Prognosis and Treatment of Primary Biliary Cholangitis : a new name – a new era

This thesis focuses on the natural history, risk stratification, and treatment of patients with primary biliary cholangitis. The aims of this thesis were to assess: 1. the predictive value of bilirubin within the normal range on transplant-free survival among patients with PBC 2. the incidence of and risk factors for cirrhosis-related complications among ursodeoxycholicacid treated patients with PBC 3. the association between ursodeoxycholic acid and transplant-free survival in patients with PBC, both in relative and absolute measures 4. biochemical response and clinical outcome of PBC patients treated with fenofibrate and bezafibrate 5. the estimated survival benefit induced by treatment with obeticholic acid in patients with PBC 6. time trends in liver transplantation for patients with PBC

Self-referred patients at the Emergency Department: patient characteristics, motivations, and willingness to make a copayment

Background In many countries, including the Netherlands, a substantial number of patients visit the Emergency Department (ED) without a referral by a general practitioner. The goal of this study was to determine the characteristics and motivations of self-referred patients (SRPs) at the ED. The secondary objective was to explore SRPs' opinion about copayments. Methods A survey, in seven different languages was performed among SRPs from October 2011 until January 2012 at an academic ED in the Netherlands. Patients were included on 21 day-, 21 evening-, and 21 nightshifts during week and weekend days equally. Patient characteristics, motivations, complaints, diagnosis, and the opinion regarding copayments were examined. Results A total of 436 SRPs were included (response rate 82%). Forty-seven percent of the ED population was self-referred. SRPs were mainly male (58%), between 18 and 35 years (54%), Dutch (67%), single without children (42%), and low-educated (73%). The most commonly presented complaints were of musculoskeletal origin (35%). Expected need for additional medical care (e.g., X-rays, blood tests) was the reason to visit the ED for 28% of the SRPs. Around 30% of the SRPs were not prepared to pay for an ED visit. Fifty percent of SRPs were prepared to pay up to 25 or 50 EUR. Highly educated patients were willing to pay more than patients with a low level of education (p < 0.05). Conclusions SRPs (47% of the total ED population) are often young men with musculoskeletal complaints. They are convinced that additional medical tests are necessary. About 70% of the SRPs are willing to make a copayment, half of the SRPs with a maximum between 25 EUR and 50 EUR. As highly educated SRPs are prepared to pay more, introducing copayments might influence equity in health care accessibility.Abstract Background In many countries, including the Netherlands, a substantial number of patients visit the Emergency Department (ED) without a referral by a general practitioner. The goal of this study was to determine the characteristics and motivations of self-referred patients (SRPs) at the ED. The secondary objective was to explore SRPs’ opinion about copayments. Methods A survey, i

Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis

Objective: The clinical benefit of ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) has never been reported in absolute measures. The aim of this study was to assess the number needed to treat (NNT) with UDCA to prevent liver transplantation (LT) or death among patients with PBC. Methods: The NNT was calculated based on the untreated LT-free survival and HR of UDCA with respect to LT or death as derived from inverse probability of treatment weighting-adjusted Cox proportional hazard analyses within the Global PBC Study Group database. Results: We included 3902 patients with a median follow-up of 7.8 (4.1-12.1) years. The overall HR of UDCA was 0.46 (95% CI 0.40 to 0.52) and the 5-year LT-free survival without UDCA was 81% (95% CI 79 to 82). The NNT to prevent one LT or death within 5 years (NNT5y) was 11 (95% CI 9 to 13). Although the HR of UDCA was similar for patients with and without cirrhosis (0.33 vs 0.31), the NNT5y was 4 (95% CI 3 to 5) and 20 (95% CI 14 to 34), respectively. Among patients with low alkaline phosphatase (ALP) (≤2× the upper limit of normal (ULN)), intermediate ALP (2-4× ULN) and high ALP (>4× ULN), the NNT5y to prevent one LT or death was 26 (95% CI 15 to 70), 11 (95% CI 8 to 17) and 5 (95% CI 4 to 8), respectively. Conclusion: The absolute clinical efficacy of UDCA with respect to LT or death varied with baseline prognostic characteristics, but was high throughout. These findings strongly emphasise the incentive to promptly initiate UDCA treatment in all patients with PBC and may improve patient compliance