Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium
Open accessAbstract
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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Best endpoints including transplant-free survival were treated as secondary outcomes in the MATCH phase II study.
“and what are now considered the best endpoints, including (transplant-free) survival were treated as secondary outcomes in the MATCH phase II study (179).”
From Targeting macrophages in liver fibrosis (2026) · cites this paper as [179]
Cites
Fernández, J., Navasa, M., Planas, R., Montoliu, S., Monfort, D., Soriano, G., Vila, C., Pardo, A., Quintero, E., Vargas, V., et al. (2007) · Gastroenterology · doi:10.1053/j.gastro.2007.06.065 · Cited by 709 · see citing works
Self-citationGinès, P., Solà, E., Angeli, P., Wong, F., Nadim, M. K., & Kamath, P. S. (2018) · Nature Reviews Disease Primers · doi:10.1038/s41572-018-0022-7 · Cited by 260 · see citing works
Self-citationGinès, P., Wong, F., Watson, H., Milutinovic, S., Ruiz del Arbol, L., & Olteanu, D. (2008) · Hepatology · doi:10.1002/hep.22293 · Cited by 193 · see citing works
Self-citationPose, E., Napoleone, L., Amin, A., Campion, D., Jimenez, C., Piano, S., Roux, O., Uschner, F. E., de Wit, K., Zaccherini, G., et al. (2020) · The Lancet Gastroenterology & Hepatology · doi:10.1016/s2468-1253(19)30320-6 · Cited by 154 · see citing works
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Targeting macrophages in liver fibrosis
González del Barrio, L., Ipsen, D. H., Pfister, D. R., Hancz, D., & Scott, C. L. (2026) · Frontiers in Immunology · doi:10.3389/fimmu.2026.1835489 · Cited by 3 · see citing works
“…score is not the most optimal endpoint for evaluating therapeutic ef fi cacy in trials targeting fi brosis progression and resolution (177, 178), and what are now considered the best endpoints, including (transplant-free) survival were treated as secondary outcomes in the MATCH phase II study (179).”
- Authors
- 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., Krag, A., Hernaez, R., Korenjak, M., Watson, H., Abraldes, J. G., Kamath, P. S., Ginès, P., 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.
- Affiliations
- Hospital Clínic de Barcelona (ES)
- Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT)
- University of Padua (IT)
- Hôpital Beaujon (FR)
- Goethe University Frankfurt (DE)
- University of Bologna (IT)
- Amsterdam UMC Location University of Amsterdam (NL)
- University College London (GB)
- University of Southern Denmark (DK)
- Aarhus University (DK)
- Mayo Clinic (US)
- University of Alberta (CA)
- Corresponding author
- Ginès, Pere
- ORCID
- Solà, Elsa
- Pose, Elisa
- Campion, Daniela
- Piano, Salvatore
- Roux, Olivier
- Simon-Talero, Macarena
- Uschner, Frank
- Zaccherini, Giacomo
- Alessandria, Carlo
- Beuers, Ulrich
- Caraceni, Paolo
- Francoz, Claire
- Mookerjee, Rajeshwar P.
- Trebicka, Jonel
- 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.
- Graupera, I.
- Napoleone, L.
- Jiménez, C.
- Juanola, A.
- Avitabile, E.
- Fabrellas, N.
- Nicolao, G.
- Forner, A.
- Helder, J.
- Cervera, M.
- Pérez-Guasch, M.
- Venue
- Journal of Hepatology
- Publisher
- Elsevier BV
- Published
- 2021-01
- Volume
- 74
- Issue
- 1
- Pages
- 200-219
- ISSN
- 0168-8278 (ISSN-L)
- OpenAlex
- W3083453401
- Cited by
- 41
- References
- 4
- Citations by year
- 2026: 9 · 2025: 5 · 2024: 14 · 2023: 4 · 2022: 3 · 2021: 6
- FWCI
- 1.91
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Journal of Hepatology (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Pure Amsterdam UMC (acceptedVersion, cc-by)
- University of Southern Denmark Research Portal (University of Southern Denmark) (acceptedVersion, cc-by-nc-nd)
- Institutional Research Information System University of Turin (University of Turin) (submittedVersion, other-oa)
- Data Archiving and Networked Services (DANS) (submittedVersion, other-oa)
- Archivio istituzionale della ricerca (Alma Mater Studiorum Università di Bologna) (submittedVersion, other-oa)
- Dipòsit Digital de la Universitat de Barcelona (Universitat de Barcelona) (submittedVersion, cc-by-nc-nd)
- UCL Discovery (University College London) (submittedVersion)
- Publication Server of Goethe University Frankfurt am Main (Goethe University Frankfurt) (submittedVersion, cc-by-nc-nd)
- Padua Research Archive (University of Padova) (submittedVersion, cc-by-nc-nd)
- Funders
- European Commission
- Topics
- Health Sciences › Medicine › Hepatology › Liver Disease and Transplantation
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Health Sciences › Medicine › Epidemiology › Liver Disease Diagnosis and Treatment
- Keywords
- decompensated cirrhosis, clinical trial design, primary endpoint, disease-modifying therapy, disease progression, cirrhosis complications, trial heterogeneity, natural history (disease)
- MeSH terms
- Ascites, Clinical Trials as Topic, Europe, Hepatic Encephalopathy, Humans, Hypertension, Portal, Liver Cirrhosis, Quality of Life, Research Design, Severity of Illness Index, Disease Progression, Disease Management, Endpoint Determination, Consensus, Secondary Prevention
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