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

Eur J Cancer. 2020 Mar;128:38-46. doi: 10.1016/j.ejca.2020.01.001. Epub 2020 Mar 5.

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.

Keywords: Anaplasia; Blastema; Cancer; Child; TP53; Wilms.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Child
  • Child, Preschool
  • Dactinomycin / therapeutic use
  • Disease Progression
  • Disease-Free Survival
  • Female
  • Humans
  • Infant
  • Kidney Neoplasms / drug therapy*
  • Kidney Neoplasms / mortality
  • Kidney Neoplasms / pathology
  • Male
  • Neoplasm Recurrence, Local / epidemiology*
  • Neoplasm Recurrence, Local / pathology
  • Neoplasm Recurrence, Local / prevention & control
  • Neoplasm Staging
  • Prospective Studies
  • Survival Rate
  • Time Factors
  • Vincristine / therapeutic use
  • Wilms Tumor / drug therapy*
  • Wilms Tumor / mortality
  • Wilms Tumor / pathology

Substances

  • Dactinomycin
  • Vincristine

Supplementary concepts

  • SIOP protocol