Long-term effectiveness, safety, and liver stiffness dynamics of PBC treatment with obeticholic acid in real-world

After the conditional approval of OCA for the treatment of PBC, the main confirmatory study failed to demonstrate OCA's ability to reduce liver-related events, leading the EMA to revoke the drug's marketing authorization. The ensuing scientific debate highlights an urgent need for further evidence from real-world practice. In the largest real-world series of patients treated with OCA to date, we confirm that the drug's effectiveness and safety profiles are maintained over a medium-to-long follow-up period. Valuable data for the management of the drug in relevant subgroups of patients, such as those with cirrhosis and autoimmune hepatitis/PBC overlap syndrome, are also provided. Our original results on liver stiffness measurement variation over time suggest a favourable impact of OCA on fibrosis progression, particularly in patients achieving a biochemical response to the drug. Overall, these data provide important insights for clinicians managing patients with PBC and contribute to the ongoing scientific debate about the effectiveness/safety profile of this drug.

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Publication Details

Journal
JHEP Reports
Published
2025-05-27
DOI
https://doi.org/10.1016/j.jhepr.2025.101448
Citations
1
Primary Topic
Liver Diseases and Immunity
Type
article
Field-Weighted Citation Impact
1.32

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article

Long-term effectiveness, safety, and liver stiffness dynamics of PBC treatment with obeticholic acid in real-world

Francesco Squeo, Pietro Pozzoni, Filomena Morisco, Rosa Cotugno et al.
1 citations
JHEP Reports
Liver Diseases and Immunity
1.32
article

Long-term effectiveness, safety, and liver stiffness dynamics of PBC treatment with obeticholic acid in real-world

Francesco Squeo, Pietro Pozzoni, Filomena Morisco, Rosa Cotugno, Giuseppe Cuccorese, G Vettori, Armando Curto, Chiara Ricci, Carlo Saitta, Lorenzo Surace, Alessandro Mussetto, Biagio Cuffari, Adriano Pellicelli, Paola Begini, Edoardo G. Giannini, Francesca Terracciani, Mauro Viganò, Francesca Colapietro, Michela Miglianti, Vincenza Calvaruso, F. Rosina, A. Castellaneta, Fabrizio Pizzolante, Luchino Chessa, Gaetano Bertino, Marco Carbone, Olivia Morelli, Francesco Losito, Lory Saveria Crocè, Andrea Galli, Luigi Muratori, Umberto Vespasiani‐Gentilucci, Cristina Rigamonti, Nicoletta De Matthaeis, L. Capodicasa, Giancarlo Gimignani, Valerio Buzzanca, V. Boccaccio, Federica Cerini, Annalisa Tortora, Rodolfo Sacco, Raffaella Tortora, Giuliano Alagna, Daniela Campion, Alessio Gerussi, Maurizio Russello, Marco Marzioni, Alberto Mattalia, Vincenzo Valiani, Federico Salomone, Marta Borghi, Annarosa Floreani, Ana Lleò, Teresa Zolfino, Ilaria Cavalli, Sara Boninsegna, Daphne D’Amato, Valentina Boano, Federico D’Amico, Elisabetta Capello, Alessandra Moretti, Laura Cristoferi, Valentina Bellia, Ludovico Abenavoli, Pietro Invernizzi, Giulia Francesca Manfredi, Rosanna Venere, Anna Morgando, Paolo Poisa, Paolo Scivetti, Paolo Rollo, Leonardo Baiocchi, Antonio De Vincentis, Nora Cazzagon, Gerardo Nardone, N. Terreni, Maurizio Pompili, Fabio Marra, Barbara Agata Coco, Grazia Anna Niro, Maurizia Rossana Brunetto, Raffaele Cozzolongo, Elisabetta Degasperi, Domenico Alvaro, Sara Labanca, Luca Cadamuro, S. Fagiuoli, Donatella Ieluzzi, Debora Angrisani, Miki Scaravaglio, Riccardo Plebani, Guido Poggi, Pietro Lampertico, Ester Vanni, Silvia Casella, Antonio Izzi, Mariarita Cannavò, Raffaella Viganò, Loredana Simone, Valeria Pace Palitti, Guido Delle Monache, Emanuela Bonaiuto, Marco Distefano, Alessia Riva
article en
1 citations

