@article{dcf417a52efa42318c0811f5cf8ef301,
title = "Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium",
abstract = "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.",
keywords = "ACLF, AKI, Ascites, Cirrhosis, Clinical trials, Endpoints, Hepatic encephalopathy, Hyponatremia, Infections, Liver transplant, Quality of life",
author = "Elsa Sol{\`a} and Elisa Pose and Daniela Campion and Salvatore Piano and Olivier Roux and Macarena Simon-Talero and Frank Uschner and {de Wit}, Koos and Giacomo Zaccherini and Carlo Alessandria and Ulrich Beuers and Paolo Caraceni and Claire Francoz and Mookerjee, {Rajeshwar P.} and Jonel Trebicka and Victor Vargas and Miquel Serra and Ferran Torres and Sara Montagnese and Aleksander Krag and Ruben Hernaez and Marko Korenjak and Hugh Watson and Abraldes, {Juan G.} and Kamath, {Patrick S.} and Pere Gin{\`e}s and Durand F and Bernardi M and Sol{\'e} C and Pich J and Graupera I and Napoleone L and Jim{\'e}nez C and Juanola A and Avitabile E and Ma A and Fabrellas N and Carol M and Palacio E and Aban M and Lanzillotti T and Nicolao G and Chiappa, {M. T.} and Esnault V and A. Forner and S. Graf-Dirmeier and Helder J and Lopez M and Cervera M and M. P{\'e}rez-Guasch",
note = "Funding Information: The meeting was organised by the LiverHope consortium with the objective of proposing recommendations for the design and selection of study populations for clinical trials in decompensated cirrhosis. LiverHope is a 7-nation consortium composed of 9 European hospitals with extensive experience in hepatology that is aimed at exploring new therapies for cirrhosis ( www.liverhope-h2020.eu ). The Consortium is funded by the European Commission under the Horizon 20/20 program. Within the consortium, patients are represented by the European Liver Patients Association (ELPA). The LiverHope consortium convened a group of experts including a body of investigators, members of ELPA, pharmaceutical industry representatives, medical statisticians, and health economists, as well as members of the LiverHope external advisory board to participate in a special workshop. The current position document summarises the areas of discussion and consensus among members of the conference. Funding Information: ES, CA, RH, EP, SM, CF, RM have nothing to declare. The other authors declare the following: PG has participated on advisory boards for the following: Grifols, Gilead, Intercept, Mallinckrodt and Ferring. He has received Investigator promoted research grants from Mallinckrodt, Gilead and Grifols. All outside the submitted work. SM research group has received consultancy fees and/or research donations from Merz Pharmaceuticals GmbH, Norgine, Alfasigma and Umecrine Cognition. VV speaking fees from Intercept Pharmaceuticals; advisory board fees from Promethera Biosciences PC Grifols SA: advisory board, speaking bureau; Octapharma. SA: speaking bureau; Takeda SA: speaking bureau. JT: reports other from - Gore, Bayer, Alexion, MSD, Gilead, Intercept, Norgine, Grifols, Versantis, Martin Pharmaceuticals outside the submitted work. PSK is Principal Investigator of the Sequana Medical sponsored POSEIDON study in North America. He does not receive any personal remuneration. HW declares he is currently and employee of EVOTEC and has shares in Sanofi, both of which are unrelated to the work submitted. JGA reports grants from Gilead, lecture fees from Ferring, and consulting fees from Gilead, Pfizer, Intercept, Boehringer-Ingelheim, Lupin and Genfit outside the submitted work. UB Research funding from South African PSC Patient Foundation, Netherlands gastroenterology and hepatology (MDLS) Foundation, Investigator initiated research funding from Intercept, USA. AK Speaker and advisory board: Norgine, Siemens. Funding Information: The work described has been funded by a H2020 Grant – LIVERHOPE – Project number: 731875 . PG, ES and EP have also been funded by a grant from AGAUR ( 2017-SGR-01281 ). Publisher Copyright: {\textcopyright} 2020 European Association for the Study of the Liver",
year = "2021",
month = jan,
doi = "10.1016/j.jhep.2020.08.009",
language = "English (US)",
volume = "74",
pages = "200--219",
journal = "Journal of hepatology",
issn = "0168-8278",
publisher = "Elsevier",
number = "1",
}