Engel Bastian, Assis David N, Bhat Mamatha, Clusmann Jan, Drenth Joost Ph, Gerussi Alessio, Londoño María-Carlota, Oo Ye Htun, Schregel Ida, Sebode Marcial, Taubert Richard
Department of Gastroenterology, Hepatology, Infectious Diseases and Endocrinology, Hannover Medical School, Hannover, Germany.
Yale School of Medicine, New Haven, CT USA.
JHEP Rep. 2024 Nov 12;7(2):101265. doi: 10.1016/j.jhepr.2024.101265. eCollection 2025 Feb.
Autoimmune hepatitis (AIH) is a rare chronic liver disease with an increasing incidence in many countries. Chronic autoimmune responses against the liver can cause hepatic and extrahepatic symptoms, decreased quality of life and reduced liver transplant-free survival if inadequately treated. Although standard treatment with corticosteroids and thiopurines improves the life expectancy of patients with AIH, remission rates and tolerability are generally overestimated and the development of alternative first-line and salvage therapies has been disappointingly slow compared to in rheumatological diseases or inflammatory bowel disease. Other gaps include the lack of disease-specific diagnostic markers for AIH. Similarly, the new entity of drug-induced autoimmune-like hepatitis underscores the need to re-evaluate previous diagnostic criteria. The International AIH Group (IAIHG) has initiated a series of research workshops over the last decade to promote the identification of research gaps and subsequently improve the pace of scientific progress by stimulating collaboration between expert centres. This review reports on the results of the 5 Research Workshop, held in Hannover, Germany in June 2024, and summarises the progress made since the 4 Workshop in 2022. Patient representatives from the European Reference Network (ERN) Rare Liver Youth Panel participated in the workshop. The specific objectives of this year's 5 Workshop were: (1) To further improve diagnostics. (2) Initiate clinical trials including knowledge transfer on drugs from extrahepatic immune-mediated diseases, including B cell-depleting CAR T cells. (3) Utilisation of multi-omics approaches to improve the understanding of disease pathogenesis. (4) Application of machine learning-based approaches established in oncology or transplantation medicine to improve diagnosis and outcome prediction in AIH.
自身免疫性肝炎(AIH)是一种罕见的慢性肝病,在许多国家发病率呈上升趋势。针对肝脏的慢性自身免疫反应可导致肝脏和肝外症状,若治疗不当,会降低生活质量并缩短无肝移植生存期。尽管使用皮质类固醇和硫嘌呤进行标准治疗可提高AIH患者的预期寿命,但缓解率和耐受性通常被高估,与风湿性疾病或炎症性肠病相比,替代一线和挽救疗法的开发进展令人失望地缓慢。其他差距包括缺乏AIH疾病特异性诊断标志物。同样,药物性自身免疫样肝炎这一新实体凸显了重新评估先前诊断标准的必要性。国际自身免疫性肝炎小组(IAIHG)在过去十年中发起了一系列研究研讨会,以促进识别研究差距,并随后通过促进专家中心之间的合作来加快科学进步的步伐。本综述报告了2024年6月在德国汉诺威举行的第5次研究研讨会的结果,并总结了自2022年第4次研讨会以来取得的进展。欧洲参考网络(ERN)罕见肝脏青年小组的患者代表参加了此次研讨会。今年第5次研讨会的具体目标是:(1)进一步改善诊断。(2)启动临床试验,包括从肝外免疫介导疾病(包括B细胞耗竭性CAR T细胞)中进行药物知识转移。(3)利用多组学方法加深对疾病发病机制的理解。(4)应用肿瘤学或移植医学中建立的基于机器学习的方法来改善AIH的诊断和结局预测。