1.
Pediatr Transplant
; 10(3): 395-9, 2006 May.
Artigo
em Inglês
| MEDLINE
| ID: mdl-16677369
RESUMO
Wiskott-Aldrich syndrome (WAS) is a primary immunodeficiency disease characterized by thrombocytopenia, eczema, impaired cellular and humoral immunity, and increased susceptibility to malignancy and autoimmunity. The only curative treatment for WAS is hematopoietic stem cell transplantation, especially in the presence of a matched sibling donor or matched unrelated donor. Here, we report the case of a 2.5-yr-old boy with WAS that resulted in mixed chimerism after having received bone marrow from his phenotypically identical grandfather. Although the patient has persistent thrombocytopenia (platelet counts 50-80 x 10(9)/L), he is currently alive and doing well at 36 months post-transplant and is free of any bleeding episodes.
Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Síndrome de Wiskott-Aldrich/genética , Síndrome de Wiskott-Aldrich/terapia , Alelos , Pré-Escolar , Quimerismo , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Masculino , Trombocitopenia/etiologia , Fatores de Tempo , Condicionamento Pré-Transplante
2.
J Genet
; 83(2): 197-9, 2004 Aug.
Artigo
em Inglês
| MEDLINE
| ID: mdl-15536260