Treatment of stage V Wilms tumor and those predisposed to developing bilateral Wilms tumor

Currently, there is not a standard approach for the treatment of stage V Wilms tumor (bilateral Wilms tumor at diagnosis) and those predisposed to developing bilateral Wilms tumor. However, for the first time, a prospective study of the treatment of patients with bilateral Wilms tumor has been completed and provides guidance for the approach.[176]

Management of a child with bilateral Wilms tumor is very challenging. The goals of therapy are to eradicate all tumor and to preserve as much normal renal tissue as possible, with the hope of decreasing the risk of chronic renal failure among these children.[288]

Historically, based on the NWTS-4 and NWTS-5 trials and trials performed in Europe, patients with bilateral Wilms tumor have had a lower EFS and OS than have patients with localized Wilms tumor. The NWTS-4 study reported that the 8-year EFS rate for patients with bilateral FH Wilms tumor was 74%, and the OS rate was 89%; for patients with anaplastic histology, the EFS rate was 40%, and the OS rate was 45%.[204] The NWTS-5 study reported that the 4-year EFS rate for all bilateral Wilms tumor patients was 56%, and the OS rate was 81%; the 4-year EFS rates for patients with FH (65%), focal anaplastic histology (76%), and diffuse anaplastic histology (25%) were also reported.[110,183] The SIOP Renal Tumor Study Group reported the results of the SIOP2001 trial for the treatment of patients with bilateral Wilms tumor. Surgery was recommended after neoadjuvant chemotherapy, which consisted of dactinomycin and vincristine, with response-adapted intensification. In 174 patients with bilateral Wilms tumor, the 5-year EFS rate was 76.1%, and the OS rate was 88.1%. With a median follow-up of 7.4 years, 87% of patients retained normal renal function without a transplant.[289]

Treatment options for stage V (bilateral) Wilms tumor include the following: