In the last few decades, significant strides have been made in the treatment of this disease. These have improved its historically dismal prognosis. This can be contributed to multi-modal therapy consisting of polychemotherapy, surgery and radiotherapy. In localized disease this has resulted in cure-rates as high as 85%. Dr. Filipas and colleagues at the University of Mainz, Germany report their experience of 107 children with rhabdomyosarcoma of pelvic organs in the BJU International 2004;93.

Between 1968 and 2001 107 children with RMS were treated, of whom 22 (20%) had tumors of the urogenital tract. 19 of these patients had RMS of the prostate and/or bladder and 3 of the vagina. Data from these patients were retrospectively analyzed and follow up details obtained. Patients were stratified by the Intergroup Rhabdomyosarcoma Study (IRS), i.e. group I (none), disease localized and completely resected; group II (six), regional disease, resected; group III (twelve), localized disease, not completely resected; and group IV (four) metastatic disease present at diagnosis. All patients received multi-agent chemotherapy according to the IRS protocol. Patients not responding well to chemotherapy received radiation prior to surgery. All 22 patients with urogenital RMS received chemotherapy. Radical surgery was used in 14 patients, but none of the girls with vaginal RMS underwent surgery. Extensive use of interoperative frozen-section analysis was utilized to determine tissue margins. Seven patients received additional postoperative radiotherapy.

The mean follow up of the 22 patients was 8.6 years. Five patients with an advanced tumor stage died of their disease. 17 children currently have no evidence of disease. The three girls with RMS of the vagina were cured by chemotherapy, either alone or in combination with radiotherapy. The two boys with RMS of the prostate, treated with chemotherapy only, or additional radiotherapy are free of tumor at follow up of 11 and 14.3 years, respectively. Of the remaining children treated with polychemotherapy and combined radical surgery, one boy presented with a relapse after 2 years and was successfully salvaged by repeat chemotherapy. In two boys radical salvage surgery was performed by doing a combined abdominoperineal approach. In one boy tumor was confined to the bladder and radical cystoprostatectomy with orthotopic urinary diversion was used. Regarding urinary diversion, in 7 children a continent ileocecal pouch to the umbilicus was constructed (Mainz Pouch I). One patient had a ureterosigmoidostomy and one patient a rectosigmoid pouch (Mainz-Pouch II). Other forms of urinary diversion included transverse colonic pouch, colonic conduit and ileocecal orthotopic bladder. Two patients experienced bladder contractions secondary to radiation and required bladder augmentation. Another boy developed ureteric implantation stenosis requiring reimplantation.

Polychemotherapy of RMS of the genitourinary tract has significantly improved the prognosis of this rare tumor in children. The increased use of chemotherapy with encouraging initial results, led to bladder-sparing nonsurgical treatments. These developments had the greatest effect on the treatment of RMS of the female genital tract where the rate of radical surgeries decreased from 100% in the mid 1970's to 13% in the 1990's. For patients with RMS of prostate and/or bladder, stratification to low-risk and high-risk has proven useful. Low-risk patients responding to chemotherapy with significant remission of the tumor can likely avoid surgery. High-risk patients not responding to chemotherapy require intensive treatment including radical surgery.

This study supports the multi-modal approach to RMS of the genitourinary tract and helps to further define the role of radical surgery in this approach. These data also demonstrate that urinary diversion can be implemented in these patients with a reasonable side-effect profile, despite the need for other therapies such as chemotherapy and radiation.

BJU Int. 2004; 93:125-129