Division of Gastroenterology, Hepatology and Nutrition, Hospital for Sick Children, Toronto, Canada.
Institute of Health Policy Management and Evaluation, University of Toronto, Toronto, Canada; Toronto Centre for Liver Disease, Toronto Western and General Hospital, University Health Network, Toronto, Canada; Department of Gastroenterology and Hepatology, Erasmus MC University Medical Centre, Rotterdam, the Netherlands.
Clin Gastroenterol Hepatol. 2020 Jun;18(7):1509-1517.e7. doi: 10.1016/j.cgh.2019.08.048. Epub 2019 Sep 4.
BACKGROUND & AIMS: Although inflammatory bowel diseases (IBD) associated with primary sclerosing cholangitis (PSC) have been well characterized in adults, there have been few pediatric studies, and these were small and produced conflicting results. We investigated features of PSC-IBD in children, compared with children with IBD without PSC.
We performed a retrospective study of 74 children with PSC-IBD, diagnosed from 2000 through 2018, who were each matched with 2 children with ulcerative colitis or IBD-unclassified (controls) based on sex, date of birth, and type of IBD. We compared IBD distribution and clinical activity (remission, medication use, hospitalization, or colectomy) and patient growth between groups. Data were extracted from each hospital contact and analyzed using mixed effects analyses or Cox proportional hazards regression, adjusting for time-dependent medication exposure.
Higher proportions of children with PSC-IBD had backwash ileitis, pancolitis, and rectal sparing, and more severe right-sided disease, than controls (P < .05). Patients with PSC-IBD were more likely to be treated with only 5-ASA, compared with controls (odds ratio [OR], 3.04; 95% CI, 1.44-6.41) and to have IBD in clinical remission (OR, 2.94; 95% CI, 1.78-4.87). Risk of colectomy or treatment with a biologic agent was lower in patients with PSC-IBD than controls (hazard ratio, 0.24; 95% CI, 0.12-0.52). However, determination of IBD severity based on symptoms underestimated severity based on endoscopic activity in patients with PSC-IBD. Among patients with IBD in clinical remission, those with PSC were less likely to have endoscopic remission (OR, 0.44; 95% CI, 0.20-0.96). Patients with PSC-IBD were shorter and had lower weight over time, compared with controls.
In a retrospective study, we found that features of IBD differed between children with vs without PSC, similar to adults. Despite the mild clinical activity of IBD in patients with PSC, lack of symptoms does not always indicate lack of mucosal inflammation. Children with PSC-IBD have greater growth impairments compared with children with ulcerative colitis or IBD-unclassified.
虽然原发性硬化性胆管炎（PSC）相关的炎症性肠病（IBD）在成人中已有很好的描述，但儿科研究较少，且这些研究规模较小，结果相互矛盾。我们研究了儿童 PSC-IBD 的特征，并与无 PSC 的 IBD 患儿进行了比较。
我们对 2000 年至 2018 年间诊断为 PSC-IBD 的 74 名儿童进行了回顾性研究，这些患儿均与溃疡性结肠炎或 IBD 未分类（对照）的 2 名患儿根据性别、出生日期和 IBD 类型进行了匹配。我们比较了各组之间的 IBD 分布和临床活动（缓解、药物使用、住院或结肠切除术）以及患者生长情况。从每次医院就诊中提取数据，并使用混合效应分析或 Cox 比例风险回归进行分析，调整了时间依赖性药物暴露。
与对照组相比，PSC-IBD 患儿中回肠炎、全结肠炎和直肠保留、右半结肠炎更严重的比例更高（P<0.05）。与对照组相比，PSC-IBD 患儿更有可能仅接受 5-ASA 治疗（比值比[OR]，3.04；95%CI，1.44-6.41），并且更有可能处于 IBD 缓解状态（OR，2.94；95%CI，1.78-4.87）。与对照组相比，PSC-IBD 患儿的结肠切除术或生物制剂治疗风险较低（风险比，0.24；95%CI，0.12-0.52）。然而，基于症状确定的 IBD 严重程度低估了 PSC-IBD 患者基于内镜活动确定的严重程度。在处于临床缓解的 IBD 患者中，PSC 患者内镜缓解的可能性较低（OR，0.44；95%CI，0.20-0.96）。与对照组相比，PSC-IBD 患儿的身高和体重随时间逐渐降低。
在一项回顾性研究中，我们发现，与无 PSC 的患儿相比，PSC 患儿的 IBD 特征不同，这与成人相似。尽管 PSC 患儿的 IBD 临床活动较轻，但缺乏症状并不总是表明没有黏膜炎症。与溃疡性结肠炎或 IBD 未分类的患儿相比，PSC-IBD 患儿的生长受损更大。
