Five-Year Survival in Heavily Pretreated Patients Receiving Nivolumab Monotherapy


Key Points

  • Overall survival at 5 years was 34.2% in patients with melanoma, 27.7% in those with RCC, and 15.6% in those with NSCLC.
  • Factors associated with likelihood of improved survival included better performance status and occurrence of treatment-related adverse events. 

As reported by Topalian et al in JAMA Oncology, long-term follow up of patients from a phase I expansion cohort study indicated 5-year overall survival rates of 34.2%, 27.7%, and 15.6% among patients who received nivolumab monotherapy for advanced melanoma, renal cell carcinoma (RCC), and non–small cell lung cancer (NSCLC), respectively.

Study Details

The study is a secondary analysis of a phase I trial (CA209-003) with expansion cohorts conducted at 13 medical centers in the United States. The trial enrolled 270 patients with advanced melanoma (n = 107), RCC (n = 34), or NSCLC (n = 129) between October 2008 and December 2011. Patients received nivolumab at 0.1, 0.3, 1.0, 3.0, or 10.0 mg/kg every 2 weeks in 8-week cycles for up to 96 weeks or until progressive disease, achievement of complete response, unacceptable toxicity, or withdrawal of consent. Patients had received one to five prior treatments for advanced or recurrent disease, with 40% having received three or more prior treatments.  

Overall Survival

Minimum follow-up for overall survival was 58.3 months for melanoma, 63.9 months for RCC, and 58.3 months for NSCLC. Median overall survival rates were 20.3 months, 22.4 months, and 9.9 months. Overall survival curves displayed an inflection toward flattening at approximately 3 years after treatment initiation. Overall survival at 3 years was 42.3% in patients with melanoma, 40.1% in patients with RCC, and 18.4% in patients with NSCLC. Overall survival at 5 years was 34.2%, 27.7%, and 15.6%.

Median overall survival was 19.8 months among patients with treatment-related adverse events of any grade and 20.3 months among those with treatment-related grade ≥ 3 adverse events vs 5.8 months among those with no treatment-related adverse events (P < .001 for both comparisons).

Factors Associated With Survival

In a multivariate analysis including all patients, liver metastases (odds ratio [OR] = 0.31, P = .02) and bone metastases (OR = 0.31, P = .04) were independently associated with reduced likelihood of 5-year survival. Eastern Cooperative Oncology Group performance status of 0 was an independent predictor of increased survival (OR = 2.74, P = .003). 

The investigators concluded, “Nivolumab treatment was associated with long-term survival in a subset of heavily pretreated patients with advanced melanoma, RCC, or NSCLC. Characterizing factors associated with long-term survival may inform treatment approaches and strategies for future clinical trial development.”

Suzanne L. Topalian, MD, of the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins Bloomberg~Kimmel Institute for Cancer Immunotherapy, is the corresponding author for the JAMA Oncology article.

Disclosure: The study was sponsored by Bristol-Myers Squibb. For full disclosures of the study authors, visit

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