Lucy Mathew, NP: Preventing PBC Progression with Timely Diagnosis, Treatment

Primary biliary cholangitis (PBC) has been a focal point in hepatology in recent months, owing largely to the notable expansion of the treatment armamentarium with the US Food and Drug Administration approvals of seladelpar (Livdelzi) and elafibranor (Iqirvo).^1,2 With more treatment options available than ever, timely diagnosis is essential for improving patient outcomes.

In a session at the 2024 annual Gastroenterology and Hepatology Advanced Practice Providers (GHAPP) conference in National Harbor, Maryland, Lucy Mathew, NP, a nurse practitioner at Cedars-Sinai, reviewed how to identify patients with PBC.

“Early identification of PBC is extremely important because if not treated, PBC can lead to liver failure in a very short period of time,” Mathew told HCPLive, describing how patients with an aggressive form of the disease can progress to liver failure in 5-10 years. “It is important that we identify early and treat them to prevent progression of the disease. [PBC] is not a curable disease, but we can slow down the progression.”

Timely identification is important but often delayed. Mathew cited the role a lack of PBC education and awareness play in diagnostic delays. However, she noted many patients with PBC present with pruritus, leading providers to believe they are facing a dermatological issue rather than a hepatic one. In many cases, patients do not present with any symptoms at all, further complicating diagnosis.

Additionally, Mathew pointed out PBC is frequently diagnosed through a blood test called anti-mitochondrial antibody (AMA) and explained that this is not a routine test done in the clinic. Although PBC can usually be diagnosed with AMA and alkaline phosphatase levels, Mathew said a liver biopsy is needed to diagnose PBC in “rare” situations.

Once diagnosed, patients with PBC are treated with ursodeoxycholic acid (UDCA), the current first-line treatment option for PBC to help improve liver function and delay liver damage. However, as many as 40% of patients with PBC have an incomplete response to UDCA and experience worse clinical outcomes, requiring additional treatment to achieve biochemical response.³

Previously, obeticholic acid (Ocaliva) was the only second-line treatment option available to patients who did not respond to or were unable to tolerate UDCA. Since June 2024, the FDA has granted accelerated approval to 2 peroxisome proliferator-activated receptor (PPAR) agonists for the second-line treatment of PBC, including seladelpar and elafibranor.^1,2

“Now we have more [treatment] options, and the more options we have, the more we can choose from,” Mathew said. “I'm very confident that with the addition of these new drugs, we will have enough options for all our patients where they can have improvement in their alkaline phosphatase and we can slow down the progression. I'm very happy and excited about trying out these new medications and seeing how they go.”

References

1. ^{Brooks, A. FDA Grants Accelerated Approval to Seladelpar (Livdelzi) for Primary Biliary Cholangitis. HCPLive. August 14, 2024. Accessed September 13, 2024. https://www.hcplive.com/view/fda-grants-accelerated-approval-to-seladelpar-livdelzi-for-primary-biliary-cholangitis}
2. ^{Brooks, A. FDA Grants Accelerated Approval to Elafibranor (Iqirvo) for PBC. June 10, 2024. Accessed September 13, 2024. https://www.hcplive.com/view/fda-grants-accelerated-approval-to-elafibranor-iqirvo-for-pbc}
3. ^{Barba Bernal R, Ferrigno B, Medina Morales E, et al. Management of Primary Biliary Cholangitis: Current Treatment and Future Perspectives. Turk J Gastroenterol. 2023;34(2):89-100. doi:10.5152/tjg.2023.22239}