A phase 2 study of vincristine and irinotecan in metastatic diffuse anaplastic Wilms tumor: Results from the Children’s Oncology Group AREN0321 study.

Abstract

10032 Background: Patients with metastatic diffuse anaplastic histology Wilms tumor (DAWT) had an unacceptably low 4-year event-free survival (EFS) and overall survival of 33% on the National Wilms Tumor Study-5. Preclinical and clinical data showed promising antitumor activity of the camptothecins in Wilms tumor. Methods: AREN0321 evaluated the activity of vincristine (Vinc) and irinotecan (Irino) in a phase 2 window in newly-diagnosed patients with Stage IV DAWT. Patients received Vinc (1.5 mg/m2/day) intravenously (IV) on days 1 and 8, and Irino (20 mg/m2/day) IV on days 1-5 and 8-12 of a 21-day cycle. In the absence of progressive disease (PD), a second cycle was administered. Tumor responses after 2 cycles of therapy were centrally reviewed and assessed using the RECIST criteria. Patients with partial response (PR) had Vinc/Irino incorporated into a chemotherapy regimen consisting of Vinc, doxorubicin, cyclophosphamide, carboplatin, and etoposide, plus local and whole lung radiation therapy. Patients...