Outcomes and potential surrogate markers for future clinical trials of non-alcoholic steatohepatitis cirrhosis
Rivera-Esteban, Jesús (Universitat Autònoma de Barcelona)
Armandi, Angelo (University of Turin)
Augustin Recio, Salvador (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Bugianesi, Elisabetta (University of Turin)

Date:	2021
Abstract:	Non-alcoholic steatohepatitis has emerged as a major public health problem, and the burden of non-alcoholic steatohepatitis cirrhosis is projected to increase by 64%-156% by 2030. The threat is aggravated by the fact that are currently no approved drugs for the treatment of non-alcoholic steatohepatitis. In this paper, we review the main challenges to drug development in patients with non-alcoholic steatohepatitis cirrhosis, and describe the opportunities brought by the advances in the understanding of the clinical and pathophysiological nuances of cirrhosis. The design of therapeutic regimens for non-alcoholic steatohepatitis cirrhosis will vary according to the specific cirrhosis substage (compensated vs decompensated), and the specific mechanistic basis of therapy, targeted either at improving aetiology-specific pathways and/or at more general aetiology-agnostic processes. The understanding of the probabilistic expectations for the whole range of potential outcomes, rooted at different mechanistic drivers at each specific substage, will be essential in order to choose adequate estimands and therapeutic strategies for clinical trials and individual patients with non-alcoholic steatohepatitis cirrhosis. Finally, we provide a summary of the main pitfalls and uncertainties in the design of clinical trials for non-alcoholic steatohepatitis cirrhosis and discuss potential biomarkers for use in trials and practice for these patients.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Biomarkers ; Clinical trials ; Liver cirrhosis ; Metabolic associated fatty liver disease (MAFLD) ; Portal hypertension ; Portal pressure
Published in:	Liver International, Vol. 41 (july 2021) , p. 1999-2008, ISSN 1478-3231

DOI: 10.1111/liv.15013
PMID: 34242466

10 p, 402.5 KB

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