Related Experiment Video
Updated: Jun 16, 2026
Surgical Treatment of an Endolymphatic Sac Tumor
Published on: May 26, 2023
Long-term outcomes for infants with very low risk Wilms tumor treated with surgery alone in National Wilms Tumor
Robert C Shamberger1, James R Anderson, Norman E Breslow
1Department of Surgery, Children's Hospital Boston and Harvard Medical School, Boston, MA, USA. robert.shamberger@childrens.harvard.edu
Insights
Surgery alone for very low-risk Wilms tumor (VLRWT) in children resulted in lower event-free survival but equivalent overall survival compared to chemotherapy. This approach minimizes chemotherapy toxicity for most patients.
Area of Science:
- Pediatric Oncology
- Surgical Oncology
- Clinical Trials
Background:
- Previous research indicated limited benefit of postoperative chemotherapy for very low-risk Wilms tumor (VLRWT).
- A cohort of 77 infants (<24 months) with small Stage I favorable histology Wilms tumors received surgery exclusively.
- The study employed stopping rules to ensure a minimum 2-year event-free survival (EFS) of 90%.
Purpose of the Study:
- To evaluate the event-free survival (EFS) and overall survival (OS) in pediatric patients with very low-risk Wilms tumor (VLRWT) treated solely with surgical intervention.
- To compare outcomes of surgery-only treatment against a regimen including dactinomycin and vincristine (EE4A).
Main Methods:
- Seventy-seven children with VLRWT were assessed for EFS and OS.
- Twenty-one patients who enrolled at study closure received salvage chemotherapy (EE4A) and were censored.
- A comparison group of 111 children treated with EE4A was analyzed.
Main Results:
- The median follow-up was 8.2 years for the surgery-only group and 5.2 years for the EE4A group.
- Five-year EFS was 84% for surgery-only versus 97% for EE4A (P=0.002).
- Five-year OS was 98% for surgery-only and 99% for EE4A (P=0.70).
Conclusions:
- While surgery-only EFS was below the target, the high salvage rate for recurrences resulted in long-term OS comparable to two-drug chemotherapy.
- This strategy avoids chemotherapy-related toxicities for the majority of VLRWT patients.
- Further research is ongoing to validate these findings in a follow-up study.
Objective:
To determine the event-free survival (EFS) and overall survival (OS) of children with very low risk Wilms tumor (VLRWT) treated with surgery only.
Background:
Previous studies suggested that postoperative chemotherapy had not improved the prognosis of children with VLRWT. A total of 77 children <24 months of age with small (<550 g) Stage I favorable histology Wilms tumors were treated with surgery only. This study was closed based on stopping rules to ensure that the 2-year EFS was > or =90%.
Methods:
A total of 77 children were assessed for EFS and OS. Of these patients, 21 enrolled at the time of closure were recalled, treated with dactinomycin and vincristine (regimen EE4A), and censored for analysis thereafter. About 111 children subsequently treated with EE4A were available for comparison.
Results:
Median follow-up of surviving patients was 8.2 years for surgery only (range, 1.9-11.8 years) and 5.2 years for the EE4A group (range, 1.6-8.9 years). The estimated 5-year EFS for surgery only was 84% (95% confidence interval [CI]: 73%, 91%); for the EE4A patients it was 97% (95% CI: 92%, 99%, P = 0.002). One death was observed in each treatment group. The estimated 5-year OS was 98% (95% CI: 87%, 99%) for surgery only and 99% (95% CI: 94%, 99%) for EE4A (P = 0.70).
Conclusion:
The surgery-only EFS was lower than anticipated but, coupled with a much higher than anticipated salvage rate of the chemotherapy naive patients whose disease recurred, led to an observed long-term OS equivalent to that seen with 2-drug chemotherapy. This approach to the treatment of patients with VLRWT eliminates the toxic side-effects of chemotherapy for a large majority of patients. A follow-up study is underway to confirm these findings.
