Title: Mo1004 Predictors and Durability of Biochemical Response to Ursodeoxycholic Acid (UDCA) in Patients With Primary Biliary Cirrhosis (PBC): A Long-Term Follow-Up Study
Abstract: Background: PBC is a chronic cholestatic liver disease that can progress to cirrhosis and liver failure.Biochemical response to treatment with UDCA is associated with improved outcomes but predictors and durability of treatment response over time are unclear.Aims: We sought to assess the durability of biochemical treatment response with long-term followup of a large cohort of patients with PBC and to identify potential predictors of treatment response.Methods: We identified patients with PBC recruited into the Mayo Clinic PBC Genetic Epidemiology Registry and Biorepository and followed at the Mayo Clinic.Medical records were reviewed retrospectively for relevant clinical and biochemical variables.Biochemical profiles including alkaline phosphatase (AP) and bilirubin were recorded at diagnosis, two years after treatment initiation, and in long-term follow-up and compared between responders and non-responders.Patients were classified as treatment responders based on the Toronto Criteria (AP <1.67X ULN at two years).To assess for prediction of treatment response, patients were stratified based on pre-treatment AP using 3X ULN as the cut-off.Survival and transplantation outcomes were summarized using the Kaplan-Meier method.Differences in lab values were assessed using the Kruskal-Wallis test.Results: 334 patients were identified whose treatment response could be ascertained.The median follow-up was 14.2 years (range 0.1-38.3).The median AP at the time of treatment response was 1.1X ULN or 206.0 U/L (n=198) in treatment responders and 2.8X ULN (n=85) or 584.0 U/L in treatment non-responders (p<0.001).The median bilirubin at the time of treatment response was 0.5 mg/dL (n=144) and 0.7 mg/dL (n=59) in treatment responders, and nonresponders respectively (p<0.001).After ten years of follow-up, the median AP among treatment responders was 1.1X ULN (n=105) or 137.0 U/L and 1.7X ULN or 281.0 U/L (n=55) in non-responders (p<0.001) and the median bilirubin was 0.5 mg/dL (n=103) and 0.7 mg/dL (n=55) in treatment responders and non-responders, respectively (p<0.001).Within response groups, patients were classified by their AP prior to treatment (≥3X ULN and <3X ULN) with 39.5% (n=132) of patients having an AP ≥3X ULN at presentation.Patients presenting with an AP ≥3X ULN were less likely to respond to treatment than those presenting with an AP <3X ULN (25.5% vs. 74.5%,p<0.001).There was no significant difference in outcomes including survival or liver transplantation among those who presented with a pre-treatment AP ≥3 or <3X ULN regardless of response to therapy.Conclusions: Initial biochemical response to UDCA persists in long-term follow-up.AP levels >3X ULN at diagnosis could predict response to therapy and identify a cohort of patients in need of additional treatment strategies.
Publication Year: 2015
Publication Date: 2015-04-01
Language: en
Type: article
Indexed In: ['crossref']
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