Selecting High-Risk Patients With Pediatric Crohn's Disease for Top-Down Anti-TNF as per the 2021 ECCO-ESPGHAN Guidelines: A 5-Year Nationwide Retrospective Analysis From Scotland (2016-2020)

Background The 2021 ECCO-ESPGHAN guideline on the medical management of pediatric Crohn's disease promotes early risk stratification and top-down anti-TNF for patients deemed high risk of severe disease course.Aims We aimed to objectively assess the risk-benefit profile of the guideline's risk stratification policy and guidance on top-down anti-TNF in a nationwide population-based cohort study.Methods Using a prospectively identified nationwide cohort of all new pediatric patients (<17 years) diagnosed with Crohn's disease in Scotland between January 1, 2016 and December 31, 2020 and retrospectively applying the current management algorithm, we explored the guideline's ability to accurately risk stratify patients. Phenotypic and treatment data were retrospectively collected from electronic medical records with a maximum follow-up of 18 months post-diagnosis.Results Four hundred and eighteen children (258/418 [62%] male; median [interquartile range {IQR}] age at diagnosis: 13.2 [11.2-14.8] years) were included. High-risk phenotype was present in 224/418 (54%) with 53/224 (24%) of high-risk patients not requiring anti-TNF therapy within 18 months of diagnosis. Conversely, 78/194 (40%) of the low-risk group received anti-TNF within 18 months. High-risk patients were more likely to require anti-TNF (171/224 [76%] vs 78/194 [40%], P < .001) and had shorter median (IQR) time to anti-TNF start (5.0 [1.0-8.0] months vs 6.5 [3.3-13.0] months, P = .01).Conclusions Our data support the guideline's ability to identify patients more likely to require early treatment escalation. However, this approach would have led to potential over- and under-treatment in a substantial proportion of patients. This underscores the importance of frequent and comprehensive monitoring, along with flexible treatment strategies that adapt to changes in disease status. In a historic nationwide cohort of patients with pediatric Crohn's disease categorized as high or low risk using the 2021 ECCO-ESPGHAN guidelines, these risk stratification criteria identify patients more likely to require early treatment escalation, but risk over-treating a proportion of patients.