Materiały źródłowe

• #1 Primary biliary cholangitis: Epidemiology, prognosis, and treatment

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10241503/

  Primary biliary cholangitis (PBC) is a chronic cholestatic autoimmune liver disease characterized by a destructive, small duct, and lymphocytic cholangitis, and marked by the presence of antimitochondrial antibodies. […] Outcomes largely depend on early recognition of the disease and prompt institution of treatment, which, in turn, are directly influenced by provider bias and socioeconomic factors. […] The transplant-free survival rate for untreated patients has been estimated at 79%, 59%, and 32% at 5, 10, and 15 years, respectively. In contrast, those treated with UDCA experience significantly higher survival rates of 90%, 78%, and 66% at 5, 10, and 15 years, respectively. […] Racial differences and health care disparities also influence survival in patients with PBC. Black and Hispanic patients have a significantly higher risk of waitlist mortality (HR: 1.26 and HR: 1.41, respectively) and removal from the transplant list due to clinical deterioration compared with Whites.

• #2 Primary biliary cholangitis: Epidemiology, prognosis, and treatment

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10241503/

  The introduction of UDCA has helped reshape the progression and prognosis of PBC. […] Symptoms do not correlate with the disease stage and can occur at any point. […] A log-linear association between serum alkaline phosphatase (ALP) and risk of LT and death has been described in patients with PBC, with higher levels translating into decreased transplant-free survival. […] Histological features that significantly predict the risk of liver fibrosis progression include ductopenia and increasing severity of interface hepatitis. […] However, the presence of advanced fibrosis (stage 3/4) at diagnosis is an independent predictor of poor transplant-free survival (HR: 2.85) even if the biochemical response has been achieved with UDCA. […] The UK-PBC and the GLOBE scores are derived from the analysis of multicenter PBC cohorts and have been externally validated in large international studies.

• #3

  https://journals.lww.com/hepcomm/fulltext/2023/06010/primary_biliary_cholangitis__epidemiology,.27.aspx

  Primary biliary cholangitis (PBC) is a chronic cholestatic autoimmune liver disease characterized by a destructive, small duct, and lymphocytic cholangitis, and marked by the presence of antimitochondrial antibodies. […] Outcomes largely depend on early recognition of the disease and prompt institution of treatment, which, in turn, are directly influenced by provider bias and socioeconomic factors. […] The transplant-free survival rate for untreated patients has been estimated at 79%, 59%, and 32% at 5, 10, and 15 years, respectively. In contrast, those treated with UDCA experience significantly higher survival rates of 90%, 78%, and 66% at 5, 10, and 15 years, respectively. […] A log-linear association between serum alkaline phosphatase (ALP) and risk of LT and death has been described in patients with PBC, with higher levels translating into decreased transplant-free survival.

• #3

  https://journals.lww.com/hepcomm/fulltext/2023/06010/primary_biliary_cholangitis__epidemiology,.27.aspx

  The introduction of UDCA has helped reshape the progression and prognosis of PBC. […] The frequency of post-transplant PBC is highly variable among studies (9%61%) given their different methodologies, sample sizes, and follow-up times. […] The use of UDCA to prevent PBC recurrence after transplant has not been studied prospectively.

• #4

  https://xiahepublishing.com/2310-8819/JCTH-2022-00347

  PBC, formerly known as primary biliary cirrhosis, is an autoimmune intrahepatic cholestatic disease. […] Early diagnosis and UDCA treatment have significantly altered the natural history of PBC. Patients with biochemical responses to UDCA have a survival similar to the matched control population. […] However, transplant-free survival of PBC patients with a suboptimal response to UDCA is significantly lower than that of healthy controls, although it is still higher than that of untreated PBC patients. […] The overall prognosis of PBC patients has been improved significantly by UDCA therapy. In China, the 5-year and 10-year transplantation-free survival rates of PBC patients treated with UDCA are estimated as 78.0-86.7% and 71.1-74.3%, respectively, and the 5-year incidence of HCC and decompensation as 1.6-2% and 3.8-14.3%, respectively.

