Appraising improvement in prediction of clinical outcomes based on different response criteria and liver stiffness measurements in patients with primary biliary cholangitis treated with second-line therapies in Journal of Hepatology

2025
ASL Asti
ASL Biella

Tipo pubblicazione

Conference Abstract

Autori/Collaboratori (100)Vedi tutti...

Vincentis AD
V. Negovsky Reanimatology Research Institute, Moscow, Russian Federation
Terracciani F
Research Unit of Internal Medicine, Universita Campus Bio-medico di Roma, Rome, Italy
D'Amato D
Research Unit of Hepatology, Universita Campus Bio-medico di Roma, Italy, Rome, Italy

et alii...

Abstract

Background and aims: The confirmation of clinical benefit for second-line therapies that showed efficacy on surrogate biomarkers may be difficult in the context of primary biliary cholangitis (PBC). For this reason, EMA suggested to adopt more stringent cut-offs of biochemical parameter used to define response criteria in forthcom¬ing clinical trials, and also to consider the variation of liver stiffness measurements (LSM). The aim of the present study was to appraise the improvement in prediction of liver-related events (LRE) based on the adoption of different response criteria and LSMs in patients with PBC treated with second-line therapies. Method: Data were from the Italian RECAPITULATE cohort, including PBC patients treated with Obeticholic acid (OCA) in centres belonging to the Italian PBC Registry and to the CLEO PBC Study group. After excluding patients with <6 months ' observation or with Child-Pugh B/C cirrhosis or previous hepatic decompensation, response to therapy was evaluated by liver enzymes and/or follow-up LSM, or by the following dichotomous criteria: ALP/ULN<1.5 with at least 40% reduction (ALP40); normal total bilirubin (TB) and ALP/ULN<1.67 with at least 15% drop (POISE); normalization of ALT, ALP and TB (normal range, NR); normalization of ALP and ALTand TB<0.7 (DEEP). LRE were defined as the occurrence of hepatic decompensation, liver transplantation or liver-related death. Cause-specific Cox regressions were fitted including the abovementioned response parameters as time-dependent covariates. Associations were evaluated through hazard ratios (HR), while predictive abilities through the c-statistics and log-partial-likelihood. Results: 677 PBC subjects (mean age 58, women 88%, cirrhotics 29%) were followed for a median of 30 months, and 31 LRE were registered. A sub-sample of 519 individuals with available LSM (total 946 LSMs) was also analysed. TB was the most strongly associated factor with LRE (HR 2.42, 95%CI 1.94-3.02, p< 0.001) a

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DOI : 10.1016/S0168-8278(25)00960-2

Keywords

bilirubin; biological marker; liver enzyme; obeticholic acid; adult; biliary cirrhosis; Child Pugh score; clinical outcome; cohort analysis; conference abstract; decompensated liver cirrhosis; diagnosis; drug therapy; effect size; female; follow up; human; liver cirrhosis; liver stiffness; liver transplantation; major clinical study; male; middle aged; prediction; primary biliary cirrhosis; second-line treatment;