Cetuximab-based treatment of metastatic anal cancer: correlation of response with KRAS mutational status
Related Experiment Video
Updated: Jun 18, 2026

Endobronchial Ultrasound-guided Intratumoral Injection of Cisplatin for the Treatment of Isolated Mediastinal Recurrence of Lung Cancer
Published on: February 12, 2017
Nadine Lukan1, Philipp Ströbel, Andreas Willer
1III. Medizinische Klinik, Universitatsmedizin Mannheim, Mannheim, Deutschland.
Background:
No standard chemotherapy regimen can be defined for patients with metastatic squamous cell carcinoma of the anus due to the low incidence of this disease and the high cure rate of localized tumors. Anal cancers universally express the epidermal growth factor receptor (EGFR) and KRAS mutations have not been reported in anal cancer thus far.
Methods:
We report on 7 patients with metastatic anal cancer treated with cetuximab - a chimeric antibody against EGFR - on a compassionate use basis along with the results of KRAS mutational analysis.
Results:
Marked tumor shrinkage was noted in several patients using cetuximab monotherapy or cetuximab/irinotecan combination as first or subsequent treatment line (usually after failure of cisplatin-based regimens). Two out of seven patients harbored KRAS mutations. Both patients had progressive disease receiving cetuximab, while the remaining 5 patients had either a partial remission (n = 3), a minor remission (n = 1) or no change lasting > or =6 months after previous rapid tumor progression.
Conclusion:
Cetuximab-based treatment appears to be a valuable treatment option for patients with metastatic KRAS wild-type anal cancer after failure of or as an alternative to cisplatin/5-fluorouracil-based therapy.
Insights
Cetuximab shows promise for metastatic anal cancer, particularly in patients without KRAS mutations. This epidermal growth factor receptor (EGFR) inhibitor offers a valuable treatment option when standard therapies fail.
Area of Science:
- Oncology
- Medical Research
Background:
- Metastatic squamous cell carcinoma of the anus lacks standard chemotherapy due to low incidence and high localized cure rates.
- Anal cancers consistently express the epidermal growth factor receptor (EGFR).
- KRAS mutations have not been previously identified in anal cancer.
Observation:
- Seven patients with metastatic anal cancer received cetuximab, an EGFR-targeting antibody, on a compassionate use basis.
- KRAS mutational analysis was performed concurrently with treatment.
- Treatment response was evaluated for cetuximab monotherapy and combination therapy with irinotecan.
Findings:
- Cetuximab treatment, alone or with irinotecan, resulted in significant tumor shrinkage in several patients.
- Two of seven patients had KRAS mutations and experienced progressive disease on cetuximab.
- Five patients without KRAS mutations achieved partial remission (3), minor remission (1), or stable disease (>6 months).
Implications:
- Cetuximab-based therapy is a potential treatment for metastatic anal cancer, especially in patients with KRAS wild-type tumors.
- This approach offers an alternative or subsequent option after failure of cisplatin/5-fluorouracil regimens.
- Further research into EGFR-targeted therapies for anal cancer is warranted.
