Genitourinary rhabdomyosarcoma (GU RMS) presents a myriad of challenges in the clinical setting and demands a nuanced understanding of its long-term impacts on patient outcomes. To this end, a comprehensive study conducted at our institution spanning 48 years provided valuable insights into the effects of various treatment modalities on this rare pediatric malignancy.¹

In this study, the greatest differences were seen when comparing radical surgery with organ-sparing approaches. We found that patients subjected to radical cystoprostatectomy (in males), cystectomy (in females), or anterior exenteration exhibited favorable outcomes in certain domains over extended periods. For example, the incidence of urinary incontinence in the radical surgery group was only 3.8% compared to 41.7% in the organ-sparing group. Although UTI rates were comparable between the 2 groups and stone formation was lower in those who were organ-spared, the higher incidence of urinary incontinence in the organ-sparing cohort warrants attention.

Furthermore, for the 29% of patients who initially underwent organ-sparing surgery (partial cystectomy, prostatectomy, or partial prostatectomy), three-quarters experienced significant genitourinary complications such as lower urinary tract symptoms, ureteral obstruction, and sexual dysfunction. Half of these patients required additional corrective surgery.

Sexual function emerged as a critical area of concern, with male and female patients who received a variety of treatment modalities reporting poor outcomes. We surmise that injury to neurovascular bundles following radical cystoprostatectomy and exenteration in males, and radiation-induced changes along with surgery for vaginal RMS in females, culminated in widespread patient-reported sexual dissatisfaction and functional impairments.

Despite the complexities and complications associated with both radical and organ-sparing approaches (Table), our study findings underscore the potential for radical surgery to achieve favorable long-term outcomes. For example, patients who underwent radical surgery and later elected to pursue continent diversion displayed resilience and a desire for improved quality of life, evident from the 71% conversion rate to continent diversion, with a mean time-to-conversion of 10.6 years. Moreover, the staged approach, which is standard at our institution and involves an initial conduit diversion followed by a delayed continent conversion, reflects a strategy tailored to the needs and readiness of patients. This approach is grounded in considerations for radiation exposure, reservoir capacity, and tissue availability.

This study was limited by its retrospective nature, small sample size (which is nonetheless significant in this rare disease population), reliance on patient-reported outcomes, and potential survivor biases. The heterogeneity of the cohort, including the inclusion of female patients with pelvic RMS, further complicates the interpretation of results. Despite such challenges, the study provides a comprehensive analysis of the long-term impacts of GU RMS treatment.

In conclusion, pediatric GU RMS management demands a nuanced and individualized approach. Radical surgery is promising regarding long-term urinary outcomes, though it is not devoid of complications. Many patients who are initially organ-spared may require future surgical interventions. Finally, the adverse effects on sexual function remain a significant challenge, underscoring the need for thorough pre- and posttreatment counseling. Future research, encompassing qualitative studies and prospective assessments, is imperative to enhance our understanding of this complex disease and refine treatment strategies for improved patient outcomes.