Study of Children with Stage IV Kidney Cancer: Comparison of Standard Preoperative Chemotherapy Consisting of Vincristine, Actinomycin D, and Doxorubicin (VAD) with Preoperative Chemotherapy Consisting of Vincristine, Carboplatin, and Etoposide (VCE)

Kidney tumors in children are rare and often go unnoticed for a long time, as patients are frequently asymptomatic even at the time of diagnosis. In some cases, tumor metastases are already present, usually in the lungs. Nephroblastoma (Wilms tumor) is by far the most common form of childhood kidney cancer, while other types of tumors are significantly less common. If the tumor has already spread, it is classified as stage IV. Treatment depends on the type of tumor, but since nephroblastoma accounts for nearly 90% of cases, chemotherapy is often initiated before surgery to reduce the size of metastases. This is followed by surgical removal of the kidney, followed by further chemotherapy and, if necessary, radiation therapy.

The goal of this Phase 3 study is to investigate whether an alternative chemotherapy regimen consisting of the drugs vincristine, carboplatin, and etoposide (VCE) is just as effective as the standard combination of vincristine, actinomycin-D, and doxorubicin (VAD), but may have fewer side effects. In both treatment groups, the chemotherapy is administered through a vein (intravenously). One of the goals is to reduce the side effects of doxorubicin on the heart in patients with metastatic disease without lowering the cure rate. This is an equivalence study; such studies are particularly important when a conventional therapy is already achieving good results but needs to be optimized. Treatment begins for both treatment groups six weeks before surgery, and patients are randomly assigned to their respective treatment groups.

Children and adolescents between the ages of 3 months and 18 years who were diagnosed with a metastatic kidney tumor (Stage IV Wilms tumor) at the time of their initial diagnosis are eligible to participate in the study. A prerequisite is that imaging has detected at least one metastasis larger than 3 mm. In addition, histological examination must confirm a nephroblastoma. Patients must not have severe heart disease, liver or kidney dysfunction, or other pre-existing conditions that would compromise safe treatment in the study. Furthermore, patients who have already undergone chemotherapy prior to the start of the study are excluded.