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Updated: Jul 4, 2026
Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
Published on: April 22, 2019
Outcomes of Epidermal Growth Factor Receptor Inhibitors in Locally Advanced and Metastatic Anal Squamous Cell
Muhammad Shaheer Mannan1, Adeena Musheer2, Ahmad Basharat1
1Department of Internal Medicine, Marshfield Clinic - Sanford Health, Marshfield, WI, 54449, USA.
Background:
Anal squamous cell carcinoma (ASCC) is a rare malignancy with rising incidence and limited systemic options in recurrent or metastatic disease. Although epidermal growth factor receptor (EGFR) inhibitors show activity in other squamous cancers, their role in ASCC remains unclear due to fragmented evidence. This systematic review and meta-analysis evaluated the efficacy and safety of EGFR inhibitors in adults with locally advanced or metastatic ASCC.
Methods:
PubMed/MEDLINE, Cochrane CENTRAL, and ClinicalTrials.gov were searched from inception to December 2025 for studies of EGFR inhibitors in ASCC. Eligible designs included randomized trials, phase II studies, prospective or retrospective cohorts, and case series. Proportional meta-analyses were performed using fixed- or random-effects models according to heterogeneity.
Results:
Twelve studies including 404 patients were analyzed. Median age ranged from 47 to 65 years, and both locally advanced and metastatic settings were represented. The EGFR inhibitors cetuximab and panitumumab were administered with chemoradiotherapy in locally advanced disease or with systemic therapy in metastatic or refractory settings. The pooled objective response rate was 52% (95% CI: 36-68; I²=84.2%, τ²=0.0518, p < 0.0001), with a complete response rate of 37% (95% CI: 15-61; I²=94.0%, τ²=0.1287, p < 0.0001). Median overall survival was 11.9 months and progression-free survival 4.5 months. Grade ≥ 3 adverse events occurred in 56% of patients; treatment discontinuation was infrequent (8%), and no treatment-related deaths were reported.
Conclusion:
EGFR inhibitors show a reproducible signal of activity in locally advanced and metastatic ASCC with manageable toxicity, though evidence is limited by nonrandomized designs and heterogeneity; prospective, biomarker-driven studies are needed.
Insights
Epidermal growth factor receptor (EGFR) inhibitors demonstrate activity in anal squamous cell carcinoma (ASCC), showing a 52% response rate. While manageable, further biomarker-driven studies are needed for this rare cancer.
Area of Science:
- Oncology
- Medical Research
Background:
- Anal squamous cell carcinoma (ASCC) is a rare cancer with increasing incidence.
- Limited systemic treatment options exist for recurrent or metastatic ASCC.
- The efficacy of epidermal growth factor receptor (EGFR) inhibitors in ASCC is not well-established.
Purpose of the Study:
- To systematically review and meta-analyze the efficacy and safety of EGFR inhibitors in adults with ASCC.
- To evaluate treatment response rates, survival outcomes, and adverse events associated with EGFR inhibitors in ASCC.
Main Methods:
- A comprehensive search of PubMed/MEDLINE, Cochrane CENTRAL, and ClinicalTrials.gov was conducted.
- Included studies comprised randomized trials, phase II studies, cohorts, and case series.
- Proportional meta-analyses were performed to synthesize data from 12 studies involving 404 patients.
Main Results:
- The pooled objective response rate (ORR) for EGFR inhibitors was 52% (95% CI: 36-68), with a complete response rate of 37% (95% CI: 15-61).
- Median overall survival was 11.9 months and progression-free survival was 4.5 months.
- Grade ≥3 adverse events occurred in 56% of patients, with low rates of discontinuation (8%) and no treatment-related deaths.
Conclusions:
- EGFR inhibitors show a promising signal of activity in locally advanced and metastatic ASCC.
- Toxicity appears manageable, but evidence is limited by study design and heterogeneity.
- Prospective, biomarker-driven trials are essential to further define the role of EGFR inhibitors in ASCC treatment.
