Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium

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 cirrh...

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Published in:Journal of hepatology 2021-01, Vol.74 (1), p.200-219
Main Authors: 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, 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., Chiappa, M.T., Esnault, V., Forner, A., Graf-Dirmeier, S., Helder, J., Lopez, M., Cervera, M., Pérez-Guasch, M.
Format: Article
Language:English
Subjects:
ACLF
AKI
Ascites
Ascites - etiology
Ascites - therapy
Cirrhosis
Clinical trials
Clinical Trials as Topic
Consensus
Consortia
Disease Management
Disease Progression
Endpoint Determination
Endpoints
Europe
Hepatic encephalopathy
Hepatic Encephalopathy - etiology
Hepatic Encephalopathy - therapy
Humans
Hypertension, Portal - etiology
Hypertension, Portal - therapy
Hyponatremia
Infections
Liver cirrhosis
Liver Cirrhosis - complications
Liver Cirrhosis - physiopathology
Liver Cirrhosis - psychology
Liver Cirrhosis - therapy
Liver transplant
Patients
Quality of Life
Research Design
Secondary Prevention - methods
Severity of Illness Index
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Summary: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.
ISSN:0168-8278
1600-0641
DOI:10.1016/j.jhep.2020.08.009