Microbiome meets immunotherapy: unlocking the hidden predictors of immune checkpoint inhibitors
Immune checkpoint inhibitors (ICIs) have revolutionized cancer immunotherapy, with the microbiome significantly influencing treatment outcomes. Analysis of 4663 studies (2014.01-2024.10) identified 71 eligible randomized controlled trials (RCTs) and cohort studies (41 viral, 30 bacterial). Analyses included subgroup assessments by cancer type, microbial taxa, and ICI regimens. Among 4663 identified studies, 71 met inclusion criteria (41 viral, 30 bacterial). Viral status, particularly hepatitis B virus (HBV) and human papillomavirus (HPV), significantly associated with ORR and DCR. Bacterial enrichment correlated with improved survival in hepatobiliary (OS: HR = 4.33, 95%CI: 2.20-8.50) and lung cancers (PFS: HR = 1.70, 95%CI: 1.04-2.78). Multi-microbiome models demonstrated superior outcome prediction, with microbial diversity correlating with improved PFS (HR = 0.64, 95%CI: 0.42-0.98). Viral status showed cancer-specific associations with SAEs. The microbiome serves as a valuable predictor of ICI outcomes. Future studies should emphasize large-scale RCTs, standardized assessment methods, and host-microbiome interactions.
Authors
- Haijun Yu (ORCID: https://orcid.org/0000-0002-3398-0880)
- X. Z. Cui (ORCID: https://orcid.org/0000-0001-8242-8964)
- Ting Wei (ORCID: https://orcid.org/0000-0001-9008-0339)
- Weiming Mou (ORCID: https://orcid.org/0009-0007-1089-6516)
- Kailai Li (ORCID: https://orcid.org/0000-0002-2368-3217)
- Aimin Jiang (ORCID: https://orcid.org/0000-0002-4092-342X)
- Anqi Lin (ORCID: https://orcid.org/0000-0002-6324-0410)
- Lihaoyun Huang (ORCID: https://orcid.org/0000-0003-3833-9117)
- Chunyan Zhang (ORCID: https://orcid.org/0000-0002-9182-0201)
- Jian Zhang (ORCID: https://orcid.org/0000-0002-7337-5124)
- Peng Luo (ORCID: https://orcid.org/0000-0002-8215-2045)
- Cui Cui
- Lingxuan Zhu
Institutions
- Sun Yat-sen University (CN)
- Chinese University of Hong Kong (HK)
- Second Military Medical University (CN)
- Shanghai Jiao Tong University (CN)
- Zhujiang Hospital (CN)
- The First People’s Hospital of Lianyungang (CN)
- Shanghai First People's Hospital (CN)
- Sun Yat-sen University Cancer Center (CN)
- State Key Laboratory of Oncology in South China
- Southern Medical University (CN)
- University of Hong Kong (HK)
Publication Details
- Journal
- npj Biofilms and Microbiomes
- Published
- 2025-09-02
- DOI
- https://doi.org/10.1038/s41522-025-00819-2
- Citations
- 12
- Primary Topic
- Cancer Immunotherapy and Biomarkers
- Type
- article
- Field-Weighted Citation Impact
- 12.54
Funders
- National Natural Science Foundation of China