Ustekinumab is safe and effective in pediatric patients with Crohn's disease
Elana B Mitchel1,2, Michael T Dolinger3,4, Brad Constant1
1Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Insights
Ustekinumab effectively treats pediatric Crohn's disease (CD), with frequent therapy escalation leading to sustained remission. This real-world data confirms its safety and durability in children with CD.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Limited real-world data exists for ustekinumab in pediatric Crohn's disease (CD).
- Evaluating ustekinumab's effectiveness, durability, and safety in children with CD is crucial.
Purpose of the Study:
- To assess the real-world effectiveness of ustekinumab in pediatric CD patients.
- To determine the long-term durability and safety profile of ustekinumab treatment in this population.
Main Methods:
- Retrospective longitudinal cohort study of 101 pediatric CD patients treated with ustekinumab (2015-2020).
- Primary outcome: steroid-free clinical remission at 1 year. Secondary outcomes: remission rates, drug escalation/discontinuation, serum levels, and adverse events.
- Statistical analyses included logistic regression and Kaplan-Meier curves.
Main Results:
- 59% of patients achieved steroid-free clinical remission at 1 year.
- Higher baseline disease activity and stricturing/penetrating phenotype were associated with lower remission rates.
- Ustekinumab escalation occurred in 70% of patients, leading to sustained remission in most.
Conclusions:
- Ustekinumab demonstrates effectiveness and a favorable safety profile for pediatric CD.
- Therapy escalation is common but leads to durable clinical responses in children with Crohn's disease.
Objectives:
Real-world data on ustekinumab for the treatment of pediatric Crohn's disease (CD) are limited. This study sought to evaluate the effectiveness, long-term durability, and safety of ustekinumab in the treatment of children with CD.
Methods:
A retrospective longitudinal cohort study of children with CD treated with ustekinumab from two large centers between 2015 and 2020 was performed. The primary outcome was frequency of steroid-free clinical remission at 1 year. Secondary outcomes included time to steroid-free clinical remission, frequency of clinical and biochemical remission, drug escalation and discontinuation, serum level data, and adverse events. Standard descriptive and comparative statistics were performed. Logistic regression was used to identify factors associated with steroid-free remission at 1 year. Kaplan-Meier curves were used to visualize time-to-event relationships for outcomes.
Results:
A total of 101 patients were included. Median follow-up time on ustekinumab was 16.6 months (interquartile range [IQR]: 8.71-31.2) with drug failure in 28% at 1 year. Fifty-nine patients were in steroid-free clinical remission at 1 year. Higher baseline disease activity (odds ratio [OR]: 0.91 (95% confidence interval [CI]: 0.84-0.97), p = 0.01) and stricturing/penetrating disease phenotype (OR: 0.14 (95% CI: 0.03-0.65), p = 0.02) were associated with decreased likelihood of steroid-free clinical remission at 1-year. Ustekinumab drug escalation occurred in 70% of patients, and after escalation, 50 (70%) achieved clinical remission, and 49 (69%) achieved steroid-free remission at the last follow-up. Adverse events were rare and did not require therapy discontinuation.
Conclusions:
Ustekinumab is effective and safe in the treatment of children with CD. Escalation of therapy occurs frequently but results in sustained durability.
Related Concept Videos
01:24Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF
01:21Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids
01:29Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents
01:29Drugs for Treatment of Ulcerative Colitis in IBD
01:29Inflammatory Bowel Disease IV: Pharmacological Management
Pharmacologic...
01:30Inflammatory Bowel Disease II: Crohn's Disease
Inflammatory bowel disease, commonly known as IBD, refers to a collection of disorders that lead to persistent inflammation of the gastrointestinal tract. The two types of IBD are ulcerative colitis, which impacts the colon, and Crohn's disease, which can involve any part of the gastrointestinal segment.
Crohn's disease
Crohn's disease is a chronic, systemic inflammatory bowel disease (IBD) that predominantly affects the gastrointestinal tract. It is marked by...