Results of a prospective randomized trial comparing neoadjuvant chemotherapy plus radiotherapy with radiotherapy alone in patients with locoregionally advanced nasopharyngeal carcinoma

Purpose: A prospective randomized trial was performed to evaluate the contribution of neoadjuvant chemotherapy in patients with to locoregionally advanced nasopharyngeal carcinoma. Patients and Methods: patients with locoregionally advanced nasopharyngeal carcinoma were treated either with radiotherapy alone (RT group) or neoadjuvant chemotherapy plus radiotherapy (CT/RT group). Neoadjuvant chemotherapy consisting of two to three cycles of cisplatin (100 mg/m(2), day 1), bleomycin (10 mg/m(2), days 1 and 5), and fluorouracil (5-FU; 800 mg/m(2), days 1 through 5, continuous infusion) followed by radiotherapy was given to the CT/RT group. All patients were treated in a uniform fashion by definitive-intent radiation therapy in both groups. Results: between July 1993 and July 1994, 456 patients were entered onto the study, with 228 patients randomised to each treatment arm, and 449 patients (225 in the RT group and 224 in the CT/RT group) were assessable. All 456 patients were included in survival analysis according to the intent-to-treat principle. The 5-year overall survival (OS) rates were 63% for the CT/RT group and 56% for the RT group (P = .11). The median relapse-free survival (RFS) time was 50 months for the RT group and not reached for the CT/RT group. the 5-year RFS rate was 49% for the RT group versus 59% for the CT/RT group (P = .05). The 5-year freedom from local recurrence rate was 82% for the CT/RT group and 74% for the RT group (P = .04). There was no significant difference in freedom from distant metastasis between the two treatment groups (CT/RT group, 79%; RT group, 75%; P = .40). Conclusion: This randomized study failed to demonstrate any significant survival benefit with the addition of neoadjuvant chemotherapy for patients with locoregionally advanced nasopharyngeal carcinoma. Therefore, neoadjuvant chemotherapy for nasopharyngeal carcinoma should not be used outside of the context of a clinical trial. J Clin Oncol 19:1350-1357. (C) 2001 by American Society of Clinical Oncology.