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Efficacy of High-dose Chemotherapy and Autologous Stem Cell transplant for Recurrent Wilms' Tumor: A Meta-analysis

Presson, Angela PhD* †; Moore, Theodore B. MD; Kempert, Pamela MD

Journal of Pediatric Hematology/Oncology: August 2010 - Volume 32 - Issue 6 - p 454-461
doi: 10.1097/MPH.0b013e3181e001c2
Original Articles

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.

*Department of Biostatistics, UCLA School of Public Health

Division of Hematology Oncology, Department of Pediatrics, Mattel Children's Hospital at UCLA, CA

Reprints: Pamela Kempert, MD, Division of Hematology Oncology, Department of Pediatrics, Mattel Children's Hospital at UCLA, room A2-410 MDCC 10833 Le Conte, Los Angeles, CA 90095-1752 (e-mail:

Received for publication October 16, 2009; accepted March 1, 2010

© 2010 Lippincott Williams & Wilkins, Inc.