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Pembrolizumab in Patients with Refractory Cutaneous Squamous Cell Carcinoma: A Phase II Trial
Renata Ferrarotto1, Luana G Sousa2, Yun Qing3
1Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. rferrarotto@mdanderson.org.
Introduction:
Patients with advanced cutaneous squamous cell carcinoma (CSCC) have a poor prognosis. Blocking the PD-1-PD-L1 axis has shown promising activity in this patient population. We assessed the safety and antitumor activity of PD-1 inhibitor pembrolizumab in patients with refractory advanced CSCC.
Methods:
This was a prespecified subgroup analysis of patients with advanced CSCC who enrolled in an open-label, phase II clinical trial for pembrolizumab in patients with refractory rare cancers during 2016-2018. Patients received pembrolizumab 200 mg intravenously every 21 days until progressive disease, unacceptable adverse event, or completion of 24 months of treatment. The primary endpoint was nonprogression rate (NPR) at 27 weeks; secondary endpoints included safety, objective response rate (ORR) per irRECIST, clinical benefit rate (CBR), progression-free survival, and overall survival.
Results:
Twenty patients with refractory CSCC enrolled; 19 were evaluable for efficacy. Median follow-up time was 44.1 months. The NPR at 27 weeks was 37% (95% CI 0.16-0.62). Three patients had a complete response (CR), three had a partial response, and one had stable disease, for an ORR of 32% and a CBR of 37%; median duration of response was 27.3 months. All three patients with a CR remained free of recurrence at the time of writing. Severe treatment-related adverse events (grade ≥ 3) occurred in 10% of patients (2/20). PD-L1 expression was not correlated with response to pembrolizumab.
Conclusion:
A long-term follow-up confirms pembrolizumab's antitumor activity and safety profile in patients with refractory CSCC. Patients with a CR may experience cure.
Trial Registration:
ClinicalTrials.gov, NCT02721732, Registered March 29, 2016.
Insights
Pembrolizumab shows significant antitumor activity and a favorable safety profile in patients with advanced cutaneous squamous cell carcinoma (CSCC). Long-term follow-up suggests that complete responses may lead to a cure for some patients.
Area of Science:
- Oncology
- Immunotherapy
- Dermatology
Background:
- Advanced cutaneous squamous cell carcinoma (CSCC) presents a poor prognosis.
- The PD-1/PD-L1 axis blockade demonstrates promising therapeutic potential in CSCC.
- Pembrolizumab is an immune checkpoint inhibitor targeting the PD-1/PD-L1 pathway.
Purpose of the Study:
- To assess the safety and antitumor activity of the PD-1 inhibitor pembrolizumab.
- To evaluate pembrolizumab in patients with refractory advanced CSCC.
- To analyze long-term outcomes including response rates and survival.
Main Methods:
- A subgroup analysis of a phase II clinical trial involving patients with advanced CSCC.
- Patients received pembrolizumab 200 mg intravenously every 21 days.
- Primary endpoint: nonprogression rate (NPR) at 27 weeks; secondary endpoints: safety, objective response rate (ORR), clinical benefit rate (CBR), progression-free survival, and overall survival.
Main Results:
- Twenty patients with refractory CSCC were enrolled; 19 were evaluable for efficacy.
- The 27-week NPR was 37%. The ORR was 32% and CBR was 37%.
- Three patients achieved a complete response (CR), with no recurrence at the time of writing. Severe adverse events occurred in 10% of patients. PD-L1 expression did not correlate with response.
Conclusions:
- Long-term follow-up confirms pembrolizumab's antitumor activity and safety in refractory advanced CSCC.
- Pembrolizumab offers a viable treatment option for patients with advanced CSCC.
- Complete responses achieved with pembrolizumab may indicate potential for cure in select patients.
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