Pediatric ulcerative colitis: early remission predictors

  • Lancet

  • International Clinical Digest
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Takeaway

  • For children with newly diagnosed ulcerative colitis (UC), early mild disease and remission by week 4 predict long-term remission.
  • Genetic, microbiome data may help predict escalation to anti-TNFα therapy.

Why this matters

  • Few robust data guide prognostication or treatment of newly diagnosed UC in children. 

Key results

  • 400 participants evaluable at week 52.
  • 38% (150/400) achieved remission.
  • Remission predictors: mild clinical severity, week-4 remission, baseline hemoglobin ≥10 g/dL in those not achieving week-4 remission.
    • Model area under curve (AUC), 0.70 (95% CI, 0.65-0.75).
    • Specificity, 77%.
    • Validation in separate cohort: mild clinical severity, week-4 remission were predictive: AUC, 0.65. 
    • Adding genetic and microbiome data: AUC, 0.75. 
  • Escalation predictors: Mayo score ≥11; lack of week-4 remission; low baseline hemoglobin, serum 25(OH)D, rectal eosinophils.
    • Model: AUC, 0.78.
    • Adding genetic and microbiome data: AUC, 0.88. 

Study design

  • Prospective multicenter inception cohort PROTECT (n=428).
  • Children ages 4-17 years with newly diagnosed UC underwent guideline-based treatment with mesalazine (Pentasa; Shire US Inc.) or corticosteroids.
  • Using baseline and 4-week data, including pretreatment rectal gene expression and gut microbiome, authors derived models to predict remission, escalation.
  • Outcome: week-52 corticosteroid-free remission with mesalazine alone.
  • Funding: NIH.

Limitations

  • Insufficient data to validate escalation model.