Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy.

Wynn, Robert F; Wraith, J Ed; Mercer, Jean; O'Meara, Anne; Tylee, Karen; Thornley, Margaret; Church, Heather J; Bigger, Brian W

Wynn, Robert F; Wraith, J Ed; Mercer, Jean; O'Meara, Anne; Tylee, Karen; Thornley, Margaret; Church, Heather J; Bigger, Brian W.

Afiliação

Wynn RF; Department of Haematology and BMT, Royal Manchester Children's Hospital, Manchester, United Kingdom. robert.wynn@cmmc.nhs.uk

J Pediatr ; 154(4): 609-11, 2009 Apr.

Article em En | MEDLINE | ID: mdl-19324223

RESUMO

We compared substrate reduction in patients with lysosomal storage disorder treated with hematopoietic stem cell transplant and found that it was significantly reduced compared with patients treated with pharmacological enzyme replacement therapy. These data might support the wider application of hematopoietic stem cell transplant in the treatment of lysosomal storage disorders.

Assuntos

Transplante de Células-Tronco Hematopoéticas; Mucopolissacaridose II/terapia; Mucopolissacaridose I/terapia; Mucopolissacaridose VI/terapia; Biomarcadores/urina; Sulfatos de Condroitina/urina; Dermatan Sulfato/urina; Terapia Enzimática; Enzimas/sangue; Humanos

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose II / Mucopolissacaridose I / Mucopolissacaridose VI / Transplante de Células-Tronco Hematopoéticas Limite: Humans Idioma: En Revista: J Pediatr Ano de publicação: 2009 Tipo de documento: Article País de afiliação: Reino Unido País de publicação: Estados Unidos

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