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

Patrick J. Stiff, Pau Montesinos, Tony Peled, Efrat Landau, Noga Rosenheimer Goudsmid, Julie Mandel, Nira Hasson, Esti Olesinski, Ela Glukhman, David A. Snyder, Einat Galamidi Cohen, Orna Srur Kidron, Dalia Bracha, Dorit Harati, Keren Ben-Abu, Etty Freind, Laurence S. Freedman, Yael C. Cohen, Liraz Olmer, Raya BarishevVanderson Rocha, Eliane Gluckman, Mary M. Horowitz, Mary Eapen, Arnon Nagler, Guillermo Sanz

Research output: Contribution to journalArticlepeer-review

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 × 10 7 cell dose/kg and 1.64 × 10 5 cell dose/kg. Median NC and CD34 + fold expansion were 400 and 77, with a mean total CD34 infused of 9.7 × 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 <.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.

Original languageEnglish
Pages (from-to)1463-1470
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Volume24
Issue number7
DOIs
StatePublished - Jul 2018

Keywords

  • Ex vivo
  • Transplantation
  • Umbilical cord blood

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