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Clinical Features and Natural History of Paediatric Patients with Ulcerative Proctitis: A Multicentre Study from the
Noa Tal1,2, Christos Tzivinikos3, Marco Gasparetto4
1Institute of Gastroenterology, Nutrition and Liver Disease, Schneider Children's Medical Center, Petah Tikva, Israel.
Insights
Pediatric ulcerative proctitis (UP) often requires treatment escalation and can progress proximally. Higher activity scores predict poorer outcomes in children with UP.
Area of Science:
- Pediatric Gastroenterology
- Inflammatory Bowel Disease Research
- Clinical Outcomes in Ulcerative Colitis
Background:
- Ulcerative proctitis (UP) is an infrequent manifestation of pediatric ulcerative colitis.
- This study characterizes the clinical features and natural history of UP in children.
- It also identifies predictors of poor outcomes in this population.
Observation:
- A retrospective study analyzed 196 pediatric UP patients (median age 14.6 years) across 37 sites.
- Common symptoms included bloody stools (95%), abdominal pain (61%), and diarrhea (47%).
- Moderate-to-severe endoscopic inflammation was frequent at diagnosis, with a median Paediatric Ulcerative Colitis Activity Index (PUCAI) score of 25.
Findings:
- Treatment escalation to biologics occurred in 10% at 1 year, 22% at 3 years, and 43% at 5 years.
- A PUCAI score ≥35 at diagnosis predicted increased risk of systemic steroids, biologics, acute severe colitis, and IBD-related admissions.
- Nearly half (48%) of patients experienced proximal disease progression, associated with higher baseline PUCAI scores and cecal patch presence.
Implications:
- Pediatric UP patients face significant rates of treatment escalation and disease extension.
- Early identification of high-risk patients using PUCAI scores is crucial for managing pediatric UP.
- Understanding UP progression aids in developing targeted therapeutic strategies for pediatric inflammatory bowel disease.
Background And Aims:
Ulcerative proctitis [UP] is an uncommon presentation in paediatric patients with ulcerative colitis. We aimed to characterize the clinical features and natural history of UP in children, and to identify predictors of poor outcomes.
Methods:
This was a retrospective study involving 37 sites affiliated with the IBD Porto Group of ESPGHAN. Data were collected from patients aged <18 years diagnosed with UP between January 1, 2016 and December 31, 2020.
Results:
We identified 196 patients with UP (median age at diagnosis 14.6 years [interquartile range, IQR 12.5-16.0]), with a median follow-up of 2.7 years [IQR 1.7-3.8]. The most common presenting symptoms were bloody stools [95%], abdominal pain [61%] and diarrhoea [47%]. At diagnosis, the median paediatric ulcerative colitis activity index [PUCAI] score was 25 [IQR 20-35], but most patients exhibited moderate-severe endoscopic inflammation. By the end of induction, 5-aminosalicylic acid administration orally, topically or both resulted in clinical remission rates of 48%, 48%, and 73%, respectively. The rates of treatment escalation to biologics at 1, 3, and 5 years were 10%, 22%, and 43%, respectively. In multivariate analysis, the PUCAI score at diagnosis was significantly associated with initiation of systemic steroids, or biologics, and subsequent acute severe colitis events and inflammatory bowel disease-associated admission, with a score ≥35 providing an increased risk for poor outcomes. By the end of follow-up, 3.1% of patients underwent colectomy. Patients with UP that experienced proximal disease progression during follow-up [48%] had significantly higher rates of a caecal patch at diagnosis and higher PUCAI score by the end of induction, compared to those without progression.
Conclusion:
Paediatric patients with UP exhibit high rates of treatment escalation and proximal disease extension.
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