Real-world experience with obeticholic acid in patients with primary biliary cholangitis.
AIH, autoimmune hepatitis
ALP, alkaline phosphatase
ALT, alanine transferase
AMA, antimitochondrial antibodies
ANA, antinuclear antibodies
AST, aspartate transferase
Autoimmunity
CRFs, case record forms
Cholestasis
Cirrhosis
EDC, electronic data capture
GGT, gamma-glutamyl transferase
OC, Overall cohort
OCA, obeticholic acid
Overlap PBC-AIH
PBC, primary biliary cholangitis
QC, quality control
RCT, randomised controlled trial
RR, risk ratio
TCC, Treatment Completer Cohort
TIPS, transjugular intrahepatic portosystemic shunt
UDCA, ursodeoxycholic acid
ULN, upper limit of normal
aRR, adjusted risk ratio
Journal
JHEP reports : innovation in hepatology
ISSN: 2589-5559
Titre abrégé: JHEP Rep
Pays: Netherlands
ID NLM: 101761237
Informations de publication
Date de publication:
Apr 2021
Apr 2021
Historique:
received:
10
09
2020
revised:
23
11
2020
accepted:
06
01
2021
entrez:
8
3
2021
pubmed:
9
3
2021
medline:
9
3
2021
Statut:
epublish
Résumé
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. 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 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 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. 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.
Sections du résumé
BACKGROUND & AIMS
OBJECTIVE
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
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
RESULTS
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
CONCLUSIONS
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
BACKGROUND
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.
Identifiants
pubmed: 33681748
doi: 10.1016/j.jhepr.2021.100248
pii: S2589-5559(21)00024-0
pmc: PMC7930359
doi:
Types de publication
Journal Article
Langues
eng
Pagination
100248Informations de copyright
© 2021 The Author(s).
Déclaration de conflit d'intérêts
The authors have no conflicts of interest to declare related to this work. Please refer to the accompanying ICMJE disclosure forms for further details.
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