An international strategy to determine the role of high dose therapy in recurrent Wilms' tumour

Tam C. Ha, Filippo Spreafico, Norbert Graf, Sandro Dallorso, Jeffrey S. Dome, Marcio Malogolowkin, Rhoikos Furtwängler, Juliet P. Hale, Veronica Moroz, David MacHin, Kathy Pritchard-Jones

Research output: Contribution to journalArticlepeer-review

40 Scopus citations


Purpose: To review event-free (EFS) and overall survival (OS) from publications describing outcome for children with relapsed Wilms' tumour. Comparisons are made between those receiving myeloablative high dose chemotherapy with autologous stem-cell rescue (HDT) and those not (NoHDT). Materials and methods: Relevant information was extracted from individual patient or summary data and 3-year EFS and OS rates established. These rates were combined in a weighted manner to derive hazard ratios (HRs). Results: Nineteen publications were identified (5 HDT, 6 NoHDT, 8 both). Pooling all studies suggested an advantage to HDT with a hazard ratio (HR) for EFS of 0.87 (95% confidence interval (CI) 0.67-1.12) and 0.94 (0.71-1.24) for OS. A stratified analysis confined to studies that provided individual patient data on both HDT and NoHDT gave HRs of 0.83 (0.56-1.24) and 0.92 (0.59-1.41). Further, analyses of risk groups, defined by treatment and/or histology prior to first relapse, suggested a HR for EFS of 0.90 (95% CI 0.62-1.31) for those of high and 0.50 (CI 0.31-0.82) for the very high risk patients. Conclusion: The evidence suggests, although there are many caveats since the information summarised here is not from randomised trials, a great deal of uncertainty concerning the role of HDT in patients following relapse after treatment for their Wilms' tumour. For each risk group we propose a randomised trial comparing a standard with a more intensive therapy with specific choice of regimen tailored to the risk group (and co-operative groups) concerned. A synthesis of updated evidence from studies in this overview together with any emerging studies and future trial information will form the basis for future evidence-based clinical decision-making.

Original languageEnglish (US)
Pages (from-to)194-210
Number of pages17
JournalEuropean Journal of Cancer
Issue number1
StatePublished - Jan 1 2013
Externally publishedYes


  • High dose chemotherapy
  • Relapsed Wilms' tumour
  • Wilms' tumour

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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