1014 The Majority of Patients With Primary Biliary Cholangitits Do Not Achieve the Ideal Treatment Response in a Real World Clinical Practice

Abstract

INTRODUCTION: The currently accepted criteria to define therapeutic response to primary biliary cholangitis (PBC) consists of alkaline phosphatase (ALP) (<180 U/L) and bilirubin (<1.0 mg/dL). New data suggests that normalization of ALP (<120 U/L) and bilirubin (<0.5 mg/dL) is associated with better overall outcomes. PBC patients are usually maintained on first line therapy of ursodeoxycholic acid (UDCA), obeticholic acid (OCA) or combination of both agents. The aim of the study was to determine the percentage of PBC patients that achieve the accepted (ALP < 180, bilirubin < 1) and ideal (ALP < 120, bilirubin < 0.5) treatment responses in a real-world setting. METHODS: All adult patients in this multi-center study had a diagnosis of PBC via ICD-9 and ICD-10 codes. Other chronic liver disease cases and those with significant alcohol consumption were excluded. Baseline demographics, clinical characteristics and laboratory data were collected. Therapy starting time was noted, assessing their lab values at baseline compared to a recent workup. A two-sided t test was done to compare continuous variables and a P value < 0.05 was considered statistically significant. RESULTS: 76 adult PBC patients were included in the final analysis. The mean age at the last follow up was 61.8 years (±11.3). 96% were female, 47% were White and 51% were of Hispanic ethnicity. 93% were on UDCA therapy alone with daily dosages between 750-1500 mg. Only 7% received both UDCA and OCA. The mean baseline ALP was 286 U/L and the mean follow up ALP was 172 U/L (±84.6), 52 U/L above the ideal value (P < 0.05). The mean baseline bilirubin was 1.04 mg/dL and the mean follow up bilirubin was 0.8 mg/dL (±1.2), 0.3 mg/dL above the ideal value (P < 0.05). 62% reached the accepted ALP value, 86% reached the accepted bilirubin value, and 57% reached the accepted level. 30% of patients achieved the ideal ALP value and 26% achieved the ideal bilirubin level with 20% reaching the ideal response. The rates for accepted and ideal responses were 80% and 40%, respectively, for those on UDCA and OCA. CONCLUSION: The majority of PBC patients in real-life settings did not achieve the ideal treatment response of ALP < 120 U/L and bilirubin < 0.5 mg/dL. Furthermore, a significant percentage did not achieve the accepted treatment response of ALP < 180 U/L and bilirubin < 1.0 mg/dL. Only a small percentage received combination therapy with UDCA/OCA. There's a need to increase awareness among healthcare providers on the optimal responses to PBC treatment.