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

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

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36 Scopus citations

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 languageEnglish (US)
Pages (from-to)352-359
Number of pages8
JournalBone marrow transplantation
Volume47
Issue number3
DOIs
StatePublished - Mar 2012

Keywords

  • alpha-mannosidosis
  • hematopoietic SCT
  • inborn errors of metabolism
  • lysosomal storage disease
  • neurodevelopment
  • outcome

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