• #4

  https://xiahepublishing.com/2310-8819/JCTH-2022-00347

  Not surprisingly, the prognosis of PBC patients with cirrhosis is poor. The 5-year transplantation-free survival rates for PBC patients with compensated and decompensated cirrhosis were reported to be 77.1% and 35.9%, respectively. […] The GLOBE score and UK-PBC score are based on data from multi-center large cohorts. The scores accurately predict the 5-, 10-, and 15-year transplantation-free survival rates of PBC patients, which have been verified in cohorts in many countries, including China. Generally, they are more predictive than other models.

• #5 Primary Biliary Cholangitis Life Expectancy and Prognosis | myPBCteam

  https://www.mypbcteam.com/resources/primary-biliary-cholangitis-life-expectancy-and-prognosis

  People who are asymptomatic (do not have PBC symptoms) are often in an early stage of the disease. […] Without treatment, half of people with symptomatic PBC are expected to live between five and eight years. […] With appropriate treatment, the life expectancy for people with PBC can be the same as that of the general population. […] People with a good response to UDCA can live as long as people without PBC. […] The article in Hepatic Medicine states that about 85 percent of people who receive a liver transplant live at least five more years. […] Your gastroenterologist may use a prognostic model to predict how well you’ll respond to treatment, your risk of needing a liver transplant, or your risk of liver-related death.

• #6 Predicting outcome in primary biliary cirrhosis | Annals of Hepatology

  https://www.elsevier.es/en-revista-annals-hepatology-16-articulo-predicting-outcome-in-primary-biliary-S1665268119308385

  Primary biliary cirrhosis (PBC) is a slowly progressive autoimmune liver disease that may ultimately result in liver failure and premature death. Predicting outcome is of key importance in clinical management and an essential requirement for patients counselling and timing of diagnostic and therapeutic interventions. The following factors are associated with progressive disease and worse outcome: young age at diagnosis, male gender, histological presence of cirrhosis, accelerated marked ductopenia in relation to the amount of fibrosis, high serum bilirubin, low serum albumin levels, high serum alkaline phosphatase levels, esophageal varices, hepatocellular carcinoma (HCC) and lack of biochemical response to ursodeoxycholic acid (UDCA). […] The ability to reliably predict outcome in patients with PBC is critically important in clinical management and an essential requirement for patient counselling and timing of diagnostic procedures and therapeutic interventions. The aim of this review is to examine established prognostic factors and available tools for estimating prognosis in individuals with PBC, including predictive scoring models for two of the most serious clinical complications, namely esophageal variceal bleeding and hepatocellular carcinoma (HCC).

• #7 The British Society of Gastroenterology/UK-PBC primary biliary cholangitis treatment and management guidelines | Gut

  https://gut.bmj.com/content/67/9/1568

  Primary biliary cholangitis (formerly known as primary biliary cirrhosis, PBC) is an autoimmune liver disease in which a cycle of immune mediated biliary epithelial cell injury, cholestasis and progressive fibrosis can culminate over time in an end-stage biliary cirrhosis. […] Male sex, younger age at onset (45) and advanced disease at presentation are baseline predictors of poorer outcome. […] Those intolerant of treatment with UDCA or those with high-risk disease as evidenced by UDCA treatment failure (frequently reflected in trial and clinical practice as an alkaline phosphatase 1.67 upper limit of normal and/or elevated bilirubin) should be considered for second-line therapy, of which OCA is the only currently licensed National Institute for Health and Care Excellence recommended agent.

• #7 The British Society of Gastroenterology/UK-PBC primary biliary cholangitis treatment and management guidelines | Gut

  https://gut.bmj.com/content/67/9/1568

  The independent predictive factors of cirrhosis development were serum bilirubin 17 mol/L, serum albumin 38g/L and moderate to severe lymphocytic interface hepatitis. […] We recommend that all patients with PBC should be offered structured life-long follow-up, recognising that different patients have different disease courses and may require different intensity of follow-up. […] To identify those at greatest risk of disease progression, we recommend that all patients have individualised risk stratification using biochemical response indices following 1year of UDCA therapy. […] We recommend that oral UDCA at 1315mg/kg/day is used as the first-line pharmacotherapy in all patients with PBC. If tolerated, treatment should usually be life-long. […] In patients with inadequate response to UDCA (or UDCA intolerance) as defined by ALP 1.67 x ULN and/or elevated bilirubin 2 x ULN, the addition of OCA has been associated with improvements in biochemical surrogates of disease activity reasonably likely to predict improved outcomes.

