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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
Nonoperative Management of Locally Advanced Resectable Cutaneous Squamous Cell Cancer of the Head and Neck with PD-1
Fergal G Kavanagh1, Ryan S Instrum1, Lillian A Boe2
1Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, New York.
Purpose:
Neoadjuvant PD-1 blockade has shown marked efficacy before surgery in patients with head and neck cutaneous squamous cell cancinoma (CSCC). However, the potential for PD-1 blockade as a definitive, nonoperative therapy remains uncertain.
Experimental Design:
We conducted a single-institution retrospective study (2018 to 2023) of patients with locally advanced resectable stage III/IV CSCC treated with cemiplimab. Patients received neoadjuvant cemiplimab followed by surgery or cemiplimab monotherapy without surgery. The primary endpoint was the objective radiologic response rate (assessed using immune Response Evaluation Criteria in Solid Tumors) and clinical response rate; the secondary endpoints included disease-specific survival (DSS), progression-free probability (PFP), histopathologic response, and exploratory genomic biomarkers.
Results:
Fifty-one patients received cemiplimab monotherapy alone (median age: 78.8 years), and 21 received neoadjuvant cemiplimab followed by surgery (median age: 73 years). The 2-year DSS and PFP for cemiplimab monotherapy were 90% [95% confidence interval (CI), 80%-100%] and 82% (95% CI, 72%-94%), respectively. The 2-year DSS and PFP for neoadjuvant cemiplimab with surgery were 95% (95% CI, 86%-100%) and 78% (95% CI, 62%-100%), respectively. Tumor-infiltrating lymphocytes (TIL) were higher in patients with complete response (CR) or partial response (PR) than in patients with progressive disease (PD) or stable disease (P = 0.005, q = 0.026), with absent TILs more frequent in nonresponders. Tumor mutational burden was higher in CR (55.7) and PR (14.9) than in PD (4.9; P = 0.02, q = 0.04).
Conclusions:
In this retrospective, nonrandomized cohort, definitive-intent cemiplimab monotherapy was associated with durable disease control in selected patients with locally advanced resectable head and neck CSCC. This study provides a promising real-world organ preservation experience for patients with advanced head and neck CSCC treated with cemiplimab monotherapy that does not compromise oncologic outcomes.
Insights
Definitive-intent cemiplimab monotherapy showed durable disease control in advanced head and neck cutaneous squamous cell cancer (CSCC). This organ-preserving approach did not compromise oncologic outcomes in selected patients.
Area of Science:
- Oncology
- Immunotherapy
- Dermatology
Background:
- Neoadjuvant PD-1 blockade demonstrates efficacy in head and neck cutaneous squamous cell cancer (CSCC) prior to surgery.
- The potential for PD-1 blockade as a non-operative, definitive therapy for CSCC remains under investigation.
Purpose of the Study:
- To evaluate the efficacy of cemiplimab monotherapy as a definitive treatment for locally advanced, resectable CSCC.
- To compare outcomes of cemiplimab monotherapy versus neoadjuvant cemiplimab followed by surgery in head and neck CSCC patients.
Main Methods:
- Retrospective analysis of 72 patients with stage III/IV CSCC treated with cemiplimab (2018-2023).
- Patients received either cemiplimab monotherapy or neoadjuvant cemiplimab followed by surgery.
- Primary endpoint: objective radiological and clinical response rate. Secondary endpoints: disease-specific survival (DSS), progression-free probability (PFP), and genomic biomarkers.
Main Results:
- The 2-year DSS and PFP for cemiplimab monotherapy were 90% and 82%, respectively.
- The 2-year DSS and PFP for neoadjuvant cemiplimab with surgery were 95% and 78%, respectively.
- Higher tumor-infiltrating lymphocytes (TILs) and tumor mutational burden correlated with better response rates.
Conclusions:
- Definitive-intent cemiplimab monotherapy achieved durable disease control in selected patients with locally advanced head and neck CSCC.
- This organ-preserving strategy offers a promising alternative for advanced CSCC without compromising oncologic outcomes.
