Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Multicenter Study
. 2025 May;45(5):e70073.
doi: 10.1111/liv.70073.

Ursodeoxycholic Acid Alone Is Effective and Safe to Treat Cholestatic Checkpoint Inhibitor-Induced Liver Injury

Lina Hountondji  1   2 , Stéphanie Faure  1 , Pascale Palassin  3 , Georges-Philippe Pageaux  1   2 , Alexandre Thibault Jacques Maria  2   4   5 , Lucy Meunier  1   2   5   6
Affiliations
Multicenter Study

Ursodeoxycholic Acid Alone Is Effective and Safe to Treat Cholestatic Checkpoint Inhibitor-Induced Liver Injury

Lina Hountondji et al. Liver Int. 2025 May.

Abstract

Introduction: Checkpoint Inhibitor-induced Liver Injury (CHILI) is a frequent complication of immune checkpoint inhibitors (ICIs). Corticosteroids are the standard treatment but have many limitations. Ursodeoxycholic acid (UDCA) offers an alternative for managing cholestatic CHILI, but its efficacy remains underexplored.

Methods: A multicenter retrospective study included 27 patients treated with first-line UDCA monotherapy. Data were collected from diagnosis to week 52, assessing liver enzyme improvement, recurrence, and outcomes.

Results: UDCA alone achieved improvement in 81.5% of patients, with an average response time of 39.3 days. Among patients, 77.8% had severe CHILI (CTCAE grade ≥ 3). Macroscopic bile duct injury was observed in 37%, associated with higher recurrence rates (75%, p < 0.001). Recurrent CHILI led to chronic CHILI in all cases. ICI rechallenge was conducted in 52% of patients, with only 23% experiencing relapse.

Conclusion: UDCA monotherapy appears effective for cholestatic CHILI, presenting a viable alternative to corticosteroids. Further prospective studies are warranted.

Keywords: DILI; Ursodeoxycholic acid; cholangitis; immune checkpoint inhibitors.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Evolution of patients treated with UDCA in first line.
See this image and copyright information in PMC

References

    1. Cunningham M., Gupta R., and Butler M., “Checkpoint Inhibitor Hepatotoxicity: Pathogenesis and Management,” Hepatology (Baltimore, Md.) 79, no. 1 (2024): 198–212. - PubMed
    1. De Martin E., Michot J. M., Papouin B., et al., “Characterization of Liver Injury Induced by Cancer Immunotherapy Using Immune Checkpoint Inhibitors,” Journal of Hepatology 68, no. 6 (2018): 1181–1190. - PubMed
    1. Haanen J., Obeid M., Spain L., et al., “Management of Toxicities From Immunotherapy: ESMO Clinical Practice Guideline for Diagnosis, Treatment and Follow‐Up,” Annals of Oncology: Official Journal of the European Society for Medical Oncology 33, no. 12 (2022): 1217–1238. - PubMed
    1. Arbour K. C., Mezquita L., Long N., et al., “Impact of Baseline Steroids on Efficacy of Programmed Cell Death‐1 and Programmed Death‐Ligand 1 Blockade in Patients With Non‐Small‐Cell Lung Cancer,” Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology 36, no. 28 (2018): 2872–2878. - PubMed
    1. Pan E. Y., Merl M. Y., and Lin K., “The Impact of Corticosteroid Use During Anti‐PD1 Treatment,” Journal of Oncology Pharmacy Practice: Official Publication of the International Society of Oncology Pharmacy Practitioners 26, no. 4 (2020): 814–822. - PubMed

Publication types

Substances