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Long-Term Course and Prognostic Factors in Pediatric Ulcerative Proctitis: A Multicenter Cohort Study
Ayako Miyazawa1, Ryusuke Nambu1, Hirotaka Shimizu2
1Division of Gastroenterology and Hepatology, Saitama Children's Medical Center, 1-2, Shintoshin, Chuo-ku, Saitama City, Saitama 330-8777, Japan.
Insights
Refractory ulcerative proctitis (UP) in children often requires immunosuppressive treatment, despite initial 5-ASA therapy. Close monitoring is crucial due to variable disease courses in pediatric UP patients.
Area of Science:
- Pediatric Gastroenterology
- Inflammatory Bowel Disease Research
- Clinical Pediatrics
Background:
- Ulcerative proctitis (UP) in children is often mild, but some cases involve proximal disease extension and necessitate immunosuppressive therapy.
- Investigated clinical characteristics and outcomes of refractory pediatric UP in a multicenter cohort.
Purpose of the Study:
- To elucidate the natural history of refractory ulcerative proctitis in children.
- To identify factors predicting the need for immunosuppressive treatment in pediatric UP.
Main Methods:
- Retrospective analysis of data from 10 pediatric inflammatory bowel disease centers (2013-2022).
- Comparison of patients receiving immunosuppressants/biologics versus 5-aminosalicylic acid (5-ASA) monotherapy.
- Utilized Pediatric Ulcerative Colitis Activity Index and endoscopic assessments.
Main Results:
- 51% of patients experienced disease extension beyond the splenic flexure.
- 37% required immunosuppressive treatment, including 18% who received biologics.
- Time to sustained clinical remission (>3 months) predicted the need for biologics.
Conclusions:
- 5-aminosalicylic acid (5-ASA) suppositories are common initial treatments for pediatric UP but have poor compliance.
- A significant proportion (37%) of pediatric UP patients ultimately require immunosuppressive or biologic agents.
- Individualized treatment adjustment and close follow-up are essential for managing pediatric UP due to its variable clinical course.
Background:
Although ulcerative proctitis (UP) in children is considered relatively mild, some patients have proximal disease extension and require immunosuppressive treatment. We investigated clinical characteristics and course of refractory UP in a multicenter pediatric cohort.
Methods:
Analyzing data obtained between 2013 and 2022 at 10 institutions specializing in pediatric inflammatory bowel disease, we elucidated natural history and factors predicting a need for immunosuppressive UP treatment. We compared patients given immunosuppressants and/or biologic agents (immunosuppressive treatment group) with those given 5-aminosalicylic acid (5-ASA) alone (5-ASA group).
Results:
Fifty-five patients were followed for 3.5 years. The median Pediatric Ulcerative Colitis Activity Index at diagnosis was 20. The commonest treatment, 5-ASA suppository monotherapy in 40% of patients, showed the worst compliance. Clinical remission was achieved at least once in 95% of all patients. Disease extension beyond the splenic flexure occurred in 51%. Immunosuppressive treatment was given to 37%; biologic agents were used for 18%. Rates of endoscopically demonstrated inflammation, including Ra/Rs at diagnosis and extension beyond the left-sided colon, were higher in the immunosuppressive treatment group (70% vs 38%, P < 0.05; 95% vs 27%, P < 0.0001). The log-rank test and multivariate Cox proportional hazards regression showed that time to first clinical remission exceeding 3 months predicted the need for biologics.
Conclusion:
The typical initial treatment of pediatric UP was 5-ASA suppositories, despite poor compliance. Biologics or other immunosuppressive treatments were needed in 37% of patients. Close follow-up with adjustment of treatment should be considered in children with UP as its clinical course varies.
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