Neoadjuvant chemotherapy prior to definitive surgery has been used widely for locally advanced oral squamous cell carcinoma (OSCC). We evaluated neoadjuvant erlotinib with platinum-docetaxel versus placebo with platinum-docetaxel in patients with stage III–IVB OSCC.

Patients and Methods:

Patients with newly diagnosed stage III, IVA, and IVB (American Joint Committee on Cancer 7th) OSCC amenable to surgical resection were included. Patients were randomized to receive up to 3 cycles of chemotherapy with concurrent erlotinib or placebo, followed by surgery. The primary endpoint was major pathologic response (MPR) rate; secondary endpoints included safety, overall survival (OS), and progression-free survival (PFS).


Fifty-two patients received at least 1 cycle of treatment and 47 were evaluable with surgical resection. MPR rate was not different between erlotinib (30%, 7/23) and placebo arms (41.7%, 10/24; P = 0.55). At median follow up of 26.5 months, there was no difference on OS or PFS between groups. Patients who received erlotinib with chemotherapy and achieved MPR (n = 7) had no recurrence. The treatment-related adverse event rates were not different between the two groups (96% vs. 96%). However, rash, mostly low grade, was more common in the erlotinib arm (79% vs. 50%). Transcriptomic analysis in the pretreatment samples indicated that genes in protein glycosylation and Wnt signaling pathways were associated with benefit in those treated with erlotinib plus chemotherapy.


The addition of erlotinib to platinum-taxane chemotherapy was well tolerated but did not induce higher rates of MPR or PFS or OS survival benefit. Patients who received chemotherapy with erlotinib and achieved major pathologic responses had excellent clinical outcome.

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