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Autoimmune Hepatitis Induced by Immune Checkpoint Inhibitors in Adults: A Systematic Review.
Sarita Chonat1, Jonathan Soldera2,3
1Acute Medicine, University of South Wales in Association with Learna Ltd., Cardiff CF37 1DL, UK.
Immune checkpoint inhibitor (ICI)-associated autoimmune hepatitis (AIH)-like liver injury often resolves with corticosteroids, with mycophenolate mofetil as a second-line option. Further research is needed to clarify diagnosis and treatment for this ICI toxicity.
Area of Science:
- Hepatology
- Oncology
- Immunology
Background:
- Immune checkpoint inhibitors (ICIs) have revolutionized cancer treatment but can cause immune-related adverse events, including liver injury.
- Some ICI-induced liver injuries mimic autoimmune hepatitis (AIH), termed AIH-like immune-mediated hepatitis, requiring careful management to balance cancer treatment and liver recovery.
Purpose of the Study:
- To systematically review the clinical characteristics, diagnosis, treatment, and outcomes of patients experiencing AIH-like liver injury due to ICIs.
- To summarize treatment strategies, response rates, and outcomes related to ICI discontinuation and rechallenge in this patient population.
Main Methods:
- A systematic PubMed search was conducted for English-language human studies on AIH or immune-mediated hepatitis following ICI exposure.
- Included studies were case reports, case series, and cohort studies, adhering to PRISMA principles.
- Data were descriptively synthesized due to heterogeneity in study designs and outcome reporting.
Main Results:
- Twenty-two studies involving 195 patients with ICI-associated AIH-like liver injury were analyzed.
- Corticosteroids were effective first-line treatment, achieving recovery in 102 of 105 patients; mycophenolate mofetil was effective for steroid-refractory cases (9/10 response).
- Most patients (133/140) recovered with treatment, and 55 patients were successfully rechallenged with ICIs without recurrent liver toxicity.
Conclusions:
- ICI-associated AIH-like liver injury is a significant immune-related toxicity, with most patients recovering, particularly with corticosteroid therapy.
- Mycophenolate mofetil is a key second-line treatment for refractory cases, but evidence quality is limited by study heterogeneity.
- Future research should standardize diagnostic criteria and outcome reporting to better differentiate AIH-like injury from classical AIH and guide management.
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