Unrelated Bone Marrow Transplantation with a Reduced Toxicity Myeloablative Conditioning Regimen in Wiskott-Aldrich Syndrome.
10.3346/jkms.2008.23.1.146
- Author:
Hyoung Jin KANG
1
;
Hee Young SHIN
;
Sun Hie KO
;
Jeong Ah PARK
;
Eun Kyung KIM
;
Jung Woo RHIM
;
Joong Gon KIM
;
Hyo Seop AHN
Author Information
1. Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea. hsahn@snu.ac.kr
- Publication Type:Case Report
- Keywords:
Wiskott-Aldrich Syndrome;
Bone Marrow Transplantation;
Busulfan;
Fludarabine
- MeSH:
*Bone Marrow Transplantation/adverse effects;
Child, Preschool;
Graft vs Host Disease/etiology;
Humans;
Male;
*Transplantation Conditioning;
Wiskott-Aldrich Syndrome/*surgery
- From:Journal of Korean Medical Science
2008;23(1):146-148
- CountryRepublic of Korea
- Language:English
-
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/m(2) 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.
