TY - JOUR
T1 - Effects of Age and Sex of Response to Ursodeoxycholic Acid and Transplant-free Survival in Patients With Primary Biliary Cholangitis
AU - Global PBC Study Group
AU - Cheung, Angela C.
AU - Lammers, Willem J.
AU - Murillo Perez, Carla F.
AU - van Buuren, Henk R.
AU - Gulamhusein, Aliya
AU - Trivedi, Palak J.
AU - Lazaridis, Konstantinos N.
AU - Ponsioen, Cyriel Y.
AU - Floreani, Annarosa
AU - Hirschfield, Gideon M.
AU - Corpechot, Christophe
AU - Mayo, Marlyn J.
AU - Invernizzi, Pietro
AU - Battezzati, Pier Maria
AU - Parés, Albert
AU - Nevens, Frederik
AU - Thorburn, Douglas
AU - Mason, Andrew L.
AU - Carbone, Marco
AU - Kowdley, Kris V.
AU - Bruns, Tony
AU - Dalekos, George N.
AU - Gatselis, Nikolaos K.
AU - Verhelst, Xavier
AU - Lindor, Keith D.
AU - Lleo, Ana
AU - Poupon, Raoul
AU - Janssen, Harry L.A.
AU - Hansen, Bettina E.
N1 - Funding Information:
This study was performed on behalf of the Global PBC Study Group and was supported by Intercept Pharmaceuticals and the Foundation for Liver and Gastrointestinal Research in Rotterdam, The Netherlands (a not-for-profit organization).
Funding Information:
This study was performed on behalf of the Global PBC Study Group and was supported by Intercept Pharmaceuticals and the Foundation for Liver and Gastrointestinal Research in Rotterdam, The Netherlands (a not-for-profit organization).
Publisher Copyright:
© 2019 AGA Institute
PY - 2019/9
Y1 - 2019/9
N2 - Background & Aims: Primary biliary cholangitis (PBC) predominantly affects middle-aged women; there are few data on disease phenotypes and outcomes of PBC in men and younger patients. We investigated whether differences in sex and/or age at the start of ursodeoxycholic acid (UDCA) treatment are associated with response to therapy, based on biochemical markers, or differences in transplant-free survival. Methods: We performed a longitudinal retrospective study of 4355 adults in the Global PBC Study cohort, collected from 17 centers across Europe and North America. Patients received a diagnosis of PBC from 1961 through 2014. We evaluated the effects of sex and age on response to UDCA treatment (based on GLOBE score) and transplant-free survival using logistic regression and Cox regression analyses, respectively. Results: Male patients were older at the start of treatment (58.3±12.1 years vs 54.3±11.6 years for women; P<.0001) and had higher levels of bilirubin and lower circulating platelet counts (P<.0001). Younger patients (45 years or younger) had increased serum levels of transaminases than older patients (older than 45 years). Patients older than 45 years at time of treatment initiation had increased odds of a biochemical response to UDCA therapy, based on GLOBE score, compared to younger patients. The greatest odds of response to UDCA were observed in patients older than 65 years (odds ratio compared to younger patients 45 years or younger, 5.48; 95% CI, 3.92–7.67; P<.0001). Risk of liver transplant or death (compared to a general population matched for age, sex, and birth year) decreased significantly with advancing age: hazard ratio for patients 35 years or younger, 14.59 (95% CI, 9.66–22.02) vs hazard ratio for patients older than 65 years, 1.39 (95% CI, 1.23–1.57) (P<.0001). On multivariable analysis, sex was not independently associated with response or transplant-free survival. Conclusion: In longitudinal analysis of 4355 adults in the Global PBC Study, we associated patient age, but not sex, with response to UDCA treatment and transplant-free survival. Younger age at time of treatment initiation is associated with increased risk of treatment failure, liver transplant, and death.
AB - Background & Aims: Primary biliary cholangitis (PBC) predominantly affects middle-aged women; there are few data on disease phenotypes and outcomes of PBC in men and younger patients. We investigated whether differences in sex and/or age at the start of ursodeoxycholic acid (UDCA) treatment are associated with response to therapy, based on biochemical markers, or differences in transplant-free survival. Methods: We performed a longitudinal retrospective study of 4355 adults in the Global PBC Study cohort, collected from 17 centers across Europe and North America. Patients received a diagnosis of PBC from 1961 through 2014. We evaluated the effects of sex and age on response to UDCA treatment (based on GLOBE score) and transplant-free survival using logistic regression and Cox regression analyses, respectively. Results: Male patients were older at the start of treatment (58.3±12.1 years vs 54.3±11.6 years for women; P<.0001) and had higher levels of bilirubin and lower circulating platelet counts (P<.0001). Younger patients (45 years or younger) had increased serum levels of transaminases than older patients (older than 45 years). Patients older than 45 years at time of treatment initiation had increased odds of a biochemical response to UDCA therapy, based on GLOBE score, compared to younger patients. The greatest odds of response to UDCA were observed in patients older than 65 years (odds ratio compared to younger patients 45 years or younger, 5.48; 95% CI, 3.92–7.67; P<.0001). Risk of liver transplant or death (compared to a general population matched for age, sex, and birth year) decreased significantly with advancing age: hazard ratio for patients 35 years or younger, 14.59 (95% CI, 9.66–22.02) vs hazard ratio for patients older than 65 years, 1.39 (95% CI, 1.23–1.57) (P<.0001). On multivariable analysis, sex was not independently associated with response or transplant-free survival. Conclusion: In longitudinal analysis of 4355 adults in the Global PBC Study, we associated patient age, but not sex, with response to UDCA treatment and transplant-free survival. Younger age at time of treatment initiation is associated with increased risk of treatment failure, liver transplant, and death.
KW - Cholestatic Liver Disease
KW - Mortality
KW - Risk Stratification
KW - Stratified Medicine
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U2 - 10.1016/j.cgh.2018.12.028
DO - 10.1016/j.cgh.2018.12.028
M3 - Article
C2 - 30616022
AN - SCOPUS:85065726746
SN - 1542-3565
VL - 17
SP - 2076-2084.e2
JO - Clinical Gastroenterology and Hepatology
JF - Clinical Gastroenterology and Hepatology
IS - 10
ER -