Symptom Burden and Treatment Response in Patients with Primary Biliary Cholangitis (PBC).

Abstract

Primary biliary cholangitis (PBC) is a chronic, cholestatic liver disease that can lead to end-stage liver disease and impairs the quality of life. At current, most data come from few large cohorts. This cross-sectional study evaluated treatment response and symptom burden in patients with PBC in Germany to expand the available data. A total of 140 PBC patients were prospectively enrolled at the outpatient liver clinic of the University Medical Center in Mainz starting in June 2016. Historic and current response rates of UDCA treatment were determined using published binary models. Symptom burden was assessed using the PBC-40 questionnaire. The primary treatment response ranged between 73 and 86% depending on the definition used. Importantly, this response rate was maintained over a median time of 5years in follow-up. The highest symptom burden was observed for fatigue and emotional (2.4 ± 1; 2.3 ± 1.1 of 5), while pruritus (1.1 ± 1.1 of 5) had the lowest scores. IgG correlated with the PBC-40 domain social (r = 0.211, p = 0.032), while HDL inversely correlated with the symptom burden of pruritus (r = - 0.236; p = 0.018). In this tertiary care cohort, 75% of the patients showed biochemical response after 1year according to the acknowledged Paris II criteria. Patients reported a significant symptom burden, and the domain fatigueof the PBC-40 was most prominently impaired.