Capecitabine induces rapid, sustained response in two patients with extensive oral verrucous carcinoma

Purpose: Oral verrucous carcinoma (VC) has been traditionally treated with surgery or radiation with frequent recurrences and significant morbidity. We describe rapid and dramatic response to oral capecitabine in two patients with advanced refractory VC. Experimental Design: VC is a rare tumor of the oral cavity. It does not metastasize but over time causes morbidity and mortality through local invasion. Radiation and surgery have been the main treatment modalities but are plagued by mutilating effects, recurrences, and possibly malignant degeneration in some cases. To date, no effective chemotherapy regimens have been described in the international literature. The clinical records of two elderly females with extensive oral VC are described. Both patients were prescribed one cycle of capecitabine, 1000 mg bid for 14 days. Response was documented by photography in one patient. Immunohistochemical evaluation of three 5-fluorouracil metabolizing enzymes on pretreatment biopsies from both patients was also performed. A review of the literature with emphasis on treatment of oral VC is presented in view of our findings. Results: Examination of the oral cavity before treatment revealed extensive involvement with an evenly spreading, exophytic, warty, whitish lesion in both patients. Microscopic examination of H&E-stained slides from biopsies of these lesions confirmed the clinical suspicion of VC. Both patients underwent treatment with oral capecitabine for one cycle (2 weeks on/1 week off) at a reduced dose of 1000 mg, p.o., bid. Both had a dramatic response with near complete resolution of their lesions within 3 weeks of initiating therapy. A durable partial response was documented at 1 year in the first patient and 6 months in the second. Immunohistochemical evaluation of pretreatment biopsies from both patients revealed a high level of expression of thymidine phosphorylase, a key enzyme in the metabolism of capecitabine. Conclusions: Oral VC is a rare entity with a progressive course over years and limited options in terms of treatment. Preliminary observations in two elderly patients demonstrate that capecitabine, an oral fluoropyrimidine, is well tolerated and may induce rapid, clinically significant response. Although not curative, it may provide a cost-effective alternative for elderly patients with a significant improvement in their quality of life.