https://pmc.ncbi.nlm.nih.gov/articles/PMC11002871/

Background

Stricturing and penetrating complications account for much of the morbidity and surgical burden in pediatric Crohn’s disease, and the prospective RISK study established which baseline factors predict them. Reproducing that result from routinely collected records would show that a distributed network can stand in for a purpose-built prospective cohort. The two complications compete with one another, so modeling either alone distorts both.

Method

ODACoR-H handles competing risks when sites disagree, allowing the baseline subdistribution hazard for each endpoint to vary by institution. A surrogate likelihood combines patient-level data at the local site with aggregated summaries from external sites, in one shot. Four national children’s hospitals within PEDSnet contributed, and the analysis followed the RISK study’s endpoint definitions so the two sets of results would be directly comparable.

Main finding

The subdistribution hazard ratios recovered from routine electronic health records were consistent with those reported by the original prospective RISK cohort, reproducing its risk stratification for penetrating and stricturing behavior. This supports using existing pediatric network data to replicate and extend prospective cohort findings in rare pediatric conditions where enrolling a new cohort is slow and expensive.