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

M. Mynarek; J. Tolar; M.H. Albert; M. Escolar; J.J. Boelens; M.J. Cowan; N. Finnegan; A. Glomstein; D.A. Jacobsohn; J.S. Kuhl; H. Yabe; J. Kurtzberg; D. Malm; P.J. Orchard; C Klein; T. Lucke; K. Sykora

doi:10.1038/bmt.2011.99

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

M. Mynarek, J. Tolar, M.H. Albert, M. Escolar, J.J. Boelens, M.J. Cowan, N. Finnegan, A. Glomstein, D.A. Jacobsohn, J.S. Kuhl, H. Yabe, J. Kurtzberg, D. Malm, P.J. Orchard, C Klein, T. Lucke, K. Sykora

Research output: Contribution to journal › Article › Academic › peer-review

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.

Original language	English
Pages (from-to)	352-359
Number of pages	8
Journal	Bone Marrow Transplantation
Volume	47
Issue number	3
DOIs	https://doi.org/10.1038/bmt.2011.99
Publication status	Published - 2012

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

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10.1038/bmt.2011.99

http://www.nature.com/bmt/journal/v47/n3/pdf/bmt201199a.pdf

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Mynarek, M., Tolar, J., Albert, M. H., Escolar, M., Boelens, J. J., Cowan, M. J., Finnegan, N., Glomstein, A., Jacobsohn, D. A., Kuhl, J. S., Yabe, H., Kurtzberg, J., Malm, D., Orchard, P. J., Klein, C., Lucke, T., & Sykora, K. (2012). Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. Bone Marrow Transplantation, 47(3), 352-359. https://doi.org/10.1038/bmt.2011.99

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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",

author = "M. Mynarek and J. Tolar and M.H. Albert and M. Escolar and J.J. Boelens and M.J. Cowan and N. Finnegan and A. Glomstein and D.A. Jacobsohn and J.S. Kuhl and H. Yabe and J. Kurtzberg and D. Malm and P.J. Orchard and C Klein and T. Lucke and K. Sykora",

year = "2012",

doi = "10.1038/bmt.2011.99",

language = "English",

volume = "47",

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Mynarek, M, Tolar, J, Albert, MH, Escolar, M, Boelens, JJ, Cowan, MJ, Finnegan, N, Glomstein, A, Jacobsohn, DA, Kuhl, JS, Yabe, H, Kurtzberg, J, Malm, D, Orchard, PJ, Klein, C, Lucke, T & Sykora, K 2012, 'Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients', Bone Marrow Transplantation, vol. 47, no. 3, pp. 352-359. https://doi.org/10.1038/bmt.2011.99

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.

AU - Boelens, J.J.

AU - Cowan, M.J.

AU - Finnegan, N.

AU - Glomstein, A.

AU - Jacobsohn, D.A.

AU - Kuhl, J.S.

AU - Yabe, H.

AU - Kurtzberg, J.

AU - Malm, D.

AU - Orchard, P.J.

AU - Klein, C

AU - Lucke, T.

AU - Sykora, K.

PY - 2012

Y1 - 2012

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

U2 - 10.1038/bmt.2011.99

DO - 10.1038/bmt.2011.99

M3 - Article

C2 - 21552297

SN - 0268-3369

VL - 47

SP - 352

EP - 359

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

IS - 3

ER -

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

Abstract

Keywords

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