Single-agent nivolumab induced durable clinical responses with a manageable safety profile in previously treated patients with locally advanced or metastatic urothelial carcinoma, according to a study published in the journal The Lancet Oncology.1

Because limited treatment options exist for patients with advanced urothelial carcinoma who have progressed after platinum-based chemotherapy, researchers sought to evaluate the activity and tolerability of nivolumab, which has demonstrated activity in a variety of cancer types, in patients with locally advanced or metastatic urothelial carcinoma.

For the international, open-label, phase 1/2 trial ( Identifier: NCT01928394), investigators enrolled 86 patients with metastatic urothelial carcinoma of the renal pelvis, ureter, bladder, or urethra. All patients received nivolumab monotherapy every 2 weeks until disease progression or unacceptable toxicity.

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At a median follow-up of 15.2 months, results showed that 24.4% (95% CI, 15.2-35.4) of 78 evaluable patients achieved a response per investigator assessment.

Researchers found that 22% experienced grade 3 to 4 treatment-related adverse events, with the most common being elevated lipase, fatigue, maculopapular rash, dyspnea, lymphopenia, and neutropenia.

In total, 10% experienced serious treatment-related adverse events and 3% discontinued nivolumab therapy and subsequently died due to treatment-related grade 4 pneumonitis and grade 4 thrombocytopenia.

The encouraging findings ultimately support the further investigation of single-agent nivolumab in this patient population.


1. Sharma P, Callahan MK, Bono P, et al. Nivolumab monotherapy in recurrent metastatic urothelial carcinoma (CheckMate 032): a multicentre, open-label, two-stage, multi-arm, phase 1/2 trial. Lancet Oncol. 2016 Oct 9. doi: 10.1016/S1470-2045(16)30496-X. [Epub ahead of print]