Outcome of patients with stage IV high-risk Wilms tumour treated according to the SIOP2001 protocol: A report of the SIOP Renal Tumour Study Group.

High-risk (HR) metastatic (stage IV) Wilms tumours (WTs) have a particular poor outcome.

Here, we report the results of HR (diffuse anaplastic [DA] or blastemal type [BT]) stage IV WT treated patients according to the HR arm in the SIOP2001 prospective study.

From January 2002 to August 2014, 3559 patients with WT were included in the SIOP2001 trial. Among the 525 patients (15%) with metastatic WT, 74 (14%) had stage IV HR-WT. The median age at diagnosis was 5.5 years (range: 1.4-18.3). Thirty-four patients (47%) had BT-WT and 40 (53%) had DA-WT. Five-year event-free survival rates were 44 ± 17% and 28 ± 15% for BT-WT and DA-WT, respectively (p = 0.09). Five-year overall survival rates were 53 ± 17% and 29 ± 16% for BT-WT and DA-WT, respectively (p = 0.03). Metastatic complete response after preoperative treatment was significantly associated with outcome in univariate and multivariate analyses (hazards ratio = 0.3; p = 0.01). Postoperative radiotherapy of metastatic sites might also be beneficial. Forty-three of 74 patients experienced a relapse or progression predominantly in the lungs (80%). The median time to relapse/progression after diagnosis was 7.3 months (range: 1.6-33.3) and 4.9 months (range: 0.7-28.4) for BT-WT and DA-WT, respectively (p = 0.67). This is the first prospective evidence of inferior survival of stage IV BT-WT as compared with historical intermediate-risk WT. Survival of patients with stage IV DA-WT has not improved compared to the previous SIOP93-01 study.

These results call for new treatment approaches for patients with HR stage IV WT.

European journal of cancer (Oxford, England : 1990). 2020 Feb 07 [Epub ahead of print]

Claudia Pasqualini, Rhoikos Furtwängler, Harm van Tinteren, Roberto A P Teixeira, Tomas Acha, Lisa Howell, Gordan Vujanic, Jan Godzinski, Patrick Melchior, Anne M Smets, Aurore Coulomb-L'Hermine, Hervé Brisse, Kathy Pritchard-Jones, Christophe Bergeron, Beatriz de Camargo, Marry M van den Heuvel-Eibrink, Norbert Graf, Arnauld C Verschuur

Children and Adolescents Oncology Department, Gustave Roussy, Villejuif, France. Electronic address: ., Department of Pediatric Haematology/Oncology, Saarland University Hospital, Homburg, Germany. Electronic address: ., Biometrics Department, Netherlands Cancer Institute, Amsterdam, the Netherlands. Electronic address: ., Instituto Do Tratamento Do Câncer Infantil, Department of Pediatrics, São Paulo University, São Paulo, Brazil. Electronic address: ., Hospital Materno-Infantil "Carlos Haya", Department of Pediatrics, Malaga, Spain. Electronic address: ., Department of Oncology, Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom. Electronic address: ., Department of Pathology, Sidra Medicine, Doha, Qatar. Electronic address: ., Department of Pediatric Surgery, Marciniak Hospital Wroclaw, Wroclaw, Poland; Department of Pediatric Traumatology and Emergency Medicine, Medical University, Wroclaw, Poland. Electronic address: ., Department of Radiation Oncology, Saarland University Hospital, Homburg, Germany. Electronic address: ., Department of Radiology and Nuclear Medicine, Academic Medical Center, Amsterdam, the Netherlands. Electronic address: ., Department of Pathology, Hospital D'Enfants Armand Trousseau, Paris, France. Electronic address: ., Department of Radiology, Curie Institut, Paris, France. Electronic address: ., Great Ormond Street Institute of Child Health, University College London, London, UK. Electronic address: ., Pediatric Onco-Haematology Department, Centre Leon Berard, Lyon, France. Electronic address: ., Instituto Nacional Do Cancer, Pediatric Onco-Haematology, Rio de Janeiro, Brazil. Electronic address: ., Oncology, Princess Maxima Centre for Pediatric Oncology, Utrecht, the Netherlands; Dutch Childhood Oncology Group, The Hague, the Netherlands. Electronic address: ., Department of Pediatric Haematology/Oncology, Saarland University Hospital, Homburg, Germany. Electronic address: ., Pediatric Onco-Haematology Department, Hopital de La Timone, AP-HM, Marseille, France. Electronic address: .