Results of novel strategies for treatment of Wilms' tumor.

Objective: To evaluate treatment outcomes in Wilms' tumor (WT).
Materials and Methods: We studied 53 children with median age of 2 years with WT, stages I-19, II-14, III-12, IV-6 and V-2. Treatment consisted of surgical excision plus adjuvant (40 children) or neoadjuvant and adjuvant chemotherapy (unresectable tumor, n=8, or caval tumor extension, n=5). Chemotherapy and radiotherapy followed protocols of Brazilian Wilms' Tumor Study Group excepting 16 cases with stage I disease that received a short duration postoperative treatment with vincristine (VCR - 11 doses) and dactinomycin (AMD - 4 doses). Relapsed WT was treated with multiagent regimens including cisplatin/carboplatin, cyclophosphamide, ifosfamide and etoposide. One patient with resistant relapsed WT was treated by high-dose conditioning chemotherapy with stem cell rescue.
Results: Overall and disease-free survival rates at 5 years were respectively 88.2 +/- 5.0% and 76.7 +/- 6.6%. Short duration therapy for stage I tumor showed a disease-free survival rate of 100% in a median time of 101 months (range 14 to 248 months). Overall and disease-free survival of 10 patients with recurrent WT at 5 years was 42.8%. The child treated with high-dose chemotherapy plus stem cell transplant is alive without evidence of disease 84 months from relapse.
Conclusion: The postoperative chemotherapy in stage I disease can be reduced without compromising the cure rate. The treatment of unfavorable stage III and IV disease or relapsed tumor remains a challenge.