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LIVER
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© 2017 by the American College of Gastroenterology The American Journal of GASTROENTEROLOGY
ORIGINAL CONTRIBUTIONS
INTRODUCTION
Primary biliary cholangitis (PBC), formerly known as pri-
mary biliary cirrhosis (1), is a chronic cholestatic liver disease
that may eventually result in severe illness, leading to death or
liver transplantation (2,3). Despite the presumed autoimmune
pathogenesis the efective therapies are those basically acting on
improving cholestasis, mainly ursodeoxycholic acid (UDCA)
(4), and more recently obeticholic acid (5). Te favorable efects
with a probability of survival free of transplantation have been
reported with UDCA, but still a signifcant proportion of
patients deserve further treatments addressed to improve out-
come (4).
In the past two decades, fbrates, and particularly bezafbrate,
commonly associated with UDCA, have demonstrated favorable
efects in PBC, in terms of improving liver biochemistries with no
or minor adverse efects (6–20). However, the studies lacked a sig-
nifcant number of patients, had low doses of UDCA, or a very
short period of treatment. Moreover, a controlled study including
a small number of patients indicated that long-term bezafbrate
treatment may result in renal insufciency (21).
We have confrmed the encouraging efects of adding bezafbrate
to UDCA for 1 year in 28 patients with PBC (20). Moreover, this
report indicates that bezafbrate alleviated itching in 12 patients,
an efect that resumed afer bezafbrate discontinuation. Four cases
Effects of Bezafibrate on Outcome and Pruritus
in Primary Biliary Cholangitis With Suboptimal
Ursodeoxycholic Acid Response
Anna Reig, MD
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, Pilar Sesé, RN
1
and Albert Parés, MD, PhD
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OBJECTIVES: Adding fibrates improves liver biochemistries in patients with primary biliary cholangitis (PBC) and
suboptimal response to ursodeoxycholic acid (UDCA). As there are no consistent data regarding
the course and outcome, we have assessed the effects of the combined treatment with UDCA and
bezafibrate on a long-term basis.
METHODS: A total of 48 patients (45 female) with PBC treated with UDCA and alkaline phosphatase (ALP)
above 1.5 times upper normal levels (×UNL) were treated with bezafibrate (400 mg/day) plus UDCA
(13–16 mg/kg/day). Changes in clinical features, liver biochemistries, and prognosis after therapy
were assessed, as well as pruritus, using a visual analog scale (43 patients) and the 5-D descriptive
pruritus scale.
RESULTS: After a median of 38 months, 26 patients (54%) had normalized ALP. In these patients, jaundice,
pruritus, and liver stiffness was lower, and age was higher than in patients who remained with
elevated ALP. Biochemical improvement was less prominent in patients without ALP normalization.
Five of these patients (23%) developed events of disease progression: 1 died, 3 were transplanted,
and 1 developed hepatocellular carcinoma. Partial or complete itching relief was reported in
all but one case with pruritus. Itching recurrence or worsening was observed after bezafibrate
discontinuation.
CONCLUSIONS: The long-term treatment with UDCA and bezafibrate results in excellent response, and is associated
with a complete or partial itching relief. Incomplete ALP normalization was observed in patients with
advanced disease who remained at risk for developing severe events. The combined treatment is
mainly effective in patients with lower fibrosis and severity of cholestasis.
Am J Gastroenterol advance online publication, 10 October 2017; doi:10.1038/ajg.2017.287
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Liver Unit, Hospital Clínic, IDIBAPS, CIBERehd, University of Barcelona, Barcelona, Spain. Correspondence: Albert Parés, MD, PhD, Liver Unit, Hospital Clínic,
University of Barcelona, C/Villarroel 170, Barcelona 08036, Spain. E-mail: pares@ub.edu
Received 16 May 2017; accepted 1 August 2017