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Abstract Details
Real-world experience with obeticholic acid in patients with primary biliary cholangitis
JHEP Rep. 2021 Jan 27;3(2):100248. doi: 10.1016/j.jhepr.2021.100248. eCollection 2021 Apr.
Daphne D'Amato1, Antonio De Vincentis2, Federica Malinverno1, Mauro Viganò3, Domenico Alvaro4, Maurizio Pompili5, Antonino Picciotto6, Valeria Pace Palitti7, Maurizio Russello8, Silvia Storato9, Marie Graciella Pigozzi10, Vincenza Calvaruso11, Elisabetta De Gasperi12, Ana Lleo13, Antonino Castellaneta14, Adriano Pellicelli15, Nora Cazzagon16, Annarosa Floreani16, Luigi Muratori17, Stefano Fagiuoli18, Grazia Anna Niro19, Valentina Feletti20, Raffaele Cozzolongo21, Natalia Terreni22, Marco Marzioni23, Rinaldo Pellicano24, Pietro Pozzoni25, Leonardo Baiocchi26, Luchino Chessa27, Floriano Rosina28, Gaetano Bertino29, Maria Vinci30, Anna Morgando24, Ester Vanni24, Gaetano Scifo31, Rodolfo Sacco32, Maria D'Antò33, Valentina Bellia34, Roberto Boldizzoni35, Silvia Casella36, Barbara Omazzi37, Guido Poggi38, Laura Cristoferi1, Alessio Gerussi1, Vincenzo Ronca1, Rosanna Venere4, Francesca Ponziani5, Maria Cannavò8, Alessandro Mussetto20, Rosanna Fontana19, Francesco Losito21, Evelise Frazzetto29, Marco Distefano31, Francesca Colapietro13, Sara Labanca6, Giulia Marconi23, Giuseppe Grassi26, Giovanni Galati2, Sarah Elizabeth O'Donnell1, Clara Mancuso1, Giacomo Mulinacci1, Andrea Palermo1, Ernesto Claar39, Antonio Izzi40, Antonio Picardi2, Pietro Invernizzi1, Marco Carbone1, Umberto Vespasiani-Gentilucci2, Italian PBC Registry and the Club Epatologi Ospedalieri (CLEO)/Associazione Italiana Gastroenterologi ed Endoscopisti Digestivi Ospedalieri (AIGO) PBC Study Group
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: Patients were recruited into the Italian PBC Registry, a multicentre, observational cohort study that monitors patients with PBC at national level. The primary endpoint was the biochemical response according to Poise criteria; the secondary endpoint was the biochemical response according to normal range criteria, defined as normal levels of bilirubin, alkaline phosphatase (ALP), and alanine aminotransferase (ALT) at 12 months. Safety and tolerability were also assessed.
Results: We analysed 191 patients until at least 12 months of follow-up. Median age was 57 years, 94% female, 61 (32%) had cirrhosis, 28 (15%) had histologically proven overlap with autoimmune hepatitis (PBC-AIH). At 12 months, significant median reductions of ALP (-32.3%), ALT (-31.4%), and bilirubin (-11.2%) were observed. Response rates were 42.9% according to Poise criteria, and 11% by normal range criteria. Patients with cirrhosis had lower response than patients without cirrhosis (29.5% vs. 49.2%, p = 0.01), owing to a higher rate of OCA discontinuation (30% vs. 12%, p = 0.004), although with similar ALP reduction (29.4% vs. 34%, p = 0.53). Overlap PBC-AIH had a similar response to pure PBC (46.4% vs. 42.3%, p = 0.68), with higher ALT reduction at 6 months (-38% vs. -29%, p = 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.
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