Abstract
Background
Among patients with nephroblastoma, those with bilateral disease are a unique population where maximising tumour control must be balanced with preserving renal parenchyma.
Methods
The SIOP 2001 protocol recommended surgery after neoadjuvant cycle(s) of Dactinomycin and Vincristine (AV) with response-adapted intensification, if needed. Adjuvant treatment was given based on the lesion with the worst histology.
Results
Three hundred and twenty seven patients with stage V disease were evaluable: 174 had bilateral Wilms tumour (BWT), 101 unilateral WT and contralateral nephroblastomatosis (NB) and 52 bilateral nephroblastomatosis. In these three groups, the estimated 5y-EFS was 76.1%, 84.6%, and 74.9%, respectively. AV chemotherapy alone was the successful chemotherapy for 58.7% of all the patients and 65.6% of the non-metastatic patients. Among the 174 patients with BWT, 149 (88.2%) had at least one nephron-sparing surgery. Twenty of 61 bilateral stage I patients were treated with four-week AV postoperatively achieving 94.4% 5y-EFS. At last follow-up, 87% of patients had normal renal function.
Conclusions
This study demonstrates that AV without anthracyclines is sufficient to achieve NSS and good survival in the majority of patients. For patients with bilateral stage I WT and intermediate risk histology, only four weeks adjuvant AV seems to be sufficient.
Clinical Trial Registration
NCT00047138
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Data availability
All data generated or analysed during this study are included in this published article [and its supplementary information files].
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Acknowledgements
We acknowledge the enormous efforts made by more than 1000 clinicians working at the 251 childhood cancer treatment centres from 26 countries who enroled and followed up with patients in this study, and the patients and their families for their participation. The SIOP Renal Tumours Study Group and all authors thank the members of the independent data monitoring committee: Max Coppes (British Columbia Cancer Agency, Canada), Otilia Dalesio (Netherlands Cancer Institute, Amsterdam), and Dietmar Schmidt (Mannheim, Germany). We also acknowledge Lucy Wilkie and Aidan McManus for editorial assistance with the journal style and formatting of the final draft.
Special Dedication We address a special memorial to Dr. Christophe Bergeron, dead in December 2023 who has initiated this work in the spectrum of BWT. He has played an important role in numerous initiatives and projects within SIOP and SFCE, including the SIOP Renal Tumour Study Group. He strongly contributed to our collaborative efforts in the field of paediatric renal tumours over many decades.
Funding
The SIOP WT 2001 study was funded by Cancer Research UK (grant C1188/A8687), the UK National Cancer Research Network and Children’s Cancer and Leukaemia Group (CCLG) (who supported the UK section), Société Française des Cancers de l’Enfant and Association Leon Berard Enfant Cancéreux and Enfant et Santé (who supported the French section), Gesellschaft fü r Pädiatrische Onkologie und Hämatologie and Deutsche Krebschilfe (grant 50-2709-Gr2, who supported the German section), Grupo Cooperativo Brasileiro para o Tratamento do Tumour de Wilms and Sociedade Brasileira de Oncologia Pediátrica (who supported the Brazilian section), the Spanish Society of Paediatric Haematology and Oncology and the Spanish Association Against Cancer (who supported the Spanish section) and SIOP-NL. K P-J is partly supported by the National Institute for Health Research Biomedical Research Centre Funding Scheme
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Contributions
Hélène Sudour-Bonnange: Conceptualisation, Investigation, Writing – Original Draft, Writing – Review & Editing., Harm van Tinteren: Methodology, Formal analysis, Writing – Review & Editing, Gema L Ramírez-Villar: Investigation, Resources, Writing – Review & Editing, Jan Godzinski: Investigation, Resources, Writing – Review & Editing, Sabine Irtan: Investigation, Resources, Writing – Review & Editing, Manfred Gessler: Investigation, Resources, Writing – Review & Editing, Tanzina Chowdhury: Investigation, Resources, Writing – Review & Editing, Georges Audry: Investigation, Resources, Writing – Review & Editing, Joerg Fuchs: Investigation, Resources, Writing – Review & Editing, Mark Powis: Investigation, Resources, Writing – Review & Editing, Cornelis P van de Ven: Investigation, Resources, Writing – Review & Editing, Bruce Okoye: Investigation, Resources, Writing – Review & Editing, Naima Smeulders: Investigation, Resources, Writing – Review & Editing, Gordan M Vujanic: Investigation, Resources, Writing – Review & Editing, Arnaud Verschuur: Investigation, Resources, Writing – Review & Editing, Aurore L’Herminé-Coulomb: Investigation, Resources, Writing – Review & Editing, Beatriz de Camargo: Investigation, Resources, Writing – Review & Editing, Joaquim Caetano de Aguirre Neto: Investigation, Resources, Writing – Review & Editing, Jens Peter Schenk: Investigation, Resources, Writing – Review & Editing, Mary M van den Heuvel-Eibrink: Investigation, Resources, Writing – Review & Editing, Kathy Pritchard-Jones: Investigation, Resources, Writing – Review & Editing, Norbert Graf: Investigation, Resources, Writing – Review & Editing, Christophe Bergeron: Conceptualisation, Investigation, and Rhoikos Furtwängler: Conceptualisation, Investigation, Writing – Review & Editing, Supervision.
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Ethics approval and consent to participate: national and local regulatory and ethical approvals were obtained according to national regulations.
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The parents or guardians signed a written informed consent for enrolment and treatment of their child in the SIOP 2001 study.
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Sudour-Bonnange, H., van Tinteren, H., Ramírez-Villar, G.L. et al. Characteristics and outcome of synchronous bilateral Wilms tumour in the SIOP WT 2001 Study: Report from the SIOP Renal Tumour Study Group (SIOP-RTSG). Br J Cancer 131, 972–981 (2024). https://doi.org/10.1038/s41416-024-02799-0
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DOI: https://doi.org/10.1038/s41416-024-02799-0
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