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Clinical outcome of ulcerative colitis with severe onset in children: a multicenter prospective cohort study
Ryusuke Nambu1, Katsuhiro Arai2, Takahiro Kudo3
1Division of Gastroenterology and Hepatology, Saitama Children's Medical Center, 1-2 Shintoshin, Chuo-Ku, Saitama, 3308777, Japan. nambee1231@gmail.com.
Insights
Children with severe ulcerative colitis often need potent treatments like calcineurin inhibitors and biologics. A trial of calcineurin inhibitors may reduce the need for biologics or surgery in steroid-resistant cases.
Area of Science:
- Pediatric Gastroenterology
- Inflammatory Bowel Disease Research
- Clinical Trial Analysis
Background:
- Best practices for treating severe pediatric ulcerative colitis are debated, especially with advanced biologic therapies.
- This study addresses the need for evidence-based treatment strategies in this challenging patient population.
Purpose of the Study:
- To prospectively investigate treatment approaches and outcomes in children with severe-onset ulcerative colitis.
- To compare outcomes between patients with severe disease (S1) and less severe disease (S0).
Main Methods:
- A multicenter cohort study using a Japanese web-based data registry (October 2012-March 2020).
- Patients were categorized into S1 (Pediatric Ulcerative Colitis Activity Index ≥65) and S0 (<65) groups.
- Follow-up duration was 3.6 ± 1.9 years for 301 children.
Main Results:
- Severe ulcerative colitis (S1) affected 25% of patients, often presenting with pancolitis at age 12.3 years.
- Colectomy-free rates were significantly lower in S1 patients (74% at 5 years) compared to S0.
- S1 patients received more calcineurin inhibitors (53%) and biologics (56%) than S0 patients.
Conclusions:
- Children with severe ulcerative colitis frequently require potent therapies, including calcineurin inhibitors and biologics, and sometimes colectomy.
- A trial of calcineurin inhibitors for steroid-resistant cases may decrease the immediate need for biologics or colectomy.
Background:
As best practices for treating children with severe-onset ulcerative colitis remain controversial in the era of biologic agents, we prospectively investigated treatments and outcomes in a multicenter cohort.
Methods:
Using a Web-based data registry maintained in Japan between October 2012 and March 2020, we compared management and treatment outcomes in an S1 group defined by a Pediatric Ulcerative Colitis Activity Index of 65 or more points at diagnosis with those in an S0 group defined by an index value below 65.
Results:
Three hundred one children with ulcerative colitis treated at 21 institutions were included, with follow-up for 3.6 ± 1.9 years. Among them, 75 (25.0%) were in S1; their age at diagnosis was 12.3 ± 2.9 years, and 93% had pancolitis. Colectomy free rates in S1 were 89% after 1 year, 79% after 2, and 74% after 5, significantly lower than for S0 (P = 0.0003). Calcineurin inhibitors and biologic agents, respectively, were given to 53% and 56% of S1 patients, significantly more than for S0 patients (P < 0.0001). Among S1 patients treated with calcineurin inhibitors when steroids failed, 23% required neither biologic agents nor colectomy, similarly to the S0 group (P = 0.46).
Conclusions:
Children with severe ulcerative colitis are likely to require powerful agents such as calcineurin inhibitors and biologic agents; sometimes colectomy ultimately proves necessary. Need for biologic agents in steroid-resistant patients might be reduced to an extent by interposing a therapeutic trial of CI rather than turning to biologic agents or colectomy immediately.
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