Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes. Biol Blood Marrow Transplant 2017 Oct;23(10):1714-1721 PMID: 28684372 PMCID: PMC5605440

Pubmed ID





The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation with 1 or 2 cord blood units (UCB) between 2006 and 2012. While the trial concluded that survival was similar regardless of number of units infused, survival was better than previously reported. This prompted a comparison of survival of trial versus nontrial patients to determine the generalizability of trial results and whether survival was better because of the trial treatment regimen. During the trial period, 396 recipients of a single UCB unit met trial eligibility but were not enrolled. Trial patients (n = 100) received total body irradiation (TBI) 1320 cGy, cyclophosphamide 120 mg/kg, and fludarabine 75 mg/m (TCF). Nontrial patients either received the same regimen (n = 62; nontrial TCF) or alternative regimens (n = 334; nontrial regimens). Five-year survival between trial and nontrial patients conditioned with TCF was similar (70% versus 62%). However, 5-year survival was significantly lower with nontrial TBI-containing (47%; hazard ratio [HR], 1.97; P = .001) and chemotherapy-only regimens (49%; HR, 1.87; P = .007). The results of BMT CTN 0501 appear generalizable to the population of trial-eligible patients. The survival difference between the trial-specified regimen and other regimens indicate the importance of conditioning regimen for UCB transplantation.

Author List

Eapen M, Kurtzberg J, Zhang MJ, Hattersely G, Fei M, Mendizabal A, Chan KW, De Oliveira S, Schultz KR, Wall D, Horowitz MM, Wagner JE


Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Mary M. Horowitz MD, MS Center Director, Professor in the Medicine department at Medical College of Wisconsin
Mei-Jie Zhang PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin


2-s2.0-85026649100   1 Citations

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

Acute Disease
Child, Preschool
Combined Modality Therapy
Cord Blood Stem Cell Transplantation
Survival Analysis
Transplantation Conditioning
Treatment Outcome
Whole-Body Irradiation
Young Adult
jenkins-FCD Prod-321 98992d628744e349846c2f62ac68f241d7e1ea70