TY - JOUR
T1 - Clinical characterization of patients with primary biliary cholangitis
T2 - A report from multiple Canadian centres
AU - Yoshida, Eric M.
AU - Swain, Mark Gordon
AU - Tsien, Cynthia
AU - Tam, Edward
AU - Bailey, Robert James
AU - Grbic, Dusanka
AU - Ko, Hin Hin
AU - Ramji, Alnoor
AU - Hilzenrat, Nir
AU - Elkhashab, Magdy
AU - Kim, Euiseok
AU - O’brien, Meaghan
AU - Puglia, Marco Amedeo
AU - Peltekian, Kevork M.
N1 - Funding Information:
Intercept Pharma Canada Inc. funded the medical writing services and statistical analysis to assist in the development of this manuscript.
Funding Information:
DISCLOSURES: EM Yoshida has received an unrestricted research grant from Paladin Laboarto-ries and clinical trials sponsorship from Gilead Sciences, Intercept, Abbvie, Merck, Genfit, Pfizer, Celgene, Allergan, and Madrigal. He has also received honoraria for CME/Ad Board lectures from Intercept, Gilead, and Merck. He is President of the Vancouver Medical Dental Allied Staff Association; C Tsien received grants/contracts from Lupin, and payment or honoraria for lectures, presentations, speaker bureaus, or educational events from Ab-bvie and Intercept; MG Swain received grants/ contracts for research studies from Gilead, Intercept, CymaBay, Genkyotex, GSK, Genfit, Novartis, Pfizer, Celgene, AstraZeneca, AbbVie, and Novo Nordisk, payment or honoraria for lectures and presentations from Gilead, Intercept, and AbbVie, and participated on the advisory boards for Gil-ead, Novartis, and Intercept; KM Peltekian participated on the advisory board for Intercept and has held a leadership or fiduciary role on the Canadian Liver Foundation board; MA Puglia has received payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing, or educational events from Intercept as well as support from them for attending meetings and/or travel and participating on their advisory board; M Elkhashab has received consulting fees and payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing, or educational events from AbbVie, Gilead, and Merck, as well as participated on the advisory boards for AbbVie, Gilead, and Merck; M O’Brien has received payment or honoraria for lectures, presentations, speaker bureaus, or educational events from Intercept; HH Ko has received payment or honoraria for lectures, presentations, speaker bureaus, or educational events from Intercept and has held a leadership or fiduciary role on the Intercept advisory board; E Tam has received consulting fees from Intercept as well as payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing, or educational events from Intercept; A Ramji has received grants/contracts from AbbVie, Allergan, Assembly, Galmed, Gilead, Janssen, Intercept, Novartis, and Merck, as well as payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing, or educational events from AbbVie, Allergan, Amgen, Celgene, Gilead, Intercept, Amgen, Janssen, No-vartis, and Merck. He has participated on advisory board for AbbVie, Gilead, Janssen, Intercept, No-vartis, Merck, and Novo Nordisk; the other authors have nothing to disclose.
Publisher Copyright:
© 2022, University of Toronto Press. All rights reserved.
PY - 2022/8/1
Y1 - 2022/8/1
N2 - BACKGROUND: Primary biliary cholangitis (PBC) is a rare, chronic autoimmune, cholestatic liver disease affecting approximately 318 per million Canadians. There is limited information regarding the characterization of this patient population in Canada. Consequently, we aim to describe a cohort of PBC patients managed across liver centres serving this type of population. METHODS: A cross-sectional examination of 1,125 PBC patient charts at 15 liver centres across Canada was conducted between January 2016 and September 2017. RESULTS: Data from 1,125 eligible patients were collected from 7 Canadian provinces. The patient population was largely female (90.2%), had a median overall age of 61.3 years, and a median overall time since diagnosis of 6.4 years. Of the patients included in the study, 89% were on ursodeoxycholic acid (UDCA) therapy at a median dose of 14.0 mg/kg/day and 4.4% were previously treated with UDCA, whereas 6.6% were never treated with UDCA. Of the patients with available data (n = 1067), 289 (27.1%) presented with alkaline phosphatase (ALP) levels ≥200 IU/L and/or total bilirubin levels ≥21 µmol/L. Assessment of UDCA treatment response revealed that 26.6% and 38.3% of patients were inadequate responders according to the Toronto and Paris-II criteria, respectively. Mortality occurred in 1.2% (14) of patients, with liver-related adverse outcomes being more commonly observed in patients who discontinued UDCA compared to those who are currently on treatment (36.3% and 19.6%, respectively). CONCLUSION: This study showed that Canadian PBC patients present with demographics and features commonly reported in the literature for this disease. Over one third of PBC patients had inadequate response to UDCA treatment or were not currently being treated with UDCA. Consequently, there is a significant unmet therapeutic need in this Canadian PBC population.
AB - BACKGROUND: Primary biliary cholangitis (PBC) is a rare, chronic autoimmune, cholestatic liver disease affecting approximately 318 per million Canadians. There is limited information regarding the characterization of this patient population in Canada. Consequently, we aim to describe a cohort of PBC patients managed across liver centres serving this type of population. METHODS: A cross-sectional examination of 1,125 PBC patient charts at 15 liver centres across Canada was conducted between January 2016 and September 2017. RESULTS: Data from 1,125 eligible patients were collected from 7 Canadian provinces. The patient population was largely female (90.2%), had a median overall age of 61.3 years, and a median overall time since diagnosis of 6.4 years. Of the patients included in the study, 89% were on ursodeoxycholic acid (UDCA) therapy at a median dose of 14.0 mg/kg/day and 4.4% were previously treated with UDCA, whereas 6.6% were never treated with UDCA. Of the patients with available data (n = 1067), 289 (27.1%) presented with alkaline phosphatase (ALP) levels ≥200 IU/L and/or total bilirubin levels ≥21 µmol/L. Assessment of UDCA treatment response revealed that 26.6% and 38.3% of patients were inadequate responders according to the Toronto and Paris-II criteria, respectively. Mortality occurred in 1.2% (14) of patients, with liver-related adverse outcomes being more commonly observed in patients who discontinued UDCA compared to those who are currently on treatment (36.3% and 19.6%, respectively). CONCLUSION: This study showed that Canadian PBC patients present with demographics and features commonly reported in the literature for this disease. Over one third of PBC patients had inadequate response to UDCA treatment or were not currently being treated with UDCA. Consequently, there is a significant unmet therapeutic need in this Canadian PBC population.
KW - Canada
KW - autoimmune liver disease
KW - characterization
KW - chart audit
KW - management
KW - national
KW - primary biliary cholangitis
KW - retrospective
KW - ursodeoxycholic acid
UR - http://www.scopus.com/inward/record.url?scp=85138323636&partnerID=8YFLogxK
U2 - 10.3138/CANLIVJ-2021-0038
DO - 10.3138/CANLIVJ-2021-0038
M3 - Article
AN - SCOPUS:85138323636
SN - 2561-4444
VL - 5
SP - 372
EP - 387
JO - Canadian Liver Journal
JF - Canadian Liver Journal
IS - 3
ER -