Peripheral blood stem cell transplantation in children with beta-thalassemia.
Bone Marrow Transplant
; 28(11): 1037-40, 2001 Dec.
Article
en En
| MEDLINE
| ID: mdl-11781613
Fifteen patients with beta-thalassemia received an allogeneic peripheral blood stem cell transplant. Median age was 3.5 years (1-15 years). Six were class I, four class II and five class III according to the Pesaro criteria. All of the donors were HLA-phenotypically identical (13 siblings and two parents). Nine patients were given BU + CY and six BU + CY plus ATG as conditioning. All patients received MTX (+1, +3, +6) and CsA (9-12 months) post transplant for GVHD prophylaxis. The median neutrophil and platelet engraftment times were day 12 and day 16, respectively. cGVHD was observed in three patients. Two patients died. Thirteen patients are well, and transfusion-independent 2-30 months after PSCT. No recurrences of thalassemia have been seen. Overall and event-free survival were 86.6%. In conclusion, we suggest that PSCT can be considered a safe and effective treatment for children with beta- thalassemia.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Talasemia beta
/
Trasplante de Células Madre Hematopoyéticas
Tipo de estudio:
Observational_studies
/
Prognostic_studies
Límite:
Adolescent
/
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Idioma:
En
Revista:
Bone Marrow Transplant
Asunto de la revista:
TRANSPLANTE
Año:
2001
Tipo del documento:
Article
País de afiliación:
Turquía
Pais de publicación:
Reino Unido