Sub-optimal therapy of patients with primary biliary cholangitis (PBC) in the real-life stetting of the German PBC cohort
- PMID: 39227008
- DOI: 10.1055/a-2382-7720
Sub-optimal therapy of patients with primary biliary cholangitis (PBC) in the real-life stetting of the German PBC cohort
Abstract
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.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.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.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.
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.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.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.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.
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Conflict of interest statement
JW: Lecturer and advisory board member for Intercept/Advanz Pharma, GSK, Ipsen TM: Ssupported by the German Research Foundation Grants MU 2864/1-3 and MU 2864/3- 1.KS: Receipt of speaker´s honoraria or advisory board: Gilead, Intercept/Advanz Pharma, Abbvie, Falk, Novo Nordisk HB: Consultant: Intercept/Advance Pharma, Ipsen GD: Consultant / speaker: AbbVie, Advanz/Intercept, Alexion, Falk Foundation, Gilead, Novartis, Orphalan, Univar PAR: Consulting and lectures fees: Astra Zeneca, BMS, Boston Scientific, CSL Behring, Gilead, Pfizer, Abbvie, Norgine JMS: Consultant: Astra Zeneca, Apollo Endosurgery, Bayer, Boehringer Ingelheim, BMS, Gilead Sciences, GSK, Intercept Pharmaceuticals, Ipsen, Inventiva Pharma, Madrigal, MSD, Northsea Therapeutics, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi, Siemens Healthineers. Research Funding: Gilead Sciences, Boehringer Ingelheim, Siemens Healthcare GmbH. Stock Options: AGED diagnostics, Hepta Bio. Speaker Honorarium: Boehringer Ingelheim, Echosens, MedPublico GmbH, Novo Nordisk, Madrigal Pharmaceuticals, Histoindex, MedPublico GmbH SZ: Speakers bureau and/or consultancy: Abbvie, BioMarin, Boehringer Ingelheim, Gilead, GSK, Intercept, Ipsen, Janssen, Madrigal, MSD/Merck, NovoNordisk, SoBi, Theratechnologies KGS: Consultant: Advance Pharma, Speaker Honorarium: AbbVie, GileadJT has received speaking and/or consulting fees from Versantis, Gore, Boehringer-Ingelheim, Falk, Grifols, Genfit and CSL Behring. WPH: Consultant or Speaker Honorarium: Intercept /Advanz, Ipsen, NovoNordisk, Gilead, Abbvie, Norgine CS: Consultant, Study support or Speaker Honorarium: Calliditas, Falk, Intercept/Advanz, Ipsen, Mirum ER: Receipt of honoraria or consultation fees/advisory board: Abbvie, Amgen, Intercept, Medac, Merz, Norgine, Falk Foundation, Gilead, Pfizer, Repha, Takeda AEK: Research grant: Intercept. Speakers bureau: Abbvie, Advanz, AOP Orphan, Bayer, BMS, CMS, CymaBay, Falk, Gilead, GSK, Intercept, Ipsen, Newbridge, Novartis, Lilly, Mirum, MSD, Roche, Zambon. Consultant: Abbvie, Advanz, Alentis, AlphaSigma, AstraZenca, Avior, Bayer, BioNTech, CymaBay, Eisai, Escient, Falk, FMC, Gilead, GSK, Guidepoint, Intercept, Ipsen, Mirum, Medscape, MSD, Myr, Roche, Takeda, Viofor MR: Consultant, or Speaker Honorarium: Intercept/Advanz, Gilead, Abbvie AT: Speaker Honorarium: Intercept/Advanz VK: Consultant Astra Zeneca, Speaker’s Honoraria from AbbVie, Gilead, Falk, Mirum, Albireo/Ipsen, Merck, MedUpdate GmbH, Sanofi, CSL Behring ER: Receipt of honoraria or consultation fees/advisory board: Abbvie, Amgen, Intercept, Medac, Merz, Norgine, Falk Foundation, Gilead, Pfizer, Repha, Takeda FT: Research grant: Allergan, BMS, Inventiva, Gilead. Speakers bureau: Gilead, Abbvie, Falk, Merz, Orphalan, Advanz. Consultant: Allergan, AstraZeneca, Gilead, Abbvie, Alnylam, BMS, Intercept / Advanz, Inventiva, Pfizer, Novartis, Novo Nordisk, Sanofi. CT: Receipt of honoraria or consultation fees/advisory board: Intercept/Advanz Pharma TB: Receipt of grants/research supports: Abbvie, BMS, Gilead, MSD/Merck, Humedics, Intercept, Merz, Norgine, Novartis, Orphalan, Sequana Medical; Receipt of honoraria or consultation fees/advisory board: Abbvie, Alexion, Albireo, Bayer, Gilead, GSK, Eisai, Enyo Pharma, HepaRegeniX GmbH, Humedics, Intercept, Ipsen, Janssen, MSD/Merck, Novartis, Orphalan, Roche, Sequana Medical, SIRTEX, SOBI, and Shionogi; Participation in a company sponsored speaker’s bureau: Abbvie, Advance Pharma, Alexion, Albireo, Bayer, Gilead, Eisai, Falk Foundation, Intercept, Ipsen, Janssen, MedUpdate GmbH, MSD/Merck, Novartis, Orphalan, Sequana Medica, SIRTEX, and SOBI Nothing to disclose: AF, RG, UN, TB, AW, MH, RG, CB, TTW, RB, HG, ND, MM, PM, JUM, AK, KP, NKB
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