Real-world experience with obeticholic acid in patients with primary biliary cholangitis
Daphne D’Amato, Antonio De Vincentis, Federica Malinverno, Mauro Viganò, Domenico Alvaro, Maurizio Pompili, A. Picciotto, Valeria Pace Palitti, Maurizio Russello, Silvia Storato, Marie Graciella Pigozzi, Vincenza Calvaruso, Elisabetta De Gasperi, Ana Lleò, A. Castellaneta, Adriano Pellicelli, Nora Cazzagon, Annarosa Floreani, Luigi Muratori, S. Fagiuoli, Grazia Anna Niro, Valentina Feletti, Raffaele Cozzolongo, N. Terreni, Marco Marzioni, Rinaldo Pellicano, Pietro Pozzoni, Leonardo Baiocchi, Luchino Chessa, F. Rosina, Gaetano Bertino, M. Vinci, Anna Morgando, Ester Vanni, Gaetano Scifo, Rodolfo Sacco, Maria D’Antò, Valentina Bellia, Roberto Boldizzoni, Silvia Casella, Barbara Omazzi, Guido Poggi, Laura Cristoferi, Alessio Gerussi, Vincenzo Ronca, Rosanna Venere, Francesca Romana Ponziani, M.R. Cannavò, Alessandro Mussetto, Rosanna Fontana, Francesco Losito, Evelise Frazzetto, Marco Distefano, Francesca Colapietro, Sara Labanca, Giulia Marconi, G Grassi, Giovanni Galati, Sarah O’Donnell, Clara Mancuso, Giacomo Mulinacci, Andrea Palermo, Ernesto Claar, Antonio Izzi, Antonio Picardi, Pietro Invernizzi, Marco Carbone, Umberto Vespasiani‐Gentilucci
Abstract
BACKGROUND & AIMS: Obeticholic acid (OCA) is the second-line treatment approved for patients with primary biliary cholangitis (PBC) and an inadequate response or intolerance to ursodeoxycholic acid. We aimed to evaluate the effectiveness and safety of OCA under real-world conditions. METHODS: , defined as normal levels of bilirubin, alkaline phosphatase (ALP), and alanine aminotransferase (ALT) at 12 months. Safety and tolerability were also assessed. RESULTS: = 0.04). Thirty-three patients (17%) prematurely discontinued OCA because of adverse events, of whom 11 experienced serious adverse events. Treatment-induced pruritus was the leading cause of OCA discontinuation (67%). CONCLUSIONS: Effectiveness and safety of OCA under real-world conditions mirror those in the Poise trial. Patients with cirrhosis had lower tolerability. Overlap PBC-AIH showed higher ALT reduction at 6 months compared with patients with pure PBC. LAY SUMMARY: Obeticholic acid (OCA) was shown to be effective in more than one-third of patients not responding to ursodeoxycholic acid in a real-world context in Italy. Patients with cirrhosis had more side effects with OCA, and this led to suspension of the drug in one-third of patients. OCA was also effective in patients who had overlap between autoimmune hepatitis and primary biliary cholangitis.