Chemotherapy as monotherapy for treatment of non-metastatic bladder/prostate embryonal rhabdomyosarcoma in children: long term outcomes.
Hesham Arab, M.Sc.,MRCS, Ahmed wadea, M.Sc., Helmy Omar, M.Sc., Ahmed AbdelHalim, M.Sc.,MRCS, Rasha Elashry, M.D, Tamer Helmy, M.D, Mohamed Dawaba, M.D, Ashraf Tarek Hafez, M.D.
Urology and nephrology center,Mansoura University, Mansoura, Egypt.
BACKGROUND: The aim of this work is to evaluate the long term outcomes of chemotherapy as monotherapy for treatment of non-metastatic bladder/prostate embryonal rhabdomyosarcoma in children, as a continuation of our published preliminary report.
Between 1996 and 2011, 35 children presented with non-metastatic RMS of the bladder/prostate at a mean age of 6.2 years (range 2 - 22). 15 patients (43%) had stage 2 disease and 20 (57%) had stage 3. The histology was embryonal in 29 patients and botryoid in 6 patients representing 83% and 17% respectively. All patients received VAC chemotherapy for a mean duration of 18 months (range 12 - 22). Second look biopsy was performed at 3 months; also CT or MRI was done at 8 weeks, every 3 months in the first year and every 6 months thereafter.
The median follow-up duration was 78 months (range 12 - 96). At 8 weeks, 17 Children (49%) showed more than 50% reduction in tumor size and the remaining 18 children (51%) showed complete disappearance on CT or MRI. At final follow-up 32 (94%) patients are living disease-free, 30 (85%) patients of them cured with chemotherapy only, with functioning bladder, one patient had persistent tumor and one patient had recurrent disease, both needed cystectomy. The remaining 3 patients died of metastatic and/or recurrent disease within 12 month (± 3.6) from start of treatment. Out of the 30 survivors with chemotherapy only, 24 (80%) had complete disappearance of the tumor at last follow-up while 6 (20%) had residual mass on CT/MRI. Tumor free in survivors was confirmed after second look biopsy. The 5-year disease-free survival was 100% for stage 2 and 75% for stage 3.
Chemotherapy as mono-therapy is effective treatment in children with stage 2 embryonal RMS of the bladder/prostate with the advantage of bladder preservation. Within stage 3, non-survivors had statistically significant larger tumors. Adjuvant therapy or more intensive chemotherapy might be required for those with large stage 3 tumors.
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