• #7 The British Society of Gastroenterology/UK-PBC primary biliary cholangitis treatment and management guidelines | Gut

  https://gut.bmj.com/content/67/9/1568

  Given the heightened awareness of poorer outcomes, attention should be given to managing high-risk, younger and UDCA non-responsive patients in specialist centres. […] The introduction of UDCA therapy, which was demonstrated in early studies to reduce serum bilirubin concentrations, has been shown not to invalidate either the absolute serum bilirubin or the Mayo risk score as prognostic markers. […] More sophisticated risk scores designed to predict prognosis in patients with PBC have been developed, and in particular recent approaches to survival analysis have addressed whether simple assessments of biochemical response to treatment, in particular with UDCA, may be useful clinically, and whether variability in such response may underpin the heterogeneity of earlier treatment and outcome studies.

• #8 Risk Stratification in Primary Biliary Cholangitis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10488776/

  Risk stratification should be performed at the time of diagnosis and re-evaluated during therapy as it allows us to predict the hazard of disease progression and the rate of response to treatment. […] The course of PBC can vary significantly among patients. […] To accurately assess each patient’s prognosis, healthcare providers must evaluate a variety of factors. […] The presence of severe interface hepatitis suggests a diagnosis of AIH or PBC-AIH OS. […] The detection of this condition is crucial due to its poorer long-term prognosis compared to pure PBC or AIH without overlap, as it demonstrates an accelerated progression of liver fibrosis. […] Identifying significant ductopenia on a liver biopsy during the initial assessment can serve as an important predictor of a poor response to the standard UDCA therapy and histological progression.

• #8 Risk Stratification in Primary Biliary Cholangitis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10488776/

  The serological profile of PBC patients can offer valuable information for both diagnosis and prognosis. […] Anti-gp210 antibodies have been found to be associated with a more aggressive form of the disease, indicating their potential prognostic value. […] The prognostic role of anti-gp210 antibodies has been recently confirmed in a large cohort study. […] Therefore, the inclusion of anti-gp210 antibodies into the UK-PBC and GLOBE scoring systems could potentially enhance their risk stratification capabilities. […] The relationship between serum ALP and the risk of LT and death in patients with PBC has been found to be log-linear, with higher levels of ALP indicating a decreased chance of transplant-free survival. […] The advent of UDCA has had a profound effect on the natural progression of PBC, as it has been found to improve LFTs, liver histology, and prolong liver transplant-free survival.

• #8 Risk Stratification in Primary Biliary Cholangitis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10488776/

  Primary Biliary Cholangitis (PBC) is a chronic cholestatic liver disease with a heterogeneous presentation, symptomatology, disease progression, and response to therapy. […] Treatment response scores after one year of therapy remain to date a major factor influencing the prognosis of PBC patients. […] Consequently, the prevailing one-size-fits-all approach is poised to be supplanted by tailored strategies, ensuring high-risk patients receive the most appropriate treatment regimen from diagnosis. […] This will require the development of a risk prediction model to assess, at the time of diagnosis, the course, outcome, and response to first and additional treatments of PBC patients. […] The risk assessment of PBC patients can significantly differ, influenced by a multitude of factors such as their age, gender, autoantibody profile, biochemical panel results at the time of diagnosis and during the treatment, and disease stage.

• #8 Risk Stratification in Primary Biliary Cholangitis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10488776/

  A lack of improvement in LFTs despite UDCA therapy is associated with a poorer prognosis. […] The GLOBE and UK-PBC scores have demonstrated superior performance in predicting death or liver transplantation compared to the Paris-I criteria. […] The GLOBE and UK-PBC scores are widely recognized as effective tools for predicting patient prognosis, but they do have some limitations. […] In conclusion, the landscape of risk stratification in PBC is evolving.

