Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome
ABSTRACT Hematopoietic stem cell transplantation (HSCT) is the only available treatment for the neurological involvement of disorders such as late-onset metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I-Hurler (MPS-IH), and X-linked cerebral adrenoleukodystrophy (CALD). Objective To...
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Thieme Revinter Publicações
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| Series: | Arquivos de Neuro-Psiquiatria |
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| author | Jonas Alex Morales Saute Carolina Fischinger Moura de Souza Fabiano de Oliveira Poswar Karina Carvalho Donis Lillian Gonçalves Campos Adriana Vanessa Santini Deyl Maira Graeff Burin Carmen Regla Vargas Ursula da Silveira Matte Roberto Giugliani Maria Luiza Saraiva-Pereira Leonardo Modesti Vedolin Lauro José Gregianin Laura Bannach Jardim |
| author_facet | Jonas Alex Morales Saute Carolina Fischinger Moura de Souza Fabiano de Oliveira Poswar Karina Carvalho Donis Lillian Gonçalves Campos Adriana Vanessa Santini Deyl Maira Graeff Burin Carmen Regla Vargas Ursula da Silveira Matte Roberto Giugliani Maria Luiza Saraiva-Pereira Leonardo Modesti Vedolin Lauro José Gregianin Laura Bannach Jardim |
| author_sort | Jonas Alex Morales Saute |
| collection | DOAJ |
| description | ABSTRACT Hematopoietic stem cell transplantation (HSCT) is the only available treatment for the neurological involvement of disorders such as late-onset metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I-Hurler (MPS-IH), and X-linked cerebral adrenoleukodystrophy (CALD). 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. |
| format | Article |
| id | doaj-art-7e3b84d8854d416ca8cff18d273cb41e |
| institution | OA Journals |
| issn | 1678-4227 |
| language | English |
| publisher | Thieme Revinter Publicações |
| record_format | Article |
| series | Arquivos de Neuro-Psiquiatria |
| spelling | doaj-art-7e3b84d8854d416ca8cff18d273cb41e2025-08-20T01:58:00ZengThieme Revinter PublicaçõesArquivos de Neuro-Psiquiatria1678-4227741295396610.1590/0004-282x20160155S0004-282X2016001200953Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndromeJonas Alex Morales SauteCarolina Fischinger Moura de SouzaFabiano de Oliveira PoswarKarina Carvalho DonisLillian Gonçalves CamposAdriana Vanessa Santini DeylMaira Graeff BurinCarmen Regla VargasUrsula da Silveira MatteRoberto GiuglianiMaria Luiza Saraiva-PereiraLeonardo Modesti VedolinLauro José GregianinLaura Bannach JardimABSTRACT Hematopoietic stem cell transplantation (HSCT) is the only available treatment for the neurological involvement of disorders such as late-onset metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I-Hurler (MPS-IH), and X-linked cerebral adrenoleukodystrophy (CALD). 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.http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2016001200953&lng=en&tlng=entransplante de células-tronco hematopoiéticasleucodistrofia metacromáticamucopolissacaridose Iadrenoleucodistrofia |
| spellingShingle | Jonas Alex Morales Saute Carolina Fischinger Moura de Souza Fabiano de Oliveira Poswar Karina Carvalho Donis Lillian Gonçalves Campos Adriana Vanessa Santini Deyl Maira Graeff Burin Carmen Regla Vargas Ursula da Silveira Matte Roberto Giugliani Maria Luiza Saraiva-Pereira Leonardo Modesti Vedolin Lauro José Gregianin Laura Bannach Jardim Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome Arquivos de Neuro-Psiquiatria transplante de células-tronco hematopoiéticas leucodistrofia metacromática mucopolissacaridose I adrenoleucodistrofia |
| title | Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome |
| title_full | Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome |
| title_fullStr | Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome |
| title_full_unstemmed | Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome |
| title_short | Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome |
| title_sort | neurological outcomes after hematopoietic stem cell transplantation for cerebral x linked adrenoleukodystrophy late onset metachromatic leukodystrophy and hurler syndrome |
| topic | transplante de células-tronco hematopoiéticas leucodistrofia metacromática mucopolissacaridose I adrenoleucodistrofia |
| url | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2016001200953&lng=en&tlng=en |
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