scholarly journals The British Society of Gastroenterology/UK-PBC primary biliary cholangitis treatment and management guidelines

Gut ◽  
2018 ◽  
Vol 67 (9) ◽  
pp. 1568-1594 ◽  
Author(s):  
Gideon M Hirschfield ◽  
Jessica K Dyson ◽  
Graeme J M Alexander ◽  
Michael H Chapman ◽  
Jane Collier ◽  
...  
Keyword(s):  
Cell Injury ◽  
Age At Onset ◽  
Symptom Burden ◽  
Response To Treatment ◽  
British Society ◽  
Primary Biliary Cholangitis ◽  
Biliary Cirrhosis ◽  
Immune Mediated ◽  
Elevated Bilirubin ◽  
End Stage

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. Both genetic and environmental influences are presumed relevant to disease initiation. PBC is most prevalent in women and those over the age of 50, but a spectrum of disease is recognised in adult patients globally; male sex, younger age at onset (<45) and advanced disease at presentation are baseline predictors of poorer outcome. As the disease is increasingly diagnosed through the combination of cholestatic serum liver tests and the presence of antimitochondrial antibodies, most presenting patients are not cirrhotic and the term cholangitis is more accurate. Disease course is frequently accompanied by symptoms that can be burdensome for patients, and management of patients with PBC must address, in a life-long manner, both disease progression and symptom burden. Licensed therapies include ursodeoxycholic acid (UDCA) and obeticholic acid (OCA), alongside experimental new and re-purposed agents. Disease management focuses on initiation of UDCA for all patients and risk stratification based on baseline and on-treatment factors, including in particular the response to treatment. 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. Follow-up of patients is life-long and must address treatment of the disease and management of associated symptoms.

scholarly journals Barriers to implementation of stratified care in primary biliary cholangitis: a scoping exercise

2019 ◽  
Vol 6 (1) ◽  
pp. e000226
Author(s):  
Margaret Corrigan ◽  
Gideon Hirschfield ◽  
Sheila Greenfield ◽  
Jayne Parry
Keyword(s):  
Primary Care ◽  
Risk Groups ◽  
Work Load ◽  
Low Risk ◽  
Response To Treatment ◽  
British Society ◽  
Primary Biliary Cholangitis ◽  
Care Pathways ◽  
Stakeholder Perspectives ◽  
Stratified Care

Patients with primary biliary cholangitis (PBC) can be stratified into low-risk and high-risk groups based on their response to treatment. Newly published guidelines from the British Society of Gastroenterology suggest low-risk patients can be managed substantially in primary care. This represents a shift from existing practice and makes assumptions about service capacity and the willingness of both patients and health care practitioners (HCPs) to make this change. The aim of this paper is to identify possible barriers to the implementation of these new care pathways through review of the PBC-specific literature and by identifying the experiences of patients and HCPs managing a different condition with comparable patients and disease characteristics. Searches of MEDLINE, CINAHL and EMBASE were undertaken. Within the existing PBC literature there is little data surrounding stakeholder perspectives on place of care. Review of the breast cancer literature highlights a number of barriers to change including primary care practitioner knowledge and work load, communication between healthcare settings, and the significance of the established doctor–patient relationship. Further research is needed to establish the extent to which these barriers may surface when changing PBC care pathways, and the actions required to overcome them.

scholarly journals Association between Circulating Growth Differentiation Factor 15 and Cirrhotic Primary Biliary Cholangitis

2020 ◽  
Vol 2020 ◽  
pp. 1-11
Author(s):  
Zhanyi Li ◽  
Yu Liu ◽  
Xiangyong Li ◽  
Yuankai Wu ◽  
Fangji Yang ◽  
...  
Keyword(s):  
Serum Levels ◽  
Response To Treatment ◽  
Primary Biliary Cholangitis ◽  
Strongly Correlated ◽  
Weight Changes ◽  
Adequate Treatment ◽  
Growth Differentiation Factor 15 ◽  
Reactive Protein ◽  
Differentiation Factor ◽  
End Stage

