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PBC Response to First Line Therapy

CE / CME

Assessing PBC Response to First-line UDCA Therapy

Pharmacists: 0.75 contact hour (0.075 CEUs)

Physicians: maximum of 0.75 AMA PRA Category 1 Credit™

Nurse Practitioners/Nurses: 0.75 Nursing contact hour

Released: March 18, 2026

Expiration: March 17, 2027

Robert Wong
Robert Wong, MD, MS, FACG, FAASLD
CME/CE Information

Activity

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Introduction

Assessing response to first-line PBC therapy with ursodeoxycholic acid (UDCA) is critical, as delays in achieving biochemical responses can translate into poor outcomes. Timely, effective therapy is key to improving PBC care and hepatic outcomes.

Transplant-Free Survival Among UDCA Nonresponders

It is not enough to treat patients with PBC; we need to measure whether they are responding to treatment, and we need to measure it early enough.

This slide depicts data from a study that stratified patients according to whether or not they responded to UDCA.1

There are many different ways to define treatment response, based on biochemical laboratory parameters such as alkaline phosphatase (ALP), total bilirubin, and, in some cases, liver enzymes. In this study, investigators assessed biochemical treatment response at 12 months using the Paris 1 criteria. The Paris 1 criteria include 3 different parameters for defining treatment response: ALP ≤3 times the upper limit of normal (ULN), aspartate aminotransferase (AST) ≤2 times the ULN, and total bilirubin ≤1 mg/dL.1,2

Here, the blue line represents those who responded, and the red line represents those who did not respond. It is very clear that patients who had a response had much better survival compared to those who did not.

These data emphasize that improvement in transplant-free survival with UDCA depends on achieving an adequate biochemical response to treatment.1

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Importance of UDCA Response to Risk of Hepatic Events

Aside from improved transplant-free survival, timely, effective PBC treatment can also reduce the risk of hepatic events, such as decompensation and hepatocellular carcinoma (HCC).3

A large, long-term study in people with PBC treated with UDCA demonstrated that the overall incidence of hepatic events (defined here as ascites, variceal bleeding, hepatic encephalopathy, and HCC) was 9.7 per 1000 patient-years, with a cumulative incidence of 9.1% after 10 years of follow-up. However, this decreased to 5.8% after the year 2000, likely due to the availability and use of other PBC treatments.1

Patients treated with UDCA who achieved a biochemical response had a significantly lower risk of hepatic decompensation.1

Furthermore, regardless of whether an individual had advanced or early disease, a biochemical response was associated with a lower incidence of hepatic events.1

These data show the importance of identifying biochemical response vs nonresponse to UDCA treatment. In those who are nonresponders to UDCA, we now have effective second-line therapies available that can be used in combination with UDCA to achieve biochemical response in additional people.

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A woman with newly diagnosed, higher-risk PBC is starting UDCA. When would you recommend that she first be assessed for UDCA response?

A woman with PBC has been taking and tolerating UDCA for 6 months. At diagnosis, her baseline ALP was 3.0 times the ULN. At today’s visit, her ALP is 2.5 times the ULN, and her bilirubin remains normal. What would you recommend?

I can evaluate biochemical response to first-line PBC therapy based on optimal timing and criteria

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