Irinotecan plus cisplatin in patients with extensive-disease poorly differentiated neuroendocrine carcinoma of the esophagus.

BACKGROUND: Poorly-differentiated neuroendocrine carcinoma (NEC) of the esophagus is a rare subtype that has a poor prognosis and is distinguished from well-differentiated neuroendocrine neoplasms in accordance with the 2010 World Health Organization classification. Irinotecan-plus-cisplatin (IP) is used as first-line chemotherapy for extensive-disease (ED) small-cell lung cancer and its use is plausible for first-line chemotherapy in ED esophageal NEC. We retrospectively analyzed the efficacy and toxicity of IP for ED esophageal NEC. PATIENTS AND METHODS: Patients with ED esophageal NEC treated with IP between 2000 and 2013 were retrospectively identified from our database. The end-points were objective response rate, progression-free survival (PFS) and overall survival (OS). Data on adverse events were also collected. RESULTS: An objective response was achieved in 50% (95% confidence interval [CI]: 25% to 75%) of 12 identified patients. Median progression-free survival was 4.0 months (95% CI: 0.9 to 7.6) and overall survival was 12.6 months (95% CI: 4.6 to 28.6). Grade 3/4 hematological toxicities included leukopenia in 50% of patients and neutropenia in 67%. The rate of febrile neutropenia was 25%. No treatment-related deaths were observed. CONCLUSION: IP appears acceptable as first-line chemotherapy for ED esophageal NEC.