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

被引：17

作者：

Kaps, Leonard ^{[1
,2
]}

Grambihler, Annette ^{[1
,2
]}

Yemane, Bethlehem ^{[2
]}

Nagel, Michael ^{[1
,2
]}

Labenz, Christian ^{[1
,2
]}

Ploch, Pascal ^{[1
,2
]}

Michel, Maurice ^{[1
,2
]}

Galle, Peter R. ^{[1
]}

Woerns, Marcus-Alexander ^{[1
]}

Schattenberg, Joern M. ^{[1
,2
]}

机构：

[1] Johannes Gutenberg Univ Mainz, Univ Med Ctr, Dept Med 1, Langenbeckstr 1, D-55131 Mainz, Germany

[2] Johannes Gutenberg Univ Mainz, Univ Med Ctr, Metab Liver Res Program, Mainz, Germany

来源：

DIGESTIVE DISEASES AND SCIENCES | 2020年 / 65卷 / 10期

关键词：

PBC; UDCA; PBC-40; Pruritus; Symptom burden; Treatment response; PLACEBO-CONTROLLED TRIAL; QUALITY-OF-LIFE; URSODEOXYCHOLIC ACID; BIOCHEMICAL RESPONSE; CIRRHOSIS; PROGNOSIS; SURVIVAL; FATIGUE; COHORT; IMPACT;

D O I：

10.1007/s10620-019-06009-3

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

Background 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. Aim This cross-sectional study evaluated treatment response and symptom burden in patients with PBC in Germany to expand the available data. Methods 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. Results 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 5 years 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). Conclusion In this tertiary care cohort, 75% of the patients showed biochemical response after 1 year according to the acknowledged Paris II criteria. Patients reported a significant symptom burden, and the domain fatigue of the PBC-40 was most prominently impaired.

引用

页码：3006 / 3013

页数：8

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