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Concept: Ursodiol


The biochemical response to ursodeoxycholic acid (UDCA) in primary biliary cirrhosis (PBC) is a strong predictor of long-term outcome and thus facilitates the rapid identification of patients needing new therapeutic approaches. Numerous criteria for predicting outcome of treatment have been studied based on biochemical response to UDCA at 1 year. We sought to determine whether an earlier biochemical response at 3 or 6 months could as efficiently identify patients at risk of poor outcome, as defined by liver-related death, liver transplantation, and complications of cirrhosis. We analyzed the prospectively collected data of 187 patients with a median follow-up of 5.8 years (range: 1.3-14 years). The survival rates without adverse outcome at 5 years and 10 years were 86% and 63%. Under UDCA therapy, laboratory liver parameters experienced a most prominent improvement in the first 3 months (P < 0.0001) and then stayed relatively stable for the following months. The Paris, Barcelona, Toronto, and Ehime, but not the Rotterdam definition applied at 3, 6, and 12 months significantly discriminated the patients in terms of long-term outcome. Compared to biochemical responses evaluated after 1 year of UDCA therapy, biochemical responses at the third month demonstrated higher positive predictive value (PPV) but lower negative predictive value (NPV) and increased negative likelihood ratio (NLR) by all definitions; biochemical responses at the sixth month showed higher or the same PPV and NPV and lower NLR by all definitions.Conclusion: For the previously published criteria, biochemical responses at the sixth month can be used in place of those evaluated after 1 year of UDCA therapy. Our findings justify a more rapid identification of patients who need new therapeutic approaches. (HEPATOLOGY 2013.).

Concepts: Bilirubin, Gastroenterology, Ursodiol, Liver, Primary biliary cirrhosis, Hepatology, Cirrhosis


BACKGROUND & AIMS:: Studies of primary biliary cirrhosis (PBC) phenotypes have largely been performed using small and used selected populations. Study size has precluded investigation of important disease sub-groups, such as men and young patients. We used a national patient cohort to obtain a better picture of PBC phenotypes. METHODS:: We performed a cross-sectional study using the United Kingdom-PBC patient cohort. Comprehensive data were collected for 2353 patients on diagnosis reports, response to therapy with ursodeoxycholic acid (UDCA), laboratory results, and symptom impact (assessed using the PBC-40 and other related measures). RESULTS:: Seventy-nine percent of the patients reported current UDCA therapy, with 80% meeting Paris response criteria. Men were significantly less likely to have responded to UDCA than women (72% vs 80% response, P <.05); male sex was an independent predictor of non-response on multivariate analysis. Age at diagnosis was strongly and independently associated with response to UDCA; response rates ranged from 90% among patients who presented with PBC when they were older than 70 y, to less than 50% for those younger than 30 y ( P <.0001). Patients who presented at younger ages were also significantly more likely not respond to UDCA therapy, based on alanine aminotransferase and aspartate aminotransferase response criteria, and more likely to report fatigue and pruritus. Women had mean fatigue scores 32% higher than men's ( P <.0001). The increase in fatigue severity in women was strongly related (r=0.58, P <.0001) to higher levels of autonomic symptoms ( P <.0001). CONCLUSIONS:: Among patients with PBC, response to UDCA treatment and symptoms are related to sex and age at presentation, with the lowest response rates and highest levels of symptoms in women presenting at <50 years of age. Increased severity of fatigue in women is related to increased autonomic symptoms, making dysautonomia a plausible therapeutic target.

Concepts: Post-concussion syndrome, Cirrhosis, Symptoms, Alanine transaminase, Symptom, Hepatology, Ursodiol, Primary biliary cirrhosis


Ursodeoxycholic acid is administered to patients with primary biliary cirrhosis, a chronic progressive inflammatory autoimmune-mediated liver disease with unknown aetiology. Despite its controversial effects, the U.S. Food and Drug Administration has approved its usage for primary biliary cirrhosis.

