Real-world experience with obeticholic acid in patients with primary biliary cholangitis.
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D'Amato Daphne, De Vincentis Antonio, Malinverno Federica, Viganò Mauro, Alvaro Domenico, Pompili Maurizio, Picciotto Antonino, Palitti Valeria Pace, Russello Maurizio, Storato Silvia, Pigozzi Marie Graciella, Calvaruso Vincenza, De Gasperi Elisabetta, Lleo Ana, Castellaneta Antonino, Pellicelli Adriano, Cazzagon Nora, Floreani Annarosa, Muratori Luigi, Fagiuoli Stefano, Niro Grazia Anna, Feletti Valentina, Cozzolongo Raffaele, Terreni Natalia, Marzioni Marco, Pellicano Rinaldo, Pozzoni Pietro, Baiocchi Leonardo, Chessa Luchino, Rosina Floriano, Bertino Gaetano, Vinci Maria, Morgando Anna, Vanni Ester, Scifo Gaetano, Sacco Rodolfo, D'Antò Maria, Bellia Valentina, Boldizzoni Roberto, Casella Silvia, Omazzi Barbara, Poggi Guido, Cristoferi Laura, Gerussi Alessio, Ronca Vincenzo, Venere Rosanna, Ponziani Francesca, Cannavò Maria, Mussetto Alessandro, Fontana Rosanna, Losito Francesco, Frazzetto Evelise, Distefano Marco, Colapietro Francesca, Labanca Sara, Marconi Giulia, Grassi Giuseppe, Galati Giovanni, O'Donnell Sarah Elizabeth, Mancuso Clara, Mulinacci Giacomo, Palermo Andrea, Claar Ernesto, Izzi Antonio, Picardi Antonio, Invernizzi Pietro, Carbone Marco, Vespasiani-Gentilucci Umberto, Italian PBC Registry and the Club Epatologi Ospedalieri (CLEO)/Associazione Italiana Gastroenterologi ed Endoscopisti Digestivi Ospedalieri (AIGO) PBC Study Group
Аннотация
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.