Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome.
J Korean Med Sci
; 23(1): 146-8, 2008 Feb.
Article
in En
| MEDLINE
| ID: mdl-18303217
ABSTRACT
Wiskott-Aldrich syndrome (WAS) is an X-linked congenital immune-deficiency syndrome, and bone marrow transplantation (BMT) has become a curative modality. However, the transplant with the alternative donor needed more intensive conditioning with increased treatment-related toxicities. Recently, fludarabine-based reduced toxicity myeloablative conditioning regimens have been developed for adult myeloid malignancies with promising results of good engraftment and low treatment-related toxicities. To increase the engraftment potential without serious complications, a boy with WAS received successful unrelated BMT with a reduced toxicity myeloablative conditioning regimen composed of fludarabine (40 mg/m2) on days -8, -7, -6, -5, -4, -3), busulfan (0.8 mg/kg i. v. q 6 hr on days -6, -5, -4, -3), and thymoglobulin (2.5 mg/kg on days -4, -3, -2). This novel conditioning regimen could improve the outcome of allogeneic transplantation for other non-malignant diseases such as congenital immune-deficiency syndromes or metabolic storage diseases.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Wiskott-Aldrich Syndrome
/
Bone Marrow Transplantation
/
Transplantation Conditioning
Type of study:
Etiology_studies
Limits:
Child, preschool
/
Humans
/
Male
Language:
En
Journal:
J Korean Med Sci
Journal subject:
MEDICINA
Year:
2008
Document type:
Article