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 Krag; Ruben 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

doi:10.1016/j.jhep.2020.08.009

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

Gastroenterology and Hepatology

Research output: Contribution to journal › Article › peer-review

2 Scopus citations

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 language	English (US)
Pages (from-to)	200-219
Number of pages	20
Journal	Journal of hepatology
Volume	74
Issue number	1
DOIs	https://doi.org/10.1016/j.jhep.2020.08.009
State	Published - 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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10.1016/j.jhep.2020.08.009

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Solà, E., Pose, E., Campion, D., Piano, S., Roux, O., Simon-Talero, M., Uschner, F., de Wit, K., Zaccherini, G., Alessandria, C., Beuers, U., Caraceni, P., Francoz, C., Mookerjee, R. P., Trebicka, J., Vargas, V., Serra, M., Torres, F., Montagnese, S., ... Pérez-Guasch, M. (2021). Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium. Journal of hepatology, 74(1), 200-219. https://doi.org/10.1016/j.jhep.2020.08.009

Solà, E, Pose, E, Campion, D, Piano, S, Roux, O, Simon-Talero, M, Uschner, F, de Wit, K, Zaccherini, G, Alessandria, C, Beuers, U, Caraceni, P, Francoz, C, Mookerjee, RP, Trebicka, J, Vargas, V, Serra, M, Torres, F, Montagnese, S, Krag, A, Hernaez, R, Korenjak, M, Watson, H, Abraldes, JG, Kamath, PS, Ginès, P, F, D, M, B, C, S, J, P, I, G, L, N, C, J, A, J, E, A, A, M, N, F, M, C, E, P, M, A, T, L, G, N, Chiappa, MT, V, E, Forner, A, Graf-Dirmeier, S, J, H, M, L, M, C & Pérez-Guasch, M 2021, 'Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium', Journal of hepatology, vol. 74, no. 1, pp. 200-219. https://doi.org/10.1016/j.jhep.2020.08.009

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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 = "Publisher Copyright: {\textcopyright} 2020 European Association for the Study of the Liver",

year = "2021",

month = jan,

doi = "10.1016/j.jhep.2020.08.009",

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AU - Solà, Elsa

AU - Pose, Elisa

AU - Campion, Daniela

AU - Piano, Salvatore

AU - Roux, Olivier

AU - Simon-Talero, Macarena

AU - Uschner, Frank

AU - de Wit, Koos

AU - Zaccherini, Giacomo

AU - Alessandria, Carlo

AU - Beuers, Ulrich

AU - Caraceni, Paolo

AU - Francoz, Claire

AU - Mookerjee, Rajeshwar P.

AU - Trebicka, Jonel

AU - Vargas, Victor

AU - Serra, Miquel

AU - Torres, Ferran

AU - Montagnese, Sara

AU - Krag, Aleksander

AU - Hernaez, Ruben

AU - Korenjak, Marko

AU - Watson, Hugh

AU - Abraldes, Juan G.

AU - Kamath, Patrick S.

AU - Ginès, Pere

AU - F, Durand

AU - M, Bernardi

AU - C, Solé

AU - J, Pich

AU - I, Graupera

AU - L, Napoleone

AU - C, Jiménez

AU - A, Juanola

AU - E, Avitabile

AU - A, Ma

AU - N, Fabrellas

AU - M, Carol

AU - E, Palacio

AU - M, Aban

AU - T, Lanzillotti

AU - G, Nicolao

AU - Chiappa, M. T.

AU - V, Esnault

AU - Forner, A.

AU - Graf-Dirmeier, S.

AU - J, Helder

AU - M, Lopez

AU - M, Cervera

AU - Pérez-Guasch, M.

PY - 2021/1

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N2 - 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.

AB - 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.

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KW - Ascites

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KW - Endpoints

KW - Hepatic encephalopathy

KW - Hyponatremia

KW - Infections

KW - Liver transplant

KW - Quality of life

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

Abstract

Keywords

ASJC Scopus subject areas

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