Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients.
Abstract
Umbilical cord blood (UCB) transplantation (UCBT) is a curative procedure for patients
with hematologic malignancies and genetic disorders and expands access to transplantation
for non-Caucasian patients unable to find a fully matched unrelated donor. In 2011,
the US Food and Drug Administration required that unrelated UCBT be performed using
either licensed UCB or unlicensed UCB under the Investigational New Drug (IND) program.
The National Marrow Donor Program manages an IND under which 2456 patients (1499 adults
and 957 children, 564 with malignant diseases and 393 with nonmalignant diseases)
underwent single or double UCBT between October 2011 and December 2016. The median
patient age was 31 years (range, <1 to 81 years), and 50% of children and 36% of adults
were non-Caucasian. The median time to neutrophil engraftment (ie, absolute neutrophil
count ≥500/mm<sup>3</sup>) was 22 days for adults, 20 days for pediatric patients
with malignant diseases, and 19 days for pediatric patients with nonmalignant diseases,
with corresponding rates of engraftment at 42 days of 89%, 88%, and 90%. In these
3 groups of patients, the incidence of acute graft-versus-host disease (GVHD) grade
II-IV was 35%, 32%, and 24%; the incidence of chronic GVHD was 24%, 26%, and 24%;
and 1-year overall survival (OS) was 57%, 71%, and 79%, respectively. In multivariate
analysis, younger age, lower Hematopoietic Cell Transplantation-Specific Comorbidity
Index, early-stage chemotherapy-sensitive disease, and higher performance score were
predictive of improved OS for adults. In a subset analysis of children with malignancies
undergoing single UCBT, the use of either licensed UCB (n = 48) or unlicensed UCB
(n = 382) was associated with similar engraftment and survival. The use of unlicensed
UCB units is safe and effective and provides an important graft source for a diverse
population.
Type
Journal articleSubject
Fetal BloodHumans
Hematologic Neoplasms
Graft vs Host Disease
Cord Blood Stem Cell Transplantation
Hematopoietic Stem Cell Transplantation
Adolescent
Adult
Aged
Aged, 80 and over
Middle Aged
Child
Child, Preschool
Infant
Young Adult
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https://hdl.handle.net/10161/24565Published Version (Please cite this version)
10.1016/j.bbmt.2019.11.016Publication Info
Ballen, Karen; Logan, Brent R; Chitphakdithai, Pintip; Kuxhausen, Michelle; Spellman,
Stephen R; Adams, Alexia; ... Miller, John P (2020). Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for
a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients. Biology of blood and marrow transplantation : journal of the American Society for
Blood and Marrow Transplantation, 26(4). pp. 745-757. 10.1016/j.bbmt.2019.11.016. Retrieved from https://hdl.handle.net/10161/24565.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina

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