Sub-optimal therapy of patients with primary biliary cholangitis (PBC) in the real-life stetting of the German PBC cohort

Abstract

Background

Real-world data on the management of patients with primary biliary cholangitis (PBC) are so far scarce in Germany. Therefore, we aimed to establish a nationwide registry and describe the clinical characteristics and therapy of PBC patients.

Methods

Three different cohorts defined as ursodeoxycholic acid (UDCA) responders, as inadequate responders according to Paris II criteria, and as newly diagnosed patients were prospectively recruited.

This manuscript includes the baseline data of the project.

Results

In total, 33/77 (43%) contacted centres (58% of university hospitals, 38% of non-university hospitals, and 24% of private practices) recruited 515 patients including 204 UDCA responders, 221 inadequate responders to UDCA, and 90 newly diagnosed patients.

All patients were treated with UDCA; however, a UDCA dosage below the recommended dosage of 13 mg/kg/d was observed in 38.5% of individuals after 12 months of treatment. UDCA dosages were lower in nonacademic compared to academic centres.

Only 75/219 (38.5%) of inadequate responders to UDCA received a second-line therapy with obeticholic acid (OCA) and/or bezafibrate (BZF). OCA (13% vs. 4.5%) and BZF (14% vs. 6.5%) were significantly more often prescribed by academic vs. nonacademic centres.

Pruritus (27% vs. 15.5%), fatigue (23% vs. 4.5%), and sicca syndrome (14% vs. 1%) were significantly more often reported by academic centres.

Conclusion

The German PBC registry could be established, which indicates suboptimal therapy in a relevant proportion of patients and shows significant differences between academic and nonacademic centres. Results are fundamental to improving clinical management at different levels of care.

Zusammenfassung

Hintergrund

Es gibt bislang wenige Real-World-Daten zur Primär Biliären Cholangitis (PBC) in Deutschland. Es war daher das Ziel, ein nationales Register zu gründen, um klinische Charakteristika von Patienten mit PBC und deren medikamentöse Therapie zu beschreiben.

Methoden

Es wurden drei Subgruppen innerhalb des Registers definiert: Patienten mit adäquatem Ansprechen auf Ursodeoxycholsäure (UDCA) nach den Paris-II-Kriterien, Patienten mit inadäquatem Therapieansprechen und neu diagnostizierte Patienten. Im vorliegenden Manuskript werden die Patientencharakteristika zum Rekrutierungszeitpunkt in das Register beschrieben.

Ergebnisse

33/77 (43%) kontaktierten Zentren (58% der Universitätskrankenhäuser, 38% der nicht-universitären Krankenhäuser, 24% der Praxen) rekrutierten 515 Patienten (204 UDCA Responder, 221 inadäquate UDCA-Responder, 90 neu diagnostizierte Patienten).

Alle Patienten wurden mit UDCA behandelt, allerdings wurde bei 38,5% der Fälle eine Dosis niedriger als die empfohlene Dosierung von 13 mg/kg/d nach 12 Monaten Therapie beobachtet. Die UDCA-Dosis war an nicht-akademischen Zentren niedriger als an akademischen Zentren.

Nur 75/219 (38.5%) Patienten mit inadäquatem UDCA-Therapieansprechen erhielten eine Behandlung mit Obeticholsäure (OCA) oder Bezafibrat (BZF). OCA (13% vs. 4,5%) und BZF (14% vs. 6,5%) wurden signifikant häufiger von akademischen Zentren verordnet.

Pruritus (27% vs. 15,5%), Müdigkeit (23% vs. 4,5%) und ein Sicca-Syndrom (14% vs. 1%) wurden signifikant häufiger an akademischen Zentren beschrieben.

Zusammenfassung

Das Deutsche PBC-Register konnte erfolgreich gegründet werden, weist auf eine sub-optimale Therapie bei vielen Patienten hin und zeigt signifikante Unterschiede zwischen akademischen und nicht-akademischen Zentren. Die Ergebnisse sind wichtig, um die Versorgung von PBC-Patienten zu verbessern.

Keywords

Ursodeoxycholic acid - obeticholic acid - bezafibrate - pruritus - extrahepatic manifestation

Schlüsselwörter

Ursodeoxycholsäure - Obeticholsäure - Bezafibrat - Juckreiz - extrahepatische Manifestation

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