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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.

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BACKGROUND: Ulcerative colitis (UC) is a chronic inflammatory bowel disease with recurrent episodes of debilitating symptoms negatively affecting work productivity and health-related quality of life (HRQoL). The use of biologics in UC treatment improves work and HRQoL but prospective long-term data concerning the treatment with TNFα inhibitor golimumab in UC patients are still rare. Therefore, our study aimed to evaluate the change in work productivity, capacity for daily activities and HRQoL in UC patients treated with golimumab in Germany. METHODS: Using the Work Productivity and Activity Impairment questionnaire, the change in work productivity and in capacity for daily activities after 3 months and over the whole observational period of 24 months were assessed (both primary endpoints). Disease-specific and health-related quality of life (QoL) were analyzed with the Inflammatory Bowel Disease Questionnaire (IBDQ), the Short-Form 12 Health Survey Questionnaire (SF-12), and the Partial Mayo Score (secondary endpoints). Further, disease-related hospitalization rates were assessed. RESULTS: This prospective non-interventional study included 286 patients. Thereof, 212 patients were employed at baseline (modified intention to treat analysis set employed at baseline, mITTe). 61.3% of the mITTe patients had moderate and 17.0% had severe UC. Three months after initiation of golimumab therapy, total work productivity impairment (TWPI) score and activity impairment score improved significantly from baseline with a mean change of - 17.3% (p < 0.0001) and - 14.4% (p < 0.0001), respectively. Results persisted over 24 months (mean change TWPI score: - 24.5%, mean change activity impairment score: - 30.0%). Disease- and health-related QoL also improved significantly under golimumab treatment as indicated by increased IBDQ [mean change: 28.0 (SD: ± 36.1, month 3), 42.1 (SD: ± 39.5, month 24)] and SF-12 scores [PCS-12: 45.9 (SD: ± 8.5), MCS-12: 4.9 (SD: ± 10.6, month 3), PCS-12: 5.9 (SD: ± 9.0), MCS-12: 6.4 (SD: ± 11.1, month 24)]. Disease-related hospitalization rate decreased from 16.0% (BL) to 4.3% at month 24 and the mean number of missed working days due to UC decreased from 8.2 (SD: 17.6, BL) to 0.7 (SD: 2.1) after golimumab induction. CONCLUSIONS: Golimumab leads to notable long-term improvements in work productivity, daily activity, HRQoL, and disease-related hospitalization rates in patients with moderate to severe UC. TRIAL REGISTRATION: PEI (Paul-Ehrlich-Institute, Langen, Germany) Registration Nr: NIS#255 ( https://www.pei.de/SharedDocs/awb/nis-0201-0300/0255.html ).

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BACKGROUND: The introduction of biologics has revolutionized the management of the chronic inflammatory bowel disease, ulcerative colitis (UC), with many patients experiencing significant improvements not only in their symptoms but in other outcomes relevant to individuals and society as a whole. In Germany, there are no prospective data > 3 mo that assess the work productivity, daily activities and quality of life (QoL) of patients with moderate-to-severe UC treated with golimumab. AIM: To assess change in work productivity, capacity for daily activities and QoL in UC patients treated with golimumab in Germany. METHODS: The validated Work Productivity Activity Impairment (WPAI) Questionnaire was used to analyze the change in work productivity, the capacity for daily activities after three months (primary endpoint) and disease specific and health related QoL (HRQoL) up to 1 year (secondary endpoints). The changes in work productivity and activity impairment were evaluated every three months until month twelve compared to baseline. Disease-specific and health-related QoL were assessed with the inflammatory bowel disease questionnaire and with the short-form 12 health survey questionnaire (SF-12). RESULTS: This prospective non-interventional study included 287 patients. The analysis population was comprised of 282 patients who had completed at least two visits. At baseline, 61% of patients had moderate UC and 18% had severe UC. Furthermore, 75% of patients worked full-time or part-time at baseline. A total of 212 patients who were employed at the start of the study (employed population) were evaluated for the primary endpoint. Golimumab significantly reduced all WPAI sub-scores compared to baseline after three, six, nine and twelve months after the start of treatment (P < 0.0001). In addition, disease-specific QoL and HRQoL, as measured by the SF-12 questionnaire, improved significantly with golimumab at all evaluation times (P < 0.0001 in each case vs baseline). CONCLUSION: Treatment of moderate-to-severe UC with golimumab leads to significant improvements in patient´s work productivity, daily activity and QoL over twelve months.

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