Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium
Elsa Solà, Elisa Pose, Daniela Campion, Salvatore Piano, Olivier Roux, Macarena Simón‐Talero, Frank Erhard Uschner, Koos de Wit, Giacomo Zaccherini, Carlo Alessandria, Ulrich Beuers, Paolo Caraceni, Claire Francoz, Rajeshwar P. Mookerjee, Jonel Trebicka, Vı́ctor Vargas, Miquel Serra‐Burriel, Ferràn Torres, Sara Montagnese, Aleksander Krag, Rubén Hernáez, Marko Korenjak, Hugh Watson, Juan G. Abraldeṣ, Patrick S. Kamath, Pere Ginès, François Durand, Mauro Bernardi, Cristina Solé, Judit Pich, Isabel Graupera, Laura Napoleone, César Jiménez, Adrià Juanola, Emma Avitabile, Ann T., Núria Fabrellas, Marta Carol, E. Palacio, Meral Aban, Tommaso Lanzillotti, Giuseppe De Nicolao, Michela Chiappa, Vincent Esnault, Alejandro Forner, Sabine Graf‐Dirmeier, Jeltje Helder, Marta López, Marta Cervera, Martina Pérez
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.