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