Reduction in Mortality after Umbilical Cord Blood Transplantation in Children Over a 20-Year Period (1995-2014).
Abstract
Infections and graft-versus-host disease (GVHD) have historically resulted in high
mortality among children undergoing umbilical cord blood transplantation (UCBT). However,
recent advances in clinical practice have likely improved outcomes of these patients.
We conducted a retrospective cohort study of children (<18years of age) undergoing
UCBT at Duke University between January 1, 1995 and December 31, 2014. We compared
2-year all-cause and cause-specific mortality during 3 time periods based on year
of transplantation (1995 to 2001, 2002 to 2007, and 2008 to 2014). We used multivariable
Cox regression to identify demographic and UCBT characteristics that were associated
with all-cause mortality, transplantation-related mortality, and death from invasive
aspergillosis after adjustment for time period. During the 20-year study period 824
children underwent UCBT. Two-year all-cause mortality declined from 48% in 1995 to
2001 to 30% in 2008 to 2014 (P = .0002). White race and nonmalignant UCBT indications
were associated with lower mortality. Black children tended to have a higher risk
of death for which GVHD (18% versus 11%; P = .06) or graft failure (9% versus 3%;
P = .01) were contributory than white children. Comparing 2008 to 2014 with 1995 to
2001, more than half (59%) of the reduced mortality was attributable to a reduction
in infectious mortality, with 45% specifically related to reduced mortality from invasive
aspergillosis. Antifungal prophylaxis with voriconazole was associated with lower
mortality from invasive aspergillosis than low-dose amphotericin B lipid complex (hazard
ratio, .09; 95% confidence interval, .01 to .76). With the decline in mortality from
invasive aspergillosis, adenovirus and cytomegalovirus have become the most frequentinfectious
causes of death in children after UCBT. Advances in clinical practice over the past
20years improved survival of children after UCBT. Reduced mortality from infections,
particularly invasive aspergillosis, accounted for the largest improvement in survival
and was associated with use of voriconazole for antifungal prophylaxis.
Type
Journal articleSubject
HumansCord Blood Stem Cell Transplantation
Survival Rate
Retrospective Studies
Cohort Studies
Time Factors
History, 20th Century
History, 21st Century
Female
Male
Permalink
https://hdl.handle.net/10161/24602Published Version (Please cite this version)
10.1016/j.bbmt.2018.11.018Publication Info
Spees, Lisa P; Martin, Paul L; Kurtzberg, Joanne; Stokhuyzen, Andre; McGill, Lauren;
Prasad, Vinod K; ... Kelly, Matthew S (2019). Reduction in Mortality after Umbilical Cord Blood Transplantation in Children Over
a 20-Year Period (1995-2014). Biology of blood and marrow transplantation : journal of the American Society for
Blood and Marrow Transplantation, 25(4). pp. 756-763. 10.1016/j.bbmt.2018.11.018. Retrieved from https://hdl.handle.net/10161/24602.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.
Collections
More Info
Show full item recordScholars@Duke
Mehreen Arshad
Assistant Professor of Pediatrics
Timothy Alan Driscoll
Assistant Professor of Pediatrics
Dr. Driscoll participates in multi-institutional studies for the treatment of high
risk neuroblastoma patients using high dose chemotherapy with stem cell transplant
and the development of new therapies for high risk neuroblastoma patients.
Matthew Kelly
Associate Professor of Pediatrics
My research is broadly focused on elucidating the complex interactions that exist
between the host microbiome and exogenous pathogens that cause infections in children.
