Long term disease burden post-transplantation: three decades of observations in 25 Hurler patients successfully treated with hematopoietic stem cell transplantation (HSCT).

Guffon, N; Pettazzoni, M; Pangaud, N; Garin, C; Lina-Granade, G; Plault, C; Mottolese, C; Froissart, R; Fouilhoux, A

Guffon, N; Pettazzoni, M; Pangaud, N; Garin, C; Lina-Granade, G; Plault, C; Mottolese, C; Froissart, R; Fouilhoux, A.

Afiliação

Guffon N; Reference Center for Inherited Metabolic Disorders, Femme Mère Enfant Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron Cedex, France. nathalie.guffon-fouilhoux@chu-lyon.fr.
Pettazzoni M; Biochemistry and Molecular Biology and Reference Center for Inherited Metabolic Disorders, Hospices Civils de Lyon, 59 boulevard Pinel, 69677 Bron cedex, France.
Pangaud N; Cardiology, Louis Pradel Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677 Bron Cedex, France.
Garin C; Orthopaedy, Femme Mère Enfant Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677 Bron Cedex, France.
Lina-Granade G; Oto-Rhino-Laryngology and Child Audiology, Femme Mère Enfant Hospital and Edouard Herriot Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677 Bron Cedex, France.
Plault C; Reference Center for Inherited Metabolic Disorders, Femme Mère Enfant Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron Cedex, France.
Mottolese C; Neurosurgery, Femme Mère Enfant Hospital and Pierre Wertheimer Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677 Bron Cedex, France.
Froissart R; Biochemistry and Molecular Biology and Reference Center for Inherited Metabolic Disorders, Hospices Civils de Lyon, 59 boulevard Pinel, 69677 Bron cedex, France.
Fouilhoux A; Reference Center for Inherited Metabolic Disorders, Femme Mère Enfant Hospital, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron Cedex, France.

Orphanet J Rare Dis ; 16(1): 60, 2021 01 31.

Article em En | MEDLINE | ID: mdl-33517895

Assuntos

Transplante de Células-Tronco Hematopoéticas; Mucopolissacaridose I; Adolescente; Adulto; Criança; Pré-Escolar; Efeitos Psicossociais da Doença; Humanos; Lactente; Mucopolissacaridose I/terapia; Qualidade de Vida; Estudos Retrospectivos; Resultado do Tratamento; Adulto Jovem

Palavras-chave

Adaptation; Cognitive; Depression; Hematopoietic cell transplantation; Long-term outcomes; Mucopolysaccharidosis type I-Hurler syndrome; Psychosocial function; Residual disease burden

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Observational_studies Limite: Adolescent / Adult / Child / Child, preschool / Humans / Infant Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: França

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