Montano-Loza Aldo J, Allegretti Jessica R, Cheung Angela, Ebadi Maryam, Jones David, Kerkar Nanda, Levy Cynthia, Rizvi Sumera, Vierling John M, Alvarez Fernando, Bai Wayne, Gilmour Susan, Gulamhusein Aliya, Guttman Orlee, Hansen Bettina E, MacParland Sonya, Mason Andrew, Onofrio Fernanda, Santamaria Pere, Stueck Ashley, Swain Mark, Vincent Catherine, Ricciuto Amanda, Hirschfield Gideon
Division of Gastroenterology and Liver Unit, University of Alberta, Edmonton, Alberta, Canada.
Division of Gastroenterology, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Can Liver J. 2021 Nov 11;4(4):401-425. doi: 10.3138/canlivj-2021-0006. eCollection 2021 Fall.
Autoimmune liver disease (AILD) spans a spectrum of chronic disorders affecting the liver parenchyma and biliary system. Three main categories of AILD are autoimmune hepatitis (AIH), primary biliary cirrhosis (PBC), and primary sclerosing cholangitis (PSC). This review condenses the presentation and discussions of the Single Topic Conference (STC) on AILD that was held in Ottawa, Ontario, in November 2019. We cover generalities regarding disease presentation and clinical diagnosis; mechanistic themes; treatment paradigms; clinical trials, including approaches and challenges to new therapies; and looking beyond traditional disease boundaries. Although these diseases are considered autoimmune, the etiology and role of environmental triggers are poorly understood. AILDs are progressive and chronic conditions that affect survival and quality of life. Advances have been made in PBC treatment because second-line treatments are now available (obeticholic acid, bezafibrate); however, a significant proportion still present suboptimal response. AIH treatment has remained unchanged for several decades, and data suggest that fewer than 50% of patients achieve a complete response and as many as 80% develop treatment-related side effects. B-cell depletion therapy to treat AIH is in an early stage of development and has shown promising results. An effective treatment for PSC is urgently needed. Liver transplant remains the best option for patients who develop decompensated cirrhosis or hepatocellular carcinoma within specific criteria, but recurrent AILD might occur. Continued efforts are warranted to develop networks for AILD aimed at assessing geo-epidemiological, clinical, and biochemical differences to capture the new treatment era in Canada.
自身免疫性肝病(AILD)涵盖了一系列影响肝实质和胆道系统的慢性疾病。AILD主要分为三类:自身免疫性肝炎(AIH)、原发性胆汁性肝硬化(PBC)和原发性硬化性胆管炎(PSC)。本综述总结了2019年11月在安大略省渥太华举行的AILD单主题会议(STC)的报告和讨论内容。我们涵盖了疾病表现和临床诊断的一般情况;发病机制主题;治疗模式;临床试验,包括新疗法的方法和挑战;以及突破传统疾病界限。尽管这些疾病被认为是自身免疫性的,但其环境触发因素的病因和作用仍知之甚少。AILD是影响生存和生活质量的进行性慢性疾病。PBC治疗已取得进展,因为现在有了二线治疗药物(奥贝胆酸、苯扎贝特);然而,仍有相当一部分患者反应欠佳。几十年来,AIH的治疗方法一直没有改变,数据表明,不到50%的患者能完全缓解,多达80%的患者会出现与治疗相关的副作用。用于治疗AIH的B细胞耗竭疗法尚处于早期开发阶段,但已显示出有希望的结果。迫切需要一种有效的PSC治疗方法。对于符合特定标准出现失代偿性肝硬化或肝细胞癌的患者,肝移植仍然是最佳选择,但可能会发生AILD复发。有必要继续努力建立AILD网络,旨在评估地理流行病学、临床和生化差异,以迎接加拿大新的治疗时代。