A Retrospective Single-Center Review of Primary Sclerosing Cholangitis in Children
A Retrospective Single-Center Review of Primary Sclerosing Cholangitis in Children
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DOI：
10.1016/j.cgh.2008.10.019
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发表时间：
2009-02-01
影响因子：
12.6
通讯作者：
Kerkar, Nanda
中科院分区：
文献类型：
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作者：
Miloh, Tamir;Arnon, Ronen;Kerkar, Nanda
背景和目标：原发性硬化性胆管炎（PSC）是一种以炎症和进行性胆管纤维化为特征的慢性胆汁淤积性肝病。关于儿科PSC的数据有限。方法：我们对47例PSC患儿进行了回顾性病历分析。结果：诊断时的平均年龄为11 ± 4.9岁。81%的患者在出现症状之前就出现了症状; 59%的患者发现了炎症性肠病，25%的患者发现了自身免疫性肝炎（重叠综合征）。磁共振胆管造影显示肝外和肝内，孤立的肝内，孤立的肝外，和无胆管受累（小管PSC）分别为40%，14%，10%和36%。晚期纤维化（>II期）占65%。结肠镜检查显示全结肠炎，直肠保留，和正常的结果分别为24%，24%和18%。所有患者均接受熊去氧胆酸（UDCA）治疗; 9例重叠综合征患者还接受了免疫抑制剂治疗。15例无重叠综合征的患者自身免疫标志物阳性，对UDCA单药治疗有反应。9例患者（3例重叠综合征和2例小导管PSC）在诊断后中位时间7年进行了肝移植。移植后10年生存率为89%。结论;在一项最大的PSC儿童单中心研究中，我们发现大多数PSC儿童在诊断时患有炎症性肠病或自身免疫性重叠和晚期纤维化。丙氨酸转氨酶和γ-谷氨酰转移酶水平在重叠综合征患者中最高，在小导管PSC患者中最低。UDCA治疗后血清肝酶水平恢复正常，包括自身免疫标志物阳性但无自身免疫性肝炎组织学特征的患者。
Background & Aims: Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease characterized by inflammation and progressive bile duct fibrosis. There are limited data on pediatric PSC. Methods: we performed a retrospective chart review of 47 pediatric patients with PSC. Results: The mean age at diagnosis was 11 +/- 4.9 years. symptoms occurred before presentation in 81% of patients; inflammatory bowel disease was found in 59% and autoimmune hepatitis (overlap syndrome) in 25% of patients. Magnetic resonance cholangiography revealed both extrahepatic and intrahepatic, isolated intrahepatic, isolated extrahepatic, and no biliary involvement (small-duct PSC) in 40%, 14%, 10%, and 36%, respectively. Advanced fibrosis (stage >II) was present in 65%. Colonoscopy revealed pancolitis, rectal sparing, and normal findings in 24%, 24%, and 18%, respectively. All patients were treated with ursodeoxycholic acid (UDCA); 9 with overlap syndrome also received immunosuppressants. Fifteen patients without overlap syndrome had positive autoimmune markers and responded to UDCA monotherapy. Liver transplantation was performed in 9 patients (3 with overlap syndrome and 2 with small-duct PSC) at a median time of 7 years after diagnosis. The 10-year post-transplant survival rate was 89%. Conclusions; In one of the largest single-center studies of children with PSC, we found that most children with PSC had inflammatory bowel disease or autoimmune overlap and advanced fibrosis at diagnosis. Levels of atanine aminotransferase and,gamma-glutamyl transferase were highest in patients with overlap syndrome and lowest in those with small-duct PSC. Levels of serum liver enzymes normalized after therapy with UDCA, including patients with positive autoimmune markers without histologic features of autoimmune hepatitis.