Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.
dc.contributor.author | Mynarek, M | |
dc.contributor.author | Tolar, J | |
dc.contributor.author | Albert, MH | |
dc.contributor.author | Escolar, ML | |
dc.contributor.author | Boelens, JJ | |
dc.contributor.author | Cowan, MJ | |
dc.contributor.author | Finnegan, N | |
dc.contributor.author | Glomstein, A | |
dc.contributor.author | Jacobsohn, DA | |
dc.contributor.author | Kühl, JS | |
dc.contributor.author | Yabe, H | |
dc.contributor.author | Kurtzberg, J | |
dc.contributor.author | Malm, D | |
dc.contributor.author | Orchard, PJ | |
dc.contributor.author | Klein, C | |
dc.contributor.author | Lücke, T | |
dc.contributor.author | Sykora, K-W | |
dc.date.accessioned | 2022-03-23T19:35:27Z | |
dc.date.available | 2022-03-23T19:35:27Z | |
dc.date.issued | 2012-03 | |
dc.date.updated | 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 | |
dc.identifier.issn | 0268-3369 | |
dc.identifier.issn | 1476-5365 | |
dc.identifier.uri | ||
dc.language | eng | |
dc.publisher | Springer Science and Business Media LLC | |
dc.relation.ispartof | Bone marrow transplantation | |
dc.relation.isversionof | 10.1038/bmt.2011.99 | |
dc.subject | Humans | |
dc.subject | alpha-Mannosidosis | |
dc.subject | Treatment Outcome | |
dc.subject | Hematopoietic Stem Cell Transplantation | |
dc.subject | Transplantation, Homologous | |
dc.subject | Retrospective Studies | |
dc.subject | Follow-Up Studies | |
dc.subject | Medical Oncology | |
dc.subject | Adolescent | |
dc.subject | Adult | |
dc.subject | Child | |
dc.subject | Child, Preschool | |
dc.subject | Infant | |
dc.subject | Female | |
dc.subject | 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 | |
pubs.organisational-group | School of Medicine | |
pubs.organisational-group | Clinical Science Departments | |
pubs.organisational-group | Institutes and Centers | |
pubs.organisational-group | Pathology | |
pubs.organisational-group | Pediatrics | |
pubs.organisational-group | Duke Cancer Institute | |
pubs.organisational-group | Institutes and Provost's Academic Units | |
pubs.organisational-group | Initiatives | |
pubs.organisational-group | Duke Innovation & Entrepreneurship | |
pubs.organisational-group | Pediatrics, Transplant and Cellular Therapy | |
pubs.publication-status | Published | |
pubs.volume | 47 |
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