1. Prediction and evaluation of high-risk patients with primary biliary cholangitis receiving ursodeoxycholic acid therapy: an early criterion
- Author
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Yang, Chunmei, Guo, Guanya, Li, Bo, Zheng, Linhua, Sun, Ruiqing, Wang, Xiufang, Deng, Juan, Jia, Gui, Zhou, Xia, Cui, Lina, Guo, Changcun, Zhou, Xinmin, Leung, Patrick SC, Gershwin, M Eric, Shang, Yulong, and Han, Ying
- Subjects
Biomedical and Clinical Sciences ,Clinical Sciences ,Liver Disease ,Clinical Research ,Digestive Diseases ,Chronic Liver Disease and Cirrhosis ,Oral and gastrointestinal ,Humans ,Ursodeoxycholic Acid ,Liver Cirrhosis ,Biliary ,Cholagogues and Choleretics ,Treatment Outcome ,Aspartate Aminotransferases ,Autoimmune liver disease ,Primary biliary cholangitis ,Adverse outcome ,Stratified therapy ,Early prediction ,Therapeutics ,Prognosis ,Biochemical response ,Complication ,Retrospective cohort study ,Gastroenterology & Hepatology ,Clinical sciences - Abstract
Background and aimsCurrent treatment guidelines recommend ursodeoxycholic acid (UDCA) as the first-line treatment for new-diagnosed primary biliary cholangitis (PBC) patients. However, up to 40% patients are insensitive to UDCA monotherapy, and evaluation of UDCA response at 12 months may result in long period of ineffective treatment. We aimed to develop a new criterion to reliably identify non-response patients much earlier.MethodsFive hundred sixty-nine patients with an average of 59 months (Median: 53; IQR:32-79) follow-up periods were randomly divided into either the training (70%) or the validation cohort (30%). The efficiency of different combinations of total bilirubin (TBIL), alkaline phosphatase (ALP), and aspartate aminotransferase (AST) threshold values to predict outcomes was assessed at 1, 3 or 6 month after the initiation of UDCA therapy. The endpoints were defined as adverse outcomes, including liver-related death, liver transplantation and complications of cirrhosis. Adverse outcome-free survival was compared using various published criteria and a proposed new criterion.ResultsA new criterion of evaluating UDCA responses at 1 month was established as: ALP ≤ 2.5 × upper limit of normal (ULN) and AST ≤ 2 × ULN, and TBIL ≤ 1 × ULN (Xi'an criterion). The 5 year adverse outcome-free survival rate of UDCA responders, defined by Xi'an criterion, was 97%, which was significantly higher than that of those non-responders (64%). An accurate distinguishing high-risk patients' capacity of Xi'an criterion was confirmed in both early and late-stage PBC.ConclusionsXi'an criterion has a similar or even higher ability to distinguish high-risk PBC patients than other published criteria. Xi'an criterion can facilitate early identification of patients requiring new therapeutic approaches.
- Published
- 2023