Solà Elsa, Pose Elisa, Campion Daniela, Piano Salvatore, Roux Olivier, Simon-Talero Macarena, Uschner Frank, de Wit Koos, Zaccherini Giacomo, Alessandria Carlo, Beuers Ulrich, Caraceni Paolo, Francoz Claire, Mookerjee Rajeshwar P, Trebicka Jonel, Vargas Victor, Serra Miquel, Torres Ferran, Montagnese Sara, Krag Aleksander, Hernaez Ruben, Korenjak Marko, Watson Hugh, Abraldes Juan G, Kamath Patrick S, Ginès Pere
Liver Unit, Hospital Clínic De Barcelona, Barcelona, School of Medicine and Health Sciences University of Barcelona, Spain; Institut d´Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEReHD), Barcelona, Spain.
Division of Gastroenterology and Hepatology, Città della Salute e della Scienza Hospital, University of Turin, Turin, Italy.
J Hepatol. 2021 Jan;74(1):200-219. doi: 10.1016/j.jhep.2020.08.009. Epub 2020 Sep 5.
Management of decompensated cirrhosis is currently geared towards the treatment of complications once they occur. To date there is no established disease-modifying therapy aimed at halting progression of the disease and preventing the development of complications in patients with decompensated cirrhosis. The design of clinical trials to investigate new therapies for patients with decompensated cirrhosis is complex. The population of patients with decompensated cirrhosis is heterogeneous (i.e., different etiologies, comorbidities and disease severity), leading to the inclusion of diverse populations in clinical trials. In addition, primary endpoints selected for trials that include patients with decompensated cirrhosis are not homogeneous and at times may not be appropriate. This leads to difficulties in comparing results obtained from different trials. Against this background, the LiverHope Consortium organized a meeting of experts, the goal of which was to develop recommendations for the design of clinical trials and to define appropriate endpoints, both for trials aimed at modifying the natural history and preventing progression of decompensated cirrhosis, as well as for trials aimed at managing the individual complications of cirrhosis.
失代偿期肝硬化的管理目前主要针对并发症出现后的治疗。迄今为止，尚无既定的疾病改善疗法旨在阻止疾病进展并预防失代偿期肝硬化患者出现并发症。针对失代偿期肝硬化患者研究新疗法的临床试验设计很复杂。失代偿期肝硬化患者群体具有异质性（即病因、合并症和疾病严重程度不同），导致临床试验纳入了多样化的人群。此外，针对包括失代偿期肝硬化患者的试验所选择的主要终点并不统一，有时可能也不合适。这导致比较不同试验结果存在困难。在此背景下，LiverHope联盟组织了一次专家会议，其目的是为临床试验设计制定建议，并确定合适的终点，这些终点既适用于旨在改变自然病程和预防失代偿期肝硬化进展的试验，也适用于旨在处理肝硬化个体并发症的试验。
