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Pathologic Response and Survival After Neoadjuvant Immunotherapy for Resectable Mucosal HNSCC: A Systematic Review
Eric V Mastrolonardo1,2, Emma De Ravin1,2, Praneet C Kaki1,2
1Department of Otolaryngology - Head and Neck Surgery, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania.
JAMA Otolaryngology-- Head & Neck Surgery
|December 18, 2025
Summary
Pathologic treatment response, including partial and major response, is linked to improved disease-free survival (DFS) in head and neck cancer patients receiving neoadjuvant immune checkpoint inhibition (ICI). This finding supports its use as a surrogate for DFS in mucosal HNSCC.
Area of Science:
- Oncology
- Immunotherapy
- Head and Neck Cancer Research
Background:
- Neoadjuvant immune checkpoint inhibition (ICI) is increasingly used for mucosal head and neck squamous cell carcinoma (HNSCC).
- Pathologic treatment response is often used as an endpoint in clinical trials, but its association with survival outcomes is not fully established.
Purpose of the Study:
- To systematically evaluate the relationship between pathologic treatment response and overall survival (OS) and disease-free survival (DFS) after neoadjuvant ICI in mucosal HNSCC.
- To determine if pathologic treatment response can serve as a meaningful surrogate endpoint for survival.
Main Methods:
- A systematic literature search was conducted across major databases (PubMed, Ovid Medline, Embase, CINAHL, Cochrane) from 2000 to 2025.
- Included were peer-reviewed studies of neoadjuvant ICI for mucosal HNSCC in adults, reporting pathologic response and survival data (OS/DFS).
- A meta-analysis of 11 trials (451 patients, 368 in meta-analysis) was performed using random-effects models to calculate hazard ratios (HRs) for DFS and OS.
Main Results:
- Partial pathologic response (PPR; ≤50% residual tumor) and major pathologic response (MPR; ≤10% residual tumor) were both significantly associated with improved DFS up to 2 years (HRs 0.53 and 0.34, respectively).
- Neither PPR nor MPR demonstrated a significant association with improved overall survival (OS).
- Nine of the 11 included studies were assessed as having a low risk of bias.
Conclusions:
- Pathologic treatment response, specifically PPR and MPR, is associated with enhanced DFS in patients with resectable mucosal HNSCC treated with neoadjuvant ICI.
- These findings support the utility of pathologic treatment response as a surrogate endpoint for DFS in this patient population.
- Further research may refine the role of pathologic response in predicting long-term outcomes for HNSCC treated with immunotherapy.
