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Paediatric lower urinary tract rhabdomyosarcoma: a single-centre experience of 30 patients
M T El-Sherbiny1, M H El-Mekresh, M A El-Baz
1Urology and Nephrology Centre, Mansoura University, Mansoura, Egypt.
Insights
Chemotherapy response in children with bladder or prostate rhabdomyosarcoma predicts outcomes. Good responders can preserve their bladder, while minimal responders require intensive treatment without bladder salvage.
Area of Science:
- Pediatric Oncology
- Urologic Oncology
- Cancer Treatment Outcomes
Background:
- Rhabdomyosarcoma of the bladder and prostate is a rare but aggressive pediatric cancer.
- Long-term outcomes for this disease have historically varied based on treatment protocols and patient response.
Purpose of the Study:
- To retrospectively analyze the long-term outcomes of pediatric patients diagnosed with bladder and/or prostate rhabdomyosarcoma.
- To evaluate the effectiveness of initial chemotherapy response in stratifying patients for risk-adapted treatment strategies.
Main Methods:
- Retrospective analysis of 30 children (26 boys, 4 girls) with bladder/prostate rhabdomyosarcoma treated over 17 years.
- All patients received initial vincristine, actinomycin D, and cyclophosphamide (VAC) chemotherapy.
- Subsequent treatment was determined by the response to initial chemotherapy, including surgery and radiotherapy.
Main Results:
- Fourteen patients showed a complete or partial response (>50% tumor reduction) to chemotherapy; 12 survived with no evidence of disease, with bladder salvage possible.
- Sixteen patients had a minimal response; 6 underwent radical cystectomy followed by chemotherapy, with all surviving disease-free.
- Ten patients with minimal response received radiotherapy, with only 3 surviving long-term.
Conclusions:
- Initial chemotherapy response is a critical factor in stratifying pediatric rhabdomyosarcoma patients.
- Complete or partial responders represent a low-risk group where bladder salvage is feasible.
- Minimal responders are high-risk and require intensive treatment, often foregoing bladder salvage attempts.
Objective:
To retrospectively analyse the long-term outcome of children with bladder and/or prostate rhabdomyosarcoma who were diagnosed at the authors' institution over the last 17 years.
Patients And Methods:
The study comprised 30 children (26 boys and four girls, mean age 5 years, range 15 days to 15 years); 23 had stage III and seven had stage II disease. The initial biopsy showed an embryonal variant in 27 and round-cell sarcoma in three patients. All patients received eight weekly doses of vincristine, actinomycin D and cyclophosphamide (VAC). Subsequent treatment depended upon the response to chemotherapy.
Results:
Fourteen patients had a complete or partial response to chemotherapy (> 50% reduction in tumour size); they were maintained on VAC chemotherapy for 2 years. Twelve patients in this group survived with no evidence of disease for 7 months to 10 years. Additional therapies were used in three patients, i.e. radical cystectomy in one and external irradiation in two. Sixteen patients had a minimal response to chemotherapy; in six, radical cystectomy was feasible and was followed by one year of chemotherapy. All patients were free of disease for 4-11 years. Radiotherapy was given to the remaining 10 patients; thereafter radical cystectomy became feasible in five while partial cystectomy was possible in three. Only three of these 10 patients survived for 4-11 years.
Conclusion:
The tumour response to initial chemotherapy can be used to stratify patients into two risk-groups, i.e. low-risk patients with a complete or partial response in whom the bladder could be salvaged, and high-risk patients with a minimal response, in whom intensive treatment should be pursued, with no attempt at bladder salvage.
