Cohort-Controlled Comparison of Umbilical Cord Blood Transplantation Using Carlecortemcel-L, a Single Progenitor-Enriched Cord Blood, to Double Cord Blood Unit Transplantation Journal Article


Authors: Stiff, P. J.; Montesinos, P; Peled, T.; Landau, E.; Goudsmid, N. R.; Mandel, J.; Hasson, N.; Olesinski, E.; Glukhman, E.; Snyder, D. A.; Cohen, E. G.; Kidron, O. S.; Bracha, D.; Harati, D.; Ben-Abu, K.; Freind, E.; Freedman, L. S.; Cohen, Y. C.; Olmer, L.; Barishev, R.; Rocha, V.; Gluckman, E.; Horowitz, M. M.; Eapen, M.; Nagler, A; Sanz, G
Article Title: Cohort-Controlled Comparison of Umbilical Cord Blood Transplantation Using Carlecortemcel-L, a Single Progenitor-Enriched Cord Blood, to Double Cord Blood Unit Transplantation
Abstract: Umbilical cord blood (UCB) transplantation has a high early mortality rate primarily related to transplanted stem cell dose. To decrease early mortality and enhance engraftment, a portion of selected cord blood units (20% to 50%) was expanded with cytokines and the copper chelator tetraethylenepentamine (carlecortemcel-L) and transplanted with the unmanipulated fraction after myeloablative conditioning. The primary endpoint was 100-day survival, which was compared with a contemporaneous double-unit cord blood transplantation (DUCBT) group. We enrolled 101 patients at 25 sites; the DUCBT comparison (n = 295) was selected from international registries using study eligibility criteria. Baseline carlecortemcel-L study group unit nucleated cell (NC) and CD34(+) were 3.06 x 10(7) cell dose/kg and 1.64 x 10(5) cell dose/kg. Median NC and CD34(+) fold expansion were 400 and 77, with a mean total CD34 infused of 9.7 x 10(5)/kg. The 100-day survival was 84.2% for the carlecortemcel-L study group versus 74.6% for the DUCBT group (odds ratio, .50; 95% CI, .26 to .95; P = .035). Survival at day 180 was similar for the 2 groups; the major cause of death after day 100 was opportunistic infections. Faster median neutrophil (21 days versus 28 days; P lt; .0001), and platelet (54 days versus 105 days; P = .008) engraftment was seen in the carlecortemcel-L study group; acute and chronic graft-versus-host disease rates were similar. In this multinational comparative study, transplanting expanded CD34(+) stem cells from a portion of a single UCB unit, with the remaining unmanipulated fraction improved 100-day survival compared with DUCBT control patients while facilitating myeloid and platelet engraftment. This trial was registered at www.clinicaltrials.gov as #NCT00469729.
Journal Title: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
ISSN: 1523-6536; 1083-8791
Publisher: The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc  
Journal Place: United States
Date Published: 2018
Language: eng
DOI/URL:
Notes: LR: 20180409; CI: Copyright (c) 2018; ClinicalTrials.gov/NCT00469729; JID: 9600628; OTO: NOTNLM; 2017/11/06 00:00 [received]; 2018/02/10 00:00 [accepted]; 2018/02/27 06:00 [pubmed]; 2018/02/27 06:00 [medline]; 2018/02/26 06:00 [entrez]; aheadofprint