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Impact

Impact

Journal: BMJ Gut

Author: Hirschfield et al.

Summary: 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.

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Journal: New England Journal of Medicine 2016

Author: Nevens et al. for the POISE Study Group*

Summary: Nevens et al. (Aug. 18 issue)1 highlight the efficacy and safety of obeticholic acid in patients with primary biliary cholangitis. The inclusion of a trial with a short follow-up of 1 year for a disease with an estimated duration of one to two decades without intervention certainly dilutes the information that is needed for the analysis of clinically relevant outcomes. Appropriate criteria that define the treatment duration and response with new regimens for primary biliary cholangitis are unknown

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Journal: Lancet. 2017

Author: Hegade et al.

Summary: In patients with primary biliary cholangitis with pruritus, 14 days of ileal bile acid transporter inhibition by GSK2330672 was generally well tolerated without serious adverse events, and demonstrated efficacy in reducing pruritus severity. GSK2330672 has the potential to be a significant and novel advance for the treatment of pruritus in primary biliary cholangitis. Diarrhoea, the most common adverse event associated with GSK2330672 treatment, might limit the long-term use of this drug.

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Transforming the Definition and Diagnosis of Sarcopenia

SARC-NET: Paving the Way for Public Participation in Sarcopenia Research

Improving Cardiovascular Outcomes in Acute Coronary Syndrome: a Joint Study with Industry

Interventions for Better Muscle Ageing

Gait Analysis as a Supportive Marker for Diagnosing Different Types of Dementia

Diagnosing and Managing Prodromal Lewy Body Dementia

Health Data Platform – Bringing Together Data to Support Patient Outcomes

Improving Diagnosis and Management of Lewy Body Dementias

Bringing Creative Activities to Those Living with Dementia, as well as the Staff and Carers Supporting Them

Towards a Better Diagnosis: Dementia with Lewy Bodies and Parkinson’s Disease Dementia

Personalised treatment for people with Primary Biliary Cholangitis (PBC)

Remote monitoring of disease and physical activity in patients with rheumatoid arthritis: a pilot study

Research and Industry Collaborate to Learn More about Fatigue

Predicting Drug-free Remission in Rheumatoid Arthritis

Autologous Tolerogenic Dendritic Cells for Rheumatoid Arthritis (AuToDeCRA)

Consolidating excellence in Rare Disease Research and Networking

Using Imaging Technology to Better Understand Diseases

Prevention of Mitochondrial Disease: Mitochondrial Donation

Translational Advances in CYLD Cutaneous Syndrome (CCS)

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

Obeticholic Acid in Primary Biliary Cholangitis

Effect of ileal bile acid transporter inhibitor GSK2330672 on pruritus in primary biliary cholangitis: a double-blind, randomised, placebo-controlled, crossover, phase 2a study

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