Advances in the clinical management of high-risk Wilms tumors


Michael V. Ortiz, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Christa Koenig, Division of Pediatric Hematology and Oncology, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.
Amy E. Armstrong, Division of Pediatric Hematology/Oncology, Washington University School of Medicine, St. Louis, Missouri, USA.
Jesper Brok, Developmental Biology and Cancer Research and Teaching Department, University College London Great Ormond Street Institute of Child Health, London, UK.
Beatriz de Camargo, Pediatric Hematology and Oncology Program, Research Center, Instituto Nacional de Cancer, Rio de Janeiro, Brazil.
Annelies M. Mavinkurve-Groothuis, Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands.
Thelma B. Herrera, Unidad Nacional de Oncologia Pediatrica, Guatemala City, Guatemala.
Rajkumar Venkatramani, Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Cancer Center, Baylor College of Medicine, Houston, Texas, USA.
Andrew D. Woods, Children's Cancer Therapy Development Institute, Beaverton, Oregon, USA.
Jeffrey S. Dome, Division of Oncology, Children's National Hospital and Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.
Filippo Spreafico, Pediatric Oncology Unit, Department of Medical Oncology and Hematology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.

Document Type

Journal Article

Publication Date



Pediatric blood & cancer








COG; SIOP; Wilms tumor; high risk; nephroblastoma; relapsed


Outcomes are excellent for the majority of patients with Wilms tumors (WT). However, there remain WT subgroups for which the survival rate is approximately 50% or lower. Acknowledging that the composition of this high-risk group has changed over time reflecting improvements in therapy, we introduce the authors' view of the historical and current approach to the classification and treatment of high-risk WT. For this review, we consider high-risk WT to include patients with newly diagnosed metastatic blastemal-type or diffuse anaplastic histology, those who relapse after having been initially treated with three or more different chemotherapeutics, or those who relapse more than once. In certain low- or low middle-income settings, socio-economic factors expand the definition of what constitutes a high-risk WT. As conventional therapies are inadequate to cure the majority of high-risk WT patients, advancement of laboratory and early-phase clinical investigations to identify active agents is urgently needed.