Pediatric Gastroenterology and Liver Unit, Sapienza University of Rome, Umberto I Hospital, Rome, Italy.
Pediatric Hepatology Gastroenterology and Transplantation, Papa Giovanni XXIII Hospital, Bergamo, Italy.
Inflamm Bowel Dis. 2024 Oct 3;30(10):1662-1669. doi: 10.1093/ibd/izad218.
Whether primary sclerosing cholangitis related to inflammatory bowel disease (PSC-IBD) diagnosed before 6 years (ie, VEO-IBD) has a distinct phenotype and disease course is uninvestigated. We aimed to analyze the characteristics and natural history of VEO-PSC-IBD, compared with early and adolescent-onset PSC-IBD.
This is a multicenter, retrospective, case-control study from 15 centers affiliated with the Porto and Interest IBD group of ESPGHAN. Demographic, clinical, laboratory, endoscopic, and imaging data were collected at baseline and every 6 months. Inflammatory bowel disease-related (clinical remission, need for systemic steroids and biologics, and surgery) and PSC-related (biliary and portal hypertensive complications, need for treatment escalation and liver transplantation, cholangiocarcinoma, or death) outcomes were compared between the 2 groups.
Sixty-nine children were included, with a median follow-up of 3.63 years (interquartile range, 1-11): 28 with VEO-PSC-IBD (23 UC [82%], 2 IBD-U [7%] and 3 [11%] CD), and 41 with PSC-IBD (37 UC [90%], 3 IBDU [7.5%] and 1 [2.5%] CD). Most patients with UC presented with pancolitis (92% in VEO-PSC-UC vs 85% in PSC-UC, P = .2). A higher number of patients with VEO-PSC-IBD were diagnosed with PSC/autoimmune hepatitis overlap syndrome than older children (24 [92%] vs 27 [67.5%] PSC-IBD, P = .03), whereas no other differences were found for PSC-related variables. Time to biliary strictures and infective cholangitis was lower in the VEO-PSC-IBD group (P = .01 and P = .04, respectively), while no difference was found for other outcomes. No cases of cholangiocarcinoma were reported.
Primary sclerosing cholangitis related to inflammatory bowel disease has similar baseline characteristics whether diagnosed as VEO-IBD or thereafter. A milder disease course in terms of biliary complications characterizes VEO-PSC-IBD.
患有炎症性肠病相关原发性硬化性胆管炎（PSC-IBD）的儿童，其发病年龄在 6 岁前（即早发性炎症性肠病，VEO-IBD）是否具有独特的表型和疾病过程，目前尚未明确。本研究旨在分析 VEO-PSC-IBD 的特征和自然病程，并与早发型和青少年发病的 PSC-IBD 进行比较。
这是一项来自 ESPGHAN 旗下 Porto 和 Interest IBD 组 15 个中心的多中心、回顾性、病例对照研究。在基线和每 6 个月时收集人口统计学、临床、实验室、内镜和影像学数据。比较两组之间与炎症性肠病相关（临床缓解、需要全身皮质类固醇和生物制剂以及手术）和 PSC 相关（胆道和门脉高压并发症、需要治疗升级和肝移植、胆管癌或死亡）的结局。
共纳入 69 名儿童，中位随访时间为 3.63 年（四分位距，1-11 年）：28 名 VEO-PSC-IBD（23 例溃疡性结肠炎 [82%]，2 例 IBD-U [7%]和 3 例 CD [11%]），41 名 PSC-IBD（37 例溃疡性结肠炎 [90%]，3 例 IBD-U [7.5%]和 1 例 CD [2.5%]）。大多数患有 UC 的患者表现为全结肠炎（VEO-PSC-UC 中为 92%，PSC-UC 中为 85%，P =.2）。与年龄较大的儿童相比，VEO-PSC-IBD 中有更多的患者被诊断为 PSC/自身免疫性肝炎重叠综合征（24 [92%]比 PSC-IBD 中 27 [67.5%]，P =.03），但在 PSC 相关变量方面没有发现其他差异。VEO-PSC-IBD 组发生胆道狭窄和感染性胆管炎的时间更短（P =.01 和 P =.04），而其他结局则无差异。没有报告胆管癌病例。
患有炎症性肠病相关原发性硬化性胆管炎的儿童，无论被诊断为 VEO-IBD 还是其他类型，其基线特征相似。VEO-PSC-IBD 的胆道并发症病程较轻。
