Therapeutic Effect of Recombinant Human Epidermal Growth Factor (RhEGF) on Mucositis in Patients Undergoing Radiotherapy, With or Without Chemotherapy, for Head And Neck Cancer A Double-blind Placebo-controlled Prospective Phase 2 Multi-institutional Clinical Trial

BACKGROUND: We evaluated the efficacy of topically applied human recombinant epidermal growth factor (rhEGF) for the treatment of oral mucositis induced by radiotherapy (RT), with or without chemotherapy, in patients with head and neck cancer. METHODS: Patients receiving definitive chemoradiotherapy, definitive RT, or postoperative RT to the oral cavity or oropharynx were recruited from 6 institutions and enrolled in a randomized, double-blind, placebo-controlled phase 2 trial. Patients were assigned to a placebo group or to 1 of 3 EGF-treatment groups (10, 50, or 100 mu g/mL doses, delivered in a spray, twice daily). The grade of mucositis was evaluated using the Radiation Therapy Oncology Group (RTOG) scoring criteria. Responders to EGF were defined as having an RTOG grade of 2 or lower at the fourth- or fifth-week examinations during RT, but an enduring RTOG grade 2 for 2 weeks was an exception. RESULTS: Of the 113 patients included in the study, 28 received placebo and 29 received EGF at 10 mu g/mL, 29 at 50 mu g/mL, and 27 at 100 mu g/mL. EGF significantly reduced the incidence of severe oral mucositis at the primary endpoint (a 64% response was observed with SO mu g/mL EGF vs a 37% response in the control group; P = .0246). CONCLUSIONS: The EGF oral spray may have potential benefit for oral mucositis in patients undergoing RT for head and neck cancer. Phase 3 studies are ongoing to confirm these results. Cancer 2009;115:3699-708. (C) 2009 American Cancer Society.