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Wilms Tumor and Other Childhood Kidney Tumors Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of Wilms Tumor

Table 3. Overview of Wilms Tumor Standard Treatment by Stage continued...

Additional Treatment Considerations

Stage I

It may be possible to treat a subset of stage I Wilms tumor patients with surgery alone without chemotherapy. The Children's Oncology Group (COG) addressed this question in the National Wilms Tumor Study-5 (NWTS-5 [COG-Q9401]) trial for children younger than 2 years at diagnosis with stage I favorable histology (FH) Wilms tumors that weigh less than 550 g. In the NWTS-5 study, the omission of adjuvant chemotherapy was tested for this group of patients.[4] Stringent stopping rules were designed to ensure closure of the study if the 2-year relapse-free survival rate was 90% or lower. The expectation was that approximately 50% of the surgery-only children would be salvaged after recurrence thus attaining the 95% predicted survival of children with very low-risk Wilms tumor treated with standard chemotherapy according to regimen EE-4A. This study was discontinued in 1998 when the predicted 2-year EFS fell below 90%.[5] Long-term follow-up of this study of the surgery-only cohort and the EE-4A group with a median follow-up of 8.2 years reported the estimated 5-year EFS for surgery only was 84% (95% confidence interval [CI], 73%–91%); for the EE-4A patients it was 97% (95% CI, 92%–99%, P = .002). One death was observed in each treatment group. The estimated 5-year overall survival (OS) was 98% (95% CI, 87%–99%) for surgery only and 99% (95% CI, 94%–99%) for EE-4A (P = .70).[4] COG study COG-AREN0532 is assessing this cohort again and is evaluating biological markers for this very low-risk group.[4,6]

Stage III

The outcome of patients with peritoneal implants treated with gross resection, three-drug chemotherapy, and total abdominal radiation (10.5 Gy) is similar to other stage III patients.[7][Level of evidence: 2A]

Stage IV

Stage IV disease is defined by the presence of hematogenous metastases to the lung, liver, bone, brain, or other sites, with the lung being the most common site. Previously, chest x-rays were used to detect pulmonary metastases. The introduction of computed tomography (CT) created controversy because many patients had lung nodules detected by chest CT scans that were not seen on chest x-rays. Management of newly diagnosed patients with FH Wilms tumor who have lung nodules detected only by CT scans (with negative chest x-ray) has elicited controversy as to whether they need to be treated with additional intensive treatment that is accompanied by acute and late toxicities. In a retrospective review of 186 patients from NWTS-4 and NWTS-5 (COG-Q9401) with CT-only detected lung nodules, patients who received doxorubicin in addition to vincristine and dactinomycin with or without lung radiation had a 5-year EFS of 80% versus 56% for patients receiving only two drugs (P = .004). There was no difference in the 5-year OS (87% vs. 86%).[8]

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