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Outcomes following autologous hematopoietic stem cell transplant for patients with relapsed Wilms' tumor: a CIBMTR retrospective analysis. Bone Marrow Transplant 2017 Nov;52(11):1549-1555

Date

09/05/2017

Pubmed ID

28869618

Pubmed Central ID

PMC5665725

DOI

10.1038/bmt.2017.178

Scopus ID

2-s2.0-85032633238 (requires institutional sign-in at Scopus site)   21 Citations

Abstract

Despite the marked improvement in the overall survival (OS) for patients diagnosed with Wilms' tumor (WT), the outcomes for those who experience relapse have remained disappointing. We describe the outcomes of 253 patients with relapsed WT who received high-dose chemotherapy (HDT) followed by autologous hematopoietic stem cell transplant (HCT) between 1990 and 2013, and were reported to the Center for International Blood and Marrow Transplantation Research. The 5-year estimates for event-free survival (EFS) and OS were 36% (95% confidence interval (CI); 29-43%) and 45% (95 CI; 38-51%), respectively. Relapse of primary disease was the cause of death in 81% of the population. EFS, OS, relapse and transplant-related mortality showed no significant differences when broken down by disease status at transplant, time from diagnosis to transplant, year of transplant or conditioning regimen. Our data suggest that HDT followed by autologous HCT for relapsed WT is well tolerated and outcomes are similar to those reported in the literature. As attempts to conduct a randomized trial comparing maintenance chemotherapy with consolidation versus HDT followed by stem cell transplant have failed, one should balance the potential benefits with the yet unknown long-term risks. As disease recurrence continues to be the most common cause of death, future research should focus on the development of consolidation therapies for those patients achieving complete response to therapy.

Author List

Malogolowkin MH, Hemmer MT, Le-Rademacher J, Hale GA, Mehta PA, Smith AR, Kitko C, Abraham A, Abdel-Azim H, Dandoy C, Angel Diaz M, Gale RP, Guilcher G, Hayashi R, Jodele S, Kasow KA, MacMillian ML, Thakar M, Wirk BM, Woolfrey A, Thiel EL

Author

Elizabeth Thiel MD Associate Professor in the Medicine department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Child
Child, Preschool
Female
Hematopoietic Stem Cell Transplantation
Humans
Infant
Male
Recurrence
Retrospective Studies
Salvage Therapy
Survival Analysis
Transplantation, Autologous
Treatment Outcome
Wilms Tumor
Young Adult