High dose therapy for recurrent Wilms' tumor.

The prognosis for most patients with newly diagnosed Wilms' tumor (WT) is excellent, but those who have recurrent or refractory disease have a significantly worse prognosis. High dose chemotherapy (HDT) requiring autologous stem-cell rescue has been tried for recurrent WT with variable results, but no randomized clinical trial has ever been performed. A recent review by T.C. Ha and colleagues of the published cases to date suggests HDT may be beneficial to the very high risk subset of patients with recurrent WT, but this conclusion is fraught with the caveats of any retrospective review. A prospective clinical trial would be challenging to complete but with careful planning and international collaboration it could be accomplished. Such a trial is likely the only way to know with certainty the safety and efficacy of HDT for recurrent WT.