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Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide.

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Date
2019-02
Authors
Horwitz, Mitchell E
Wease, Stephen
Blackwell, Beth
Valcarcel, David
Frassoni, Francesco
Boelens, Jaap Jan
Nierkens, Stefan
Jagasia, Madan
Wagner, John E
Kuball, Jurgen
Koh, Liang Piu
Majhail, Navneet S
Stiff, Patrick J
Hanna, Rabi
Hwang, William YK
Kurtzberg, Joanne
Cilloni, Daniela
Freedman, Laurence S
Montesinos, Pau
Sanz, Guillermo
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Abstract
<h4>Purpose</h4>Increasing the number of hematopoietic stem and progenitor cells within an umbilical cord blood (UCB) graft shortens the time to hematopoietic recovery after UCB transplantation. In this study, we assessed the safety and efficacy of a UCB graft that was expanded ex vivo in the presence of nicotinamide and transplanted after myeloablative conditioning as a stand-alone hematopoietic stem-cell graft.<h4>Methods</h4>Thirty-six patients with hematologic malignancies underwent transplantation at 11 sites.<h4>Results</h4>The cumulative incidence of neutrophil engraftment at day 42 was 94%. Two patients experienced secondary graft failure attributable to viral infections. Hematopoietic recovery was compared with that observed in recipients of standard UCB transplantation as reported to the Center for International Blood and Marrow Transplant Research (n = 146). The median time to neutrophil recovery was 11.5 days (95% CI, 9 to 14 days) for recipients of nicotinamide-expanded UCB and 21 days (95% CI, 20 to 23 days) for the comparator ( P < .001). The median time to platelet recovery was 34 days (95% CI, 32 to 42 days) and 46 days (95% CI, 42 to 50 days) for the expanded and the comparator cohorts, respectively ( P < .001). The cumulative incidence of grade 2 to 4 acute graft-versus-host disease (GVHD) at day 100 was 44%, and grade 3 and 4 acute GVHD at day 100 was 11%. The cumulative incidence at 2 years of all chronic GVHD was 40%, and moderate/severe chronic GVHD was 10%. The 2-year cumulative incidences of nonrelapse mortality and relapse were 24% and 33%, respectively. The 2-year probabilities of overall and disease-free survival were 51% and 43%, respectively.<h4>Conclusion</h4>UCB expanded ex vivo with nicotinamide shortens median neutrophil recovery by 9.5 days (95% CI, 7 to 12 days) and median platelet recovery by 12 days (95% CI, 3 to 16.5 days). This trial establishes feasibility, safety, and efficacy of an ex vivo expanded UCB unit as a stand-alone graft.
Type
Journal article
Subject
Neutrophils
Fetal Blood
Humans
Hematologic Neoplasms
Graft vs Host Disease
Niacinamide
Disease-Free Survival
Transplantation Conditioning
Cord Blood Stem Cell Transplantation
Graft Survival
Adolescent
Adult
Middle Aged
Female
Male
Young Adult
Permalink
https://hdl.handle.net/10161/24579
Published Version (Please cite this version)
10.1200/jco.18.00053
Publication Info
Horwitz, Mitchell E; Wease, Stephen; Blackwell, Beth; Valcarcel, David; Frassoni, Francesco; Boelens, Jaap Jan; ... Sanz, Guillermo (2019). Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 37(5). pp. 367-374. 10.1200/jco.18.00053. Retrieved from https://hdl.handle.net/10161/24579.
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Scholars@Duke

Horwitz

Mitchell Eric Horwitz

Professor of Medicine
Allogeneic stem cell transplantation using umbilical cord blood grafts; Allogenic stem cell transplantation for Sickle Cell Disease; Prevention of acute graft versus host disease through donor stem cell graft manipulation; Improving immune recovery following alternative donor stem cell transplantation using donor graft manipulation or third party thymus transplantation.
Kurtzberg

Joanne Kurtzberg

Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg conducts both clinical and laboratory-based translational research efforts, all involving various aspects of normal and malignant hematopoiesis. In the laboratory, her early work focused on studies determining the mechanisms that regulate the choice between the various pathways of differentiation available to the pluripotent hematopoietic stem cell. Her laboratory established a CD7+ cell line, DU.528, capable of multilineage differentiation as well as self-renewal, and subse
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