, Amanda [Auteur]
Saubermann, Lawrence [Auteur]
Sathya, Pushpa [Auteur]
Shteyer, Eyal [Auteur]
Smolka, Vratislav [Auteur]
Tanaka, Atsushi [Auteur]
Varier, Raghu [Auteur]
Venkat, Veena [Auteur]
Vitola, Bernadette [Auteur]
Woynarowski, Marek [Auteur]
Guthery, Stephen [Auteur]
Titre de la revue :
Journal of Pediatric Gastroenterology and Nutrition
Nom court de la revue :
J. Pediatr. Gastroenterol. Nutr.
Numéro :
70
Pagination :
e12-e17
Date de publication :
2020-01
ISSN :
1536-4801
Mot(s)-clé(s) en anglais :
primary sclerosing cholangitis
prognosis
pediatric
risk stratification
natural history
prognosis
pediatric
risk stratification
natural history
Discipline(s) HAL :
Sciences du Vivant [q-bio]
Résumé en anglais : [en]
Natural history models for primary sclerosing cholangitis (PSC) are derived from adult patient data, but have never been validated in children. It is unclear how accurate such models are for children with PSC.
We utilized ...
Lire la suite >Natural history models for primary sclerosing cholangitis (PSC) are derived from adult patient data, but have never been validated in children. It is unclear how accurate such models are for children with PSC. We utilized the pediatric PSC consortium database to assess the Revised Mayo Clinic, Amsterdam-Oxford, and Boberg models. We calculated the risk stratum and predicted survival for each patient within each model using patient data at PSC diagnosis, and compared it with observed survival. We evaluated model fit using the c-statistic. Model fit was good at 1 year (c-statistics 0.93, 0.87, 0.82) and fair at 10 years (0.78, 0.75, 0.69) in the Mayo, Boberg, and Amsterdam-Oxford models, respectively. The Mayo model correctly classified most children as low risk, whereas the Amsterdam-Oxford model incorrectly classified most as high risk. All of the models underestimated survival of patients classified as high risk. Albumin, bilirubin, AST, and platelets were most associated with outcomes. Autoimmune hepatitis was more prevalent in higher risk groups, and over-weighting of AST in these patients accounted for the observed versus predicted survival discrepancy. All 3 models offered good short-term discrimination of outcomes but only fair long-term discrimination. None of the models account for the high prevalence of features of autoimmune hepatitis overlap in children and the associated elevated aminotransferases. A pediatric-specific model is needed. AST, bilirubin, albumin, and platelets will be important predictors, but must be weighted to account for the unique features of PSC in children.Lire moins >
Lire la suite >Natural history models for primary sclerosing cholangitis (PSC) are derived from adult patient data, but have never been validated in children. It is unclear how accurate such models are for children with PSC. We utilized the pediatric PSC consortium database to assess the Revised Mayo Clinic, Amsterdam-Oxford, and Boberg models. We calculated the risk stratum and predicted survival for each patient within each model using patient data at PSC diagnosis, and compared it with observed survival. We evaluated model fit using the c-statistic. Model fit was good at 1 year (c-statistics 0.93, 0.87, 0.82) and fair at 10 years (0.78, 0.75, 0.69) in the Mayo, Boberg, and Amsterdam-Oxford models, respectively. The Mayo model correctly classified most children as low risk, whereas the Amsterdam-Oxford model incorrectly classified most as high risk. All of the models underestimated survival of patients classified as high risk. Albumin, bilirubin, AST, and platelets were most associated with outcomes. Autoimmune hepatitis was more prevalent in higher risk groups, and over-weighting of AST in these patients accounted for the observed versus predicted survival discrepancy. All 3 models offered good short-term discrimination of outcomes but only fair long-term discrimination. None of the models account for the high prevalence of features of autoimmune hepatitis overlap in children and the associated elevated aminotransferases. A pediatric-specific model is needed. AST, bilirubin, albumin, and platelets will be important predictors, but must be weighted to account for the unique features of PSC in children.Lire moins >
Langue :
Anglais
Audience :
Internationale
Vulgarisation :
Non
Établissement(s) :
CHU Lille
Inserm
Université de Lille
Inserm
Université de Lille
Date de dépôt :
2024-01-30T10:28:33Z
2024-02-06T10:16:25Z
2024-02-06T10:16:25Z