Ustekinumab shows benefit in kids with refractory Crohn's disease

  • Chavannes M & al.
  • J Crohns Colitis
  • 12 Dec 2018

  • curated by Miriam Davis, PhD
  • Clinical Essentials
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Takeaway

  • A cohort study finds that subcutaneous ustekinumab (UST) has multiple benefits for children with medically refractory Crohn's disease (CD), including a clinical remission rate of 38.6%.
  • Medically refractory CD is defined as having failed ≥1 biologic agent.

Why this matters

  • UST is currently being used off-label in children with medically refractory CD.
  • Findings suggest that a randomized controlled trial is warranted.

Study design

  • Multicenter prospective cohort (n=44) with medically refractory pediatric CD given UST.
  • Primary outcome at 3 and 12 months was improvement on abbreviated Pediatric Crohn's Disease Activity Index (aPCDAI) (scores: 15-25 moderate disease; >25 severe disease).
  • Clinical response defined as decrease of ≥15 on aPCDAI.
  • Funding: None.

Key results

  • 38.6% of cohort achieved clinical remission at 12 months; 47.8% achieved clinical response.
  • UST had a statistically and clinically significant benefit by linear mixed model analysis (χ2[1], 42.7; P=1.2×10−8).
    • aPCDAI scores were lowered by 16±2.7 (standard error) at 3 months; 19.6±2.9 at 12 months.
  • Adverse events: 12.4 per 1000 patient-months.
  • At 12 months, the probability of staying on UST was 76.9%.
  • Mean weight z-scores increased from baseline to 12 months by 0.48±0.13 (P=.0008); mean BMI z-score increased by 0.66±0.16 (P=.0003).

Limitations

  • Uncontrolled, open-label, observational design.
  • Clinical benefit lacks confirmatory endoscopy.

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