Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort

Author:

Atia Ohad12ORCID,Klomberg Renz C. W.3,de Ridder Lissy3ORCID,Kemos Polychronis4,Ruemmele Frank M.5,Kang Ben6ORCID,Choi Sujin6,Choe Byung‐Ho6,Kang Youra6,Shouval Dror S.78ORCID,Focht Gili12,Ledder Oren12,Lev‐Tzion Raffi1,Carmon Natalie1,Berger Tal David9,Turner Dan12,Croft Nicholas M.4ORCID,Orlanski‐Meyer Esther12

Affiliation:

1. Shaare Zedek Medical Centre Jerusalem Israel

2. The Hebrew University of Jerusalem Jerusalem Israel

3. Erasmus University Medical Centre – Sophia Children's Hospital Rotterdam the Netherlands

4. Blizard Institute Barts and the London School of Medicine, Queen Mary University of London London UK

5. Université Paris Descartes, Sorbonne Paris Cité, APHP, Hôpital Necker Enfants Malades Paris France

6. Department of Paediatrics, School of Medicine Kyungpook National University Daegu Republic of Korea

7. Institute of Gastroenterology, Nutrition, and Liver Diseases, Schneider Children's Medical Centre of Israel Petach Tikva Israel

8. Sackler Faculty of Medicine Tel Aviv University Tel Aviv Israel

9. Sheba Medical Centre Tel Aviv University Tel Aviv Israel

Abstract

SummaryBackgroundSeveral 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 cohortsAimTo explore these models in a prospective multicentre inception cohortMethodsChildren 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).ResultsOf 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.ConclusionsThe 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.

Publisher

Wiley

Subject

Pharmacology (medical),Gastroenterology,Hepatology

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