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40
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Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
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URL Address
<a href="http://doi.org/10.1016/j.bbmt.2007.01.082" target="_blank" rel="noreferrer">http://doi.org/10.1016/j.bbmt.2007.01.082</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy
Publisher
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Biology Of Blood And Marrow Transplantation : Journal Of The American Society For Blood And Marrow Transplantation
Date
A point or period of time associated with an event in the lifecycle of the resource
2007
Subject
The topic of the resource
Child; Humans; Male; Survival Rate; Treatment Outcome; Cognition; Predictive Value of Tests; Motor Activity; Preschool; Outcomes; Adrenoleukodystrophy/complications/mortality/physiopathology/therapy; Cord Blood Stem Cell Transplantation/methods; Graft Survival; Graft vs Host Disease; Histocompatibility Testing; Language Development; Myeloablative Agonists/therapeutic use; Neurophysiology; Transplantation Conditioning/methods
Creator
An entity primarily responsible for making the resource
Beam D; Poe MD; Provenzale JM; Szabolcs P; Martin PL; Prasad V; Parikh S; Driscoll T; Mukundan S; Kurtzberg J; Escolar ML
Description
An account of the resource
Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents in young boys and adolescent boys. Allogeneic bone marrow transplantation has been used to halt progression of the disease. However, many patients lack suitable HLA- matched related donors and must rely on unmatched donors for a source of stem cells. The purpose of this study was to evaluate outcomes of unrelated donor umbilical cord blood transplantation after chemotherapy-based myeloablative conditioning and retrospectively determine if baseline studies correlate and help predict outcome. Between November 22, 1996, and November 3, 2005, 12 boys with X-linked ALD who lacked HL- matched related donors were referred to Duke University Medical Center for transplantation. These children were conditioned with myeloablative therapy including busulfan, cyclophosphamide, and antithymocyte globulin before receiving umbilical cord-blood transplants from unrelated donors. Baseline studies of neurophysiologic, neuroimaging, and neurodevelopmental status were performed and patients were subsequently evaluated for survival, engraftment, graft-versus-host disease, and neurodevelopmental outcomes. A substudy evaluated whether baseline neuroimaging and neurophysiologic studies correlated with cognitive and motor function and if these studies were predictive of posttransplantation outcomes. The umbilical cord blood grafts had normal levels of very long chain fatty acids. They delivered a median of 6.98 x 10(7) nucleated cells per kilogram of recipient body weight and were discordant for up to 4 of 6 HLA markers. Neutrophil engraftment occurred at a median of 22.9 days after transplantation. Three patients had grade II-IV acute graft-versus-host disease; 2 had extensive chronic graft-versus-host disease. Cumulative incidence of overall survival of the group at 6 months is 66.7% (95% confidence interval 39.9-93.3%). Median follow-up was 3.3 years (range 12 days to 6.3 years). As previously reported with bone marrow transplantation, symptomatic patients faired poorly with lower survival and rapid deterioration of neurologic function. This study included 3 patients transplanted at a very young age (2.6-3.5 years) before the onset of clinical symptoms who continue to develop at a normal rate for 3-5 years posttransplant. Although baseline Loes scores correlated with cognitive and motor outcome, neurophysiologic studies failed to show statistically significant differences. Transplantation of boys with X-linked ALD using partial HLA-matched umbilical cord blood yields similar results to those previously reported after bone marrow transplantation. Superior outcomes were seen in neurologically asymptomatic boys less than 3.5 years of age at the time of transplantation. Baseline Loes scores were a strong predictor of cognitive and motor outcome.
2007
Identifier
An unambiguous reference to the resource within a given context
<a href="http://doi.org/10.1016/j.bbmt.2007.01.082" target="_blank" rel="noreferrer">10.1016/j.bbmt.2007.01.082</a>
Rights
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Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Type
The nature or genre of the resource
Journal Article
2007
Adrenoleukodystrophy/complications/mortality/physiopathology/therapy
Backlog
Beam D
Biology Of Blood And Marrow Transplantation : Journal Of The American Society For Blood And Marrow Transplantation
Child
Cognition
Cord Blood Stem Cell Transplantation/methods
Driscoll T
Escolar ML
Graft Survival
Graft vs Host Disease
Histocompatibility Testing
Humans
Journal Article
Kurtzberg J
Language Development
Male
Martin PL
Motor Activity
Mukundan S
Myeloablative Agonists/therapeutic use
Neurophysiology
Outcomes
Parikh S
Poe MD
Prasad V
Predictive Value of Tests
Preschool
Provenzale JM
Survival Rate
Szabolcs P
Transplantation Conditioning/methods
Treatment Outcome
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Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
Backlog
URL Address
<a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=16538790" target="_blank" rel="noreferrer">http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=16538790</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
Surviving cancer in childhood can mean lifelong health issues
Publisher
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Lancet Oncology
Date
A point or period of time associated with an event in the lifecycle of the resource
2006
Subject
The topic of the resource
Child; Humans; Adult; Follow-Up Studies; Health Status; adolescent; PedPal Lit; Pain/etiology; Graft vs Host Disease; Survivors; Cataract/etiology; Hearing Loss/etiology; Hematopoietic Stem Cell Transplantation/adverse effects; Vision Disorders/etiology
Creator
An entity primarily responsible for making the resource
Senior K
Rights
Information about rights held in and over the resource
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Type
The nature or genre of the resource
Journal Article
Description
An account of the resource
2006
2006
Adolescent
Adult
Backlog
Cataract/etiology
Child
Follow-up Studies
Graft vs Host Disease
Health Status
Hearing Loss/etiology
Hematopoietic Stem Cell Transplantation/adverse effects
Humans
Journal Article
Lancet Oncology
Pain/etiology
PedPal Lit
Senior K
Survivors
Vision Disorders/etiology