Pediatric urologic rhabdomyosarcoma

To review on the most significant and recent developments in basic research, diagnostic, and therapeutic aspects of genitourinary rhabdomyosarcomas (RMS) in children.

Collaborative studies have dramatically improved therapy of genitourinary RMS with a remarkable improvement of survival. However, refractory or relapsed disease is still the most demanding challenge. Advances in our understanding of RMS molecular biology evolve new risk stratification and new therapeutic targets such as growth factors and their receptors. High-quality imaging is crucial and integrated fluorodeoxyglucose positron emission tomography/computed tomography significantly increased sensitivity. Alternative chemotherapeutical strategies were proven. Mutilating primary surgery should be avoided. Modern irradiation modalities with decrease of local radiation dose and, therefore, less long-term effects are new tools in the therapeutic arsenal of therapists.

Therapy-associated morbidity after current RMS treatment can be significantly decreased by including modern strategies, especially in the field of irradiation. However, the follow-up is too short so far to make a distinct decision. The advances in understanding tumor biology may give rise to clinically relevant new therapeutic targets in the near future.