Z Gastroenterol 2020; 58(08): e162
DOI: 10.1055/s-0040-1716167
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Implementation of a national registry for patients with primary biliary cholangitis (PBC): the German PBC cohort

J Wiegand
1   Universitätsklinikum Leipzig, Hepatologie, Leipzig, Deutschland
,
R Günther
2   Universitätsklinikum Schleswig Holstein, Kiel, Deutschland
,
U Naumann
3   UBN Praxis, Berlin, Deutschland
,
U Stölzel
4   Klinikum Chemnitz, Chemnitz, Deutschland
,
C Berg
5   Universitätsklinikum Tübingen, Tübingen, Deutschland
,
H Bantel
6   Medizinische Hochschule Hannover, Hannover, Deutschland
,
K Stein
7   Praxis Hepatologie Magdeburg, Magdeburg, Deutschland
,
H Heinzow
8   Universitätsklinikum Münster, Münster, Deutschland
,
Kremer AE
9   Universitätsklinikum Erlangen, Erlangen, Deutschland
,
Hofmann WP
10   Praxis Gastroenterologie am Bayerischen Platz, Berlin, Deutschland
,
A Kautz
11   Deutsche Lebehilfe, Köln, Deutschland
,
U Beuers
12   Academic Medical Center, University of Amsterdam, Amsterdam, Niederlande
,
C Trautwein
13   Universitätsklinikum Aachen, Aachen, Deutschland
,
A Franke
14   Zentrum für Klinische Studien Leipzig, Leipzig, Deutschland
,
K Piotrowski
14   Zentrum für Klinische Studien Leipzig, Leipzig, Deutschland
,
T Berg
1   Universitätsklinikum Leipzig, Hepatologie, Leipzig, Deutschland
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Background Primary biliary cholangitis (PBC) is characterized by inflammation and destruction of bile ducts which may progress to biliary cirrhosis. Due to low prevalence and slow disease progression it is difficult to investigate the prognosis of subgroups or to evaluate the effectiveness of therapeutic interventions on clinical outcomes. National registry data for Germany do not exist so far. Thus, the German PBC Cohort was implemented as a non-interventional study (NIS)

Methods A confirmed PBC diagnosis according to EASL guidelines (at least two of three criteria positive: elevated alkaline phosphatase (AP), AMA-M2 positivity, PBC compatible liver biopsy) is requested and treatment with at least one licensed PBC medication. Subgroups are classified according to their response to therapy with ursodeoxychocholic acid (UDCA) as responders or primary or secondary incomplete responders (Paris II criteria). Newly diagnosed patients were diagnosed within the last six months prior to inclusion.

Results Recruitment started in September 2019. As of 12 March 2020, 13 initiated of 37 participating centers (n=7 academic, n=2 non-academic hospitals, n=4 outpatient practices) recruited 72 patients (90.3% female, mean (SD) age 59.8 (11.3) years, BMI 27.4 (5.4), 9% (N=6) with cirrhosis). PBC diagnosis was confirmed by elevated AP and AMA-M2 positivity in 83.3% of cases, 45.8% underwent biopsy, 41.7% fulfilled all three diagnostic criteria.

PBC patients were classified as responders (N=40), primary (N=10) or secondary (N=13) incomplete responders, respectively. Median disease duration was 7 [4;10] vs. 7 [2;9] vs. 6 [4;14] years, respectively. 2 of the 6 cirrhotic patients were in the incomplete responder groups. 9 patients were newly diagnosed.

All patients were treated with UDCA (median daily dosage 12.8 [10.9; 14.8] mg/kg), 11/68 received obeticholic acid (OCA), 1/68 bezafibrate, and 1/68 budesonide. OCA was used in 10/20 incomplete responders.

Chronic fatigue was reported in 11.8% of patients, itching of mild (45%) or moderate (55%) severity in 29.4% of cases.

Conclusion The German PBC Cohort has been successfully established as a prospective nationwide registry. Current recruitment and clinical data will be presented at the meeting.



Publication History

Article published online:
08 September 2020

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