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Reduction in Mortality after Umbilical Cord Blood Transplantation in Children Over a 20-Year Period (1995-2014).

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Date
2019-04
Authors
Spees, Lisa P
Martin, Paul L
Kurtzberg, Joanne
Stokhuyzen, Andre
McGill, Lauren
Prasad, Vinod K
Driscoll, Timothy A
Parikh, Suhag H
Page, Kristin M
Vinesett, Richard
Severyn, Christopher
Sung, Anthony D
Proia, Alan D
Jenkins, Kirsten
Arshad, Mehreen
Steinbach, William J
Seed, Patrick C
Kelly, Matthew S
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(18 total)
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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 article
Subject
Humans
Cord 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/10161/24602
Published Version (Please cite this version)
10.1016/j.bbmt.2018.11.018
Publication 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.
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Scholars@Duke

Arshad

Mehreen Arshad

Assistant Professor of Pediatrics
Driscoll

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.
Kelly

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
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
Martin

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
Page

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
Parikh

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
Prasad

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
Proia

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
Steinbach

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.
Sung

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
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Alphabetical list of authors with Scholars@Duke profiles.
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