A Multicenter Phase II Trial of Axitinib in Patients With Recurrent or Metastatic Noneclear-cell Renal Cell Carcinoma Who Had Failed Prior Treatment With Temsirolimus

We conducted a multicenter phase II trial of axitinib for patients with advanced noneclear-cell renal cell carcinoma who had failed prior treatment with temsirolimus. Axitinib showed promising efficacy in terms of objective response rate and progression-free survival in recurrent or metastatic noneclear-cell renal cell carcinoma when used after failure with temsirolimus. Background: The optimal treatment option for noneclear-cell renal cell carcinoma (nccRCC) is not established. We conducted a multicenter phase II trial of axitinib for patients with advanced nccRCC who had failed prior treatment with temsirolimus. Patients and Methods: Patients with histologically confirmed metastatic or recurrent nccRCC received 5 mg axitinib twice daily. Prior use of vascular endothelial growth factor pathway inhibitors was not allowed. The primary endpoint was progression-free survival (PFS), and the secondary endpoints were objective response rate (ORR), disease control rate, overall survival, and safety. Results: Forty patients were included between January 2013 and December 2016. The median age was 59 years (range, 22-84 years). Eastern Cooperative Oncology Group performance status were 0 (7.5%) and 1 (92.5%), and 82.5% of patients had undergone prior nephrectomy. Papillary type 2 (60.0%) was the most common histology, and patients belonged to favorable (12.5%), intermediate (72.5%), and poor (15.0%) risk groups according to the International Metastatic Renal Cell Carcinoma Database Consortium risk stratification. With a median follow-up duration of 14.7 months (95% confidence interval, [ CI], 10.8-18.6 months), the median PFS was 7.4 months (95% CI, 5.2-9.5 months). The ORR was 37.5%, and the disease control rate was 67.5%. The median overall survival was 12.1 months (95% CI, 6.4-17.7 months). Most adverse events were manageable, and no unexpected toxicities were found. Conclusion: Axitinib showed promising efficacy in terms of ORR and PFS in recurrent or metastatic nccRCC when used after failure with temsirolimus.