Symptom burden and treatment response in patients with primary biliary cholangitis (PBC) – real life data form Germany

Background:

Primary biliary cholangitis (PBC) is a chronic, cholestatic liver disease that can lead to end-stage liver disease. Beside physical symptoms, PBC can impact on the social live and creates psychological distress promoting a significant burden of disease [P. Blackburn et. al., Liver Int. 2007]. The management of PBC includes bile acid based therapeutic regimes, primarily with ursodeoxycholic acid (UDAC). Only limited data about the response rates in relation to the quality of life in patients with PBC in Germany is known.

Aim:

The aim of the study was to evaluate the biochemical response rates in PBC and PBC/AIH overlap patients scored by acknowledged binary models (Paris I/IIb, Rotterdam and Barcelona criteria) [G. Hierschfield, J. Hep. 2017]. Further, biochemical markers were correlated with the six domains of the PBC-40 score which relate to fatigue, emotional, social, and cognitive function, general symptoms, and itch.

Results and methods:

A total of 151 patients, 140 PBC (92%) and 11 (7%) PBC/AIH overlap patients, were prospectively studied at the outpatient clinic of the University Medical Center in Mainz. One year after UDAC treatment was initiated, biochemical serum markers were assessed and treatment response was scored by the acknowledged binary models. According to the Paris-I criteria, exhibiting the best prediction for liver transplantation or death among the tested binary models, 137 (91%) patients responded to the treatment. As assessed by the PBC-40 survey, patients primarily suffered from fatigue (26/100 points) followed by social impairments (14/100 points), while pruritus received the lowest score (5/100 points). Biochemical serum markers, serum proteins, blood cell counts and lipids were correlated with the single domains of the PBC-40 score. Here, IgG, as a marker of the disease activity, significantly correlated with the emotional and social burden of the patients as assessed by PBC-40 (p < 0.05).

Conclusion:

UDAC treatment is an effective treatment for PBC, as proven by a high response rate of up to 91%. The disease's activity correlates significantly with the psychological distress of the patients. PBC and PBC/AIH-overlap patients suffer above all from fatigue, while pruritus is less of a burden.