@article{
 title = {Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and hurler syndrome},
 type = {article},
 year = {2016},
 keywords = {Adrenoleukodystrophy,Hematopoietic stem cell transplantation,Leukodystrophy,Metachromatic,Mucopolysaccharidosis I},
 pages = {953-966},
 volume = {74},
 id = {6ba40835-397d-369f-b71e-fa1c1fc08977},
 created = {2018-01-16T17:29:08.206Z},
 file_attached = {false},
 profile_id = {a1b2ded6-b257-3e56-ac7c-9d438762d170},
 last_modified = {2021-10-19T17:32:18.228Z},
 read = {false},
 starred = {false},
 authored = {true},
 confirmed = {true},
 hidden = {false},
 private_publication = {false},
 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.},
 bibtype = {article},
 author = {Saute, Jonas Alex Morales and De Souza, Carolina Fischinger Moura and De Oliveira Poswar, Fabiano and Donis, Karina Carvalho and Campos, Lillian Gonçalves and Deyl, Adriana Vanessa Santini and Burin, Maira Graeff and Vargas, Carmen Regla and Da Silveira Matte, Ursula and Giugliani, Roberto and Saraiva-Pereira, Maria Luiza and Vedolin, Leonardo Modesti and Gregianin, Lauro José and Jardim, Laura Bannach},
 doi = {10.1590/0004-282x20160155},
 journal = {Arquivos de Neuro-Psiquiatria},
 number = {12}
}

{"_id":"ARxWPzp64HStwe72L","bibbaseid":"saute-desouza-deoliveiraposwar-donis-campos-deyl-burin-vargas-etal-neurologicaloutcomesafterhematopoieticstemcelltransplantationforcerebralxlinkedadrenoleukodystrophylateonsetmetachromaticleukodystrophyandhurlersyndrome-2016","authorIDs":[],"author_short":["Saute, J., A., M.","De Souza, C., F., M.","De Oliveira Poswar, F.","Donis, K., C.","Campos, L., G.","Deyl, A., V., S.","Burin, M., G.","Vargas, C., R.","Da Silveira Matte, U.","Giugliani, R.","Saraiva-Pereira, M., L.","Vedolin, L., M.","Gregianin, L., J.","Jardim, L., B."],"bibdata":{"title":"Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and hurler syndrome","type":"article","year":"2016","keywords":"Adrenoleukodystrophy,Hematopoietic stem cell transplantation,Leukodystrophy,Metachromatic,Mucopolysaccharidosis I","pages":"953-966","volume":"74","id":"6ba40835-397d-369f-b71e-fa1c1fc08977","created":"2018-01-16T17:29:08.206Z","file_attached":false,"profile_id":"a1b2ded6-b257-3e56-ac7c-9d438762d170","last_modified":"2021-10-19T17:32:18.228Z","read":false,"starred":false,"authored":"true","confirmed":"true","hidden":false,"private_publication":false,"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.","bibtype":"article","author":"Saute, Jonas Alex Morales and De Souza, Carolina Fischinger Moura and De Oliveira Poswar, Fabiano and Donis, Karina Carvalho and Campos, Lillian Gonçalves and Deyl, Adriana Vanessa Santini and Burin, Maira Graeff and Vargas, Carmen Regla and Da Silveira Matte, Ursula and Giugliani, Roberto and Saraiva-Pereira, Maria Luiza and Vedolin, Leonardo Modesti and Gregianin, Lauro José and Jardim, Laura Bannach","doi":"10.1590/0004-282x20160155","journal":"Arquivos de Neuro-Psiquiatria","number":"12","bibtex":"@article{\n title = {Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and hurler syndrome},\n type = {article},\n year = {2016},\n keywords = {Adrenoleukodystrophy,Hematopoietic stem cell transplantation,Leukodystrophy,Metachromatic,Mucopolysaccharidosis I},\n pages = {953-966},\n volume = {74},\n id = {6ba40835-397d-369f-b71e-fa1c1fc08977},\n created = {2018-01-16T17:29:08.206Z},\n file_attached = {false},\n profile_id = {a1b2ded6-b257-3e56-ac7c-9d438762d170},\n last_modified = {2021-10-19T17:32:18.228Z},\n read = {false},\n starred = {false},\n authored = {true},\n confirmed = {true},\n hidden = {false},\n private_publication = {false},\n 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.},\n bibtype = {article},\n author = {Saute, Jonas Alex Morales and De Souza, Carolina Fischinger Moura and De Oliveira Poswar, Fabiano and Donis, Karina Carvalho and Campos, Lillian Gonçalves and Deyl, Adriana Vanessa Santini and Burin, Maira Graeff and Vargas, Carmen Regla and Da Silveira Matte, Ursula and Giugliani, Roberto and Saraiva-Pereira, Maria Luiza and Vedolin, Leonardo Modesti and Gregianin, Lauro José and Jardim, Laura Bannach},\n doi = {10.1590/0004-282x20160155},\n journal = {Arquivos de Neuro-Psiquiatria},\n number = {12}\n}","author_short":["Saute, J., A., M.","De Souza, C., F., M.","De Oliveira Poswar, F.","Donis, K., C.","Campos, L., G.","Deyl, A., V., S.","Burin, M., G.","Vargas, C., R.","Da Silveira Matte, U.","Giugliani, R.","Saraiva-Pereira, M., L.","Vedolin, L., M.","Gregianin, L., J.","Jardim, L., B."],"biburl":"https://bibbase.org/service/mendeley/a1b2ded6-b257-3e56-ac7c-9d438762d170","bibbaseid":"saute-desouza-deoliveiraposwar-donis-campos-deyl-burin-vargas-etal-neurologicaloutcomesafterhematopoieticstemcelltransplantationforcerebralxlinkedadrenoleukodystrophylateonsetmetachromaticleukodystrophyandhurlersyndrome-2016","role":"author","urls":{},"keyword":["Adrenoleukodystrophy","Hematopoietic stem cell transplantation","Leukodystrophy","Metachromatic","Mucopolysaccharidosis I"],"metadata":{"authorlinks":{}},"downloads":0},"bibtype":"article","creationDate":"2020-02-05T21:53:19.727Z","downloads":0,"keywords":["adrenoleukodystrophy","hematopoietic stem cell transplantation","leukodystrophy","metachromatic","mucopolysaccharidosis i"],"search_terms":["neurological","outcomes","hematopoietic","stem","cell","transplantation","cerebral","linked","adrenoleukodystrophy","late","onset","metachromatic","leukodystrophy","hurler","syndrome","saute","de souza","de oliveira poswar","donis","campos","deyl","burin","vargas","da silveira matte","giugliani","saraiva-pereira","vedolin","gregianin","jardim"],"title":"Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and hurler syndrome","year":2016,"biburl":"https://bibbase.org/service/mendeley/a1b2ded6-b257-3e56-ac7c-9d438762d170","dataSources":["KeLrgK49soyCLiQgv","ya2CyA73rpZseyrZ8","2252seNhipfTmjEBQ"]}