Early PBC prognosis advocated

Biochemical response to ursodeoxycholic acid (UDCA) at 6 months is a better predictor for primary biliary cirrhosis (PBC) prognosis than the current standard of 1 year, study results suggest.

The authors say their findings indicate that early determination of prognosis should be used in both clinical practice and trials to identify patients who will benefit the most from alternative therapies.

The study included 187 patients diagnosed with PBC between 1995 and 2012 who were treated with UDCA 13-15 mg/kg daily.

Over a median follow-up of 5.8 years, 12 (6.4%) patients died or underwent liver transplantation, and 25 (13.4%) developed cirrhosis or hepatocellular carcinoma.

The authors observed a rapid decline in liver function parameters (serum alkaline phosphatase, gamma-glutamyl transferase, aspartate aminotransferase, and alanine aminotransferase), bilirubin, and immunoglobulin M, and an elevation of albumin, during the first 3 months of UDCA treatment, after which time values remained fairly stable during the first year.

By all definitions of clinical response, except for the Rotterdam criteria, biochemical response at 3, 6, and 12 months significantly discriminated between patients' long-term outcomes.

The authors subsequently used the Paris, Barcelona, Toronto, and Ehime definitions of response to assess its predictive value at 3, 6, and 12 months. In comparison to biochemical response at 1 year, the response at 3 months had a higher positive predictive value (PPV), a lower negative predictive value (NPV), and increased negative likelihood ratio (NLR) for all criteria. This means that response at 3 months would be effective at identifying patients with good prognosis but poor for selecting at-risk patients, say the authors.

Conversely, biochemical response at 6 months could more accurately predict long-term outcome than could response at 1 year, leading to the same or higher PPV and NPV, and lower NLR, in comparison.

Non-response to UDCA therapy is well known to predict poor prognosis in PBC. However, Feng-Chun Zhang (Peking Union Medical Colleages Hospital, Beijing, China) and colleagues say that, although previously published criteria have relied on response at 1 or 2 years, there is increasing recognition that at-risk patients should be identified within the first 6 months of treatment to optimize outcomes.

"Our findings provide important information that will be helpful in clinical evaluation of PBC patients. It may also facilitate a more rapid identification of patients who need new therapeutic approaches," they conclude in Hepatology.

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