Primary biliary cholangitis (PBC) is a common condition that usually shows a progressive course towards cirrhosis without adequate treatment. Growth differentiation factor 15 (GDF15) plays multiple roles in various pathological conditions. The overall role of circulating GDF15 in cirrhotic PBC requires further investigation. Twenty patients with cirrhotic PBC, 26 with non-cirrhotic PBC, and 10 healthy subjects were enrolled between 2014 and 2018, and the serum levels of GDF15 were measured via enzyme immunoassay. The correlations between serum GDF15, weight, biochemical parameters, and the prognosis were analysed. Serum levels of GDF15 were significantly higher in cirrhotic PBC patients than in non-cirrhotic PBC patients or healthy controls ( p = 0.009 and p < 0.001 , respectively). The circulating GDF15 levels strongly correlated with weight changes ( r = − 0.541 , p = 0.0138 ), albumin ( r = − 0.775 , p < 0.0001 ), direct bilirubin ( r = − 0.786 , p < 0.0001 ), total bile acids ( r = 0.585 , p = 0.007 ), and C-reactive protein ( r = 0.718 , p = 0.0005 ). Moreover, circulating GDF15 levels strongly correlated with the Mayo risk score ( r = 0.685 , p = 0.0009 ) and Model for End-stage Liver Disease score ( r = 0.687 , p = 0.0008 ). Determined by the area under the receiver operating characteristic curves, the overall diagnostic accuracies of GDF15 were as follows: cirrhosis = 0.725 (>3646.55 pg/mL, sensitivity: 70.0%, specificity: 69.2%), decompensated   cirrhosis = 0.956 (>4073.30 pg/mL, sensitivity: 84.62%, specificity: 100%), and cirrhotic biochemical non-responders = 0.835 (>3479.20 pg/mL, sensitivity: 71.43%, specificity: 92.31%). GDF15 may be a useful and integrated biochemical marker to evaluate not only the disease severity and prognosis but also the nutrition and response to treatment of cirrhotic PBC patients, and its overall performance is satisfactory. Therapy targeting GDF15 is likely to benefit cirrhotic PBC patients and is worth further research.

scholarly journals Primary Biliary Cholangitis: Predictors of Poor Response to Ursodeoxycholic Acid after 1 Year of Treatment in Moroccan Patients

2021 ◽  
pp. 990-995
Author(s):  
Hakima Abid ◽  
Inssaf Akoch ◽  
Maria Lahlali ◽  
Nada Lahmidani ◽  
Mounia El Yousfi ◽  
...  
Keyword(s):  
Ursodeoxycholic Acid ◽  
Biological Response ◽  
Poor Response ◽  
Overlap Syndrome ◽  
Response To Therapy ◽  
Response To Treatment ◽  
Decompensated Cirrhosis ◽  
Primary Biliary Cholangitis ◽  
Biliary Cirrhosis ◽  
Average Value

Introduction: Primary biliary cholangitis (PBC), the new dominance of primary biliary cirrhosis, is a cholestatic disease of autoimmune etiology and represents the leading cause of intra-hepatic cholestasis. Treatment is mainly based on ursodeoxycholic acid. The biological response to treatment is the main predictor of survival without liver transplantation. The Globe-score has been recently validated as the main prognostic factor. Materials and methods: This is a retrospective study carried out in our department collating all cases of PBC followed in consultation. The aim of our work is to research the predictors of poor response to UDCA. Results: 46 patients were collected. The mean age of the patients was 58.82 years, with a predominance of women (n = 43, 93.5%). 34.78% of patients were in the stage of cirrhosis. Anti-M2 mitochondria antibodies were positive in 44 patients (95.65%). An overlap syndrome was found in 11 patients (23.9%). Treatment was based on UDCA combined with corticosteroid therapy and immunosuppressant for overlap syndrome. A biochemical response at 1 year of treatment according to the Paris II criteria was found in 47.8%. The average value of the globe score was 1.35. A score greater than 0.30 was objectified in 20 cases (43.47%). Nineteen cirrhotic patients (41.30%) had a globe score> 0.30. Factors associated with poor response to therapy were: stage of decompensated cirrhosis, elevated pre-therapy total bilirubin greater than 30 g / l and hypoalbunemia less than 35 g / l. The study of the correlation between Globe score and Paris II showed a strong and significant association with a correlation coefficient estimated at 67%. The Paris II score was significantly correlated with the response to treatment (p = 0.001). Conclusion: In accordance with the data in the literature, the globe-score and Paris II are two similar predictive means for evaluating the response at 1 year of treatment in Moroccan context. Keywords: Morocco, Predictors of response, Primary biliary cholangitis, Ursodeoxycholic acid

