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Dr George Mells

Consultant Hepatologist

Treatment for patients with a rare and chronic liver condition could be significantly improved following a nationwide audit of NHS centres.

Primary biliary cholangitis (PBC) is a rare liver disease that occurs in fewer than one in 2,000 people. It is a leading indication for liver transplantation in the UK. Hepatologists from almost all NHS liver centres in the UK have recently completed an audit of management of PBC. It has raised important questions about the provision of health care for rare liver diseases in the NHS.

Slow progression

PBC is an autoimmune liver disease, resulting from a person’s immune system damaging the small bile ducts in the liver. “There is a slow progression over time and in a substantial proportion of patients, PBC eventually leads to cirrhosis. Some patients develop chronic liver failure and require a liver transplant,” says Dr Mells from Addenbrooke’s Hospital in Cambridge. It is an important cause of liver disease.

First-line treatment

The first-line treatment for PBC is ursodeoxycholic acid, which is effective in most but not all patients. Second-line treatments are available in the UK for patients whose PBC is not controlled by ursodeoxycholic acid alone.

Prescription of second-line treatment for PBC is monitored by the NHS. This has shown that the number of prescriptions issued for second-line treatments is “substantially fewer” than the number of patients predicted to need it. “That was the real prompt to do the audit,” adds Dr Mells. “It needed to be national because we wanted to be able to compare the management of PBC in specialist centres to district general hospitals and across different regions of the UK.” Dr Mells explains there were also growing concerns that PBC was not being managed in accordance with the care quality standards outlined in guidelines from the British Society of Gastroenterology (BSG).

While data from the audit are still being analysed, Dr Mells says one of the key findings is that only half of the patients eligible for second-line treatment are receiving it. “That means that patients are missing out on the treatments they need. That puts them at risk of progressive liver disease and future complications of cirrhosis,” he says.

PBC patients may also have no symptoms, so the observations that should trigger a referral for second-line treatment might be overlooked.

Lack of familiarity

Not unexpectedly, the audit also shows that prescribing rates are higher for patients managed in specialist centres compared to smaller hospitals. The reason for this disparity is unclear. One possibility, however, is that due to its rarity, treatment pathways for PBC might be less well-rehearsed in smaller centres than in the UK’s major liver hubs.

Expanding on how this might lead to patients with PBC being overlooked, Dr Mells says that specialist centres frequently have clinics focussed on the condition. Conversely, in smaller centres, rare patients with PBC are interspersed amongst those with more prevalent liver conditions.

PBC patients may also have no symptoms, so the observations that should trigger a referral for second-line treatment might be overlooked. If effective treatment is delivered earlier, however, it can prevent patients from developing complications from liver disease. “Effective treatment needs to start as early as possible to prevent progression of the disease and avoid the future need for a liver transplant,” he says.

Audit data

The audit covered all aspects of PBC management and was co-led by the Addenbrooke’s team, which found that most UK centres willingly contributed data for a national report. Project manager, Steven Flack, explains: “Each centre is going to receive their data back to identify gaps within their own centre to put procedures in place to improve treatment.” Dr Mells adds: “The broad aim is to improve care for PBC and make sure that patients eligible for second-line treatment are identified and referred to the local multi-disciplinary team.”

While this audit has identified issues that must be addressed, there is hope that it will lead to measurable improvement in care for patients with this rare liver disease in the UK.

This article has been sponsored by Intercept Pharma UK & Ireland Limited (“Intercept”). Intercept has recommended the potential authors for this article, however, with the exception of a factual accuracy check, Intercept has had no editorial control over the content of this article.

Date of Prep: March 22

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