Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.

dc.contributor.author

Mynarek, M

dc.contributor.author

Tolar, J

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Albert, MH

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Escolar, ML

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Boelens, JJ

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Cowan, MJ

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Finnegan, N

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Glomstein, A

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Jacobsohn, DA

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Kühl, JS

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Yabe, H

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Kurtzberg, J

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Malm, D

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Orchard, PJ

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Klein, C

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Lücke, T

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Sykora, K-W

dc.date.accessioned

2022-03-23T19:35:27Z

dc.date.available

2022-03-23T19:35:27Z

dc.date.issued

2012-03

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2022-03-23T19:35:27Z

dc.description.abstract

Alpha-mannosidosis is a rare lysosomal storage disease. Hematopoietic SCT (HSCT) is usually recommended as a therapeutic option though reports are anecdotal to date. This retrospective multi institutional analysis describes 17 patients that were diagnosed at a median of 2.5 (1.1-23) years and underwent HSCT at a median of 3.6 (1.3-23.1) years. In all, 15 patients are alive (88%) after a median follow-up of 5.5 (2.1-12.6) years. Two patients died within the first 5 months after HSCT. Of the survivors, two developed severe acute GvHD (>=grade II) and six developed chronic GvHD. Three patients required re-transplantation because of graft failure. All 15 showed stable engraftment. The extent of the patients' developmental delay before HSCT varied over a wide range. After HSCT, patients made developmental progress, although normal development was not achieved. Hearing ability improved in some, but not in all patients. We conclude that HSCT is a feasible therapeutic option that may promote mental development in alpha-mannosidosis.

dc.identifier

bmt201199

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0268-3369

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1476-5365

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https://hdl.handle.net/10161/24659

dc.language

eng

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Springer Science and Business Media LLC

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Bone marrow transplantation

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10.1038/bmt.2011.99

dc.subject

Humans

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alpha-Mannosidosis

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Treatment Outcome

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Hematopoietic Stem Cell Transplantation

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Transplantation, Homologous

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Retrospective Studies

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Follow-Up Studies

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Medical Oncology

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Adolescent

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Adult

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Child

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Child, Preschool

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Infant

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Female

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Male

dc.title

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.

dc.type

Journal article

duke.contributor.orcid

Kurtzberg, J|0000-0002-3370-0703

pubs.begin-page

352

pubs.end-page

359

pubs.issue

3

pubs.organisational-group

Duke

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School of Medicine

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Clinical Science Departments

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Institutes and Centers

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Pathology

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Pediatrics

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Duke Cancer Institute

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Institutes and Provost's Academic Units

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Initiatives

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Duke Innovation & Entrepreneurship

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Pediatrics, Transplant and Cellular Therapy

pubs.publication-status

Published

pubs.volume

47

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