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 Simon-Talero, Frank Uschner, Koos de Wit, Giacomo Zaccherini, Carlo Alessandria, Ulrich Beuers, Paolo Caraceni, Claire Francoz, Rajeshwar P. Mookerjee, Jonel Trebicka, Victor Vargas, Miquel Serra, Ferran Torres, Sara Montagnese, Aleksander KragRuben Hernaez, Marko Korenjak, Hugh Watson, Juan G. Abraldes, Patrick S. Kamath, Pere Ginès, Durand F, Bernardi M, Solé C, Pich J, Graupera I, Napoleone L, Jiménez C, Juanola A, Avitabile E, Ma A, Fabrellas N, Carol M, Palacio E, Aban M, Lanzillotti T, Nicolao G, M. T. Chiappa, Esnault V, A. Forner, S. Graf-Dirmeier, Helder J, Lopez M, Cervera M, M. Pérez-Guasch

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Pages (from-to)200-219
Number of pages20
JournalJournal of hepatology
Volume74
Issue number1
DOIs
StatePublished - Jan 2021

Keywords

  • ACLF
  • AKI
  • Ascites
  • Cirrhosis
  • Clinical trials
  • Endpoints
  • Hepatic encephalopathy
  • Hyponatremia
  • Infections
  • Liver transplant
  • Quality of life

ASJC Scopus subject areas

  • Hepatology

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