Randomized phase III study of 2 cisplatin-based chemoradiation regimens in locally advanced head and neck squamous cell carcinoma: Impact of changing disease epidemiology on contemporary trial design

Cristina P. Rodriguez, David J. Adelstein, Lisa A. Rybicki, Panayiotis Savvides, Jerrold P. Saxton, Shlomo A. Koyfman, John F. Greskovich, Min Yao, Joseph Scharpf, Pierre Lavertu, Benjamin G. Wood, Brian B. Burkey, Robert R. Lorenz, Rod P. Rezaee, Chad A. Zender, Denise I. Ives

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Background Chemoradiotherapy results in excellent outcomes in locally advanced head and neck squamous cell carcinoma (HNSCC). This trial compared 2 chemoradiotherapy regimens. Methods Patients with locally advanced HNSCC were treated with radiation (70-74.4 Gy), and randomized to arm A: cisplatin 100 mg/m2 on radiotherapy (RT) days 1, 22, and 43, or arm B: cisplatin (20 mg/m2/day) and 5-fluorouracil (5-FU; 1000 mg/m2/day) continuous 96-hour infusions on RT weeks 1 and 4. The primary endpoint was relapse-free survival (RFS). Results Between February 2008 and October 2011, 69 patients were enrolled in this study. The study prematurely closed when a scheduled interim analysis showed superior outcomes in both arms and futility of continuation. Eighty-three percent of patients had oropharyngeal cancer, of these, 86% were human papillomavirus (HPV)/p16+. The 3-year Kaplan-Meier outcome estimates (median follow-up, 41 months) for arms A and B were: RFS 87% versus 80% (p=.24), overall survival 97% versus 85% (p=.013), locoregional control 96% versus 94% (p=.52), and distant metastatic control 91% versus 87% (p=.9). Conclusion Multiagent was not superior to single-agent chemoradiotherapy. Overrepresentation of HPV/p16+ patients resulted in better than expected outcomes.

Original languageEnglish (US)
Pages (from-to)1583-1589
Number of pages7
JournalHead and Neck
Issue number11
StatePublished - Nov 2015

All Science Journal Classification (ASJC) codes

  • Otorhinolaryngology

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