Real-World Management of Patients with Primary Biliary Cholangitis-A Retrospective Study from a Tertiary Medical Center in Israel.

Eyal Yehezkel,Ehud Zigmond,Inbal Houri,Oren Shibolet,Moshe Leshno,Inbal Israel

doi:10.3390/jcm10194551

Eyal Yehezkel, Ehud Zigmond + Show 4 more

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https://doi.org/10.3390/jcm10194551

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Abstract

Background: Primary biliary cholangitis (PBC) is a rare autoimmune liver disease with variation in prevalence, phenotype and prognosis across different geographical regions. Little is known about PBC in Israel. Our aim was to characterize the demography, clinical presentation, treatment patterns and prognosis in a cohort of PBC patients followed in a referral center in central Israel. Methods: Clinical, demographic and laboratory data were collected from the medical records of PBC patients followed at Tel Aviv Medical Center in the years 2003–2020. Results: We have identified 189 patients with a confirmed diagnosis of PBC; 92.6% were female and the mean age at diagnosis was 54.7 years. Thirty-nine percent were diagnosed with another autoimmune disease and 5.9% were diagnosed with a PBC-AIH (autoimmune hepatitis) variant syndrome. Ninety-six percent were treated with ursodeoxycholic acid (UDCA) at a mean dose of 13.3 mg/kg. A total of 28.1% were found with inadequate response to UDCA according to the Toronto criteria, and 53% of the UDCA non-responders were treated with bezafibrate. Younger age at diagnosis, higher baseline levels of alkaline phosphatase (ALP) and gamma-glutamyltransferase (GGT), AIH-PBC variant and positive anti-smooth muscle antibodies (ASMA) were associated with an inadequate UDCA response. In a multivariable analysis, higher ALP at diagnosis (OR = 1.92 CI 1.11–3.20 per 50-unit change, p = 0.018) and ASMA (OR = 27.6 CI 2.58–295, p = 0.006) independently predicted inadequate UDCA response. Higher alanine transaminase (ALT), ALP and GGT, lower albumin, younger age at diagnosis and pruritus conferred an increased risk for disease progression. Conclusions: Disease characteristics, treatment patterns, response to therapy and prognosis of a PBC patient cohort in a tertiary center in central Israel were revealed. The results highlight the importance of risk stratification in PBC, specifically in younger patients, those presenting with a high level of liver enzymes and in ASMA-positive patients with an assumed diagnosis of the AIH-PBC variant.

Highlights

Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease of the small intrahepatic bile ducts
A total of 215 patients with a diagnosis of PBC according to ICD-9 coding were found in a digital database search for patients who visited the outpatient liver clinic of the gastroenterology department in Tel Aviv Medical Center between January 2003 and May
Revision of patients’ medical records found 26 patients without a probable diagnosis of PBC according to EASL guidelines [11] or with an alternative diagnosis; a total of 189 patients with PBC were included for further analysis (Figure 1)

Summary

Introduction

Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease of the small intrahepatic bile ducts. PBC may progress to cirrhosis and hepatic failure, and eventually, may require liver transplantation. Ursodeoxycholic acid (UDCA) therapy was shown to improve liver-transplant-free survival and is considered the cornerstone of treatment [5,6]. UDCA was the only therapeutic option for PBC patients; several new treatment options have recently been added to the treatment armamentarium as adjuncts to UDCA, in patients with inadequate response. The availability of these second line drugs, obeticholic acid and fibrates, varies greatly between different geographical regions

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