TY - JOUR
T1 - Validation of predictive models for disease outcomes in paediatric ulcerative colitis
T2 - A multicentre prospective inception cohort
AU - Atia, Ohad
AU - Klomberg, Renz C.W.
AU - de Ridder, Lissy
AU - Kemos, Polychronis
AU - Ruemmele, Frank M.
AU - Kang, Ben
AU - Choi, Sujin
AU - Choe, Byung Ho
AU - Kang, Youra
AU - Shouval, Dror S.
AU - Focht, Gili
AU - Ledder, Oren
AU - Lev-Tzion, Raffi
AU - Carmon, Natalie
AU - Berger, Tal David
AU - Turner, Dan
AU - Croft, Nicholas M.
AU - Orlanski-Meyer, Esther
N1 - Publisher Copyright:
© 2023 The Authors. Alimentary Pharmacology & Therapeutics published by John Wiley & Sons Ltd.
PY - 2023/7
Y1 - 2023/7
N2 - Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts. Aim: To explore these models in a prospective multicentre inception cohort. Methods: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC). Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively. SFR rate was 35% at 3 months and 47% at 12 months (62% of those with mild disease at diagnosis vs. 41% in moderate–severe disease; p = 0.01). Thirty-six (16%) children developed ASC during the first month after diagnosis, and 53 (24%) during the first year. The AUC of the PROTECT model for predicting SFR at 3 and 12 months was 0.78 [95% CI 0.65–0.92] and 0.57 [95% CI 0.47–0.66], respectively. The sensitivity/specificity/PPV/NPV of Schechter's criteria to predict sustained SFR at 12 months was 50%/60%/35%/74%. ASC was predicted only by the PUCAI score at diagnosis and at 3 months. Conclusions: The PROTECT model had a good predictive utility for SFR at 3 months, but not at 12 months. The other predictive models did not achieve sufficient accuracy, which was far from that reported in the original studies. This highlights the necessity for external validation of any prediction model prior to its implementation into clinical practice.
AB - Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts. Aim: To explore these models in a prospective multicentre inception cohort. Methods: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC). Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively. SFR rate was 35% at 3 months and 47% at 12 months (62% of those with mild disease at diagnosis vs. 41% in moderate–severe disease; p = 0.01). Thirty-six (16%) children developed ASC during the first month after diagnosis, and 53 (24%) during the first year. The AUC of the PROTECT model for predicting SFR at 3 and 12 months was 0.78 [95% CI 0.65–0.92] and 0.57 [95% CI 0.47–0.66], respectively. The sensitivity/specificity/PPV/NPV of Schechter's criteria to predict sustained SFR at 12 months was 50%/60%/35%/74%. ASC was predicted only by the PUCAI score at diagnosis and at 3 months. Conclusions: The PROTECT model had a good predictive utility for SFR at 3 months, but not at 12 months. The other predictive models did not achieve sufficient accuracy, which was far from that reported in the original studies. This highlights the necessity for external validation of any prediction model prior to its implementation into clinical practice.
UR - http://www.scopus.com/inward/record.url?scp=85161219969&partnerID=8YFLogxK
U2 - 10.1111/apt.17544
DO - 10.1111/apt.17544
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C2 - 37248642
AN - SCOPUS:85161219969
SN - 0269-2813
VL - 58
SP - 182
EP - 190
JO - Alimentary Pharmacology and Therapeutics
JF - Alimentary Pharmacology and Therapeutics
IS - 2
ER -