儿童溃疡性直肠炎患者的临床特征和自然病史：来自 ESPGHAN 的 Porto 儿童 IBD 小组的一项多中心研究。

Clinical Features and Natural History of Paediatric Patients with Ulcerative Proctitis: A Multicentre Study from the Paediatric IBD Porto Group of ESPGHAN.

机构信息

Institute of Gastroenterology, Nutrition and Liver Disease, Schneider Children's Medical Center, Petah Tikva, Israel.

Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

出版信息

J Crohns Colitis. 2023 Dec 30;17(12):1939-1948. doi: 10.1093/ecco-jcc/jjad111.

DOI:10.1093/ecco-jcc/jjad111

PMID:37392064

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.

摘要

背景和目的

溃疡性直肠炎（UP）是儿童溃疡性结肠炎中一种不常见的表现。我们旨在描述儿童 UP 的临床特征和自然史，并确定不良结局的预测因素。

方法

这是一项回顾性研究，涉及 ESPGHAN 的 IBD 波尔图组的 37 个站点。数据收集自 2016 年 1 月 1 日至 2020 年 12 月 31 日期间诊断为 UP 的<18 岁患者。

结果

我们确定了 196 例 UP 患者（中位诊断年龄为 14.6 岁[四分位距，IQR 12.5-16.0]），中位随访时间为 2.7 年[IQR 1.7-3.8]。最常见的首发症状是血性粪便[95%]、腹痛[61%]和腹泻[47%]。在诊断时，中位数儿童溃疡性结肠炎活动指数[PUCAI]评分 25[IQR 20-35]，但大多数患者表现为中重度内镜炎症。在诱导结束时，口服、局部或两者联合使用 5-氨基水杨酸的临床缓解率分别为 48%、48%和 73%。1、3 和 5 年内生物制剂治疗升级的比例分别为 10%、22%和 43%。多变量分析显示，诊断时的 PUCAI 评分与全身性类固醇或生物制剂的起始、随后的急性重度结肠炎事件和炎症性肠病相关入院显著相关，评分≥35 提示不良结局的风险增加。在随访结束时，3.1%的患者接受了结肠切除术。在随访过程中出现近端疾病进展的 UP 患者[48%]，在诊断时更有可能存在盲肠斑块，诱导结束时的 PUCAI 评分也更高，与未进展的患者相比。