Predictors of serious adverse events and non-response in cirrhotic patients with primary biliary cholangitis treated with obeticholic acid.


Journal

Liver international : official journal of the International Association for the Study of the Liver
ISSN: 1478-3231
Titre abrégé: Liver Int
Pays: United States
ID NLM: 101160857

Informations de publication

Date de publication:
11 2022
Historique:
revised: 24 05 2022
received: 17 02 2022
accepted: 23 06 2022
pubmed: 7 8 2022
medline: 28 9 2022
entrez: 6 8 2022
Statut: ppublish

Résumé

Obeticholic acid (OCA) has recently been restricted in patients with primary biliary cholangitis (PBC) with "advanced cirrhosis" because of its narrow therapeutic index. We aimed to better define the predicting factors of hepatic serious adverse events (SAEs) and non-response in cirrhotic patients undergoing OCA therapy. Safety and efficacy of treatment were evaluated in a cohort of consecutive PBC cirrhotic patients started with OCA. OCA response was evaluated according to the Poise criteria. Risk factors for hepatic SAEs and non-response were reported as risk ratios (RR) with 95% confidence intervals (CIs). One hundred PBC cirrhotics were included, 97 Child-Pugh class A and 3 class B. Thirty-one had oesophageal varices and 5 had a history of ascites. Thirty-three per cent and 32% of patients achieved a biochemical response at 6 and 12 months respectively. Male sex (adjusted-RR 1.75, 95%CI 1.42-2.12), INR (1.37, 1.00-1.87), Child-Pugh score (1.79, 1.28-2.50), MELD (1.17, 1.04-1.30) and bilirubin (1.83, 1.11-3.01) were independently associated with non-response to OCA. Twenty-two patients discontinued OCA within 12 months: 10 for pruritus, 9 for hepatic SAEs (5 for jaundice and/or ascitic decompensation; 4 for upper digestive bleeding). INR (adjusted-RR 1.91, 95%CI 1.10-3.36), lower albumin levels (0.18, 0.06-0.51), Child-Pugh score (2.43, 1.50-4.04), history of ascites (3.5, 1.85-6.5) and bilirubin (1.30, 1.05-1.56), were associated with hepatic SAEs. A total bilirubin≥1.4 mg/dl at baseline was the most accurate biochemical predictor of hepatic SAEs under OCA. An accurate baseline assessment is crucial to select cirrhotic patients who can benefit from OCA. Although OCA is effective in one third of cirrhotics, bilirubin level ≥1.4 mg/dl should discourage from its use.

Sections du résumé

BACKGROUND & AIMS
Obeticholic acid (OCA) has recently been restricted in patients with primary biliary cholangitis (PBC) with "advanced cirrhosis" because of its narrow therapeutic index. We aimed to better define the predicting factors of hepatic serious adverse events (SAEs) and non-response in cirrhotic patients undergoing OCA therapy.
METHODS
Safety and efficacy of treatment were evaluated in a cohort of consecutive PBC cirrhotic patients started with OCA. OCA response was evaluated according to the Poise criteria. Risk factors for hepatic SAEs and non-response were reported as risk ratios (RR) with 95% confidence intervals (CIs).
RESULTS
One hundred PBC cirrhotics were included, 97 Child-Pugh class A and 3 class B. Thirty-one had oesophageal varices and 5 had a history of ascites. Thirty-three per cent and 32% of patients achieved a biochemical response at 6 and 12 months respectively. Male sex (adjusted-RR 1.75, 95%CI 1.42-2.12), INR (1.37, 1.00-1.87), Child-Pugh score (1.79, 1.28-2.50), MELD (1.17, 1.04-1.30) and bilirubin (1.83, 1.11-3.01) were independently associated with non-response to OCA. Twenty-two patients discontinued OCA within 12 months: 10 for pruritus, 9 for hepatic SAEs (5 for jaundice and/or ascitic decompensation; 4 for upper digestive bleeding). INR (adjusted-RR 1.91, 95%CI 1.10-3.36), lower albumin levels (0.18, 0.06-0.51), Child-Pugh score (2.43, 1.50-4.04), history of ascites (3.5, 1.85-6.5) and bilirubin (1.30, 1.05-1.56), were associated with hepatic SAEs. A total bilirubin≥1.4 mg/dl at baseline was the most accurate biochemical predictor of hepatic SAEs under OCA.
CONCLUSIONS
An accurate baseline assessment is crucial to select cirrhotic patients who can benefit from OCA. Although OCA is effective in one third of cirrhotics, bilirubin level ≥1.4 mg/dl should discourage from its use.

