Japanese Phase II Trial of Nivolumab in Previously Treated Advanced Esophageal Cancer


Key Points

  • In heavily pretreated patients with advanced esophageal squamous cell carcinoma, nivolumab produced response in 17% and stable disease in 25%.
  • Median duration of response had not been reached at the time of this analysis.

In a Japanese phase II trial reported in The Lancet Oncology, Kudo et al found that nivolumab (Opdivo) had activity in heavily pretreated patients with advanced esophageal squamous cell carcinoma.

Study Details

In the study, with enrollment between February and November 2014, 65 patients intolerant of or with disease refractory to fluoropyrimidine-, platinum-, and taxane-based chemotherapy received nivolumab at 3 mg/kg once every 2 weeks in 6-week cycles. The primary endpoint was centrally assessed objective response. Patients had a median age of 62 years, 82% were male, 68% had received at least 3 prior chemotherapy regimens, 68% had prior surgery, and 68% had prior radiotherapy.


Median follow-up was 10.8 months. Among 64 patients evaluable for efficacy, response was observed in 11 patients (17%, 95% confidence interval [CI] = 10%–28%), including a complete response in 1. Stable disease was observed in an additional 16 patients (25%). Median duration of response on central assessment had not been reached at the time of analysis, with durations ranging from 2.6 to 11.6 months at cutoff. On investigator assessment, response was observed in 14 patients (22%), including complete response in 2, and stable disease was observed in 20 patients (31%).

Adverse Events

Grade 3 or 4 adverse events occurred in 17 patients (26%), with the most common being lung infection (8%), decreased appetite (3%), increased creatine phosphokinase (3%), and dehydration (3%). Serious adverse events occurred in 22%, including lung infection (6%), dehydration (3%), and interstitial lung disease (3%). Treatment was interrupted due to adverse events in 23% and discontinued in 11%. There were no treatment-related deaths.

The investigators concluded: “Nivolumab showed promising activity with a manageable safety profile. This drug could offer a potential new treatment approach for patients with treatment-refractory advanced squamous-cell carcinoma.”

The study was funded by Ono Pharmaceutical and Bristol-Myers Squibb.

Yasuo Hamamoto, MD, of the Keio University School of Medicine, is the corresponding author of The Lancet Oncology article.

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