Abstract
Recent clinical trials have revealed several unanticipated complexities in the optimal management of genitourinary rhabdomyosarcoma (RMS). Improvement in outcomes for low- and intermediate-risk RMS over the past several decades led to the design of clinical trials aimed at reducing acute and late toxicity from extirpative surgeries, conventional radiotherapy, and cytotoxic chemotherapy. Results from these studies are mixed and have illuminated areas where historical risk stratification strategies need refining. Although radiotherapy has now become the standard for local control for most patients with genitourinary RMS, recent studies are demonstrating that there may be opportunities to minimize radiation toxicity while maintaining acceptable failure-free survival. A reduction in cyclophosphamide exposure may benefit select low-risk RMS patients but recent results illustrate that decreasing therapy intensity for most genitourinary RMS patients will require careful consideration in future prospective trials. Finally, recent studies highlight differences in perspective between European and North American investigators regarding the optimal balance of increased local failure rates but less toxicity versus improved event-free survival at a cost of higher toxicity. This review focuses on the results from the most recent RMS clinical trials and discusses their implications for the management of pediatric genitourinary RMS.