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

Publication ,  Journal Article
Mynarek, M; Tolar, J; Albert, MH; Escolar, ML; Boelens, JJ; Cowan, MJ; Finnegan, N; Glomstein, A; Jacobsohn, DA; Kühl, JS; Yabe, H; Malm, D ...
Published in: Bone Marrow Transplant
March 2012

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.

Duke Scholars

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Published In

Bone Marrow Transplant

DOI

EISSN

1476-5365

Publication Date

March 2012

Volume

47

Issue

3

Start / End Page

352 / 359

Location

England

Related Subject Headings

  • alpha-Mannosidosis
  • Treatment Outcome
  • Transplantation, Homologous
  • Retrospective Studies
  • Medical Oncology
  • Male
  • Infant
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
 

Citation

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Chicago
ICMJE
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Mynarek, M., Tolar, J., Albert, M. H., Escolar, M. L., Boelens, J. J., Cowan, M. J., … Sykora, K.-W. (2012). Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. Bone Marrow Transplant, 47(3), 352–359. https://doi.org/10.1038/bmt.2011.99
Mynarek, M., J. Tolar, M. H. Albert, M. L. Escolar, J. J. Boelens, M. J. Cowan, N. Finnegan, et al. “Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.Bone Marrow Transplant 47, no. 3 (March 2012): 352–59. https://doi.org/10.1038/bmt.2011.99.
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 Transplant. 2012 Mar;47(3):352–9.
Mynarek, M., et al. “Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.Bone Marrow Transplant, vol. 47, no. 3, Mar. 2012, pp. 352–59. Pubmed, doi:10.1038/bmt.2011.99.
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. Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. Bone Marrow Transplant. 2012 Mar;47(3):352–359.

Published In

Bone Marrow Transplant

DOI

EISSN

1476-5365

Publication Date

March 2012

Volume

47

Issue

3

Start / End Page

352 / 359

Location

England

Related Subject Headings

  • alpha-Mannosidosis
  • Treatment Outcome
  • Transplantation, Homologous
  • Retrospective Studies
  • Medical Oncology
  • Male
  • Infant
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation