Observational study to predict the efficacy and optimal duration of nivolumab treatment in patients with previously treated advanced or recurrent non-small cell lung cancer

BACKGROUND: Immune checkpoint inhibitors, including nivolumab, are essential agents for treating non-small cell lung cancer. However, predictive markers are currently lacking, especially using factors based on patient-reported outcomes. METHODS: We conducted a prospective observational study of 244 patients with advanced or recurrent non-small cell lung cancer treated with second- or later-line nivolumab from August 2016 to December 2017. Patient-reported outcomes, including quality of life, were evaluated by the EQ-5D-5L before and during nivolumab treatment. To predict the efficacy of nivolumab during the early treatment phase, we also analyzed the patients’ clinical characteristics, responses and immune-related adverse events at 9 weeks of therapy. The primary endpoint was the disease control rate at 25 weeks after the initiation of nivolumab. RESULTS: The objective response and disease control rates at 25 weeks were 18.5 and 41.2%, respectively. The emergence of immune-related adverse events at 9 weeks did not significantly affect the disease control rate at 6 months. The response at 9 weeks and patient-reported quality of life were potentially predictive of disease control at week 25. Disease control on week 9 and patients-reported outcomes were potential predictive factors for the overall survival. CONCLUSIONS: This study found no new baseline factors predicting the outcome of nivolumab treatment in patients with non-small cell lung cancer, but response to nivolumab was a robust predictor of overall efficacy. In addition, patient-perceived quality of life could predict the durable efficacy of immune checkpoint inhibitors.