Nivolumab for chemorefractory oesophageal squamous cell carcinoma

Although immunotherapy has radically changed the treatment paradigm for a variety of tumours including melanoma, non-small cell lung cancer and renal cell carcinoma, until now it has been less successful in tumours of the gastrointestinal tract. In a recent report in Lancet Oncology, Kudo et al. described the results of the first trial assessing immune checkpoint blockade in OSCC patients (1). The study enrolled 65 Japanese patients with platinum and taxane refractory OSCC in a standard open-label phase II design, the primary endpoint of which was centrally assessed objective response rate (ORR). OSCC patients treated with nivolumab on the trial had an ORR of 17% [95% confidence interval (CI), 10–28%] by central radiological review. However, a further proportion of patients appeared to benefit from anti-PD-1 therapy but did not reach RECIST criteria for response; a reduction in overall tumour burden was observed in 45% of participants by the trial investigators. In keeping with many studies of immunotherapy, median progression-free survival (PFS) did not appear to be substantially improved by nivolumab (median PFS, 1.5 months; 95% CI, 1.4–2.8 months), however, median overall survival (OS) was promising for a chemorefractory patient population at 10.8 months (95% CI, 7.4–13.3 months). These results are potentially important because if validated in a randomised controlled trial, nivolumab could provide a new treatment option for advanced OSCC, a cancer for which very little evidence is available to guide clinical management.