Efficacy of high-dose chemotherapy and autologous stem cell transplant for recurrent Wilms' tumor: a meta-analysis

J Pediatr Hematol Oncol. 2010 Aug;32(6):454-61. doi: 10.1097/MPH.0b013e3181e001c2.


Long-term survival of relapsed Wilms' tumor patients is about 40% to 70%. Modern second-line treatment consists of either (a) salvage chemotherapy+/-radiation therapy (CT) or (b) chemotherapy followed by high-dose chemotherapy and autologous hematopoietic stem cell rescue (ASCR). Here, we conduct an individual patient data meta-analysis on 100 patients collected from 6 studies to determine characteristics that predict survival in relapsed patients who received ASCR therapy. We compare these results with survival data on 118 CT treated patients from 2 recently published studies. Four year overall survival among the combined ASCR treated patients was 54.1% (95% CI: 42.8-64.1%). The ASCR patients who only relapsed in the lungs had higher 4-years survival rates 77.7% (58.6% to 88.8%) than those who relapsed in other locations and/or suffered multiple relapses 41.6% (24.8% to 57.6%). Although lung-only relapse is considered a favorable prognostic factor, there was no clear advantage for the patients treated with salvage chemotherapy. Four-year survival rates among stage I-II patients were about 30% higher with CT than ASCR, but the 2 were comparable for stage III-IV patients. These findings suggest salvage chemotherapy is typically the better choice for relapsed Wilms' tumor patients, ASCR could be considered for stage III-IV patients with a lung-only relapse.

Publication types

  • Meta-Analysis

MeSH terms

  • Adolescent
  • Antineoplastic Agents / therapeutic use*
  • Child
  • Child, Preschool
  • Combined Modality Therapy
  • Female
  • Humans
  • Kaplan-Meier Estimate
  • Kidney Neoplasms / mortality
  • Kidney Neoplasms / therapy*
  • Male
  • Neoplasm Recurrence, Local / mortality
  • Neoplasm Recurrence, Local / therapy*
  • Neoplasm Staging
  • Salvage Therapy / methods*
  • Stem Cell Transplantation
  • Wilms Tumor / mortality
  • Wilms Tumor / therapy*


  • Antineoplastic Agents