Arquivos de neuro-psiquiatria · 2016
Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome.
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Saute Jonas Alex Morales, Souza Carolina Fischinger Moura de, Poswar Fabiano de Oliveira, Donis Karina Carvalho, Campos Lillian Gonçalves, Deyl Adriana Vanessa Santini, Burin Maira Graeff, Vargas Carmen Regla, Matte Ursula da Silveira, Giugliani Roberto, Saraiva-Pereira Maria Luiza, Vedolin Leonardo Modesti, Gregianin Lauro José, Jardim Laura Bannach
Аннотация
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.
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.
Предметные рубрики MeSH
AdolescentAdrenoleukodystrophyAdultAge of OnsetBrainBrazilChildChild, PreschoolFemaleHematopoietic Stem Cell TransplantationHumansLeukodystrophy, MetachromaticMagnetic Resonance ImagingMaleMucopolysaccharidosis IPedigreeRetrospective StudiesTissue DonorsTransplantation ConditioningTreatment OutcomeWhite MatterYoung Adult