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Case Reports
. 2016 Dec;74(12):953-966.
doi: 10.1590/0004-282X20160155.

Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome

Jonas Alex Morales Saute  1   2   3 , Carolina Fischinger Moura de Souza  1 , Fabiano de Oliveira Poswar  4 , Karina Carvalho Donis  1   5 , Lillian Gonçalves Campos  6   3 , Adriana Vanessa Santini Deyl  7 , Maira Graeff Burin  1 , Carmen Regla Vargas  1   8 , Ursula da Silveira Matte  4   9   10 , Roberto Giugliani  1   5   3   4   9   10 , Maria Luiza Saraiva-Pereira  1   2   4   11 , Leonardo Modesti Vedolin  6   3 , Lauro José Gregianin  7   12 , Laura Bannach Jardim  1   2   5   3   4   13
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Free article
Case Reports

Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome

Jonas Alex Morales Saute et al. Arq Neuropsiquiatr. 2016 Dec.
Free article

Abstract

Objective: To describe survival and neurological outcomes after HSCT for these disorders.

Methods: Seven CALD, 2 MLD and 2 MPS-IH patients underwent HSCT between 2007 and 2014. Neurological examinations, magnetic resonance imaging, molecular and biochemical studies were obtained at baseline and repeated when appropriated.

Results: Favorable outcomes were obtained with 4/5 related and 3/6 unrelated donors. Two patients died from procedure-related complications. Nine transplanted patients were alive after a median of 3.7 years: neurological stabilization was obtained in 5/6 CALD, 1/2 MLD, and one MPS-IH patient. Brain lesions of the MPS-IH patient were reduced four years after HSCT.

Conclusion: Good outcomes were obtained when HSCT was performed before adulthood, early in the clinical course, and/or from a related donor.

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