UPDATE: The BSG/UK-PBC Primary Biliary Cholangitis Treatment and Management Guidelines

UPDATEThese guidelines have now been published in Gut and can be accessed here: https://bit.ly/2v6zvSh

Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease. It continues to have a burden of morbidity and mortality that spans both the consequences of a sometimes progressive biliary injury, alongside a symptom profile notably encompassing pruritus, sicca complex, fatigue, abdominal discomfort and arthralgias/bone pain. UK-PBC and the BSG have partnered to develop a comprehensive guideline document to provide detailed advice and recommendations on the best approaches to management of disease. A series of recommendations and audit standards are proposed, to ensure that patients are offered timely licenced therapy (ursodeoxycholic acid [UDCA], obeticholic acid [OCA]) in addition to actively managed for symptoms, as well as complications of progressive liver disease.

The guidelines have now been submitted, and we are awaiting publication. In the meantime, we have summarised the key recommendations below:

1. That the presence of anti-mitochondrial antibodies (greater than 1 in 40) or highly PBC-specific anti-nuclear antibodies, in the appropriate context of cholestatic liver biochemistry, without alternate explanation, is usually sufficient for confidently reaching the diagnosis of PBC.

2. That all patients with PBC should be offered structured lifelong follow-up, recognising that different patients have different disease courses, and may require different intensity of follow up.

3. That risk assessment should evaluate disease severity and activity at baseline and on treatment. We recommend a combination of serum liver tests (to identify those with an elevated bilirubin, a platelet count <150, or biochemical disease activity on treatment), imaging (liver ultrasound to identify overt cirrhosis and splenomegaly; transient elastography to identify increased liver stiffness) and recognition of young age at disease onset (<45 years), and male sex.

4. That to identify those at greatest risk of disease progression, we recommend that all patients have individualised risk stratification using biochemical response indices following one year of UDCA therapy. We suggest that UDCA treated patients with an alkaline phosphatase (ALP) >1.67x ULN and/or elevated bilirubin <2x ULN, represent a group of high-risk patients in whom there is randomised controlled trial evidence for the addition of second-line therapy.

5. That we recommend oral UDCA at 13-15mg/kg/day is used as the first-line pharmaco-therapy in all patients with PBC. If tolerated, treatment should usually be lifelong.

6. That in patients with inadequate response to UDCA (or UDCA intolerance) as defined by ALP >1.67x ULN and/or elevated Bilirubin <2x ULN, the addition of OCA has been associated with improvements in biochemical surrogates of disease activity reasonably likely to predict improved outcomes. We therefore recommend, in keeping with the NICE evaluation of OCA, that the addition of OCA for patients with an inadequate response to UDCA, or intolerant of UDCA, is considered. We recommend dose adjustment in patients with advanced liver disease as per the drug label.

7. That we recommend all patients should be evaluated for the presence of symptoms, particularly fatigue and itch. Clinicians should recognise that severity of symptoms does not correlate with stage of disease.

8. That true overlap with autoimmune hepatitis is probably rare and we suggest that when suspected, liver biopsy, with expert clinico-pathological review is needed to make the diagnosis and guide treatment.

9. That we recommend that patients with PBC should be offered the chance to seek support from patient support groups.

10. That we recommend that clinicians caring for patients with PBC should consider introducing clinical audit tools to document and improve the quality of care delivered to patients.

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