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Research Article
12 August 2026

Real-world treatment patterns and clinical outcomes of patients with primary biliary cholangitis in the United States

Authors: Nisreen Shamseddine https://orcid.org/0000-0001-8068-7786, Hongbo Yang https://orcid.org/0000-0003-2741-8418, Su Zhang https://orcid.org/0000-0002-2056-8838, Dongni Ye https://orcid.org/0000-0002-0355-0636, Shravanthi Seshasayee https://orcid.org/0000-0002-1692-7121, Jingyi Chen https://orcid.org/0009-0007-0141-2241, Sonal Kumar https://orcid.org/0000-0002-6873-6933, and Kris V Kowdley https://orcid.org/0000-0002-8553-3652 [email protected]Author Info & Affiliations
Volume 15, Number 9
https://doi.org/10.57264/cer-2025-0172
PDFFormats

Abstract

Background & aim: Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease that can lead to increased morbidity and mortality. This study described real-world treatment patterns and clinical outcomes by line of treatment among patients with PBC in the US. Materials & methods: Adults (≥18 years) diagnosed with PBC on or after 1 January 17 were identified in the IQVIA PharMetrics® Plus database and grouped into newly diagnosed, first-line (1L) and second-line or more (2L+) cohorts. Index date was initial PBC diagnosis or initiation of 1L or 2L therapy; follow-up continued until the earliest of end of continuous enrollment, death or data end. Time to treatment initiation, treatment discontinuation and negative clinical outcomes were assessed with Kaplan–Meier analysis. Results: The newly diagnosed, 1L and 2L+ cohorts included 1748, 1659 and 181 patients, respectively (average age at index: 52.7–54.5 years; female: 84.2–89.0%). Of the newly diagnosed cohort, 34.8% did not initiate PBC treatment within 1.5 years post-diagnosis. In the 1L cohort, median time from diagnosis to 1L initiation was 1.2 months; median time from 1L initiation to 1L discontinuation/2L initiation was nearly 5 years. In the 2L+ cohort, median time from 2L initiation to 2L discontinuation was approximately 4 years. In the untreated, 1L, and 2L+ cohorts, 18.9%, 14.4% and 19.3% of patients developed ≥1 negative clinical outcome post-index (usually cirrhosis). Conclusion: Results of this US population-based study demonstrate a potential unmet need for early intervention and effective treatment options for patients with PBC, as one in three patients with PBC remain untreated years after diagnosis.

Plain language summary: How primary biliary cholangitis is treated in clinical practice in the US and patient outcomes

What is this article about?

Primary biliary cholangitis (PBC) is a rare liver disease that gradually worsens over time and can be potentially life-threatening. People with PBC may try multiple medications to manage their disease and/or treat their symptoms because the first one does not always work.

What methodology is described?

In this article, we describe a study that looked at how patients with PBC were treated with medication in the US and what the results of these treatments were.

What were the results?

The majority (65.2%) of patients with newly diagnosed PBC started at least one medication for PBC. Almost half (48.0%) of patients with newly diagnosed PBC started a medication within 1 month of diagnosis and nearly half of patients stopped their first medication within 4 years of starting it. About one in three newly diagnosed patients did not start any PBC medication by one and a half years after their diagnosis. Additionally, about one in five patients had a negative health complication, regardless of the treatment they started. Usually, this meant their disease got worse and led to severe liver scarring, known as cirrhosis.

What do the results mean?

This study shows that patients with PBC may not be receiving timely or effective treatment with medications for PBC. More awareness of PBC and its treatment options is important to help connect patients with the right care at the right time.

Supplementary Material

File (supplementary materials.docx)

References

Papers of special note have been highlighted as: • of interest; •• of considerable interest
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