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Penile cancer is a rare disease with approximately 2,070 new cases each year in the U.S. Treatment options are limited and typically begin with platinum chemotherapy. Immune checkpoint inhibitors (ICIs) are novel therapies that are active in several cancer types but are understudied in penile cancer. Clinical trials to evaluate ICIs for penile cancer are challenging to run because the cancer is so rare.

Investigators from Dana-Farber Cancer Institute, Yale Cancer Center, AdventHealth and other centers retrospectively looked at a multi-institutional, international cohort of 92 patients with advanced to assess the efficacy of ICIs.

The most common ICIs given were pembrolizumab, nivolumab, and cemiplimab but some patients received combination treatment with nivolumab and ipilimumab. The authors found that 13% of all patients and 35% of patients with metastases limited to lymph nodes responded to ICI therapy. Few experienced durable benefits from ICIs.

This retrospective study, published in JNCI: Journal of the National Cancer Institute, identifies a subset of patients with penile cancer that may benefit from ICIs. More research is needed to understand why ICIs work in some patients and not others.

More information: Talal El Zarif et al, Safety and efficacy of immune checkpoint inhibitors in advanced penile cancer: report from the Global Society of Rare Genitourinary Tumors, JNCI: Journal of the National Cancer Institute (2023). DOI: 10.1093/jnci/djad155