• #9 Primary Biliary Cholangitis (Primary Biliary Cirrhosis): Practice Essentials, Background, Pathophysiology

  https://emedicine.medscape.com/article/171117-overview

  The most reliable determinants of patient prognosis in primary biliary cholangitis are the degree of serum bilirubin elevation and the Mayo risk score. […] Published reports indicate that, when serum bilirubin values are constantly above 2, the mean survival rate is 4.1 years. […] When bilirubin levels are constantly above 6 mg/dL, the mean survival rate is 2.1 years. […] When bilirubin levels are constantly above 10 mg/dL, the mean survival rate is 1.4 years. […] The Mayo risk score is calculated as follows: R = 0.871 loge (bilirubin in mg/dL) + (2.53) loge (albumin in g/dL) + 0.039 age in years + 2.38 loge (prothrombin time in seconds) + 0.859 (edema score of 0, 0.5, or 1). […] A report reassessed the Mayo risk score, taking into consideration other factors found to be important in the timing of transplantation in patients with chronic cholestatic liver disease.

• #9 Primary Biliary Cholangitis (Primary Biliary Cirrhosis): Practice Essentials, Background, Pathophysiology

  https://emedicine.medscape.com/article/171117-overview

  Neither the degree of serum bilirubin elevation nor the Mayo risk score are influenced by UDCA therapy. Treatment with UDCA after liver transplantation may improve outcomes in patients with recurrent primary biliary cholangitis. […] Bjrnsson et al determined that fatigue levels may be a prognostic indicator for primary biliary cholangitis. […] Among the 208 patients with the disease (16 males, 192 females, median age 59 years), the investigators recorded an overall baseline score of 28 on the fatigue impact scale (FIS). […] In their report, Bjrnsson et al suggested that high fatigue levels can aid in the predictions of liver-related mortality or liver transplantation need in patients with primary biliary cholangitis. […] Age [hazard ratio (HR) 1.1 (confidence interval (CI) 1.0-1.2)] and aspartate aminotransferase [HR 2.0 (CI 1.3-3.0)] were independently associated with decreased survival on multivariate analysis.

• #10 Risk Stratification in Primary Biliary Cholangitis

  https://www.mdpi.com/2077-0383/12/17/5713

  The relationship between serum ALP and the risk of LT and death in patients with PBC has been found to be log-linear, with higher levels of ALP indicating a decreased chance of transplant-free survival. […] Therefore, ALP level is a reliable marker of treatment response, with lower levels indicating a better prognosis, decreased mortality, and longer transplant-free survival. […] The advent of UDCA has had a profound effect on the natural progression of PBC, as it has been found to improve LFTs, liver histology, and prolong liver transplant-free survival. […] A lack of improvement in LFTs despite UDCA therapy is associated with a poorer prognosis. […] The GLOBE and UK-PBC scores have demonstrated superior performance in predicting death or liver transplantation compared to the Paris-I criteria.

• #10 Risk Stratification in Primary Biliary Cholangitis

  https://www.mdpi.com/2077-0383/12/17/5713

  Primary Biliary Cholangitis (PBC) is a chronic cholestatic liver disease with a heterogeneous presentation, symptomatology, disease progression, and response to therapy. […] Treatment response scores after one year of therapy remain to date a major factor influencing the prognosis of PBC patients. […] The risk assessment of PBC patients can significantly differ, influenced by a multitude of factors such as their age, gender, autoantibody profile, biochemical panel results at the time of diagnosis and during the treatment, and disease stage. Each of these elements plays a critical role in shaping an individual’s risk profile and can be instrumental in guiding treatment decisions. […] Risk stratification should be performed at the time of diagnosis and re-evaluated during therapy as it allows us to predict the hazard of disease progression and the rate of response to treatment.

