PortalPublikationenAllogeneic hematopoietic SCT for alpha-mannosidosis

Allogeneic hematopoietic SCT for alpha-mannosidosis

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Allogeneic hematopoietic SCT for alpha-mannosidosis : an analysis of 17 patients. / Mynarek, M; Tolar, J; Albert, M H; Escolar, M L; Boelens, J J; Cowan, M J; Finnegan, N; Glomstein, A; Jacobsohn, D A; Kühl, J S; Yabe, H; Kurtzberg, J; Malm, D; Orchard, P J; Klein, C; Lücke, T; Sykora, K-W.

in: BONE MARROW TRANSPL, Jahrgang 47, Nr. 3, 03.2012, S. 352-9.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

Mynarek, M, Tolar, J, Albert, MH, Escolar, ML, Boelens, JJ, Cowan, MJ, Finnegan, N, Glomstein, A, Jacobsohn, DA, Kühl, JS, Yabe, H, Kurtzberg, J, Malm, D, Orchard, PJ, Klein, C, Lücke, T & Sykora, K-W 2012, 'Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients', BONE MARROW TRANSPL, Jg. 47, Nr. 3, S. 352-9. https://doi.org/10.1038/bmt.2011.99

APA

Mynarek, M., Tolar, J., Albert, M. H., Escolar, M. L., Boelens, J. J., Cowan, M. J., Finnegan, N., Glomstein, A., Jacobsohn, D. A., Kühl, J. S., Yabe, H., Kurtzberg, J., Malm, D., Orchard, P. J., Klein, C., Lücke, T., & Sykora, K-W. (2012). Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. BONE MARROW TRANSPL, 47(3), 352-9. https://doi.org/10.1038/bmt.2011.99

Vancouver

Mynarek M, Tolar J, Albert MH, Escolar ML, Boelens JJ, Cowan MJ et al. Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. BONE MARROW TRANSPL. 2012 Mär;47(3):352-9. https://doi.org/10.1038/bmt.2011.99

Bibtex

@article{5706cafbdec44021a0362992c98bbaba,
title = "Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients",
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.",
keywords = "Adolescent, Adult, Child, Child, Preschool, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Infant, Male, Medical Oncology, Retrospective Studies, Transplantation, Homologous, Treatment Outcome, alpha-Mannosidosis, Journal Article, Multicenter Study",
author = "M Mynarek and J Tolar and Albert, {M H} and Escolar, {M L} and Boelens, {J J} and Cowan, {M J} and N Finnegan and A Glomstein and Jacobsohn, {D A} and K{\"u}hl, {J S} and H Yabe and J Kurtzberg and D Malm and Orchard, {P J} and C Klein and T L{\"u}cke and K-W Sykora",
year = "2012",
month = mar,
doi = "10.1038/bmt.2011.99",
language = "English",
volume = "47",
pages = "352--9",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "3",

}

RIS

TY - JOUR

T1 - Allogeneic hematopoietic SCT for alpha-mannosidosis

T2 - an analysis of 17 patients

AU - Mynarek, M

AU - Tolar, J

AU - Albert, M H

AU - Escolar, M L

AU - Boelens, J J

AU - Cowan, M J

AU - Finnegan, N

AU - Glomstein, A

AU - Jacobsohn, D A

AU - Kühl, J S

AU - Yabe, H

AU - Kurtzberg, J

AU - Malm, D

AU - Orchard, P J

AU - Klein, C

AU - Lücke, T

AU - Sykora, K-W

PY - 2012/3

Y1 - 2012/3

N2 - 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.

AB - 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.

KW - Adolescent

KW - Adult

KW - Child

KW - Child, Preschool

KW - Female

KW - Follow-Up Studies

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - Infant

KW - Male

KW - Medical Oncology

KW - Retrospective Studies

KW - Transplantation, Homologous

KW - Treatment Outcome

KW - alpha-Mannosidosis

KW - Journal Article

KW - Multicenter Study

U2 - 10.1038/bmt.2011.99

DO - 10.1038/bmt.2011.99

M3 - SCORING: Journal article

C2 - 21552297

VL - 47

SP - 352

EP - 359

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 3

ER -