Claudia Pasqualini1, Rhoikos Furtwängler2, Harm van Tinteren3, Roberto A P Teixeira4, Tomas Acha5, Lisa Howell6, Gordan Vujanic7, Jan Godzinski8, Patrick Melchior9, Anne M Smets10, Aurore Coulomb-L'Hermine11, Hervé Brisse12, Kathy Pritchard-Jones13, Christophe Bergeron14, Beatriz de Camargo15, Marry M van den Heuvel-Eibrink16, Norbert Graf17, Arnauld C Verschuur18. 1. Children and Adolescents Oncology Department, Gustave Roussy, Villejuif, France. Electronic address: claudia.pasqualini@gustaveroussy.fr. 2. Department of Pediatric Haematology/Oncology, Saarland University Hospital, Homburg, Germany. Electronic address: rhoikos.furtwaengler@uks.eu. 3. Biometrics Department, Netherlands Cancer Institute, Amsterdam, the Netherlands. Electronic address: h.v.tinteren@nki.nl. 4. Instituto Do Tratamento Do Câncer Infantil, Department of Pediatrics, São Paulo University, São Paulo, Brazil. Electronic address: rapteixeira@uol.com.br. 5. Hospital Materno-Infantil "Carlos Haya", Department of Pediatrics, Malaga, Spain. Electronic address: tomas.acha.sspa@juntadeandalucia.es. 6. Department of Oncology, Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom. Electronic address: lisa.howell@alderhey.nhs.uk. 7. Department of Pathology, Sidra Medicine, Doha, Qatar. Electronic address: gvujanic@sidra.org. 8. Department of Pediatric Surgery, Marciniak Hospital Wroclaw, Wroclaw, Poland; Department of Pediatric Traumatology and Emergency Medicine, Medical University, Wroclaw, Poland. Electronic address: jgodzin@wp.pl. 9. Department of Radiation Oncology, Saarland University Hospital, Homburg, Germany. Electronic address: patrick.melchior@uks.eu. 10. Department of Radiology and Nuclear Medicine, Academic Medical Center, Amsterdam, the Netherlands. Electronic address: a.m.smets@amc.uva.nl. 11. Department of Pathology, Hospital D'Enfants Armand Trousseau, Paris, France. Electronic address: aurore.coulomb@aphp.fr. 12. Department of Radiology, Curie Institut, Paris, France. Electronic address: herve.brisse@curie.fr. 13. Great Ormond Street Institute of Child Health, University College London, London, UK. Electronic address: k.pritchard-jones@ucl.ac.uk. 14. Pediatric Onco-Haematology Department, Centre Leon Berard, Lyon, France. Electronic address: christophe.bergeron@ihope.fr. 15. Instituto Nacional Do Cancer, Pediatric Onco-Haematology, Rio de Janeiro, Brazil. Electronic address: bdecamar@terra.com.br. 16. Oncology, Princess Maxima Centre for Pediatric Oncology, Utrecht, the Netherlands; Dutch Childhood Oncology Group, The Hague, the Netherlands. Electronic address: m.m.vandenheuvel-eibrink@prinsesmaximacentrum.nl. 17. Department of Pediatric Haematology/Oncology, Saarland University Hospital, Homburg, Germany. Electronic address: graf@uks.eu. 18. Pediatric Onco-Haematology Department, Hopital de La Timone, AP-HM, Marseille, France. Electronic address: arnauld.verschuur@ap-hm.fr.
Abstract
INTRODUCTION: High-risk (HR) metastatic (stage IV) Wilms tumours (WTs) have a particular poor outcome. METHODS: 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. RESULTS: 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. CONCLUSION: These results call for new treatment approaches for patients with HR stage IV WT.
INTRODUCTION: High-risk (HR) metastatic (stage IV) Wilms tumours (WTs) have a particular poor outcome. METHODS: 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. RESULTS: 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. CONCLUSION: These results call for new treatment approaches for patients with HR stage IV WT.
Authors: Kevin X Liu; Yu-Hui Chen; David Kozono; Raymond H Mak; Patrick J Boyle; Katherine A Janeway; Elizabeth A Mullen; Karen J Marcus Journal: Adv Radiat Oncol Date: 2020-09-23
Authors: Prakriti Roy; Sophie E van Peer; Martin M de Witte; Godelieve A M Tytgat; Henrike E Karim-Kos; Martine van Grotel; Cees P van de Ven; Annelies M C Mavinkurve-Groothuis; Johannes H M Merks; Roland P Kuiper; Janna A Hol; Geert O R Janssens; Ronald R de Krijger; Marjolijn C J Jongmans; Jarno Drost; Alida F W van der Steeg; Annemieke S Littooij; Marc H W A Wijnen; Harm van Tinteren; Marry M van den Heuvel-Eibrink Journal: PLoS One Date: 2022-01-13 Impact factor: 3.240