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Management of Paediatric Patients With Medically Refractory Crohn's Disease Using Ustekinumab: A Multi-Centred Cohort
Mallory Chavannes1,2, Christine Martinez-Vinson3, Lara Hart4
1Department of Paediatrics, Sainte-Justine UHC, University of Montreal, QC, Canada.
Insights
Ustekinumab effectively treats pediatric Crohn's disease, improving symptoms and growth in children who have not responded to other biologic therapies. This study highlights its potential as a valuable treatment option for this patient group.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Ustekinumab (UST) is an established treatment for moderate to severe Crohn's disease (CD) in adults.
- Limited data exists regarding UST efficacy and safety in pediatric CD patients.
Purpose of the Study:
- To assess the effectiveness of subcutaneous Ustekinumab in children with moderate to severe Crohn's disease.
- To evaluate clinical response, remission rates, growth parameters, and adverse events.
Main Methods:
- A multicenter retrospective cohort study of pediatric patients (<18 years) with CD treated with open-label subcutaneous UST.
- Primary outcomes included changes in abbreviated Pediatric Crohn's Disease Activity Index (aPCDAI) and clinical remission rates at 3 and 12 months.
- Secondary outcomes assessed clinical response, C-reactive protein (CRP), albumin, growth (weight and BMI z-scores), and adverse events.
Main Results:
- UST significantly reduced aPCDAI scores by 16 points at 3 months and 19.6 points at 12 months.
- At 12 months, 38.6% achieved clinical remission and 47.8% achieved clinical response.
- Significant improvements were observed in weight (z-score +0.48) and BMI (z-score +0.66); 76.9% remained on UST at 12 months.
Conclusions:
- Subcutaneous Ustekinumab is a viable therapeutic option for pediatric patients with Crohn's disease refractory to other biologic agents.
- Further investigation through prospective randomized trials is warranted to confirm these findings.
Background:
Ustekinumab [UST] is effective in the treatment of adults with moderate to severe Crohn's disease [CD]. There is a paucity of data on its use in children.
Aim:
To evaluate the response to UST in children with moderate to severe CD.
Methods:
This multicentre retrospective cohort study identified children under 18 years old with CD, who received open-labelled subcutaneous UST. The primary outcome was changes in mean abbreviated Paediatric Crohn's Disease Activity Index [aPCDAI] between baseline and 3 and 12 months, and rate of clinical remission at 3 and 12 months. Secondary outcomes were clinical response at the same time points, changes in C-reactive protein [CRP] and albumin, improvement in growth parameters, and rate of adverse events.
Results:
A total of 44 patients who failed at least one biological treatment were identified. Linear mixed model [LMM] analysis revealed a statistically significant effect of UST (χ2[1] = 42.7, p = 1.2 × 10-8) which lowered the aPCDAI scores by about 16 ± 2.7 at 3 months, and 19.6 ± 2.9 at 12 months. At 12 months, 38.6% of the patients achieved clinical remission and 47.8% achieved clinical response. There was a significant increase in mean weight z-score of 0.48 [±0.13] [p <0.001] and in mean body mass index [BMI] z score of 0.66 [±0.16] [p <0.001]. The probability of remaining on UST at 12 months was 76.9%. The rate of adverse events was 12.4 per 1000 patient-months.
Conclusions:
Subcutaneous UST should be considered a viable therapeutic option for paediatric patients who are refractory to other biological agents. Prospective randomised trials are needed.