We have several ongoing projects evaluating: 1) the impact of the upper respiratory
microbiome on the risk of colonization and invasion by bacterial respiratory pathogens
among infants in Botswana; 2) associations between the gut microbiome of pediatric
stem cell transplant recipients and the risk of infections (bloodstream infec
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
Paul Langlie Martin
Professor of Pediatrics
For most of my career in Pediatric Hematology/Oncology I have focused on the use of
stem cell transplant for the treatment of pediatric leukemias (ALL, AML, CML and JMML)
and other non-malignant blood disorders, such as sickle cell disease, hemaphagocytic
disorders, Wiskott-Aldrich, aplastic anemia, Diamond-Blackfan Anemia, as well as inherited
metabolic diseases. In addition to focusing on determining the best use of stem cell
transplants for these disorders, I have also been involved in clinic
Kristin Marie Page
Assistant Professor of Pediatrics
Stem cell transplantation and/or cellular therapies can be used to treat a variety
of pediatric diseases including malignancies such as leukemia, lymphoma and myelodysplastic
syndrome in addition to certain non-malignant conditions (such as immune deficiencies,
inherited metabolic diseases, hemoglobinopathies, and bone marrow failure syndromes).
As the Director of the Pediatric Transplant and Cellular Therapy Survivorship Clinic,
my goal is optimize the care of survivors of pediatric stem cell t
Suhag H. Parikh
Associate Professor of Pediatrics
Stem cell transplantation for a variety of disorders - ranging from malignant diseases
such as leukemia, lymphoma and myelodysplastic syndrome to nonmalignant diseases such
as sickle cell disease, thalassemias, aplastic anemia, histiocytosis and leukodystrophies.
My clinical research interest is stem cell transplantation for children with primary
immune deficiency disorders and hemoglobinopathies such as sickle cell anemia,thalassemia
and other non-malignant disorders. In addition,I am intereste
Vinod K. Prasad
Professor of Pediatrics
1. Expanding the role of umbilical cord blood transplants for inherited metabolic
disorders. 2. Impact of histocompatibility and other determinants of alloreactivity
on clinical outcomes of unrelated cord blood transplants. 3. Studies to analyse the
impact of Killer Immunoglobulin receptors on the outcomes of hematopoietic stem cell
transplantation utilizing haploidentical, CD34 selected, familial grafts. 4. Propective
longitudinal study of serial monitoring of adenovirus in
Alan David Proia
Professor of Pathology
My research interests are focused on the pathology and genesis of diseases affecting
the eye and adjacent structures.
Patrick Casey Seed
Associate Professor of Pediatrics
We are studying human microbial ecology and the molecular basis for different bacterial
infections that are of relevance to both children and adults. Summaries of the research
areas are described below: 1. THE MOLECULAR BASIS FOR VIRULENCE OF UROPATHOGENIC
ESCHERICHIA COLI AND URINARY TRACT INFECTIONS. Uropathogenic Escherichia coli (UPEC)
is the leading cause of community-acquired urinary tract infections (UTIs). Over
100 million UTIs occur annually throughout the world inclu
William J. Steinbach
Samuel L. Katz Distinguished Professor of Pediatrics
My overall clinical and research focus is to improve the diagnosis, treatment, and
outcomes of immunocompromised patients, specifically those with invasive fungal infections.
My research enterprise is divided between my basic, translational, and clinical approaches.
My laboratory focuses on the molecular pathogenesis of Aspergillus fumigatus, a leading
invasive fungal killer, and the development of novel antifungal drugs and diagnostic
assays. I also oversee numerous translatio
This author no longer has a Scholars@Duke profile, so the information shown here reflects
their Duke status at the time this item was deposited.
Anthony D Sung
Associate Professor of Medicine
I am dedicated to the treatment of hematologic malignancies through cellular therapies
such as hematopoietic stem cell transplantation (HCT). My research focuses on strategies
to reduce complications of HCT and ranges from preclinical studies using murine models
of HCT to Phase 1 and Phase 2 clinical trials. Areas of interest include the role
of the microbiota (the trillions of bacteria living in and on our bodies), nutrition,
and exercise in modulating HCT outcomes such as graft-versus-host
Alphabetical list of authors with Scholars@Duke profiles.

Articles written by Duke faculty are made available through the campus open access policy. For more information see: Duke Open Access Policy
Rights for Collection: Scholarly Articles
Works are deposited here by their authors, and represent their research and opinions, not that of Duke University. Some materials and descriptions may include offensive content. More info