Identifiants

pubmed: 35932095
doi: 10.1111/liv.15386
pmc: PMC9804305
doi:

Substances chimiques

Albumins 0
obeticholic acid 0462Z4S4OZ
Chenodeoxycholic Acid 0GEI24LG0J
Bilirubin RFM9X3LJ49

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

2453-2465

Investigateurs

Valentina Feletti (V)
Alessandro Mussetto (A)
Rosanna Venere (R)
Giulia Bernaccioni (G)
Marie Graciella Pigozzi (M)
Stefano Fagiuoli (S)
Natalia Terreni (N)
Pietro Pozzoni (P)
Leonardo Baiocchi (L)
Giuseppe Grassi (G)
Maria Vinci (M)
Valentina Bellia (V)
Roberto Boldizzoni (R)
Silvia Casella (S)
Barbara Omazzi (B)
Guido Poggi (G)

Commentaires et corrections

Type : CommentIn

Informations de copyright

© 2022 The Authors. Liver International published by John Wiley & Sons Ltd.

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Auteurs

Antonio De Vincentis (A)

Internal Medicine and Hepatology, University Campus Bio-Medico of Rome, Rome, Italy.

Daphne D'Amato (D)

Gastroenterology Unit, Città della salute e della scienza, Turin, Italy.

Laura Cristoferi (L)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Alessio Gerussi (A)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Federica Malinverno (F)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Ana Lleo (A)

Internal Medicine and Hepatology, Humanitas Clinical and Research Center IRCCS, Humanitas University, Milan, Italy.

Francesca Colapietro (F)

Internal Medicine and Hepatology, Humanitas Clinical and Research Center IRCCS, Humanitas University, Milan, Italy.

Fabio Marra (F)

Internal Medicine and Hepatology Unit, Department of Experimental and Clinical Medicine, University of Firenze, Firenze, Italy.

Andrea Galli (A)

Department of Experimental and Clinical Biochemical Sciences, University of Florence, Florence, Italy.

Cecilia Fiorini (C)

Department of Experimental and Clinical Biochemical Sciences, University of Florence, Florence, Italy.

Barbara Coco (B)

Hepatology Unit, University Hospital of Pisa, Pisa, Italy.

Maurizia Brunetto (M)

Hepatology Unit, University Hospital of Pisa, Pisa, Italy.

Grazia Anna Niro (GA)

Gastroenterology Unit, Fondazione Casa Sollievo Della Sofferenza IRCCS, San Giovanni Rotondo, Italy.

Rosa Cotugno (R)

Gastroenterology Unit, Fondazione Casa Sollievo Della Sofferenza IRCCS, San Giovanni Rotondo, Italy.

Carlo Saitta (C)

Division of Medicine and Hepatology, University Hospital of Messina "Policlinico G. Martino", Messina, Italy.

Raffaele Cozzolongo (R)

Gastroenterology Unit, National Institute of Gastroenterology "S de Bellis" Research Hospital, Castellana Grotte, Italy.

Francesco Losito (F)

Gastroenterology Unit, National Institute of Gastroenterology "S de Bellis" Research Hospital, Castellana Grotte, Italy.

Edoardo Giovanni Giannini (EG)

Gastroenterology Unit, Department of Internal Medicine, University of Genova, IRCCS Ospedale Policlinico San Martino, Genova, Italy.

Sara Labanca (S)

Gastroenterology Unit, Department of Internal Medicine, University of Genova, IRCCS Ospedale Policlinico San Martino, Genova, Italy.

Marco Marzioni (M)

Clinic of Gastroenterology and Hepatology, Università Politecnica delle Marche, Ancona, Italy.

Giulia Marconi (G)

Clinic of Gastroenterology and Hepatology, Università Politecnica delle Marche, Ancona, Italy.

Anna Morgando (A)

Gastroenterology Unit, Città della salute e della scienza, Turin, Italy.

Rinaldo Pellicano (R)

Gastroenterology Unit, Città della salute e della scienza, Turin, Italy.

Ester Vanni (E)

Gastroenterology Unit, Città della salute e della scienza, Turin, Italy.

Nora Cazzagon (N)

Gastroenterology Unit, Department of Surgery, Oncology and Gastroenterology, Padua University Hospital, Padua, Italy.

Annarosa Floreani (A)

Gastroenterology Unit, Department of Surgery, Oncology and Gastroenterology, Padua University Hospital, Padua, Italy.

Luchino Chessa (L)

Liver Unit, University Hospital of Cagliari, Cagliari, Italy.

