Division of Liver Diseases, Icahn School of Medicine at Mount Sinai, New York, NY, 10029, USA.
Hepatol Int. 2018 Nov;12(6):510-519. doi: 10.1007/s12072-018-9906-6. Epub 2018 Nov 14.
Primary sclerosing cholangitis (PSC) is a chronic inflammatory disease of the biliary tree of unknown etiology leading to stricturing and dilation. There is currently no effective medical therapy for PSC and liver transplantation (LT) remains the ultimate treatment for severe disease defined as repeated episodes of cholangitis, decompensated biliary cirrhosis or in exceptional cases, cholangiocarcinoma (CCA). Patients often present with a "dominant" stricture and the therapeutic endoscopist plays an important role in management to improve biliary patency using a variety of techniques that involve sampling, balloon dilation and temporary stenting. Newer modalities such as self-expanding metal stents or magnetic compression anastomosis that have been used in other diseases may have a role to play in PSC but should remain investigational. Liver transplantation for PSC is curative in most cases but the optimal timing remains unclear. The lifetime risk of CCA is 10-15% in PSC patients and LT is often not possible at the time of diagnosis. Multiple studies have tried to identify risk factors and to diagnose CCA at an early stage when surgical resection may be possible or LT can be performed. However, deceased donor organs for LT remain in short supply throughout the world so even identifying PSC patients with CCA at an early stage may not be beneficial unless a live donor organ is available.
原发性硬化性胆管炎（PSC）是一种病因不明的胆道慢性炎症性疾病，可导致胆管狭窄和扩张。目前尚无有效的医学治疗方法，肝移植（LT）仍然是严重疾病的最终治疗方法，这些严重疾病的定义为反复发作的胆管炎、失代偿性胆汁性肝硬化或在特殊情况下的胆管癌（CCA）。患者通常表现为“优势”狭窄，治疗性内镜医生在管理中起着重要作用，可使用各种技术来改善胆汁通畅性，这些技术包括取样、球囊扩张和临时支架置入。在其他疾病中使用的新型方法，如自膨式金属支架或磁压缩吻合术，可能在 PSC 中发挥作用，但仍需进一步研究。大多数情况下，肝移植对 PSC 是有效的，但最佳时机仍不清楚。PSC 患者的终身患癌风险为 10-15%，且在诊断时往往无法进行 LT。多项研究试图确定风险因素，并在可能进行手术切除或 LT 时早期诊断 CCA。然而，全世界供 LT 的已故供体器官仍然短缺，因此，即使能早期识别出 PSC 合并 CCA 的患者，如果没有活体供体器官，可能也无济于事。