Concepts: Ursodiol, Liver, Liver biopsy, Bilirubin, Primary sclerosing cholangitis, Primary biliary cirrhosis, Cirrhosis, Hepatology


We performed a meta-analysis to evaluate the effects of ursodeoxycholic acid (UDCA) on pruritus, liver test results, and outcomes of babies born to women with intrahepatic cholestasis of pregnancy (ICP).

Concepts: Ursodiol, Fetus, Hepatology, Childbirth, Obstetrics, Cholestasis, Progressive familial intrahepatic cholestasis, Intrahepatic cholestasis of pregnancy


The efficacy of ursodeoxycholic acid (UDCA) on long-term outcome of primary biliary cirrhosis (PBC) has been less documented in Chinese cohort. We aimed to assess the therapeutic effect of UDCA on Chinese patients with PBC. In the present study, 67 patients with PBC were treated with UDCA (13-15 mg·kg(-1)·day(-1)) and followed up for 2 years to evaluate the changes of symptoms, laboratory values and histological features. As the results indicated, fatigue and pruritus were obviously improved by UDCA, particularly in patients with mild or moderate symptoms. The alkaline phosphatase and γ-glutamyl transpetidase levels significantly declined at year 2 comparing to baseline values, with the most profound effects achieved in patients at stage 2. The levels of alanine aminotransferase and aspartate aminotransferase significantly decreased whereas serum bilirubin and immunoglobulin M levels exhibited no significant change. Histological feature was stable in patients at stages 1-2 but still progressed in patients at stages 3-4. The biochemical response of patients at stage 2 was much better than that of patients at stages 3-4. These data suggest that, when treated in earlier stage, patients in long-term administration of UDCA can gain favorable results not only on symptoms and biochemical responses but also on histology. It is also indicated that later histological stage, bad biochemical response and severe symptom may be indicators of poor prognosis for UDCA therapy.

Concepts: Amino acid, Primary sclerosing cholangitis, Liver function tests, Cirrhosis, Ursodiol, Hepatology, Bilirubin, Primary biliary cirrhosis


We evaluated the efficacy and safety of obeticholic acid (OCA, α-ethylchenodeoxycholic acid) in a randomized controlled trial of patients with primary biliary cirrhosis (PBC) who had an inadequate response to ursodeoxycholic acid (UDCA) therapy.

Concepts: Autoimmune hepatitis, Bilirubin, Primary sclerosing cholangitis, Cirrhosis, Hepatology, Ursodiol, Randomized controlled trial, Primary biliary cirrhosis



As primary biliary cholangitis (PBC) is a heterogeneous disease, we hypothesized that there is a population of patients with early PBC who do not need prompt treatment with ursodeoxycholic acid (UDCA).

Concepts: Primary biliary cirrhosis, Ursodiol


Approximately one-third of patients with primary biliary cholangitis (PBC) fail to respond to ursodeoxycholic acid (UDCA) and are at risk for progression to biliary cirrhosis and end-stage liver disease. In this paper by Pares et al., the authors evaluate the effect of long-term use of bezafibrate in patients with primary biliary cholangitis (PBC) and inadequate response to UDCA. They found that addition of bezafibrate led to normalization of serum alkaline phosphatase in half of the study subjects and major improvement in pruritus. Here we discuss these findings and place them in context with current knowledge about fibrates in PBC.

Concepts: Liver function tests, Primary sclerosing cholangitis, Ursodiol, Cirrhosis, Liver, Alkaline phosphatase, Hepatology, Primary biliary cirrhosis


In this era of near universal ursodeoxycholic acid (UDCA) treatment for primary biliary cholangitis (PBC), progression to cirrhosis still occurs in an important proportion of patients. The aim of this study was to describe the incidence of cirrhosis-associated complications in patients with PBC and assess risk factors and impact on survival.

Concepts: Hepatic encephalopathy, Ursodiol, Ammonia, Primary biliary cirrhosis, Liver