Timing, Management, and Outcomes of Liver Transplantation in Primary Sclerosing Cholangitis

2017 ◽  
Vol 37 (04) ◽  
pp. 305-313 ◽  
Author(s):  
Cynthia Levy ◽  
Eric Martin
Keyword(s):  
Liver Transplantation ◽  
Liver Disease ◽  
Primary Sclerosing Cholangitis ◽  
Graft Survival ◽  
Sclerosing Cholangitis ◽  
Cholestatic Liver Disease ◽  
Biliary Cirrhosis ◽  
Immune Mediated ◽  
End Stage ◽  
Patient And Graft Survival

AbstractPrimary sclerosing cholangitis (PSC) is a chronic, immune-mediated cholestatic liver disease that often progresses to secondary biliary cirrhosis and end-stage liver disease. Short of liver transplantation (LT), there is no effective treatment for PSC. PSC accounts for approximately 5% of total adult LTs in the US and is currently the fifth most common indication for LT. Patient and graft survival for PSC is among the highest for all indications for LT. The main factors that impact outcomes after LT for PSC include biliary strictures, rejection, and recurrence of PSC. Recurrent PSC (rPSC) develops in 20% of LT recipients within 5 years of LT and is associated with negative patient and graft survival. LT is a viable option for recipients who develop rPSC and progress to graft failure.

Patient ownership of primary biliary cholangitis long-term management

2020 ◽  
pp. flgastro-2019-101324
Author(s):  
Jessica Leighton ◽  
Collette Thain ◽  
Robert Mitchell-Thain ◽  
Jessica K Dyson ◽  
David E Jones
Keyword(s):  
Alkaline Phosphatase ◽  
Symptom Burden ◽  
Clinic Visit ◽  
Response To Treatment ◽  
Biochemical Response ◽  
Primary Biliary Cholangitis ◽  
Response Measure ◽  
Key Aspects ◽  
Term Management

ObjectivePatient ownership of disease is vital in rare diseases like primary biliary cholangitis (PBC). This survey of UK members of the PBC foundation aimed to assess patients’ perception of their disease management, focusing on key biomarkers and problematic symptoms.DesignRegistered PBC foundation members were surveyed on their experiences on their most recent clinic visit, covering the type of hospital and clinician and whether biochemical response and symptom burden were discussed, including who initiated these conversations. Respondents were also asked about their willingness to initiate these conversations.ResultsAcross 633 respondents, 42% remembered discussing alkaline phosphatase, the key biochemical response measure, and the majority of discussions were initiated by the healthcare provider. 56% of respondents remembered discussing itch, a key PBC symptom. There was no distinction between the grade of healthcare professional, but both patients and clinicians were significantly more likely to discuss symptoms over disease progression. Reassuringly, 84% of respondents felt willing to initiate conversations about their illness, regardless of the grade of managing clinician.ConclusionsThis work lays a positive foundation for patient education and empowerment projects, likely to improve clinical outcomes. Key aspects of management (biochemical response to treatment and symptom burden) should be emphasised as topics of discussion to both patients and clinicians managing PBC. We suggest a simple cue card to prompt patient-led discussion.

