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

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.