Assessing the Validity of Adult-Derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children.

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F61989592%3A15110%2F20%3A73596343" target="_blank" >RIV/61989592:15110/20:73596343 - isvavai.cz</a>

Výsledek na webu

<a href="https://journals.lww.com/jpgn/Fulltext/2020/01000/Assessing_the_Validity_of_Adult_derived_Prognostic.27.aspx" target="_blank" >https://journals.lww.com/jpgn/Fulltext/2020/01000/Assessing_the_Validity_of_Adult_derived_Prognostic.27.aspx</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1097/MPG.0000000000002522" target="_blank" >10.1097/MPG.0000000000002522</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Assessing the Validity of Adult-Derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children.

Popis výsledku v původním jazyce

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 to observed survival. We evaluated model fit using the c-statistic. RESULTS: Model fit was good at one year (c-statistics 0.93, 0.87, 0.82) and fair at ten 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 vs. predicted survival discrepancy. CONCLUSION: All three 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.

Název v anglickém jazyce

Assessing the Validity of Adult-Derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children.

Popis výsledku anglicky

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 to observed survival. We evaluated model fit using the c-statistic. RESULTS: Model fit was good at one year (c-statistics 0.93, 0.87, 0.82) and fair at ten 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 vs. predicted survival discrepancy. CONCLUSION: All three 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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30219 - Gastroenterology and hepatology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2020

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

ISSN

0277-2116

e-ISSN

—

Svazek periodika

70

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

6

Strana od-do

12-17

Kód UT WoS článku

000561369900006

EID výsledku v databázi Scopus

2-s2.0-85077298792