Abstract

After the conditional approval of OCA for the treatment of PBC, the main confirmatory study failed to demonstrate OCA's ability to reduce liver-related events, leading the EMA to revoke the drug's marketing authorization. The ensuing scientific debate highlights an urgent need for further evidence from real-world practice. In the largest real-world series of patients treated with OCA to date, we confirm that the drug's effectiveness and safety profiles are maintained over a medium-to-long follow-up period. Valuable data for the management of the drug in relevant subgroups of patients, such as those with cirrhosis and autoimmune hepatitis/PBC overlap syndrome, are also provided. Our original results on liver stiffness measurement variation over time suggest a favourable impact of OCA on fibrosis progression, particularly in patients achieving a biochemical response to the drug. Overall, these data provide important insights for clinicians managing patients with PBC and contribute to the ongoing scientific debate about the effectiveness/safety profile of this drug.

JHEP ReportsVol. 7(8)
Università Cattolica del Sacro Cuore (IT), University of Messina (IT), University of Modena and Reggio Emilia (IT), Marche Polytechnic University (IT), Università degli Studi del Piemonte Orientale “Amedeo Avogadro” (IT), Humanitas University (IT), University of Padua (IT), University of Trieste (IT), Università Campus Bio-Medico (IT), University of Sassari (IT), University of Cagliari (IT), University of Perugia (IT), Casa Sollievo della Sofferenza (IT), Agostino Gemelli University Polyclinic (IT), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT), Magna Graecia University (IT), Ospedale San Paolo (IT), Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT), Ospedale Maggiore (IT), Ospedale Antonio Cardarelli (IT), Ospedale Santa Chiara (IT), Ospedale degli Infermi (IT), ASL Roma (IT), Istituti Clinici Scientifici Maugeri (IT), Ospedali Riuniti di Foggia (IT), Ospedale "Santa Maria delle Croci" di Ravenna (IT), Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT), Azienda Ospedaliera Universitaria Integrata Verona (IT), 5T Torino (Italy) (IT), IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola (IT), Ospedale San Filippo Neri (IT), Azienda Unità Sanitaria Locale di Ferrara (IT), Ospedale Sacro Cuore Don Calabria (IT), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Ospedale A. Perrino (IT), Nuovo Ospedale San Giovanni di Dio (IT), Azienda Ospedaliero Universitaria di Sassari (IT), Ospedale Garibaldi (IT), Ospedale Papa Giovanni XXIII (IT), Ospedale di Santo Spirito (IT), Centro Clinico Nemo (IT), Azienda Sanitaria Locale di Asti (IT), Ospedale San Paolo (IT), Alessandro Manzoni Hospital (IT), Federico II University Hospital (IT), Azienda Ospedaliero Universitaria Maggiore della Carita (IT), Presidio Ospedaliero (IT), Azienda Ospedaliera Sant'Andrea (IT), Arcispedale Sant'Anna (IT), Policlinico Universitario di Catania (IT), IRCCS San Camillo Hospital (IT), Azienda Sanitaria Ospedaliera S.Croce e Carle Cuneo (IT), Lawrence Livermore National Security (US), Guglielmo da Saliceto Hospital (IT), Policlinico Tor Vergata (IT), University of Palermo (AR), Campus Bio Medico University Hospital (IT), Ospedale Policlinico San Martino (IT), Azienda Ospedale - Università Padova (IT), Ospedale San Giuseppe (IT), Ospedale D. Cotugno (IT), University of the Sacred Heart (JP), Ospedale degli Infermi (IT), Azienda Socio Sanitaria Territoriale della Valtellina e Alto Lario (IT), Gastroenterology Hospital "Saverio de Bellis" (IT), Ospedale Valduce (IT), IRCCS Humanitas Research Hospital (IT), Azienda Sanitaria Universitaria Integrata di Trieste (IT), Ospedali Riuniti Umberto I (IT), Azienda Ospedaliera Universitaria Pisana (IT), Istituti Ospitalieri di Cremona (IT), Erregierre (Italy) (IT), Azienda Ospedaliera G. Brotzu (IT), University of Florence (IT), University of Bari Aldo Moro (IT), University of Milano-Bicocca (IT), University of Naples Federico II (IT), University of Genoa (IT), Sapienza University of Rome (IT), University of Palermo (IT)
Università degli Studi di Napoli Federico II, Università Degli Studi di Modena e Reggio Emila, Università degli Studi di Milano-Bicocca, Università Campus Bio-Medico di Roma, Università degli Studi di Palermo
Openalex Percentile: Top 20%
Liver Diseases and Immunity
1.32
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