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Updated: Mar 7, 2026

Through the Looking Glass: Time-lapse Microscopy and Longitudinal Tracking of Single Cells to Study Anti-cancer Therapeutics
Published on: May 14, 2016
Checkpoint inhibition for advanced mucosal melanoma
Julia Thierauf1, Johannes A Veit1, Jochen Hess2
1Department of Otorhinolaryngology, Head and Neck Surgery, University Medical Center Ulm, Frauensteige 12, 89075 Ulm, Germany.
Background:
Whereas anti-PD-1 therapy has demonstrated a significant and durable response against advanced cutaneous melanoma, conventional chemotherapies have shown only minor benefit against advanced mucosal melanoma.
Objectives:
To investigate the efficacy of anti-PD-1 therapy in a small cohort of patients with mucosal melanoma of the head and neck.
Materials & Methods:
We analysed five patients with mucosal melanoma of the head and neck who received nivolumab or pembrolizumab, at an advanced stage. Expression of PD-L1 and PD-1 in all tumour samples was evaluated immunohistochemically.
Results:
All patients received at least two cycles of nivolumab or pembrolizumab. The most severe adverse events were categorised as CTCAE (common terminology criteria for adverse events) Grade 2. All patients showed progressive disease after restaging at three and six months, and no partial or complete response was observed. Immunohistochemical staining demonstrated PD-L1 expression in less than 5% of tumour cells.
Conclusion:
Systemic therapy with either nivolumab or pembrolizumab showed no clinical response, however, tumour progression was identified in all patients using Response Evaluation Criteria In Solid Tumors (RECIST) v1.1 and immune-related response criteria (irRC) to evaluate tumour response.
Insights
Anti-PD-1 therapy (programmed cell death protein 1) showed no clinical response in advanced mucosal melanoma of the head and neck. All patients experienced tumor progression despite treatment with nivolumab or pembrolizumab.
Area of Science:
- Oncology
- Immunotherapy
- Melanoma Research
Background:
- Advanced cutaneous melanoma responds well to anti-PD-1 therapy.
- Conventional chemotherapies offer limited benefit for advanced mucosal melanoma.
Purpose of the Study:
- Investigate the efficacy of anti-PD-1 therapy in patients with head and neck mucosal melanoma.
- Evaluate nivolumab and pembrolizumab in a small patient cohort.
Main Methods:
- Analysis of five patients with advanced mucosal melanoma of the head and neck.
- Treatment with nivolumab or pembrolizumab.
- Immunohistochemical evaluation of PD-L1 and PD-1 expression in tumor samples.
Main Results:
- All patients received at least two cycles of anti-PD-1 therapy.
- No partial or complete response observed; all patients showed progressive disease.
- PD-L1 expression was less than 5% in tumor cells.
Conclusions:
- Systemic nivolumab or pembrolizumab demonstrated no clinical response in this cohort.
- Tumor progression was confirmed by RECIST v1.1 and irRC criteria.
- Anti-PD-1 therapy may not be effective for advanced mucosal melanoma of the head and neck.

