A phase II trial of gefitinib for recurrent or metastatic cancer of the esophagus or gastroesophageal junction

Background Conventional chemotherapeutic agents are of limited benefit in patients with recurrent or metastatic cancer of the esophagus or gastroesophageal junction (GEJ). We report results from a phase II trial in this population using gefitinib, an oral epidermal growth factor receptor inhibitor. Patients and methods Eligibility required a diagnosis of esophageal or GEJ adenocarcinoma or squamous cell carcinoma, which was either metastatic or recurrent and incurable after initial therapy. No more than one prior chemotherapy regimen was permitted. Treatment consisted of gefitinib 250 mg daily for a minimum of 8 weeks. Results Between April 2003 and January 2010, 58 patients, including 18 who were chemotherapy-na < ve, were entered into this trial. Toxicity was modest, although most experienced grade 1-2 diarrhea and/or skin rash. There were 4 partial responders (7%) and 10 patients with stable disease (17%). The clinical benefit (partial response and stable disease) lasted for a median 6.1 months. Median survival for all patients was 5.5 months with survival projections at 1-year of 24.6% and at 2-years of 12.5%. Conclusion Gefitinib was well tolerated but of limited efficacy in patients with recurrent or metastatic esophageal or GEJ cancer. Further study of this or similar agents will require better patient selection.