TW2003, the 3(rd) Italian prospective study on Wilms tumor (WT), aimed to improve survival in patients with stage III-IV tumors, de-escalate therapy for stage I-II non-anaplastic tumors, refine the risk stratification of therapy, and develop a national infrastructure for biobanking and central pathology review.
TW2003 recruited children ≤18 years old with primary intrarenal tumors. Local physicians chose nephrectomy with or without preoperative chemotherapy as the initial treatment, based on the risk of unsafe and/or incomplete immediate surgery. The main drivers for adjuvant therapy were tumor stage and diffuse anaplasia. A new risk stratification schema was investigated, incorporating patient's age, reason for stage III designation, and completeness of lung nodule response in the case of stage IV disease.
We report on 453 patients with unilateral WT. Preoperative chemotherapy was administered to 42% of patients. Five-year event-free survival (EFS) and overall survival (OS) rates were respectively: 89.1% (95% confidence interval [CI]: 83.6%-94.9%) and 97.0% (93.7%-100%) for stage I; 85.1% (79.6%-91.1%) and 94.0% (90.1%-98.1%) for stage II (n=160); 82.7% (75.3%-90.8%) and 90.9% (85.0%-97.1%) for stage III (n=101); 72.1% (61.9%-84.0%) and 82.5% (73.1%-93.1%) for stage IV (n=69). On multivariable analysis, only anaplasia was significant for EFS (hazard ratio 2.68, 95%CI: 1.48-4.86; P=.001; bias-corrected c-index = 0.580) and OS (hazard ratio 5.29, 95%CI: 2.52-11.12; P<.001; bias-corrected c-index = 0.697).
The survival rates achieved, and the proposed risk stratification schema, provide a basis for future comparisons of WT treatment burden and patient outcome.
The Journal of urology. 2017 Jun 24 [Epub ahead of print]
Filippo Spreafico, Davide Biasoni, Salvatore Lo Vullo, Lorenza Gandola, Paolo D'Angelo, Monica Terenziani, Maurizio Bianchi, Provenzi Massimo, Paolo Indolfi, Pession Andrea, Nantron Marilina, Andrea Di Cataldo, Carlo Morosi, Daniela Perotti, Serena Catania, Franca Fossati Bellani, Paola Collini, AIEOP Wilms Tumor Working Group
Pediatric Oncology Unit, Department of Hematology and Pediatric Hematology-Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy. Electronic address: ., Urology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy., Clinical Epidemiology and Trials Organization Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy., Department of Radiology and Radiotherapy, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy., Pediatric Hematology and Oncology Unit, Ospedale dei Bambini G. di Cristina, Palermo, Italy., Pediatric Oncology Unit, Department of Hematology and Pediatric Hematology-Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy., Pediatric Onco-Hematology, Stem Cell Transplantation and Cellular, Therapy Division, Regina Margherita Children's Hospital, Torino, Italy., Pediatric Unit, Ospedali Riuniti Bergamo, Bergamo, Italy., Pediatric Oncology, Pediatric Department, II University, Napoli, Italy., Pediatric Hematology and Oncology Unit "Lalla Serà gnoli", Bologna University, Bologna, Italy., Department of Hematology and Oncology, Istituto G. Gaslini, Genova, Italy., Pediatric Hematology and Oncology, Catania University, Catania, Italy., Department of Predictive & Preventive Medicine, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy., Department of Pathology and Laboratory Medicine, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.