Title |
Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome
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Published in |
Arquivos de Neuro-Psiquiatria, December 2016
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DOI | 10.1590/0004-282x20160155 |
Pubmed ID | |
Authors |
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 |
Abstract |
To describe survival and neurological outcomes after HSCT for these disorders. 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. 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. Good outcomes were obtained when HSCT was performed before adulthood, early in the clinical course, and/or from a related donor. |
X Demographics
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United States | 1 | 100% |
Demographic breakdown
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Scientists | 1 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 50 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Bachelor | 10 | 20% |
Other | 5 | 10% |
Student > Ph. D. Student | 5 | 10% |
Student > Postgraduate | 4 | 8% |
Student > Doctoral Student | 3 | 6% |
Other | 8 | 16% |
Unknown | 15 | 30% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 13 | 26% |
Biochemistry, Genetics and Molecular Biology | 6 | 12% |
Neuroscience | 5 | 10% |
Nursing and Health Professions | 2 | 4% |
Agricultural and Biological Sciences | 2 | 4% |
Other | 4 | 8% |
Unknown | 18 | 36% |