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

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2012-03

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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.

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10.1038/bmt.2011.99

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Mynarek, M, J Tolar, MH Albert, ML Escolar, JJ Boelens, MJ Cowan, N Finnegan, A Glomstein, et al. (2012). Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. Bone marrow transplantation, 47(3). pp. 352–359. 10.1038/bmt.2011.99 Retrieved from https://hdl.handle.net/10161/24659.

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