Department of Pediatrics, Division of Gastroenterology and Hepatology, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA.
Paediatr Drugs. 2011 Apr 1;13(2):87-95. doi: 10.2165/11586500-000000000-00000.
Primary sclerosing cholangitis (PSC) is a chronic cholestatic condition affecting the extrahepatic and intrahepatic biliary tree. The incidence is seemingly increasing in children as a result of increased use of cholangiographic screening techniques in children with inflammatory bowel disease. The clinical presentation of PSC in childhood is variable and frequently without obvious cholestatic features, and cholangiography is essential for the diagnosis of this disorder. Histologic findings may help to exclude autoimmune sclerosing cholangitis. The underlying pathogenesis remains poorly understood and, as a result, therapeutic agents that halt disease progression and improve prognosis are lacking. PSC treatment is mainly supportive and directed at controlling cholestatic symptoms and preventing complications. Ursodeoxycholic acid is helpful in inducing biochemical improvement; long-term pediatric studies to determine a benefit of this agent in young patients are lacking, although results from adult studies have not been promising. Some agents such as antibiotics are under investigation with some promising results. Liver transplantation is required for children who progress to end-stage liver disease. Prospective multicenter trials in children with PSC are needed.
原发性硬化性胆管炎（PSC）是一种影响肝外和肝内胆管的慢性胆汁淤积性疾病。由于在炎症性肠病患儿中越来越多地使用胆管造影筛查技术，PSC 的发病率似乎在儿童中有所增加。儿童 PSC 的临床表现多种多样，且常无明显胆汁淤积特征，胆管造影对于诊断这种疾病至关重要。组织学发现有助于排除自身免疫性硬化性胆管炎。其潜在发病机制仍不清楚，因此缺乏能阻止疾病进展和改善预后的治疗药物。PSC 的治疗主要是支持性的，旨在控制胆汁淤积症状和预防并发症。熊去氧胆酸有助于诱导生化改善；缺乏针对年轻患者的该药物疗效的长期儿科研究，尽管来自成人研究的结果并不理想。一些药物，如抗生素，正在研究中，并有一些有希望的结果。对于进展为终末期肝病的儿童需要进行肝移植。需要在 PSC 患儿中开展前瞻性多中心试验。
