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Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
Published on: April 22, 2019
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Pathologic Treatment Effect and Survival in HPV-Negative HNSCC Following Neoadjuvant Nivolumab
Annie E Moroco1, Kathryn Nunes1, Angela Alnemri1
1Department of Otolaryngology-Head and Neck Surgery, Thomas Jefferson University, Philadelphia, Pennsylvania.
JAMA Otolaryngology-- Head & Neck Surgery
|July 31, 2025
Summary
Pathologic treatment effect (pTE) response to neoadjuvant nivolumab in head and neck squamous cell carcinoma (HNSCC) may predict survival. HPV-negative patients showing a pTE response had significantly better disease-free and overall survival.
Area of Science:
- Oncology
- Immunotherapy
- Head and Neck Cancer Research
Background:
- Neoadjuvant immunotherapy, specifically nivolumab, shows promise for head and neck squamous cell carcinoma (HNSCC).
- Pathologic treatment effect (pTE) is a potential measure of treatment response, but its correlation with survival outcomes requires further investigation.
Purpose of the Study:
- To determine if pathologic response to neoadjuvant nivolumab in HNSCC correlates with improved survival outcomes.
- To assess the utility of pTE as a surrogate marker for treatment efficacy in HNSCC.
Main Methods:
- Pooled analysis of two multi-institutional neoadjuvant clinical trials (NCT03238365, NCT03854032) involving patients with resectable HNSCC.
- Patients received neoadjuvant nivolumab with or without additional immunomodulating medications.
- Kaplan-Meier survival analysis was used to compare outcomes between pTE responders and non-responders, with a pTE threshold determined by recursive partitioning analysis.
Main Results:
- A pTE threshold of 57% was identified to distinguish responders from non-responders.
- In HPV-negative HNSCC patients, pathologic responders (pTE >57%) demonstrated significantly improved 3-year disease-free survival (100% vs. 66.8%) and overall survival (100% vs. 73.3%).
- In HPV-positive HNSCC patients, both responders and non-responders exhibited high 3-year disease-free survival rates (90% and 92.4%, respectively).
Conclusions:
- Pathologic response (pTE >57%) to neoadjuvant nivolumab may indicate improved survival outcomes in HPV-negative HNSCC patients.
- Pathologic treatment effect shows potential as a surrogate marker for treatment response in this setting.
- The neoadjuvant immunotherapy strategy is associated with improved survival in HNSCC.
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