Symptom burden and treatment response in patients with primary biliary cholangitis (PBC)

Background:

Primary biliary cholangitis (PBC) is a chronic, cholestatic liver disease that can lead to end-stage liver disease, provoking not alone physical symptoms.

Aim: This cross-sectional study evaluated treatment response and symptom burden in PBC patients with early and advanced disease in Germany.

Methods:

A total of 151 PBC patients were prospectively enrolled at the outpatient liver clinic of the University Medical Center in Mainz starting in June 2016. By chart review, historic response rates 12 months after initiation of UDCA treatment were determined using published binary models. Symptom burden was assessed using the PBC-40 questionnaire.

Results:

The prevalence of early disease with normal bilirubin, INR and albumin was 69%. Treatment response ranged between rates at 12 months ranging from 45% according to Barcelona up to 88% according to Paris-I-criteria. Importantly, this rate was maintained over a median time of 9 years. The symptom burden was high across all scored categories with the domains fatigue and emotional (2.4 ± 1; 2.3 ± 1.1 of 5) exhibiting the highest and the domain pruritus (1.5 ± 0.9 of 5) and cognition (1.9 ± 0.9 of 5) the lowest scores. The markers of cholestatic liver injury ALP and gGT correlated with the PBC-40 domain social (r =-0.270, p = 0.004; r = 0.194, p = 0.04).

Conclusion:

The mean response rate to UDCA treatment in Germany at one year after treatment initiation and over a median time of 9 years is ˜72%. The PBC-related symptom burden is high and non-response correlates with social concerns highlighting that symptoms in patients with PBC can be indicative of inadequate response.