The present study aimed to identify significant correlations between gene expression and
chemotherapy response to
5-fluorouracil (5-FU)/
cisplatin in
head and neck squamous cell carcinoma (
HNSCC), and to identify patients who would benefit from
induction chemotherapy for both organ preservation and survival. A total of 64 patients who underwent radical treatment for
HNSCC were enrolled. All patients received
induction chemotherapy with 5-FU/
cisplatin and
tumor responses were evaluated. Pretreatment biopsy specimens from all patients were assayed for
mRNA expression of
thymidylate synthase,
dihydropyrimidine dehydrogenase (DPD),
orotate phosphoribosyltransferase, tymidine
phosphorylase,
glutathione S-transferase-pi, p53, RB Transcriptional
Corepressor 1,
B-cell lymphoma 2 (Bcl-2), Bcl-xL,
E2F Transcription Factor 1,
epidermal growth factor receptor,
human epidermal growth factor receptor 2,
phosphoinositide 3-kinase,
phosphatase and
tensin homolog,
vascular endothelial growth factor (
VEGF),
cyclooxygenase-2, XPA, DNA Damage Recognition And Repair Factor, excision repair cross-complementing 1 (ERCC1), multidrug resistance gene 1 (MDR1),
multidrug resistance-associated protein 1,
equilibrative nucleoside transporter 1 and β-
tubulin by reverse transcription-quantitative polymerase chain reaction, and the association between the expression levels of these genes and patient response to
chemotherapy was determined. The complete response (CR) group and non-CR group for
induction chemotherapy comprised 32.8 and 67.2% of patients, respectively. The 5-year overall survival rate was significantly higher for the CR group (95%) compared with the non-CR group (57%). According to univariate analysis,
chemotherapy response was associated with T-class and
mRNA expressions of DPD, ERCC1, XPA, p53, Bcl-2,
VEGF and MDR1. Multivariate analysis identified ERCC1 expression and T-class as significant predictors of response to
chemotherapy, indicating that
a DNA-repair pathway and apoptosis pathway are pivotal mechanisms governing response to
chemotherapy. The findings suggest that ERCC1 expression could be a predictive
biomarker for
chemotherapy response to 5-FU/
cisplatin in
HNSCC. Assessing
mRNA expression is a standard method for these studies, however further investigations examining polymorphisms and mutations in addition to apoptotic responses are required to determine target gene activation in
HNSCC.