• #10 Risk Stratification in Primary Biliary Cholangitis

  https://www.mdpi.com/2077-0383/12/17/5713

  The risk score calculators are accessible online at the respective websites. […] The landscape of risk stratification in PBC is evolving. The traditional approach of sequential therapy based on therapeutic response scores at 12 months is giving way to a more proactive, top-down approach that considers the patient’s risk at the time of diagnosis.

• #11 Development and external validation of an early prediction model to identify irresponsive patients and prognosis of UDCA treatment in primary biliary cholangitis | Scientific Reports

  https://www.nature.com/articles/s41598-024-82854-1

  Ursodeoxycholic acid (UDCA) is the first-line treatment for primary biliary cholangitis (PBC), but 20-40% of patients do not respond well to UDCA. […] Total cholesterol, alkaline phosphatase (ALP), and neutrophil-to-lymphocyte ratio (NLR) were the three independent risk factors associated with early biochemical nonresponse to UDCA treatment. […] In summary, we developed an early predictive model that could identify potential nonresponse factors to UDCA at baseline, which could facilitate risk evaluation and stratification for PBC patients. […] The 87 patients with a poor treatment response of UDCA had a significantly lower survival compared to the responders (7.77 vs. 8.93 years, P=0.023). […] We found that PBC patients with high total cholesterol, neutrophil lymphocyte rate (NLR), and alkaline phosphatase (ALP) at the time of diagnosis were associated with irresponse to UDCA, which generally has a poor prognosis, such as esophageal and gastric varices, ascites, and even death.

• #11 Development and external validation of an early prediction model to identify irresponsive patients and prognosis of UDCA treatment in primary biliary cholangitis | Scientific Reports

  https://www.nature.com/articles/s41598-024-82854-1

  Constructing a Model for UDCA nonresponse uses the above three variables exhibited good predictive value for inadequate biochemical response, its AUC reaching 0.862. […] Predicting early patients of PBC response to UDCA allows for timely adjustments to the treatment plan, which can improve patient prognosis. […] In summary, we developed a predictive model that could identify potential nonresponse factors to UDCA at baseline, which could facilitate risk evaluation and stratification for PBC patients.

• #12 Primary Biliary Cholangitis Life Expectancy and Prognosis | myPBCteam

  https://www.mypbcteam.com/resources/primary-biliary-cholangitis-life-expectancy-and-prognosis

  Primary biliary cholangitis (PBC), formerly called primary biliary cirrhosis, is a progressive liver disease that can eventually cause liver failure over a span of 15 to 20 years. […] Your doctor may be able to predict your prognosis (outcome) by looking at several factors, including: Your PBC stage, The results of your liver function tests, Your symptoms, Your PBC treatment. […] Doctors may use your PBC stage, in addition to other factors of your PBC and overall health, when predicting your outcome and deciding on your treatment plan. […] Bilirubin levels may help predict how much longer you live. […] According to research, the average survival time (or time a person lives after diagnosis) based on bilirubin level is as follows: Bilirubin constantly above 2 mg/dL Four years, Bilirubin constantly above 6 mg/dL Two years, Bilirubin constantly above 10 mg/dL 1.4 years.

• #13 Long-term outcomes in patients with primary biliary cholangitis complicated with CREST syndrome | Scientific Reports

  https://www.nature.com/articles/s41598-024-64976-8

  Primary biliary cholangitis (PBC) is frequently associated with autoimmune disease. […] The survival rates without LT (3/5/10 years) were 92/87/80% for the PBC-alone group and 98/96/96% for the PBC-CREST group, with a significantly better prognosis in the PBC-CREST group (log-rank P=0.0172). […] The predicted 5/10/15-year risks of liver-related death or LT based on the UK-PBC score were significantly lower in the PBC-CREST group (2.4/7.6/13.2%) than in the PBC-alone group (4.8/11.8/18.8%) (P<0.05). [...] The predicted 3/5-year LT-free survival rates based on the GLOBE score were significantly higher in the PBC-CREST group (93/88%) than in the PBC-alone group (88/81%) (P<0.05). [...] Patients with PBC-CREST may have better long-term outcomes than those with PBC alone. [...] In this study, the 3/5/10-year LT-free survival rates were 92/87/80% in the PBC alone group and 98/96/96% in the PBC-CREST group, with a significantly better prognosis in the PBC-CREST group. [...] Moreover, the predicted risk of LRD and LT was significantly lower for patients with PBC-CREST than for those with PBC alone. [...] Multivariate analysis of the prevalence of cirrhosis showed that the presence of CREST syndrome was an independent protective factor.

