A pharmacovigilance data–driven approach to reveal high fatal adverse events following checkpoint immunotherapy

Z Zhen Sun X Xuan Deng (Department of Laboratory Medicine, Huashan Hospital, Fudan University) J Jianglong Guo M Mengsi Liu (Guangdong Cardiovascular Institute) S Siying Wang (State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital, Shanghai Jiao Tong University School of Medicine) J Jian Wang J Jiaojiao Zheng (State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital, Shanghai Jiao Tong University School of Medicine) K Kongyan Niu (State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital, Shanghai Jiao Tong University School of Medicine) W Weiming Shen (College of Chemistry and Molecular Sciences) Z Zhen Ye C Chongming Zheng (Zhejiang Key Laboratory of intelligent Cancer Biomarker Discovery & Translation, Department of Hepatopancreatobiliary Surgery, The First Affiliated Hospital of Wenzhou Medical University) H Hongye Wang (Department of interventional oncology, Renji Hospital, Shanghai Jiao Tong University School of Medicine) F Fan Yang Z Zhouzhou Bao (Shanghai Key Laboratory of Gynecologic Oncology, Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine) K Kui Ming Chan (Department of Biomedical Sciences, City University of Hong Kong) G Gang Chen M Ming Guan (Department of Laboratory Medicine, Huashan Hospital, Fudan University) H Haojie Jin (State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital)

Abstract

Immune checkpoint inhibitors (ICIs) have significantly improved the outcome of cancer treatment, but they also expose most patients to a variety of treatment-related adverse events (AEs). This study introduces a modified pharmacovigilance approach to identify “ICI-related high-mortality AEs”, a subset of treatment-related complications that are disproportionately reported with elevated fatality during ICI therapy. Utilizing large-scale pharmacovigilance data from 148,972 ICI-treated cases in the FDA AEs Reporting System and 142,645 ICI-treated cases in the WHO global VigiBase, we found 63 types of ICI-related high-mortality AE, such as interstitial lung disease, myositis, and hepatic failure, which necessitate heightened clinical vigilance. Patients who experienced these events had significantly higher fatality reporting rates compared to other cases (36.25% vs. 10.66%). These findings were corroborated in the additional clinical datasets to ensure their generalizability. In summary, identifying these ICI-related high-mortality AEs provides crucial insights for developing proactive monitoring strategies and prioritizes specific events for prevention and intervention to mitigate fatality risk.

Article Details

Volume / Issue Vol. 123, Issue 10
Published March 10, 2026
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (18)

Z

Zhen Sun

X

Xuan Deng

Department of Laboratory Medicine, Huashan Hospital, Fudan University

J

Jianglong Guo

M

Mengsi Liu

Guangdong Cardiovascular Institute

S

Siying Wang

State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital, Shanghai Jiao Tong University School of Medicine

J

Jian Wang

J

Jiaojiao Zheng

State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital, Shanghai Jiao Tong University School of Medicine

K

Kongyan Niu

State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital, Shanghai Jiao Tong University School of Medicine

W

Weiming Shen

College of Chemistry and Molecular Sciences

Z

Zhen Ye

C

Chongming Zheng

Zhejiang Key Laboratory of intelligent Cancer Biomarker Discovery & Translation, Department of Hepatopancreatobiliary Surgery, The First Affiliated Hospital of Wenzhou Medical University

H

Hongye Wang

Department of interventional oncology, Renji Hospital, Shanghai Jiao Tong University School of Medicine

F

Fan Yang

Z

Zhouzhou Bao

Shanghai Key Laboratory of Gynecologic Oncology, Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine

K

Kui Ming Chan

Department of Biomedical Sciences, City University of Hong Kong

G

Gang Chen

M

Ming Guan

Department of Laboratory Medicine, Huashan Hospital, Fudan University

H

Haojie Jin

State Key Laboratory of Systems Medicine for Cancer, Shanghai Cancer Institute, Renji Hospital