scholarly journals Primary biliary cholangitis

2021 ◽  
Vol 23 (1) ◽  
pp. 162-170
Author(s):  
I. M. Iljinsky ◽  
O. M. Tsirulnikova
Keyword(s):  
Bile Ducts ◽  
Geographic Location ◽  
Primary Biliary Cholangitis ◽  
Biliary Cirrhosis ◽  
Second Stage ◽  
Third Stage ◽  
Organ Specific ◽  
End Stage ◽  
Portal Tracts ◽  
Made In

Primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis, is an organ-specific autoimmune disease predominantly affecting middle-aged women. It does not occur in children. PBC prevalence varies depending on the geographic location of the country. Over the past 30 years, there has been an increased incidence of PBC, while significant progress has been made in understanding the pathogenesis of PBC due to the development of innovative technologies in molecular biology, immunology and genetics. The presence of antimitochondrial antibodies and cholestasis on biochemical analysis is sufficient to make a diagnosis, without the need for liver biopsy. Small- and medium-sized bile ducts are the targets of PBC. In the first stage of the disease, granulomatous destruction of the bile ducts occurs; in the second stage, loss of bile ducts, their proliferation, increased size of the portal tracts with chronic inflammation; in the third stage - fibrosis with septal formation, loss of bile ducts and cholestasis; in the fourth stage - liver cirrhosis. Previously, the survival rate of PBC patients ranged from 7.5 to 16 years. However, it has improved significantly with ursodeoxycholic acid and obeticholic acid treatment. If there is no effect from treatment and end-stage liver failure sets in, liver transplantation is performed.

scholarly journals Cholestatic liver diseases: new targets, new therapies

2018 ◽  
Vol 11 ◽  
pp. 175628481878740 ◽  
Author(s):  
Priscila Santiago ◽  
Andrew R. Scheinberg ◽  
Cynthia Levy
Keyword(s):  
Liver Diseases ◽  
Treatment Options ◽  
Standard Of Care ◽  
Primary Biliary Cholangitis ◽  
Biliary Cirrhosis ◽  
Free Survival ◽  
Improved Outcomes ◽  
Emerging Treatments ◽  
Pharmacologic Treatments ◽  
End Stage

Cholestatic liver diseases result from gradual destruction of bile ducts, accumulation of bile acids and self-perpetuation of the inflammatory process leading to damage to cholangiocytes and hepatocytes. If left untreated, cholestasis will lead to fibrosis, biliary cirrhosis, and ultimately end-stage liver disease. Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are the two most common chronic cholestatic liver diseases affecting adults, and their etiologies remain puzzling. While treatment with ursodeoxycholic acid (UDCA) has significantly improved outcomes and prolonged transplant-free survival for patients with PBC, treatment options for UDCA nonresponders remain limited. Furthermore, there is no available medical therapy for PSC. With recent advances in molecular biochemistry specifically related to bile acid regulation and understanding of immunologic pathways, novel pharmacologic treatments have emerged. In this review, we discuss the standard of care and emphasize the various emerging treatments for PBC and PSC.

scholarly journals Palliation Versus Dialysis for End-Stage Renal Disease in the Oldest Old: What are the Considerations?

2017 ◽  
Vol 10 ◽  
pp. 117822421773508
Author(s):  
Lyle S Walton ◽  
Gregory D Shumer ◽  
Björg Thorsteinsdottir ◽  
Theodore Suh ◽  
Keith M Swetz
Keyword(s):  
Renal Disease ◽  
End Stage Renal Disease ◽  
Oldest Old ◽  
Functional Decline ◽  
Symptom Burden ◽  
Vulnerable Population ◽  
Management Approach ◽  
The Us ◽  
Stage Renal Disease ◽  
End Stage

