Department of Paediatrics, University Malaya Medical Center, Kuala Lumpur, Malaysia; Paediatric and Child Health Research Group, University Malaya, Kuala Lumpur, Malaysia.
Khoo Teck Puat National University Children's Medical Institute, National University Hospital, Singapore.
Pediatr Neonatol. 2019 Aug;60(4):396-404. doi: 10.1016/j.pedneo.2018.09.007. Epub 2018 Oct 2.
Current knowledge on the clinical features and natural history of childhood primary sclerosing cholangitis - inflammatory bowel disease in Asia is limited. We described the presenting features and natural history of primary sclerosing cholangitis-inflammatory bowel disease seen in a cohort of Southeast Asian children.
We conducted a retrospective review of childhood primary sclerosing cholangitis-inflammatory bowel disease from three tertiary centers in Singapore and Malaysia.
Of 24 patients (boys, 58%; median age at diagnosis: 6.3 years) with primary sclerosing cholangitis-inflammatory bowel disease (ulcerative colitis, n = 21; Crohn's disease, n = 1; undifferentiated, n = 2), 63% (n = 15) were diagnosed during follow-up for colitis, and 21% (n = 5) presented with acute or chronic hepatitis, 17% (n = 4) presented simultaneously. Disease phenotype of liver involvement showed 79% had sclerosing cholangitis-autoimmune hepatitis overlap, 54% large duct disease, and 46% small duct disease. All patients received immunosuppression therapy. At final review after a median [±S.D.] duration follow-up of 4.7 [±3.8] years, 12.5% patients had normal liver enzymes, 75% persistent disease, and 12.5% liver failure. The proportion of patients with liver cirrhosis increased from 13% at diagnosis to 29%; 21% had portal hypertension, and 17% had liver dysfunction. One patient required liver transplant. Transplant-free survival was 95%. For colitis, 95% had pancolitis, 27% rectal sparing, and 11% backwash ileitis at initial presentation. At final review, 67% patients had quiescent bowel disease with immunosuppression. One patient who had UC with pancolitis which was diagnosed at 3 years old developed colorectal cancer at 22 years of age. All patients survived.
Liver disease in primary sclerosing cholangitis-inflammatory bowel disease in Asian children has variable severity. With immunosuppression, two-thirds of patients have quiescent bowel disease but the majority have persistent cholangitis and progressive liver disease.
目前亚洲儿童原发性硬化性胆管炎-炎症性肠病的临床特征和自然史的知识有限。我们描述了在新加坡和马来西亚的三个三级中心的一组东南亚儿童中所见的原发性硬化性胆管炎-炎症性肠病的表现特征和自然史。
我们对来自新加坡和马来西亚的三个三级中心的 24 名儿童原发性硬化性胆管炎-炎症性肠病患者（男，58%；诊断时的中位年龄：6.3 岁）进行了回顾性分析。
24 名患者中，21 名（溃疡性结肠炎，n=21；克罗恩病，n=1；未分化，n=2）患有原发性硬化性胆管炎-炎症性肠病，63%（n=15）在结肠炎随访期间被诊断，21%（n=5）表现为急性或慢性肝炎，17%（n=4）同时表现出肝脏和肠道疾病。肝脏受累的疾病表型显示 79%有硬化性胆管炎-自身免疫性肝炎重叠，54%有大胆管疾病，46%有小胆管疾病。所有患者均接受免疫抑制治疗。在中位（±SD）随访 4.7（±3.8）年后的最终复查中，12.5%的患者肝功能正常，75%的患者持续患病，12.5%的患者发生肝衰竭。肝硬化患者比例从诊断时的 13%增加到 29%；21%有门静脉高压，17%有肝功能障碍。1 名患者需要进行肝移植。无移植生存率为 95%。对于结肠炎，95%的患者初次就诊时表现为全结肠炎，27%的患者有直肠保留，11%的患者有回肠反流性肠炎。在最终复查时，67%的患者用免疫抑制剂治疗后肠道疾病处于缓解状态。1 名溃疡性结肠炎患儿，3 岁时诊断为全结肠炎，22 岁时发展为结直肠癌。所有患者均存活。
亚洲儿童原发性硬化性胆管炎-炎症性肠病的肝脏疾病严重程度不同。通过免疫抑制治疗，三分之二的患者肠道疾病处于缓解状态，但大多数患者仍有胆管炎和进行性肝病。
