Engel Bastian, Taubert Richard, Jaeckel Elmar, Manns Michael P
Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Hannover, Germany.
European Reference Network on Hepatological Diseases (ERN RARE-LIVER).
Liver Int. 2020 Feb;40 Suppl 1:149-153. doi: 10.1111/liv.14378.
Autoimmune liver diseases (AILD), namely autoimmune hepatitis (AIH), primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC), are rare diseases. These days, patients with PBC almost never require liver transplantation. When treated early with ursodeoxycholic acid patients have a normal life expectancy if the disease is diagnosed at an early stage and the patients respond to treatment. Patients with AIH often go into remission with first-line therapy including corticosteroids alone or in combination with azathioprine. Nevertheless, about one quarter of patients already developed cirrhosis at diagnosis. Those who do not respond to first line standard of care (SOC) have significant liver-related morbidity and mortality. No approved second- or third-line treatments are available and the drugs are selected based on limited case series and personal experience. Larger trials are needed to develop efficient therapies for difficult-to-treat AIH patients. No treatment has been found to alter the natural course of disease in patients with PSC except for liver transplantation. Identifying PSC patients at risk of developing cholangiocarcinoma (CCA) is another unmet need. Current research in all AILD including AIH, PBC and PSC, focuses on improving our understanding of the underlying disease process and identifying new therapeutic targets to decrease morbidity and mortality.
自身免疫性肝病（AILD），即自身免疫性肝炎（AIH）、原发性胆汁性胆管炎（PBC）和原发性硬化性胆管炎（PSC），是罕见疾病。如今，PBC患者几乎从不需进行肝移植。如果在疾病早期诊断且患者对治疗有反应，用熊去氧胆酸早期治疗的患者预期寿命正常。AIH患者采用包括单独使用皮质类固醇或与硫唑嘌呤联合使用的一线治疗往往会缓解。然而，约四分之一的患者在诊断时已发展为肝硬化。那些对一线标准治疗（SOC）无反应的患者有显著的肝脏相关发病率和死亡率。目前尚无获批的二线或三线治疗方法，药物选择基于有限的病例系列和个人经验。需要开展更大规模的试验来为难治性AIH患者研发有效的治疗方法。除肝移植外，尚未发现有治疗方法能改变PSC患者的疾病自然进程。识别有发展为胆管癌（CCA）风险的PSC患者是另一个未满足的需求。目前对包括AIH、PBC和PSC在内的所有AILD的研究都集中在增进我们对潜在疾病过程的理解以及确定新的治疗靶点以降低发病率和死亡率。
