title: Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome.
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.
authors.affiliation: Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
authors.forename: Hyoung Jin
authors.initials: HJ
authors.lastname: Kang
authors.forename: Hee Young
authors.initials: HY
authors.lastname: Shin
authors.forename: Sun Hie
authors.initials: SH
authors.lastname: Ko
authors.forename: Jeong Ah
authors.initials: JA
authors.lastname: Park
authors.forename: Eun Kyung
authors.initials: EK
authors.lastname: Kim
authors.forename: Jung Woo
authors.initials: JW
authors.lastname: Rhim
authors.forename: Joong Gon
authors.initials: JG
authors.lastname: Kim
authors.forename: Hyo Seop
authors.initials: HS
authors.lastname: Ahn
mesh_terms: D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002675:Child, Preschool; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D008297:Male; D019172:Transplantation Conditioning; D014923:Wiskott-Aldrich Syndrome / Q000601:surgery*