The effects of early and late bone marrow transplantation in siblings with alpha-mannosidosis. Is early haematopoietic cell transplantation the preferred treatment option?
J Inherit Metab Dis
; 33 Suppl 3: S123-7, 2010 Dec.
Article
em En
| MEDLINE
| ID: mdl-20165920
ABSTRACT
This article documents both the neurological and physical outcomes of the first published set of siblings undergoing transplantation at differing ages for α-mannosidosis. The older brother, the index case, was diagnosed at the age of 3 years and underwent transplantation at 13 years for the treatment of increasing somatic problems and recurrent infections. The younger brother had undergone transplantation pre-symptomatically at 6 months of age. Their clinical, radiological and developmental outcomes are documented and compared with the previous published cases, with the case for early transplantation being weighted against other potential therapies.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Células-Tronco Hematopoéticas
/
Tempo para o Tratamento
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Alfa-Manosidose
Tipo de estudo:
Diagnostic_studies
/
Etiology_studies
/
Risk_factors_studies
Limite:
Adolescent
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Humans
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Infant
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Male
Idioma:
En
Revista:
J Inherit Metab Dis
Ano de publicação:
2010
Tipo de documento:
Article
País de afiliação:
Reino Unido