Assessing the Validity of Adult-derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children.
J Pediatr Gastroenterol Nutr
; 70(1): e12-e17, 2020 01.
Article
em En
| MEDLINE
| ID: mdl-31651664
ABSTRACT
BACKGROUND:
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.METHODS:
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.RESULTS:
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.CONCLUSIONS:
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Pediatria
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Colangite Esclerosante
/
Modelos Estatísticos
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Medição de Risco
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Gastroenterologia
Tipo de estudo:
Etiology_studies
/
Evaluation_studies
/
Prognostic_studies
/
Risk_factors_studies
Limite:
Child
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Female
/
Humans
/
Male
Idioma:
En
Revista:
J Pediatr Gastroenterol Nutr
Ano de publicação:
2020
Tipo de documento:
Article