Clinical Features and Natural History of Paediatric Patients with Ulcerative Proctitis: A Multicentre Study from the Paediatric IBD Porto Group of ESPGHAN
Noa Tal, Christos Tzivinikos, Marco Gasparetto, Daniela Elena Serban, Eyal Zifman, Iva Hojsak, Oren Ledder, Anat Yerushalmy Feler, Helena Rolandsdotter, Marina Aloi, Matteo Bramuzzo, Stephan Buderus, Paolo Lionetti, Lorenzo Norsa, Christoph Norden, Darja Urlep, Claudio Romano, Ron Shaoul, Christine Martinez‐Vinson, Anna Karoliny, Elisabeth De Greef, Ben Kang, Eva VIčková, Patrizia Alvisi, Michal Kori, Marta Tavares, Batia Weiss, Séamus Hussey, Maria E Qamhawi, L M Palomino Pérez, Paul Henderson, Raj Parmar, Erasmo Miele, Firas Rinawi, Ana Lozano-Ruf, Veena Zamvar, Kaija‐Leena Kolho, Dror S. Shouval
Abstract
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.