Study on Combination Chemotherapy and Surgery for Treating Young Patients with Wilms Tumor

Program Overview

This study is investigating the effectiveness of using a combination of chemotherapy drugs along with surgery to treat young patients with Wilms tumor, a type of kidney cancer. Chemotherapy drugs work by killing cancer cells, stopping them from dividing, or preventing them from spreading. By using more than one drug, the treatment may be more effective in reducing the tumor size before surgery and in eliminating any remaining cancer cells after surgery.

Description

The main goals of this study are:

1. To improve the 4-year event-free survival rate to 73% for young patients with bilateral Wilms tumor (BWT).
2. To avoid the complete removal of at least one kidney in 50% of patients with BWT by using a three-drug chemotherapy regimen before surgery.
3. To evaluate how well chemotherapy can preserve kidney function in children with a condition called diffuse hyperplastic perilobar nephroblastomatosis (DHPLN) and prevent the development of Wilms tumor.
4. To enable partial kidney removal instead of complete removal in 25% of children with unilateral tumors and certain genetic conditions by using a two-drug chemotherapy regimen before surgery.
5. To have 75% of patients with BWT undergo definitive surgery by 12 weeks after starting chemotherapy.

Patients are divided into three groups:

• Group 1 (Bilateral Wilms Tumors): Patients receive a three-drug chemotherapy regimen and are evaluated at 6 and 12 weeks to see if partial kidney surgery is possible. Surgery is performed at week 12, followed by additional chemotherapy and possibly radiation, depending on the tumor type and stage. Treatment lasts for 25 or 31 weeks, and patients are followed for up to 10 years.

• Group 2 (Unilateral High-Risk Tumors): Patients receive either a two-drug or three-drug chemotherapy regimen and are evaluated at 6 and 12 weeks for the possibility of partial kidney surgery. Surgery is performed at week 12, followed by additional chemotherapy.

• Group 3 (DHPLN): Patients with this rare condition receive a two-drug chemotherapy regimen. They are reassessed at 6 and 12 weeks. If the disease responds or remains stable, chemotherapy continues for 19 weeks. If the disease progresses, a biopsy is done to adjust treatment, which may include surgery, chemotherapy, or radiation.

Chemotherapy Regimens:

• VAD Regimen: Vincristine is given intravenously over 1 minute on specific days, and dactinomycin and doxorubicin are given intravenously over 15-120 minutes on specific days.

• EE4A Regimen: Vincristine is given intravenously over 1 minute on specific days, and dactinomycin is given intravenously over 1-5 minutes on specific days.

After completing the study treatment, patients are followed up periodically for 10 years.

Eligibility Criteria