Olivia Morelli (O)

Clinic of Gastroenterology and Hepatology, Department of Medicine, Università degli Studi di Perugia, Perugia, Italy.

Luigi Muratori (L)

DIMEC Università di Bologna, Policlinico di Sant'Orsola, Bologna, Italy.

Adriano Pellicelli (A)

Liver Unit, San Camillo Hospital, Rome, Italy.

Maurizio Pompili (M)

Internal Medicine and Hepatology Unit, Policlinico Gemelli, Sapienza University, Rome, Italy.

Francesca Ponziani (F)

Internal Medicine and Hepatology Unit, Policlinico Gemelli, Sapienza University, Rome, Italy.

Annalisa Tortora (A)

Internal Medicine and Hepatology Unit, Policlinico Gemelli, Sapienza University, Rome, Italy.

Floriano Rosina (F)

Medical Team Torino, Torino, Italy.

Maurizio Russello (M)

Liver Unit, Arnas Garibaldi, Catania, Italy.

Mariarita Cannavò (M)

Liver Unit, Arnas Garibaldi, Catania, Italy.

Loredana Simone (L)

Department of Gastroenterology, University Hospital Sant'Anna, Ferrara, Italy.

Silvia Storato (S)

IRCCS Sacro Cuore Institute Don Calabria, Gastroenterology, Negrar, Italy.

Mauro Viganò (M)

Hepatology Unit, San Giuseppe Hospital, Milan, Italy.

Ludovico Abenavoli (L)

Department of Health Sciences, University "Magna Graecia" of Catanzaro, Italy.

Maria D'Antò (M)

Hepatology Unit, Santa Maria delle Grazie Hospital, Pozzuoli, Italy.

Elisabetta De Gasperi (E)

Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico - Division of Gastroenterology and Hepatology - CRC "A.M. and A. Migliavacca" Center for Liver Disease, Milan, Italy.

Marco Distefano (M)

Department of Infectious Diseases, Umberto I Hospital, Syracuse, Italy.

Gaetano Scifo (G)

Department of Infectious Diseases, Umberto I Hospital, Syracuse, Italy.

Teresa Zolfino (T)

Department of Gastroenterology, Brotzu Hospital, Cagliari, Italy.

Vincenza Calvaruso (V)

Gastroenterology and Hepatology Unit, University of Palermo, Palermo, Italy.

Giuseppe Cuccorese (G)

Internal Medicine Ospedale "R. Dimiccoli", Barletta, Italy.

Valeria Pace Palitti (VP)

Hepatology Unit, Santo Spirito Hospital, Pescara, Italy.

Rodolfo Sacco (R)

Gastroenterology Unit, Ospedali Riuniti, Foggia, Italy.

Gaetano Bertino (G)

Gastroenterology and Hepatology Unit, University Hospital Policlinico Vittorio Emanuele, Catania, Italy.

Evelise Frazzetto (E)

Gastroenterology and Hepatology Unit, University Hospital Policlinico Vittorio Emanuele, Catania, Italy.

Domenico Alvaro (D)

Department of Translational and Precision Medicine, University La Sapienza, Rome, Italy.

Giacomo Mulinacci (G)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Andrea Palermo (A)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Miki Scaravaglio (M)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Francesca Terracciani (F)

Internal Medicine and Hepatology, University Campus Bio-Medico of Rome, Rome, Italy.

Giovanni Galati (G)

Internal Medicine and Hepatology, University Campus Bio-Medico of Rome, Rome, Italy.

Vincenzo Ronca (V)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Massimo Zuin (M)

Liver and Gastroenterology Unit, Department of Health Sciences, Universita' degli Studi di Milano, Milan, Italy.
ASST Santi Paolo e Carlo, University Hospital San Paolo, Milan, Italy.

Ernesto Claar (E)

Department of Hepatology, Betania Hospital, Napoli, Italy.

Antonio Izzi (A)

Department of Infectious Diseases, D. Cotugno Hospital, Napoli, Italy.

Antonio Picardi (A)

Internal Medicine and Hepatology, University Campus Bio-Medico of Rome, Rome, Italy.

Pietro Invernizzi (P)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

Umberto Vespasiani-Gentilucci (U)

Internal Medicine and Hepatology, University Campus Bio-Medico of Rome, Rome, Italy.

Marco Carbone (M)

Division of Gastroenterology, Centre for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, European Reference Network on Hepatological Diseases (ERN RARE-LIVER), San Gerardo Hospital, Monza, Italy.

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