AI Summary
Patients with stage IV Wilms tumor (FHWT) and extrapulmonary metastases (EPM) have poor outcomes. The optimal treatment for these patients is unknown. A study reviewed the experiences of patients with EPM on different treatment protocols to gather more information. The pooled results of all patients showed a 4-year survival rate of 77.3%. Patients treated on protocol AREN0533 had similar outcomes to those on protocol NWTS-5. Age and lung response were associated with worse outcomes. Further trials with standardized treatment and detailed data are needed.
Abstract
Background
Patients with stage IV favorable histology Wilms tumor (FHWT) with extrapulmonary metastases (EPM) constitute a small subset of patients with FHWT. Because of their rarity and heterogeneity, optimal FHWT treatment is not well understood. Children’s Oncology Group protocol AREN0533 assigned patients with FHWT and EPM to intensified chemotherapy, regimen M, after initial DD-4A chemotherapy. To improve understanding of prognostic factors and best therapies, experiences of patients with EPM on AREN0533, as well as on protocols AREN03B2 and NWTS-5, were reviewed.
Methods
Combined outcomes for patients with EPM from NWTS-5, AREN0533, and AREN03B2 were determined. Those treated on AREN0533 were compared with those treated on NWTS-5. Prognostic factors were explored in the pooled cohort.
Results
Forty-seven patients with FHWT with EPM enrolled on AREN0533, 37 enrolled on NWTS-5, and 64 were followed only on AREN03B2. The pooled cohort of all 148 patients demonstrated a 4-year event-free survival (EFS) of 77.3% (95% CI, 70.8–84.4) and 4-year overall survival of 88.9% (95% CI, 83.9–94.2). Four-year EFS of patients with EPM treated on AREN0533 was 76.0% (95% CI, 64.6–89.4) vs 64.9% (95% CI, 51.7–82.2) on NWTS-5; hazard ratio, 0.64, p = .26; no difference in overall survival was observed. Increasing linear age and slow incomplete lung response were associated with worse EFS in a pooled cohort.
Conclusions
Outcomes for patients with EPM are among the lowest for children with FHWT. Further trials with standardized surgical and radiation treatment to metastatic sites, and prospectively collected biologic and treatment details are needed.
Clinical trial registration
Clinical Trials.gov identifiers: NCT00379340, NCT00898365, and NCT00002611.