• #14 Does neutrophil-to-lymphocyte ratio predict 1-year mortality in patients with primary biliary cholangitis? Results from a retrospective study with validation cohort | BMJ Open

  https://bmjopen.bmj.com/content/7/7/e015304

  Neutrophil-to-lymphocyte ratio (NLR) has been used to predict prognosis in various liver diseases, but its role in primary biliary cholangitis (PBC) is not clarified. […] NLR is closely related to short-term mortality in patients with PBC. […] Our results demonstrated that elevated baseline NLR of hospitalised patients with PBC was associated with poor survival. […] The patients with NLR 2.18 exhibited lower mortality, whereas NLR 2.18 was indicative of increased death risk. […] The predictive value of NLR was confirmed in a validation cohort. […] In conclusion, NLR, an affordable, widely available and reproducible index, is closely related to short-term mortality in patients with PBC.

• #15 CT and MR imaging of primary biliary cholangitis: a pictorial review | springermedizin.de

  https://www.springermedizin.de/ct-and-mr-imaging-of-primary-biliary-cholangitis-a-pictorial-rev/26227650

  PBC with baseline cirrhosis is widely recognized as a strong predictor of worse long-term outcomes, and liver transplantation should be considered once symptoms fail to resolve. […] A recent meta-analysis showed that the pooled HCC incidence in patients with PBC cirrhosis at baseline was 13.05 per 1000 person-years, which was significantly higher than that of PBC patients without cirrhosis. […] In PBC, liver stiffness measured by MRE can be used to identify histologically advanced fibrosis and performs better than biochemical indicators. […] The ability of hepatobiliary phase imaging to differentiate different etiologies of liver cirrhosis (i.e. PBC or viral hepatitis) needs further investigation.

• #15 CT and MR imaging of primary biliary cholangitis: a pictorial review | springermedizin.de

  https://www.springermedizin.de/ct-and-mr-imaging-of-primary-biliary-cholangitis-a-pictorial-rev/26227650

  Primary biliary cholangitis (PBC) is a rare chronic autoimmune-mediated cholestatic liver disease involving medium and small bile ducts that can lead to liver fibrosis and cirrhosis. […] Moreover, CT and MRI can be used to monitor the disease progression after treatment of PBC (e.g. the onset of cirrhotic decompensation or HCC) to guide the clinical decisions for liver transplantation. […] With the optimization of imaging technology, magnetic resonance elastography (MRE) offers additional information on liver stiffness, allows for the identification of early cirrhosis in PBC and provides a basis for predicting prognosis. […] The presence of liver surface nodularity was correlated with advanced stages of the disease and can be used for differentiating stage III and stage III-IV fibrosis in PBC.

• #16 Clinical manifestations, diagnosis, and prognosis of primary biliary cholangitis – UpToDate

  https://www.uptodate.com/contents/clinical-manifestations-diagnosis-and-prognosis-of-primary-biliary-cholangitis

  Clinical manifestations, diagnosis, and prognosis of primary biliary cholangitis […] Primary biliary cholangitis (PBC; previously referred to as primary biliary cirrhosis) is characterized by a T-lymphocyte-mediated attack on small intralobular bile ducts. A continuous assault on the bile duct epithelial cells leads to their gradual destruction and eventual disappearance (picture 1). The sustained loss of intralobular bile ducts causes the signs and symptoms of cholestasis and eventually may result in cirrhosis and liver failure. […] With the advent of treatment with ursodeoxycholic acid, the majority of patients now have normal life expectancies and only a minority of patients develops cirrhosis. […] This topic will review the clinical manifestations and diagnosis of PBC. It will also review factors associated with prognosis. […] The American Association for the Study of Liver Diseases issued a practice guideline for PBC in 2018.

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