As the US population continues to age, new cases of end-stage renal disease (ESRD) in individuals, aged 85 years or older (the oldest old), are increasing. Many patients who begin hemodialysis despite questionable benefit may struggle with high symptom burden and rapid functional decline. This article reviews the history regarding the funding and development of the Medicare ESRD program, reviews current approaches to the oldest old with ESRD, and considers strategies to improve the management approach of this vulnerable population.

scholarly journals The Management of Cholestatic Liver Diseases: Current Therapies and Emerging New Possibilities

2021 ◽  
Vol 10 (8) ◽  
pp. 1763
Author(s):  
Marta Mazzetti ◽  
Giulia Marconi ◽  
Martina Mancinelli ◽  
Antonio Benedetti ◽  
Marco Marzioni ◽  
...  
Keyword(s):  
Liver Diseases ◽  
Immunosuppressive Drugs ◽  
Farnesoid X Receptor ◽  
New Drugs ◽  
Primary Biliary Cholangitis ◽  
Peroxisome Proliferator ◽  
Biliary Cirrhosis ◽  
Peroxisome Proliferator Activated Receptor ◽  
Multiple Drugs ◽  
New Treatments

Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are two chronic cholestatic liver diseases affecting bile ducts that may progress to biliary cirrhosis. In the past few years, the increasing knowledge in the pathogenesis of both diseases led to a growing number of clinical trials and possible new targets for therapy. In this review, we provide an update on the treatments in clinical use and summarize the new drugs in trials for PBC and PSC patients. Farnesoid X Receptor (FXR) agonists and Pan-Peroxisome Proliferator-Activated Receptor (PPAR) agonists are the most promising agents and have shown promising results in both PBC and PSC. Fibroblast Growth Factor 19 (FGF19) analogues also showed good results, especially in PBC, while, although PBC and PSC are autoimmune diseases, immunosuppressive drugs had disappointing effects. Since the gut microbiome could have a potential role in the pathogenesis of PSC, recent research focused on molecules that could change the microbiome, with good results. The near future of the medical management of these diseases may include new treatments or a combination of multiple drugs targeting different signaling pathways at different stages of the diseases.

Renal Osteodystrophy in Children with Chronic Renal Failure: An Unexpectedly Common and Incapacitating Complication

PEDIATRICS ◽  
1982 ◽  
Vol 70 (5) ◽  
pp. 742-750 ◽  
Author(s):  
Anthony C. Hsu ◽  
Sang Whay Kooh ◽  
Donald Fraser ◽  
William A. Cumming ◽  
Victor L. Fornasier
Keyword(s):  
Renal Failure ◽  
Chronic Renal Failure ◽  
Renal Osteodystrophy ◽  
Age At Onset ◽  
Growth Zone ◽  
Growth Patterns ◽  
Bone Lesions ◽  
Older Children ◽  
Before And After ◽  
End Stage

The incidence, age at onset, and progression of the biochemical, radiographic, and histologic characteristics of renal osteodystrophy were studied in 50 children in whom chronic renal failure had been recently diagnosed. During a ten-year observation period, 19 patients progressed to end-stage renal failure and radiographic signs of renal osteodystrophy developed in 15 of these (79%). Renal osteodystrophy developed in all nine patients whose chronic renal failure was diagnosed before 3 years of age and in six of the ten children with later onset of failure. The mean interval from diagnosis of renal failure to development of osteodystrophy was 1.4 years. Radiographically, growth zone lesions predominated in the younger children, whereas cortical erosions were more prevalent in the older children. Histologic examination, performed in 38 patients, showed both defective mineralization and excessive resorption and was a more sensitive diagnostic index than radiography. Noticeable deformities developed in one third of the patients with osteodystrophy, despite medical treatment including vitamin D2 therapy. Deformities were particularly frequent and Severe in patients whose renal failure developed in infancy. In all 13 patients whose growth patterns were studied before and after osteodystrophy developed, the onset of bone lesions was associated with a deterioration of growth, indicating that osteodystrophy plays a major role in causing the growth retardation commonly observed in children with chronic renal failure.

Sign in / Sign up

Export Citation Format

Share Document