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

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• 作者：Tam C. Ha^a ; ^k ; ^{ha.tam.cam@nccs.com.sg} ; Filippo Spreafico^b ; Norbert Graf^c ; Sandro Dallorso^d ; Jeffrey S. Dome^e ; Marcio Malogolowkin^f ; Rhoikos Furtwä ; ngler^c ; Juliet P. Hale^g ; Veronica Moroz^h ; David Machinⁱ ; Kathy Pritchard-Jones^j ; ^{k.pritchard-jones@ich.ucl.ac.uk}
• 关键词：Wilms&rsquo ; tumour ; Relapsed Wilms&rsquo ; tumour ; High dose chemotherapy
• 刊名：European Journal of Cancer
• 出版年：2013
• 出版时间：January, 2013
• 年：2013
• 卷：49
• 期：1
• 页码：194-210
• 全文大小：666 K

文摘

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.